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Growth Hormone Therapy in Hypophosphatemic Rickets

Darrell M. Wilson, MD; Phillip D. K. Lee, MD; Alan H. Morris, MD; Edward O. Reiter, MD; Joseph M. Gertner, MB, MRCP; Robert Marcus, MD; Valerie E. Quarmby, PhD; Ron G. Rosenfeld, MD
Am J Dis Child. 1991;145(10):1165-1170. doi:10.1001/archpedi.1991.02160100097031.
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• The effects of growth hormone therapy on the biochemical measures of bone metabolism were studied in 11 children aged 3.5 to 17 years who had familial hypophosphatemic rickets; five were male. Subjects were maintained on a regimen of stable doses of conventional therapy (calcitriol and phosphate). Subjects were studied at baseline receiving conventional therapy and during three sequential treatment periods: no therapy (4 weeks), growth hormone only (0.05 mg/kg per day for 4 weeks), and conventional therapy plus growth hormone (2 weeks). The nine youngest subjects were continued on a regimen of triple therapy for an additional 24 weeks. Serum phosphate averaged 0.93±0.13 mmol/L (mean±SD) at entry and decreased when the subjects were not receiving any therapy. During the 4 weeks of growth hormone only treatment, phosphate rose in all 11 subjects (0.70±0.08 mmol/L to 0.83±0.08 mmol/L). With triple therapy, phosphate remained higher than with no therapy. Calcitriol, osteocalcin, and parathyroid hormone increased as the subjects received growth hormone alone. Insulinlike growth factor I z scores rose significantly in response to growth hormone therapy alone. All nine subjects receiving 6 months of triple therapy increased their growth rate z scores. Exogenous growth hormone therapy may be useful in familial hypophosphatemic rickets.

(AJDC. 1991;145:1165-1170)

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