Inborn Errors Working Party (IEWP)
IEWP Newsletter April 2019
by Arjan Lankester
Last month many of us have met during the very well attended Inborn Errors WP sessions at the annual EBMT meeting in Frankfurt. The excellent presentations covered a broad spectrum of both ‘state-of-the art’ and innovative treatment modalities on stem cell transplantation and gene therapy strategies for inherited immune disorders.
In 2019, IEWP will continu its study activities in collaboration with SCETIDE, EBMT, PIDTC and other partners. Several studies are still recruiting. These include: clinical features and SCT outcomes in patients with CD27/CD70 deficiency, clinical features and SCT outcomes in patients with ICF syndrome, SCT outcomes in patients with hypomorphic RAG deficiency, and SCT outcome in IL7receptor deficient SCID. In case you would like to contribute to these studies with your patients and have not been approached or in contact yet, please contact me and I will connect you to the respective study coördinators.
Finally, I would like to announce our annual IEWP autumn meeting which will take place October 11-13, in London (local host: Paul Veys). The program will include an educational part on the first day. Further details on the program will be made available soon.
I look forward to continue our collaborations and hope to meet you during our next meetings.