title: Treatment of sinusoidal obstruction syndrome with defibrotide: a single-center experience.
abstract: Sinusoidal obstruction syndrome (SOS) is a frequent, troubling, and potentially fatal complication of hematopoietic stem cell transplantation. Despite promising results with defibrotide (DF), no treatment has been established as standard. DF is a single-stranded polydeoxyribonucleotide, obtained from controlled depolymerization of porcine intestinal mucosal cells. It has antithrombotic, antiischemic, antiinflammatory, and thrombolytic properties without significant side effects. We retrospectively evaluated the charts of 80 consecutive patients, with 89 hematopoietic stem cell transplants for hematologic malignancies. The results of early initiation of DF treatment in 14 patients with SOS are presented in this study. Fourteen patients, 8 males and 6 females % median age 40.5 years (range, 16-46 years) were diagnosed to have SOS. Disease severity was classified as severe in 6 (42.85%), moderate in 4 (28.57%), and mild in 4 (28.57%) patients. We treated 14 patients with DF for a median of 21.5 days (range, 4-39 days). All 14 patients received DF after the diagnosis of SOS. Three patients with severe and all of the patients with mild to moderate SOS responded to treatment with complete resolution of SOS-related signs and symptoms. All patients responding to DF were alive at 100 days posttransplantation. There was no significant drug-related side effect among patients treated with DF. With an overall response rate of 78.56% and a 50% complete response rate in severe SOS cases and minimal side effects, we suggest that DF is the best available agent to treat SOS.
authors.affiliation: Department of Hematology, Gazi University Faculty of Medicine, Besevler, Ankara, Cankaya 06500, Turkey. aysucak@gazi.edu.tr
authors.forename: G T
authors.initials: GT
authors.lastname: Sucak
authors.forename: Z S
authors.initials: ZS
authors.lastname: Aki
authors.forename: M
authors.initials: M
authors.lastname: Yagcí
authors.forename: Z A
authors.initials: ZA
authors.lastname: Yegin
authors.forename: Z N
authors.initials: ZN
authors.lastname: Ozkurt
authors.forename: R
authors.initials: R
authors.lastname: Haznedar
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D001787:Blood Group Incompatibility; D005260:Female; D005343:Fibrinolytic Agents / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006493:Heparin / Q000627:therapeutic use; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy* / Q000209:etiology*; D006801:Humans; D007938:Leukemia / Q000628:therapy; D008223:Lymphoma / Q000628:therapy; D008297:Male; D008875:Middle Aged; D011089:Polydeoxyribonucleotides / Q000627:therapeutic use*; D012189:Retrospective Studies; D014184:Transplantation, Homologous
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide; D006493:Heparin