Reusz G S, Miltényi G, Stubnya G, Szabó A, Horváth C, Byrd D J, Péter F, Tulassay T
Ist Department of Pediatrics, Semmelweis Medical University, Budapest, Hungary.
Pediatr Nephrol. 1997 Oct;11(5):573-7. doi: 10.1007/s004670050340.
The impact of recombinant human growth hormone (rhGH) treatment on growth, bone mineral metabolism, and bone mineral density (BMD) was evaluated in six children (3 girls, 3 boys) with familial hypophosphatemic rickets (XLH). Five were prepubertal (aged 6-8.8 years), one 15.3-year-old boy had combined XLH and GH deficiency, but had not been treated with rhGH previously. rhGH was administered daily for 1 year, at a dose of 1 IU/kg per week, combined with 1,25-dihydroxyvitamin D3 and oral phosphate therapy. Z scores for growth velocity and height improved significantly (-2.9 vs. 2.5, P < 0.01, and -2.2 vs. -1.5, P < 0.01, respectively). However, the ratio of Z score for height to that of subischial leg length decreased significantly (0.65 vs. 0.43, P < 0.01), indicating disproportionate growth in favor of the trunk. The height-corrected BMD Z increased slightly (-0.99 vs. -0.94, P < 0.05). A slight increase in serum phosphate occurred (0.78 vs. 0.88 mmol/l, P < 0.02). Tubular reabsorption of phosphate/glomerular filtration rate increased from 0.45 mmol/l to 0.55 mmol at 6 months (P < 0.02), but returned to the initial level at 12 months. These results indicate that children with XLH can benefit from the positive effect of rhGH on growth, however treatment could aggravate the already existing tendency to disproportionate growth. GH production should be evaluated in poorly growing patients with XLH, because it can mask GH deficiency. rhGH can be safely combined with conventional treatment in XLH. Further studies are needed to determine the effect of treatment on final height and maximal BMD.
在六名患有家族性低磷性佝偻病（XLH）的儿童（3名女孩，3名男孩）中评估了重组人生长激素（rhGH）治疗对生长、骨矿物质代谢和骨矿物质密度（BMD）的影响。五名儿童处于青春期前（年龄6 - 8.8岁），一名15.3岁男孩同时患有XLH和生长激素缺乏症，但此前未接受过rhGH治疗。rhGH每周以1 IU/kg的剂量每日给药1年，同时联合1,25 - 二羟维生素D3和口服磷酸盐治疗。生长速度和身高的Z评分显著改善（分别为 - 2.9对2.5，P < 0.01，以及 - 2.2对 - 1.5，P < 0.01）。然而，身高Z评分与坐骨结节下腿长Z评分的比值显著降低（0.65对0.43，P < 0.01），表明生长不成比例，躯干生长更占优势。身高校正后的BMD Z评分略有增加（ - 0.99对 - 0.94，P < 0.05）。血清磷酸盐略有升高（0.78对0.88 mmol/l，P < 0.02）。6个月时磷酸盐的肾小管重吸收/肾小球滤过率从0.45 mmol/l增加到0.55 mmol（P < 0.02），但在12个月时恢复到初始水平。这些结果表明，XLH患儿可从rhGH对生长的积极作用中获益，然而治疗可能会加剧已有的生长不成比例的倾向。对于生长缓慢的XLH患者应评估生长激素分泌情况，因为它可能掩盖生长激素缺乏症。rhGH可与XLH的传统治疗安全联合使用。需要进一步研究以确定治疗对最终身高和最大BMD的影响。
