CurrentAlliance HealthPolicy Clinical Coverage Policy No: 11A-10

Hematopoietic Stem-Cell Transplantation (HSCT) for Central Nervous System (CNS) Embryonal Tumors & Ependymoma

NC Medicaid clinical coverage policy defining when HSCT (autologous and tandem autologous) is covered or not covered for central nervous system embryonal tumors and ependymoma, provider billing requirements, and claims attachments. Applies to Medicaid beneficiaries; EPSDT exceptions for <21 years noted.

Policy Summary

PayerAlliance Health

PolicyHematopoietic Stem-Cell Transplantation (HSCT) for Central Nervous System (CNS) Embryonal Tumors & Ependymoma

Policy CodePolicy Clinical Coverage Policy No: 11A-10

Change TypeRevised (template update, prior approval removed, coverage additions/exclusions)

Effective DateJan 1, 1994

Next Review Date

Key ActionProviders must document how the requested HSCT meets medical necessity and, for beneficiaries under 21, how EPSDT criteria are satisfied when requesting services that exceed policy limitations.

SourceLink

POLICY UPDATE CHANGES

Policy posted 8/15/2023 with an effective date of 4/1/2023 and template language updated due to NC Health Choice move to Medicaid.

Prior approval requirement removed effective 07/01/2021.

Added coverage for tandem autologous HSCT for high-risk CNS embryonal tumors (11/01/2018).

Added tandem autologous HSCT for average-risk CNS embryonal tumors to non-coverage (11/01/2018).

3Covered Indications (single autologous, recurrent, tandem for high-risk)

4Specific Not Covered Situations

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Prior Approval Required

Coverage Summary

This NC Medicaid coverage policy (Clinical Coverage Policy No: 11A-10) addresses Hematopoietic Stem-Cell Transplantation (HSCT) for Central Nervous System (CNS) embryonal tumors and ependymoma. Effective date: 1994-01-01; last review/amended: 2023-08-15. Coverage stance: mixed — single autologous HSCT and tandem autologous HSCT for specified indications are covered while several HSCT applications are explicitly not covered. For beneficiaries under age 21, the EPSDT provision may allow exceptions to policy limitations when provider documentation shows the service is medically necessary to correct or ameliorate a health problem.

Policy facts

Policy numberClinical Coverage Policy No: 11A-10

SubjectHematopoietic Stem-Cell Transplantation (HSCT) for CNS Embryonal Tumors & Ependymoma

Effective date1994-01-01

Last review / Amended dateAmended Date: August 15, 2023 (last_review 2023-08-15)

StatusCURRENT

Medical-Necessity Criteria

General Coverage Criteria

Medicaid shall cover the procedure, product, or service related to this policy when medically necessary, and:

ALL of the following

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
The procedure, product, or service can be safely furnished, and no equally effective and more conservative or less costly treatment is available statewide
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

Specific Criteria Covered

Medicaid shall cover single autologous HSCT or tandem autologous HSCT when determined medically necessary as:

Not Covered / Exclusions

General Not Covered Criteria

Medicaid shall not cover when any of the following apply:

ANY of the following

Beneficiary does not meet the eligibility requirements listed in Section 2.0
Beneficiary does not meet the criteria listed in Section 3.0
The procedure, product, or service duplicates another provider's procedure, product, or service
The procedure, product, or service is experimental, investigational, or part of a clinical trial

Specific Not Covered Criteria

Provider Actions

Documentation Required

Medical necessity documentation

Providers must document how the requested HSCT meets medical necessity. For beneficiaries under age 21, provider documentation must also show how EPSDT criteria are satisfied when requesting services that exceed policy limitations.

EPSDT documentation showing medical necessity to correct or ameliorate a health problem for beneficiaries under 21 (see EPSDT Special Provision)

Prior Authorization

Prior approval not required

No prior approval is required for Hematopoietic Stem-Cell Transplantation (HSCT) for Central Nervous System (CNS) embryonal tumors and ependymoma.

Coding

Claim Type / Billing Formsmixed

CMS-1500/837P	Professional claim form/transaction
UB-04/83711	Institutional claim form/transaction; institutional claims must follow National Uniform Billing Guidelines

Coding Guidancemixed

ICD-10-CM/PCS	Report to the highest level of specificity using the current ICD-10-CM and PCS edition in effect at the time of service; specific codes are not listed in this policy
CPT/HCPCS	Report the most specific CPT or HCPCS code in effect at the time of service; if no specific code exists use appropriate unlisted procedure/service codes and follow CPT/HCPCS special report instructions

Background

Hematopoietic stem-cell transplantation (HSCT) refers to infusion of hematopoietic stem cells to restore bone marrow after bone-marrow–toxic doses of cytotoxic drugs and can use autologous, allogeneic, syngeneic, or umbilical cord blood sources harvested from marrow, peripheral blood, or cord blood.

For brain tumors, autologous HSCT is used to permit escalation of chemotherapy doses above marrow tolerance (rescue transplant) and has been tried in patients with high-risk brain tumors to eradicate residual disease and improve cure rates; tandem autologous HSCT is a preplanned sequence of transplants and may be used for high-risk embryonal tumors. Allogeneic HSCT is uncommonly used for solid tumors; its theoretical rationale is a graft-versus-tumor effect and it may be considered only if an autologous graft cannot be cleared of tumor or cannot be harvested.

