CurrentAlliance HealthPolicy 11A-5

Allogeneic Hematopoietic Stem Cell Transplantation for Genetic Diseases and Acquired Anemias

State Medicaid clinical coverage policy governing when allogeneic hematopoietic stem cell transplantation (HSCT) for genetic diseases and acquired anemias is covered for North Carolina Medicaid beneficiaries, including EPSDT considerations for beneficiaries under 21.

Policy Summary

PayerAlliance Health

PolicyAllogeneic Hematopoietic Stem Cell Transplantation for Genetic Diseases and Acquired Anemias

Policy CodePolicy 11A-5

Change TypePrior approval removed

Effective Date08/15/2023

Next Review Date

Key ActionObtain and retain documentation showing medical necessity and beneficiary ability to comply with pre- and post-transplant care; verify Medicaid eligibility before billing.

SourceLink

POLICY UPDATE CHANGES

Prior approval requirement removed.

Removed all CPT, HCPCS, and ICD-10 codes from attachments.

UB-04 accepted as an institutional claims form.

Updated ICD-10 codes.

NC Medicaidapplicable program

EPSDTEPSDT applies for <21

Not requiredprior approval

07/01/2021Date prior approval removed

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Aug 15, 2023amended date

Coverage Criteria

General Criteria Covered

Medicaid shall cover the procedure, product, or service when medically necessary and when ALL of the following are met:

General coverage conditions: a. the procedure, product, or service is individualized, specific, and consistent with symptoms or confirmed diagnosis of the illness or injury under treatment, and not in excess of the beneficiary's needs; b. the procedure, product, or service can be safely furnished, and no equally effective and more conservative or less costly treatment is available statewide; c. the procedure, product, or service is furnished in a manner not primarily intended for the convenience of the beneficiary, the beneficiary's caretaker, or the provider.

From Subsection 3.1

Covered Indications (Initial therapy)

Covered when ALL of the following general medical necessity requirements AND one of the specific disorder indications are met:

General medical necessity: (a) individualized, specific, consistent with symptoms or confirmed diagnosis and not in excess of beneficiary's needs; (b) can be safely furnished and no equally effective/conservative/less costly treatment available statewide; (c) furnished not primarily for convenience of beneficiary, caretaker, or provider.

from Subsection 3.1

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Covered Indications (Initial therapy)

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Medicaid covers the service only when it is medically necessary and when all general criteria are satisfied. Specifically, the procedure must be individualized, specific, and consistent with the beneficiary's symptoms or confirmed diagnosis, not be in excess of the beneficiary's needs, and be able to be safely furnished. Coverage also requires that no equally effective, more conservative, or less costly treatment be available statewide and that the service not be provided primarily for the convenience of the beneficiary, caretaker, or provider.

Allogeneic HSCT is not covered for diagnoses other than those listed in Subsection 3.2. Coverage is also denied when the beneficiary's psychosocial history or current beneficiary/caretaker non-compliance would make adherence to required pre- and post-transplant medical care improbable, since the ability to comply with the disciplined peri‑transplant regimen is essential for a successful outcome.

Medicaid will not reimburse expenses related to unsuccessful donor searches. Only actual donor transplant‑related medical expenses billed on the beneficiary's transplant claim using the beneficiary's Medicaid ID are eligible for reimbursement.

Services are considered not medically necessary when they are not individualized, not consistent with the beneficiary's diagnosis, or are excessive relative to the beneficiary's needs. Coverage also requires that services be safe and that no equally effective, more conservative, or less costly statewide alternative exists; failure of these conditions supports denial as not medically necessary.

The procedure is not medically necessary (and may be denied) when the beneficiary fails the program eligibility requirements in Section 2.0, does not meet the specific coverage criteria in Section 3.0, when the service duplicates another provider's service, or when it is experimental or part of a clinical trial. Additionally, procedures outside the diagnoses listed in Subsection 3.2 or those where psychosocial factors or non‑compliance make required care improbable are not covered.

