Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial
Year: 2016
Type: article
Abstract: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of stem cell transplantation (SCT). Severe cases, defined by multi-organ dysfunction (MOD), may be associated with >80% mortality. There is no FDA-approved treatment for VOD/SOS. Here we present the final analysis of day+100 survival and day+100 complete response (CR) of the pivotal phase 3 trial of DF in VOD/SOS with MOD, including additional data obtained at the request of US health authorities.
Authors: Paul G. Richardson, Nancy A. Kernan, Joel A. Brochstein, Shin Mineishi, Sally Arai, Stephan A. Grupp, Eva Guinan, Paul L. Martin, Gideon Steinbach, Amrita Krishnan, Eneida R. Nemecek, Reggie E. Duerst, Joseph H. Antin, Leslie Lehmann, Alfred P. Gillio, Rajinder Bajwa, Maja Miloslavsky, Robin Hume, Massimo Iacobelli, Bijan Nejadnik, Alison L. Hannah, Robert J. Soiffer
Institutions: Harvard University, Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center, Cohen Children's Medical Center, University of Alabama at Birmingham, Stanford Medicine, Children's Hospital of Philadelphia, Duke Medical Center, University of Washington, Fred Hutch Cancer Center, City Of Hope National Medical Center, City of Hope, Oregon Health & Science University, Doernbecher Children's Hospital, Northwestern University, Lurie Children's Hospital, Boston Children's Hospital, Hackensack University Medical Center, Nationwide Children's Hospital, Jazz Pharmaceuticals (United States), Villa Melitta, Vatican Observatory
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2337987352