Recombinant PTH Infusion in a Child With Sanjad-Sakati Syndrome Refractory to Conventional Therapy

Ibrahim Bali1,2, Reem Al Khalifah1

  • 1Division of Pediatric Endocrinology, Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia, 11421- P. O. Box 800.

JCEM Case Reports
|April 24, 2024
PubMed

Insights

Hypoparathyroidism in Sanjad-Sakati syndrome can cause severe hypocalcemia. A newborn responded to parathyroid hormone (PTH) subcutaneous infusion, demonstrating its effectiveness for refractory cases.

Area of Science:

  • Pediatric Endocrinology
  • Genetics
  • Metabolic Disorders

Background:

  • Sanjad-Sakati syndrome is a rare genetic disorder.
  • Hypoparathyroidism is a common endocrinological manifestation in this syndrome.
  • Standard treatment involves calcium and active vitamin D, which can be insufficient.

Observation:

  • A newborn with Sanjad-Sakati syndrome presented with severe, persistent hypocalcemia from birth.
  • Initial treatments with high-dose oral/IV calcium and vitamin D analogues were ineffective.
  • Intermittent parathyroid hormone (PTH) subcutaneous injections provided temporary improvement.

Findings:

  • Continuous subcutaneous PTH infusion via a pump initially improved calcium levels but led to iatrogenic hypercalcemia.
  • Subsequent management involved lower doses of calcium carbonate and alfacalcidol, meeting average requirements.
  • PTH subcutaneous infusion demonstrated potential efficacy in refractory hypocalcemia, aiding hospital discharge.

Implications:

  • Continuous PTH infusion may be a viable option for severe, refractory hypocalcemia in Sanjad-Sakati syndrome.
  • Careful monitoring and dosage adjustments are crucial to prevent complications like hypercalcemia.
  • This case highlights the potential of advanced PTH delivery systems in managing complex pediatric endocrine disorders.