ModifiedColorado Rocky Mountain Health PlansPolicy UN-CSTRANSPT002.E

Hematopoietic Stem Cell Transplantation

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Clinical guideline governing medical necessity and coverage criteria for autologous and allogeneic hematopoietic stem cell transplantation for Ohio members; describes indications, contraindications, evaluation and center requirements referenced to Ohio Administrative Code and InterQual® criteria.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyHematopoietic Stem Cell Transplantation

Policy CodePolicy UN-CSTRANSPT002.E

Change TypeMaterial updates to coverage for TA‑TMA, pediatric Ryoncil dosing, and VOD guidance

Effective DateAug 1, 2026

Last ReviewJun 10, 2026

Next Review DateAug 1, 2026

Key ActionObtain prior authorization with disease‑specific documentation (prognostic scores, diagnostic confirmation, and Day‑28 response plan for pediatric Ryoncil where applicable).

SourceLink

POLICY UPDATE CHANGES

Narsoplimab‑wuug (Yartemlea) is FDA‑approved (Dec 2025) and is considered medically necessary for TA‑TMA in patients ≥2 years when criteria are met.

Remestemcel‑L‑rknd (Ryoncil) is considered medically necessary for steroid‑refractory acute GVHD in pediatric patients 2 months to 17 years with specified dosing and Day‑28 response‑guided retreatment rules.

Defibrotide coverage clarified: covered for treatment of active hepatic VOD with renal or pulmonary dysfunction post‑HCT and not covered for VOD prophylaxis.

Ohio-onlyjurisdiction applicability

MRD standard

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ModifiedColorado Rocky Mountain Health PlansPolicy UN-CSTRANSPT002.E

Hematopoietic Stem Cell Transplantation

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Sign up for Colorado Rocky Mountain Health Plans Policy UN-CSTRANSPT002.E alerts

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Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyHematopoietic Stem Cell Transplantation

Policy CodePolicy UN-CSTRANSPT002.E

Change TypeMaterial updates to coverage for TA‑TMA, pediatric Ryoncil dosing, and VOD guidance

Effective DateAug 1, 2026

Last ReviewJun 10, 2026

Next Review DateAug 1, 2026

Key ActionObtain prior authorization with disease‑specific documentation (prognostic scores, diagnostic confirmation, and Day‑28 response plan for pediatric Ryoncil where applicable).

SourceLink

POLICY UPDATE CHANGES

Narsoplimab‑wuug (Yartemlea) is FDA‑approved (Dec 2025) and is considered medically necessary for TA‑TMA in patients ≥2 years when criteria are met.

Defibrotide coverage clarified: covered for treatment of active hepatic VOD with renal or pulmonary dysfunction post‑HCT and not covered for VOD prophylaxis.

Ohio-onlyjurisdiction applicability

MRD standard

2mo–17yRyoncil age range

≥2yYartemlea minimum age

10Center volume requirement

A–KAppendices included

Coverage Criteria and Indications

Allogeneic transplant — Leukemias

Allogeneic hematopoietic stem cell transplantation may be considered medically necessary for the following leukemia-related indications

Allogeneic leukemia indications: ALL: first complete remission (CR1); second or subsequent complete remission; any relapse; consolidation post-CAR T therapy. AML: adverse-risk disease; intermediate-risk disease; favorable-risk disease with inadequate clearance of measurable residual disease (MRD). CLL. CML. Prolymphocytic leukemia.

See Appendix A for AML genetic risk classification; MRD referenced in policy.

Autologous transplant — Leukemias

Autologous hematopoietic stem cell transplantation may be considered medically necessary in limited circumstances for leukemia

Autologous leukemia indications: Prolymphocytic leukemia. ALL or AML in certain adults when no suitable allogeneic donor exists (refer to Medical Director). In pediatric ALL certain high‑risk cytogenetic features (e.g., hypodiploidy, iAMP21) may influence decision to transplant in CR1 (refer to Medical Director).

Medical Director consultation required for donor-unsuitable cases.

Allogeneic transplant — MDS and related

Allogeneic hematopoietic stem cell transplantation may be considered medically necessary for the following myelodysplastic and related disorders

Allogeneic MDS indications: Myelodysplastic syndromes (MDS). Juvenile myelomonocytic leukemia/juvenile chronic myelogenous leukemia (JMML/JCML). Chronic myelomonocytic leukemia (CMML).

Medicare NCD 110.23 expands coverage by prognostic risk scores (see Appendix B).

Allogeneic transplant — Myelofibrosis

Allogeneic hematopoietic stem cell transplantation may be considered medically necessary in myeloproliferative disorders when disease‑risk criteria are met

Myelofibrosis indications: Primary myelofibrosis and related conditions (including secondary myelofibrosis) in patients with DIPSS-Plus Intermediate‑2 or High‑Risk score; selected secondary myelofibrosis (post-polycythemia vera or ET) after transplant evaluation approval.DIPSS-Plus Intermediate-2 or High

See Appendix C for DIPSS / DIPSS‑Plus scoring.

Transplant — Brain tumors — coverage stance

Brain tumor transplant stance: Allogeneic HSCT is considered unproven and not medically necessary for listed brain tumors. Autologous HSCT may be considered medically necessary for medulloblastoma, oligodendroglioma, pineoblastoma, ETMR, and glioblastoma multiforme (GBM) — GBM may be considered in infants (refer to Medical Director).

Specific tumor lists and exceptions are in policy.

Myelofibrosis — specific allogeneic HSCT criteria

Allogeneic HSCT may be considered medically necessary when ALL of the following for myelofibrosis are met:

Myelofibrosis allogeneic criteria: Diagnosis of primary myelofibrosis or related condition AND DIPSS‑Plus risk category Intermediate‑2 or High AND patient deemed appropriate transplant candidate after comprehensive evaluation (including consideration of age, comorbidity, and donor availability).DIPSS‑Plus Intermediate‑2 or High

Refer to Appendix C for scoring details.

Brain Tumors — autologous HSCT criteria

Autologous HSCT may be considered medically necessary for selected brain tumors when ALL of the following apply:

Autologous HSCT for brain tumors: Medulloblastoma OR Oligodendroglioma OR Pineoblastoma OR Embryonal tumor with multilayered rosettes (ETMR) OR Glioblastoma multiforme (GBM; may be considered in infants with Medical Director referral).

High‑dose autologous therapy limited to chemo‑responsive patients without bulky residual disease.

Germ Cell Tumors — autologous HSCT coverage criteria

Autologous HSCT may be considered medically necessary for germ cell tumors for the listed tumor types.

Autologous HSCT for germ cell tumors: Testicular germ cell tumor OR Extragonadal germ cell tumor OR Seminoma OR Choriocarcinoma OR Embryonal carcinoma OR Mixed germ cell tumor OR Teratoma OR Yolk‑sac tumor OR Ovarian germ cell tumor — high‑dose therapy with autologous support when disease is chemo‑responsive and without bulky residual tumor.

Allogeneic HSCT is considered unproven for germ cell tumors.

Multiple Myeloma — allogeneic and autologous criteria

Allogeneic HSCT may be considered medically necessary for multiple myeloma in selected high‑risk or specific tandem transplant circumstances

Allogeneic MM criteria: Initial therapy in newly diagnosed patients with high‑risk myeloma (high‑risk cytogenetics: del(17p), t(4;14), t(14;16) per IMWG or Mayo Clinic additions) AND otherwise fit for transplant; early relapse (<24 months) after primary therapy that included an autologous SCT with favorable salvage response; reduced‑intensity MRD/MUD as second transplant of a planned tandem transplant.

Refer all allogeneic MM requests to Medical Director.

Autologous MM allowed: Single autologous transplant; tandem autologous or tandem autologous→allogeneic in early relapse or per specified clinical scenarios.

Autologous HCT considered at diagnosis and at progression/relapse.

Plasma cell disorders and MGRS — coverage status

Plasma cell and related disorders — autologous or allogeneic HSCT coverage status:

Autologous HSCT allowed: AL‑amyloidosis OR POEMS OR Waldenstrom macroglobulinemia OR MGRS (only after failed clone‑targeting chemotherapy and when renal function permits high‑dose chemotherapy).

Allogeneic HSCT unproven in MGRS; autologous may be considered when criteria met.

Allogeneic HSCT allowed: Waldenstrom macroglobulinemia; AL‑amyloidosis may be appropriate in a clinical trial setting. Allogeneic HSCT is unproven and not medically necessary for MGUS and solitary plasmacytoma.

Hodgkin Lymphoma — coverage stance

Hodgkin lymphoma coverage stance:

Hodgkin lymphoma: Autologous HSCT is considered medically necessary for Hodgkin lymphoma. Allogeneic HSCT is also considered medically necessary in appropriate circumstances.

Non-Hodgkin Lymphoma — subtype-specific transplant coverage

Non‑Hodgkin lymphoma (NHL) — transplant coverage by subtype:

Allogeneic HSCT for NHL: Small B‑cell lymphocytic lymphoma; Follicular lymphoma; Lymphoplasmacytoid lymphoma/immunocytoma; Marginal zone lymphoma; Burkitt lymphoma; Diffuse large B‑cell lymphoma; Mantle cell lymphoma; Precursor B‑cell leukemia/lymphoma; T‑cell lymphoma.

Autologous HSCT for NHL: Follicular lymphoma; Lymphoplasmacytoid lymphoma/immunocytoma; Marginal zone lymphoma; Burkitt lymphoma; Diffuse large cell lymphoma; Mantle cell lymphoma; Precursor B‑cell leukemia/lymphoma; T‑cell lymphoma — autologous transplants must be for chemosensitive tumors per Cheson criteria.

Autologous HSCT not standard for small lymphocytic lymphoma; refer to Medical Director.

Other malignancies — coverage stance

Other malignancies — coverage stance:

Allogeneic HSCT allowed: Blastic plasmacytoid dendritic cell neoplasm; Rhabdomyosarcoma/soft tissue sarcoma may be appropriate in a clinical trial with Medical Director referral.

Many other solid tumors listed are unproven and not medically necessary for allogeneic HSCT.

Autologous HSCT allowed: Ewing sarcoma; Neuroblastoma; Retinoblastoma; Supratentorial ependymoma; Atypical teratoid rhabdoid tumor; Rhabdomyosarcoma (in clinical trials); Wilms tumor (relapsed disease in trials) — high‑dose autologous therapy only for chemo‑responsive disease without bulky residual tumor.

Tandem autologous may be indicated in trials.

Hematological Disorders — allogeneic HSCT indications

Hematological disorders — allogeneic HSCT may be considered when diagnosis matches list:

Hematological disorders: Aplastic anemia; Diamond‑Blackfan syndrome; Chronic granulomatous disease; Kostmann syndrome (congenital agranulocytosis); Congenital amegakaryocytic thrombocytopenia; Dyskeratosis congenita; Fanconi anemia; Paroxysmal nocturnal hemoglobinuria (PNH); Shwachman‑Diamond syndrome; Sickle cell disease; Thalassemia major.

