Effectiveness of defibrotide in the prevention of hepatic venooclusive disease among adult patients receiving allogeneic hematopoietic cell transplantation: A retrospective single center experience. (February 2022)

• Record Type: Journal Article
• Authors: Kayikci, Omur; Akpinar, Seval; Tekgunduz, Emre
• Is Part Of: Transfusion and apheresis science. Volume 61:Number 1(2022)
• Publication Date: 2022-02
• Subjects: Hepatic veno-occlusive disease -- Sinusoidal obstruction syndrome -- Hematopoietic stem cell transplantation -- Defibrotide -- Prophylaxis; Blood -- Transfusion -- Periodicals; Hemapheresis -- Periodicals; 615.39
• DOI: 10.1016/j.transci.2022.103369
• Languages: English
• ISSNs: 1473-0502

Abstract

Abstract: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is one of the most life-threatening early complications following hematopoietic cell transplantation (HCT). Due to the high mortality rate of severe VOD/SOS accompanied with multiorgan failure, there is a great interest in preventive strategies. The efficacy of defibrotide (DF) on the prevention of VOD/SOS has been clearly shown in high-risk pediatric patients, but evidence-based data on adults is scarce. In this report, we aimed to assess the impact of DF on the incidence of VOD/SOS in our center by posttransplant day 30 among patients who were treated with allogeneic HCT (allo−HCT). The study included a total of 56 patiens (28 males, 28 females). The median age of the study cohort was 43 (20−68). The daily dose of DF was 10 mg/kg and 25 mg/kg in 53 (94.6 %) and 3 (5.3 %) patients, respectively. Patients also recieved oral ursodeoxycolic acid (UDCA) 250 mg three-times daily started with conditioning until D + 90. Twenty-three (41.1 %) patients had at least one major EBMT-defined risk factor for development of VOD/SOS. One patient who belonged to a very high-risk group (with at least two major risk factors) developed very-severe VOD/SOS at posttransplant D + 20 and died as a result of multiorgan failure. The cumulative incidence of VOD/SOS at D + 30 was 1.9 %. Our findings indicate that 10 mg/kg daily intravenous DF combined with UDCA is quite effective in prevention of VOD/SOS in patients who underwent first allo-HSCT.