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.
Bibliographical noteFunding Information:
All authors contributed to the acquisition, analysis or interpretation of the data, provided critical review of the manuscript, and gave final approval of the manuscript. This analysis was funded by Jazz Pharmaceuticals. Medical writing and editorial support were provided by William Perlman, PhD, and Erica Chevalier‐Larsen, PhD, of SciFluent Communications, and were financially supported by Jazz Pharmaceuticals.
Paul G. Richardson has served on advisory committees for Jazz Pharmaceuticals. Nancy A. Kernan received grants from Gentium during the conduct of the study, and her research was supported by the National Cancer Institute of the National Institutes of Health under award number P30 CA008748; the content is solely the responsibility of the author and does not necessarily represent the official views of the National Institutes of Health. Robert J. Soiffer has consulted for Celgene, Gilead, Jazz Pharmaceuticals, Mana Therapeutics, and Neovii; he has served on the data and safety advisory board for Juno and is on the board of directors for Kiadis. William Tappe is a former employee of Jazz Pharmaceuticals and holds stock ownership in Jazz Pharmaceuticals. Robert J. Ryan is an employee of and holds stock ownership in Jazz Pharmaceuticals. Stephan Grupp receives study support from Novartis, Kite, and Servier; he has consulted for Novartis, Roche, GSK, Cure Genetics, Humanigen, and CBMG and is on study steering committees or scientific advisory boards for Jazz, Adaptimmune, TCR2, Eureka, Cellectis, Juno, and Vertex; he holds a patent (Toxicity management for anti‐tumor activity of CARs, WO 2014011984 A1) that is managed according to the University of Pennsylvania patent policy. Angela R. Smith and Leslie Lehmann have nothing to disclose.
© 2020 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd
- multi-organ dysfunction
- veno-occlusive disease/sinusoidal obstruction syndrome