Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: A Multi-centre, Multinational, Prospective Observational Registry to Collect Safety and Outcome Data in Patients Diagnosed With Severe Hepatic VOD Following Hematopoietic Stem Cell Transplantation (HS
Brief Summary:
Following the licencing of a new drug, Defitelio®, indicated for the treatment of severe Veno-Occlusive Disease of the liver (sVOD), a rare but serious complication of haematopoietic stem cell transplantation (HSCT), the pharmaceutical company that manufactures Defitelio® (Gentium, a Jazz Pharmaceuticals Company) - as a specific obligation (SOB) - has been required by PRAC to set up a disease registry to collect safety and outcome data, and to assess patterns of utilization of Defitelio® in the post-approval setting. This registry is a Post Authorization Safety Study (PASS), which is being coordinated through the European Society for Blood and Marrow Transplantation (EBMT). Following appropriate consent, data from patients who undergo haematopoietic stem cell transplantation are routinely collected at the local centre for the EBMT database. For this study, the sites are being asked to collect additional anonymised clinical data from patients who develop this complication or who have been treated with Defitelio® for other reasons than sVOD. The study simply requires collection of data which is already recorded in the patient charts, but not routinely collected.
The study DOES NOT involve decisions about treatment, which are clinical decisions, but merely collection of data for patients who develop this complication, whether or not they receive treatment and for patients who are treated with Defitelio® for any other reasons.