Autologous HSCT is unproven for these disorders.

Immunodeficiency and lysosomal storage diseases — allogeneic HSCT

Primary immunodeficiency syndromes — allogeneic HSCT may be considered for the listed disorders:

Immunodeficiency syndromes allogeneic: CD40 ligand deficiency; Chediak‑Higashi syndrome; Hemophagocytic lymphohistiocytosis (HLH/FEL); Leukocyte adhesion deficiency; Omenn syndrome; Wiskott‑Aldrich syndrome; X‑linked lymphoproliferative syndrome; Severe combined immunodeficiency (SCID); selected lysosomal storage diseases (Gaucher type I, Niemann‑Pick type B, fucosidosis) when criteria met.

Autologous HSCT is unproven for these conditions.

Autoimmune Diseases — coverage logic

Autoimmune diseases — coverage logic:

Allogeneic HSCT for autoimmune disease: Allogeneic HSCT is unproven and not medically necessary for multiple sclerosis and systemic sclerosis; allogeneic HSCT may be considered only in clinical trial contexts for Crohn's disease, rheumatoid arthritis, or SLE when the member's benefit supports trial participation.

Autologous HSCT for autoimmune disease: Autologous HSCT may be considered for Crohn's disease (in trials), multiple sclerosis (meets RR or SP definition; EDSS 2.0–6.0; failed ≥1 disease‑modifying therapy; and either ≥2 relapses in prior 12 months or 1 relapse plus MRI gadolinium‑enhancing lesion in prior 12 months), systemic sclerosis meeting specific criteria (age ≥18, diffuse cutaneous disease, disease duration ≤5 years from first non‑Raynaud symptom, mRSS ≥15–20, progressive internal organ involvement), and RA or SLE in trial settings.EDSS 2.0–6.0 for MS; mRSS ≥15–20 and disease duration ≤5 years for systemic sclerosis

Systemic sclerosis excludes listed physiologic contraindications (FVC <45% predicted, creatinine clearance <40 ml/min, pulmonary arterial hypertension, constrictive pericarditis, cardiac tamponade, LVEF <50%).

Inherited metabolic disorders — allogeneic HSCT

Allogeneic hematopoietic stem cell transplantation may be considered medically necessary for the following inherited metabolic disorders when clinically appropriate:

Inherited metabolic disorders list: Adrenoleukodystrophy; Epidermolysis bullosa; Globoid cell leukodystrophy (Krabbe); Hurler syndrome (MPS I); Hunter syndrome (MPS II); Mannosidosis; Maroteaux‑Lamy syndrome (MPS VI); Metachromatic leukodystrophy; Mitochondrial neurogastrointestinal encephalopathy (MNGIE); Osteopetrosis; Rett syndrome.

Autologous HSCT is unproven for these inherited metabolic disorders.

Omidubicel — hematologic malignancy and SAA indications

Omidubicel (Omisirge) is covered for selected indications when ALL specified criteria are met

Omidubicel (hematologic malignancy): Patient is ≥12 years of age; candidate for myeloablative allogeneic HSCT; has no readily available matched sibling or matched unrelated donor; Omidubicel is being requested to reduce time to neutrophil recovery and incidence of infection; following comprehensive evaluation umbilical cord blood is felt to be the best cell source for this patient.age >= 12 years

Supported by randomized trial showing faster neutrophil/platelet recovery and fewer infections.

Omidubicel (severe aplastic anemia): Patient is ≥6 years of age; has no readily available matched sibling or matched unrelated donor; candidate for reduced‑intensity conditioning; Omidubicel is being requested to reduce time to neutrophil recovery and incidence of infection; following comprehensive evaluation umbilical cord blood is felt to be the best cell source.age >= 6 years

Based on single‑center SAA experience with neutrophil recovery endpoints.

Donor lymphocyte infusion — coverage criteria

Donor Lymphocyte Infusion (DLI) is medically necessary following a medically necessary allogeneic HSCT for a hematologic malignancy when ALL of the following are met

DLI indication: Donor lymphocytes are collected from the original HSCT donor AND intended for management of refractory, persistent, or relapsed hematologic malignancy OR to enhance conversion from mixed to full donor chimerism in confirmed relapse OR as a planned strategy to prevent relapse in patients at elevated relapse risk.

Evidence includes multicenter retrospective data showing benefit for prophylactic DLI in high‑risk acute leukemia.

Cardiac conditions — not medically necessary (top-level)

Cardiac conditions: Allogeneic and autologous HSCT are unproven and not medically necessary for cardiac conditions; transplantation considered only in clinical trial when the member's benefit permits trial participation.

Ryoncil (remestemcel-L-rknd) — pediatric SR‑aGVHD criteria

Remestemcel‑L‑rknd (Ryoncil) is considered medically necessary for pediatric steroid‑refractory acute GVHD when ALL of the following are met

Ryoncil pediatric SR-aGVHD criteria: Patient age 2 months to 17 years; steroid‑refractory acute GVHD grade B–D (excluding skin‑only grade B); remestemcel‑L‑rknd administered at 2 x 10^6 MSCs/kg IV twice weekly for 4 consecutive weeks; Day 28 ±2 assessment performed to guide further therapy.

Day‑28 response rules: CR → stop; PR/MR → once weekly x4; No response → consider alternatives; recurrence after CR → twice weekly x4. Further regimens beyond these are unproven/not medically necessary.

Yartemlea (narsoplimab-wuug) — TA‑TMA criteria

Narsoplimab‑wuug (Yartemlea) is considered medically necessary for TA‑TMA when ONE of the following is met:

TA‑TMA diagnostic acceptance: Biopsy‑proven TA‑TMA (kidney or GI).

TA‑TMA clinical criteria: Meets ≥4 of the specified 7 clinical/laboratory criteria at two consecutive time points within 14 days. The 7 criteria include: anemia (failure to achieve transfusion independence, hemoglobin drop ≥1 g/dL, or new transfusion dependence with other causes ruled out); thrombocytopenia (failure platelet engraftment, refractory to transfusion, ≥50% reduction after engraftment, or increased transfusion needs); elevated LDH > ULN for age; presence of schistocytes; hypertension (>99th percentile for age in <18 years OR SBP ≥140 mmHg or DBP ≥90 mmHg in ≥18 years); elevated sC5b‑9 ≥ ULN; proteinuria rUPCR ≥1 mg/mg.≥4 of 7

Approval beyond initial 6‑month certification based on continued active disease or inadequate response per Jodele criteria.

Defibrotide — treatment vs prophylaxis stance

Defibrotide coverage: Defibrotide is covered for treatment of active hepatic veno‑occlusive disease (VOD) with renal or pulmonary dysfunction following HSCT.

Defibrotide is not covered for VOD prophylaxis.

CLL — transplant indications

Chronic lymphocytic leukemia (CLL) indications for HCT consideration:

CLL indications: Resistance or intolerance to BTK inhibitors and/or BCL2 inhibitors OR Richter's transformation.

These features identify CLL patients for transplant consideration.

AML — transplant consultation/consideration criteria

Acute myeloid leukemia (AML) — HCT consultation/consideration when ANY of the following apply:

AML indications list: High‑resolution HLA typing recommended at diagnosis; early HCT consultation for age <2 years at diagnosis; primary induction failure; measurable residual disease after initial therapy; CR1 except favorable‑risk AML; monosomy 5 or 7; treatment‑related leukemia; first relapse; CR2+ if not previously evaluated.

Favorable‑risk AML defined by specific cytogenetic/molecular abnormalities (see Appendix A).

ALL (≤39 years) — HCT consideration criteria

Acute lymphoblastic leukemia (ALL) (age ≤39 years) — HCT consideration when ANY of the following apply:

Infant ALL and high‑risk features: Infant at diagnosis with unfavorable genetics; age <3 months with any WBC or <6 months with WBC >300,000; any infant with MRD+ after consolidation.

Induction/response indications: Primary induction failure after achieving MRD‑negative status; presence of MRD after initial therapy (MRD ≥0.01% following consolidation at 9–12 weeks); high/very high‑risk CR1 including Ph+ slow‑TKI responders with IKZF1 deletions, iAMP21 if MRD+ after consolidation, 11q23 rearrangement if MRD+ after consolidation.MRD ≥0.01% after consolidation

Relapse indications: First relapse with aim to transplant in CR2; CR2+ including all young adults in CR2; early relapse (≤36 months medullary or ≤18 months EMD); MRD+ (>0.1% medullary or equivalent for EMD) after re‑induction (4–8 weeks); T‑cell ALL; CR3+.MRD >0.1% after re‑induction

MDS — transplant considerations

Myelodysplastic syndromes (MDS):

MDS: HCT consideration at diagnosis for all subtypes of MDS.

Medicare prognostic score thresholds described in Appendix B apply for Medicare coverage determinations.

JMML — transplant criteria

Juvenile myelomonocytic leukemia (JMML):

JMML: Allogeneic HCT consideration at diagnosis.

Lymphomas — subtype‑specific transplant indications

Lymphomas — subtype‑specific transplant indications:

Follicular lymphoma: Poor response to initial treatment; initial remission duration <24 months; at relapse; transformation to diffuse large B‑cell lymphoma.

Diffuse large B‑cell lymphoma: Primary induction failure (including residual PET‑avid disease); first relapse; CR2+; double or triple hit at diagnosis (MYC and BCL‑2 and/or BCL‑6); primary CNS lymphoma at diagnosis PIF or first relapse.

High‑grade B‑cell lymphoma: MYC and BCL‑2 and/or BCL‑6 rearrangements; primary induction failure; CR1; first relapse; CR2+.

Mantle cell lymphoma: At diagnosis; first relapse; BTK‑intolerant or resistant disease.

Germ cell tumors — coverage

Germ cell tumors — autologous high‑dose therapy may be considered when specified conditions are met:

Germ cell tumors: High‑dose therapy with autologous stem cell support for non‑germinomatous germ cell tumors refractory after induction if chemo‑responsive and without bulky residual tumor; germinoma/NGGCT chemo‑responsive to re‑induction and without bulky residual tumor.

Allogeneic HSCT considered unproven for germ cell tumors.

Neuroblastoma / Medulloblastoma / embryonal tumors — coverage and timing

Neuroblastoma and embryonal tumors — autologous HSCT indications and timing:

Neuroblastoma indications: INRGSS L2 at diagnosis with MYCN amplification or age >18 months with unfavorable histology and segmental chromosome aberration; INRGSS stage M at diagnosis with MYCN amplification or age >18 months; age 12–18 months with unfavorable histology, segmental chromosome aberrations, or diploid DNA content.

All allogeneic and pediatric autologous neuroblastoma recipients should be screened weekly for TA‑TMA during first 100 days post‑HCT per policy.

Medulloblastoma/embryonal tumors: High‑dose autologous therapy may be standard first‑line for several infant (<3 years) embryonal tumors (medulloblastoma, AT/RT, ETMR, PNET‑NOS) and may be considered at relapse if chemoresponsive without bulky residual disease.

