Malignant infantile osteopetrosis is a rare hereditary disorder of osteoclast function, which can be reversed by hematopoietic stem cell transplantation (SCT).
The study noted that a high level of VOD was observed in transplanted pediatric patients, with seven patients out of 11 experiencing VOD after transplantations done consecutively between 1996 and 2001. In this group, only one patient was diagnosed with moderate VOD.
“The lack of reliable diagnostic tools to identify VOD early impedes pre-emptive therapeutic intervention. Prophylactic use of Defibrotide in a high-risk pediatric population is therefore an encouraging modality supported by earlier studies and the benign adverse event profile,” the study concluded.
“We are currently in the midst of a European phase II/III trial of Defibrotide to prevent VOD in pediatric patients and look forward to demonstrating equally compelling clinical results in this devastating disease for which there are no currently approved treatments and which, in its severe form, is most often fatal,” said Dr Laura Ferro, president and CEO of Gentium.
The study was led by the Department of Pediatrics, University of Ulm in Germany.