Modifiedpriority healthPolicy N/A

Stem cell or bone marrow transplantation

Defines Priority Health coverage, criteria, and limits for autologous and allogeneic stem cell, bone marrow, peripheral blood, and other blood cell transplants for various indications; applies to members whose plans include this benefit and providers seeking prior authorization.

Policy Summary

Payerpriority health

PolicyStem cell or bone marrow transplantation

Policy CodePolicy N/A

Change TypeAdded coverage statement for AHSCT in multiple sclerosis

Effective DateJun 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization on the specified transplant form and ensure procedures are performed at a Priority Health approved facility.

SourceLink

POLICY UPDATE CHANGES

Autologous hematopoietic stem cell transplantation for the treatment of multiple sclerosis may be considered medically necessary when criteria are met.

New Related policies section

1 yearfollow-up coverage post-transplant

18-45age limit for AHSCT (MS)

<10 yrsdisease duration limit (MS)

3max tandem HSCTs (neuroblastoma)

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

transplant-related mortality (AHSCT in MS)

12 studiessystematic review size (Crohn's aHSCT)

Coverage Criteria and Indications

Acute Lymphocytic Leukemia (ALL) and Chronic Lymphocytic Leukemia (CLL)/SLL

Allogeneic stem cell transplantation is a covered benefit for the treatment of acute lymphocytic leukemia (ALL) and for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) in specific clinical circumstances. Autologous transplantation and repeat transplantation have specified coverage rules described below.

Allogeneic stem cell transplantation is a covered benefit for the treatment of acute lymphocytic leukemia (ALL), including primary refractory ALL (i.e., leukemia that does not achieve a complete remission after conventional dose chemotherapy), except for refractory relapse defined as persons in relapse who are unresponsive to 3 or more months of adequate chemotherapy.

A non-myeloablative allogeneic hematopoietic cell transplantation (mini-allograft or reduced intensity conditioning transplant) is a covered benefit for the treatment of ALL for members with no persistent disease who meet the general selection criteria for transplantation. Note: Persons with persistent disease should not be candidates for a mini-allograft transplant.

A second myeloablative allogeneic HSCT from an appropriately-matched HLA donor is a covered benefit for the treatment of ALL when relapsed disease occurs more than six months after the first allogeneic SCT.

Autologous stem cell transplantation is a covered benefit for ALL when no suitable donor is available.

Tandem stem cell transplant for ALL is considered experimental and not a covered benefit.

Allogeneic stem-cell transplantation is a covered benefit for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) that is not responsive to standard therapy.

Autologous stem cell transplantation is a covered benefit for CLL in an individual in complete or good partial remission.

For both ALL and CLL/SLL, repeat allogeneic (ablative or non-myeloablative) hematopoietic cell transplantation is a covered benefit when the first transplant was unsuccessful due to primary graft failure or failure to engraft or when relapse occurs and the member otherwise meets selection criteria.

For ALL and CLL/SLL, autologous transplantation as initial therapy where evidence supports its use is addressed above; however, high-dose chemotherapy with autologous stem cell support is considered investigational for certain leukemias (see policy sections on AML and CML) and should be evaluated in the context of the specific diagnosis and available donor options.

All transplants must meet the General Coverage Criteria: adequate major organ function, predicted ability to tolerate procedure and posttransplant therapy, emotional/psychiatric stability and support, ability to understand risks, and absence of exclusions such as active substance abuse, psychiatric disease interfering with compliance, or significant history of medical noncompliance. All transplants require prior authorization and must be performed at Priority Health–approved facilities.

