The EBMT and Gentium s.r.l. (formerly Gentium SpA), a Jazz Pharmaceuticals Company, have developed a strong collaboration to comply with the post-authorization requirements of PRAC by setting up a disease registry to collect data in patients developing sVOD following HSCT.
This observational registry study protocol has the scope to collect patients' data on specific SAEs of interest (including fatalities) as well as endpoints in relation to clinical outcome and standard baseline information. The original registry study was aiming to stop enrolling in June 2017 and it was planned to stop the recruitment when 300 patients with severe VOD treated with Defitelio® and a minimum of 300 patients managed through supportive care (controls) were included.
- Following discussions started in 2017 with EMA, a decision to cease patient recruitment into the study was agreed by the EMA end of June 2018. This was communicated to all active sites to cease patient recruitment as of July 18th 2018.
- Currently the study is active in Italy (19 sites), France (22 sites), Portugal (1 site) and United Kingdom (11 sites) where sites are open and following up.
- In total, in August 2018, 170 enrolled patients were reported in the database so far! It is a great achievement and we wish to thank all of you for your participation and constant effort to make it possible. Please register your latest patients URGENTLY now.
- The next exportation of patient data is planned on October, 18th 2018 so be ready to complete on time your visits and queries! Thank you.