Living with hypoparathyroidism: development of the Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact)
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Living with hypoparathyroidism: development of the Hypoparathyroidism Patient Experience Scale‑Impact (HPES‑Impact) Meryl Brod1 · Laura Tesler Waldman1 · Alden Smith2 · David Karpf2 Accepted: 7 August 2020 © The Author(s) 2020
Abstract Purpose Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone (PTH). Research indicates that HP patients on conventional therapy may have a reduced quality of life. The study’s purpose was to develop a new disease-specific measure of the impacts of hypoparathyroidism on functioning and well-being and provide evidence for its content validity based on rigorous qualitative research methodologies for patient-reported outcomes development. Methods Semi-structured, individual concept elicitation (CE) interviews were conducted with 5 clinical experts and 42 adults in the USA with HP to identify impacts of relevance and importance to the target population. Transcripts were coded and analyzed using an adapted grounded theory approach common to qualitative research. Following item generation, the draft measure was cognitive debriefed in an independent sample of 16 adults with HP. Results Analyses identified four impact domains: physical functioning, including ability to exercise (n = 32, 76%) and mobility (n = 21, 50%); daily life, including ability to do things around the home (n = 33, 79%), and interference with work productivity (n = 18, 43%); psychological well-being, including feeling anxious/anxiety (n = 34, 81%) and frustrated (n = 27, 64%); and social, including ability to participate in social activities (n = 33, 79%) and relationships (n = 32, 76%). Twentyseven impacts were identified and included in the preliminary measure. After the cognitive debriefing, a validation-ready, 26-item Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact) was generated. Conclusion Findings provide substantial evidence of content validity for the validation-ready HPES-Impact in adults with HP. Keywords Hypoparathyroidism · Patient-reported outcome measures · Quality of life · Adult
Background Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone (PTH) [1, 2]. Low levels or the absence of PTH circulating in the bloodstream can lead to hypocalcemia (low blood calcium levels), hyperphosphatemia (elevated blood phosphate levels), hypercalciuria David Karpf: Affiliation when research was conducted. * Meryl Brod [email protected] 1
The Brod Group, 219 Julia Ave., Mill Valley, CA 94941, USA
Ascendis Pharma, Inc., Palo Alto, CA, USA
2
(elevated urinary calcium levels), and overly-mineralized bone [1, 3]. Significant physical and cognitive symptoms are associated with the condition, including fatigue, muscle cramping/spasms, paresthesia (sensation of tingling and/or numbness), cognitive dysfunction, and sleep disturbances [1, 4–8]. Based on data from 2007–2009, the prevalence of HP in the USA has bee
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