Psychometric Validation of the PAINS-pNF Target Plexiform Neurofibroma (PN) Pain Severity Score Using Data From KOMET (NCT04924608)
Author(s)
Adeyemi A1, Wolters P2, Norquist J3, McMurtry E4, Yang X3, Martin S2, Annas P5, Bunod L6, de la Rosa Rodriguez R1, Regnault A6
1Alexion Pharmaceuticals Inc, Boston, MA, USA, 2Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD, USA, 3Merck & Co., Inc., Rahway, NJ, USA, 4Clinical Pharmacology & Safety Sciences, AstraZeneca, Cambridge, UK, 5Alexion Pharmaceuticals, Inc., Copenhagen, Denmark, 6Modus Outcomes, a THREAD company, Lyon, France
Presentation Documents
OBJECTIVES: The PAin INtensity Scale for Plexiform Neurofibromas (PAINS-pNF) is a patient-reported outcome measure adapted from Numeric Rating Scale-11 using qualitative research to assess pain intensity in individuals with neurofibromatosis type 1-associated PN. This study aimed to quantitatively demonstrate PAINS-pNF target PN pain scores as fit-for-purpose to evaluate KOMET endpoints.
METHODS: KOMET participants (n=145) completed the PAINS-pNF daily. The chronic target PN pain score was obtained by aggregating responses over 28 days. Spike pain was defined as the maximum available daily PAINS-pNF spike pain intensity score over 28 days. Distribution/completeness of daily PAINS-pNF scores were assessed. Test–retest reliability of the scores was evaluated using intraclass correlation coefficients (ICC). Construct validity was evaluated using ANOVA and Spearman coefficients for association of PAINS-pNF with Patient’s Global Impression of Severity (PGIS) and measures of pain interference with daily life. The ability to detect change was assessed using PGIS (primary anchor) and Patient’s Global Impression of Change (PGIC). Meaningful score difference (MSD) was evaluated from baseline–cycle (C) 12 using both anchors.
RESULTS: Overall, <4% of PAINS-pNF scores were missing across cycles, except for C12 (7%). Good-to-excellent ICC: 0.92–>0.95 and adequate-to-excellent: 0.82–>0.95 test–retest reliability was demonstrated with PAINS-pNF chronic and spike target PN pain scores, respectively. Spearman correlations (>0.3) between the changes in PAINS-pNF scores and PGIS/PGIC indicated good construct validity. PAINS-pNF chronic target PN pain score showed good ability to capture improvement between baseline and C12. PAINS-pNF chronic pain score for half of patients with a one-category improvement in PGIS (baseline−C12; n=18) had decreased by >2.16; scores for <10% of participants with no change in PGIS (n=25) decreased by >2.00.
CONCLUSIONS: PAINS-pNF chronic and spike target PN pain measures demonstrated strong psychometric performance. An MSD estimate of a two-point decrease is reasonable for the chronic target pNF pain score.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
MSR8
Topic
Methodological & Statistical Research
Topic Subcategory
PRO & Related Methods
Disease
Drugs, Neurological Disorders, Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)