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.
Paul Gerard RichardsonAngela R SmithNancy A KernanLeslie LehmannRobert J SoifferRobert J RyanWilliam TappeStephan GruppPublished in: British journal of haematology (2020)
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.
Keyphrases
- end stage renal disease
- chronic kidney disease
- cell therapy
- peritoneal dialysis
- extracorporeal membrane oxygenation
- newly diagnosed
- early onset
- single cell
- acute respiratory distress syndrome
- free survival
- oxidative stress
- mechanical ventilation
- prognostic factors
- cardiovascular events
- randomized controlled trial
- intensive care unit
- stem cells
- coronary artery disease
- bone marrow
- clinical trial
- cell proliferation
- mesenchymal stem cells
- study protocol
- open label