Design and Methodological Issues of Within-Person (Split-Body) Randomized Controlled Trials Evaluating a Topical Treatment: A Systematic Review.

Intra-individual comparison Randomized clinical trial Split-body design Topical treatment Within-person design

Journal

Dermatology (Basel, Switzerland)
ISSN: 1421-9832
Titre abrégé: Dermatology
Pays: Switzerland
ID NLM: 9203244

Informations de publication

Date de publication:
2023
Historique:
received: 14 12 2022
accepted: 06 03 2023
medline: 12 10 2023
pubmed: 30 3 2023
entrez: 29 3 2023
Statut: ppublish

Résumé

Topical drugs are often used as first-line treatment for dermatological conditions. A within-person design may then be well adapted: it consists of randomizing lesions/body sites rather than patients, which are then concomitantly treated by the different drugs compared, reducing inter-group variability and therefore requiring fewer patients than the classical parallel-group trial. The aim of this review was to provide a methodological overview of within-person randomized trials (WP-RCTs) in dermatology. We searched for eligible trials published between 2017 and 2021 in MEDLINE, Embase, and Central in dermatology journals and the 6 highest-impact-factor general medical journals. Two authors selected publications and extracted data independently. From 1,034 articles identified, we included 54 WP-RCTs, mainly for acne vulgaris, psoriasis, actinic keratosis, and atopic dermatitis. In most of the trials, patients had only 2 lesions/body sites. In none of the trials, did we detect a potential carry-across effect (known to be the major methodological problem in WP-RCTs). Twelve studies reported a care provider applying the treatment, and in 26 studies, the patients themselves applied the treatment. Finally, we also highlight statistical issues for the statistical analysis: overall, 14 (26.9%) studies used a test for independent observations, thus ignoring the between-lesion correlation. Our systematic review highlights that despite the publication of the CONSORT checklist extension for WP-RCTs in 2017, this design is rarely used, and when it is, there are methodological and reporting concerns.

Sections du résumé

BACKGROUND BACKGROUND
Topical drugs are often used as first-line treatment for dermatological conditions. A within-person design may then be well adapted: it consists of randomizing lesions/body sites rather than patients, which are then concomitantly treated by the different drugs compared, reducing inter-group variability and therefore requiring fewer patients than the classical parallel-group trial.
OBJECTIVES OBJECTIVE
The aim of this review was to provide a methodological overview of within-person randomized trials (WP-RCTs) in dermatology.
METHODS METHODS
We searched for eligible trials published between 2017 and 2021 in MEDLINE, Embase, and Central in dermatology journals and the 6 highest-impact-factor general medical journals. Two authors selected publications and extracted data independently.
RESULTS RESULTS
From 1,034 articles identified, we included 54 WP-RCTs, mainly for acne vulgaris, psoriasis, actinic keratosis, and atopic dermatitis. In most of the trials, patients had only 2 lesions/body sites. In none of the trials, did we detect a potential carry-across effect (known to be the major methodological problem in WP-RCTs). Twelve studies reported a care provider applying the treatment, and in 26 studies, the patients themselves applied the treatment. Finally, we also highlight statistical issues for the statistical analysis: overall, 14 (26.9%) studies used a test for independent observations, thus ignoring the between-lesion correlation.
CONCLUSION CONCLUSIONS
Our systematic review highlights that despite the publication of the CONSORT checklist extension for WP-RCTs in 2017, this design is rarely used, and when it is, there are methodological and reporting concerns.

Identifiants

pubmed: 36990057
pii: 000530149
doi: 10.1159/000530149
doi:

Types de publication

Systematic Review Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

720-731

Informations de copyright

© 2023 S. Karger AG, Basel.

Auteurs

Sophie Leducq (S)

Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France.
Department of Dermatology and Reference Center for Rare Diseases and Vascular Malformations (MAGEC), CHRU Tours, Tours, France.

Amandine Dugard (A)

Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France.

Aude Allemang-Trivalle (A)

Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France.

Bruno Giraudeau (B)

Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France.
INSERM CIC 1415, Tours, France.

Annabel Maruani (A)

Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France.
Department of Dermatology and Reference Center for Rare Diseases and Vascular Malformations (MAGEC), CHRU Tours, Tours, France.

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Classifications MeSH