European Phase II/III Clinical Trial: Prevention of Veno-Occlusive Disease in Pediatric Patients
The Phase 2/3 European pediatric prevention trial was a prospective, multi-center (28 BMT/SCT centers in Europe and Israel), open label, randomized clinical trial to evaluate the prophylactic use of Defibrotide in patients under 18 years of age who were undergoing stem cell transplantation and were at high risk for developing hepatic Veno-Occlusive Disease (VOD).
Patients randomized in the prophylaxis arm received 25 mg/kg/day of Defibrotide in four divided doses beginning at the start of conditioning and until 30 days post transplant. Patients randomized to the control arm received no VOD prophylactic therapy. Patients were permitted to receive Defibrotide as therapy if they developed VOD. The primary endpoint of the study was the development of VOD within 30 days post stem cell transplantation (SCT), based on the modified Seattle criteria. Other endpoints were the development of severe VOD (VOD associated with MOF), Day + 100 mortality, and the incidence and severity of GVHD. A blinded independent review committee of three expert haematologists confirmed the diagnoses of VOD.
Enrolment completed with 180 patients in the treatment arm and 176 in the control arm.
Final clinical trial results for this study were presented at the American Society of Haematology Conference in New Orleand on 5-8 December 2009 and, at the European Blood and Marrow Transplantation (EBMT) 36th Annual Meeting in Vienna. March 21, 2010.