Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation.
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TLDR
Defibrotide appears to act through multiple mechanisms to restore thrombo-fibrinolytic balance and protect endothelial cells, and there are promising data on the use of defibrotides for VOD/SOS prophylaxis in high-risk children undergoing HSCT.Abstract:
Introduction: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a complication that is typically associated with conditioning for hematopoietic stem cell transplantation (HSCT). In patients with concomitant multi-organ dysfunction, mortality may be >80%. Recently, the European Society for Blood and Marrow Transplantation established separate criteria for diagnosis and severity of VOD/SOS for adults and children, to better reflect current understanding of the disease.Areas covered: This review provides an overview of post-HSCT hepatic VOD/SOS and defibrotide, including its pharmacological, clinical, and regulatory profile. In children and adults following HSCT, defibrotide is approved for the treatment of hepatic VOD/SOS with concomitant renal or pulmonary dysfunction in the United States and for the treatment of severe hepatic VOD/SOS in the European Union. Day +100 survival rates with defibrotide are superior to those of historical controls receiving best supportive care...read more
Citations
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Journal ArticleDOI
Risk Factors for Development of and Progression of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome.
TL;DR: The latest studies reporting VOD/SOS risk factors support previously published data, although the importance of patient-related factors, such as acute kidney injury, increased international normalized ratio, female sex (in children), and platelet refractoriness, is given greater emphasis in the recent data.
Journal ArticleDOI
Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).
TL;DR: This analysis provides the largest assessment of survival in patients treated with defibrotide for VOD/SOS with or without MOD, and shows survival was 41% and 70%, respectively, for the population of patients receiving any dose of defib rotide.
Journal ArticleDOI
Liver Stiffness Measurement Allows Early Diagnosis of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome in Adult Patients Who Undergo Hematopoietic Stem Cell Transplantation: Results from a Monocentric Prospective Study
Antonio Colecchia,Antonio Colecchia,Federico Ravaioli,Mariarosaria Sessa,Vanessa Luigina Alemanni,Elton Dajti,Giovanni Marasco,Amanda Vestito,Rocco Maurizio Zagari,Francesco Barbato,Mario Arpinati,Michele Cavo,Davide Festi,Francesca Bonifazi +13 more
TL;DR: LSM by TE can be considered a promising method to perform an early, preclinical diagnosis and follow-up of VOD/SOS, and Interestingly, LSM values did not increase significantly in patients experiencing hepatobiliary complications (according to the Common Terminology Criteria) other than VOD /SOS.
Journal ArticleDOI
Early Clinical Predictors of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Myeloablative Stem Cell Transplantation.
Lindsey E. Roeker,Haesook T. Kim,Brett Glotzbecker,Prashant Nageshwar,Sarah Nikiforow,John Koreth,Philippe Armand,Corey Cutler,Edwin P. Alyea,Joseph H. Antin,Paul G. Richardson,Robert J. Soiffer,Vincent T. Ho +12 more
TL;DR: Acute renal dysfunction, platelet refractoriness, and elevated or abnormal tacrolimus levels are dynamic clinical markers that should alert clinicians to the development of VOD/SOS before the presence of classical diagnostic criteria.
Journal ArticleDOI
Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease after Autologous or Allogeneic Hematopoietic Stem Cell Transplantation in Children: a retrospective study of the Italian Hematology-Oncology Association-Hematopoietic Stem Cell Transplantation Group
Maura Faraci,Alice Bertaina,Roberto Luksch,Elisabetta Calore,Edoardo Lanino,Francesco Saglio,Arcangelo Prete,Mariacristina Menconi,Giusy De Simone,Veronica Tintori,Simone Cesaro,Stella Santarone,Maria Grazia Orofino,Franco Locatelli,Marco Zecca +14 more
TL;DR: Based on the application of new EBMT criteria, the overall incidence of SOS/VOD recorded in this large Italian pediatric retrospective study was 2%, and nonrelapse mortality was significantly higher in patients who developed SOS/ VOD.
References
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Journal ArticleDOI
Veno-occlusive Disease of the Liver and Multiorgan Failure after Bone Marrow Transplantation: A Cohort Study of 355 Patients
George B. McDonald,Mary S. Hinds,Lloyd D. Fisher,Howard G. Schoch,John L. Wolford,Banaji M,Barbara J. Hardin,Howard M. Shulman,Reginald A. Clift +8 more
TL;DR: The clinical impression is that the current incidence of VOD at the institution is much higher than the 21% rate reported 9 years ago and that more patients have severe liver disease, which may explain the apparent increased incidence and severity of this complication.
Journal ArticleDOI
Venoocclusive disease of the liver following bone marrow transplantation
Richard J. Jones,Kamthorn S. K. Lee,William E. Beschorner,Victor G. Vogel,Louise B. Grochow,Hayden G. Braine,Georgia B. Vogelsang,Lyle L. Sensenbrenner,George W. Santos,Rein Saral +9 more
TL;DR: VOD is a common complication of bone marrow transplantation and has a specific clinical presentation, which usually allows diagnosis without the need of liver biopsy, and is the third leading cause of death in allogeneic graft recipients, and the second leading cause in patients receiving autologous transplants.
Journal ArticleDOI
Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome.
Jason Coppell,Paul G. Richardson,Robert J. Soiffer,Paul L. Martin,Nancy A. Kernan,Allen R. Chen,Eva C. Guinan,Georgia B. Vogelsang,Amrita Krishnan,Sergio Giralt,Carolyn Revta,Nicole A. Carreau,Massimo Iacobelli,Enric Carreras,Tapani Ruutu,Tiziano Barbui,Joseph H. Antin,Dietger Niederwieser +17 more
TL;DR: The evolution of MOF in the setting of VOD after SCT can be considered a reliable indication of severity and a predictor of poor outcome, and the methods used to evaluate severity are discussed.
Journal ArticleDOI
The syndrome of hepatic veno-occlusive disease after marrow transplantation.
TL;DR: Therapeutic drug monitoring of busulfan and pharmacokinetic dose adjustments appear to be useful in reducing the incidence of VOD in patients receiving this agent and treatment with recombinant tissue plasminogen activator has promise.
Journal ArticleDOI
Revised diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: a new classification from the European Society for Blood and Marrow Transplantation
M. Mohty,Florent Malard,M. Abecassis,Erik Aerts,Ahmed Alaskar,M Aljurf,Mutlu Arat,Peter Bader,Frédéric Baron,Ali Bazarbachi,Didier Blaise,Fabio Ciceri,Selim Corbacioglu,J.-H. Dalle,Fiona L Dignan,Takahiro Fukuda,Anne Huynh,Tamás Masszi,Mauricette Michallet,Arnon Nagler,Mairead NiChonghaile,S. Okamoto,A Pagliuca,C Peters,Finn Bo Petersen,Paul G. Richardson,Tapani Ruutu,Bipin N. Savani,E. Wallhult,Ibrahim Yakoub-Agha,R. F. Duarte,Enric Carreras +31 more
TL;DR: A new definition for diagnosis, and a severity-grading system for SOS/VOD in adult patients, are proposed on behalf of the European Society for Blood and Marrow Transplantation.
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