Multiple myeloma — covered indications (selected)

Multiple myeloma and related plasma cell disorders — covered indications summary:

Multiple myeloma (autologous): Autologous HCT considered at diagnosis and at progression/relapse.

Diagnostic confirmation per IMWG/CRAB required for transplant candidacy.

Other plasma cell disorders: AL‑amyloidosis and POEMS may be considered for autologous HCT; Waldenstrom macroglobulinemia may be considered for allogeneic HCT in select cases; MGRS autologous HCT only after failed clone‑targeting chemotherapy and with sufficient renal function.

Covered Indications (partial list)

Covered indications (partial list) — disease groups and timing/conditions:

Malignant and CNS tumors: Medulloblastoma; multiple myeloma (at diagnosis and at progression/relapse); plasma cell disorders; POEMS syndrome (at diagnosis).

See disease‑specific entries for timing and response requirements.

Inherited metabolic and immune deficiency disorders: Inherited metabolic disorders (e.g., Hurler, ALD) and primary immune deficiency diseases (e.g., SCID, Wiskott‑Aldrich, Omenn) — often covered at diagnosis or newborn screening detection for appropriate disorders.

Appendix K flags indicate autologous vs allogeneic permissibility.

Sickle cell disease: Coverage includes children (especially <13) with matched sibling donors and patients with aggressive course or listed complications; alternative donors acceptable if listed complications present.Includes TCD and TRJ numeric thresholds

Numeric thresholds: TCD ≥200 cm/sec (or >185 cm/sec with intracranial vasculopathy); TRJ ≥2.7 m/sec; chronic pain ≥6 months.

Marrow failure syndromes — covered when progression/complications occur

Marrow failure syndromes — covered when disease‑specific progression or complications occur:

Other marrow failure syndromes: Diamond‑Blackfan anemia with continued transfusion‑dependent anemia after steroid therapy, significant infections, or progression to MDS/AML; Shwachman‑Diamond, Fanconi anemia, Dyskeratosis congenita with development of cytopenias, transfusion dependence, significant infections, high‑risk cytopenic clones, high‑risk somatic mutation patterns, or MDS/AML.

Autoimmune and systemic disease criteria

Autoimmune and systemic disease criteria — covered with specified severity or functional criteria:

Systemic sclerosis: At diagnosis or with diffuse disease: increasing mRSS and evidence of decrease to <80% predicted in FVC and/or DLCO; additional criteria per systemic sclerosis entry must be met and contraindications absent.mRSS increase; FVC/DLCO <80% predicted

See Appendix H for mRSS documentation.

Multiple sclerosis: After MS relapse with ≥2 relapse episodes in past 3 years while on disease‑modifying therapy; patient must be able to walk 100 meters (unilateral assistance allowed).≥2 relapses in 3 years; ambulatory 100 m

See Appendix G for EDSS scoring.

Pediatric/hematologic diagnoses — covered at diagnosis

Other pediatric and hematologic disorders — covered at diagnosis:

Transfusion‑dependent thalassemias: Coverage indicated at diagnosis.

Hemophagocytic lymphohistiocytosis (HLH): Coverage indicated at diagnosis.

Medicare MDS prognostic score requirement

Medicare coverage rule for allogeneic HCT in MDS (NCD 110.23):

Medicare MDS HCT coverage: Coverage requires meeting at least one prognostic‑score threshold: IPSS ≥ 1.5 OR IPSS‑R ≥ 4.5 OR IPSS‑M ≥ 0.5.IPSS >=1.5 OR IPSS-R >=4.5 OR IPSS-M >=0.5

See Appendix B for scoring details; absence of qualifying scores implies noncoverage for Medicare members.

Multiple myeloma diagnostic criteria (IMWG/CRAB)

Diagnostic criteria for multiple myeloma relevant to HCT consideration — all three required:

Multiple myeloma diagnostic requirement: (1) Monoclonal plasma cells in bone marrow >10% and/or biopsy‑proven plasmacytoma; (2) Monoclonal protein present in serum and/or urine (exceptions detailed for nonsecretory disease); (3) One or more myeloma‑related organ dysfunction (CRAB) OR at least one IMWG myeloma‑defining event (≥60% clonal plasma cells; involved/uninvolved FLC ratio ≥100 with involved FLC ≥100 mg/L; or >1 focal MRI lesion ≥5 mm).see text

IMWG MDEs may substitute for CRAB features; imaging and lab documentation required for prior authorization.

Appendix A — AML genetic risk classification

Appendix A — AML genetic risk classification (used to inform transplant decisions):

AML risk groups: Patients are categorized as Favorable, Intermediate, or Adverse based on specified cytogenetic and molecular abnormalities (examples enumerated in Appendix A). Initial risk assignment may change with MRD results.N/A

See Appendix A for detailed abnormality listings (t(8;21), inv(16), NPM1, FLT3‑ITD, TP53, complex karyotype, etc.).

DIPSS / DIPSS-Plus risk criteria

DIPSS / DIPSS‑Plus risk criteria (Appendix C) — informs myelofibrosis transplant thresholds:

DIPSS‑based risk categorization: DIPSS factors include age >65, Hb <10 g/dL, WBC >25x10^9/L, peripheral blasts >1%, constitutional symptoms; DIPSS categories: Low, Int‑1, Int‑2, High. DIPSS‑Plus adds adverse karyotype, platelets <100x10^9/L, and RBC transfusion need.See Appendix C

DIPSS‑Plus Intermediate‑2 or High indicates transplant consideration.

Multiple myeloma — diagnostic classification criteria

Multiple myeloma diagnostic classification elements (used to determine transplant evaluation urgency):

Multiple myeloma diagnostic classification: All 3 required: (1) Monoclonal plasma cells in bone marrow >10% and/or biopsy‑proven plasmacytoma; (2) Monoclonal protein in serum and/or urine (or nonsecretory criteria); (3) CRAB features OR an IMWG myeloma‑defining event.see children

IMWG MDEs listed separately (≥60% marrow plasma cells; FLC ratio ≥100 with involved FLC ≥100 mg/L; >1 focal MRI lesion ≥5 mm).

IMWG myeloma‑defining events

IMWG myeloma‑defining events (any one sufficient for diagnosis regardless of CRAB):

IMWG myeloma‑defining events: Any one of: (a) ≥60% clonal plasma cells in bone marrow; (b) serum involved/uninvolved free light chain ratio ≥100 with involved FLC ≥100 mg/L; (c) >1 focal MRI lesion ≥5 mm.see items

Each marker associated with high short‑term risk of organ damage and may prompt urgent transplant evaluation.

MGUS — diagnostic criteria

MGUS diagnostic criteria (all required):

MGUS: (1) Low serum and/or urine monoclonal protein levels; (2) Bone marrow monoclonal plasma cells <10%; (3) Normal serum calcium, hemoglobin, and creatinine, no bone lesions or amyloidosis features.see text

Defines non‑malignant monoclonal gammopathy and excludes active myeloma.

Smoldering myeloma — diagnostic criteria

Smoldering (indolent) myeloma criteria (all required):

Smoldering myeloma: (1) Monoclonal protein in serum and/or urine; (2) Monoclonal plasma cells in bone marrow and/or tissue biopsy; (3) Does not meet criteria for MGUS, active multiple myeloma, or solitary plasmacytoma and absence of IMWG myeloma‑defining events; upper marrow clonal plasma cell limit 60%.upper limit 60% marrow plasma cells

Patients with smoldering myeloma are monitored rather than immediately offered HCT unless progression occurs.

Lymphoma response criteria

Lymphoma response criteria (used to assess chemosensitivity and remission status):

Complete Remission (CR): Disappearance of all evidence of disease; bone marrow infiltrate cleared on repeat biopsy; PET guidance for previously FDG‑avid disease.NA

From revised response criteria for malignant lymphoma (Appendix E).

Partial Remission (PR): Regression of measurable disease with no new sites; >50% decrease in sum of products of diameters (SPD) of dominant masses or equivalent PET/SUV decrease when size measurements unavailable.>50% decrease in SPD

Used to document chemo‑responsiveness prior to autologous HCT.

Appendix K — disease-to-transplant coverage mapping

Appendix K — disease‑to‑transplant coverage mapping (usage note):

Disease‑to‑transplant mapping: Each disease row in Appendix K has two boolean flags: '1' indicates autologous transplant permissibility and '2' indicates allogeneic transplant permissibility. Coverage is consistent with the flag 'Yes' for the requested transplant type—providers must match the member diagnosis to the appendix row and request the transplant type indicated.flag == Yes

When appendix flag for requested transplant type is 'No', HSCT is not supported by Appendix K quick reference.

Autologous hematopoietic stem cell transplantation (autologous HSCT) is described in the guideline as appropriate in a limited set of indications but is explicitly unproven and not medically necessary for many conditions. Examples cited where autologous HSCT is not considered appropriate include most myelodysplastic syndromes and many leukemia subtypes unless a specific, limited exception applies (for example, prolymphocytic leukemia or when no suitable allogeneic donor is available and Medical Director consultation supports autologous HSCT). Providers should therefore confirm disease-specific coverage language and obtain prior authorization when autologous HSCT is requested for leukemia or MDS presentations that are not listed as accepted indications.

The policy states that allogeneic HSCT is considered unproven and not medically necessary for germ cell tumors and for many solid tumors listed in the document. For these diagnoses (for example, many primary brain tumors and most germ cell tumor entries) allogeneic transplant requests are expected to be denied unless there is an explicit, documented rationale and Medical Director review. Autologous HSCT may be considered for selected solid tumor indications (for example certain germ cell tumor presentations and several pediatric embryonal brain tumors) but allogeneic HSCT is not a standard covered option for germ cell and most solid tumor types.

Autologous HSCT is unproven and not medically necessary for primary immunodeficiency syndromes and many lysosomal storage/inherited metabolic diseases listed in the guideline. By contrast, the policy identifies specific primary immunodeficiency and inherited metabolic disorders for which allogeneic HSCT may be medically necessary (e.g., SCID, Wiskott-Aldrich, Hurler syndrome). Requests for autologous HSCT in these disorders should be supported by clear evidence when provided, but the default policy stance is that autologous HSCT is not supported for these conditions.

The guideline explicitly states that HSCT (autologous or allogeneic) for cardiac conditions is unproven and not medically necessary and would only be considered within a clinical trial if the member’s benefit permits trial participation. Clinical teams should not plan HSCT for cardiac disease outside approved investigational settings unless the Medical Director authorizes an exception.

Remestemcel-L (Ryoncil) is included with specific pediatric steroid-refractory aGVHD criteria and response-guided continuation rules; the policy notes that further use of Ryoncil beyond the specified Day 28–guided continuation/retreatment regimens is considered unproven and not medically necessary. Providers must document dosing, Day 28 response assessment, and planned retreatment strategy consistent with the policy to support authorization.

High-dose autologous therapy (high-dose chemotherapy with autologous stem cell rescue) is supported only for patients with chemo-responsive disease and without bulky residual tumor. The policy specifically requires that patients be demonstrably chemotherapy-responsive and that there be no bulky residual disease before proceeding with high-dose autologous approaches for germ cell tumors or embryonal pediatric tumors; presence of bulky residual disease is a contraindication to high-dose autologous therapy in these sections.