Candidate Selection and Contraindications

Candidate Requirements

Candidate evaluation requirements and indication-specific criteria

Candidate baseline requirements: Adequate liver, cardiac, pulmonary, and renal function; predicted ability to tolerate the procedure and immunosuppression; emotional/psychiatric stability with documented social work evaluation; ability to understand risks; transportation/lodging not covered

General prerequisites for transplant candidacy

Behavioral contraindications: Active substance or alcohol abuse; psychiatric disease interfering with regimen; significant medical noncompliance; unwillingness or inability to adhere to post‑transplant restrictions

These conditions preclude coverage

Candidate criteria (disease-specific)

Coverage decisions by disease‑specific candidate criteria summarized as statements in policy sections:

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior Authorization Required. All bone marrow, peripheral stem cell, or other blood cell transplants (including allogeneic and autologous procedures) must be preauthorized by Priority Health and performed at a Priority Health–approved facility. When prior authorization is required, the provider must submit the Bone Marrow/Peripheral Stem Cell or Other Blood Cell Transplant prior authorization form and demonstrate that the requested procedure is medically necessary per policy criteria.

Preauthorization form: Bone Marrow/Peripheral Stem Cell or Other Blood Cell Transplant prior authorization form.
Approved facility: Transplants must be performed at a Priority Health approved facility; referrals should be to in-network or contracted transplant centers (see Provider Manual).
When required: Providers must document medical necessity and supply requested clinical documentation (see documentation callouts).

Prior Authorization

Prior Authorization for AHSCT in Multiple Sclerosis

Prior authorization is required for consideration of autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis (MS). Requests must include documentation that the member meets specialty guideline-based candidate criteria (see clinical guidelines cited) — e.g., relapsing forms of MS with ongoing clinical relapses or MRI activity despite high‑efficacy disease‑modifying therapies, appropriate EDSS and age/duration considerations, or aggressive MS with rapid disability progression. Providers must show prior trials of appropriate DMTs (or justify exceptions) and supply neurologic assessments, MRI reports, and treatment history.

Coding and Billing

ICD-10 Codes that may applyICD-10

C40.00 - C40.92	Malignant neoplasm of bone and articular cartilage of limbs (Ewings's sarcoma)
C56.1 - C56.9	Malignant neoplasm of ovary
C62.0 - C62.92	Malignant neoplasm of testis
C64.1 - C64.9	Malignant neoplasm of kidney, except renal pelvis (Wilms tumor)
C69.20 - C69.22	Malignant neoplasm of retina
C71.0 - C71.9	Malignant neoplasm of brain
C81.00 - C81.99	Hodgkin lymphoma
C82.00 - C82.99	Follicular lymphoma
C83.00 - C83.99	Small cell B-cell lymphoma
C84.60 - C84.69	Anaplastic large cell lymphoma, ALK-positive

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NOT COVERED INDICATIONS (ICD-10 examples)ICD-10Not Covered

C11.0 - C11.9	Malignant neoplasm of nasopharynx
C15.3 - C15.9	Malignant neoplasm of esophagus
C16.0 - C16.9	Malignant neoplasm of stomach
C18.0 - C20	Malignant neoplasm of the colon
C23	Malignant neoplasm of gallbladder
C24.0 - C24.9	Malignant neoplasm of other and unspecified parts of biliary tract
C25.0 - C25.9	Malignant neoplasm of pancreas
C31.0 - C31.9	Malignant neoplasm of accessory sinuses
C34.00 - C34.92	Malignant neoplasm of bronchus and lung
C37	Malignant neoplasm of thymus

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CPT/HCPCS Codes (selected)mixedCovered

38204	Management of recipient hematopoietic progenitor cell donor search and cell acquisition
38205	Blood-derived hematopoietic progenitor cell harvesting for transplantation, per collection; allogeneic
38206	Blood-derived hematopoietic progenitor cell harvesting for transplantation, per collection; autologous
38207	Transplant preparation of hematopoietic progenitor cells; cryopreservation and storage
38208	Transplant preparation of hematopoietic progenitor cells; thawing of previously frozen harvest, without washing, per donor
38209	Transplant preparation of hematopoietic progenitor cells; thawing of previously frozen harvest, with washing, per donor
38210	Transplant preparation of hematopoietic progenitor cells; specific cell depletion within harvest, T-cell depletion
38211	Transplant preparation of hematopoietic progenitor cells; tumor cell depletion
38212	Transplant preparation of hematopoietic progenitor cells; red blood cell removal
38213	Transplant preparation of hematopoietic progenitor cells; platelet depletion

