Inborn Errors Working Party (IEWP)
IEWP Newsletter December 2019
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by Arjan Lankester
Dear colleagues and friends,
In 2019, the IEWP has continued and further expanded its activities in the field of stem cell therapies for inherited disorders of immunity both within ESID and EBMT.
During this years’ ESID focus meeting in Brussels on Malignancy in PID, a very well attended IEWP session was fully dedicated to optimizing the use of registries for PID with presentations on the value of clinical score in PID (M Seidel), registration of malignancies in PID (F Porta) and a novel initiative for registry of XIAP patients (S Burns).
On October 11-13, the Inborn Errors Working Party Annual Conference took place in London (host: Paul Veys), and special attention was given to the 40th anniversary of the stem cell therapy program in Great Ormond Street Children’s Hospital. On the first educational day, a series of international experts presented excellent overviews on developments in the field of newborn screening for SCID, state-of-the art in SCT and gene therapy in SCID and non-SCID PID, developments in gene editing, and novel treatment modalities in primary HLH.
The second and third day included high-quality and interactive sessions on the evolving position of cellular therapy in treatment of immune regulatory disorders, challenges in AT and VEO-IBD, recent advances in the field of individualized conditioning and reports on ongoing IEWP studies. Cutting-edge presentations were given on thymic development (G Hollander) and the keynote lecture on RAG deficiency (L Notarangelo).
The meeting was attended by many international colleagues reflecting both the excellent quality of the meeting as well as the strong international collaboration in the field of stem cell therapy for patients with these rare inherited disorders.
In 2019, fifteen excellent manuscripts were published on behalf of IEWP, including a series bundled as Golden Research Topic in Frontiers in Pediatrics on HSCT for Primary Immunodeficiencies and Rare Metabolic Diseases.
A number of new studies is still recruiting including HSCT outcome in hypomorphic RAG deficiency, IFNgR deficiency and AYA-PID, as well as studies on CD27/70 deficiency and ICF syndrome. You are all invited to participate in these studies and contribute with your patients. For 2020, IEWP has the aim to further extend its study activities, retrospective, prospective as well as biological, in collaboration with other WPs, SCETIDE, PIDTC and other international partners.
I look forward continuing our collaborations and wish you all a successful 2020.
Inborn Errors Working Party