{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,30]],"date-time":"2026-07-30T21:33:10Z","timestamp":1785447190672,"version":"3.56.0"},"reference-count":35,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2018,6,6]],"date-time":"2018-06-06T00:00:00Z","timestamp":1528243200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000054","name":"National Cancer Institute","doi-asserted-by":"publisher","award":["P30 CA008748"],"award-info":[{"award-number":["P30 CA008748"]}],"id":[{"id":"10.13039\/100000054","id-type":"DOI","asserted-by":"publisher"}]},{"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":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2018,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Hepatic veno\u2010occlusive disease\/sinusoidal obstruction syndrome (VOD\/SOS) is a potentially fatal complication of conditioning for hematopoietic stem cell transplantation (HSCT) but can occur after nontransplant\u2010associated chemotherapy. Following HSCT, VOD\/SOS with multi\u2010organ dysfunction (MOD) may be associated with\u00a0&gt;80% mortality. Defibrotide is approved to treat severe hepatic VOD\/SOS post\u2010HSCT in patients aged\u00a0&gt;1 month in the European Union and hepatic VOD\/SOS with renal or pulmonary dysfunction post\u2010HSCT in the United States. Prior to US approval, defibrotide was available to treat VOD\/SOS through an expanded\u2010access treatment (T\u2010IND) program. A post hoc analysis of nontransplant\u2010associated VOD\/SOS patients treated with defibrotide initiated within 30 days of starting chemotherapy and followed for 70 days is presented.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>Patients were diagnosed by Baltimore or modified Seattle criteria or biopsy, and received defibrotide 25\u00a0mg\/kg\/day in four divided doses (\u226521 days recommended).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Of the 1,154 patients in the T\u2010IND, 137 had nontransplant\u2010associated VOD\/SOS, 82 of whom developed VOD\/SOS within 30 days of starting chemotherapy. Of them, 66 (80.5%) were aged \u226416 years. Across all the 82 patients, Kaplan\u2013Meier estimated day +70 survival was 74.1%, 65.8% in patients with MOD (n\u00a0=\u00a038), and 81.3% in patients without MOD (n\u00a0=\u00a044). By age group, Kaplan\u2013Meier estimated day +70 survival was 80.1% in pediatric patients (n\u00a0=\u00a066) and 50.0% in adults (n\u00a0=\u00a016). Treatment\u2010related adverse events occurred in 26.8%.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In this post hoc analysis of 82 patients initiating defibrotide within 30 days of starting chemotherapy, Kaplan\u2013Meier estimated survival was 74.1% at 70 days after defibrotide initiation. Safety profile was consistent with prior defibrotide studies.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.27269","type":"journal-article","created":{"date-parts":[[2018,6,6]],"date-time":"2018-06-06T15:14:21Z","timestamp":1528298061000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Defibrotide for the treatment of hepatic veno\u2010occlusive disease\/sinusoidal obstruction syndrome following nontransplant\u2010associated chemotherapy: Final results from a post hoc analysis of data from an expanded\u2010access program"],"prefix":"10.1002","volume":"65","author":[{"given":"Nancy A.","family":"Kernan","sequence":"first","affiliation":[{"name":"Pediatric Bone Marrow Transplantation Service, Department of Pediatrics Memorial Sloan Kettering Cancer Center  New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul G.","family":"Richardson","sequence":"additional","affiliation":[{"name":"Jerome 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