Rozendaal L, le Cessie S, Wit J M, Hennekam R C M
Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
J Pediatr. 2005 Nov;147(5):674-9. doi: 10.1016/j.jpeds.2005.06.011.
To determine the accuracy and precision of 2 height-prediction methods in Marfan syndrome and to assess the growth-reductive effect and side effects of sex hormone treatment.
In a retrospective study in 31 untreated (17 boys) and 43 treated patients (21 boys) with Marfan syndrome, we assessed bone age and predicted adult height by 2 methods. The accuracy of the methods was assessed in the untreated group. The effect of therapy was corrected for outcome in the untreated group and other confounding variables with multivariate analysis.
Accuracy strongly varied with sex, chronological age, and prediction method. Overall precision was low. Treatment was started at a mean age of 12.8 +/- 1.4 years (boys) and 11.4 +/- 1.2 years (girls). With multiple regression analysis, a statistically significant effect was observed only in boys using a pharmacologic dosage (5.5 cm, 95% CI 0.96-10.1 cm; P = .02). Side effects were worsening of acne and weight and muscle gain.
In adolescents with Marfan syndrome, the accuracy and precision of 2 height-prediction methods were limited. The apparent growth-reductive effect of sex hormone treatment appears similar to earlier reports on adolescents with constitutional tall stature. There were no clinically important short-term side effects.
确定两种身高预测方法在马凡综合征中的准确性和精确性，并评估性激素治疗的生长抑制作用和副作用。
在一项对31例未接受治疗的（17例男孩）和43例接受治疗的马凡综合征患者（21例男孩）的回顾性研究中，我们通过两种方法评估骨龄并预测成年身高。在未治疗组中评估这些方法的准确性。通过多变量分析对未治疗组的结果以及其他混杂变量进行校正，以评估治疗效果。
准确性因性别、实足年龄和预测方法而有很大差异。总体精确性较低。治疗开始时男孩的平均年龄为12.8±1.4岁，女孩为11.4±1.2岁。通过多元回归分析，仅在使用药物剂量的男孩中观察到有统计学意义的效果（5.5厘米，95%可信区间0.96 - 10.1厘米；P = 0.02）。副作用包括痤疮加重、体重增加和肌肉量增加。
在患有马凡综合征的青少年中，两种身高预测方法的准确性和精确性有限。性激素治疗明显的生长抑制作用似乎与早期关于体质性身材高大青少年的报道相似。没有临床上重要的短期副作用。
