1. A multicentre, multinational, prospective, observational registry study of defibrotide in patients diagnosed with veno-occlusive disease/sinusoidal obstruction syndrome after haematopoietic cell transplantation: an EBMT study
- Author
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Marco Zecca, Didier Blaise, Mohamad Mohty, Vian Amber, Natalia Maximova, Raj Hanvesakul, Simone Cesaro, Fabio Ciceri, Robert J. Ryan, Elisabetta Calore, Myriam Labopin, Sarah Lawson, Robert Wynn, Marta Lisa Battista, Katia Perruccio, Cecile Renard, Centre de Recherche en Cancérologie de Marseille (CRCM), Aix Marseille Université (AMU)-Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Mohty, M., Battista, M. L., Blaise, D., Calore, E., Cesaro, S., Maximova, N., Perruccio, K., Renard, C., Wynn, R., Zecca, M., Labopin, M., Hanvesakul, R., Amber, V., Ryan, R. J., Lawson, S., and Ciceri, F.
- Subjects
medicine.medical_specialty ,Hepatic Veno-Occlusive Disease ,[SDV.CAN]Life Sciences [q-bio]/Cancer ,Defibrotide ,03 medical and health sciences ,Polydeoxyribonucleotides ,0302 clinical medicine ,Internal medicine ,medicine ,Clinical endpoint ,Humans ,Prospective Studies ,Registries ,Adverse effect ,Survival rate ,ComputingMilieux_MISCELLANEOUS ,Transplantation ,business.industry ,Incidence (epidemiology) ,Hematopoietic Stem Cell Transplantation ,Hematology ,030220 oncology & carcinogenesis ,bacteria ,Population study ,Observational study ,business ,Complication ,030215 immunology ,medicine.drug - Abstract
Severe hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic cell transplantation (HCT). This multinational, prospective, observational study (NCT03032016), performed by the EBMT, enrolled patients treated with defibrotide from April 2015 to July 2018. This analysis focused on defibrotide-treated patients with VOD/SOS post-HCT. The primary endpoint was incidence of serious adverse events (SAEs) of interest up to 12 months post-HCT in patients with severe VOD/SOS. Overall, 104 defibrotide-treated patients with VOD/SOS post-HCT were enrolled: 62 had severe VOD/SOS and comprised the primary study population, including 36 with multi-organ dysfunction/failure (MOD/MOF). SAEs of interest occurred in 20 of 62 (32%) severe VOD/SOS patients; the most common by category were infection (24%) and bleeding (13%). In patients with severe VOD/SOS, the Kaplan–Meier–estimated Day 100 survival rate was 73% (95% CI: 60%, 82%) with VOD/SOS resolution by Day 100 in 45 of 62 (73%) patients. MOD/MOF resolved in 19 of 36 (53%) patients with MOD/MOF at VOD/SOS diagnosis. Results from this multicentre registry study build on prior defibrotide studies supporting the utility of defibrotide for the treatment of VOD/SOS post-HCT. These results provide additional real-world evidence of the effectiveness and safety of defibrotide in patients with VOD/SOS post-HCT.
- Published
- 2021