Evaluation and Validation of the Hypoparathyroidism Daily Diary of Symptom Experience (HPT-DD-SE) and the Hypoparathyroidism Life Impact Questionnaire (HPT-LIQ)
Author(s)
Diane Whalley, PhD1, Rebecca Crawford, MA1, Nadège Tremel, MSc2, Caroline Robo, PharmD2, Lynda Doward, MRes1, Soraya Allas, MD, PhD2.
1RTI Health Solutions, Manchester, United Kingdom, 2Alexion Pharma France, Écully, France.
1RTI Health Solutions, Manchester, United Kingdom, 2Alexion Pharma France, Écully, France.
OBJECTIVES: The Hypoparathyroidism Daily Diary of Symptom Experience (HPT-DD-SE) and Hypoparathyroidism Life Impact Questionnaire (HPT-LIQ) are novel patient-reported outcome measures assessing symptoms and health-related quality of life (HRQoL) in chronic hypoparathyroidism (HypoPT). This study evaluated the psychometric properties and meaningful change thresholds for the scale scores.
METHODS: Data collected in a randomized, placebo-controlled, phase 3 trial of eneboparatide in adults with chronic HypoPT were used. The analysis sample included all randomized patients with HPT-DD-SE or HPT-LIQ data at baseline and week 12 and a subset of patients with data at week 24. Analyses included test-retest reliability intraclass correlation coefficients (ICCs), validity correlations, analysis of variance for group differences, and descriptive change scores by Patient Global Impression of Severity and Change anchors to estimate meaningful within-patient change (MWPC) and minimal important difference (MID) thresholds.
RESULTS: The analysis sample included 189 patients at baseline and week 12 and 151 patients at week 24. Scale scores demonstrated good test-retest reliability (ICCs>0.7) and discriminated between known and change groups. Most correlations with external measures were as hypothesized. Results supported computation of weekly HPT-DD-SE scale scores with more than half of daily scores available. MWPC thresholds for improvement in scores at week 24 were 1.2-1.9 for HPT-DD-SE Core Physical Symptoms, 1.1-1.8 for HPT-DD-SE Overall Physical Symptoms, 1.4-2.9 for HPT-DD-SE Cognitive Symptoms, and 2.1-3.4 for HPT-LIQ Physical Functioning. MID thresholds for differences in change at week 24 were 0.8 for HPT-DD-SE Core Physical Symptoms, 0.8 for HPT-DD-SE Overall Physical Symptoms, 0.7 for HPT-DD-SE Cognitive Symptoms, and 2.0 for HPT-LIQ Physical Functioning. Some items demonstrated ceiling effects due to variable symptomatic experiences in chronic HypoPT, but effects were minor for most scale scores.
CONCLUSIONS: HPT-DD-SE and HPT-LIQ scale scores demonstrate strong reliability, construct validity, and responsiveness to assess chronic HypoPT symptoms and impacts in clinical trials.
METHODS: Data collected in a randomized, placebo-controlled, phase 3 trial of eneboparatide in adults with chronic HypoPT were used. The analysis sample included all randomized patients with HPT-DD-SE or HPT-LIQ data at baseline and week 12 and a subset of patients with data at week 24. Analyses included test-retest reliability intraclass correlation coefficients (ICCs), validity correlations, analysis of variance for group differences, and descriptive change scores by Patient Global Impression of Severity and Change anchors to estimate meaningful within-patient change (MWPC) and minimal important difference (MID) thresholds.
RESULTS: The analysis sample included 189 patients at baseline and week 12 and 151 patients at week 24. Scale scores demonstrated good test-retest reliability (ICCs>0.7) and discriminated between known and change groups. Most correlations with external measures were as hypothesized. Results supported computation of weekly HPT-DD-SE scale scores with more than half of daily scores available. MWPC thresholds for improvement in scores at week 24 were 1.2-1.9 for HPT-DD-SE Core Physical Symptoms, 1.1-1.8 for HPT-DD-SE Overall Physical Symptoms, 1.4-2.9 for HPT-DD-SE Cognitive Symptoms, and 2.1-3.4 for HPT-LIQ Physical Functioning. MID thresholds for differences in change at week 24 were 0.8 for HPT-DD-SE Core Physical Symptoms, 0.8 for HPT-DD-SE Overall Physical Symptoms, 0.7 for HPT-DD-SE Cognitive Symptoms, and 2.0 for HPT-LIQ Physical Functioning. Some items demonstrated ceiling effects due to variable symptomatic experiences in chronic HypoPT, but effects were minor for most scale scores.
CONCLUSIONS: HPT-DD-SE and HPT-LIQ scale scores demonstrate strong reliability, construct validity, and responsiveness to assess chronic HypoPT symptoms and impacts in clinical trials.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR85
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases