Current Transition Practice for Primary Immunodeficiencies and Autoinflammatory Diseases in Europe: a RITA-ERN Survey

Muskan Israni, Bethany Nicholson, RITA-ERN Transition Working Group Consortium, Nizar Mahlaoui, Laura Obici, Linda Rossi-Semerano, Helen Lachmann, Georgia Hayward, Mojca Zajc Avramovič, Aurelien Guffroy, Virgil Dalm, Rachel Rimmer, Leire Solis, Carlotta Villar, Andrew R. Gennery, Stephanie Skeffington, Julia Nordin, Klaus Warnatz, Anne Sophie Korganow, Jordi AntónMarco Cattalini, Tania Amin, Stephan Berg, Pere Soler-Palacin, Siobhan O. Burns*, Mari Campbell*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

1 Downloads (Pure)

Abstract

Background: Due to the absence of curative treatments for inborn errors of immunity (IEI), children born with IEI require long-term follow-up for disease manifestations and related complications that occur over the lifespan. Effective transition from pediatric to adult services is known to significantly improve adherence to treatment and long-term outcomes. It is currently not known what transition services are available for young people with IEI in Europe. Objective: To understand the prevalence and practice of transition services in Europe for young people with IEI, encompassing both primary immunodeficiencies (PID) and systemic autoinflammatory disorders (AID). Methods: A survey was generated by the European Reference Network on immunodeficiency, autoinflammatory, and autoimmune diseases Transition Working Group and electronically circulated, through professional networks, to pediatric centers across Europe looking after children with IEI. Results: Seventy-six responses were received from 52 centers, in 45 cities across 17 different countries. All services transitioned patients to adult services, mainly to specialist PID or AID centers, typically transferring up to ten patients to adult care each year. The transition process started at a median age of 16–18 years with transfer to the adult center occurring at a median age of 18–20 years. 75% of PID and 68% of AID centers held at least one joint appointment with pediatric and adult services prior to the transfer of care. Approximately 75% of PID and AID services reported having a defined transition process, but few centers reported national disease-specific transition guidelines to refer to. Conclusions: Transition services for children with IEI in Europe are available in many countries but lack standardized guidelines to promote best practice.

Original languageEnglish
Pages (from-to)206-216
Number of pages11
JournalJournal of Clinical Immunology
Volume43
Issue number1
DOIs
Publication statusE-pub ahead of print - 12 Oct 2022

