Defibrotide – pro
Defibrotide (Defitelio, Gentium) is a deoxyribonucleic acid derivative (single-stranded) derived from cow lung or porcine mucosa. It is an anticoagulant with a multiple mode of action. In April 2016 the US FDA approved it for the treatment of hepatic VOD after hematopoietic stem cell transplantation (HSCT). DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
This drug was approved without evidence from randomized controlled trials. it may be reasonable to restrict its sue for Venous Occlusive Disease after stem cell transplantation only and not from other organ transplantation, subject to existing off-label policies.
P. Rishardson, Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease. Ther Adv Hematol. 2012 August; 3(4): 253–265.
Defitelio, Prescribing Information 2017
Paul G. Richardson et al, Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease and Multiorgan Failure after Stem Cell Transplantation: A Multicenter, Randomized, Dose-Finding Trial. Biol Blood Marrow Transplant. 2010 July; 16(7): 1005–1017.