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Allogeneic BMT or SCT

Source: Chronic Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: March 13, 2025 · Captured 2026-09-09.

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Context: Treatment of Chronic-Phase CML / Treatment Options for Chronic-Phase CML

Allogeneic BMT or SCT is the only consistently successful curative treatment for patients with CML.[28-30] Patients younger than 60 years with an identical twin or with HLA–matched siblings can consider BMT early in the chronic phase. Although the procedure is associated with considerable acute morbidity and mortality, 50% to 70% of patients who undergo transplant in the chronic phase appear to be cured. The results are better in younger patients, especially for those younger than 20 years. The outcomes of patients who undergo transplant in the accelerated and blastic phases of the disease are progressively worse.[31,32] Most transplant series suggest improved survival when the procedure is performed within 1 year of diagnosis.[33-35][Level of evidence C1] The data supporting early transplant, however, have never been confirmed in controlled trials.

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Evidence (allogeneic SCT):

In a randomized clinical trial, patients underwent allogeneic SCT after receiving preparative therapy with either cyclophosphamide and total-body irradiation (TBI) or busulfan and cyclophosphamide without TBI. The following results were reported:[36][Level of evidence A1]

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Disease-free survival and OS were comparable between arms.

Busulfan and cyclophosphamide without TBI was associated with less graft-versus-host disease (GVHD) and fewer fevers, hospitalizations, and hospital days.

A retrospective review of 2,444 patients who underwent myeloablative allogeneic SCT reported the following:[37]

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A retrospective review of 2,444 patients who underwent myeloablative allogeneic SCT reported the following:[37]

The 15-year OS rates were 88% (95% CI, 86%–90%) for sibling-matched transplant recipients and 87% (95% CI, 83%–90%) for unrelated-donor transplant recipients.

A retrospective review of 2,444 patients who underwent myeloablative allogeneic SCT reported the following:[37]

The cumulative incidences of relapse were 8% (95% CI, 7%–10%) for sibling-matched transplant recipients and 2% (95% CI, 1%– 4%) for unrelated-donor transplant recipients.

In a prospective trial of 354 patients 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 patients also underwent a matched unrelated-donor SCT in remission.[38][Level of evidence B4]

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With a 9-year median follow-up, survival still favored the drug treatment arm (P = .049), but most of the benefit was early from transplant-related mortality, with the survival curves converging by 8 years.

Although most relapses occur within 5 years of transplant, relapses have occurred as late as 15 years after a BMT.[39] In a molecular analysis of 243 patients who underwent allogeneic BMT over a 20-year interval, only 15% had no detectable BCR::ABL1 transcript by PCR analysis.[40] The risk of relapse appears to be less in patients who underwent transplant early in disease and in patients who developed chronic GVHD.[32,41] In a retrospective review, patients with relapsed disease after allogeneic transplant who received TKI therapy had a 3-year OS rate of 60%.[42][Level of evidence C1]

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With the introduction of asciminib, imatinib, dasatinib, bosutinib, and nilotinib therapy, the timing and sequence of allogeneic BMT or SCT has been questioned.[43] Allogeneic SCT is the preferred choice for certain patients presenting with blastic-phase disease, those with a T315I variant and resistance to ponatinib (an oral TKI), and for patients with complete intolerance to the pharmacological options.[44] Similar outcomes were seen in patients who underwent allogeneic SCT because of TKI intolerance or nonadherence.[45]

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Preserved source evidence · Not medical advice