Purpose of Technology: Patients with chronic myelogenous leukemia (CML) have an overproduction of partially mature white blood cells of the myeloid lineage. High doses of chemotherapy and/or radiotherapy can destroy bone marrow producing the abnormal cells but normal cells are also affected. Allogeneic hematopoietic stem cell transplantation (HSCT) is a procedure that uses stem cells derived from the peripheral blood or bone marrow of matched related or unrelated donors, which are infused into the transplant recipient where they migrate to the bone marrow and help reestablish normal hematopoiesis. When the highly effective tyrosine kinase inhibitor (TKI) drugs were introduced, the use of HSCT as a primary therapy declined among patients with CML, particularly those in the chronic phase of the disease. While TKIs are highly effective, allogeneic HSCTs provide the only potentially curative therapy for CML.

Relevant Questions:

  • Does HSCT effectively improve survival compared with TKIs or other drug-based therapies?

  • Do patients achieve hematologic, cytogenetic, and/or molecular responses more often with HSCT compared with TKIs or other drug-based therapies?

  • Are HSCTs and drug-based therapies comparatively safe?

  • Have definitive patient selection criteria been established for HSCT versus TKI or other drug therapy for CML?


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