ECE2020 Audio ePoster Presentations Bone and Calcium (121 abstracts)
Baseline characteristics from the observational paradighm registry of patients with chronic hypoparathyroidism
Bart L Clarke 1 , Lars Rejnmark 2 , Steven Ing 3 , Maria Luisa Brandi 4 , Sigridur Björnsdottir 5 , Stefanie Hahner 6 , Lorenz C Hofbauer 7 , Pascal Houillier 8 , Aliya A Khan 9 , Michael A Levine 10 , Michael Mannstadt 11 , Dolores M Shoback 12,13 , Tamara J Vokes 14 , Pinggao Zhang 15 , Claudio Marelli 16 , John Germak 17 & Neil Gittoes 18
1Mayo Clinic Rochester, Division of Endocrinology, Diabetes, Metabolism, & Nutrition, Minnesota, United States; 2Aarhus University and Aarhus University Hospital, Endocrinology and Internal Medicine, Aarhus, Denmark; 3Ohio State University Wexner Medical Center, Endocrinology, Diabetes and Metabolism, Ohio, United States; 4University Hospital of Careggi, Endocrinology and Metabolic Diseases, Florence, Italy; 5Karolinska Institutet, Molecular Medicine and Surgery, Stockholm, Sweden; 6University of Würzburg, Department of Medicine I, Endocrinology and Diabetology, Würzburg, Germany; 7Technische Universität Dresden, Endocrinology, Diabetes, and Osteology, Dresden, Germany; 8Georges Pompidou European Hospital and Paris University, Metabolism and Physiology, Paris, France; 9McMaster University, Medicine, Divisions of Endocrinology and Metabolism and Geriatric Medicine, Ontario, Canada; 10Children’s Hospital of Philadelphia, Endocrinology and Diabetes, Pennsylvania, United States; 11Massachusetts General Hospital and Harvard Medical School, Endocrine Unit, Massachusetts, United States; 12San Francisco Veterans Affairs Medical Center; University of California, San Francisco, Endocrine Research Unit − 111N, California, United States; 13University of California, San Francisco, California, United States; 14University of Chicago, Section of Endocrinology, Illinois, United States; 15Shire Human Genetic Therapies, Inc., a Takeda company, Biostatistics, Massachusetts, United States; 16Shire International GmbH, Takeda company, Global Medical Affairs, Glattpark-Opfikon (Zurich), Switzerland; 17Shire Human Genetic Therapies, Inc., Takeda company, US Medical Affairs, Massachusetts, United States; 18University of Birmingham, Centre for Endocrinology, Diabetes and Metabolism (CEDAM), Birmingham, United Kingdom
PARADIGHM is an actively recruiting, prospective, observational registry (NCT01922440/EUPAS16927). The primary objective is to evaluate the safety and effectiveness of recombinant human parathyroid hormone, rhPTH(1-84), treatment in patients with chronic hypoparathyroidism under routine clinical care. The secondary objective is tocharacterize the clinical course of chronic hypoparathyroidism under conditions of routine clinical practice. At enrollment, registry inclusion criteria are patients having a hypoparathyroidism diagnosis >6 months and receiving conventional therapy (CT; eg, calcium supplements and active vitamin D), rhPTH(1-84) plus CT, or rhPTH(1-84). We present baseline characteristics of patients as of a June 30 2019 data cut. Baseline was defined as the value entered at the time of enrollment (Visit 1). Baseline symptom data exclude patients who initiated rhPTH(1-84) prior to enrollment (n = 68) and are herein presented as two groups: those subsequently prescribed with rhPTH(1-84) after enrollment or those treated with CT. All data are summarized descriptively. Patient data from 64 centers in Europe and North America were analyzed. In the analysis population (n = 737), 587 patients (79.6%) were female, 620 (84.1%) were white, and the mean (SD) age was 49.1 (16.45) years. The mean (SD) BMI was 19.3 (5.73) kg/m2and 30.0 (7.72) kg/m2in patients aged <18 (n = 25) and ≥18 (n = 587) years, respectively. The primary cause of hypoparathyroidism was thyroid surgery (n = 547 [74.2%]; of these, 281 [60.0%] underwent surgery for thyroid cancer). Endocrinologists were the prescribing specialists for most patients (n = 660 [89.6%]). Vitamin D and analogs were prescribed for 90.1% of patients (calcitriol, 74.2%, native vitamin D, 47.4%, alfacalcidol, 7.9%), calcium for 81.0% (calcium carbonate, 57.9%, calcium citrate, 27.1%), and thyroid hormones for 71.2% (levothyroxine, 73.4%; liothyronine, 5.8%). Symptoms reported at enrollment for the rhPTH (1-84) (n = 66) and the CT groups (n = 603), respectively, included fatigue (53.0%, 39.3%), paresthesia (48.5%, 29.2%), muscle twitching (48.5%, 21.1%), muscle cramping (40.9%, 33.0%), headaches (33.3%, 17.6%), anxiety (28.8%, 20.1%), muscle pain (28.8%, 19.2%), tetany (28.8%, 12.1%), and brain fog (27.3%, 16.3%). The baseline data for the overall population appear to be representative of patients with chronic hypoparathyroidism. Baseline data suggest that at enrollment patients prescribed rhPTH(1-84) after enrollment appear to have an increased burden of disease than patients receiving CT based on symptoms. PARADIGHM will be a valuable resource of real-world longitudinal data for patients with chronic hypoparathyroidism.
Funding: Shire, a Takeda company
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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