Introduction: The Patient-Oriented Eczema Measure (POEM) assesses patient-reported severity of atopic dermatitis (AD) symptoms, whereas the Children's Dermatology Life Quality Index (CDLQI) measures how AD affects health-related quality of life (HRQoL) in children. Although the POEM and CDLQI have established thresholds for clinically meaningful within-patient change in adolescents (aged 12-17 years), there are no defined within-patient responder thresholds for clinically meaningful change in children aged 6-11 years.
Methods: Data from the LIBERTY AD PEDS phase 3 randomized, double-blind, placebo-controlled trial of dupilumab in children aged 6-11 years with severe AD were used to define the threshold for within-patient meaningful change in POEM and CDLQI scores. Anchor-based methods were applied to estimate mean change in POEM and CDLQI scores from baseline to week 16, with anchors of a 1-point improvement in the Patient Global Impression of Disease (PGID) scale and an improvement in score of ''A little better'' on the Patient Global Impression of Change (PGIC) scale. The distribution-based methods, a one-half standard deviation (SD) at baseline and a standard error mean (SEM) were also used.
Results: The mean POEM change scores associated with the anchors were a change of - 8.40 with the PGID anchor and - 6.30 with the PGIC anchor. Distribution-based estimates for POEM were one-half SD at baseline of 2.76, with a SEM of 3.32. Mean CDLQI change scores corresponding to the PGID and PGIC anchors were - 7.30 and - 6.80, respectively, while distribution-based estimates for CDLQI were a one-half SD at baseline of 3.69, with a SEM of 3.52.
Conclusions: In children with severe AD, an appropriate minimum threshold of clinically meaningful within-patient change was estimated as 6 points for both the POEM and CDLQI scores.
Trial registration: ClinicalTrials.gov Identifier: NCT03345914. What is the clinically meaningful within-person change in Patient-Oriented Eczema Measure and Children's Dermatology Life Quality Index scores in children 6 to 11 years old with severe atopic dermatitis? (MP4 289443 KB).
Keywords: Atopic dermatitis; CDLQI; Clinically meaningful; Dupilumab; POEM; Pediatric; Psychometric; Responder threshold; Validation.
© 2021. The Author(s).