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Allogeneic Hematopoietic Cell Transplant
Source: Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 14, 2025 · Captured 2026-09-09.
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Context: Treatment of Refractory or Recurrent AML
When patients with relapsed disease are treated aggressively, they may have extended disease-free survival (DFS); however, patients with relapsed disease can only be cured with HCT.[25][Level of evidence C2] Allogeneic HCT for patients in their second CR provides better DFS rates than transplant for patients in relapse.[26][Level of evidence C1]
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Evidence (allogeneic HCT):
Current transplant outcomes are reported by the Center for International Blood and Marrow Transplantation Registry. The group reported the following outcomes for transplants done in the United States between 2008 and 2018:[27][Level of evidence C2]
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Current transplant outcomes are reported by the Center for International Blood and Marrow Transplantation Registry. The group reported the following outcomes for transplants done in the United States between 2008 and 2018:[27][Level of evidence C2]
The 3-year survival rate was 53% for patients with AML in second or subsequent CR versus 31% for patients with relapsed disease (or never in a CR) who underwent a matched sibling donor transplant.
Current transplant outcomes are reported by the Center for International Blood and Marrow Transplantation Registry. The group reported the following outcomes for transplants done in the United States between 2008 and 2018:[27][Level of evidence C2]
The 3-year survival rate was 50% for patients with AML in second or subsequent CR versus 30% for patients with relapsed disease (or who never achieved CR) who underwent a matched unrelated donor transplant.
Allogeneic HCT can be effective salvage therapy in some patients whose disease fails to go into remission with intensive chemotherapy (primary refractory leukemia). A number of retrospective studies have demonstrated the ability of allogeneic HCT to induce remission in primary refractory disease.[28]
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Evidence (allogeneic HCT to induce remission):
In one retrospective analysis of 168 patients with AML and primary refractory disease who underwent allogeneic HCT, the 5-year OS rate was 22%.[29]
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Another analysis was conducted among patients enrolled in a prospective cooperative group trial, SWOG S0106 (NCT00085709).[30]
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Another analysis was conducted among patients enrolled in a prospective cooperative group trial, SWOG S0106 (NCT00085709).[30]
Of the 589 patients treated in the cooperative group trial, 150 (25%) had primary refractory disease.
Another analysis was conducted among patients enrolled in a prospective cooperative group trial, SWOG S0106 (NCT00085709).[30]
Among the 64 patients with primary refractory disease who received an allogeneic HCT, the 4-year survival rate was 48% compared with 4% among the 86 patients who did not undergo transplant.[30][Level of evidence C3]
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Randomized trials testing the efficacy of this approach are not available.
Studies cited in this source section
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Preserved source evidence · Not medical advice