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Chronic Myelogenous Leukemia Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Chronic-Phase Chronic Myelogenous Leukemia (CML)


About 20% of otherwise eligible CML patients lack a suitably matched sibling donor.[37] HLA-matched unrelated donors or donors mismatched at one-HLA antigen can be found for about 50% of eligible participants through the National Marrow Donor Program.[37] A retrospective review of 2,444 patients who received myeloablative allogeneic SCT showed OS at 15 years of 88% (95% confidence interval [CI], 86%-90%) for sibling-matched transplant and of 87% (95% CI, 83%-90%) for unrelated donor transplant.[38] The cumulative incidences of relapse were 8% (95% CI, 7%-10%) for sibling-matched transplant and 2% (95% CI, 1%- 4%) for unrelated donor transplant.[38]

Although the majority of relapses occur within 5 years of transplantation, relapses have occurred for as long as 15 years following BMT.[39] In a molecular analysis of 243 patients who underwent allogeneic BMT over a 20-year interval, only 15% had no detectable BCR/ABL transcript by PCR analysis.[40] The risk of relapse appears to be less in patients transplanted early in disease and in patients who develop chronic graft-versus-host disease.[30,41]

With the advent of imatinib, dasatinib, and nilotinib, the timing and sequence of allogeneic BMT or SCT has been cast in doubt.[26] Allogeneic SCT is the preferred choice for patients presenting with accelerated-phase or blast-phase disease, for patients with a T3151 mutation (resistant to currently available tyrosine kinase inhibitors), and for patients with complete intolerance to the pharmacologic options.[42]

In a prospective trial of 354 patients aged younger than 60 years, 123 of 135 patients with a matched, related donor underwent early allogeneic SCT while the others received interferon-based therapy and imatinib at relapse; some also underwent a matched, unrelated-donor transplant in remission.[43] With a 9-year median follow-up, survival still favored the drug treatment arm (P = .049), but most of the benefit was early as a result of transplant-related mortality, with the survival curves converging by 8 years.[43][Level of evidence: 2A] Among the many unanswered questions are the following:

  • Should younger eligible patients move quickly toward allogeneic SCT after induction failure by imatinib mesylate?
  • Does the substantial toxicity and mortality of allogeneic transplantation render its early use obsolete?
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