Healthcare Industry News: liver failure
News Release - July 11, 2006
Gentium Announces Publication of Two Independent Pediatric Studies of DefibrotideVILLA GUARDIA (COMO), Italy--(HSMN NewsFeed)--July 11, 2006--Gentium S.p.A. (Nasdaq:GENT ; the Company) today announced the publication of two independent studies of the company's lead product candidate, Defibrotide, for the treatment of veno-occlusive disease (VOD) in children, in two separate peer-reviewed journals.
One study, led by Ursula Haussmann of the Division of Immunology/ Hematology/BMT, University Children's Hospital, Zurich, Switzerland; Growth and Development Centre, University Children's Hospital, Zurich, Switzerland, entitled, "Hepatic veno-occlusive disease in pediatric stem cell transplantation: impact of pre-emptive anti-thrombin III replacement and combined anti-thrombin III/Defibrotide therapy," was published in the peer-reviewed journal, "Haematologica/The Hematology Journal 2006."
This prospective case study comprised two phases. In the first phase, 71 children did not receive any specific VOD prophylaxis or therapy (controls). In the second phase, 91 children were given pre-emptive anti-thrombin III (ATIII) replacement in case of decreased ATIII activity. If VOD was diagnosed clinically (according to modified Seattle criteria), high dose Defibrotide (60 mg/day) and ATIII replacement therapy were combined. The severity of VOD was determined according to the degree of multiple organ dysfunction. The incidence of VOD was similar in both groups -- 13 out of 71 patients or 18% versus 14 out of 91 patients or 15%. All 14 patients in the second group who developed VOD showed decreased ATIII activity not more than 1 day prior to the clinical diagnosis of VOD. The resulting short duration of pre-emptive ATIII therapy failed to prevent VOD and none of the patients (n=72) maintaining normal ATIII levels developed VOD. All 14 patients with VOD who received combined Defibrotide and ATIII therapy achieved complete remission and 93% (13 out of 14 patients) survived until day +100, compared with only six survivors (46%) in the first group. The study showed that "pre-emptive ATIII administration did not alter the incidence of VOD, whereas combination treatment with ATIII and Defibrotide was safe and yielded excellent remission and survival rates."
"Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease in Children," was published in the peer-reviewed journal, Pediatric Blood Cancer. The study, led by Sean R. Bulley, M.D., MSc, ABIM, FRCPC, Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada, is a retrospective analysis of the use of defibrotide in children with VOD following hematopoietic progenitor cell transplant (HPCT). Fourteen children who underwent HPCT during the study period received Defibrotide for the treatment of VOD. Defibrotide was started on transplant day, with a median initial Defibrotide dose of 33 mg/kg/day. The median duration of Defibrotide therapy was 16 days. The survival rate for patients treated with Defibrotide to day +100 was 79%. The study concluded that Defibrotide appeared to be an effective and relatively safe treatment for children with HVOD.
Commenting on the two publications, Dr. Laura Ferro, chairman and chief executive officer of Gentium, said, "These published results are very encouraging and underscore the potential of Defibrotide in treating pediatric patients with VOD. We are currently conducting a 270-patient Phase II/III trial to prevent VOD in pediatric patients in Europe, and hope to demonstrate compelling clinical results in preventing this devastating disease among these very sick pediatric patients."
Veno-occlusive disease (VOD) is a potentially life-threatening condition. Certain high dose chemotherapy and radiation therapies and stem cell transplantation (SCT) can damage cells of the blood vessels and result in VOD, a blockage of the small veins of the liver that can lead to liver failure and the failure of other organs (Severe VOD). SCT is a frequently used treatment following high dose chemotherapy and radiation therapy. The International Bone Marrow Transplant Registry estimated that approximately 45,000 people received blood and bone marrow transplants, which are types of SCT, in 2002. Based on the Company's review of more than 200 published papers, it believes that approximately 20% of patients who undergo SCT develop VOD, approximately one-third of those who develop VOD progress to Severe VOD, and approximately 80% of Severe VOD patients die within 100 days of the SCT. The Company believes that there are no approved therapies to treat or prevent VOD in the U.S. or the EU.
Gentium S.p.A. is a biopharmaceutical company located in Villa Guardia (Como), Italy that is focused on the research, discovery and development of drugs derived from DNA extracted from natural sources, and drugs that are synthetic derivatives, to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate in the U.S., is an investigational drug that has been granted Orphan Drug status by the U.S. FDA to treat Severe VOD and Fast Track designation for the treatment of Severe VOD in recipients of stem cell transplants.
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