Media type: E-Article Title: Treosulfan/fludarabine as an allogeneic hematopoietic stem cell transplant conditioning regimen for high‐risk patients Contributor: Baronciani, Donatella; Rambaldi, Alessandro; Iori, Anna Paola; Di Bartolomeo, Paolo; Pilo, Federica; Pettinau, Martina; Depau, Cristina; Mico, Caterina; Santarone, Stella; Angelucci, Emanuele imprint: Wiley, 2008 Published in: American Journal of Hematology Language: English DOI: 10.1002/ajh.21240 ISSN: 0361-8609; 1096-8652 Keywords: Hematology Origination: Footnote: Description: <jats:title>Abstract</jats:title><jats:p>In recent years, new conditioning regimens have been explored in patients not eligible for conventional transplant with the aim to reduce transplant‐related mortality. In a phase II multicentric prospective trial, we investigated the safety and feasibility of the treosulfan–fludarabine combination prior to allogeneic hematopoietic stem cell transplant in patients with various hematological malignancies not eligible for conventional regimens because of previous intensive treatment, older age, and comorbidities. Forty‐six consecutive patients, median age 48 years (range 17–69), were enrolled. Sixteen of them were in complete remission, and 20 had a HSCT comorbidity index ≥ 1. Forty‐four patients had regular and sustained engraftment, and 39 out of 40 evaluable patients developed complete chimerism. Nonhematological toxicity was limited. Risk of transplant‐related mortality was 9% (95% CI, 2–17%) at day +100 and plotted at 15% (95% CI, 7–22%) after 7 months. The estimated overall survival and progression‐free survival with a median follow‐up of 20 months were 51% and 38%, respectively. The estimated 30 months progression‐free survival for patients transplanted in remission was 56%. The treosulfan–fludarabine combination is a reduced‐toxicity but myeloablative regimen that can be proposed to patients not fitting criteria for conventional transplant regimens. Longer follow‐up and further prospective studies are necessary to evaluate this regimen. © 2008 Wiley‐Liss, Inc. Am. J. Hematol., 2008.</jats:p> Access State: Open Access