Lindgren A C, Ritzén E M
Department of Woman and Child Health, Karolinska Institute, Stockholm, Sweden.
Acta Paediatr Suppl. 1999 Dec;88(433):109-11. doi: 10.1111/j.1651-2227.1999.tb14416.x.
The authors have followed 18 prepubertal children (3-12 years of age) with Prader-Willi syndrome during 5 years of growth hormone (GH) treatment. Initially, all the children participated in a randomized, controlled GH trial, conducted to assess the effects of GH treatment on growth, body composition and behaviour. GH was administered to group A (n = 9) at a dose of 0.1 IU/kg/day (0.033 mg/kg/day) for 2 years. Group B (n = 9) was untreated for the first year, but the children were given GH at a dose of 0.2 IU/kg/day (0.066 mg/kg/day) during the second year. Thereafter, all children stopped GH treatment for 6 months and were then restarted with GH at a dose of 0.1 IU/kg/day (0.033 mg/kg/day). During the first year of GH treatment, there was a dramatic increase in height SDS in both groups. The attained height percentile was maintained during the continued GH treatment. Five years after the start of GH treatment, mean height SDS is still above average for age. Four children have reached final height, all within 2 SD of target height. During the first year of GH treatment, body mass index (BMI) SDS decreased significantly from 3.0 to 1.5 SDS in group A and from 2.8 to 1.2 SDS in group B, but it increased again during the 6-month period without treatment. Following the restart of GH treatment, BMI SDS has stabilized at 1.7 SDS for group A and 2.5 SDS for group B. In 16 of 18 patients, fasting insulin, glucose and the A1c fraction of glycosylated haemoglobin remained within normal ranges during 5 years of GH treatment. Following a period of rapid weight gain, two children have developed non-insulin-dependent diabetes mellitus. Glucose homeostasis returned to normal when GH treatment was withdrawn. In conclusion, GH treatment has a proven favourable effect on growth and body composition in patients with Prader-Willi syndrome. Treatment should be individualized, and close surveillance of glucose homeostasis is needed, especially if the patient is severely obese.
作者对18名患有普拉德-威利综合征的青春期前儿童（3至12岁）进行了为期5年的生长激素（GH）治疗随访。最初，所有儿童都参与了一项随机对照GH试验，该试验旨在评估GH治疗对生长、身体成分和行为的影响。A组（n = 9）以0.1 IU/kg/天（0.033 mg/kg/天）的剂量接受GH治疗，为期2年。B组（n = 9）在第一年未接受治疗，但在第二年儿童接受了0.2 IU/kg/天（0.066 mg/kg/天）剂量的GH治疗。此后，所有儿童停止GH治疗6个月，然后以0.1 IU/kg/天（0.033 mg/kg/天）的剂量重新开始使用GH治疗。在GH治疗的第一年，两组儿童的身高标准差评分（SDS）都有显著增加。在持续的GH治疗期间，所达到的身高百分位数得以维持。GH治疗开始5年后，平均身高SDS仍高于年龄平均水平。4名儿童已达到最终身高，均在目标身高的2个标准差范围内。在GH治疗的第一年，A组的体重指数（BMI）SDS从3.0显著降至1.5 SDS，B组从2.8降至1.2 SDS，但在未治疗的6个月期间又再次升高。重新开始GH治疗后，A组的BMI SDS稳定在1.7 SDS，B组稳定在2.5 SDS。在18名患者中的16名中，空腹胰岛素、血糖和糖化血红蛋白的A1c分数在5年的GH治疗期间保持在正常范围内。在经历了一段时间的快速体重增加后，两名儿童患上了非胰岛素依赖型糖尿病。停用GH治疗后，葡萄糖稳态恢复正常。总之，GH治疗对普拉德-威利综合征患者的生长和身体成分有已证实的有利影响。治疗应个体化，并且需要密切监测葡萄糖稳态，特别是如果患者严重肥胖。