Within the provided excerpts the document does not list an exhaustive set of explicit denials labeled as exclusions in some sections; many sections summarize covered indications and qualifying conditions rather than enumerating blanket, standalone exclusions. Users should reference the disease-specific coverage statements and Appendix K flags to determine when a requested transplant will not meet quick-reference coverage criteria.

Medicare coverage for allogeneic HCT in MDS is conditional on prognostic-score thresholds. For Medicare members the policy references the CMS/NCD criteria requiring at least one of the following: IPSS ≥ 1.5, IPSS-R ≥ 4.5, or IPSS‑M ≥ 0.5. Failure to meet one of these thresholds is expected to render the allogeneic HCT request non‑covered under Medicare rules.

The document distinguishes monoclonal gammopathy categories relevant to transplant: MGUS requires low serum/urine monoclonal protein and bone marrow plasma cells <10%; smoldering myeloma meets intermediate criteria without myeloma‑defining events and has an upper marrow plasma cell limit of 60%; and active multiple myeloma requires all three diagnostic elements (bone marrow/plasmacytoma, monoclonal protein in serum/urine or nonsecretory criteria, and CRAB features or an IMWG myeloma‑defining event). These distinctions determine whether HSCT evaluation or transplant is indicated.

The quick reference (Appendix K) is intended only to identify standard-of-care indications and has limitations: it does not capture potential exceptions such as clinical trial enrollment or required Medical Director escalation. The policy instructs users to consult the full guideline for complete criteria and exceptions rather than relying solely on the quick-reference table.

Appendix K uses binary flags to indicate transplant permissibility by disease and transplant type. Several diseases in the appendix are explicitly flagged as 'No' for autologous and/or allogeneic transplant (for example, many hematological congenital disorders are No for autologous but Yes for allogeneic). When an appendix row shows 'No' for the requested transplant type, the quick-reference indicates that the HSCT request will not meet the appendix-based indication and the full guideline should be consulted for any potential exceptions.

The policy states that allogeneic HSCT is considered unproven and not medically necessary for leukemia types that are not specifically listed as covered indications. Similarly, autologous HSCT is considered unproven and not medically necessary for many leukemia subtypes and for brain tumors not specifically enumerated as covered. Requests for HSCT in these circumstances require clear justification and Medical Director review if pursued.

Autologous HSCT is explicitly described as unproven and not medically necessary for primary and secondary myelofibrosis. Allogeneic HSCT is the indicated curative option for myelofibrosis when patients meet appropriate DIPSS/DIPSS‑Plus risk thresholds; autologous transplant is not supported for these disorders in routine practice.

The policy identifies allogeneic HSCT as unproven and not medically necessary for listed brain tumors and for germ cell tumors. Autologous HSCT may be considered medically necessary for specific brain tumor subtypes (medulloblastoma, oligodendroglioma, pineoblastoma, ETMR, and in selected infants with GBM) and for specified germ cell tumor presentations, but allogeneic HSCT is not a supported approach for these tumor groups in routine coverage.

For inherited metabolic disorders the guideline specifies that allogeneic HSCT may be medically necessary for a defined list of disorders (for example, Hurler syndrome, Krabbe disease, adrenoleukodystrophy), and it explicitly states that autologous HSCT is unproven and not medically necessary for these inherited metabolic disorders. Providers should therefore plan for allogeneic approaches when transplant is indicated for these conditions.

The policy reiterates that remestemcel‑L (Ryoncil) retreatment rules are response‑guided around the Day 28 assessment; the guideline emphasizes that any use of Ryoncil beyond the described Day 28–guided continuation or retreatment regimens is considered unproven and not medically necessary.

In many sections the document lists covered indications and qualifying clinical conditions rather than providing a separate list of explicit 'not medically necessary' items within the same chunk. The appendices and the disease-specific coverage statements function together to identify non‑covered scenarios (e.g., when Appendix K flags show 'No'). Readers should therefore cross‑reference the disease statements, exclusions, and Appendix K flags rather than assuming the absence of an explicit exclusion in the immediate text means coverage.

Appendices focus on defining diagnostic categories and risk/response criteria rather than serving as explicit denials. For example, the IMWG/CRAB definitions and MGUS/smoldering myeloma criteria are presented to classify disease status, and they do not themselves constitute explicit coverage denial language; rather, they inform whether a patient meets the diagnostic threshold for transplant consideration.

When Appendix K flags the requested transplant type as 'No' for the member’s diagnosis, the quick‑reference indicates that HSCT would not meet the appendix‑based indication. Appendix K is organized as a mapping table (autologous = 1, allogeneic = 2) where each disease row shows a Yes/No flag for each transplant type; providers must match the member diagnosis and requested transplant type to the appendix entry and document accordingly when submitting a prior authorization request.

Candidate Selection and Referral Criteria

Prior Authorization, Documentation and Provider Requirements

Prior Authorization

Prior authorization and Medical Director review required (Ohio Consortium)

Prior authorization is required for HSCT and reimbursement is contingent on prospective review and recommendation by the Ohio Hematopoietic Transplant and Cellular Therapy Consortium (or compliance with the Ohio agency performance standards and volume criteria); tandem transplants and third transplants require Medical Director review per the listed exceptions.

Reimbursement contingent on review/recommendation by the Ohio Hematopoietic Transplant and Cellular Therapy Consortium and department authorization.
Centers must be Consortium members or comply with agency 3701 performance standards and meet the 10-transplant volume threshold where applicable.
Tandem transplants require Medical Director review except specified pediatric/oncology conditions; third transplants require Medical Director review.

Prior Authorization

Document disease‑specific clinical criteria with prior authorization

Prior authorization requests must document disease-specific eligibility criteria and that the treating facility considers the patient an appropriate transplant candidate (examples include DIPSS Intermediate-2/High for myelofibrosis, IMWG/Mayo high‑risk cytogenetics for myeloma, and EDSS with relapse history for MS).

Coding, Thresholds and Key Clinical Values

Referenced CPTCPT

38243

Hematopoietic progenitor cell (HPC); transplantation or infusion (used in guideline to reference 'boost')

MRD sensitivity thresholds discussed

MRD sensitivity levels discussed10^-5 and 10^-6 sensitivity referenced for MRD testing and trial endpoints

Clinical useMRD is part of standard evaluation of response to HSCT for several disorders

Contextual guidanceUltrahigh-sensitivity NGS MRD recommended for select AML subgroups prior to allogeneic HCT

Proteinuria (rUPCR) threshold

Proteinuria threshold (rUPCR)≥ 1 mg/mg random protein-to-creatinine ratio

Role in TA-TMA criteriaOne of 7 clinical/laboratory criteria used to diagnose TA-TMA when present at two time points within 14 days

Pre-Approval Evaluation and Testing Requirements

Note

Transplant Center and Program Requirements

Note

Post-Transplant Management, Complications and Coverage

Maintenance therapy considered part of transplant event and covered when evidence supports

Maintenance therapy coverageMaintenance therapy is considered part of the transplant event and is covered when supported by adequate clinical evidence

Policy implicationWhen maintenance therapy is clinically supported, it may be included in the transplant benefit rather than billed as a separate unrelated event

DocumentationClinical evidence supporting maintenance therapy should be provided with authorization requests

Stem cell boost / HSCI — not a new transplant event; CPT 38243 referenced

Stem cell boost (HSCI) not a new transplant eventStem cell boost is not considered a new transplant event and is distinguished from a transplant; CPT 38243 referenced

Definition

Contraindications and Exclusionary Conditions

The policy notes that contraindications and suitability considerations are described in later sections and in disease‑specific criteria; reviewers should therefore consult those sections for absolute and relative contraindications and for the complete contraindication language prior to making coverage determinations.

For systemic sclerosis patients considered for autologous HSCT the policy lists specific physiologic contraindications. A patient is not suitable if they have any of: FVC <45% predicted, creatinine clearance <40 ml/min, pulmonary arterial hypertension, constrictive pericarditis, cardiac tamponade, or left ventricular ejection fraction <50%. These thresholds are required exclusions for autologous HSCT candidacy in systemic sclerosis assessments.

This placeholder refers to contraindication content described in other sections of the full guideline. Specific absolute and relative contraindications for additional diagnoses are provided elsewhere in the policy and should be consulted for authorization decisions.

This placeholder indicates that further contraindication details exist in the full policy text; reviewers should reference the complete guideline for explicit exclusionary criteria per diagnosis.

This is a placeholder noting the presence of additional contraindication entries in other policy sections not excerpted here.

This block is reserved for contraindication detail that is located in sections of the policy beyond the provided excerpts; consult the main document for full information.

This placeholder acknowledges that absolute and relative contraindications exist across indications but are not all captured in the extracted chunks; complete contraindication lists are in the full policy.

Definitions and Scoring Systems

Primary vs Secondary graft failure definitions

Primary vs Secondary graft failure definitionsPrimary failure: failure to reach three consecutive days with ANC > 500/µL after SCT; Secondary failure: initial engraftment for ≥3 days with ANC > 500/µL then subsequent decrease below that level

UseDefinitions used to distinguish engraftment failure types for post‑transplant management

DocumentationANC trend and timing should be included in clinical records when assessing graft failure

Stem cell boost definition

Stem cell boost definitionHematopoietic stem cell infusion to assist recovery or declining donor chimerism, not preceded by preparative regimen and not considered a new transplant event

CodingCPT 38243 referenced for boost procedures

Background and Evidence Summary

Background: Hematopoietic stem cell transplantation (autologous and allogeneic) is used to treat hematologic malignancies and selected non‑malignant inherited disorders. The policy frames allogeneic HCT as the only curative option for many disorders (e.g., MDS, inherited metabolic and primary immunodeficiency diseases) while autologous HCT remains standard for specific disease states (for example, many plasma cell disorders and selected pediatric solid tumors). The guideline integrates modern prognostic scoring systems and MRD assessments to guide transplant candidacy and timing.

The policy references a broad literature base to support indications and expected outcomes after HSCT. These citations and systematic reviews underpin the disease‑specific recommendations and are provided to inform reviewers about evidence sources used in drafting the coverage criteria; specific outcome data and trial details are available in the referenced publications.

Transplant‑associated thrombotic microangiopathy (TA‑TMA) treatment guidance was updated in the review cycle: new therapeutics (including narsoplimab‑wuug/Yartemlea) and other treatment notes were added to policy review notes. The document lists diagnostic criteria and documentation requirements tied to prior authorization for these agents; reviewers should follow the specified diagnostic thresholds and documentation checklists when evaluating treatment requests for TA‑TMA.

