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Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Neuroblastoma: Final Data from the Defibrotide Expanded-Access Program (T-IND)

Year: 2018
Type: article
Abstract: Background: VOD/SOS is a life-threatening complication of HSCT conditioning. VOD/SOS with multi-organ dysfunction (MOD) may be associated with >80% mortality. Defibrotide is approved to treat hepatic VOD/SOS with renal/pulmonary dysfunction post-HSCT in the US and severe hepatic VOD/SOS post-HSCT patients (pts) aged >1 month in the EU. There are few published data on survival of neuroblastoma pts with VOD/SOS post-HSCT. We report Day +100 survival and safety post hoc for the neuroblastoma post-HSCT subgroup in the T-IND.
DOI: 10.1016/j.bbmt.2017.12.070
Authors: Stephan A. Grupp, Araz Marachelian, Sonata Jodele, Robert Ryan, Robin Hume, William Tappe, Paul G. Richardson
Institutions: Children's Hospital of Philadelphia, Children's Hospital of Los Angeles, Jazz Pharmaceuticals (United States), Harvard University, Dana-Farber Cancer Institute, Dana-Farber/Harvard Cancer Center
Language: English
Subfield: Hematology
Field: Medicine
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2786352895
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