Fierro-Pineda JC, Tsai H-L, Blackford AL, et al. Prospective PTCTC Trial of Myeloablative HaploBMT with Post-Transplant Cyclophosphamide for Pediatric Acute Leukemias. Blood Advances. 2023; (doi: 10.1182/bloodadvances.2023010281).

New research shows that myeloablative conditioning followed by haploidentical bone marrow transplant (haploBMT) using post-transplantation cyclophosphamide (PTCy) did not lead to transplant-related mortality (TRM) or severe acute graft-versus-host-disease (aGVHD) among pediatric and young adult patients with high-risk acute leukemias or myelodysplastic syndrome (MDS). In the prospective study for the Pediatric Transplantation and Cellular Therapy Consortium, 32 patients (median age 12 years) with acute leukemias or MDS received myeloablative conditioning (MAC), haploBMT with PTCy, mycophenolate mofetil, and tacrolimus. Fifteen patients were diagnosed with acute myeloid leukemia, 11 with acute lymphoid leukemia, one with mixed lineage leukemia, and five with MDS. At 180 days post-transplant, the rate of TRM — the primary objective — was 0%. The cumulative incidence (CuI) of aGVHD grade II at day 100 was 13%.  No patient developed aGVHD grades III-IV.  The 1-year CuI of moderate-severe chronic GVHD was 4%. In all, 84% of patients experienced donor engraftment. Three patients who received suboptimal bone marrow grafts had primary graft failures that were corrected after second transplants. At 1 year, the CuI of relapse was 32%, with greater frequency in patients with pre-BMT minimal residual disease. At 1 year and 2 years, overall survival was 77% and 73%, while the respective rates of event-free survival were 68% and 64%.

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Tags: GVHD, acute, Acute GVHD, aGVHD, PTCy