ECE2019 Guided Posters Calcium and Bone 1 (11 abstracts)
Recombinant human parathyroid hormone 1–84 for the treatment of adults with chronic hypoparathyroidism: Six-year safety and efficacy results of the RACE study
John P Bilezikian 1 , Henry Bone 2 , Bart L Clarke 3 , Douglas Denham 4 , John Germak 5 , Hak-Myung Lee 6 , Michael A Levine 7 , Michael Mannstadt 8 , Munro Peacock 9 , Jeffrey G Rothman 10 , Nicole Sherry 11 , Dolores M Shoback 12 , Tamara J Vokes 13 , Mark L Warren 14 & Nelson B Watts 15
1College of Physicians and Surgeons, Columbia University, New York, USA. 2Michigan Bone & Mineral Clinic, PC, Detroit, USA; 3Mayo Clinic Division of Endocrinology, Diabetes, Metabolism, and Nutrition, Rochester, USA; 4Clinical Trials of Texas, Inc., San Antonio, USA; 5Shire International GmbH, a member of the Takeda group of companies, Zug, Switzerland; 6Shire Human Genetic Therapies, Inc., a member of the Takeda group of companies, Lexington, USA; 7Children’s Hospital of Philadelphia, Philadelphia, USA; 8Massachusetts General Hospital and Harvard Medical School, Boston, USA; 9Indiana University School of Medicine, Indianapolis, USA; 10University Physicians Group – Research Division, Staten Island, USA; 11Shire Human Genetic Therapies, Inc., a member of the Takeda group of companies, Cambridge, USA; 12San Francisco Veterans Affairs Medical Center, University of California, San Francisco, USA; 13University of Chicago Medicine, Chicago, USA; 14Physicians East, PA, Greenville, USA; 15Mercy Health, Cincinnati, USA.
Background: RACE is an open-label study that assessed the long-term safety and efficacy of recombinant human parathyroid hormone 1–84 (rhPTH[1–84]) for the treatment of hypoparathyroidism in adults (ClinicalTrials.gov identifier NCT01297309). Here, we present 6-year safety and efficacy data.
Methods: Patients initially received 25 or 50 μg of rhPTH(1–84) subcutaneously, once daily, with stepwise dose adjustments of 25 μg (up or down) to a maximum of 100 μg/day. rhPTH(1–84) could be titrated and oral calcium (Ca) and calcitriol doses adjusted at any time during the study to maintain albumin-corrected serum Ca levels in the target range (2.0–2.25 mmol/L). A composite efficacy endpoint was the proportion of patients who achieved at least a 50% reduction from baseline (BL) in oral Ca dose (or Ca ≤500 mg/day) and at least a 50% reduction from BL in calcitriol dose (or calcitriol ≤0.25 μg/day), while normalising or maintaining albumin-corrected serum Ca compared with BL value and not exceeding the upper limit of normal for the central laboratory. Data are summarised with descriptive statistics (mean±SD).
Results: The study cohort consisted of 49 patients enrolled at 12 US centres (mean age, 48.1±9.78 years; 81.6% female); data from 34 patients (69.4%) who completed 72 months (M72) of treatment with rhPTH(1–84) as of July 17, 2018, are presented here. Oral Ca and calcitriol doses were reduced by 40.4% and 72.2% at M72, respectively, and albumin-corrected serum Ca levels were maintained within the target range (BL, 2.1±0.17 mmol/L; M72, 2.1±0.17 mmol/L). At M72, 22 of 34 patients (64.7%) achieved the composite efficacy endpoint. Urinary Ca excretion declined from above normal at BL to within the normal range (BL, 8.9±5.01 mmol/d; M72, 5.3±3.22 mmol/d). Mean serum creatinine levels remained stable (BL, 84.7±18.16 μmol/L; M72, 79.7±18.76 μmol/L), as did estimated glomerular filtration rate (BL, 77.7±17.67 mL/min/1.73 m2; M72, 79.4±18.39 mL/min/1.73 m2). Serum phosphorus levels declined from above normal at BL to within normal range (BL, 1.6±0.19 mmol/L; M72, 1.3±0.20 mmol/L); calcium-phosphorus product levels also declined (BL, 3.4±0.51 mmol2/L2; M72, 2.7±0.40 mmol2/L2). Treatment-emergent adverse events and treatment-emergent serious adverse events were reported in 98.0% and 26.5% of patients, respectively; no new safety concerns were identified.
Conclusions: Continuous use of rhPTH(1–84) over 6 years resulted in a favourable safety profile, was effective, and improved key measurements of mineral homeostasis, notably normalisation of urinary calcium.
Volume 63
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Endocrine Abstracts
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