Inborn Errors Working Party (IEWP)

IEWP Newsletter December 2018

by Arjan Lankester

Dear colleagues,

At the end of 2018 we can look back at a very interesting and productive year with many IEWP events (see also our previous newsletter). In September (6-7th, Barcelona), IEWP has actively participated in the EBMT International Training Course with presentations on SCT in PID (Arjan Lankester) and SCT in hemoglobinopathies (Josu de la Fuente). On September 14th (London), the 3rd thymus workshop was organized by CWP and IEWP. The meeting was attended by a group of international experts in preclinical and clinical research, and a meeting report is in preparation.

The annual IEWP meeting was hosted by the Leiden team with special attention to the 50th anniversary of the first successful SCT in a SCID patient in Europe. On the first educational day of the meeting a series of international experts presented excellent overviews on developments in the field of SCT and gene therapy in SCID and non-SCID PID, SCID newborn screening, and long term outcome after SCT. The second and third day included sessions on various aspects of stem cell therapies and SCT indications in PID, hemoglobinopathies and inherited metabolic diseases. In addition, reports on ongoing IEWP studies and new proposals were presented. The meeting was very well attended by 150 participants.

During the ESID Lisbon meeting, a very well attended IEWP breakfast session was fully dedicated to current management of CGD patients including conventional treatment (Steve Holland), SCT (Tayfun Güngör) and gene therapy (Claire Booth) followed by a panel discussion on CGD cases.

Looking forward to 2019, IEWP will continu its study activities in collaboration with SCETIDE, EBMT, PIDTC and other partners. IEWP studies on SCT outcomes in SCID, CGD and WAS are being completed. Recently, several new study have been launched. These include clinical features and SCT outcomes in patients with CD27/CD70 deficiency, patients with ICF syndrome, 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.

In 2019 IEWP will participate in and organize several events. It is a pleasure to announce that 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 before the summer.

It has been a great pleasure to work with many of you this year. I look forward to continue our current and new collaborations and hope to see you during our meetings.

I wish you all good health and a collaborative and successful 2019.

Arjan Lankester
Chair IEWP