Author
CARPENTER, T - YALE UNIV, NEW HAVEN, CT | |
INSOGNA, K - YALE UNIV, NEW HAVEN, CT | |
GLORIEUX, F - MCGILL UNIV | |
TRAVERS, R - YALE UNIV, NEW HAVEN, CT | |
CAREY, R - MCGILL UNIV | |
Horst, Ronald | |
COMITE, F - SHRINERS HOSP, MONTREAL | |
KELLER, M - SHRINERS HOSP, MONTREAL |
Submitted to: American Society for Bone and Mineral Research
Publication Type: Abstract Only Publication Acceptance Date: 9/13/1995 Publication Date: N/A Citation: N/A Interpretive Summary: Technical Abstract: Oral calcitriol and phosphate salts are considered standard therapy (SRx) for X-linked hypophosphatemic rickets (XLH), but result in incomplete skeletal healing and the frequent complication of secondary hyperparathyroidism. To seek improved outcomes in XLH, we performed a prospective, placebo-controlled trial of 24,25(OH)2D3 (24,25D), 10 ug daily, in combination with SRx. 15 patients (3-58 yr) on SRx were monitored every 4 hr during 1 day for serum PTH, Ca, and P, and then begun on placebo(pl)_SRx. monitoring was repeated after 1 yr of SRx+pl, and again after one year of SRx+24,25D. Leg radiographs were obtained each year in children. Each of 6 adults underwent 2 bone biopsies (before and after SRx+24,25D). The single- blinded, sequential (placebo-first) study design eliminated any carry- over effects of 24,25D treatment. As shown below, 24,25D, but not placebo, significantly decreased PTH (P<0.0005), whereas Ca and P did not significantly change: Study Entry SRx+Placebo SRx+24,25D Ca (mg/dl)(-/xSEM) 9.20 +/- 0.06 9.21 +/- 0.04 9.29 +/- 0.04 P (mg/dl) 3.05 +/- 0.10 2.93 +/- 0.07 3.13 +/- 0.08 PTH (pM) 32 +/- 2.6 29 +/- 2.1 20 +/- 1.7 |