24,25 dihydroxyvitamin D supplementation corrects hyperparathyroidism and improves skeletal abnormalities in X-linked hypophosphatemic rickets : A clinical research center study

Therapy for X-linked hypophosphatemia (XLH) only partially corrects skeletal lesions and is often complicated by hyperparathyroidism. 24,25(OH)2 D3 improves skeletal lesions in a murine model of XLH and suppresses PTH secretion in animals. Therefore, we undertook a placebo-controlled trial of 24,25(...

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Published in:The journal of clinical endocrinology and metabolism 1996-06, Vol.81 (6), p.2381-2388
Main Authors: CARPENTER, T. O, KELLER, M, GLORIEUX, F. H, GUNDBERG, C. M, POOLE, A. R, INSOGNA, K. L, SCHWARTZ, D, MITNICK, M, SMITH, C, ELLISON, A, CAREY, D, COMITE, F, HORST, R, TRAVERS, R
Format: Article
Language:English
Subjects:
24,25-Dihydroxyvitamin D 3 - adverse effects
24,25-Dihydroxyvitamin D 3 - therapeutic use
Adolescent
Adult
Biological and medical sciences
Bone and Bones - drug effects
Child
Child, Preschool
Female
General and cellular metabolism. Vitamins
Genetic Linkage
Humans
Hyperparathyroidism - drug therapy
Hypophosphatemia, Familial - drug therapy
Hypophosphatemia, Familial - genetics
Male
Medical sciences
Middle Aged
Pharmacology. Drug treatments
Prospective Studies
Single-Blind Method
X Chromosome
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Summary:Therapy for X-linked hypophosphatemia (XLH) only partially corrects skeletal lesions and is often complicated by hyperparathyroidism. 24,25(OH)2 D3 improves skeletal lesions in a murine model of XLH and suppresses PTH secretion in animals. Therefore, we undertook a placebo-controlled trial of 24,25(OH)2 D3 supplementation to standard treatment in patients with XLH to improve bone disease and reduce hyperparathyroid complications. Fifteen subjects with XLH receiving standard treatment [1,25(OH)2 D3 or dihydrotachysterol plus phosphate] were evaluated, supplemented with placebo, and reevaluated one yr later. 24,25(OH)2 D3 supplementation was then begun and studies repeated after another year. Each patient underwent a detailed evaluation of calcium homeostasis over a 24-h period. Rachitic abnormalities were assessed radiographically in children. Adults underwent bone biopsies. 24,25(OH)2 D3 normalized PTH values in nine subjects (peak PTH was 46.5 +/- 6.6 pmol/L at entry, 42.3 +/- 5.9 pmol/L after placebo, and 23.3 +/- 5.4 pmol/L after 24,25(OH)2 D3). Nephrogenous cAMP decreased at night, coincident with the decrease in PTH, and serum phosphorus was slightly greater with 24,25(OH)2 D3. Radiographic features of rickets improved during 24,25(OH)2 D3 supplementation in children, and osteoid surface decreased in adults. 24,25(OH)2 D3 is a useful adjunct to standard therapy in XLH by effecting correction of hyperparathyroidism and improvement of rickets and osteomalacia.
ISSN:0021-972X
1945-7197
DOI:10.1210/jc.81.6.2381