A new paradigm for hypoparathyroidism

Abstract

The management of most hormone deficiency states is straightforward: suspect hormone deficiency based upon clinical presentation, confirm deficiency with laboratory testing, treat with exogenous hormone, and assess for clinical and laboratory improvement. This approach has been the standard for generations for conditions such as type 1 diabetes, growth hormone deficiency, hypothyroidism, and others.One notable exception to this approach has been hypoparathyroidism. With no “hormone replacement,” children with hypoparathyroidism have been treated with combinations of calcitriol, phosphate binders, and thiazide diuretics. This often is a tricky business, with a number of possible complications. A recombinant human parathyroid hormone (PTH) product has been available and used in adults for quite some time. There have been many reports of its off-label use in children. The current issue of the Journal includes an important article that expands the use of the drug.Winer et al, at the National Institutes of Health, describe 12 children with hypoparathyroidism treated with recombinant PTH 1-34 by continuous pump infusion, analogous to the approach increasingly used in type 1 diabetes. In theory, such delivery makes sense in light of the known diurnal variation in PTH levels. When compared with twice-daily administration, pump therapy provided much better metabolic control in this group of patients.Although this therapy is not yet ready for prime time, the study by Winer et al is important. It sets the stage for infantile and pediatric hypoparathyroidism finally fitting into the therapeutic paradigm that has been employed for decades in other deficiency states.Article page 556 The management of most hormone deficiency states is straightforward: suspect hormone deficiency based upon clinical presentation, confirm deficiency with laboratory testing, treat with exogenous hormone, and assess for clinical and laboratory improvement. This approach has been the standard for generations for conditions such as type 1 diabetes, growth hormone deficiency, hypothyroidism, and others. One notable exception to this approach has been hypoparathyroidism. With no “hormone replacement,” children with hypoparathyroidism have been treated with combinations of calcitriol, phosphate binders, and thiazide diuretics. This often is a tricky business, with a number of possible complications. A recombinant human parathyroid hormone (PTH) product has been available and used in adults for quite some time. There have been many reports of its off-label use in children. The current issue of the Journal includes an important article that expands the use of the drug. Winer et al, at the National Institutes of Health, describe 12 children with hypoparathyroidism treated with recombinant PTH 1-34 by continuous pump infusion, analogous to the approach increasingly used in type 1 diabetes. In theory, such delivery makes sense in light of the known diurnal variation in PTH levels. When compared with twice-daily administration, pump therapy provided much better metabolic control in this group of patients. Although this therapy is not yet ready for prime time, the study by Winer et al is important. It sets the stage for infantile and pediatric hypoparathyroidism finally fitting into the therapeutic paradigm that has been employed for decades in other deficiency states. Article page 556 Effects of Pump versus Twice-Daily Injection Delivery of Synthetic Parathyroid Hormone 1-34 in Children with Severe Congenital HypoparathyroidismThe Journal of PediatricsVol. 165Issue 3PreviewTo compare the response with synthetic human parathyroid hormone (PTH) 1-34 delivered by twice-daily injection vs insulin pump in children with severe congenital hypoparathyroidism due to calcium receptor mutation or autoimmune polyglandular syndrome type 1. Full-Text PDF