Scope: Clinical review criteria governing medical necessity, indications, contraindications, documentation, and coding for stem cell (blood and marrow) transplantation including autologous, allogeneic, cord blood, haploidentical, myeloablative and non-myeloablative (mini) transplants; includes MTAC technology assessment summaries and procedure code guidance (CPT/HCPCS set).

Coverage stance: Mixed — many hematologic and selected non-malignant indications are covered when the policy criteria are met, while multiple specific indications and non-myeloablative approaches are not supported by MTAC; stem cell storage (long-term) is not medically necessary unless the patient is scheduled for transplant and requires case-by-case clinical review.

Subject: Stem Cell Transplant / Bone Marrow Transplant — criteria address eligibility, general principles, disease-specific indications, contraindications, required documentation, and applicable procedure codes.

High-level policy position: Transplantation is considered medically necessary for listed indications when all applicable criteria are met and documentation submitted; conversely, several indications and non-myeloablative uses do not meet Kaiser Permanente MTAC and are not recommended. Stem cell storage is explicitly limited to patients scheduled to receive a transplant and may be denied if not meeting that condition.

Background: Stem cell transplantation (also called bone marrow transplant or cord blood transplant) involves collection of hematopoietic stem cells from the patient (autologous) or a donor (allogeneic), possible cryopreservation, administration of high-dose chemotherapy ± radiation (conditioning), then reinfusion of stem cells to reconstitute hematopoiesis; peripheral blood, bone marrow, or cord blood are common sources.

Transplant types and process: Autologous transplants use the patient’s own cells (harvested, frozen, and reinfused after high-dose therapy); allogeneic transplants use donor cells and can provide graft-versus-tumor effects but carry risks such as graft-versus-host disease. Non-myeloablative (reduced-intensity or “mini”) regimens use lower intensity conditioning to allow donor engraftment with less toxicity and are intended for older or less fit patients.

Rationale: Myeloablative allogeneic HSCT aims to eradicate disease and rescue marrow, and is an effective curative approach for many hematologic malignancies in appropriately selected patients. Non-myeloablative approaches seek to exploit graft-versus-tumor effects while reducing regimen-related mortality for patients ineligible for full myeloablation.

MTAC evidence assessments: Historical MTAC reviews summarized condition-specific evidence — examples include CML (autologous SCT did not meet MTAC based on limited evidence), multiple sclerosis and breast cancer (HDC with SCT did not meet MTAC), mixed conclusions for multiple myeloma over time, and generally insufficient evidence to support non-myeloablative transplants for several solid tumor and hematologic indications. MTAC determinations and literature reviews (case series, registries, some nonrandomized trials) form the evidence base summarized in the background.

Administrative notes: Applicable CPT/HCPCS codes for harvesting, preparation, transplantation, and storage are listed and claims for long-term storage often face denial unless the member is scheduled for transplant; documentation requirements include last 6 months of clinical notes and other prior-authorizations as required by plan.

Myeloablative conditioning: High-dose chemo/radiotherapy regimen intended to eradicate bone marrow prior to transplant; requires stringent organ function thresholds (contraindications include EF <45%, FEV1 <50% or DLCO <50% predicted, creatinine clearance <60 ml/min with limited exceptions, and bilirubin >3.0 mg/dL or transaminases >3x ULN).

Non-myeloablative (mini) transplant: Reduced-intensity conditioning regimen intended to allow donor engraftment with less toxicity; may have less stringent organ function requirements and was reviewed as investigational for many indications per MTAC (insufficient high-quality evidence for most hematologic and solid tumor uses).

NST definition (Non-myeloablative / mini transplant): Conditioning regimens not resulting in permanent marrow aplasia, causing only mild myelodepression but potent immunosuppression and often inducing mixed chimerism; designed to allow relatively prompt hematopoietic recovery without full ablation.

MTAC: Kaiser Permanente Medical Technology Assessment Criteria (MTAC) is the assessment framework used to evaluate evidence and determine whether specific transplant indications or approaches meet criteria for coverage.