Complication Screening, Management and Therapeutics

Remestemcel-L (Ryoncil) dosing and response-guided retreatment rules

Ryoncil dosing & response rules (summary)2 x 10^6 MSCs/kg IV twice weekly x4 weeks; Day 28 ±2 response assessment directs continuation/retreatment: CR stop; PR/MR continue weekly x4; recurrence after CR → twice weekly x4; other retreatments unproven

IndicationFDA‑approved for pediatric steroid‑refractory acute GVHD (≥2 months)

Authorization requirementsPrior authorization requires age, dosing plan, Day 28 assessment plan, and rationale for continued/repeat dosing

Policy Revision History

2026-08-01policy_effectiveLatest

Policy effective date; incorporates recent review notes and literature updates through mid-2026 including product approvals and guideline updates.

2026-06-10last_review

Document last reviewed on 2026-06-10 as noted in review history; review included literature and product updates.

2026-05-15revision

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyHematopoietic Stem Cell Transplantation

Policy CodePolicy UN-CSTRANSPT002.E

Change TypeMaterial updates to coverage for TA‑TMA, pediatric Ryoncil dosing, and VOD guidance

Effective DateAug 1, 2026

Last ReviewJun 10, 2026

Next Review DateAug 1, 2026

Key ActionObtain prior authorization with disease‑specific documentation (prognostic scores, diagnostic confirmation, and Day‑28 response plan for pediatric Ryoncil where applicable).

SourceLink

On This Page

CAR‑T context: Patients who receive CD19 4‑1BB and achieve MRD‑negative CR if they have not already received HCT; patients receiving CD22 or other investigational therapies may be considered.

Primary refractory disease; first or subsequent relapse; brentuximab vedotin and checkpoint inhibitor refractory/intolerant disease (allo HCT).

Include relevant prognostic or scoring results (e.g., DIPSS/DIPSS‑Plus for myelofibrosis).
Provide disease-specific data such as cytogenetics, MRD levels, EDSS and relapse history as applicable.
Confirm the treating facility deems the patient an appropriate candidate.

Prior Authorization

Prior authorization required for Omidubicel and for Donor Lymphocyte Infusion (DLI)

Prior authorization is required for requests for Omidubicel (Omisirge) and for donor lymphocyte infusion (DLI); requests must state patient age, donor‑availability status, transplant conditioning intent, and the clinical rationale.

Omidubicel requests must confirm age criteria (≥12 years for malignancy indication; ≥6 years for severe aplastic anemia where applicable) and lack of readily available matched donors.
For DLI, document that donor lymphocytes are from the original HSCT donor and the intended indication (relapse management, chimerism conversion, or planned relapse prevention).
State the intended benefit (e.g., reduce neutrophil recovery time/infection risk for Omidubicel).

Prior Authorization

Prior authorization for narsoplimab (Yartemlea): diagnostic criteria and dosing plan required

Prior authorization for narsoplimab (Yartemlea) is required and must include documentation that TA‑TMA diagnostic criteria are met (either biopsy‑proven TA‑TMA or fulfillment of ≥4 of 7 clinical/lab criteria at two consecutive timepoints within 14 days), baseline severity/risk stratification, and the planned dosing schedule.

If using clinical criteria, show ≥4 of the specified 7 criteria documented at two consecutive time points within 14 days (or provide biopsy confirmation).
Provide baseline severity and risk stratification and the intended dose/weight‑based schedule and frequency.

Prior Authorization

Remestemcel‑L (Ryoncil) prior authorization: pediatric age and Day‑28 plan required

Prior authorization for remestemcel‑L (Ryoncil) is required; requests must document pediatric age eligibility and the Day‑28 response assessment and planned continuation/retreatment strategy per the policy.

Confirm patient age meets the indication (2 months to 17 years).
Document planned dosing (2 x 10^6 MSCs/kg IV twice weekly for 4 weeks) and Day 28 ±2 response assessment to guide further dosing.
State the planned follow‑up actions based on Day‑28 response (CR → stop; PR/MR → once weekly x4; no response → consider alternatives; recurrence after CR → twice weekly x4).

Prior Authorization

Prior authorization required when disease‑specific criteria are met (examples provided)

Prior authorization is required when the policy’s disease‑specific criteria are met for listed indications (for example, AML with MRD or CR1 except favorable‑risk; ALL meeting MRD thresholds or relapse criteria).

Submit documentation demonstrating the specific criterion that triggers HCT consideration (e.g., MRD values, CR timing, chemo‑responsiveness).
For tumor indications, show evidence of chemosensitivity and absence of bulky residual disease where required.

Prior Authorization

Prior authorization expected for listed indications — reference the specific indication

Prior authorization is expected for HSCT for the listed covered indications (e.g., multiple myeloma at diagnosis or relapse, inherited metabolic disorders, sickle cell disease meeting numeric thresholds); requests should reference the applicable indication.

Match the request to the covered indication (e.g., MM at diagnosis or progression; inherited metabolic disorders at diagnosis).
For sickle cell disease, include numeric thresholds when applicable (TCD, TRJ velocity, history of stroke, etc.).

Prior Authorization

Reference prognostic scores and disease‑specific diagnostic criteria in prior authorization

Use established prognostic scores and disease‑specific diagnostic classifications in prior authorization submissions (e.g., IPSS/IPSS‑R/IPSS‑M for MDS; DIPSS/DIPSS‑Plus for myelofibrosis; IMWG criteria for multiple myeloma).

For Medicare patients with MDS, provide the IPSS, IPSS‑R or IPSS‑M score meeting the stated thresholds when applicable.
Include DIPSS/DIPSS‑Plus scoring documentation for myelofibrosis evaluations.

Documentation Required

Diagnostic documentation required for plasma cell disorders prior authorization

Prior authorization for transplant evaluation or HSCT for plasma cell disorders must be supported by diagnostic documentation that meets the three required myeloma elements (bone marrow/plasmacytoma, monoclonal protein in serum/urine or nonsecretory criteria, and one or more CRAB features or an IMWG myeloma‑defining event).

Provide bone marrow plasma cell percentage or biopsy‑proven plasmacytoma.
Include quantification of monoclonal protein in serum and/or urine or nonsecretory disease criteria.
Document CRAB features or an IMWG myeloma‑defining event (≥60% clonal plasma cells, FLC ratio ≥100 with involved FLC ≥100 mg/L, or >1 focal MRI lesion ≥5 mm).

Note

Quick‑reference (Appendix K) not sufficient — escalate or document exceptions

Do not rely on the Appendix K quick reference alone for authorization decisions; prior authorization requests should reference the full guideline and escalate to medical director when exceptions or unusual circumstances apply.

Appendix K is a quick reference and does not capture exceptions such as clinical trial enrollment or medical director escalation rules.
If criteria are ambiguous or a requested transplant is outside standard flags, refer to the full guideline and Medical Director as instructed.

Prior Authorization

Reference the indication and transplant type per Appendix K in prior authorization

Prior authorization requests should specifically identify the disease/indication and whether autologous (1) or allogeneic (2) HSCT is being requested to match the Appendix K mapping and support coverage determination.

Include the exact diagnosis and indicate the transplant type requested (autologous = 1; allogeneic = 2).
Confirm that the appendix row for the diagnosis is flagged 'Yes' for the requested transplant type or provide rationale/medical director escalation.

Billing Rule

Planned‑sequence whole‑course approval allowed when protocolized

When the transplant is part of a planned sequence (for example, protocolized sequential high‑dose chemotherapy with rescue infusions), providers may request approval for the entire course up front and must document that the sequence is protocolized.

State that the transplants are part of a planned/treatment protocol (e.g., tandem or sequence) and include protocol details and timing.
Tandem sequences still require Medical Director review except where specified as exceptions in the guideline.

Prior Authorization

MGRS: autologous HSCT only after failed clone‑directed chemo and adequate renal function

For MGRS, autologous HSCT may be considered only after failure of chemotherapy targeting the underlying B‑cell clone and when renal function is sufficient to tolerate high‑dose chemotherapy; allogeneic HSCT is unproven for MGRS.

Document prior clone‑directed chemotherapy and its failure.
Document renal function showing suitability for high‑dose chemotherapy.

Note

Sequence notes: DLI prophylactic/preemptive use and Omidubicel when matched donor unavailable

Therapy sequencing expectations: DLI may be used prophylactically or preemptively per clinical criteria; Omidubicel is considered when no matched donor is readily available and when the intent is to reduce neutrophil recovery time and infection risk—document donor availability and intent.

For DLI, indicate whether the use is prophylactic, preemptive, or for relapse management, and document donor identity (original HSCT donor).
For Omidubicel, document lack of suitable matched sibling or unrelated donor and rationale for cord blood product choice.

Step Therapy

Ryoncil Day‑28 response‑guided continuation/retreatment rules

Remestemcel‑L (Ryoncil) continuation or retreatment decisions must follow the Day‑28 response‑guided algorithm: stop for complete response; repeat once weekly x4 for partial or mixed response; consider alternatives for no response; repeat twice weekly x4 for recurrence after CR.

Document Day‑28 ±2 assessment and planned next steps in the prior authorization submission.
Other retreatment regimens beyond these rules are considered unproven and not medically necessary.

Documentation Required

Document prior therapy and refractoriness for lymphoma transplant considerations

Prior lines of therapy and refractoriness are relevant for some lymphoma indications; prior authorization should document prior therapies, responses, and refractory status (for example, brentuximab/checkpoint inhibitor‑refractory Hodgkin lymphoma).

List prior systemic therapies, dates, and response (including refractory or intolerant status).
Provide evidence of chemo‑responsiveness when autologous high‑dose therapy is being considered.

Step Therapy

Expectations for prior disease‑modifying therapy and documented relapses (stepwise prior therapy)

For indications that expect stepwise prior therapy (such as MS), prior authorization should document prior disease‑modifying therapies, documented relapses, and the patient’s ambulatory capacity (e.g., ability to walk 100 meters).

Provide DMT history and evidence of treatment failure or relapse frequency (≥2 relapses in 12 months or other specified windows).
Document EDSS score or functional evidence and confirm 100‑meter ambulation capability.

Note

Use guideline and product PI references to justify therapy sequencing

Sequencing should be informed by clinical guidelines (NCCN, NMDP timing guidance) and product prescribing information for adjunct cellular products (e.g., Omidubicel, Ryoncil); include citations or guidance used to support sequencing decisions in the request.

Reference applicable NCCN or NMDP guidance when asserting timing or sequencing rationale.
Cite product PI dosing/continuation rules when requesting Omidubicel or remestemcel‑L.

Note

Appendices guide classification not sequencing — interpretive note

Interpretive note: the appendices provide classification and diagnostic detail rather than prescriptive step‑therapy sequences; authorization decisions should therefore rely on the full guideline and clinical evidence rather than assuming sequencing protocols from appendices alone.

Use appendices to confirm diagnostic classification, then apply full guideline and clinical context for sequencing decisions.
When sequencing is unclear, escalate to the Medical Director.

Step Therapy

Step therapy: none specified in the appendix — document prior therapies as relevant

No formal step therapy protocols are specified in these appendices; the quick reference does not establish mandatory pre‑authorization therapeutic steps—document prior therapies and consult the full guideline for sequencing requirements.

