Related Experiment Videos
Response to crystalline 1alpha-hydroxyvitamin D3 in vitamin D dependency
Pediatric Research
|July 1, 1975
Summary
1alpha-hydroxycholecalciferol (1alpha-OH-D3) effectively treated autosomal recessive vitamin D dependency (ARVDD). This vitamin D analog rapidly corrected hypocalcemia and healed rickets, showing promise for similar genetic disorders.
Area of Science:
- Endocrinology
- Metabolic Bone Disease
Background:
- Autosomal recessive vitamin D dependency (ARVDD) requires high doses of vitamin D2 for management.
- Vitamin D2 withdrawal leads to the recurrence of vitamin D deficiency symptoms.
Purpose of the Study:
- To evaluate the therapeutic efficacy of 1alpha-hydroxycholecalciferol (1alpha-OH-D3) in patients with ARVDD.
Main Methods:
- Three patients with ARVDD were treated with oral 1alpha-OH-D3 at 1-3 mug/24 hr.
- Clinical and biochemical parameters, including serum calcium, phosphorus, and amino acid excretion, were monitored.
Main Results:
- 1alpha-OH-D3 treatment rapidly corrected hypocalcemia and suppressed parathyroid hormone-dependent renal amino acid loss.
- Rickets healed within 7-9 weeks of treatment with 1alpha-OH-D3 alone.
- Serum calcium levels decreased within two weeks of 1alpha-OH-D3 withdrawal.
Conclusions:
- 1alpha-OH-D3 is an effective therapeutic agent for ARVDD, offering a simpler synthesis and comparable efficacy to vitamin D hormone analogs.
- 1alpha-OH-D3 is recommended as the treatment of choice for ARVDD and analogous conditions.