Protocol for a prospective, observational, longitudinal study in paediatric patients with moderate-to-severe atopic dermatitis (PEDISTAD): study objectives, design and methodology

Abstract

Introduction: Atopic dermatitis (AD) is a chronic inflammatory skin disease often associated with atopic comorbidities and has significant impact on children and their families. There is a lack of robust and longitudinal long-term data on disease characteristics and typical clinical practice with currently available treatments in children with moderate-to-severe AD. Hence, an observational study is needed to evaluate AD characteristics and progression in paediatric patients with moderate-to-severe AD.

Methods and analysis: Pediatric Study in Atopic Dermatitis (PEDISTAD) is a prospective, observational, longitudinal study in paediatric patients with moderate-to-severe AD who are currently receiving systemic or topical treatment and whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are not medically advisable. 1300 children at 100-150 sites in approximately 20 countries worldwide will be enrolled and followed for 5 years. AD therapy is at the discretion of the investigator. Data collected will include: AD disease characteristics and comorbidities; current therapy for AD and initiation of new treatments/changes in current treatment; patient-reported/caregiver-reported outcomes; days missed from school/work for the patient/caregiver; healthcare professional visits; safety and biomarkers.

Ethics and dissemination: This study is conducted in accordance with the principles established by the 18th World Medical Assembly and all subsequent amendments and the guidelines for Good Epidemiology Practice. Each individual country assures that ethics approval has been received and local regulatory requirements are met. Ethics approval has been obtained in all countries currently participating in PEDISTAD. Study data will be disseminated in manuscripts submitted to peer-reviewed medical journals as well as in abstracts submitted to congresses and in the resulting posters and presentations.

Trial registration number: NCT03687359; pre-results.

Keywords: atopic dermatitis; observational; paediatric; systemic treatment.

Conflict of interest statement

Competing interests: ASP: AbbVie, AnaptysBio, Eli Lilly, Galderma, Incyte, Janssen, LEO Pharma, Novartis, Regeneron Pharmaceuticals, Inc., Sanofi – investigator; AbbVie, Amgen, Asana, Dermavant, Dermira, Galderma, Eli Lilly, Forte, LEO Pharma, Matrisys Bioscience, Menlo Therapeutics, Morphosys/Galapagos, Novartis, Pfizer, Regeneron Pharmaceuticals, Inc., Sanofi – consultant. EG-Y: AbbVie, Celgene, Eli Lilly, Galderma, GlaxoSmithKline, Glenmark, LEO Pharma, Pfizer, Regeneron Pharmaceuticals, Inc., Sanofi – investigator; AbbVie, Anacor, Asana Biosciences, Daiichi Sankyo, DBV, Dermira, Eli Lilly, Galderma, GlaxoSmithKline, Glenmark, Kiniksa Pharmaceuticals, Kyowa, LEO Pharma, Menlo Therapeutics, Novartis, Pfizer, Realm, Regeneron Pharmaceuticals, Inc., Sanofi – consultant; AbbVie, Celgene, Dermira, Galderma, Innovaderm, Janssen, LEO Pharma, Novartis, Pfizer, Regeneron Pharmaceuticals, Inc., Sanofi – research grants. ADI: AbbVie, Chugai Pharma, Genentech, Janssen, Novartis, Pfizer, Regeneron Pharmaceuticals, Inc., Sanofi Genzyme – consultant. EB: Almirall – speaker; AbbVie, Eli Lilly, Pfizer – investigator; Pierre Fabre Dermatology – investigator, consultant; Regeneron Pharmaceuticals, Inc., Sanofi Genzyme – consultant; Venthera – co-founder, consultant. MdeB-W: Regeneron Pharmaceuticals, Inc., Sanofi Genzyme – investigator, advisory board member, speaker, consultant; AbbVie, Pfizer – investigator, advisory board member; Eli Lilly, UCB – advisory board member SJ, AZ, ER, ZEO: Sanofi – employees, may hold stock and/or stock options in the company. PM-O: Regeneron Pharmaceuticals, Inc. – employee and shareholder.

© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1

Patients and study locations.

Figure 2

Data collection schedule. AD, atopic dermatitis; HCP, healthcare professional. *Therapy switch visits will take place at each initiation of a new systemic therapy and post-therapy switch will take place approximately 1 month later. Therapy switch visits are not required if the patient stays on topical therapy, stays on the same systemic drug through the study or if the visit overlaps with another scheduled visit.†Blood samples are collected every 12 months.