CNS embryonal tumors are a heterogeneous group (including medulloblastoma and related entities such as ETMR, AT/RT, pineoblastoma, and others) that tend to disseminate via CSF pathways and are increasingly subclassified by molecular genetics for risk stratification and treatment planning. Medulloblastoma treatment centers on maximal surgical resection followed by craniospinal radiotherapy and risk-adapted chemotherapy; average-risk and high-risk groups have distinct prognoses and treatment regimens, and in children under three years alternative strategies (including high-dose chemotherapy with autologous HSCT) have been explored to delay or avoid radiotherapy.

Recurrent CNS embryonal tumors may be treated with high-dose chemotherapy and autologous HSCT as salvage therapy; objective response rates of 50%–75% have been reported but long-term disease control is seen in fewer than 30% of patients, primarily those in first relapse with localized disease.

Ependymoma arises from ependymal cells, is usually managed initially with maximal surgical resection followed by radiotherapy, and generally shows poor response to conventional-dose chemotherapy. Because relapse is common and further surgery or radiation may not be feasible, high-dose chemotherapy with autologous HSCT has been investigated as a possible salvage option, but HSCT for ependymoma is specifically addressed in coverage exclusions.

Revision History

1994-01-01revised

Original effective date established (policy effective January 1, 1994).

07/01/2005revised

Policy updated to include coverage criteria effective with approved State Plan amendment; template and coverage updates across the policy.

10/01/2015revised

All sections and attachments revised; updated policy template language and added ICD-10 codes to comply with federally mandated implementation.

Policy Summary

PayerAlliance Health

PolicyHematopoietic Stem-Cell Transplantation (HSCT) for Central Nervous System (CNS) Embryonal Tumors & Ependymoma

Policy CodePolicy Clinical Coverage Policy No: 11A-10

Change TypeRevised (template update, prior approval removed, coverage additions/exclusions)

Effective DateJan 1, 1994

Next Review Date

SourceLink

On This Page

Covered HSCT indications

Single autologous HSCT as consolidation therapy for previously untreated embryonal tumors of the CNS that show partial or complete response to induction chemotherapy, or stable disease after induction therapy
Single autologous HSCT as treatment for recurrent CNS embryonal tumors
Tandem autologous HSCT as treatment for high-risk embryonal tumors of the CNS

Medicaid shall not cover HSCT for ANY of the following specific situations:

ALL of the following

Allogeneic HSCT to treat embryonal tumors of the CNS
Tandem autologous HSCT for the treatment of average-risk embryonal tumors of the CNS
Autologous, tandem autologous and allogeneic HSCT to treat ependymoma
Autologous, tandem autologous and allogeneic HSCT to treat other CNS tumors (e.g., astrocytoma, oligodendroglioma, glioblastoma multiforme)

Billing Rule

Claim form and coding adherence

Providers shall submit professional claims on CMS-1500/837P or institutional claims on UB-04/83711. Institutional claims must follow the National Uniform Billing Guidelines. All claims must comply with National Coding Guidelines and providers must report the most specific billing codes in effect at the time of service.

CMS-1500/837P (Professional claim)
UB-04/83711 (Institutional claim)

Billing Rule

Place of service

Place of service for HSCT is inpatient hospital or outpatient hospital, as applicable.

Documentation Required

Provider eligibility to bill

To bill for HSCT, providers must meet Medicaid qualifications for participation, have a current signed DHHS Provider Administrative Participation Agreement, and bill only for procedures, products, and services within their licensed scope of clinical practice.

Billing Rule

Unlisted procedure reporting

If no specific CPT or HCPCS code exists for the procedure, providers shall report the service using the appropriate unlisted procedure or service code and comply with the CPT/HCPCS instructions for unlisted procedures and special report requirements in effect at the time of service.

Definitions (core terms)

HSCTHematopoietic stem-cell transplantation from autologous, allogeneic, syngeneic, or umbilical cord blood sources.

Induction TherapyThe first treatment given for a disease (first-line therapy), often surgery, chemotherapy, and/or radiation.

Consolidation TherapyTreatment given after initial therapy to kill remaining cancer cells; may include stem cell transplant.

Rescue TransplantReplacing blood-forming stem cells destroyed by high-dose anticancer treatment to help bone marrow recover (stem cell rescue).

Salvage TherapyTreatment given after the cancer has not responded to other treatments.

Tandem TransplantsPreplanned sequential hematopoietic stem cell transplants.

11/01/2018revised

Multiple substantive revisions: policy renamed to HSCT for CNS Embryonal Tumors & Ependymoma; Section 1.0 updated with WHO classifications; definitions added; coverage expanded to add tandem autologous HSCT for high-risk CNS embryonal tumors; tandem autologous HSCT for average-risk added to non-coverage; other edits to attachments and requirements.

07/01/2021revised

Section 5.0 revised and prior approval requirement removed (no prior approval required for HSCT for CNS embryonal tumors & ependymoma).

2023-04-01revised

Policy posted with an effective date of April 1, 2023 as part of updates due to NC Health Choice move to Medicaid.

2023-08-15revisedLatest

Amended August 15, 2023: Section revisions across all sections and attachments; updated policy template language due to North Carolina Health Choice Program's move to Medicaid (policy posted 8/15/2023).