Candidate Clinical Indications and Criteria

Candidate clinical indications

Clinical indications described in this portion of the policy include inherited hemoglobinopathies, bone marrow failure syndromes, primary immunodeficiencies, inherited metabolic diseases, and infantile malignant osteopetrosis.

Indications for consideration: Allogeneic HSCT may be indicated for: (1) thalassemia major and sickle cell disease as definitive therapy or cure; (2) bone marrow failure syndromes including Fanconi anemia, Shwachman-Diamond, Diamond-Blackfan; (3) primary immunodeficiencies including SCID, Wiskott-Aldrich; (4) inherited metabolic diseases such as lysosomal and peroxisomal storage disorders; (5) infantile malignant osteopetrosis where HSCT is curative.

Clinical indications described in policy text

Candidate criteria

Candidates must have one of the specified disorders; additional general medical necessity requirements apply.

Candidate disorders: Sickle cell anemia (children/young adults with prior stroke or increased risk of stroke or end-organ damage); homozygous beta-thalassemia; aplastic anemia (hereditary or acquired); primary immunodeficiencies with absent/defective T-cell, NK, or neutrophil function; lysosomal and peroxisomal storage disorders except Hunter, Sanfilippo, Morquio; infantile malignant osteopetrosis.

Contraindications

No contraindications are specified in this excerpt of the policy.

A contraindication to coverage exists when the beneficiary's psychosocial history or current beneficiary/caretaker non‑compliance would make adherence to pre‑ and post‑transplant care improbable. In such cases, Medicaid will not cover allogeneic HSCT because the required disciplined medical regimen cannot reasonably be followed.

Evaluation and Pre-transplant Requirements

Denial Risk

HLA Compatibility and Pre-transplant Evaluation

Immunologic compatibility established by HLA typing is critical prior to allogeneic HSCT. Providers must document HLA typing results and donor–recipient match level in the medical record. Lack of documented HLA compatibility or missing/unsupported HLA testing may result in denial of coverage.

Document HLA typing and match level in pre-transplant evaluation.
Accepted methods: cellular, serologic, or molecular HLA typing.
Umbilical cord blood may have less stringent HLA matching requirements; document rationale.

Documentation Required

Preparative Conditioning Documentation

Selection of a preparative conditioning regimen (myeloablative vs reduced-intensity conditioning) must be supported in the chart with the clinical indication and rationale. Missing documentation of regimen selection rationale, regimen details (drugs, doses, radiation), or intent (myeloablative vs RIC) may lead to claim denial.

Provider Actions, Documentation, and Billing Rules

Prior Authorization

Obtain prior approval when required

If a service requires prior approval, obtain prior approval before providing the service; EPSDT status for beneficiaries under 21 does not remove prior approval requirements when they apply.

Check whether prior approval is required for the specific service and secure approval per NCTracks instructions.

Prior Authorization

Prior approval not required for this HSCT policy

No prior approval is required for Allogeneic Hematopoietic Stem Cell Transplantation for Genetic Diseases and Acquired Anemias under this Medicaid policy.

Providers may bill for HSCT under this policy without obtaining transplant‑specific prior approval.

Prior Authorization

Coding and Claiming

Claims-related sections and coding attachments presentmixed

No codes listed

CPT/HCPCS/ICD-10 codesmixed

No codes listed

Coding guidancemixed

No codes listed

EPSDT age threshold

EPSDT age thresholdEPSDT special provision applies for beneficiaries 21 years of age or younger (<= 21 years).

Statutory basisCites 42 U.S.C. § 1396d(r) (1905(r) of the Social Security Act) as the EPSDT authority.

Documentation requirementFor beneficiaries under 21, provider documentation must show the service is medically necessary to correct or ameliorate a health problem to qualify under EPSDT.