If step therapy is clinically relevant for the indication, provide documented prior attempts and reasons for progression to HSCT.
Absent explicit step therapy in the appendices, rely on disease‑specific criteria and guideline references.

Documentation Required

Regulatory and criteria references required with requests (Ohio OAC and InterQual)

The Ohio Administrative Code (Rule 5160‑1‑01) governs medical necessity determinations and InterQual® transplant modules are the referenced clinical coverage criteria; include compliance references and any InterQual® module used in the prior authorization submission.

Indicate which InterQual® module(s) were used to assess medical necessity (allogeneic or autologous pediatric/adult as applicable).
State compliance with Ohio Administrative Code requirements where relevant.

Documentation Required

Document high‑risk features for allogeneic SCT in multiple myeloma

Requests for allogeneic SCT in multiple myeloma should include supporting evidence of high‑risk disease (e.g., del(17p), t(4;14), t(14;16) or Mayo Clinic additions) or documentation of early relapse within 24 months and response to salvage therapy.

Provide cytogenetic/FISH results and relevant molecular data supporting high‑risk classification.
If citing early relapse as rationale, include dates of prior transplant/therapy and response to salvage treatment.

Documentation Required

Required documentation: infectious disease, psychosocial and caregiver/support evaluations

Required evaluations and documentation include infectious disease consultation and evidence of HIV viral suppression when applicable, psychosocial evaluation and caregiver/support availability, and documentation of financial ability to obtain post‑transplant medications.

Include an infectious disease consult and viral suppression documentation for HIV‑positive patients.
Submit psychosocial evaluation details and caregiver/support plan; note any remediation for adherence concerns.
Document assessment of ability to obtain post‑transplant medications.

Prior Authorization

Ryoncil prior authorization: include pediatric age, dosing and Day‑28 response plan

For Ryoncil approvals, provide pediatric age, the planned dosing regimen (2 x 10^6 MSCs/kg IV twice weekly ×4 weeks), and a Day‑28 ±2 response assessment with rationale for continued or repeat dosing per policy‑specified rules.

Confirm patient age is within 2 months to 17 years.
Include the exact dosing plan and Day‑28 assessment method.
State planned continuation/retreatment actions tied to Day‑28 response.

Documentation Required

Include disease status, MRD and genetic/cytogenetic risk in documentation

Clinical documentation should include disease status (e.g., CR1/CR2), MRD levels where referenced, and genetic/cytogenetic risk details (e.g., AML favorable/adverse definitions, MYCN in neuroblastoma, double/triple‑hit lymphoma) to support authorization.

Provide remission status, MRD assay results with thresholds where applicable, and cytogenetic/molecular findings.
Include imaging or pathology confirming chemo‑responsiveness when required for autologous high‑dose therapy.

Documentation Required

Required documentation: diagnostic confirmation, disease course and numeric thresholds

Clinical documentation must include diagnostic confirmation and disease course (e.g., number and timing of relapses, progression status) and measured values for any numeric thresholds cited in the policy (TCD, TRJ, FVC/DLCO, mRSS, ambulation distance).

Provide numeric test results (e.g., TCD velocity, TRJ velocity, % predicted FVC/DLCO) with dates.
Document relapse timeline and prior therapy attempts to substantiate transplant timing.

Documentation Required

Multiple myeloma prior authorization must document all three diagnostic elements

For multiple myeloma prior authorization, demonstrate all three required diagnostic elements: >10% clonal plasma cells and/or biopsy‑proven plasmacytoma; monoclonal protein in serum/urine (or nonsecretory criteria); and at least one CRAB feature or an IMWG myeloma‑defining event.

Supply bone marrow biopsy percentage, serum/urine monoclonal protein quantification or nonsecretory evidence, and documentation of CRAB features or IMWG MDEs.
If using imaging to document lesions, include modality and follow‑up imaging if equivocal per IMWG guidance.

Documentation Required

Document marrow plasma cell percentage and protein quantification for plasma cell disorder requests

Providers should document bone marrow plasma cell percentage, presence or absence of biopsy‑proven plasmacytoma, quantification of monoclonal protein in serum/urine (including free light chain results), and evidence of CRAB features or IMWG myeloma‑defining events when requesting transplant evaluation for plasma cell disorders.

Include clonal plasma cell percentage and any focal lesion imaging reports.
Provide involved/uninvolved free light chain ratio and absolute involved FLC level when relevant.

Documentation Required

mRSS exam documentation expectations for systemic sclerosis

mRSS assessments for systemic sclerosis should be documented site‑by‑site with a total score, exam date, examiner name/signature, and supporting notes as part of pre‑transplant evaluation.

Complete the 17‑site mRSS form with individual site scores and total.
Include examiner identification and date of exam in the record.

Documentation Required

Match request to Appendix K indication and transplant type in documentation

Ensure the prior authorization request references the specific disease/indication and the transplant type requested (autologous = 1; allogeneic = 2) so the reviewer can match the request to Appendix K flags.

Explicitly state the appendix row and flag used to justify the request when applicable.
If the appendix flag is 'No' for the requested transplant type, provide supporting rationale or escalate to the Medical Director.

Denial Risk

Denial risk: requests for unproven or quantity‑limited services will be evaluated and may be denied

Requests for services stated as unproven, or for which there is a coverage or quantity limit, will be evaluated for medical necessity under Ohio Administrative Code 5160‑1‑01 and may be denied if not supported by the guidelines or Consortium review.

If the intervention is listed as unproven or quantity‑limited in the policy (e.g., off‑label retreatment regimens), provide evidence or rationale for medical necessity and expect heightened scrutiny.
Reimbursement decisions follow Consortium recommendation and department authorization.

Denial Risk

Denial risk: allogeneic HSCT unproven for germ cell tumors

Allogeneic HSCT is considered unproven and not medically necessary for germ cell tumors; requests for allogeneic transplant for these diagnoses may be denied unless compelling justification and Medical Director escalation are provided.

For germ cell tumor indications, autologous high‑dose therapy may be appropriate; allogeneic SCT is not supported by the policy.
If an allogeneic approach is proposed, document exceptional rationale and seek Medical Director review.

Denial Risk

Denial risk: allogeneic HSCT unproven for listed brain tumors

Allogeneic HSCT is considered unproven and not medically necessary for the listed brain tumors (e.g., GBM, anaplastic astrocytoma) and such requests may be denied without strong justification and Medical Director review.

Autologous HSCT may be considered for certain brain tumors (medulloblastoma, oligodendroglioma, pineoblastoma, ETMR, GBM in infants); document specifics if proposing autologous approach.
Allogeneic requests for listed brain tumors should include explicit justification and be escalated.

Denial Risk

Denial risk: physiologic exclusion thresholds and unresolved relative contraindications

Transplant may be denied if patients meet listed physiologic exclusion thresholds or unresolved relative contraindications (examples: FVC <45% predicted, creatinine clearance <40 ml/min, LVEF <50%, uncontrolled infection, severe end‑stage organ damage, lack of psychosocial support).

Confirm FVC, creatinine clearance, and LVEF meet policy‑specified minimums and resolve uncontrolled infections prior to request.
Provide psychosocial assessment and caregiver/support documentation to mitigate denial risk.

Denial Risk

Denial risk for narsoplimab: inadequate TA‑TMA diagnostic documentation

For narsoplimab (Yartemlea), lack of documented TA‑TMA diagnostic criteria—either biopsy confirmation or fulfillment of ≥4 of 7 criteria at two consecutive time points within 14 days—may trigger denial of the request.

Provide biopsy results if available; otherwise document the 7 clinical/lab criteria with dates demonstrating persistence at two timepoints within 14 days.
Include baseline severity and planned monitoring/follow‑up data.

Denial Risk

Denial risk: absence of early HCT consultation for high‑risk AML

Early HCT consultation is expected for indicated high‑risk AML patients (e.g., primary induction failure, persistent MRD, CR1 except favorable‑risk); failure to document early transplant consultation may delay authorization or risk denial.

Document date and content of HCT consultation or rationale if referral is pending.
If early consultation was not done, explain timing and any attempts to expedite evaluation.

Denial Risk

Denial risk: missing diagnostic or indication documentation may trigger denial

Requests lacking documented indication, diagnostic confirmation, or required diagnostic elements for plasma cell disorders or multiple myeloma (all three required elements) may be denied; include full diagnostic evidence to avoid denial.

Ensure bone marrow, protein studies, and CRAB/MDE documentation are all present for multiple myeloma cases.
For inherited metabolic disorders or hemoglobinopathies, provide diagnostic evidence per appendix entries.

Denial Risk

MDS (Medicare): prognostic‑score dependent coverage — include scores

Medicare coverage for allogeneic HCT in MDS depends on meeting specified prognostic score thresholds (IPSS ≥1.5; IPSS‑R ≥4.5; IPSS‑M ≥0.5); failure to meet these scores may result in noncoverage for Medicare members.

For Medicare patients, include the prognostic score used and the score value meeting the stated threshold.
If scores do not meet thresholds, provide alternative justification or plan for Medical Director review.

Denial Risk

Denial risk: incomplete multiple myeloma diagnostic documentation may lead to denial

Failure to document all three required diagnostic elements for multiple myeloma (monoclonal plasma cells >10% or plasmacytoma; monoclonal protein in serum/urine or nonsecretory criteria; CRAB feature or IMWG MDE) may lead to denial of transplant eligibility claims.

Provide complete marrow, protein, and organ‑dysfunction/MDE documentation in the submission.
If any element is absent, state the reason and supply supporting evidence or request Medical Director review.

Note

Quick reference limitations — consult full guideline and Medical Director when needed

The Appendix K quick reference is limited and should not be used alone for authorization decisions; exceptions (such as clinical trial enrollment) and medical director escalation paths are detailed in the full guideline and must be followed.

If relying on Appendix K, attach supporting full‑guideline documentation when exceptions apply.
Follow Medical Director escalation instructions for cases not clearly supported by the quick reference.

Denial Risk

Appendix K mismatch may trigger coverage denial — document or escalate

If the member’s diagnosis does not match an Appendix K row flagged 'Yes' for the requested transplant type, coverage may be denied; providers must document support for the indicated transplant type or request Medical Director review.

Explicitly state the appendix row and its autologous/allogeneic flags in the prior authorization.
Provide rationale or supporting evidence if requesting a transplant type not flagged 'Yes' in Appendix K.

