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

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.

Living with hypoparathyroidism: development of the Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact)

“…As with all research, there are study limitations to consider when interpreting findings. These have been noted in a prior publication but should be reiterated here [28]. While efforts were made to recruit a diverse sample of participants, the majority were female and had post-surgical HP, which is similar to the sample compositions in other studies of patients with this condition [5,8,33].…”

“…A detailed description of the demographic characteristics of the patient study sample has previously been reported [ 28 ]. To briefly summarize these findings, 42 participants were included in data analysis.…”

“…The HPES-Impact was developed in accordance with the FDA guidance [29] on best research practices for PRO measure development [22][23][24][25]. The methods used to carry out this research, including eligibility criteria, have been described in detail in the HPES-Symptom article [28]. To briefly summarize, qualitative, individual concept elicitation (CE) interviews conducted by telephone with clinical experts and adults with HP were used to identify key concepts, the impacts of HP, that were both relevant and important to people with this condition to inform the development of the PRO measure and develop the preliminary theoretical model [24].…”

“…The purpose of this study was to develop disease-specific measures of the symptoms and health-related QOL (HRQOL) impacts of HP, and provide evidence for the content validity of the measure based on rigorous qualitative research methodologies for patient-reported outcomes (PRO) development [22][23][24][25][26][27]. The development of the first of these measures, the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom) has been previously reported [28]. This paper describes the development of the Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact).…”

MANAGEMENT OF ENDOCRINE DISEASE: Postsurgical hypoparathyroidism: current treatments and future prospects for parathyroid allotransplantation

“…Recently, the Hypoparathyroidism Patient Experience Scale-Symptom has been developed as a specific tool for patient-reported outcomes in hypoPT ( 8 ). One hundred per cent of the concept elicitation patient group experienced physical symptoms attributed to hypoPT, such as tingling, numbness and paresthesia (88%), muscle cramps (86%), physical fatigue (83%), cognitive dysfunction (86%), impaired memory (57%), impaired ability to have a conversation (50%), and a lack of concentration/focus (43%).…”