Hypoparathyroidism is a rare endocrine disorder characterized by hypocalcaemia and hyperphosphatemia, due to absent or inappropriately low parathyroid hormone levels. For management of chronic hypoparathyroidism, current treatment options involve oral calcium and vitamin D. This standard treatment cannot resolve all problematic aspects of the disease, such as abnormal bone remodeling and reduced quality of life, and is associated with long-term complications, including nephrolithiasis, nephrocalcinosis, renal impairment, cataracts and cerebral calcifications. AREAS COVERED: In 2015, the FDA (Food and Drug Administration) approved rhPTH (1-84), named Natpara®, a bioengineered recombinant human PTH, for the management of hypoparathyroidism of any etiology, except Autosomal Dominant Hypocalcemia, not well controlled with calcium and active vitamin D. Herein, the authors review its chemistry, pharmacodynamics, pharmacokinetics and clinical efficacy for hypoparathyroidism. EXPERT OPINION: Replacement therapy with rhPTH (1-84) is a fundamental step in the treatment of chronic hypoparathyroidism in cases not well controlled by standard therapy, providing the natural hormone that is lacking for the maintenance of normal calcium levels, and reducing long-term risks associated with conventional therapy. Nevertheless, given the chronic nature of the disease, in the future, further studies will have to be performed to evaluate long-term efficacy and safety of the drug.

Natpara for the treatment of hypoparathyroidism / Marcucci, G; Della Pepa, G; Brandi, MARIA LUISA. - In: EXPERT OPINION ON BIOLOGICAL THERAPY. - ISSN 1471-2598. - ELETTRONICO. - 16(11):(2016), pp. 1417-1424. [10.1080/14712598.2016.1238455]

Natpara for the treatment of hypoparathyroidism

Marcucci, G;BRANDI, MARIA LUISA
2016

Abstract

Hypoparathyroidism is a rare endocrine disorder characterized by hypocalcaemia and hyperphosphatemia, due to absent or inappropriately low parathyroid hormone levels. For management of chronic hypoparathyroidism, current treatment options involve oral calcium and vitamin D. This standard treatment cannot resolve all problematic aspects of the disease, such as abnormal bone remodeling and reduced quality of life, and is associated with long-term complications, including nephrolithiasis, nephrocalcinosis, renal impairment, cataracts and cerebral calcifications. AREAS COVERED: In 2015, the FDA (Food and Drug Administration) approved rhPTH (1-84), named Natpara®, a bioengineered recombinant human PTH, for the management of hypoparathyroidism of any etiology, except Autosomal Dominant Hypocalcemia, not well controlled with calcium and active vitamin D. Herein, the authors review its chemistry, pharmacodynamics, pharmacokinetics and clinical efficacy for hypoparathyroidism. EXPERT OPINION: Replacement therapy with rhPTH (1-84) is a fundamental step in the treatment of chronic hypoparathyroidism in cases not well controlled by standard therapy, providing the natural hormone that is lacking for the maintenance of normal calcium levels, and reducing long-term risks associated with conventional therapy. Nevertheless, given the chronic nature of the disease, in the future, further studies will have to be performed to evaluate long-term efficacy and safety of the drug.
2016
16(11)
1417
1424
Marcucci, G; Della Pepa, G; Brandi, MARIA LUISA
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Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1068119
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