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Recommendations on hematopoietic stem cell transplantation for patients with Diamond–Blackfan anemia. On behalf of the Pediatric Diseases and Severe Aplastic Anemia Working Parties of the EBMT

Diaz-de-Heredia, Cristina ; Bresters, Dorine ; Faulkner, Lawrence ; Yesilipek, Akif ; Strahm, Brigitte ; Miano, Maurizio ; Dalle, Jean-Hugues ; Peffault de Latour, Régis ; Corbacioglu, Selim


Diamond Blackfan anemia (DBA) is a rare congenital syndrome presenting primarily as pure red cell aplasia with constitutional abnormalities and cancer predisposition. Established treatment options are corticosteroids, regular erythrocyte transfusions with iron chelation therapy, and hematopoietic stem cell transplantation (HSCT). To date, HSCT is the only definitive curative treatment for the ...


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