PURPOSE: To develop patient reported outcome (PRO) measures for hypoparathyroidism (HPT) patients to assess the effect of investigational treatment on symptoms and health-related quality of life (HRQOL).
METHODS: The PRO measures were developed using industry standard methods. Literature reviews (published/social media) provided discussion topics for concept elicitation (CE) interviews with US adult patients with self-reported HPT for ≥12 months. Thematic analysis of CE interview data generated content for 2 PRO measures: Hypoparathyroidism Daily Diary of Symptom Experience (HPT-DD-SE) and Hypoparathyroidism Life Impact Questionnaire (HPT-LIQ). Hybrid CE/cognitive debriefing (CD) interviews evaluated content validity of the HPT-DD-SE and HPT-LIQ. A US-based patient advocate (PA) interview further supported the measures’ content validity.
RESULTS: Literature review data generated key symptom and HRQOL topic areas for discussion in 10 CE interviews (90% female; mean age=51.8 years, age range 27-76 years). Key HPT symptoms reported by ≥7 patients included cognitive dysfunction (n=10;100%), pain (n=10;100%), muscle cramps (n=10;100%), fatigue (n=9;90%), muscles twitches/spasms (n=9;90%), temperature sensitivity (n=9;90%), tingling (n=8;80%), muscles weakness (n=8;80%), and muscle stiffness (n=7;70%). Patient-reported HRQOL impacts of HPT included physical functioning (n=9;90%), daily activities (n=10;100%), social functioning/relationships (n=10;100%), emotional impact (n=10;100%), work (n=8;80%), and sleep (n=5;50%). Draft measures (HPT-DD-SE: 19 items/HPT-LIQ: 34 items) included some duplicate items to allow patient-selection of most appropriate phrasing during CD interviews. 6 hybrid CE/CD interviews (100% female; mean age=47 years, age range 26-64 years) and 12 CD interviews (92% female; mean age=49.3 years, age range 32-72 years) were conducted. Similar symptoms and HRQOL impacts emerged from the hybrid CE/CD interviews and were confirmed by the PA. CD interviews confirmed content validity, resulting in a 15-item HPT-DD-SE and a 27-item HPT-LIQ. Changes were based on patient preferences for item relevance and comprehension. The final versions were acceptable to patients.
CONCLUSIONS: The rigorous development process yielded 2 HPT-specific PRO measures: the HPT-DD-SE assesses severity of key symptoms (last 24 hours), and the HPT-LIQ assesses HRQOL impacts of HPT (last 7-days). Further work is needed to assess the psychometric properties of the new measures.
