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Efficacy and Safety of Defibrotide in Pediatric Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results From the Expanded-Access Program (T-IND)

Year: 2018
Type: article
Abstract: Background: VOD/SOS is a complication of HSCT conditioning or chemotherapy. VOD/SOS with multi-organ dysfunction (MOD) may be associated with >80% mortality. For pediatric (PED) and adult patients, defibrotide is approved to treat hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT in the US and to treat severe hepatic VOD/SOS post-HSCT in patients aged >1 month in the EU. Reported here are outcomes from the largest PED VOD/SOS cohort treated with defibrotide.
DOI: 10.1016/j.bbmt.2017.12.334
Authors: Nancy A. Kernan, Angela R. Smith, Leslie Lehmann, Robert Ryan, Wei Liang, Robin Hume, William Tappe, Stephan A. Grupp
Institutions: Memorial Sloan Kettering Cancer Center, University of Minnesota, Harvard University, Dana-Farber Cancer Institute, Dana-Farber/Harvard Cancer Center, Jazz Pharmaceuticals (United States), Children's Hospital of Philadelphia
Language: English
Subfield: Hematology
Field: Medicine
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2793706133
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