Coding specificity

Coding specificity requirementReport ICD-10-CM and PCS to the highest level of specificity that supports medical necessity.

Edition guidance

Provider and Center Requirements

Prior Authorization

Verify beneficiary eligibility at each encounter

Verify each Medicaid beneficiary's eligibility every time a service is rendered and follow any applicable prior approval processes as directed.

Confirm eligibility before scheduling or delivering HSCT-related services.
Follow any specified prior approval processes when they apply despite general policy removal.

Billing Rule

Maintain provider qualifications and use correct claim forms

Providers must meet Medicaid participation qualifications, maintain a signed DHHS Provider Administrative Participation Agreement, and bill using the correct claim forms within the scope of their clinical practice.

Ensure enrollment and agreements are current and bill on UB‑04 or CMS‑1500 as appropriate.

Post-Transplant Coverage and Donor Billing

Post-transplant services referenced in attachments

Attachments presentClaims-related information and coding attachments are provided in Attachment A (codes, modifiers, billing units, place of service, donor expense billing).

Claim types referencedAttachment A lists claim types and requirements for professional (CMS-1500) and institutional (UB-04) claims.

Reference to billing guidanceProviders must comply with the NCTracks Provider Claims and Billing Assistance Guide and Medicaid bulletins referenced in attachments.

Post-transplant care (implicit requirement for coverage)

Coverage conditional on complianceCoverage requires the beneficiary's ability to comply with required pre- and post-transplant medical care; non-compliance can make the procedure not covered.

Definitions

Allogeneic HSCT

DefinitionAllogeneic HSCT: infusion of hematopoietic progenitor cells obtained from a donor to restore bone marrow function.

Cell sourcesDonor cells may be harvested from bone marrow, peripheral blood, or umbilical cord blood (and placenta shortly after delivery).

Compatibility requirementImmunologic compatibility established by HLA typing is a critical factor for achieving a good outcome.

Reduced-intensity conditioning (RIC)

DefinitionReduced-intensity conditioning (RIC) refers to chemotherapy regimens that partially eradicate the patient's hematopoietic ability to permit donor engraftment while reducing myelotoxic adverse effects compared with full myeloablation.

Clinical effect

Background

Allogeneic hematopoietic stem‑cell transplantation (HSCT) uses hematopoietic progenitor cells obtained from a donor to restore bone marrow function and can be curative for several inherited and acquired hematologic disorders. Preparative conditioning regimens range from myeloablative to reduced‑intensity conditioning (RIC), with the choice balancing toxicity and the need for donor engraftment; immunologic compatibility (HLA typing) and appropriate donor selection are critical components of the evaluation.

Policy Summary

PayerAlliance Health

PolicyAllogeneic Hematopoietic Stem Cell Transplantation for Genetic Diseases and Acquired Anemias

Policy CodePolicy 11A-5

Change TypePrior approval removed

Effective Date08/15/2023

Next Review Date

Key ActionObtain and retain documentation showing medical necessity and beneficiary ability to comply with pre- and post-transplant care; verify Medicaid eligibility before billing.

SourceLink

On This Page

Include regimen name, agents, doses, dates, and intent (myeloablative or reduced-intensity) in pre-transplant notes.
Document expected impact on engraftment and rationale tied to patient comorbidities and disease status.
If RIC is used, document expected chimerism plan and follow-up monitoring.

Prior Authorization

Claims, Billing, and Compliance Requirements

Providers must comply with NC Medicaid clinical coverage policies, CMS/DHHS guidance, NCTracks claims and billing guidance, and other applicable laws and contractual requirements at the time of service. Failure to follow applicable coverage policies, billing rules, prior authorization procedures, or to submit required forms and supporting documentation may result in denial or recoupment.

