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

Abstract

IntroductionAtopic 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 analysisPediatric 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 disseminationThis 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 numberNCT03687359; pre-results.

Disease characteristics, comorbidities, treatment patterns and quality of life impact in children <12 years old with atopic dermatitis: Interim results from the PEDISTAD Real-World Registry

“…This ongoing, international, longitudinal, 5-year registry assesses the disease course, comorbidities, treatment, and disease burden in children with moderate-to-severe AD. 1 Here, we present baseline interim data.…”

“…The study design and inclusion/exclusion criteria for PEDISTAD have been reported. 1 Briefly, PEDISTAD included children aged \12 years at baseline with investigator-assessed moderate-to-severe disease receiving systemic medications for AD (including biologics, cyclosporine, methotrexate, azathioprine, mycophenolate mofetil, and corticosteroids), phototherapy, or topical treatment (but otherwise candidates for systemic therapy).…”

Recommendations for Vaccination in Children with Atopic Dermatitis Treated with Dupilumab: A Consensus Meeting, 2020

“…Regarding measles, mumps, rubella, and varicella (MMR-V) booster vaccination during dupilumab therapy, which is of utmost interest to the panel as some AD pediatric patients would be in the age range when they may require a booster, safety data are insufficient to make a positive recommendation [ 51 ]. Nonetheless, the panel recommends an individualized assessment considering the benefit of MMR-V immunization against the risk of infection in patients already taking dupilumab.…”

EAACI Biologicals Guidelines—dupilumab for children and adults with moderate‐to‐severe atopic dermatitis

“…The Paediatric 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. Data collected will include disease characteristics and co‐morbidities, current therapy for AD and initiation of new treatments/changes in current treatment, safety and biomarkers, patient‐oriented outcomes and health‐economics parameters 151 …”