&lt;p&gt;Psychometric evaluation of the hypoparathyroidism symptom diary&lt;/p&gt;

Coles, Theresa; Chen, Kristina; Nelson, Lauren; Harris, Nimanee; Vera-Llonch, Montserrat; Krasner, Alan; Martin, Susan

doi:10.2147/prom.s179310

Cited by 20 publications

(13 citation statements)

References 15 publications

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“…In addition to developing the HPES-Symptom measure, the findings on impacts were used to develop a separate disease-specific, patient-reported impact measure on how HP affects daily functioning and well-being (to be reported separately). The symptoms identified in this study are similar to those included in a recently developed symptom diary for this condition, lending support to the validity of our findings [37].…”

Section: Discussionsupporting

confidence: 86%

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

Brod

Waldman

Smith³

et al. 2019

Patient

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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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Section: Discussionsupporting

confidence: 86%

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

Brod

Waldman

Smith³

et al. 2019

Patient

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“…The measure is relatively brief and would be reasonable to administer on a daily basis in the context of clinical studies. Further research is under way to develop the scoring algorithm and evaluate the measure’s psychometric properties in the context of observational studies in this patient population [14].…”

Section: Resultsmentioning

confidence: 99%

Development of a Patient-Reported Outcome Measure for Chronic Hypoparathyroidism

et al. 2019

Self Cite

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IntroductionA patient-reported outcome (PRO) measure specific to chronic hypoparathyroidism is lacking to facilitate the evaluation of treatment. A PRO measure that followed the recommendations of the US Food and Drug Administration (FDA) PRO guidance was created to address key hypoparathyroidism symptoms.MethodsA literature review was conducted to identify symptoms of hypoparathyroidism and any existing PRO measures appropriate for evaluating these symptoms, followed by concept elicitation interviews involving six individuals with hypoparathyroidism. On the basis of the results of the literature review and interviews, a draft item pool was developed and refined through two sets of cognitive debriefing interviews with six additional patients. A translatability assessment was also conducted to evaluate concepts, phrases, or components of the items that could be problematic in future translations and to identify culturally specific phrasing.ResultsNo PRO measures appropriate to address hypoparathyroidism symptoms documented in the literature were identified. Qualitative research participants included 11 women and one man, with an average age of 49 years; the majority (10) of these participants were white. Concept elicitation interview results were generally consistent with the results of the literature review; the most commonly reported symptoms included issues with cognition, often described as “brain fog” (n = 6), muscle cramping (n = 5), tingling (n = 5), and muscle spasms or twitching (n = 4). The initial draft item pool included 20 items; based upon participant feedback, the final Hypoparathyroidism Symptom Diary comprised 13 items and was found to be easily understood and relevant to the participants. No significant issues were identified by the translatability assessment.ConclusionThe Hypoparathyroidism Symptom Diary was developed following the recommendations of the FDA’s PRO guidance. The measure addresses a comprehensive set of symptoms, as well as key impacts of hypoparathyroidism deemed important by patients.FundingShire Human Genetic Therapies, Inc., Lexington, MA, USA, a member of the Takeda group of companies.

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“…This study importantly provides evidence for content validity of the impacts captured in the HPES-Impact. Previous disease-specific measures that have been developed for this condition have focused primarily on symptoms and not captured the broad spectrum of both symptom and disease impacts [32]. We believe the HPES-Impact measure is the first disease-specific measure to capture the broad spectrum of disease impacts experienced by adult patients with HP.…”

Section: Discussionmentioning

confidence: 99%

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

Brod

Waldman

Smith³

et al. 2020

Qual Life Res

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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.

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<p>Psychometric evaluation of the hypoparathyroidism symptom diary</p>

Cited by 20 publications

References 15 publications

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

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

Development of a Patient-Reported Outcome Measure for Chronic Hypoparathyroidism

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

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