Follow NC Medicaid clinical coverage policies and CMS/DHHS guidance effective on service date.
Adhere to NCTracks Provider Claims and Billing Assistance Guide, Medicaid bulletins, and fee schedules.
Obtain and document prior authorization when required by payer-specific rules.
Attach required forms and supporting medical records (HLA typing, transplant summary, conditioning regimen details, consent) to the claim as instructed.
General denial triggers: missing prior authorization, missing medical records, noncompliance with coverage policy criteria.
Specific denial triggers: undocumented HLA compatibility, undocumented conditioning regimen details, services not meeting NC Medicaid coverage criteria, billing with incorrect codes or modifiers.

Prior approval requirement removed (effective 07/01/2021)

The prior approval requirement for this policy was removed effective 07/01/2021; prior authorization is not required per this revision.

No transplant prior authorization is required starting 07/01/2021 per policy revision history.

Step Therapy

Ensure no equally effective conservative alternative exists

Coverage is contingent on the service being individualized, medically necessary, and that no equally effective, more conservative, or less costly statewide treatment is available.

Document why less conservative or less costly alternatives are not appropriate for the beneficiary.

Documentation Required

Document EPSDT medical necessity for beneficiaries <21

For beneficiaries under 21, provider documentation must demonstrate the requested service is medically necessary to correct or ameliorate a health problem to qualify under EPSDT.

Include documentation showing how the service corrects, improves, or prevents worsening of the beneficiary's health condition.

Documentation Required

Maintain enrollment, agreements, and compliance documentation

Providers must meet Medicaid qualifications for participation, have a signed DHHS Provider Administrative Participation Agreement, bill only within the scope of their clinical practice, and comply with applicable agreements, laws (including HIPAA), record retention, and NC Medicaid clinical policies.

Maintain a current signed DHHS Provider Administrative Participation Agreement.
Retain medical records and comply with HIPAA and record retention requirements.

Billing Rule

Retain and submit claims per UB‑04/CMS‑1500 and NCTracks guidance

Providers must retain required claims documentation per National Uniform Billing Guidelines and NCTracks guidance; institutional claims must be submitted on UB‑04 and professional claims on CMS‑1500 as applicable.

Use UB‑04 for institutional claims and CMS‑1500 for professional claims unless otherwise directed.
Ensure claims comply with National Coding Guidelines and NCTracks instructions.

Denial Risk

Risk of denial if service is not individualized or excessive

Services that are not individualized, not consistent with the beneficiary's symptoms/diagnosis, excessive to the beneficiary's needs, unsafe, or where an equally effective and more conservative or less costly treatment exists statewide may be denied.

Document individualized medical necessity and rationale when higher‑risk or higher‑cost services are provided.

Denial Risk

Verify eligibility and criteria to avoid denial

A claim may be denied if the beneficiary does not meet eligibility in Section 2.0, does not meet the policy criteria in Section 3.0, or if the service duplicates another provider's service.

Verify beneficiary eligibility at each service and confirm criteria in Section 3.0 are met prior to providing HSCT.

Denial Risk

Denial risk for unlisted diagnoses or psychosocial/non‑compliance

Transplantation may be denied when the diagnosis is not among those listed in Subsection 3.2 or when psychosocial history or beneficiary/caretaker non‑compliance would make adherence to pre‑ and post‑transplant care unlikely.

Assess and document psychosocial readiness and likelihood of adherence to pre‑ and post‑transplant regimens.

Billing Rule

Use highest‑specificity ICD‑10‑CM and PCS codes

Report ICD‑10‑CM and PCS codes to the highest level of specificity that supports medical necessity; failure to do so may result in claim issues or denial.

Use the current ICD‑10 edition in effect at time of service and the most specific codes available.
Refer to CPT instructions for unlisted procedures as needed.

Use the current ICD-10 edition and any subsequent editions in effect at the time of service.

Code descriptionsProviders shall refer to the applicable ICD-10 edition for code descriptions; code descriptions are not documented in the policy.

