Study Explores Clinically Meaningful Changes in Pediatric AD Assessment Scores

A post-hoc analysis of the Phase 3 LIBERTY AD PEDS trial determined clinically meaningful within-patient changes in atopic dermatitis severity scores for pediatric patients.

Thresholds for clinically meaningful changes in the Patient-Oriented Eczema Measure (POEM) assessments and Children’s Dermatology Life Quality Index (CDLQI) have already been established for 12-17 years old patients. Similarly, thresholds for POEM and Dermatology Life Quality Index (DLQI) have been established for adults.

However, the investigators noted a particular clinical gap in established thresholds for children aged 6-11.

As such, the team, led by Eric Simpson, MD, MCR, of Oregon Health & Science University, evaluated data from the randomized, double-blind placebo-controlled trial of dupilumab in the pediatric patient population.

Defining Thresholds

Patients in the LIBERTY AD PEDS trial included those with severe atopic dermatitis that could not be controlled with topical medications.

Enrolled participants (n = 367) were randomized 1:1:1 to dupilumab added to topical corticosteroids every 2 weeks, ever 4 weeks, or placebo for 16 weeks. The average age of the population was 8.5 years, and the majority was female (50.1%) and white (69.2%).

Simpson and colleagues used anchor-based and distribution-based methods to estimate thresholds for within-patient changes in POEM and CDLQI scores.

In the anchor-based approach, they calculated the mean change scores for both assessments based on a defined improvement in selected anchors.

“In line with recommendations that patient-reported anchors (often global assessments) are the most appropriate, a 1-point improvement on the Patient Global Impression of Disease (PGID) scale and ‘‘a little better’’ on the Patient Global Impression of Change (PGIC) scale from baseline to week 16 were selected as the anchors,” the investigators wrote.

The distribution-based method was considered supportive to the anchor-based approach.

Overall, 357 patients completed POEM assessments at baseline and week 16; likewise, 356 completed CDLQI at both time periods.

The magnitude of change correlations of POEM to PGID (|r| = 0.65) and PGIC (|r| = 0.60), and CDLQI to PGID (|r| = 0.58) and PGIC (|r| = 0.44) affirmed the anchors to be appropriate.

As such, they calculated that the mean POEM change scores associated with PGID improvement of 1 point was -8.40; the mean POEM change associated with a PGIC of a “little better improvement” was -6.30.

For CDLQI, the mean change in score associated with the minimum changes in PGID and PGIC were -7.30 and -6.80, respectively.

The distribution-based estimates were a one-half standard deviation at baseline of 2.76 and a standard error of measurement of 3.32 for PEOM. As for CDLQI, the one-half standard deviation was 3.69 at baseline and standard error of measurement was 3.52.

A paper authored by Simpson indicated that a change of 6-8 points in POEM and CDLQI scores for adolescents may be a reasonable threshold to show response and clinically meaningful change.

However, for this present study, the derivation of thresholds from empirically driven data was a limitation; as such the findings may not be generalized to other age ranges or conditions. Nevertheless, the results were considered to line up closely with the previous literature.

“In children with severe AD, a minimum individual within-patient change of 6 points in POEM and CDLQI scores is proposed as an appropriate threshold for defining a clinically meaningful within-patient change,” Simpson and colleagues concluded.

The study, “Definition of Clinically Meaningful Within-Patient Changes in POEM and CDLQI in Children 6 to 11 Years of Age with Severe Atopic Dermatitis,” was published online in Dermatology and Therapy.