Documentation requirementMeasured rUPCR should be provided in prior authorization for narsoplimab

Hypertension threshold (pediatric/adult)

Hypertension thresholds>99th percentile for age (<18 years) OR systolic BP ≥140 mmHg OR diastolic BP ≥90 mmHg (≥18 years)

Use in TA-TMA criteriaAge-based hypertension is one of the 7 diagnostic criteria for TA-TMA

Prior authorization noteBlood pressure measurements and age thresholds should be documented when requesting TA-TMA therapy

Elevated LDH threshold (> ULN for age)

Elevated LDH> upper limit of normal (ULN) for age

Role in TA-TMA diagnosisElevated LDH > ULN is one of the 7 clinical/lab criteria; requires documentation on two consecutive time points or biopsy

Interpretation noteLDH should be interpreted against age‑specific ULN and correlated with other hemolysis markers

sC5b-9 threshold

sC5b-9 threshold≥ ULN

Use in TA-TMA criteriaElevated soluble C5b‑9 (sC5b‑9) at or above ULN is one of the 7 diagnostic criteria

DocumentationLaboratory result with reference range required for prior authorization of narsoplimab

ALL MRD threshold after consolidation

ALL MRD after consolidationMRD ≥ 0.01% following consolidation (9–12 weeks from diagnosis)

TimingApplied after consolidation phase (9–12 weeks) to guide HCT consideration in ALL (age ≤39 years)

DocumentationMRD assay sensitivity and numeric result should be included in authorization records

ALL MRD threshold after re-induction for relapse

ALL MRD after re-induction for relapseMRD+ defined as >0.1% for medullary disease (or equivalent for extramedullary disease) after re-induction (4–8 weeks from relapse)

ContextUsed to determine transplant candidacy in relapsed ALL aiming for CR2+

DocumentationSpecify timing of MRD measurement relative to re-induction and assay sensitivity

Transcranial Doppler velocity threshold for sickle cell considerations

Transcranial Doppler (TCD) velocity threshold≥ 200 cm/sec OR > 185 cm/sec with intracranial vasculopathy

Use in sickle cell HSCT candidacyAbnormal TCD velocity is an aggressive-feature trigger for transplant consideration in SCD

DocumentationProvide TCD report with measured velocities and presence/absence of vasculopathy in prior authorization

Tricuspid regurgitant jet (TRJ) velocity threshold

Tricuspid regurgitant jet (TRJ) velocity≥ 2.7 m/sec

Clinical contextListed as an adult-focused threshold indicative of cardiopulmonary risk in sickle cell disease transplant consideration

DocumentationEchocardiography report with measured TRJ velocity should be included for transplant evaluation

Pulmonary function decline threshold (FVC/DLCO <80% predicted)

Pulmonary function decline thresholdDecrease to < 80% predicted in FVC and/or DLCO

Use in systemic sclerosis candidacyFVC/DLCO decline to <80% predicted is part of internal organ involvement criteria for autologous HSCT

DocumentationProvide pre- and serial PFTs with percent predicted values for authorization

Ambulation requirement (walk 100 m) for MS candidacy

Ambulation requirement for MS candidacyAble to walk 100 meters (unilateral assistance allowed)

Use in MS criteriaRequired functional threshold before considering autologous HSCT after relapse history

DocumentationDocumented walking distance and assistance level (e.g., cane) required for prior authorization

Chronic pain duration threshold (≥6 months)

Chronic pain duration threshold≥ 6 months

Context in SCDChronic pain ≥6 months (e.g., leg ulcers, avascular necrosis) is an indication feature for transplant consideration

DocumentationClinical notes documenting chronic pain duration and related complications should be submitted

IPSS threshold for Medicare MDS coverage (≥1.5)

IPSS threshold (Medicare MDS)IPSS ≥ 1.5 (Intermediate-2 or High) required for Medicare coverage of allogeneic HCT in MDS

Policy linkageMedicare NCD requires meeting at least one prognostic-score threshold for coverage

DocumentationInclude IPSS score calculation and source data in prior authorization for Medicare members

IPSS-R threshold for Medicare MDS coverage (≥4.5)

IPSS-R threshold (Medicare MDS)IPSS-R ≥ 4.5 (High or Very High) required for Medicare coverage of allogeneic HCT in MDS

Application noteOne of three acceptable prognostic-score thresholds for Medicare coverage (NCD)

DocumentationProvide IPSS-R score worksheet and underlying labs/cytogenetics for authorization reviews

IPSS-M threshold for Medicare MDS coverage (≥0.5)

IPSS-M threshold (Medicare MDS)IPSS-M ≥ 0.5 (High or Very High) required for Medicare coverage of allogeneic HCT in MDS

ContextIPSS-M is an acceptable prognostic metric per CMS NCD for Medicare coverage determination

DocumentationInclude molecular testing results and IPSS-M calculation when used for authorization

Clonal plasma cell percentage guidance (MGUS <10%; MDE if ≥60%)

Clonal plasma cell percentage guidanceMGUS: bone marrow clonal plasma cells <10%; Myeloma MDE if ≥60%; Smoldering myeloma upper marrow plasma cell limit 60%

Diagnostic role≥60% clonal plasma cells is an IMWG myeloma‑defining event sufficient for diagnosis regardless of CRAB

DocumentationReport bone marrow plasma cell percentage and correlate with IMWG criteria in transplant authorization

Serum involved/uninvolved free light chain ratio threshold (≥100)

Serum involved/uninvolved free light chain ratio threshold≥ 100 with involved FLC ≥ 100 mg/L (IMWG MDE)

Diagnostic significanceOne of three IMWG myeloma-defining events — sufficient for myeloma diagnosis regardless of CRAB

DocumentationProvide free light chain numeric results with reference ranges when used for transplant candidacy

Calcium threshold for CRAB (>10.5 mg/dl or above ULN)

Calcium threshold (CRAB)>10.5 mg/dl or above the laboratory upper limit of normal

Use in myeloma diagnosisHypercalcemia meeting this threshold is a CRAB feature qualifying for myeloma-related organ dysfunction

DocumentationReport serum calcium value and reference range in diagnostic documentation

Creatinine threshold for CRAB (>2 mg/dl)

Creatinine threshold (CRAB)> 2 mg/dl

Use in myeloma diagnosisRenal insufficiency with creatinine >2 mg/dl is a CRAB feature for myeloma-defining organ dysfunction

DocumentationInclude serum creatinine and baseline renal function when asserting CRAB criterion

Hemoglobin threshold for CRAB (<10 g/dl or ≥2 g below normal)

Hemoglobin threshold (CRAB)Hemoglobin < 10 g/dl OR ≥ 2 g/dl below normal

Use in myeloma diagnosisAnemia meeting this threshold constitutes a CRAB feature for myeloma-defining organ dysfunction

DocumentationProvide hemoglobin values and normal reference range in diagnostic documentation

mRSS per-site score (0–3)

mRSS per-site score range0–3 per site (17 sites assessed)

Use in systemic sclerosismRSS site scores summed and documented; thresholds for candidacy referenced in systemic sclerosis criteria

DocumentationComplete mRSS form with site scores, total, examiner name and date expected in evaluation records

EDSS range (0–10)

EDSS score range0–10 scale

Use in MS candidacyEDSS used to document neurologic disability; policy references EDSS range and ambulatory thresholds (100 m walk) for HSCT consideration

DocumentationInclude EDSS score and date in prior authorization for MS patients considered for HSCT

Boost is an infusion to assist hematopoietic recovery or declining donor chimerism and is not preceded by preparative regimen

Coding noteCPT 38243 cited as existing code for boost procedures

Donor lymphocyte infusion — medically necessary post-allogeneic HSCT when indicated

Donor lymphocyte infusion (DLI) coverage stanceDLI is medically necessary post-allogeneic HSCT when intended for relapse management, chimerism conversion, or planned relapse prevention with donor-derived lymphocytes

RequirementsDonor lymphocytes must be from the original HSCT donor; indication and intent should be documented for authorization

EvidenceMulticenter retrospective data (n=271) support prophylactic/preemptive strategies in high-risk acute leukemia

Remestemcel-L-rknd (Ryoncil) for pediatric SR-aGVHD — FDA-approved indication and dosing summary

Ryoncil FDA indication and dosingFDA-approved for pediatric steroid‑refractory acute GVHD in patients 2 months to 17 years; 2 x 10^6 MSCs/kg IV twice weekly for 4 weeks (infusions ≥3 days apart)

Day 28 response‑guided retreatment rulesDay 28 ±2 assessment: CR → stop; PR/MR → once weekly x4 additional infusions; recurrence after CR → twice weekly x4; no response → consider alternatives; other retreatments unproven

Supporting evidencePhase 3 single-arm pediatric study (n=54) with Day 28 OR primary endpoint and follow‑up outcomes described

Screening for TA-TMA — weekly for first 100 days for all allogeneic and pediatric autologous neuroblastoma recipients

TA-TMA screening periodWeekly screening for TA-TMA during the first 100 days post-HCT for all allogeneic and pediatric autologous neuroblastoma recipients

RationaleConsensus recommendation from expert panels to detect and risk-stratify TA-TMA early

DocumentationScreening schedule and results should be available in the transplant chart for post‑transplant surveillance

Allogeneic HCT consideration post-CAR-T — MRD-negative CR recipients may be considered for HCT

Allogeneic HCT consideration post‑CAR‑TCAR‑T recipients who achieve MRD‑negative CR but have not had HCT may be considered for subsequent allogeneic HCT

ContextApplied in selected CD19 and other CAR‑T therapy responders when further consolidation with HCT is under consideration

DocumentationProvide CAR‑T product, response status (MRD‑negative CR), and rationale for proceeding to HCT in authorization materials

General HSCT follow-up and outcomes references cited

References and outcomes supportMultiple literature references cited to support indications and outcomes for HSCT across diseases

ImplicationClinical decisions and prior authorization should reference the cited guideline and evidence sources when relevant

No explicit durationsAppendix references provide support but do not define post‑transplant coverage durations in these chunks

Disease response assessment methods for post-transplant monitoring

Disease response assessment methodsPost‑transplant monitoring should use established response criteria including imaging, marrow biopsy, and PET per lymphoma response appendix

ExamplesComplete remission and partial remission criteria (Cheson/revised criteria) referenced for lymphoma assessment

DocumentationProvide imaging modality, date, and response interpretation in post‑transplant follow‑up records

Acute GVHD staging and grading assessments — use MAGIC criteria

Acute GVHD staging/grading assessmentUse MAGIC criteria to stage target‑organ involvement and assign overall clinical grade I–IV

DocumentationRecord organ stages and overall grade per MAGIC criteria for management and authorization of GVHD therapies

PurposeStandardized staging supports consistency for treatment decisions and prior authorization reviews

TA-TMA treatment — new treatments (narsoplimab/Yartemlea) noted in review

TA‑TMA treatments notedPolicy review notes include newer treatments for TA‑TMA such as narsoplimab (Yartemlea) in addition to review of prior therapies

Evidence noteNarsoplimab received FDA approval (Dec 2025) and real‑world pediatric/adult data referenced in appendices

ImplicationPrior authorization for TA‑TMA therapies requires diagnostic criteria documentation and treatment rationale

The appendices emphasize diagnostic classifications and response criteria rather than explicit transplant exclusions. As such, the policy does not list all absolute or relative contraindications in the appendix entries; those contraindications are described in the disease‑specific sections and pre‑transplant suitability criteria.

Across the disease‑specific sections the document references physiologic thresholds and exclusionary conditions (for example cardiopulmonary and renal thresholds for systemic sclerosis). However, the provided chunks do not compile a single comprehensive list of absolute and relative contraindications; the policy therefore notes that absolute and relative contraindications are not fully detailed in these excerpts and reviewers must consult the full policy for the complete contraindication framework.

