Safety and effects of prophylactic defibrotide for sinusoidal obstruction syndrome in hematopoietic stem cell transplantation

Ann Transplant. 2013 Jan 28:18:36-42. doi: 10.12659/AOT.883811.

Abstract

Background: Sinusoidal obstruction syndrome (SOS) is a serious complication of hematopoietic stem cell transplantation (HSCT), with a mortality rate of up to 90%. We report our experience on the use of defibrotide for SOS prophylaxis in HSCT.

Material and methods: We retrospectively reviewed data of 49 patients who received defibrotide as SOS prophylaxis during the course of HSCT at the National Cancer Center, Goyang, Korea, between August 2005 and July 2008.

Results: Thirty-four patients (69.4%) were classified as a high-risk group for developing SOS. Defibrotide was well-tolerated, without any grade 3 or 4 toxicity. The median value of maximum total bilirubin within 100 days after HSCT was within the normal range. SOS was diagnosed in only 1 patient, who underwent autologous HSCT due to relapsed medulloblastoma. There was no day 100 treatment-related mortality in our study.

Conclusions: Defibrotide appears to be a safe prophylaxis for SOS. This study suggests that it could be effective to use prophylactic defibrotide in advance to improve HSCT outcomes in patients at risk of SOS.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Female
  • Fibrinolytic Agents / adverse effects
  • Fibrinolytic Agents / therapeutic use*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Hepatic Veno-Occlusive Disease / etiology*
  • Hepatic Veno-Occlusive Disease / prevention & control*
  • Humans
  • Male
  • Middle Aged
  • Polydeoxyribonucleotides / adverse effects
  • Polydeoxyribonucleotides / therapeutic use*
  • Retrospective Studies
  • Risk Factors
  • Treatment Outcome
  • Young Adult

Substances

  • Fibrinolytic Agents
  • Polydeoxyribonucleotides
  • defibrotide