A L Bosma1, P I Spuls1, I Garcia-Doval2,3, L Naldi4, D Prieto-Merino5,6, F Tesch7, C J Apfelbacher8, B W M Arents9, S Barbarot10, E Baselga11, M Deleuran12, L F Eichenfield13, L A A Gerbens1, A D Irvine14,15,16, A Manca17, P Mendes-Bastos18, M A Middelkamp-Hup1, A Roberts19, J Seneschal20, Å Svensson21, J P Thyssen22, T Torres23, F M Vermeulen1, C Vestergaard12, L B von Kobyletzki24,25, D Wall26,27, S Weidinger28, J Schmitt7,29, C Flohr30. 1. Amsterdam UMC, Location AMC, University of Amsterdam, Department of Dermatology, Amsterdam Public health, Infection and Immunity, Amsterdam, the Netherlands. 2. Research Unit, Academia Española de Dermatología y Venereología, Madrid, Spain. 3. Dermatology Department, Complexo Hospitalario Universitario de Vigo, Vigo, Spain. 4. Centro Studi GISED, Bergamo, Italy. 5. Applied Statistics in Medical Research Group, Catholic University of Murcia (UCAM), Murcia, Spain. 6. Faculty of Epidemiology and Population Health, London School of Hygiene and Tropical Medicine, London, U.K. 7. Center for Evidence-based Healthcare, Medizinische Fakultät Carl Gustav Carus, TU Dresden, Dresden, Germany. 8. Institute of Epidemiology and Preventive Medicine, University of Regensburg, Regensburg, Germany. 9. Dutch Association for People with Atopic Dermatitis, Nijkerk, the Netherlands. 10. Department of Dermatology, CHU Nantes, Nantes, France. 11. Department of Dermatology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain. 12. Department of Dermatology, Aarhus University Hospital, Aarhus, Denmark. 13. Department of Dermatology and Pediatrics, University of California, San Diego, CA, U.S.A. 14. Department of Clinical Medicine, Trinity College Dublin, Dublin, Ireland. 15. National Children's Research Centre, Dublin, Ireland. 16. Department of Paediatric Dermatology, Our Lady's Children's Hospital, Dublin, Ireland. 17. Centre for Health Economics, University of York, York, U.K. 18. Dermatology Centre, Hospital CUF Descobertas, Lisboa, Portugal. 19. Nottingham Support Group for Carers of Children with Eczema, Nottingham, U.K. 20. Department of Dermatology and Pediatric Dermatology, National Reference Center for Rare Skin Diseases, University Hospital of Bordeaux, Bordeaux, France. 21. Department of Dermatology and Venereology, Skane University Hospital, Malmö, Sweden. 22. Department of Dermatology and Allergy, Herlev-Gentofte Hospital, University of Copenhagen, Hellerup, Denmark. 23. Department of Dermatology, Centro Hospitalar Universitário Porto, Porto, Portugal. 24. Centre for Clinical Research, Lund University, Malmö, Sweden. 25. Centre for Clinical Research, Örebro University, Örebro, Sweden. 26. St James's Hospital, Dublin, Ireland. 27. Irish Skin Foundation, Dublin, Ireland. 28. Department of Dermatology and Allergy, University Hospital Schleswig-Holstein, Campus Kiel, Kiel, Germany. 29. University Allergy Center, University Hospital Carl Gustav Carus Dresden, Dresden, Germany. 30. Unit for Population-Based Dermatology Research, St John's Institute of Dermatology, Guy's & St Thomas' NHS Foundation Trust and King's College London, London, U.K.
Abstract
BACKGROUND: A long-term prospective observational safety study is essential to characterize fully the safety profile of systemic immunomodulating therapies for patients with atopic eczema. The TREatment of ATopic eczema (TREAT) Registry Taskforce offers a large platform to conduct such research using national registries that collect the same data using a predefined core dataset. OBJECTIVES: To present a protocol for a safety study comparing dupilumab with other systemic immunomodulating therapies in children and adults with moderate-to-severe atopic eczema, to assess the long-term safety risk of these therapies in a routine clinical care setting. METHODS: We describe a registry-embedded international observational prospective cohort study. Adult and paediatric patients who start treatment with dupilumab or another systemic immunomodulating agent for their atopic eczema will be included. The primary end point is the incidence of malignancies (excluding nonmelanoma skin cancer) compared between the treatment groups. Secondary end points include other serious adverse events and adverse events of special interest, such as eye disorders and eosinophilia. CONCLUSIONS: This protocol delineates a safety study for dupilumab in adult and paediatric patients with atopic eczema, using a standardized methodological approach across several national registries. The protocol could also be used for other novel systemic immunomodulating therapies, and could provide licensing and reimbursement authorities, pharmaceutical companies and clinicians with safety evidence from a routine clinical care setting. What's already known about this topic? There is a need for long-term data on the safety of systemic immunomodulating therapies in patients with atopic eczema. Regulatory bodies, such as the European Medicines Agency, increasingly stipulate the collection of such data as part of the licensing agreement for new treatments, to assess the new agent's long-term safety profile against established therapies. Large numbers of patients with a long duration of follow-up are necessary in order to detect rare events like malignancies. What does this study add? The TREAT Registry Taskforce offers a platform to conduct such research with a network of multiple national atopic eczema research registries. We present a protocol for an investigator-initiated multicentre safety study comparing dupilumab with other systemic immunomodulating therapies in adults and subsequently adolescents and children with moderate-to-severe atopic eczema. This protocol can be used as a framework for similar studies for other novel systemic immunomodulating therapies across both adult and paediatric populations.
