{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T03:25:10Z","timestamp":1790220310306,"version":"4.1.0"},"reference-count":122,"publisher":"American Society of Hematology","issue":"12","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2018,6,26]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Hepatic veno-occlusive disease\/sinusoidal obstruction syndrome (VOD\/SOS) is a potentially life-threatening complication of conditioning during hematopoietic stem cell transplantation (HSCT) or chemotherapy without HSCT, with a historically reported mean incidence of 13.7% post-HSCT. Typical symptoms of VOD\/SOS may include hyperbilirubinemia, painful hepatomegaly, weight gain, and ascites. Defibrotide, a polydisperse mixture of predominantly single-stranded polydeoxyribonucleotides, is currently the only therapy approved to treat hepatic VOD\/SOS with pulmonary\/renal dysfunction (ie, multiorgan dysfunction\/multiorgan failure [MOD\/MOF]) following HSCT in the United States and to treat severe hepatic VOD\/SOS post-HSCT in the European Union. In preclinical and human studies, defibrotide has demonstrated profibrinolytic, antithrombotic, anti-inflammatory, and angio-protective actions, thus promoting an anticoagulant phenotype of the endothelium that protects and stabilizes the function of endothelial cells. In a phase 3, historically controlled, multicenter trial in adults and children with VOD\/SOS and MOD\/MOF (defibrotide: n = 102; controls treated before defibrotide availability: n = 32), defibrotide resulted in significantly greater day +100 survival following HSCT (38.2%) vs controls (25.0%; propensity analysis-estimated between-group difference: 23%; P = .0109). The most common adverse events (AEs) were hypotension and diarrhea; rates of common hemorrhagic AEs were similar in the defibrotide and historical control group (64% and 75%, respectively). In a phase 3 prophylaxis trial, defibrotide was found to lower incidence of VOD\/SOS in children (not an approved indication) and reduce the incidence of graft-versus-host disease. This review describes the development and clinical applications of defibrotide, focusing on its on-label use in patients with VOD\/SOS and MOD\/MOF after HSCT.<\/jats:p>","DOI":"10.1182\/bloodadvances.2017008375","type":"journal-article","created":{"date-parts":[[2018,6,26]],"date-time":"2018-06-26T15:55:14Z","timestamp":1530028514000},"page":"1495-1509","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":69,"title":["The use of defibrotide in blood and marrow transplantation"],"prefix":"10.1182","volume":"2","author":[{"given":"Paul G.","family":"Richardson","sequence":"first","affiliation":[{"name":"Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Enric","family":"Carreras","sequence":"additional","affiliation":[{"name":"Spanish Bone Marrow Donor Program, Josep Carreras Leukemia Foundation and Leukemia Research Institute, Barcelona, Spain;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimo","family":"Iacobelli","sequence":"additional","affiliation":[{"name":"Techitra s.r.l., Milan, Italy; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bijan","family":"Nejadnik","sequence":"additional","affiliation":[{"name":"Galena Biopharma, San Ramon, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","published-online":{"date-parts":[[2018,6,26]]},"reference":[{"key":"2019111616025292000_B1","unstructured":"Gentium\n              SA\n            \n          . 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