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Title of Journal: Pediatr Nephrol

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Abbravation: Pediatric Nephrology

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Springer Berlin Heidelberg

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DOI

10.1016/j.arth.2016.04.004

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ISSN

1432-198X

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Cinacalcet as adjunctive therapy in pseudohypopara

Authors: Tarak Srivastava Jeffrey Krudys Neil J Mardis Judith SebestyenVanSickle Uri S Alon
Publish Date: 2015/12/01
Volume: 31, Issue: 5, Pages: 795-800
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Abstract

In patients with pseudohypoparathyroidism type 1b PHP1b due to a tissuespecific imprinting defect in the Gprotein αsubunit skeletal disorders can arise from the bones being sensitive to parathyroid hormone PTH while the kidneys remain resistant to this hormoneWe report a 48yearold girl with PHP1b who presented with an abnormal gait severe skeletal changes and elevated levels of serum PTH 2844 pg/ml phosphate 72 mg/dl and bone turnover markers Traditional treatment with calcium and calcitriol failed to suppress PTH secretion which was still elevated at 2877 pg/ml after 14 months of therapy nor did it correct the other clinical biochemical and radiographic abnormalities The addition of cinacalcet to the treatment regimen over the subsequent 32 months resulted in normalization of serum PTH 58 ng/ml phosphate 49 mg/dl and bone turnover markers and resolution of the radiographic changes with no adverse effects notedInformed consent was given by the family of our patient to share our experience with cinacalcet in PHP1b We appreciate their support and confidence in us over these years All authors have contributed to either the patient’s clinical care data analysis and/or writing up of the manuscript The study was supported by the Sam and Helen Kaplan Research Fund in Pediatric Nephrology and Eric McClure Research Fund in Pediatric Bone and Mineral Disorders

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