ArticleBlood advances2026
PTCy-based allo-BMT platform as a curative, accessible alternative for pediatric and young adult severe aplastic anemia.
Article in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
abstractSevere aplastic anemia (SAA) is a life-threatening bone marrow failure syndrome. Allogeneic bone marrow transplantation (allo-BMT) is the most definitive curative treatment for SAA and is prioritized when matched sibling donors (MSDs) are available. Haploidentical BMT (haplo-BMT) with posttransplant cyclophosphamide (PTCy) has emerged as a promising alternative, expanding donor access while mitigating historically high rates of graft failure and graft-versus-host disease (GVHD). We report the outcomes of 31 consecutive pediatric, adolescent, and young adult patients (aged ≤21 years) with acquired SAA who underwent allo-BMT at a single institution (2014-2024). Patients with treatment-naïve (TN) and relapsed/refractory (R/R) SAA were included. Patients received reduced-intensity conditioning (RIC) haplo-BMT or MSD-BMT with PTCy, mycophenolate mofetil, and tacrolimus (PTCy cohort, n = 23), or standard-of-care (SOC) MSD-BMT with cyclophosphamide/antithymocyte globulin conditioning and calcineurin inhibitor/methotrexate-based GVHD prophylaxis (SOC cohort, n = 8). Overall survival was 97% (PTCy, 96%; SOC, 100%). GVHD rates were low, with only 1 case of grade 2 acute GVHD and 1 case of extensive chronic GVHD (cGVHD) in the PTCy group, and 1 case of extensive cGVHD in the SOC group. No patients developed clonal hematopoiesis. Late effects were infrequent, and limited to menstrual dysfunction and BK virus-associated nephropathy in 1 patient. Robust donor chimerism was achieved in the PTCy cohort, in contrast to uniformly mixed chimerism in the SOC group. These findings demonstrate that RIC haplo-BMT with PTCy is a safe and effective curative approach for pediatric patients with both R/R and TN SAA, supporting its use as a frontline option even when MSDs are available.
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