Jazz Pharmaceuticals to Present Data on Defibrotide, an Investigational Treatment, in Patients with Hepatic Veno-Occlusive Disease (VOD) at BMT Tandem Meetings
Data from the three defibrotide studies will be presented today in an oral abstract session at the 2015 BMT (Bone Marrow Transplantation) Tandem meetings, the combined annual meetings of the
"VOD is a potentially life-threatening complication in patients undergoing HSCT therapy, and there are currently no approved therapies for VOD in the U.S," said
The three defibrotide oral presentations will be presented today during the BMT Tandem "Scientific" meeting, Session F - Allogeneic Transplants & Supportive Care & Graft Processing in the Harbor Ballroom ABC at the
Paul G. Richardson, M.D., "Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival." Dr. Richardson and co-authors will present an NNT analysis from a Phase 3 study that formed the basis of the approval of defibrotide for the treatment of severe VOD following HSCT in the European Union. Dr. Richardson is the clinical program leader and director of clinical research at the LeBow Institute for Myeloma Therapeuticsand the Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Instituteand the RJ Corman Professor of Medicine at Harvard Medical School. Paul G. Richardson, M.D., "Updated Results from the Ongoing US Treatment IND Study Using Defibrotide for Patients with Hepatic Veno-Occlusive Disease." Researchers will present an interim analysis update from the T-IND study, which is gathering data on the safety and efficacy of defibrotide in patients with severe and non-severe VOD post-HSCT, as well as post-chemotherapy.
- Selim Corbachioglu, M.D., "Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease: An Update from the International Compassionate Use Program in 710 Patients." Dr. Corbachioglu, of the
University of Regensburg, Germany, will present the final results of a large, international defibrotide compassionate use program conducted from 1998-2009 in Europe, the U.S., Asiaand the Middle East.
Full details of the 2015 BMT Tandem meetings can be found at https://bmt.confex.com/tandem/2015/webprogram/programs.html.
Defibrotide is marketed under the name Defitelio®▼(defibrotide) in
Please consult the Defitelio Summary of Product Characteristics (
The most frequent adverse events observed during pre-marketing use were hemorrhage, hypotension and coagulopathy, as noted in the Defitelio SmPC. Please consult the Defitelio SmPC for the full list of all side effects reported with Defitelio.
▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the
An ongoing expanded access program through a Treatment IND is enrolling patients diagnosed with VOD in the U.S. Expanded access programs are part of an effort by the
VOD, also known as sinusoidal obstruction syndrome (SOS), is a complication in patients undergoing HSCT therapy. The condition is characterized by blockage in some of the small blood vessels in the liver, resulting in decreased blood flow within the liver and possibly leading to liver damage. Signs and symptoms include weight gain, jaundice and enlargement of the liver. VOD may also occur after a patient undergoes radiation therapy to the liver or receives anticancer chemotherapy before HSCT.1
In its severe form, VOD can be life-threatening and is associated with multi-organ failure, and can be fatal in over 80% of patients.2,3 Although the disease appears most often within 21 days post-HSCT, its onset can occur later in the course of the patient's recovery from HSCT, even after discharge from the hospital.2 Early clinical signs such as percent weight gain, mean total bilirubin (indicative of jaundice) and the rate of change in these measures may predict progression to severe VOD.4,5 However, other conditions may mimic VOD, making early differential diagnosis challenging, yet crucial to successful patient outcomes.6
Specific groups of HSCT patients, including children, people who have suffered a previous injury to the liver, and recipients of allogenic (i.e., from another individual) hematopoietic stem cells, are at higher risk of developing VOD.3 Current treatment approaches usually involve monitoring of clinical lab values, as well as supportive care.
Studies have reported a wide range of incidence rates for VOD in HSCT patients, from as low as 0% to as high as 62%.3 Generally, data indicate that approximately 14% of patients undergoing HSCT develop VOD.2,7,8
About Jazz Pharmaceuticals plc
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to the effectiveness of defibrotide for treating severe VOD,
2 Carreras E. EBMT-ESH Handbook 2012. Chapter 11: Early complications after HSCT.
3 Coppell JA, et al. Biol Blood Marrow Transplant. 2010;16:157–168.
4 McDonald GB,
5 Bearman SI. Blood. 1995;85(11):3005-3020.
6 Carreras E. Eur J Haematol. 2000;64(5):281-291.
7 Carreras E, et al. Blood. 1998;92(10)3599-604.
8 Corbaciouglu S, et al. Lancet. 2012;379(9823):131-9.
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