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Review
. 2024 Jul 15;8(1):73.
doi: 10.1186/s41687-024-00751-7.

Capturing patient-reported sleep disturbance in atopic dermatitis clinical trials

Carla Dias-Barbosa  1 , Jonathan I Silverberg  2 , Sonja Ständer  3 , Danielle Rodriguez  4 , Fatoumata Fofana  5 , Dina Filipenko  6 , Liliana Ulianov  7 , Christophe Piketty  7 , Jorge Puelles  7
Affiliations
Review

Capturing patient-reported sleep disturbance in atopic dermatitis clinical trials

Carla Dias-Barbosa et al. J Patient Rep Outcomes. .

Abstract

Background: Patient-focused approaches to capturing day-to-day variability in sleep disturbance are needed to properly evaluate the sleep benefits of new treatments. Such approaches rely on patient-reported outcome (PRO) measures validated in the target patient population.

Methods: Using atopic dermatitis (AD) as an example of a disease in which sleep is commonly disturbed, we developed a strategy for measuring sleep disturbance in AD trials. In developing this strategy, we conducted a targeted literature review and held concept elicitation interviews with adolescents and adults with AD. We subsequently identified potentially suitable PRO measures and cognitively debriefed them. Finally, we evaluated their psychometric properties using data from phase 2b (NCT03100344) and phase 3 (NCT03985943 and NCT03989349) clinical trials.

Results: The literature review confirmed that sleep disturbance is a key impact of AD but failed to identify validated PRO measures for assessing fluctuations in sleep disturbance. Subsequent concept elicitation interviews confirmed the multidimensional nature of sleep disturbance in AD and supported use of a single-item measure to assess overall sleep disturbance severity, complemented by a diary to capture individual components of sleep disturbance. The single-item sleep disturbance numerical rating scale (SD NRS) and multi-item Subject Sleep Diary (SSD)-an AD-adapted version of the Consensus Sleep Diary-were identified as potentially suitable PRO measures. Cognitive debriefing of the SD NRS and SSD demonstrated their content validity and their understandability to patients. Psychometric analyses based on AD trial data showed that the SD NRS is a well-defined, reliable, and fit-for-purpose measure of sleep disturbance in adults with AD. Furthermore, the SD NRS correlated with many SSD sleep parameters, suggesting that most concepts from the SSD can be covered using the SD NRS.

Conclusions: Using these findings, we developed an approach for measuring sleep disturbance in AD trials. Subject to further research, the same approach could also be applied to future trials of other skin diseases where itch causes sleep disturbance.

Keywords: Atopic; Content validity; Dermatitis; Patient reported outcome measures; Psychometrics; Sleep Wake disorders.

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Conflict of interest statement

CDB, DR, FF, and DF are employees of Evidera, which was paid by Galderma for work relating to this study. JIS has received grants as a consultant and/or advisory board member for Abbvie, Alamar, Aldena, Amgen, AObiome, Arcutis, Arena, Asana, Aslan, BioMX, Biosion, Bodewell, Boehringer-Ingelheim, Cara, Castle Biosciences, Celgene, Connect Biopharma, Dermavant, Dermira, Dermtech, Eli Lilly, Galderma, GlaxoSmithKline, Incyte, Kiniksa, Leo Pharma, Menlo, Novartis, Optum, Pfizer, RAPT, Regeneron, Sanofi-Genzyme, Shaperon, Union; speaker for Abbvie, Eli Lilly, Leo Pharma, Pfizer, Regeneron, Sanofi-Genzyme; institution received grants from Galderma, Incyte, Pfizer. SS has received grants/contracts from Almirall, Beiersdorf, the German Research Foundation (DFG), the European Academy of Dermatology and Venereology (EADV), the German Federal Ministry of Education and Research (BMBF), the Interdisciplinary Center for Clinical Research Münster (IZKF), Leo Pharma, Menlo, Novartis, Sanofi, and Trevi Therapeutics; consulting fees from Abbvie, Almirall, Beiersdorf, Bellus Health, Benevolent, Bionorica, Cara, Celgene, CelloHealth, Clexio, DS Biopharma, Eli Lilly, Escient, Galderma, Grünenthal, Kiniksa, Klinge Pharma, Menlo, Sanofi, Sienna, Trevi, the P.G. Unna Academy, Perrigo, Pfizer, Vanda, Vifor, and WebMD; and payment/honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Almirall, Eli Lilly, Sanofi, Galderma, Menlo, Omnicuris, Beiersdorf, Leo Pharma, Novartis, the P. G. Unna Academy, Pfizer, and Pierre Fabre. She has also participated in advisory boards for AbbVie, Almirall, Beiersdorf, Bellus Health, Benevolent, Bionorica, Cara, Celgene, CelloHealth, Clexio, DS Biopharma, Eli Lilly, Escient, Galderma, Grünenthal, Kiniksa, Klinge Pharma, Menlo, Sanofi, Sienna, Trevi, the P.G. Unna Academy, Perrigo, Pfizer, Vanda, Vifor, and WebMD. LU, CP, and JP are employees of Galderma, which funded this study.

Figures

Fig. 1
Fig. 1
Four-step strategy for measuring sleep disturbance in clinical trials. PRO patient-reported outcome
Fig. 2
Fig. 2
Conceptual framework for measuring sleep disturbance in AD. AD atopic dermatitis, NRS numerical rating scale, PRO patient-reported outcome, SD NRS sleep disturbance numerical rating scale, SSD Subject Sleep Diary
See this image and copyright information in PMC

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