TY - JOUR TI - Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children AU - Murillo-Sanjuán, L. AU - González-Vicent, M. AU - Argilés Aparicio, B. AU - Badell Serra, I. AU - Rodríguez Villa, A. AU - Uria Oficialdegui, M. L. AU - López-Duarte, M. AU - Beléndez-Bieler, C. AU - Sastre Urgelles, A. AU - Sevilla Navarro, J. AU - Diaz-de-Heredia, C. T2 - Bone Marrow Transplantation AB - Hematopoietic stem cell transplantation (HSCT) is currently the only curative option for hematological manifestations in patients with Fanconi anemia (FA). We report the outcome of 34 patients with FA inside a collaborative multicenter national study based on recommendations of Spanish Working Group for Bone Marrow Transplantation in Children (GETMON) between 2009 and 2016. Fludarabine-based conditioning regimen was carried out in all patients, with low dose total body irradiation in unrelated transplants. Disease status before HSCT was bone marrow failure (BMF) in 30 patients and myelodysplastic syndrome (MDS) in four. Donors were matched siblings donors (MSD) in 18, matched unrelated donors (MUD) in 15, and one haploidentical donor. All except one patient engrafted. Cumulative incidence of grades II-IV acute graft-versus-host disease (GVHD) was 29% and 11% for chronic GVHD. Median follow-up after HSCT was 6.5 years. Seven patients (21%) died due to transplant-related causes, two (6%) because of MDS relapse, and one (3%) after a squamous cell carcinoma. Overall survival (OS) was 73% at 5 years post-transplant, with no differences between MSD and MUD transplants. OS for patients with BMF was 80% while for MDS was 25%. Our data suggest HSCT can cure hematologic manifestations of most FA patients with BMF. DA - 2020/12/10/ PY - 2020 DO - 10.1038/s41409-020-01172-y DP - www.nature.com SP - 1 EP - 4 LA - en SN - 1476-5365 ST - Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia UR - https://www.nature.com/articles/s41409-020-01172-y Y2 - 2020/12/10/17:23:26 ER -