Bibliographical note

Funding Information:
BN was contracted to work on this project by Royal Free Hospital and Great Ormond Street Hospital; SB has received grant support from the European Union, National Institute of Health Research, UCLH and GOSH/ICH Biomedical Research Centers, and CSL Behring and personal fees or travel expenses from Immunodeficiency Canada/IAACI, CSL Behring, Baxalta US Inc, and Biotest; MC has received grant support from the National Institute of Health Research, PIDUK, CSL Behring and the Royal Free Charity and personal fees or travel expenses from Biotest, BPL, CSL Behring, Grifols, HAEUK, Shire, and Takeda; LR-S has received consulting fees from Pfizer and AbbVie, and support for attending meetings or travel expenses from Novartis, Sobi, Medac, AbbVie, and Pfizer; ARG has received research funding from Mallinckrodt and JAZZ Pharmaceuticals; AG and ASK receive research funding from CSL Behring and Takeda; SB has received payment or honoraria for speakers bureaus from Sobi and Novartis; PSP has received grant support from the European Union, Instituto Carlos III, Grifols, and CSL Behring and personal fees or travel expenses from CSL Behring, Takeda, and Grifols; JA has received grant support from the European Union, Insituto Carlos III, Fundación Daniel Bravo, research support from Novartis, Sobi, Novimmune, Roche, Pfizer, Lilly, AbbVie, and Amgen, and personal fees or travel expenses from Novartis, Sobi, Roche, Pfizer, Lilly, AbbVie, and Gebro; MC has received speaking fees from Novartis Farma, AbbVie, Sobi, and Pfizer; KW is on the advisory boards for Shire Deutschland GmbH and LFB biomedicals, and has received grant support from BMS, honoraria for lectures, symposiums, and meetings from CSL Behring and Shire, and travel expenses from Shire; HL is the President-Elect for ISSAID, Council Member of the Nephrology section of the Royal Society of Medicine, and Vice-Chair of MHRA EAG; HL has received grant support for ImmunAID from EUH2020, consulting fees from Novartis, Sobi, and Roche, payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Novartis and Sobi, and support for attending meetings and/or travel from DADA2 patient society and Sobi; IM is the president of European Society for Immunodeficiencies, a board member of Astra Zeneca Foundation, and a senior clinical investigator at FWO Vlaanderen, IM has received grant support from CSL Behring Primary Immunodeficiencies Chair, European Research Council under the European Union’s Horizon 2020 research and innovation program, FWO Vlaanderen, VIB Grand Challenges Program, KU Leuven Onderzoeksraad, and the Jeffrey Modell Foundation, IM has received honoraria for lecturing by CSL Behring; JVDH has received consulting fees from Novartis and Sobi, and payment for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Sobi; LG is a participant on the Data Safety Monitoring board or Advisory Board for Novartis, and has received payment for presentations from Novartis, and support for attending meetings and/or travel expenses from AbbVie and Novartis; ŠF has received payment or honoraria for presentations and meeting/travel expenses from Sobi; AS has received payment or honoraria for presentations, lectures, speakers bureaus, educational events, or manuscript writing from Takeda, CSL Behring, and Octapharma; TH has received payment for lectures from Novartis, and support for attending meetings from Sobi; TRL is a participant on the advisory board for Sanofi-Pasteur, and has received support for attending meetings and/or travel from Takeda; VM is the president of the Vaccine Committee of the Italian Society of Pediatric Allergy and Immunology, and has received personal fees from Takeda and CSL Behring, and travel/meeting expenses from CSL Behring; JAMvL has received payment for expert testimony from Amgen and Sobi; NMW is the coordinator of ERN-RITA, and a participant in an investigator-initiated study on the effect of intranasal administration of palivizumab on respiratory syncytial virus-associated infection. NMW has received grant support from ZONMW and ReumaNederland, and consulting fees from Sanofi-Genzyme and UCB; FOR has received support for attending meetings and/or travel from Novartis; PČ has received support for attending meetings and/or travel from Takeda; CC is a pediatric representative on the Committee of primary Immunodeficiencies Hospital Universitario y Politecnico La Fe, and has received support for this manuscript from Hospital Universitario y Politecnico La Fe—Valencia, and support for attending meetings and/or travel expenses from CSL Behring; JS-M has received payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Pfizer, and meeting/travel expenses from AbbVie; LA has received consulting fees from CSL Behring, Grifols, Shire, and Takeda, honoraria for lectures from CSL Behring, Shire, and Takeda, and meeting/travel expenses from CSL Behring, Grifols, Shire, and Takeda; MERS participates on the Data Safety Monitoring Board or Advisory Board for Takeda has received meeting/travel expenses from CSL Behring and ALK, and payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Takeda and CSL Behring; LIG-G is a medical advisor for AEDIP and has received grant support from Fondo de Investigación Sanitaria, co-financed with FEDER funds; OE has received grant support from the Swedish Research Council; LAD has received funding to attend an educational event from CSL, and support to attend a speaker meeting for GPs from ALK; AJJW is a participant on the advisory board for Orchard Therapeutics; PB is a trustee of UK charity Societi, treasurer of International Society of Systemic Autoinflammatory Diseases, and participant on the NOVARTIS gene therapy advisory board, PB has received grant support from the European Union for an investigator-led clinical trial of Prednisolone in Kawasaki disease, consulting fees from Sobi and Novartis, payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Sobi, Roche, and Novartis, and payment for expert testimony from KD Scotland; SS is the Chair of UKPIN, and has received grant support from CSL Behring, and consulting fees from Takeda and Novartis; SF is a participant on the advisory boards for AstraZeneca, Medimmune, Sanofi, Pfizer, Seqirus, Sandoz, Merck, and J&J, and was the chair of UK NICE Sepsis (2014–2016) and Lyme Disease (2016–2018) Guidelines, SF has received the NIHR Senior Investigator Award, grant support from Pfizer, Sanofi, GSK, J&J, Merck, AstraZeneca, and Valneva, and payment for symposiums from Pfizer. No other authors have competing interests. 1 1 2 2

Funding:
This study was supported by the ERN-RITA.

Publisher Copyright:
© 2022, The Author(s).

Fingerprint

Dive into the research topics of 'Current Transition Practice for Primary Immunodeficiencies and Autoinflammatory Diseases in Europe: a RITA-ERN Survey'. Together they form a unique fingerprint.

Cite this