Pooled analysis of Day 100 survival for defibrotide-treated patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome and ventilator or dialysis dependence following haematopoietic cell transplantation

Br J Haematol. 2020 Aug;190(4):583-587. doi: 10.1111/bjh.16552. Epub 2020 Mar 10.

Abstract

For patients with untreated hepatic veno-occlusive disease (VOD)/sinusoidal obstruction syndrome (SOS) with multi-organ dysfunction (MOD), mortality is >80%. We conducted a pooled analysis of three studies that assessed Day 100 survival in relationship to MOD severity, with dialysis and/or ventilator dependence representing the most severe organ dysfunction. All patients in the analysis were diagnosed using Baltimore criteria/biopsy. This analysis of patients with VOD/SOS and MOD after haematopoietic cell transplantation (HCT; n = 651) demonstrated higher Day 100 survival rates amongst defibrotide-treated patients with VOD/SOS with less versus more severe forms of MOD. Even patients with severe forms of MOD post-HCT benefitted from defibrotide.

Keywords: defibrotide; dialysis; multi-organ dysfunction; veno-occlusive disease/sinusoidal obstruction syndrome; ventilator.

Publication types

  • Meta-Analysis
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Kidney Injury / etiology
  • Acute Kidney Injury / mortality
  • Acute Kidney Injury / therapy
  • Adolescent
  • Adult
  • Aged
  • Child
  • Child, Preschool
  • Clinical Trials, Phase II as Topic / statistics & numerical data
  • Clinical Trials, Phase III as Topic / statistics & numerical data
  • Confidence Intervals
  • Female
  • Fibrinolytic Agents / adverse effects
  • Fibrinolytic Agents / therapeutic use*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Hemorrhage / chemically induced
  • Hepatic Veno-Occlusive Disease / drug therapy*
  • Hepatic Veno-Occlusive Disease / etiology
  • Hepatic Veno-Occlusive Disease / mortality
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Middle Aged
  • Multicenter Studies as Topic
  • Multiple Organ Failure / drug therapy
  • Multiple Organ Failure / etiology
  • Multiple Organ Failure / mortality
  • Odds Ratio
  • Polydeoxyribonucleotides / adverse effects
  • Polydeoxyribonucleotides / therapeutic use*
  • Renal Dialysis*
  • Respiration Disorders / etiology
  • Respiration Disorders / mortality
  • Respiration Disorders / therapy
  • Respiration, Artificial*
  • Retrospective Studies
  • Transplantation Conditioning / adverse effects*
  • Young Adult

Substances

  • Fibrinolytic Agents
  • Polydeoxyribonucleotides
  • defibrotide