Continuous Subcutaneous Recombinant Parathyroid Hormone (1-34) Infusion in the management of childhood Hypoparathyroidism associated with Malabsorption

Background/ Aims: Hypoparathyroidism associated with malabsorption can be particularly challenging to manage due to limited and erratic intestinal absorption of calcium and vitamin D analogues, resulting in episodes of hypo- or hypercalcaemia. We evaluated the role of continuous subcutaneous recombinant parathyroid (rhPTH 1-34) hormone infusion (CSPI) in children with hypoparathyroidism associated with intestinal malabsorption resistant to conventional therapy.
Method: Four patients (8 - 13 years), with symptomatic hypocalcaemia resistant to conventional therapy were started on CSPI (follow up 3-8 years), in two paediatric endocrinology units in Europe.
Results: Serum calcium normalised within 48 hours of commencing treatment in all 4 patients. An average rhPTH 1-34 dose of 0.4 µg/kg/day resulted in a substantial reduction in symptomatic hypocalcaemia and hypo/ hypercalcaemia-related hospital admissions. An increased alkaline phosphatase activity was noted in the first six months on CSPI, indicating increase in bone turnover. In 2 patients with elevated urinary calcium excretion pre CSPI, this normalised in the first year on treatment. No significant side effects were noticed in the short or long term, with patient-reported preference of CSPI over conventional treatment.
Conclusion: CSPI is a promising and effective treatment option for managing hypocalcaemia and hyperphosphatemia in children with hypoparathyroidism associated with intestinal malabsorption.