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10.1111/bjh.15267

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C6032999!6032999!29767845
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suck abstract from ncbi


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pmid29767845      Br+J+Haematol 2018 ; 181 (6): 816-27
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  • Final results from a defibrotide treatment?IND study for patients with hepatic veno?occlusive disease/sinusoidal obstruction syndrome #MMPMID29767845
  • Kernan NA; Grupp S; Smith AR; Arai S; Triplett B; Antin JH; Lehmann L; Shore T; Ho VT; Bunin N; Iacobelli M; Liang W; Hume R; Tappe W; Soiffer R; Richardson P
  • Br J Haematol 2018[Jun]; 181 (6): 816-27 PMID29767845show ga
  • Hepatic veno?occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life?threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi?organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded?access treatment programme (T?IND; NCT00628498). In the completed T?IND, the Kaplan?Meier estimated Day +100 survival for 1000 patients with documented defibrotide treatment after HSCT was 58·9% [95% confidence interval (CI), 55·7?61·9%]. Day +100 survival was also analysed by age and MOD status, and post hoc analyses were performed to determine Day +100 survival by transplant type, timing of VOD/SOS onset (?21 or >21 days) and timing of defibrotide treatment initiation after VOD/SOS diagnosis. Day +100 survival in paediatric patients was 67·9% (95% CI, 63·8?71·6%) and 47·1% (95% CI, 42·3?51·8%) in adults. All patient subgroups without MOD had higher Day +100 survival than those with MOD; earlier defibrotide initiation was also associated with higher Day +100 survival. The safety profile of defibrotide in the completed T?IND study was similar to previous reports.
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