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Not Covered (listed CPT/HCPCS)CPT | HCPCSNot Covered

0263T	Intramuscular autologous bone marrow cell therapy, with preparation of harvested cells, multiple injections, 1 leg; complete procedure including unilateral or bilateral bone marrow harvest
0264T	Intramuscular autologous bone marrow cell therapy, with preparation of harvested cells, multiple injections, 1 leg; complete procedure excluding bone marrow harvest
0265T	Intramuscular autologous bone marrow cell therapy; unilateral or bilateral bone marrow harvest only for intramuscular autologous bone marrow cell
S2150	Bone marrow or blood-derived stem cells (peripheral or umbilical), allogeneic or autologous, harvesting, transplantation, and related complications; global procedure including pheresis, cell preparation/storage, marrow ablative therapy, drugs, supplies, hospitalization and outpatient follow-up

inv-39: Expanded Disability Status Scale (EDSS)

EDSS score rangeExpanded Disability Status Scale (EDSS) score of 2.0 to 6.0 required for AHSCT consideration in MS

Purpose of EDSS thresholdUsed to define ambulatory and disability range appropriate for AHSCT candidacy

DocumentationPre-transplant evaluation should document neurologic baseline including EDSS

inv-40: Maximum tandem HSCTs for high-risk neuroblastoma

Maximum tandem transplantsA maximum of three (3) tandem autologous HSCTs are covered for high-risk neuroblastoma

Pre-Transplant Evaluation and Testing

Documentation Required

Pre‑transplant evaluation and social work documentation

An approved Bone Marrow, Peripheral Stem Cell, or other Blood Cell Transplant includes one pre‑transplant evaluation (one evaluation per transplant) and requires formal social work documentation of support network as part of candidate assessment.

One pre‑transplant evaluation per transplant is covered.
Formal social work evaluation to document support network and psychosocial stability is required.

Documentation Required

Evaluation requirements — prior therapy failure and HLA‑matched donor when applicable

For some indications (for example intermediate/high‑risk MDS), coverage requires prior therapy failure and an available HLA‑matched donor; prior authorization may require documentation of these elements.

Show that prior therapies failed and that an HLA‑matched donor is available for allogeneic transplants.
Include donor matching and prior treatment records with the authorization request.

Transplant Center and Facility Requirements

Billing Rule

Facility requirement — perform transplants at Priority Health approved facility

All transplants must be performed at a Priority Health approved facility; referrals are directed to network or contracted transplant facilities and an approved transplant includes follow‑up care covered at the transplant facility for one year post‑transplant.

Schedule transplant at an approved Priority Health facility.
Follow referral direction to network/contracted facilities per Provider Manual.

Documentation Required

Center selection and standard transplant center practices

Policy references selection criteria and standard transplant center practices (matching, NDMP criteria) and implies centers should have appropriate expertise; centers performing AHSCT for autoimmune diseases should have relevant accreditation and experience.

Post-Transplant Follow-up and Aftercare

inv-102: Follow-up care and services — routine follow-up coverage at transplant facility for one year

Routine follow-up coverage periodFollow-up care and services are covered at the transplant facility for one year following the transplant (both contracted and non-contracted facilities)

Included servicesPre-transplant evaluation (one per transplant), transplant care, facility and professional fees, donor harvesting, and post-transplant immunosuppressant drug therapy (if group has outpatient drug coverage)

Second opinion provisionA second opinion transplant evaluation may be approved at a contracted or in-network transplant facility if previously turned down for transplant

inv-103: Extended follow-up care — coverage beyond one year at contracted facilities

