David Hélène, Aupiais Camille, Louveau Baptiste, Quartier Pierre, Jacqz-Aigrain Evelyne, Carel Jean-Claude, Simon Dominique
Assistance Publique-Hôpitaux de Paris, Hôpital Robert Debré, Service d'Endocrinologie Diabétologie Pédiatrique, Centre de Référence des Maladies Endocriniennes Rares de la Croissance, France.
Assistance Publique-Hôpitaux de Paris, Hôpital Robert Debré, Unité d'Epidémiologie Clinique Université Paris Diderot, Sorbonne Paris-Cité, INSERM U1123 et CIC-EC 1426, France.
J Clin Endocrinol Metab. 2017 Dec 1;102(12):4578-4587. doi: 10.1210/jc.2017-01455.
Growth hormone (GH) therapy may improve statural growth outcomes in patients with severe juvenile idiopathic arthritis (JIA).
To evaluate the effect of GH treatment on adult height and to identify determinants of growth outcomes in JIA.
Data from 58 patients with JIA, including 53 receiving GH, enrolled in three prospective clinical trials between 1997 and 2002 were analyzed.
GH (0.056 mg/kg/d [interquartile range (IQR), 0.050 to 0.062]) for a median duration of 6.5 years (IQR, 4.7 to 7.9 years).
Factors associated with a favorable growth outcome (adult height - target height ≤ -1.5 standard deviations) were identified by multivariate logistic regression.
Adult height was available for 48 patients 8.6 years after GH initiation (IQR, 6.0 to 10.2 years). Height standard deviation score (SDS) increased from -2.9 (IQR, -4.4 to -1.6) at baseline to -1.7 (IQR, -3.9 to -0.1) in adulthood (P < 0.001). Median adult height was below target height [SDS, -0.2 (IQR, -1.4 to 0.4); P < 0.001]. Corrected adult height SDS was -1.3 (IQR, -3.0 to -0.2). Growth outcome was favorable in 24 (52.2%) patients. Significant independent determinants of growth outcome were age at GH initiation [adjusted odds ratio (aOR), 0.68 per additional year; 95% confidence interval (CI), 0.47 to 0.99], height at GH initiation (aOR, 2.6 per additional SDS; 95% CI, 1.15 to 5.9), and mean C-reactive protein levels during follow up (aOR, 0.51 per additional 10 mg/L; 95% CI, 0.28 to 0.92).
Long-term GH treatment significantly increased growth in patients with JIA but did not fully restore the genetic growth potential. The response showed marked interindividual variability and was weaker in patients with severe inflammation.
生长激素(GH)治疗可能改善重度幼年特发性关节炎(JIA)患者的身高增长结局。
评估GH治疗对成年身高的影响,并确定JIA患者生长结局的决定因素。
分析了1997年至2002年间纳入三项前瞻性临床试验的58例JIA患者的数据,其中53例接受了GH治疗。
GH(0.056mg/kg/d[四分位数间距(IQR),0.050至0.062]),中位治疗持续时间为6.5年(IQR,4.7至7.9年)。
通过多因素logistic回归确定与良好生长结局(成年身高-目标身高≤-1.5标准差)相关的因素。
在开始使用GH后8.6年(IQR,6.0至10.2年)有48例患者获得了成年身高数据。身高标准差评分(SDS)从基线时的-2.9(IQR,-4.4至-1.6)增加至成年时的-1.7(IQR,-3.9至-0.1)(P<0.001)。成年身高中位数低于目标身高[SDS,-0.2(IQR,-1.4至0.4);P<0.001]。校正后的成年身高SDS为-1.3(IQR,-3.0至-0.2)。24例(52.2%)患者生长结局良好。生长结局的显著独立决定因素为开始使用GH时的年龄[校正优势比(aOR),每增加一岁为0.68;95%置信区间(CI),0.47至0.99]、开始使用GH时的身高(aOR,每增加一个SDS为2.6;95%CI,1.15至5.9)以及随访期间的平均C反应蛋白水平(aOR,每增加10mg/L为0.51;95%CI,0.28至0.92)。
长期GH治疗显著增加了JIA患者的生长,但未完全恢复其遗传生长潜力。反应存在明显的个体差异,在严重炎症患者中较弱。