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.
19 - 22 May 2018
European Society of Endocrinology