Current Transition Practice for Primary Immunodeficiencies and Autoinflammatory Diseases in Europe: a RITA-ERN Survey
RITA-ERN Transition Working Group Consortium
(2023) Journal of Clinical Immunology, volume 43, issue 1, pp. 206 - 216
(Article)
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.
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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.
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Keywords: Autoinflammatory diseases, Network, Primary immunodeficiencies, Transition, Immunology and Allergy, Immunology
ISSN: 0271-9142
Publisher: Springer New York
Note: Funding Information: This study was supported by the ERN-RITA. 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 Information: RITA-ERN Transition Working Group Consortium Collaborators Wouters, C. MD PhD30,31, Meyts, I. MD PhD30,31, van der Werff ten Bosch, J. E. MD, PhD32, Goffin, L. MD33, Ogunjimi, B. MD PhD34-36, Gilliaux, O. MD37,38, Kelecic, J. MD39, Jelusic, M. MD PhD40, Fingerhutová, Š. MUDr41, Sediva, A. MD PhD42, Herlin, T. MD PhD43, Seppänen Mikko R. J. MD PhD44, Aalto, K. MD PhD45, Ritterbusch, H. RN19, Insalaco, A. MD46, Moschese, V. MD PhD47, Plebani, A. Professor of Pediatrics MD PhD23, Cimaz. R. MD48, Canessa, C. MD49, Dellepiane, R. M. MD50, Carrabba, M. MD PhD51, Barzaghi, F. MD52, Tommasini, A. MD PhD53, van Laar, J.A.M. MD PhD11, Wulffraat, N. M. Prof MD PhD54, Marques L. MD55, Carreras, C. MD56, Sánchez-Manubens J. MD PhD57, Alsina, L. MD PhD58,59, Seoane Reula, M. E. MD PhD60, Mendez-Echevarria, A. MD PhD61,62, Gonzales-Granado, L. I. MD63-65, Santamaria M. MD PhD66, Neth, O. MD PhD67, Ekwall, O. MD PhD25,68, Brodszki, N. MD PhD69, Hague, R. MD70, Devlin, L. A. MB BChBAO MRCPCH MD FRCPath71, Brogan, P. MBChB PhD72, Arkwright, P.D. MB PhD73, Riordan, A. MD74, McCann, L. MRCPCH MMedSc74, McDermott, E. FRCP, FRCPath, DM75, Faust, S. N. PhD76,77, Carne, E. MSc7830Department of Microbiology and Immunology, Pediatric Immunology, KU Leuven - University of Leuven, B-3000, Leuven, Belgium31Department of Pediatrics, Division Pediatric Rheumatology, University Hospitals Leuven, B-3000, Leuven, Belgium32Department of Pediatrics UZ Brussel, Brussels, Belgium33Pediatric Rheumatology Department, Hôpital Universitaire des Enfants Reine Fabiola (HUDERF) – Université Libre de Bruxelles, Brussels, Belgium34Division of Paediatric Rheumatology, Department of Paediatrics, Antwerp University Hospital, Antwerp, Belgium35Division of Paediatric Rheumatology, Department of Rheumatology, Ziekenhuis Netwerk Antwerpen, Antwerp, Belgium35Division of Paediatric Rheumatology, Department of Paediatrics, Universitair Ziekenhuis Brussel, Jette, Belgium36Centre for Health Economics Research and Modeling Infectious Diseases, University of Antwerp, Antwerp, Belgium37Pediatric department, CHU of Charleroi (Hôpital civil Marie Curie), Charleroi, Belgium38Laboratory of experimental medicine (ULB222), Medicine faculty, Université libre de Bruxelles, ISPPC, CHU of Charleroi, Charleroi (Lodelinsart), Belgium.39University Hospital Center Zagreb, Department of Pediatrics, Division of Clinical Immunology, Allergology, Respiratory Diseases and Rheumatology, Kišpatićeva 12, 10000 Zagreb, Croatia40Professor of Paediatrics, University of Zagreb, School of Medicine, Croatia41Centre for Paediatric Rheumatology and Autoinflammatory Diseases, Department of Paediatrics and Inherited Metabolic Disorders, 1st Faculty of Medicine, Charles University, General University Hospital in Prague, Ke Karlovu 2, 128 08 Prague 2, Czech Republic42Department of Immunology, Motol University Hospital, 2nd Faculty of Medicine, Charles University, Prague, Czech Republic43Department of Pediatrics, Aarhus University Hospital, Aarhus, Denmark44Rare