Extended follow-up beyond one yearFollow-up care beyond one year is covered at contracted transplant facilities; for non-contracted facilities only physician services are covered while testing, labs, and imaging are covered in-network only

Background and Evidence Context

Stem cell and bone marrow transplantation (autologous and allogeneic) are established therapies for hematologic malignancies and certain non‑malignant hematologic disorders. The policy emphasizes that transplant candidates must have adequate organ function, psychosocial stability, and documented support; transplants require preauthorization and performance at an approved Priority Health transplant facility. Follow-up care at the transplant facility is covered for one year post‑transplant.

Autologous stem cell transplantation for Crohn's disease is discussed as an evidence topic: a 2024 systematic review of 12 studies (609 records) reported reductions in Crohn Disease Activity Index (CDAI) and some fistula benefit but also notable adverse events including infections, acute renal failure, and deaths. Despite these signals of efficacy in some studies, autologous SCT for Crohn's disease is listed in the policy as experimental and not a covered benefit.

The policy summarizes investigational or high‑risk transplant indications where evidence is limited or harms may outweigh benefits. Examples include ALS (low‑certainty evidence with minimal functional improvements), type 1 diabetes (conditioning risks outweigh potential glycemic benefits), recessive dystrophic epidermolysis bullosa (high‑risk allogeneic BMT with some wound‑healing benefit but substantial adverse events), and other indications where trial evidence is insufficient to support routine coverage.

Definitions

inv-68: Transplant evaluation (definition)

DefinitionTransplant evaluation: pre-transplant evaluation performed as part of pre-transplant care; one evaluation per transplant is covered

Documentation elementsEvaluation includes assessment of organ function, psychosocial evaluation (formal social work), and determination of ability to tolerate procedure and immunosuppression

Second opinionA second opinion consult to determine transplant candidacy may be approved at a contracted or in-network transplant facility if previously turned down

inv-69: EDSS 2.0 to 6.0 definition

EDSS range defined for eligibilityEDSS 2.0 to 6.0 specifies the required Expanded Disability Status Scale range for AHSCT candidacy in MS

Clinical implication

Policy Changes and Revision History

2025-06-01policy_effectiveLatest

Policy effective date; autologous hematopoietic stem cell transplantation for multiple sclerosis added as a medically necessary option when criteria are met.

section_added

Related policies section added listing policy numbers 91117, 91448, and 91606.

Policy status: This document is for informational purposes only and is not an authorization, certification, explanation of benefits, or contract. The policy's effective and review dates are documented in the policy header; eligibility and benefit coverage are determined in accordance with the member's plan in effect on the date services are rendered.

Policy Summary

Payerpriority health

PolicyStem cell or bone marrow transplantation

Policy CodePolicy N/A

Change TypeAdded coverage statement for AHSCT in multiple sclerosis

Effective DateJun 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization on the specified transplant form and ensure procedures are performed at a Priority Health approved facility.

SourceLink

On This Page

HLA‑matched donor requirement for MDS: Allogeneic SCT is covered for intermediate/high‑risk MDS when an available HLA‑matched donor exists and prior therapy has failed.

ALL - mini‑allograft candidate: Non‑myeloablative allogeneic (mini‑allograft) is for members with no persistent disease who meet selection criteria; persons with persistent disease should not be candidates.

Transplant candidate criteria

Candidate selection notes drawn from evidence and guideline excerpts in this section:

Crohn's disease candidate: Severe, refractory Crohn's disease with failure of conventional medical and surgical therapies; patient considered for autologous HSCT after multidisciplinary evaluation and documentation of refractory disease.

