ESPEYB19 5. Bone, Growth Plate and Mineral Metabolism Advances in clinical practice (5 abstracts)
5.4. PTH infusion for seizures in autosomal dominant hypocalcemia type 1
Sastre A , Valentino K , Hannan FM , Lines KE , Gluck AK , Stevenson M , Ryalls M , Gorrigan RJ , Pullen D , Buck J , Sankaranarayanan S , Allgrove J , Thakker RV & Gevers EF
N Engl J Med. 2021 Jul 8;385(2):189-191.Abstract: https://pubmed-ncbi-nlm-nih-gov.proxy.kib.ki.se/34233101/
In brief: This study retrospectively analysed a cohort of patients with autosomal dominant hypocalcemia type 1 and recurrent hypocalcemic seizures treated with continuous subcutaneous PTH (1-34) infusions using insulin pumps. Compared to conventional therapy, PTH (1-34) infusion resulted in higher mean serum calcium and magnesium, lower mean serum phosphorus and fewer seizures.
Commentary: Autosomal dominant hypocalcemia type 1 (ADH1) is caused by gain-of-function mutations in the calcium-sensing receptor gene (CASR). CaSRs are expressed in the parathyroid glands regulate parathyroid hormone (PTH) secretion whereas CaSRs expressed in the kidney regulate calcium reabsorption. Conventional treatment with calcium and vitamin D analogs tend to exacerbate hypercalciuria and increase the risk for nephrocalcinosis and renal complications in patients with ADH1.
The study reports a retrospective review of six ADH1 patients with recurrent hypocalcemic seizures on conventional treatment who were selected for treatment with continuous subcutaneous PTH (1-34) infusions. PTH infusion therapy has previously been assessed in a clinical trial and shown to modestly improve calcium homeostasis and bone turnover markers compared to twice daily PTH (1). In the Sastre A et al. study, they found that PTH (1-34) infusion resulted in higher mean serum calcium and magnesium, lower mean serum phosphorus and fewer seizures compared to calcium and vitamin D analogue treatment. However, the variability in serum calcium levels remained high with infusion therapy. One benefit of the current study is that it shows that continuous PTH pump treatment can be feasible in clinical settings and therefore could be considered in selected patients. Even more useful for this group of patients, long-acting PTH and maybe especially calciolytic drugs may soon be available and help improve the management and outcome for patients with ADH1 (2).
References: 1. Winer KK, Fulton KA, Albert PS, Cutler GB Jr. Effects of pump versus twice-daily injection delivery of synthetic parathyroid hormone 1-34 in children with severe congenital hypoparathyroidism. J Pediatr. 2014 Sep;165(3):556–63.e1. doi: 10.1016/j.jpeds.2014.04.060.2. Roberts MS, Gafni RI, Brillante B, Guthrie LC, Streit J, Gash D, Gelb J, Krusinska E, Brennan SC, Schepelmann M, Riccardi D, Bin Khayat ME, Ward DT, Nemeth EF, Rosskamp R, Collins MT. Treatment of Autosomal Dominant Hypocalcemia Type 1 With the Calcilytic NPSP795 (SHP635). J Bone Miner Res. 2019 Sep;34(9):1609–1618. doi: 10.1002/jbmr.3747.
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ESPE Yearbook of Paediatric Endocrinology
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