Disease and Pediatric Research Centers, University of Helsinki and Helsinki University Central Hospital, Finland45Pediatric Rheumatologist, Pediatric Rheumatology, New Children’s Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland46Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, 00146 Roma, Italy47Pediatric Immunopathology and Allergology Unit, University of Rome Tor Vergata, Policlinico Tor Vergata, Rome, Italy, Saint Camillus International University of Health and Medical Sciences, Italy48Department of Clinical Sciences and Community Health, University of Milano, Italy49Division of Pediatric Immunology, Department of Health Sciences, University of Florence and Meyer Children’s Hospital, Florence, Italy50Department of Pediatrics Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico Milano, Italy51Immunodeficiency and Autoinflammatory Disorders Centre, Internal Medicine Department. Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico of Milan, Italy52San Raffaele Telethon Institute for Gene Therapy, Pediatric Immunohematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy53Institute for Maternal and Child Health IRCCS Burlo Garofolo and University of Trieste, Trieste, Italy54Department of Pediatric Rheumatology, Pediatrics Division, University Medical Center Utrecht, University Utrecht, the Netherlands55Paediatric Immunodefciencies and Infectious Diseases Unit, Paediatric Department, Department Centro Materno-Infantil do Norte, CHUPorto, Porto, Portugal56Hospital Universitario y Politécnico La Fe, Valencia57Pediatric Rheumatology Unit, Department of Pediatrics, Hospital Universitari Parc Taulí Sabadell, Spain58Pediatric Clinical Immunology and Primary Immunodeficiencies Unit, Hospital Sant Joan de Déu, Barcelona, Spain59Functional Unit of Immunology, Sant Joan de Déu Hospital, Institut de Recerca Sant Joan de Déu, University of Barcelona, Barcelona, Spain60Multidisciplinary Immunodeficiencies Unit of adults and children, Gregorio Marañon Hospital, Complutense University of Madrid, Madrid, Spain61Department of Pediatric Infectious Diseases, La Paz University Hospital, 28046 Madrid, Spain62Translational Research Network in Pediatric Infectious Diseases, 28009 Madrid, Spain63Primary Immunodeficiencies Unit, Pediatrics Hospital 12 octubre, Madrid, Spain64Instituto de Investigación, Hospital 12 octubre (imas12), Madrid, Spain65School of Medicine, Complutense University, Madrid, Spain66Prof Immunology, H.U. Reina Sofia, Faculty of Medicine, University of Cordoba, Cordoba, Spain67Pediatric Infectious Diseases, Rheumatology and Immunology Unit, Hospital Universitario Virgen del Rocío, Institute of Biomedicine of Seville (IBIS)/ Universidad de Sevilla/CSIC, Red de Investigación Translacional en Infectología Pediátrica RITIP, Seville, Spain68The Department of Rheumatology and Inflammation Research, The Sahlgrenska Academy at University of Gothenburg, Sweden69Department of Pediatric Oncology and Immunology, Children’s Hospital, Skåne University Hospital, Lund, Sweden70Royal Hospital for Children, Glasgow, Scotland71Regional Immunology Service, Royal Victoria Hospital, Belfast, BT126BA72UCL GOS Institute of Child Health, London UK73Department of Paediatric Allergy & Immunology, Royal Manchester Children’s Hospital, Manchester, UK74Alder Hey Children’s Foundation Trust, Liverpool, UK75Clinical Immunology and Allergy Department, Nottingham University Hospitals NHS Trust76NIHR Southampton Clinical Research Facility and Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UK77Faculty of Medicine and Institute for Life Sciences, University of Southampton, Southampton, SO17 1BJ, UK78Immunodeficiency Centre for Wales, Cardiff, UK Publisher Copyright: © 2022, The Author(s).
(Peer reviewed)