~25% of CD patients may be refractory; systematic review showed CDAI reduction but notable adverse events

MS candidate: MS patients with inflammatory disease activity not responding to approved DMTs; younger and ambulatory patients preferred for autologous HSCT per EBMT guidance; documentation of relapses or MRI activity and prior DMT failure recommended.EDSS 2.0–6.0

EBMT recommends AHSCT for RRMS not responding to DMTs and for selected SPMS/PPMS with activity

AHSCT candidate criteria (MS)

MS‑specific AHSCT candidate features recommended by professional societies and supported by trial data

Recommended candidate features for AHSCT in MS: Evidence of inflammatory activity (clinical relapses and/or Gd‑enhancing or new T2 MRI lesions); relapsing forms of MS (RRMS) or PPMS/SPMS with superimposed activity; failure of or contraindication to high‑efficacy DMTs OR aggressive disease course; ambulatory (EDSS ≤5.5), younger age (preferably <45–50 years), and disease duration <10 years for optimal outcomes; documented disability progression in prior 12 months when applicable.EDSS ≤5.5; age preferably <45–50; disease duration <10 years

Based on EBMT, ASTCT/ABMTR, NMSS statements and randomized trial (Burt et al.)

Umbilical cord blood stem cell transplant (UCBSCT) is covered only for patients who meet all eligibility requirements for an allogeneic stem cell transplant. UCBSCT is not covered for patients who do not meet patient selection criteria for allogeneic transplantation. Nonmyeloablative transplants in patients who do not meet selection criteria due to age or comorbidities are considered investigational.

Significant concern about conditioning‑related toxicity (for example, risk of bone marrow aplasia, sepsis, or other life‑threatening complications) may contraindicate HSCT for certain indications. The policy cites these conditioning‑related risks as a reason against HSCT for high‑risk non‑established indications (eg, routine treatment of type 1 diabetes) and as a safety consideration when assessing candidacy.

Required documentation for AHSCT in MS: neurological evaluation including EDSS score, MRI demonstrating active inflammatory disease (new/enhancing T2 lesions), detailed DMT history and reason for failure/intolerance, disease duration, age, and ambulatory status.
Guideline alignment: ASTCT, EBMT/JACIE, and NMSS recommendations support AHSCT for select relapsing MS patients with high inflammatory activity despite DMTs.
Prior authorization must precede scheduling of AHSCT and demonstration of medical necessity is required.

Denial Risk

Experimental / Investigational / Unproven Procedures (Not Covered)

Experimental, investigational, or unproven indications are not covered. Procedures listed below are considered experimental/unproven and will be denied unless coverage is granted under Priority Health's Experimental/Investigational/Unproven Care Exception policies or applicable clinical trials exceptions.

Autologous stem cell transplantation for Crohn's disease (except per separate investigational review pathway)
Stem cell therapy for erectile dysfunction
Autologous bone marrow cells (including transendocardial delivery) for coronary artery disease, left ventricular dysfunction, heart failure, or angina
Age‑related macular degeneration
Amyotrophic lateral sclerosis
Type 1 diabetes mellitus
Essential thrombocythemia
Polycythemia vera
Recessive dystrophic epidermolysis bullosa
Retinitis pigmentosa
Thrombotic thrombocytopenic purpura

Denial Risk

Safety‑Triggered Denials

Safety concerns may trigger denial of transplant requests. High treatment‑related risk, inability to tolerate conditioning regimens, or evidence of organ dysfunction or comorbidities that increase the risk of treatment‑related mortality are grounds for noncoverage.

Safety‑triggered denials may occur for procedures with unacceptable risk (for example, severe organ dysfunction, active uncontrolled infection, or other contraindications to conditioning).
Evidence of severe treatment‑related adverse events in the literature (including trial reports of renal failure, thrombotic microangiopathy, pulmonary veno‑occlusive disease, and treatment‑related deaths) underscores the need to document that the member can tolerate conditioning and post‑transplant care.

Denial Risk

Safety‑Related Denial Risk

Providers should be aware of specific safety‑related denial risks associated with transplantation conditioning and patient selection. Documented inadequate organ function, active substance abuse, uncontrolled psychiatric disease, or significant noncompliance history may result in denial due to unacceptable peri‑transplant risk.

