非永久性特发性生长激素缺乏症患者成人身高的评价

A. Murianni, A. Lussu, C. Guzzetti, A. Ibba, Letizia Casula, M. Salerno, M. Cappa, S. Loche
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摘要

背景:一些研究已经评估了IGF-1在生长激素缺乏症(GHD)诊断中的作用。根据最近的一项研究,IGF-1浓度的- 1.5标准差评分(SDS)似乎是区分GHD儿童和正常儿童的最佳截止值。由于刺激试验和IGF-1测定本身的诊断准确性较差,因此该值应与诊断GHD的其他临床和生化参数一起解释。我们的研究旨在评估身高矮小且基线IGF-1浓度≤- 1.5 SDS的儿童的成人身高(AH)。设计:本回顾性分析包括52名儿童和青少年,在过去30年里因身材矮小和/或生长速度减慢而接受诊断程序,以评估可能的GHD。只有在第一次测试时基线IGF-1值≤- 1.5 SDS的患者被纳入研究。有遗传性/器质性GHD或基础疾病的患者不包括在内。方法:病例组为非永久性、特发性、孤立性GHD患者24例(男孩13例,女孩11例)(经精氨酸(Arg)、胰岛素耐量试验(ITT)、可拉定(Clo)两种刺激试验后峰值GH < 10 μg/L,或经GHRH+精氨酸(GHRH+Arg)试验后峰值GH <20 μg/L;正常的核磁共振;正常的GH;和/或正常的IGF-1浓度接近ah)。这些患者给予生长激素(25 ~ 35 μg/kg/例)治疗,直至接近ah。对照组包括28例特发性身材矮小患者(23名男孩和5名女孩)(ISS,刺激性测试后GH峰值正常,没有其他原因导致他们矮小的证据)。两组基础IGF-1≤- 1.5 SDS。结果:两组AH和身高增加具有可比性。在病例组中,诊断时的平均IGF-1 SDS明显低于重新检测时的水平。结论:在本研究中,接受治疗的特发性GHD患者和未接受治疗的ISS患者均达到相似的近ahs(在目标身高范围内),且身高SDS的增加相似。因此,用rhGH治疗这些患者的疗效可能是值得怀疑的。这可能是由于患有ISS的儿童经常被误诊为GHD。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Evaluation of Adult Height in Patients with Non-Permanent Idiopathic GH Deficiency
Background: Several studies have evaluated the role of IGF-1 in the diagnosis of growth hormone deficiency (GHD). According to a recent study, an IGF-1 concentration of a −1.5 standard deviation score (SDS) appeared to be the best cut-off for distinguishing between children with GHD and normal children. This value should always be interpreted in conjunction with other clinical and biochemical parameters for the diagnosis of GHD, since both stimulation tests and IGF-1 assays have poor diagnostic accuracy by themselves. Our study was designed to evaluate the adult height (AH) in children with short stature and baseline IGF-1 concentration ≤ −1.5 SDS. Design: This retrospective analysis included 52 children and adolescents evaluated over the last 30 years for short stature and/or deceleration of the growth rate who underwent diagnostic procedures to evaluate a possible GHD. Only the patients who had baseline IGF-1 values ≤−1.5 SDS at the time of the first test were included in the study. Patients with genetic/organic GHD or underlying diseases were not included. Method: The case group consisted of 24 patients (13 boys and 11 girls) with non-permanent, idiopathic, and isolated GHD (peak GH < 10 μg/L after two provocative tests with arginine (Arg), insulin tolerance test (ITT), and clonidine (Clo), or <20 μg/L after GHRH + Arginine (GHRH+Arg); normal MRI; normal GH; and/or normal IGF-1 concentrations at near-AH). These patients were treated with GH (25–35 μg/kg/die) until near-AH. The control group consisted of 28 patients (23 boys and 5 girls) with idiopathic short stature (ISS, normal peak GH after provocative testing, no evidence of other causes for their shortness). Both groups had basal IGF-1 ≤−1.5 SDS. Results: AH and height gain in both groups were comparable. In the group of cases, mean IGF-1 SDS at the time of diagnosis was significantly lower than the levels found at the time of retesting. Conclusions: In this study, both treated patients with idiopathic GHD and untreated patients with ISS reached similar near-AHs (within target height) and showed similar increases in SDS for their height. Thus, the efficacy of treatment with rhGH in these patients may be questionable. This could be due to the fact that children with ISS are frequently misdiagnosed with GHD.
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