1alpha-hydroxyvitamin D3 in the treatment of nutritional and metabolic rickets and osteomalacia.
Five patients with nutritional osteomalacia or rickets and six children with rickets unresponsive to physiological doses of vitamin D were treated with 1alpha-hydroxyvitamin D3 (1alpha-OHD3). Patients with nutritional osteomalacia responded to 1--2 microgram/day of 1alpha-OHD3. The most striking findings were rises in plasma calcium and, in one case, a decrease in faecal calcium. In some cases there was a rise in plasma phosphorus, alkaline phosphatase remained unchanged. There was radiological healing. In three patients with cystinosis and one with hypophosphataemia and Barrter's syndrome 2 microgram of 1alpha-OHD3 produced healing of rickets. Plasma phosphate rose on treatment, possibly by a suppression of parathyroid activity. The response to such low doses of 1alpha-OHD3 suggests impaired 1alpha-hydroxylation of 25-hydroxyvitamin D in these patients. A patient with intestinal malabsorption was resistant to high doses of 1alpha-OHD3 by mouth but responded to parenteral administration. A boy with osteopetrosis and the biochemical changes of rickets was resistant to large doses of 1alpha-OHD3 presumably because of failure of osseous response.
D000293:Adolescent; D000328:Adult; D000368:Aged; D002118:Calcium / Q000378:metabolism; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D006887:Hydroxycholecalciferols / Q000627:therapeutic use*; D007015:Hypophosphatemia, Familial / Q000188:drug therapy*; D007223:Infant; D008297:Male; D008875:Middle Aged; D010018:Osteomalacia / Q000188:drug therapy*; D010710:Phosphates / Q000378:metabolism; D014808:Vitamin D Deficiency / Q000188:drug therapy*
D006887:Hydroxycholecalciferols; D010710:Phosphates; D002118:Calcium