Placeholder for contraindication content not included in the excerpted chunks.

Placeholder for additional contraindication entries; refer to the full policy document for complete lists.

Clinical noteBoost may be used for poor graft function or declining chimerism per treating center judgment

Myelofibrosis — brief definition

Myelofibrosis definitionA rare disorder with splenomegaly, anemia, and marrow fibrosis caused by clonal hematopoietic stem cell mutations

EpidemiologyIncidence approx. 0.25 per 100,000

Clinical implicationMay be indication for allogeneic HSCT when DIPSS‑Plus Intermediate‑2 or High risk

Multiple myeloma (MM) definition

Multiple myeloma (MM) definitionPlasma cell malignancy with monoclonal plasma cell proliferation in marrow, M protein, bone destruction, and displacement of other hematopoietic lineages

Disease spectrumIncludes MGUS and smoldering myeloma as precursor states

Clinical implicationDiagnosis guides autologous/allogeneic HSCT considerations per IMWG/CRAB criteria

DLI definition

DLI definitionDonor lymphocyte infusion (DLI) is adoptive immunotherapy using lymphocytes from original HSCT donor to induce graft‑versus‑tumor effect

Use casesManagement of relapsed/persistent disease, chimerism conversion, or prophylaxis in high‑risk patients

RequirementDonor lymphocytes must be from original donor per policy criteria

Omidubicel / Omisirge definition

Omidubicel/Omisirge definitionAn ex vivo expanded hematopoietic progenitor cell product derived from a single umbilical cord blood unit used to reduce time to neutrophil recovery and infections after myeloablative HSCT

Age-based approvalsApproved for hematologic malignancy in patients ≥12 years; approved for SAA in patients ≥6 years under specified conditions

Clinical useConsidered when no readily available matched donor and goal is to reduce neutrophil recovery time

Partial response (PR) definition

Partial response (PR) definitionRegression of measurable disease and no new sites; specific radiologic/biopsy criteria apply

UseUsed in response assessment post‑therapy/transplant per lymphoma criteria

DocumentationImaging and marrow biopsy results supporting PR should be included in records

Mixed response (MR) definition

Mixed response (MR) definitionImprovement in at least one evaluable organ with worsening in another organ (GVHD context) or heterogeneous tumor responses in oncology contexts

UseMR designation impacts retreatment rules for Ryoncil and other therapies

DocumentationSpecify organ‑level changes or lesion‑level changes when using MR designation

Measurable residual disease (MRD) definition and use

MRD definition and useMeasurable residual disease (MRD) denotes disease detected at low levels after therapy; MRD thresholds are used to guide transplant decisions (e.g., ALL and AML) and assay sensitivity may be 10^-5 or 10^-6

ContextMRD informs timing of HCT consultation and candidacy across leukemias

DocumentationInclude numeric MRD result, assay sensitivity, and timing relative to therapy in authorization records

CR1/CR2 definition

CR1/CR2 definitionComplete remission 1 or 2 (first or second complete remission) used as timing markers for considering HCT

UseCR1/CR2 status specified across leukemia indications and impacts transplant timing

DocumentationProvide documentation of remission status and dates in authorization requests

TCD abnormal velocity definition

TCD abnormal velocity definitionAbnormal TCD velocity defined as ≥ 200 cm/sec or >185 cm/sec with intracranial vasculopathy

UseUsed as an aggressive‑feature criterion for sickle cell disease transplant consideration

DocumentationProvide TCD study report including measured velocities and vessel status for authorization

TRJ velocity definition

TRJ velocity definitionTricuspid regurgitant jet velocity threshold noted as ≥ 2.7 m/sec (adult‑focused)

UseConsidered among cardiopulmonary complications in sickle cell disease transplant assessment

DocumentationEchocardiogram TRJ measurement should be included in candidate evaluation

Mobility requirement for MS (walk 100 m)

Mobility requirement for MSPatient must be able to walk 100 meters (may use unilateral assistance) to be considered for transplant after relapse history

Relation to EDSSAmbulatory capacity aligns with EDSS functional thresholds used in candidacy assessment

DocumentationProvide objective walking test results or clinician assessment documenting 100‑meter ability

AML genetic risk groups definition

AML genetic risk groupsFavorable, Intermediate, or Adverse risk groups based on specific cytogenetic and molecular abnormalities as listed in Appendix A

UseRisk group informs transplant consideration (e.g., CR1 exceptions for favorable risk) and MRD interpretation

DocumentationReport cytogenetic/molecular findings and assigned risk group in authorization materials

MDS prognostic score thresholds (Medicare) definition

MDS prognostic score thresholds (Medicare)Medicare coverage for allogeneic HCT contingent on IPSS ≥1.5 OR IPSS‑R ≥4.5 OR IPSS‑M ≥0.5

SourceCMS NCD referenced in Appendix B

DocumentationProvide scoring worksheets and underlying data for Medicare member authorization

DIPSS / DIPSS-Plus definition

DIPSS / DIPSS‑Plus definitionRisk scoring systems for primary myelofibrosis combining age, hemoglobin, WBC, blasts, platelet count and additional variables (DIPSS‑Plus) to categorize risk

UseDIPSS categories (Low, Int‑1, Int‑2, High) inform transplant candidacy; DIPSS‑Plus adds adverse karyotype, platelets <100, RBC transfusion need

DocumentationProvide DIPSS/DIPSS‑Plus score and component data when used for HCT authorization

CRAB criteria definition

CRAB criteria definitionMyeloma‑related organ dysfunction: hypercalcemia (calcium >10.5 mg/dl or above ULN), renal insufficiency (creatinine >2 mg/dl), anemia (Hb <10 g/dl or ≥2 g below normal), and bone disease (lytic lesions)

UseCRAB features constitute myeloma‑related organ dysfunction required for diagnosis alongside marrow/protein criteria

DocumentationProvide laboratory and imaging evidence for CRAB features in transplant evaluations

IMWG myeloma-defining events (MDEs) definition

IMWG myeloma-defining events (MDEs)Any one of: ≥60% clonal plasma cells in bone marrow; involved/uninvolved serum FLC ratio ≥100 with involved FLC ≥100 mg/L; >1 focal MRI lesion ≥5 mm — each sufficient for diagnosis regardless of CRAB

Clinical impactPresence of an MDE qualifies for myeloma diagnosis and influences urgency for transplant consideration

DocumentationProvide quantitative bone marrow %, FLC numeric results, and MRI lesion descriptions when citing MDEs

MGUS definition

MGUS definitionLow‑level serum/urine monoclonal protein, bone marrow plasma cells <10%, normal calcium/hemoglobin/creatinine, urine monoclonal protein <1.0 g/24 h, and no bone lesions or amyloidosis

Clinical implicationMGUS is distinguished from smoldering myeloma and active myeloma for transplant eligibility

DocumentationProvide serum/urine protein quantification and bone marrow percentage to support MGUS classification

Smoldering myeloma definition

Smoldering myeloma definitionMonoclonal protein and clonal plasma cells in marrow/tissue without meeting criteria for MGUS or active myeloma; upper marrow clonal plasma cell limit 60%

Clinical implicationSmoldering myeloma patients should lack myeloma‑defining events or amyloidosis; helps determine timing of transplant evaluation

DocumentationProvide marrow plasma cell percentage, monoclonal protein levels, and absence of MDEs for classification

Expanded Disability Status Scale (EDSS) definition

EDSS definitionExpanded Disability Status Scale is a non‑linear 0–10 scale for evaluating neurologic disability in MS (0 normal, 10 MS‑related death)

UseEDSS used to document disability in MS transplant candidacy evaluations

DocumentationInclude EDSS score and date in authorization materials for MS patients

Modified Rodnan Skin Score (mRSS) definition

mRSS definitionModified Rodnan Skin Score: 17‑site palpation instrument grading skin thickness 0–3 per site, summed to a total

UsemRSS used to quantify skin involvement in systemic sclerosis and inform transplant candidacy

DocumentationComplete mRSS form with site scores, total, examiner, and date expected in pre‑transplant evaluation

MAGIC criteria (acute GVHD) definition

MAGIC criteria definitionStaging of acute GVHD target‑organ involvement (skin, liver, upper/lower GI) with numeric stages and overall clinical grade I–IV

UseRecommended for staging and grading acute GVHD to guide management and documentation

DocumentationRecord organ stages and overall grade per MAGIC in GVHD treatment authorization

Autologous vs Allogeneic flags — appendix indexing explanation

Autologous vs Allogeneic flags (Appendix indexing)Appendix K rows include two flags: '1' for autologous candidacy and '2' for allogeneic candidacy; each flagged Yes or No to indicate coverage permissibility

UseProviders must match member diagnosis and requested transplant type to Appendix K flags for quick‑reference coverage mapping

DocumentationIndicate requested transplant type (autologous=1, allogeneic=2) on authorization requests to align with Appendix K

Administrative review entry noted (16.0) in May 2026 reflecting content updates and approval workflow entries.

2025-12regulatory_update

FDA approved narsoplimab-wuug (Yartemlea) for treatment of TA-TMA in patients ≥2 years, and the policy incorporated this approval and related TA-TMA diagnostic/coverage criteria.

2024-12-26evidence_update

Health technology assessment (Hayes) on remestemcel-L-rknd (Ryoncil) for acute GVHD informed formalization of pediatric age, dosing, and Day-28 response–guided continuation/retreatment rules included in the policy.

2024-05-21evidence_publication

Real-world experience publication on narsoplimab safety and efficacy in pediatric and adult TA-TMA patients cited in the policy bibliography and used to inform coverage language.

2017-2025literature_updates

Multiple referenced literature items (clinical trials, guidelines, consensus documents) from 2017–2025 were added to support indications and criteria across the policy (bibliography entries).

2026-08-01material_revisionLatest

Material policy changes implemented: narsoplimab (Yartemlea) FDA approval and TA-TMA coverage criteria added; remestemcel-L (Ryoncil) pediatric dosing and Day-28 response–guided retreatment rules formalized; defibrotide coverage clarified to be treatment-only (not prophylaxis).

2025-12drug_approval_added

Narsoplimab-wuug (Yartemlea) FDA approval for TA-TMA (Dec 2025) incorporated into the policy with diagnostic criteria and age threshold (≥2 years).

2024-12-26clinical_policy_update

Remestemcel-L-rknd (Ryoncil) pediatric SR-aGVHD coverage language added with explicit age range (2 months–17 years), dosing regimen, and Day 28 response–guided continuation/retreatment rules per product assessments and evidence review.

2026-06-10review_note

Review and approval notes in June 2026 document the incorporation of the above material changes into the policy revision history.

Document version and approvals: This policy was updated during the 2026 annual review cycle and approved by the relevant expert panels and committees. The review history documents the inclusion of new indications and treatment additions (e.g., narsoplimab/Yartemlea) and the dates of the most recent annual reviews (May–June 2026).