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Report of the Inborn Errors Working Party Spring School on Cellular Therapy for Inborn Errors of Immunity and Hematopoiesis 2023

Inborn Errors Working Party (IEWP)

Summary written by Michael Albert, Secretary IEWP

The first IEWP Spring School Meeting 2023 was held on the island of Frauenchiemsee, Germany, from April 2nd to April 5th. The Spring School was an educational event aimed at providing in-depth knowledge and understanding of hematopoietic stem cell transplantation (HSCT) for immune dysregulation and immunodeficiency disorders. The meeting brought together 23 participants from different parts of the world who had a keen interest in learning about the latest developments in this field. The program was designed to cover a wide range of topics related to HSCT for IEI, with presentations by seven faculty members (Benedicte Neven, Emma Morris, Arjan Lankester, Manfred Hönig, Carsten Speckmann, Andrew Gennery and Michael Albert).

The meeting commenced with a presentation on the fundamental principles of Hematopoietic Stem Cell Transplantation (HSCT) for Immunodeficiency, followed by a thorough discussion of the indications for HSCT. Subsequently, the attendees deliberated on the optimal timing for performing a transplant, and then focused on exploring various sources of grafts and selecting donors. The meeting featured a presentation on selecting an appropriate conditioning regimen and bridging strategies to HSCT. Attendees were also informed about HSCT for SCID, Chronic Granulomatous Disease (CGD), Combined Immunodeficiencies (CIDs), and Hemophagocytic Lymphohistiocytosis (HLH). The discussion extended to include HSCT for DNA breakage disorders, as well as haploidentical HSCT with PTCY or TCR alpha/beta depletion. Furthermore, the meeting covered the short to medium-term follow-up post-HSCT, which included immune reconstitution and vaccinations. The session also included a presentation on HSCT for adults with IEI. The attendees engaged in a comprehensive discussion on managing infections before, during, and after HSCT. Gene therapy for Immunodeficiency, as well as the use of T-cell depleting serotherapy, were also presented and discussed. The meeting concluded with two presentations - one on long-term follow-up, which included disease and fertility, and the other on secondary procedures. In addition to the formal presentations, the program included case studies presented by each participant, which were thoroughly discussed.

In conclusion, the IEWP Spring School 2023 was a successful educational event that provided a comprehensive overview of HSCT for immune dysregulation and immunodeficiency disorders. The presentations were informative and allowed for extensive interactive discussion and networking, providing participants with a deeper understanding of the complex issues involved in HSCT for IEI. Overall, the meeting was a great opportunity for the participants to learn from each other and the experts and to enhance their knowledge and skills.

IEWP23 Group Photo