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10.1016/j.bbmt.2014.08.020

http://scihub22266oqcxt.onion/10.1016/j.bbmt.2014.08.020
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C4324724!4324724!25196857
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suck abstract from ncbi


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pmid25196857      Biol+Blood+Marrow+Transplant 2014 ; 20 (12): 1996-2003
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  • Treosulfan-Based Conditioning and Hematopoietic Cell Transplantation for Nonmalignant Diseases: A Prospective Multi-Center Trial #MMPMID25196857
  • Burroughs LM; Nemecek ER; Torgerson TR; Storer BE; Talano JA; Domm J; Giller RH; Shimamura A; Delaney C; Skoda-Smith S; Thakar MS; Baker KS; Rawlings DJ; Englund JA; Flowers MED; Deeg HJ; Storb R; Woolfrey AE
  • Biol Blood Marrow Transplant 2014[Dec]; 20 (12): 1996-2003 PMID25196857show ga
  • Hematopoietic cell transplantation (HCT) is effective in the treatment of patients with nonmalignant diseases and for many is the only known cure. Conventional myeloablative regimens have been associated with unacceptably high early transplant-related mortality (TRM) particularly in patients with co-morbid conditions. This prospective multicenter trial was designed to determine the safety and engraftment efficacy of treosulfan-based conditioning in patients with nonmalignant diseases. Thirty-one patients received HLA-matched related (n=4) or unrelated (n=27) grafts following conditioning with treosulfan (total dose: 42 g/m2), fludarabine (total dose: 150 mg/m2), ± thymoglobulin (6 mg/kg; n=22). Graft-versus-host disease (GVHD) prophylaxis consisted of tacrolimus and methotrexate. All patients engrafted. Day-100 TRM was 0%. With a median follow-up of 2 years, the 2-year survival was 90%. Three patients died of GVHD, recurrent hemophagocytic lymphohistiocytosis, and a surgical complication, respectively. The cumulative incidences of grades II?IV and III?IV acute GVHD at day 100 and chronic GVHD at 2 years were 62%, 10% and 21%, respectively. Patients who received thymoglobulin had a significantly lower incidence of grade III-IV acute GVHD (0% versus 33%; P = 0.005). These results indicate that the combination of treosulfan, fludarabine, and thymoglobulin is effective at establishing donor engraftment with low toxicity and improved survival in patients with nonmalignant diseases, and support the need for future disease-specific clinical trials.
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