Stamoyannou L, Karachaliou F, Neou P, Papataxiarchou K, Pistevos G, Bartsocas C S
Department of Pediatrics, Faculty of Nursing, University of Athens, Greece.
Am J Med Genet. 1997 Oct 3;72(1):71-6.
The efficacy and safety of recombinant human growth hormone (hGH) administration was studied in children with achondroplasia. Fifteen children with achondroplasia, seven boys (4.8-12.2 years of age) and 12 girls (5.7-2.2 years of age), were treated daily with hGH at a dosage of 1 IU/kg/week. Auxological assessments were performed 6 months before, at initiation of, and at 6, 12, and 24 months following initiation of growth hormone (GH) therapy. Before initiating GH therapy, hypothalamic-pituitary and thyroid functions were evaluated. Levels of serum insulin-like growth factor (IGF)-I and IGF binding protein (BP)-3 (IGFBP-3) were assessed, as was GH response to provocative stimuli. GH responses in two stimulation tests were normal for all but three children. During the first semester of GH treatment, a significant increase in height velocity (HV), from 3.2 to 8.3 cm/year, was observed in all children. However, during the second semester, a relative decrease in growth rate was observed. By the end of the first year, HV had increased from 3.2 to 6.9 cm/year (mean, 3.7 cm/year; range, 1.1-8 cm/year) in 13 children and remained unchanged in two children. HV declined progressively during the next 12 months and, by the end of the second year of treatment, had increased in seven of the nine children who had completed 2 years of therapy (mean increase, 3.1 cm/year); two children did not respond to GH therapy, as shown by the lack of increase in HV. Sitting-height (SH) to standing-height ratio % (SH%) remained unchanged throughout GH therapy, and no significant change in skeletal maturation was observed. In conclusion, hGH treatment resulted in an increased growth rate in some children with achondroplasia; however, this increase waned during the second year of treatment. Children with the lowest pretreatment HVs seemed to benefit most from GH therapy. Nonetheless, the usefulness of GH treatment in achondroplasia will be known only when a study of final height is completed.
研究了重组人生长激素（hGH）治疗软骨发育不全患儿的疗效和安全性。15名软骨发育不全患儿，7名男孩（4.8 - 12.2岁）和12名女孩（5.7 - 12.2岁），每天接受剂量为1 IU/kg/周的hGH治疗。在生长激素（GH）治疗开始前6个月、开始时以及开始后6、12和24个月进行了体格评估。在开始GH治疗前，评估了下丘脑 - 垂体和甲状腺功能。评估了血清胰岛素样生长因子（IGF）-I和IGF结合蛋白（BP）-3（IGFBP - 3）水平，以及GH对刺激试验的反应。除3名儿童外，所有儿童在两项刺激试验中的GH反应均正常。在GH治疗的第一学期，所有儿童的身高增长速度（HV）均显著增加，从3.2厘米/年增至8.3厘米/年。然而，在第二学期，观察到生长速率相对下降。到第一年末，13名儿童的HV从3.2厘米/年增至6.9厘米/年（平均，3.7厘米/年；范围，1.1 - 8厘米/年），2名儿童的HV保持不变。在接下来的12个月中，HV逐渐下降，到治疗第二年末，完成2年治疗的9名儿童中有7名的HV有所增加（平均增加3.1厘米/年）；2名儿童对GH治疗无反应，表现为HV未增加。在整个GH治疗过程中，坐高（SH）与身高比百分比（SH%）保持不变，未观察到骨骼成熟有显著变化。总之，hGH治疗使一些软骨发育不全患儿的生长速率增加；然而，这种增加在治疗的第二年减弱。治疗前HV最低的儿童似乎从GH治疗中获益最大。尽管如此，只有在完成最终身高研究后，才能知道GH治疗在软骨发育不全中的有用性。
