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. 2020 Apr;13(2):151-162.
doi: 10.1007/s40271-019-00388-5.

Assessing the Patient Experience of Hypoparathyroidism Symptoms: Development of the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom)

Meryl Brod  1 , Laura Tesler Waldman  2 , Alden Smith  3 , David Karpf  3
Affiliations

Assessing the Patient Experience of Hypoparathyroidism Symptoms: Development of the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom)

Meryl Brod et al. Patient. 2020 Apr.

Abstract

Background and objective: Hypoparathyroidism is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone. Patients with hypoparathyroidism receiving standard-of-care therapy report debilitating physical and cognitive symptoms, which may indicate a reduced health-related quality of life. The purpose of this study was to develop a new disease-specific measure of the signs and symptoms of hypoparathyroidism, the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom), and provide evidence for the content validity of items in the measure based on rigorous qualitative research methodologies for patient-reported outcome development.

Methods: Semi-structured, individual concept elicitation interviews were conducted with five clinical experts and 42 adults with hypoparathyroidism in the USA to identify the signs and symptoms of relevance and importance to those with the condition. Transcripts were coded and analyzed using an adapted grounded theory approach. Following item generation, cognitive debriefing interviews of the draft measure were conducted in an independent sample of 16 adults with hypoparathyroidism.

Results: One hundred percent of the concept elicitation patient sample reported experiencing physical symptoms that were attributed to hypoparathyroidism, including tingling/numbness/paresthesia (n = 37, 88%), muscle cramping (n = 36, 86%), and physical fatigue (n = 35, 83%). The majority of patients (n = 36, 86%) further reported experiencing cognitive dysfunction, including impaired memory (n = 24, 57%), impaired ability to have a conversation (n = 21, 50%), and lack of concentration/focus (n = 18, 43%). Seventeen major signs and symptoms were identified during item generation and included in the preliminary measure. After the cognitive debriefing, the 17-item HPES-Symptom was generated.

Conclusions: The findings provided evidence of content validity for the HPES-Symptom in US adults with hypoparathyroidism. Additional research is needed to validate the measure in patients with hypoparathyroidism to assess its psychometric properties.

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Conflict of interest statement

Meryl Brod and Laura Tesler Waldman are consultants to the pharmaceutical industry, including Ascendis Pharma. Alden Smith is an employee of Ascendis Pharma and received stock options as an employee of Ascendis Pharma. David Karpf is an employee of Ascendis Pharma and received stock/stock options as an employee of Ascendis Pharma.

Figures

Fig. 1
Fig. 1
Preliminary theoretical model of the signs and symptoms of hypoparathyroidism
See this image and copyright information in PMC

References

    1. Mannstadt M, Bilezikian JP, Thakker RV, Hannan FM, Clarke BL, Rejnmark L, et al. Hypoparathyroidism. Nat Rev Dis Primers. 2017;3:17080. doi: 10.1038/nrdp.2017.80. - DOI - PubMed
    1. Bilezikian JP, Khan A, Potts JT, Jr, Brandi ML, Clarke BL, Shoback D, et al. Hypoparathyroidism in the adult: epidemiology, diagnosis, pathophysiology, target-organ involvement, treatment, and challenges for future research. J Bone Miner Res. 2011;26(10):2317–2337. doi: 10.1002/jbmr.483. - DOI - PMC - PubMed
    1. Cusano NE, Rubin MR, Bilezikian JP. PTH(1–84) replacement therapy for the treatment of hypoparathyroidism. Expert Rev Endocrinol Metab. 2015;10(1):5–13. doi: 10.1586/17446651.2015.971755. - DOI - PMC - PubMed
    1. Parfitt AM. The actions of parathyroid hormone on bone: relation to bone remodeling and turnover, calcium homeostasis, and metabolic bone diseases. II. PTH and bone cells: bone turnover and plasma calcium regulation. Metabolism. 1976;25(8):909–955. doi: 10.1016/0026-0495(76)90124-4. - DOI - PubMed
    1. Kroll MH. Parathyroid hormone temporal effects on bone formation and resorption. Bull Math Biol. 2000;62(1):163–188. doi: 10.1006/bulm.1999.0146. - DOI - PubMed

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