BACKGROUND: A long-term prospective observational safety study is essential to characterize fully the safety profile of systemic immunomodulating therapies for patients with atopic eczema. The TREatment of ATopic eczema (TREAT) Registry Taskforce offers a large platform to conduct such research using national registries that collect the same data using a predefined core dataset. OBJECTIVES: To present a protocol for a safety study comparing dupilumab with other systemic immunomodulating therapies in children and adults with moderate-to-severe atopic eczema, to assess the long-term safety risk of these therapies in a routine clinical care setting. METHODS: We describe a registry-embedded international observational prospective cohort study. Adult and paediatric patients who start treatment with dupilumab or another systemic immunomodulating agent for their atopic eczema will be included. The primary end point is the incidence of malignancies (excluding nonmelanoma skin cancer) compared between the treatment groups. Secondary end points include other serious adverse events and adverse events of special interest, such as eye disorders and eosinophilia. CONCLUSIONS: This protocol delineates a safety study for dupilumab in adult and paediatric patients with atopic eczema, using a standardized methodological approach across several national registries. The protocol could also be used for other novel systemic immunomodulating therapies, and could provide licensing and reimbursement authorities, pharmaceutical companies and clinicians with safety evidence from a routine clinical care setting. What's already known about this topic? There is a need for long-term data on the safety of systemic immunomodulating therapies in patients with atopic eczema. Regulatory bodies, such as the European Medicines Agency, increasingly stipulate the collection of such data as part of the licensing agreement for new treatments, to assess the new agent's long-term safety profile against established therapies. Large numbers of patients with a long duration of follow-up are necessary in order to detect rare events like malignancies. What does this study add? The TREAT Registry Taskforce offers a platform to conduct such research with a network of multiple national atopic eczema research registries. We present a protocol for an investigator-initiated multicentre safety study comparing dupilumab with other systemic immunomodulating therapies in adults and subsequently adolescents and children with moderate-to-severe atopic eczema. This protocol can be used as a framework for similar studies for other novel systemic immunomodulating therapies across both adult and paediatric populations.
Authors: Dmitri Wall; Raed Alhusayen; Bernd Arents; Christian Apfelbacher; Esther A Balogh; Laita Bokhari; Manja Bloem; Angela L Bosma; Tim Burton; Leslie Castelo-Soccio; Nicole Fagan; Steven R Feldman; Godfrey Fletcher; Carsten Flohr; Esther Freeman; Lars E French; Christopher E M Griffiths; George J Hruza; John R Ingram; Michael D Kappelman; Irene Lara-Corrales; Henry W Lim; Nekma Meah; Devon E McMahon; Satveer K Mahil; Ian McNicoll; Annelie Musters; Haley B Naik; Rodney Sinclair; Catherine H Smith; Phyllis Spuls; Desmond J Tobin; Katherine York; Alan D Irvine Journal: Clin Dermatol Date: 2021-04-06 Impact factor: 3.541
Authors: Jonathan I Silverberg; H Chih-Ho Hong; Jacob P Thyssen; Brian M Calimlim; Avani Joshi; Henrique D Teixeira; Eric B Collins; Marjorie M Crowell; Scott J Johnson; April W Armstrong Journal: Dermatol Ther (Heidelb) Date: 2022-04-18
Authors: Carla J Jonker; Sieta T de Vries; H Marijke van den Berg; Patricia McGettigan; Arno W Hoes; Peter G M Mol Journal: Drug Saf Date: 2021-06-06 Impact factor: 5.606
Authors: Katrina Abuabara; Jonathan I Silverberg; Eric L Simpson; Amy S Paller; Lawrence F Eichenfield; Robert Bissonnette; James Krueger; John E Harris; Laura Dalfonso; Stephanie E Watkins; Julie M Crawford; D Thaçi; Emma Guttman-Yassky Journal: BMJ Open Date: 2020-11-27 Impact factor: 2.692