Impact of periprocedural subcutaneous parathyroid hormone on control of hypocalcaemia in APS‐1/APECED patients undergoing invasive procedures. (26th December 2020)

• Record Type: Journal Article
• Authors: Winer, Karen K.; Schmitt, Monica M.; Ferre, Elise M. N.; Fennelly, Kevin P.; Olivier, Kenneth N.; Heller, Theo; Lionakis, Michail S.
• Is Part Of: Clinical endocrinology. Volume 94:Number 3(2021)
• Page Start: 377
• Page End: 383
• Publication Date: 2020-12-26
• Subjects: Endocrinology -- Periodicals; 616.4005
• DOI: 10.1111/cen.14335
• Languages: English
• ISSNs: 0300-0664

Abstract

Abstract: Context: The monogenic disorder autoimmune polyendocrine syndrome type 1 (APS‐1) or autoimmune polyendocrinopathy‐candidiasis‐ectodermal dystrophy (APECED) manifests frequently with hypoparathyroidism, which requires treatment with oral supplementation with calcium and active vitamin D analogs. The majority of APS‐1/APECED patients also suffer from intestinal malabsorption, which complicates the management of hypoparathyroidism and may lead to refractory severe hypocalcaemia. In such situations, reliance on intravenous calcium carries a high risk of nephrocalcinosis and renal damage. Methods: Here, we report our experience of periprocedural subcutaneous administration of recombinant human parathyroid hormone (rhPTH 1‐34) in APS‐1/APECED patients. Serum calcium was measured up to five times within the 36‐hour period starting the evening before the scheduled procedure and ending the morning following the procedure. Results: Twenty‐seven APS‐1/APECED patients with hypoparathyroidism (aged 4‐67 years) underwent 31 invasive gastrointestinal and/or pulmonary procedures. The patients received an average rhPTH1‐34 dose of 9.6 ± 1.4 µg by subcutaneous injection. 92% of the adults and 54% of children in our cohort had evidence of nephrocalcinosis. Mean calcium levels remained stable and ranged from 2.06 to 2.17 mmol/L with minimal fluctuation. None of our patients experienced periprocedural adverse events connected with hypocalcaemia. Conclusion: rhPTH 1‐34 is an alternative to conventional therapy in patients with APS‐1/APECED and hypoparathyroidism undergoing invasive procedures. Subcutaneous PTH1‐34 given directly before and after procedures resulted in well‐controlled serum calcium levels maintained in the low‐normal range and avoided the need for intravenous calcium which may contribute to renal calcifications and tubular damage.