Billing Rule

Follow NCTracks, bulletins, and fee schedules for coverage/reimbursement

Providers must comply with NCTracks guidance, Medicaid bulletins, fee schedules, and NC Medicaid clinical coverage policies for specific coverage and reimbursement rules.

Regularly review NCTracks and Medicaid updates to ensure billing and coverage requirements are met.

Psychosocial assessment relevance

A beneficiary's psychosocial history that limits ability to comply with pre- and post-transplant care is a basis for non-coverage.

General denial linkServices may be denied if the beneficiary does not meet eligibility or criteria in Sections 2.0 and 3.0, including inability to adhere to required care.

Donor-related medical services

Billing on beneficiary claimDonor transplant-related medical expenses are billed on the Medicaid beneficiary's transplant claim using the beneficiary's Medicaid ID.

Reimbursable donor expensesMedicaid reimburses only for the actual donor's transplant-related medical expenses.

Unreimbursable itemsMedicaid does not reimburse for unsuccessful donor searches.

RIC patients initially demonstrate donor cell engraftment and mixed chimerism, with many converting to full-donor chimerism.

Regimen spectrumRIC regimens exist on a continuum from nearly totally myeloablative to minimally myeloablative with lymphoablation.

Hemoglobinopathies

DefinitionHemoglobinopathies include sickle cell anemia (children or young adults with prior stroke or at increased risk of stroke or end-organ damage) and homozygous beta-thalassemia (thalassemia major).

Sickle cell specificsSickle cell anemia is covered for children/young adults with history of prior stroke or increased stroke/end-organ damage risk.

ThalassemiaHomozygous beta-thalassemia (thalassemia major) is listed as a covered indication.

Bone marrow failure syndromes

DefinitionBone marrow failure syndromes include aplastic anemia (hereditary forms such as Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond, Diamond-Blackfan) and acquired forms (e.g., secondary to drug or toxin exposure).

Hereditary examplesHereditary examples explicitly named: Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond, Diamond-Blackfan.

Acquired formsAcquired aplastic anemia (secondary to drug or toxin exposure) is also included.

Primary immunodeficiencies

DefinitionPrimary immunodeficiencies include disorders with absent or defective T-cell, natural killer (NK), or neutrophil function (examples: severe combined immunodeficiency, Wiskott-Aldrich syndrome, X-linked lymphoproliferative syndrome, Chediak-Higashi, Kostmann syndrome, chronic granulomatous disease, leukocyte adhesion defect).

T-cell defects examplesExamples of absent/defective T-cell function include SCID and Wiskott-Aldrich syndrome.

Other immune cell defectsExamples of NK and neutrophil function defects are listed (e.g., Chediak-Higashi; Kostmann syndrome; chronic granulomatous disease).

Inherited metabolic disease

DefinitionInherited metabolic disease covers lysosomal and peroxisomal storage disorders, excluding Hunter, Sanfilippo, and Morquio syndromes.

InclusionsLysosomal and peroxisomal storage disorders are included as indications for HSCT when appropriate.

ExclusionsHunter, Sanfilippo, and Morquio syndromes are explicitly excluded from coverage under inherited metabolic disease criteria.

Genetic skeletal disorders

DefinitionGenetic skeletal disorders include conditions affecting skeletal tissue such as infantile malignant osteopetrosis (Albers-Schonberg disease or marble bone disease).

Specific exampleInfantile malignant osteopetrosis is listed as a covered genetic skeletal disorder indication for HSCT.

Therapeutic intentHSCT is indicated when it can be curative for these genetic skeletal disorders.

Donor expense billing

Billing practiceDonor transplant-related medical expenses must be billed on the beneficiary's transplant claim using the beneficiary's Medicaid identification number.

Reimbursement limitationMedicaid reimburses only actual donor transplant-related medical expenses; unrelated or excessive donor costs are not reimbursed.

Unsuccessful searchesMedicaid does not reimburse for unsuccessful donor searches.