ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Endocrine Abstracts (2018) 56 P241 | DOI: 10.1530/endoabs.56.P241

Burden of illness among patients with chronic hypoparathyroidism not adequately controlled with standard therapy by self-perception

Heide Siggelkow1, Bart L. Clarke2, Helen Dahl-Hansen3, Elizabeth Glenister4, Davneet Judge5, Nawal Bent-Ennakhil5, Katie Gibson5, John Germak6, Kristina Chen7, Claudio Marelli6 & Jens Bollerslev8


1Department of Gastroenterology and Endocrinology, University of Göttingen, Göttingen, Germany; 2Mayo Clinic Division of Endocrinology, Diabetes, Metabolism, and Nutrition, Rochester, USA; 3Nordic hypoPARA Organisation, Oslo, Norway; 4Hypopara UK, East Grinstead, UK; 5Adelphi Real-World, Bollington, UK; 6Shire International GmbH, Zug, Switzerland; 7Shire Human Genetic Therapies, Inc., Lexington, USA; 8Section of Specialized Endocrinology, Oslo University Hospital, Oslo, Norway.


Significant knowledge gaps exist regarding the humanistic effects of chronic hypoparathyroidism (HPT), a rare, debilitating condition. We provide interim results from a global survey conducted to characterise the burden associated with chronic HPT from the patient perspective. An anonymous, self-reported survey (online or paper) was conducted in patients with chronic HPT, not adequately controlled with standard therapy by self-perception, from 12 countries through physicians or patient associations. Symptoms and impact of HPT were assessed via HPT Symptom Diary, a disease-specific, patient-reported outcome tool that recorded severity of symptoms experienced in the last 7 days. Health-related quality of life (HRQoL) was evaluated by 2 validated instruments: SF-36 v2 and EQ-5D-5L. Patient demographics and clinical characteristics were also captured. Data were obtained from 226 patients (mean age, 51.6 years; 77% women; mean time since diagnosis, 7.8 years; surgery as main cause of HPT, 81%), of whom 94% had persistent symptoms despite treatment and 63% had been told by a physician that their calcium levels were poorly controlled. Most patients were receiving oral calcium (77%) and/or activated vitamin D (74%). Self-perceived overall symptom severity was reported as mild, moderate, or severe in 56 (25%), 138 (61%), and 30 (13%) patients, respectively. Per symptom diary, 98% of patients reported physical fatigue (symptom severity: mild, 22%; moderate, 42%; severe, 26%; very severe, 9%), 90% reported muscle cramps (mild, 31%; moderate, 42%; severe, 13%; very severe, 4%), and 90% reported heaviness in limbs (mild, 24%; moderate, 46%; severe, 16%; very severe, 4%). An impact on daily life, rated as ‘somewhat’ or ‘very much’, was reported by 89% of patients for ability to exercise, 85% for sleep, 80% for ability to work, and 72% for family relationships. An apparent inverse relationship was observed between scores of HPT symptom severity and both HRQoL assessments – the higher the severity scores, the lower the health status. Mean SF-36 summary scores were 44.3/36.6/28.2 (physical component summary) and 44.4/33.9/31.4 (mental health component summary) for patients reporting mild/moderate/severe HPT symptoms, respectively. Mean EQ-5D scores were 0.8/0.7/0.3 for patients reporting mild/moderate/severe HPT symptoms [n=132]). Findings from this interim analysis of a global survey demonstrated that there is a spectrum of symptom severity within a cohort of patients self-reporting inadequately-controlled HPT on standard therapy. The magnitude of symptom severity as reported by patients correlated with extent of impact on daily life and reduction in HRQoL.