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Cesaro, Simone; Pillon, Marta; Sauer, Martin; Smiers, Frans; Faraci, Maura; de Heredia, Cristina Diaz; Wynn, Robert; Greil, Johann; Locatelli, Franco; Veys, Paul; Uyttebroeck, Anne; Ljungman, Per; Chevalier, Patrice; Ansari, Marc; Badell, Isabel; Guengoer, Tayfun; Salim, Rahuman; Tischer, Johanna; Tecchio, Cristina; Russell, Nigel; Chybicka, Alicja; Styzynski, Jan; Krivan, Gergely; Smith, Owen; Stein, Jerry; Afanasyev, Boris; Pochon, Cecile; Menconi, Maria Cristina; Bosman, Paul; Mauro, Margherita; Tridello, Gloria; de Latour, Regis Peffault and Dufour, Carlo (2020): Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT). In: Bone Marrow Transplantation, Vol. 55, No. 9: pp. 1796-1809

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Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative procedure in patients with Shwachman-Diamond syndrome (SDS) with bone marrow abnormalities. The results of 74 patients with SDS (6 acute myeloid leukemia, 7 myelodysplastic syndrome, and 61 bone marrow failure) treated with HSCT between 1988 and 2016 are reported. The donor source was: 24% sibling, 8% parent, and 68% unrelated donor. The stem cell source was: 70% bone marrow, 19% peripheral blood stem cells, and 11% cord blood. The conditioning regimen was myeloablative in 54% and reduced intensity in 46%. Neutrophil engraftment was achieved in 84% of patients after a median time of 17.5 days. Graft failure occurred in 15% of HSCTs. Grades I-IV acute and chronic GVHD were observed in 55% and 20% of patients, respectively. After a median follow-up of 7.3 years (95% CI 4.8-10.2), 28 patients died for progression/relapse (7) or toxicity (21). The 5-year overall survival and nonrelapse mortality were 63.3% (95% CI 50.8-73.4) and 19.8% (95% CI 10.8-30.8), respectively. In conclusion, this is the largest series so far reported and confirms that HSCT is a suitable option for patients with SDS. Further efforts are needed to lower transplant-related toxicity and reduce graft failure.

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