Humanistic Burden of Chronic Hypoparathyroidism - Results of a Systematic Literature Review

Author(s)

Smith A1, Harricharan S2, Hubscher E3, Eng WF4, Forsythe A5
1Ascendis Pharma, Inc., Palo Alto, CA, USA, 2Purple Squirrel Economics, Toronto, ON, Canada, 3Purple Squirrel Economics, Apex, NC, USA, 4Ascendis Pharma, Inc., Hercules, CA, USA, 5Purple Squirrel Economics, New York, NY, USA

OBJECTIVES : Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone. Significant physical and cognitive symptoms are associated with HP, which may result in reduced health-related quality of life (HRQoL). A systematic literature review was conducted to evaluate the humanistic burden of chronic HP.

METHODS : EMBASE, MEDLINE, Cochrane Library, and relevant congresses were searched in accordance with the Preferred Reporting Item for Systematic Reviews and Meta-Analyses (PRISMA) guidelines using Population, Intervention & Comparators, Outcomes and Study Design (PICOS) criteria to identify relevant HRQoL studies published between 2010 and search date (12/08/2020).

RESULTS : Among 1,075 records screened using PICOS-based criteria, 33 studies evaluating HRQoL were selected. According to studies from the US, UK, and Norway, patients with chronic HP have significantly lower HRQoL scores than the general population. In 10 studies, patients with chronic HP experienced debilitating symptoms, including fatigue, muscle cramps, numbness and tingling, memory and processing speed deficits, and poor well-being. Increasing symptom severity was associated with diminished overall health status; EQ-5D-5L utility scores among patients reporting moderate and severe symptoms were 0.7 and 0.4, respectively. In one study, the incidence of anxiety and depression was significantly higher among patients with chronic HP (P = 0.02). Both patients and caregivers reported reduced work productivity and activity interference, as well as negative impacts on interpersonal relationships. Conventional treatment with calcium and vitamin D supplementation negatively impacted HRQoL. In one survey, most patients rated pill burden as moderate to severe; nearly 90% reported concern regarding treatment-related complications, and 25% indicated extreme concern about potential organ damage.

CONCLUSIONS : Despite treatment, patients with chronic HP experience a substantial symptom burden that interferes with daily activities, work function, and relationships, as well as negatively impacting emotional well-being. As a result, patients experience significantly diminished HRQoL relative to the general population.

Conference/Value in Health Info

2021-05, ISPOR 2021, Montreal, Canada

Value in Health, Volume 24, Issue 5, S1 (May 2021)

Code

PRO54

Topic

Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

Rare and Orphan Diseases

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