Inborn Errors Working Party (IEWP)

IEWP Newsletter November 2020

by Michael Albert

Dear colleagues and friends,

I hope all of you stay safe and healthy during this second wave of the Coronavirus pandemic.

Thank you for electing me to be the chair of the IEWP for the upcoming four years. I really appreciate your trust and support.

First, let me report on another study on behalf of IEWP that has just been published. With a reported overall survival of 100% in children with familial HLH after HSCT, it again expands our horizon on what we can achieve for children with severe inborn errors of immunity:

Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Felber et al, on behalf of the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT) and the European Society of Immunodeficiencies (ESID). Blood Adv (2020) 4 (9): 1998–2010.

I would especially like to thank all of you for your supporting this true team effort with your contributions. This study also nicely highlights the impact of the soon-to-be-updated IEWP guidelines for HSCT in PID.

During the postponed and virtual annual EBMT meeting, held from Aug 30th to Sep 1st, IEWP had two well attended sessions with lively Q&A sessions.

Unfortunately, also the annual IEWP fall meeting had to be cancelled. Instead we held an “extended IEWP business meeting” on September 25th with two scientific presentations by the Milan team followed by a business meeting in which we updated everybody on IEWP activities, studies, study proposals etc. In 2021 we intend to meet in person in Milano.

Finally, we all just met online at the ESID biennial meeting, which was supposed to take place in Birmingham. We had a nicely interactive scientific IEWP session on Thursday and were involved in an educational session on “Gene Therapy and Treatment in “New” or Unknown PID” on Wednesday as well as a debate session on “HSCT or not in adult PID” on Thursday.

Looking ahead to the new year, we will again have an all-virtual EBMT meeting in March. We still hope that in-person scientific exchange will again be possible sometime next year.

Best wishes

Michael Albert, chair IEWP