LCD L39513: Allogeneic Hematopoietic Cell Transplantation for Primary Refractory or Relapsed Hodgkin and Non-Hodgkin Lymphoma with B-cell or T-cell Origin

Stem cell transplantation is a process in which s tem cells are harvested from either a patient’s (autologous) or donor’s (allogeneic) bone marrow or peripheral blood for intravenous infusion. Autologous s tem cell transplantation (AuSCT) is a technique for restoring stem cell s using the patient's own previously stored cells. AuSCT must be used to effect hematopoietic reconstitution following severely myelotoxic doses of chemotherapy (HDCT) and/or radiotherapy used to treat various malignancies.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a procedure in which a portion of a healthy donor's stem cells or bone marrow is obtained and prepared for intravenous infusion. Hematopoietic stem cells are multi-potent cells that give rise to all the blood cell types; these stem cells form blood and immune cells. A hematopoietic stem cell is a cell isolated from blood or bone marrow that can renew itself, differentiate to a variety of specialized cells, can mobilize out of the bone marrow into circulating blood, and can undergo programmed cell death, called apoptosis - a process by which cells that are unneeded or detrimental will self-destruct.

The Centers for Medicare & Medicaid Services (CMS) has clarified that bone marrow and peripheral blood stem cell transplantation is a process which includes mobilization, harvesting, and transplant of bone marrow or peripheral blood stem cells and the administration of high dose chemotherapy or radiotherapy prior to the actual transplant. When bone marrow or peripheral blood stem cell transplantation is covered, all necessary steps are included in coverage.

NCD 110.23 Stem Cell Transplantation covers allo-HSCT for the following conditions, when reasonable and necessary:

• Leukemia

• Aplastic Anemia

• Severe Combined Immunodeficiency disease (SCID)

• Wiskott-Aldrich Syndrome

Allo-HSCT is covered for Medicare beneficiaries with the following indications only when participating in approved prospective clinical studies meeting specific criteria under the Coverage with Evidence Development (CED) paradigm:

• Myelodysplastic Syndromes

• Multiple myeloma with Durie-Salmon Stage II or III disease, or International Staging System (ISS) Stage II or Stage III disease

• Myelofibrosis (MF) with Dynamic International Prognostic Scoring System (DIPSSplus) intermediate-2 or High primary or secondary disease; or

• Sickle cell disease (SCD) that is severe and symptomatic

(Please refer to CMS Internet-Only Manual, Pub. 100-03, Medicare National Coverage Determinations Manual , Chapter 1, Part 2, §110.23 Stem Cell Transplantation)

Per the NCD, all other indications for s tem c ell transplantation not otherwise noted as covered or non-covered remain at local Medicare Administrative Contractor (MAC) discretion.

This policy describes additional locally covered indications for allo-HSCT for primary refractory or relapsed Hodgkin and non-Hodgkin lymphomas with B-cell or T-cell origin that are medically necessary in patients for whom there are no other curative intent options.

Documentation to support the reasonable and necessary nature of allo-HSCTs must speak to the serious illness of the patient, the reasons why the patient is considered to have relapsed or refractory disease, relevant clinical contextual information such as age, frailty, performance status, cardiopulmonary function, any associated organ dysfunction that could impact complications or recovery, screening for infectious diseases that could impact transplant care, nutritional status and patient specific psychosocial and financial support structures. Risk assessment scoring, such as the European Society for Blood and Bone Marrow Transplantation (EBMT) or the HCT Comorbidity Index (HCT-CI), should be strongly considered and in the case of any nationally covered indications per NCD 110.23 would of course be required.

Allogeneic hematopoietic cell transplantation (allo-HCT has been increasingly used for a variety of hematologic neoplasms and non-malignant bone marrow disorders. Eligibility for allo-HCT varies amongst institutions and is usually based on a case-by-case basis dependent upon a risk-benefit assessment, and the needs and wishes of the patient. 7

Although historically allo-HCT was offered to patients who had exhausted all other treatment modalities, currently the decision to perform a transplant is dependent upon an assessment if the transplant will offer an outcome superior to other treatment options. 7

The CMS National Coverage Determination (NCD 110.23) for Stem Cell Transplantation describes nationally covered indications for SCT, the details of which will not be fully repeated within this policy. This policy describes additional locally covered indications for allogeneic stem cell for primary refractory or relapsed Hodgkin's and non-Hodgkin's lymphomas with B-cell or T-cell origin, for whom there are no other curative intent options, and are medically necessary.

Multiple other disorders are under investigation as part of clinical trials and are not covered unless the clinical trial meets the criteria of NCD 310.1 Routine Costs in Clinical Trials.