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Comparative Effectiveness Review of the Influence of Stem Cell Source on Allogeneic Stem Cell Transplant Effectiveness for Treatment of Chronic Myelogenous Leukemia

June 30, 2016

Purpose of Technology: Patients with chronic myelogenous leukemia (CML) overproduce partially mature, abnormal white blood cells of the myeloid lineage. Allogeneic hematopoietic stem cell transplantation (HSCT) is a procedure in which patients undergo high-dose chemotherapy and/or radiotherapy to destroy their abnormal bone marrow and leukemia cells. This is followed by transplantation of stem cells harvested from a healthy donor to reestablish normal hematopoiesis and exert a graft-versus-leukemia effect. Donor stem cells for transplant are harvested from bone marrow, peripheral blood, or umbilical cord blood. The optimal donor choice is a matched sibling donor; alternatives are matched related or unrelated donors. HSCT outcomes are influenced by multiple factors, including donor factors, and stem cell source.

Controversy: The risk of transplant-related morbidity and mortality are a major concern and a barrier for many patients. Additionally, the optimal timing of transplant is under debate, particularly for patients in chronic phase (CP) CML. The relative access to a fully matched related donor (RD) or matched unrelated donor (MURD) may strongly influence the outcomes of the transplant. Questions remain regarding how differences in the composition of graft sources, i.e., peripheral blood stem cell transplantation (PBSCT) or bone marrow transplantation (BMT), influence outcomes.

Relevant Questions:

  • How do remission, survival, and relapse rates following allogeneic HSCT for CML compare between the use of BMT and PBSCT?
  • How do remission, survival, and relapse compare between HSCT performed with stem cells harvested from an human leukocyte antigen (HLA)-matched sibling donor and those using grafts from related and unrelated donors, or from mismatched donors?
  • How do safety outcomes compare between allogeneic HSCT using different graft and donor sources?
  • Have definitive patient selection criteria been established for the use of BMT versus PBSCT, and for the various donor graft sources?