Common safety‑related denial triggers: inadequate hepatic, cardiac, pulmonary, or renal function; active substance or alcohol abuse; psychiatric conditions interfering with adherence; history of medical noncompliance.
Conditioning toxicity risk: providers must document anticipated ability to tolerate conditioning regimen and provide social work evaluation supporting post‑transplant support and adherence.

Denial Risk

Not Covered Diagnoses

Not covered diagnoses and indications will be denied. When an ICD‑10 code or clinical indication appears in the policy's Not Covered or Experimental lists, the transplant request is not a covered benefit unless an exception is approved per policy.

Umbilical cord blood stem cell transplant (UCBSCT) is covered only when patient meets allogeneic transplant eligibility; UCBSCT is not covered if patient selection criteria for allogeneic transplant are not met.
Examples of not covered / experimental indications include (see Experimental list): autologous SCT for Crohn's disease, stem cell therapy for erectile dysfunction, certain cardiac indications, AMD, ALS, type 1 diabetes, and hematologic conditions listed as not covered.
ICD‑10: Refer to the policy's Not Covered ICD‑10 list; claims submitted with listed codes will be subject to noncoverage determination.

Documentation Required

Authorization and Documentation Expectations

Documentation requirements and second‑opinion pathway. An approved transplant authorization includes pre‑transplant evaluation documentation; one transplant evaluation is covered per transplant. A second opinion transplant evaluation to determine candidacy may be approved at a contracted or in‑network transplant facility if the member was previously turned down for transplant.

Authorization includes coverage for one pre‑transplant evaluation; second transplant evaluation for candidacy requires prior approval and is limited to contracted/in‑network transplant facilities.
Required documentation: organ function testing, psychiatric/social work evaluation, evidence of adherence support, detailed treatment history, and any specialty testing (e.g., HLA typing, chimerism, stem cell counts) as requested.

Applies to autologous stem-cell transplantation for treatment of high-risk neuroblastoma

Allogeneic pathwayAllogeneic SCT from an appropriately matched HLA donor is covered if individual is not a candidate for autologous HSCT

inv-41: Duration of observed benefit in studies (retinitis pigmentosa MSC studies)

Observed duration of benefitMost MSC studies in retinitis pigmentosa reported sustained improvements up to 12 months

Evidence baseSystematic review included 11 studies and 355 RP patients reporting short-term BCVA and retinal function improvements

LimitationsLong-term efficacy beyond 12 months is less conclusive; further large-scale trials needed

Documentation Required

Pre‑transplant evaluation covered — social work required

One pre‑transplant evaluation per transplant is covered; documentation should include social work evaluation to document support network and psychosocial readiness.

Document social support and psychosocial stability in the pre‑transplant evaluation.
Evaluation coverage is limited to one per transplant.

Documentation Required

Pre‑transplant evaluation for MS — EDSS, MRI, DMT history, ambulatory status, age

Pre‑transplant evaluation for MS candidates should document neurologic baseline (EDSS), MRI disease activity, prior DMT history and failure, ambulatory status, age, and disease duration to support AHSCT candidacy.

Record baseline EDSS and ambulatory capability.
Provide MRI evidence of inflammatory activity and prior DMT exposure/failure; document age and disease duration.

Documentation Required

Pre‑ and post‑transplant lab and HLA testing referenced

Pre‑ and post‑transplant laboratory testing and HLA testing (HLA typing, crossmatch, chimerism analysis, CD34/stem cell counts) are referenced as expected parts of the transplant workup and monitoring.

Include HLA typing and crossmatch results in authorization and claims documentation.
Provide chimerism analysis and CD34/stem cell count testing records when applicable.

Provide documentation of center selection criteria and matching processes when requested.

Accreditation and program expertise (eg, EBMT/JACIE standards) are implied for centers performing AHSCT.

