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Busulfan–fludarabine- or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major

Lüftinger, Roswitha ; Zubarovskaya, Natalia ; Galimard, Jacques-Emmanuel ; Cseh, Annamaria ; Salzer, Elisabeth ; Locatelli, Franco ; Algeri, Mattia ; Yesilipek, Akif ; de la Fuente, Josu ; Isgrò, Antonella ; Alseraihy, Amal ; Angelucci, Emanuele ; Smiers, Frans J. ; La La Nasa, Giorgia ; Zecca, Marco ; Fisgin, Tunc ; Unal, Emel ; Kleinschmidt, Katharina ; Peters, Christina ; Lankester, Arjan ; Corbacioglu, Selim



Zusammenfassung

Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients' life expectancy. However, transfusion-associated iron overload remains a significant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of ...

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