Outcomes of Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide-Based GVHD Prophylaxis in Acute Myeloid Leukemia and Myelodysplastic Syndromes Patients Over 70 Years of Age: A Comparative Analysis by Age Group.
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BACKGROUND: We aimed to study outcomes of allogeneic hematopoietic cell transplantation in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients aged above 70 with using post-transplant cyclophosphamide (PTCy) graft-versus-host disease (GVHD) prophylaxis. METHODS: We performed a retrospective multicenter analysis on the Center for International Blood and Marrow Transplant Research registry of adult patients with acute myeloid leukemia or myelodysplastic syndromes who underwent their first allogeneic hematopoietic cell transplantation (2012-2017) with post-transplant cyclophosphamide-based GVHD prophylaxis. Patients were stratified by age (>70 vs. 18-70 years; the comparator was predominantly older, with a median age of 61.9). Primary outcomes included overall survival (OS), disease-free survival (DFS), relapse, nonrelapse mortality (NRM), acute and chronic GVHD, and GVHD-free relapse-free survival. Multivariable Cox and cause-specific hazards models assessed the independent effect of age >70 years. RESULTS: Of 431 patients, 71 (16%) were aged >70 years. Haploidentical donors were used in 84% of older versus 69% of younger patients. With a median follow-up of 4.0 years, 1-year OS was 54% in patients >70 years versus 63% in those 18 to 70 years; 1-year DFS was 38% versus 42%. In multivariable analysis, age >70 years was associated with inferior OS (hazard ratio 1.57, 95% CI 1.06-2.34) and higher NRM (hazard ratio 2.21, 95% CI 1.29-3.80), but not with relapse, DFS, GVHD-free relapse-free survival, acute GVHD, chronic GVHD, or neutrophil engraftment. CONCLUSION: Allogeneic hematopoietic cell transplantation with PTCy is feasible in carefully selected patients aged >70 years with AML/MDS. Although higher NRM and modestly inferior OS persist, comparable relapse rates, DFS, and GVHD control support individualized rather than age-based decision-making for transplantation in this population.