Billing Rule

Approved facility and referral direction for transplants

Transplants must be performed at a Priority Health approved facility; referrals are directed to network or contracted facilities and an approved transplant includes follow‑up care covered for one year at the transplant facility.

Follow Priority Health referral and facility approval requirements.
One year of follow‑up care at the transplant facility is included with an approved transplant.

Documentation Required

Accreditation and program standards (EBMT/JACIE)

Guidelines and referenced position statements (EBMT/JACIE) imply that centers should meet accreditation and specialized program standards to perform HSCT, particularly for autoimmune disease indications.

Center accreditation and specialized program standards are expected for HSCT programs.
Provide center accreditation information if requested during review.

ScopeApplies to services beyond the initial 1-year post-transplant follow-up period

Facility requirementExtended coverage beyond one year limited to contracted transplant facilities for full range of services

inv-104: second myeloablative allogeneic HSCT — covered for ALL when relapse >6 months after first SCT

Coverage conditionA second myeloablative allogeneic HSCT is covered for ALL when relapse occurs more than six months after the first allogeneic SCT

Donor requirementSecond myeloablative allogeneic HSCT should be from an appropriately-matched HLA donor

ScopeApplies specifically to ALL (acute lymphoblastic leukemia) per policy statements

inv-105: Inpatient post-transplant monitoring and complication management

Inpatient monitoring necessityPost-transplant care must include monitoring and management of short-term complications (infections, organ toxicities) given high rates of serious adverse events reported in trials

Typical complicationsReported serious adverse events include renal failure from thrombotic microangiopathy, pulmonary veno-occlusive disease, severe infections, and treatment-related mortality

Documentation expectationFacility should document capability to manage acute transplant complications as part of candidate selection and authorization

inv-106: Outcomes monitoring and complication surveillance — survival statistics and transplant-related mortality

Key outcomes to monitorPost-transplant monitoring should include survival, event-free survival, relapse-free and MRI-activity–free survival metrics (policy cites pooled overall survival ~94% and 4% transplant-related mortality after AHSCT in MS studies)

Use of registry/aggregate dataPolicy references pooled estimates from systematic reviews/meta-analyses to inform expected outcomes and transplant-related mortality rates

Surveillance focusOngoing surveillance for new disease activity, graft failure, and transplant-related complications is recommended

inv-107: post-transplant aftercare/status coding

Aftercare encounter codeZ48.290 — Encounter for aftercare following bone marrow transplant

Transplant status codesZ94.81 — Bone marrow transplant status; Z94.89 — Stem cells transplant status

PurposeThese codes are referenced for billing and tracking post-transplant services and status

This EDSS range reflects ambulatory patients with sufficient disability to consider AHSCT but not severely disabled patients unlikely to tolerate procedure

DocumentationPre-transplant evaluation should document neurologic baseline EDSS as part of authorization for AHSCT

inv-70: Tandem Transplantation definition

DefinitionTandem Transplantation: two or more planned courses of high-dose chemotherapy and stem cell support, administered typically at intervals of two to six months

DistinctionMultiple cycles with longer intervals differ from tandem transplants which are planned sequential procedures

Typical useTandem transplants may be covered for specific conditions when explicitly listed in policy (eg, multiple myeloma, neuroblastoma) with stated limits

inv-71: Partial remission definition

DefinitionPartial remission: at least a 50% reduction in tumor burden

ContextUsed to define response categories relevant to transplant eligibility and coverage decisions

Related termsResponsive includes complete or partial remission as defined in policy

inv-72: Relapse definition

DefinitionRelapse: tumor recurrence after a prior complete remission

Policy relevanceRelapse timing (eg >6 months after first SCT) is used to determine coverage for repeat transplants in certain indications

ExampleA second myeloablative allogeneic HSCT is covered for ALL when relapse occurs >6 months after first SCT

inv-73: Refractory disease definition

DefinitionRefractory disease: failure to attain a complete or partial response (primary or secondary) to therapy

