PARADIGHM, a global, prospective, observational registry of patients with chronic hypoparathyroidism (HPT), began enrolment in 2013 to collect data on the natural history of chronic HPT (ClinicalTrials.gov NCT01922440). Since initiation, recombinant human PTH, rhPTH(1-84), has been approved in the United States and Europe as an adjunctive treatment for adult patients with HPT. The protocol for the registry (now a European Medicines Agencydesignated postmarketing commitment) was amended to capture rhPTH(1-84) use in HPT patients under routine clinical care (EUPAS16927). Patient recruitment continues with a global enrolment goal of ≥900 patients, including ≥300 receiving rhPTH(1-84). Follow-up data collection on each patient is planned for ≥10 years. Patients with a diagnosis of HPT of ≥6 months are eligible for inclusion; exclusions include inability to provide informed consent, enrolment in any interventional study, or active PTH(1-34) therapy. Treatment regimens are determined by the patients physician, per usual clinical practice, and can be conventional calcium/vitamin D supplements and/or rhPTH(1-84). Primary outcome variables are HPT lab tests, including renal function; renal and cardiovascular events; soft tissue calcification or bone fractures; presence of cataracts; and adverse events, including those considered to be related to rhPTH(1-84) treatment. Secondary outcome variables include health-related quality of life, disease-specific patient-reported measures, and hospitalisations and emergency room visits. Additional data to be collected include demographics, medical history, HPT management, and concomitant medications. Data are collected every 6 months, and the database uploads are via electronic data capture. A steering committee reviews scientific reports and evaluates requests for analyses. Prior to starting the new protocol, a registry data cut on December 1, 2016, was completed. Forty-one investigator sites and 492 patients (49±17 years of age; 30±9 kg/m2 body mass index) had been enrolled. Notably, 93% of patients reported ≥1 symptom within the previous 6 months despite prescribed conventional therapy of calcium (91%) and vitamin D (84%). Only 7% were recorded as receiving rhPTH(1-84) in a clinical trial setting (ie, data capture was prior to US Food and Drug Administration approval). Data from PARADIGHM will provide physicians with needed information on the natural history of HPT in patients prescribed conventional treatment and in those prescribed rhPTH(1-84).
19 - 22 May 2018
European Society of Endocrinology