{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T22:26:30Z","timestamp":1789079190154,"version":"build-2803163510"},"reference-count":32,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2018,5,16]],"date-time":"2018-05-16T00:00:00Z","timestamp":1526428800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100011096","name":"Jazz Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011096","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2018,6]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Hepatic veno\u2010occlusive disease\/sinusoidal obstruction syndrome (VOD\/SOS) is a potentially life\u2010threatening 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\u2010organ dysfunction (MOD)] after HSCT in the United States and severe VOD\/SOS after HSCT in patients aged older than 1\u00a0month in the European Union. Defibrotide was available as an investigational drug by an expanded\u2010access treatment programme (T\u2010IND; NCT00628498). In the completed T\u2010IND, the Kaplan\u2013Meier estimated Day +100 survival for 1000 patients with documented defibrotide treatment after HSCT was 58\u00b79% [95% confidence interval (CI), 55\u00b77\u201361\u00b79%]. Day +100 survival was also analysed by age and MOD status, and <jats:italic>post hoc<\/jats:italic> analyses were performed to determine Day +100 survival by transplant type, timing of VOD\/SOS onset (\u226421 or &gt;21\u00a0days) and timing of defibrotide treatment initiation after VOD\/SOS diagnosis. Day +100 survival in paediatric patients was 67\u00b79% (95% CI, 63\u00b78\u201371\u00b76%) and 47\u00b71% (95% CI, 42\u00b73\u201351\u00b78%) 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\u2010IND study was similar to previous reports.<\/jats:p>","DOI":"10.1111\/bjh.15267","type":"journal-article","created":{"date-parts":[[2018,5,16]],"date-time":"2018-05-16T12:54:36Z","timestamp":1526475276000},"page":"816-827","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":120,"title":["Final results from a defibrotide treatment\u2010<scp>IND<\/scp> study for patients with hepatic veno\u2010occlusive disease\/sinusoidal obstruction syndrome"],"prefix":"10.1111","volume":"181","author":[{"given":"Nancy A.","family":"Kernan","sequence":"first","affiliation":[{"name":"Memorial Sloan\u2010Kettering Cancer Center New York NY USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephan","family":"Grupp","sequence":"additional","affiliation":[{"name":"The Children's Hospital of Philadelphia Philadelphia PA 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