Impact of periprocedural subcutaneous parathyroid hormone on control of hypocalcaemia in APS-1/APECED patients undergoing invasive procedures.

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.

• Authors: Karen K Winer, Monica M Schmitt, Elise M N Ferre, Kevin P Fennelly, Kenneth N Olivier, Theo Heller, Michail S Lionakis
• Journal: Clinical endocrinology (Clin Endocrinol (Oxf)) Vol. 94 Issue 3 Pg. 377-383 (03 2021) ISSN: 1365-2265 [Electronic] England
• PMID: 32955743 (Publication Type: Journal Article, Research Support, N.I.H., Intramural)
• Copyright: © 2020 John Wiley & Sons Ltd.

Chemical References

• Parathyroid Hormone
• Calcium

Topics

• Adult
• Calcium (blood)
• Child
• Humans
• Hypocalcemia (drug therapy)
• Hypoparathyroidism (drug therapy)
• Parathyroid Hormone (administration & dosage)
• Polyendocrinopathies, Autoimmune (blood, drug therapy)