Title :

The PID Odyssey 2030: outlooks, unmet needs, hurdles, and opportunities - proceedings from the IPOPI global multi-stakeholders' summit (June 2022).

Author(s) :

Tadros, Susan [Auteur]
Royal Free London NHS Foundation Trust
Prévot, Johan [Auteur]
Meyts, Isabelle [Auteur]
Catholic University of Leuven = Katholieke Universiteit Leuven [KU Leuven]
University Hospitals Leuven [Leuven]
Sánchez-Ramón, Silvia [Auteur]
Instituto de Investigación Sanitaria del Hospital Clínico San Carlos [Madrid, Spain] [IdISSC]
Erwa, Nahla H. [Auteur]
University of Khartoum
Fischer, Alain [Auteur]
Centre de Référence Déficits Immunitaires Héréditaires [CEREDIH]
Collège de France [CdF (institution)]
Hôpital Necker - Enfants Malades [AP-HP]
Imagine - Institut des maladies génétiques (IHU) [Imagine - U1163]
Lefevre, Guillaume [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Hotchko, Matthew [Auteur]
Jaworski, Peter M. [Auteur]
Georgetown University [Washington] [GU]
Leavis, Helen [Auteur]
University Medical Center [Utrecht] [UMCU]
Boersma, Cornelis [Auteur]
University of Groningen [Groningen]
Open University of the Netherlands [Heerlen]
Drabwell, Jose [Auteur]
Van Hagen, P. Martin [Auteur]
Erasmus University Medical Center [Rotterdam] [Erasmus MC]
Van Coillie, Samya [Auteur]
Pergent, Martine [Auteur]
Burns, Siobhan O. [Auteur]
Royal Free London NHS Foundation Trust
Mahlaoui, Nizar [Auteur]
Centre de Référence Déficits Immunitaires Héréditaires [CEREDIH]
Hôpital Necker - Enfants Malades [AP-HP]

Journal title :

Frontiers in Immunology

Abbreviated title :

Front Immunol

Volume number :

14

Pages :

1245718

Publication date :

2023-09-08

ISSN :

1664-3224

English keyword(s) :

primary immunodeficiencies/inborn errors of immunity
immunoglobulin replacement therapy/plasma derived medicinal products
hematopoietic stem cell transplantation
gene therapy
newborn screening
next generation sequencing
quality of life
targeted therapies/personalized medicine

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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 ...
Show more >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.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-01-11T22:16:25Z
2024-03-08T10:44:38Z