Clinical implicationRefractory disease documentation is required for consideration of HSCT in many indications

ExamplesUsed to determine investigational vs covered status for indications such as Crohn's disease and certain malignancies

inv-74: Crohn's disease (CD) definition

DefinitionCrohn's disease (CD): chronic inflammatory bowel disease affecting any part of the digestive tract with transmural granulocytic infiltrates causing diarrhea, abdominal pain, rectal bleeding and weight loss

Policy noteAutologous SCT for Crohn's disease is considered experimental and not covered despite some evidence of benefit

Evidence expectationCandidate selection requires documented failure of conventional medical and surgical therapies

inv-75: Multiple sclerosis (MS) definition

DefinitionMultiple sclerosis (MS): chronic inflammatory and neurodegenerative CNS disease with demyelination and variable clinical courses (RRMS, SPMS, PPMS)

Clinical descriptorsDisease activity is evidenced by clinical relapses or MRI lesions; disability progression reflects increasing neurological dysfunction

Policy relevanceAHSCT is recommended for selected inflammatory MS phenotypes per EBMT guidance and is covered when criteria are met

inv-76: Autologous hematopoietic stem cell transplantation (aHSCT) definition

DefinitionAutologous hematopoietic stem cell transplantation (aHSCT): procedure involving ablation of the patient's immune system and reconstitution with the patient’s own harvested hematopoietic stem cells

Clinical intentUsed to induce remission in severe autoimmune diseases by resetting the immune system

Safety noteTrials report substantial serious adverse events prompting careful candidate selection and center expertise

inv-77: AHSCT description and recommended phenotypes

DescriptionAHSCT involves ablation of the patient's aberrant immune system followed by infusion of autologous hematopoietic stem cells to reconstitute a new immune system; recommended for selected inflammatory MS phenotypes

Recommended phenotypesPrimarily recommended for relapsing forms of MS with inflammatory activity and for select SPMS with active inflammation

Candidate profileOptimal candidates are younger, ambulatory patients with disease duration <10 years and failure of high-efficacy DMTs

inv-78: Nonmyeloablative HSCT definition and risks

DefinitionNonmyeloablative HSCT: a conditioning strategy with reduced-intensity chemotherapy prior to stem cell reinfusion

RisksAssociated with significant conditioning-related risks including marrow aplasia and sepsis; safety concerns may limit use in some indications (eg, type 1 diabetes)

Use casesMay be considered for patients who meet selection criteria but are not candidates for myeloablative regimens due to comorbidities or age

inv-79: Thrombotic thrombocytopenic purpura (TTP) definition

DefinitionThrombotic thrombocytopenic purpura (TTP): rare thrombotic microangiopathy caused by ADAMTS13 deficiency (congenital or acquired) associated with high untreated mortality and organ ischemic complications

Clinical urgencyAcute episodes are medical emergencies with high mortality if untreated

Policy noteListed among autoimmune and hematologic conditions in the policy; SCT for some related indications may be investigational

inv-80: Retinitis pigmentosa (RP) definition

DefinitionRetinitis pigmentosa (RP): group of hereditary retinal dystrophies with progressive photoreceptor degeneration causing night blindness, visual field constriction, and decreased acuity

Therapeutic contextMesenchymal stem cell therapies have shown short-term improvements in visual function up to 12 months in systematic reviews

Evidence gapLong-term benefit beyond 12 months remains less conclusive and requires larger trials

inv-81: Reference list for transplant indications (references cited)

References includedPolicy cites references and guidelines for neuroblastoma, HSCT indications, and systematic reviews (see reference list)

ExamplesIncludes COG neuroblastoma schema, UpToDate reviews, EBMT position statements, and multiple systematic reviews/meta-analyses

PurposeReferences support indication-specific coverage statements, candidate selection, and evidence summaries within the policy