TY - JOUR
T1 - The PID Odyssey 2030
T2 - outlooks, unmet needs, hurdles, and opportunities — proceedings from the IPOPI global multi-stakeholders’ summit (June 2022)
AU - Tadros, Susan
AU - Prévot, Johan
AU - Meyts, Isabelle
AU - Sánchez-Ramón, Silvia
AU - Erwa, Nahla H.
AU - Fischer, Alain
AU - Lefevre, Guillaume
AU - Hotchko, Matthew
AU - Jaworski, Peter M.
AU - Leavis, Helen
AU - Boersma, Cornelis
AU - Drabwell, Jose
AU - van Hagen, Martin
AU - Van Coillie, Samya
AU - Pergent, Martine
AU - Burns, Siobhan O.
AU - Mahlaoui, Nizar
N1 - Publisher Copyright:
Copyright © 2023 Tadros, Prévot, Meyts, Sánchez-Ramón, Erwa, Fischer, Lefevre, Hotchko, Jaworski, Leavis, Boersma, Drabwell, van Hagen, Van Coillie, Pergent, Burns and Mahlaoui.
PY - 2023/8/15
Y1 - 2023/8/15
N2 - IPOPI held its first Global Multi-Stakeholders’ Summit on 23-24 June 2022 in Cascais, Portugal. This IPOPI initiative was designed to set the stage for a stimulating forward-thinking meeting and brainstorming discussion among stakeholders on the future priorities of the PID community. All participants were actively engaged in the entire Summit, bringing provocative questions to ensure a high level of discussion and engagement, and partnered in identifying the outlooks, unmet needs, hurdles and opportunities of PIDs for 2030. The topics that were covered include diagnosis (e.g., newborn screening [NBS], genomic sequencing— including ethical aspects on the application of genomics on NBS, the role of more accurate and timely diagnostics in impacting personalized management), treatment (e.g., the therapeutic evolution of immunoglobulins in a global environment, new therapies such as targeted therapies, new approaches in curative therapies), the interactions of Primary ID with Secondary ID, Autoinflammatory Diseases and other diseases as the field experiences an incessant evolution, and also the avenues for research in the field of humanities and human sciences such as Patient-Reported Outcome Measures (PROMs), Patient-Reported Experience Measures (PREMs), and Health-Related Quality Of Life (HRQoL). During this meeting, all participants contributed to the drafting of recommendations based on our common understanding of the future opportunities, challenges, and scenarios. As a collection of materials, perspectives and summaries, they are succinct and impactful and may help determine some of the next key steps for the PID community.
AB - IPOPI held its first Global Multi-Stakeholders’ Summit on 23-24 June 2022 in Cascais, Portugal. This IPOPI initiative was designed to set the stage for a stimulating forward-thinking meeting and brainstorming discussion among stakeholders on the future priorities of the PID community. All participants were actively engaged in the entire Summit, bringing provocative questions to ensure a high level of discussion and engagement, and partnered in identifying the outlooks, unmet needs, hurdles and opportunities of PIDs for 2030. The topics that were covered include diagnosis (e.g., newborn screening [NBS], genomic sequencing— including ethical aspects on the application of genomics on NBS, the role of more accurate and timely diagnostics in impacting personalized management), treatment (e.g., the therapeutic evolution of immunoglobulins in a global environment, new therapies such as targeted therapies, new approaches in curative therapies), the interactions of Primary ID with Secondary ID, Autoinflammatory Diseases and other diseases as the field experiences an incessant evolution, and also the avenues for research in the field of humanities and human sciences such as Patient-Reported Outcome Measures (PROMs), Patient-Reported Experience Measures (PREMs), and Health-Related Quality Of Life (HRQoL). During this meeting, all participants contributed to the drafting of recommendations based on our common understanding of the future opportunities, challenges, and scenarios. As a collection of materials, perspectives and summaries, they are succinct and impactful and may help determine some of the next key steps for the PID community.
KW - gene therapy
KW - hematopoietic stem cell transplantation
KW - immunoglobulin replacement therapy/plasma derived medicinal products
KW - newborn screening
KW - next generation sequencing
KW - primary immunodeficiencies/inborn errors of immunity
KW - quality of life
KW - targeted therapies/personalized medicine
UR - http://www.scopus.com/inward/record.url?scp=85169329593&partnerID=8YFLogxK
U2 - 10.3389/fimmu.2023.1245718
DO - 10.3389/fimmu.2023.1245718
M3 - Article
C2 - 37654496
AN - SCOPUS:85169329593
SN - 1664-3224
VL - 14
JO - Frontiers in Immunology
JF - Frontiers in Immunology
M1 - 1245718
ER -