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AML and Checkpoint Inhibitors: Outcomes With Cyclophosphamide After Transplantation

By: Kayci Reyer
Posted: Monday, July 27, 2020

According to research published in the journal Cancer, hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) who have previously been treated with checkpoint inhibitors may be feasible. Betül Oran, MD, of MD Anderson Cancer Center, Houston, and colleagues sought to determine whether the use of cyclophosphamide after transplantation would affect the prevalence of post-transplantation acute graft-versus-host disease.

The retrospective study included 43 patients with AML and/or myelodysplastic syndromes who had received treatment with anti–PD-1 blockade (n = 32), anti–CTLA-4 blockade (n = 9), or both (n = 2) before transplantation. Patients who received cyclophosphamide following transplantation (n = 22) had lower instances of grade 3 or 4 acute graft-versus-host disease than those who did not (n = 21), despite a comparable rate of occurrence of grade 2 to 4 acute graft-versus-host disease. Patients who had received more than four rounds of checkpoint inhibitor treatment prior to transplantation and who did not receive prophylactic cyclophosphamide had a higher rate of grade 3 or 4 acute graft-versus-host disease than those who did (43% vs. 12%).

At 1-year follow-up, progression-free survival among patients who had undergone prior checkpoint inhibitor treatment was superior for patients who had received cyclophosphamide than for those who had not (55% vs. 22%). Cyclophosphamide appeared to lead to improvements in graft-versus-host disease and did not seem to compromise disease control.

Disclosure: For full disclosures of the study authors, visit

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