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描绘转变历程:一项针对170例儿童期起病的生长激素缺乏症患者的单中心研究

Mapping the journey of transition: a single-center study of 170 childhood-onset GH deficiency patients.

作者信息

Doknic Mirjana, Stojanovic Marko, Soldatovic Ivan, Milenkovic Tatjana, Zdravkovic Vera, Jesic Maja, Todorovic Sladjana, Mitrovic Katarina, Vukovic Rade, Miljic Dragana, Savic Dragan, Milicevic Mihajlo, Stanimirovic Aleksandar, Bogosavljevic Vojislav, Pekic Sandra, Manojlovic-Gacic Emilija, Djukic Aleksandar, Grujicic Danica, Petakov Milan

机构信息

Neuroendocrine Department, Clinic for Endocrinology, Diabetes and Metabolic Diseases, University Clinical Center of Serbia, Belgrade, Serbia.

Faculty of Medicine, University of Belgrade, Belgrade, Serbia.

出版信息

Endocr Connect. 2021 Aug 13;10(8):935-946. doi: 10.1530/EC-21-0274.

Abstract

OBJECTIVE

To analyze metabolic parameters, body composition (BC), and bone mineral density (BMD) in childhood-onset GH deficiency (COGHD) patients during the transition period (TP).

DESIGN

Single- center, retrospective study was performed on 170 consecutive COGHD patients (age 19.2 ± 2.0 years, range 16-25) transferred after growth completion from two pediatric clinics to the adult endocrine unit. Two separate analyses were performed: (i) cross-sectional analysis of hormonal status, metabolic parameters, BC, and BMD at first evaluation after transfer from pediatrics to the adult department; (ii) longitudinal analysis of BC and BMD dynamics after 3 years of GH replacement therapy (rhGH) in TP.

RESULTS

COGHD was of a congenital cause (CONG) in 50.6% subjects, tumor-related (TUMC) in 23.5%, and idiopathic (IDOP) in 25.9%. TUMC patients had increased insulin and lipids levels (P < 0.01) and lower Z score at L-spine (P < 0.05) compared to CONG and IDOP groups. Patients treated with rhGH in childhood demonstrated lower fat mass and increased BMD compared to the rhGH-untreated group (P < 0.01). Three years of rhGH after growth completion resulted in a significant increase in lean body mass (12.1%) and BMD at L-spine (6.9%), parallel with a decrease in FM (5.2%).

CONCLUSION

The effect of rhGH in childhood is invaluable for metabolic status, BC, and BMD in transition to adulthood. Tumor-related COGHD subjects are at higher risk for metabolic abnormalities, alteration of body composition, and decreased BMD, compared to those with COGHD of other causes. Continuation of rhGH in transition is important for improving BC and BMD in patients with persistent COGHD.

摘要

目的

分析儿童期起病的生长激素缺乏症(COGHD)患者在过渡期(TP)的代谢参数、身体成分(BC)和骨密度(BMD)。

设计

对170例连续的COGHD患者(年龄19.2±2.0岁,范围16 - 25岁)进行单中心回顾性研究,这些患者在生长完成后从两家儿科诊所转至成人内分泌科。进行了两项独立分析:(i)从儿科转至成人科室后首次评估时激素状态、代谢参数、BC和BMD的横断面分析;(ii)TP期生长激素替代治疗(rhGH)3年后BC和BMD动态变化的纵向分析。

结果

50.6%的受试者COGHD病因是先天性(CONG),23.5%是肿瘤相关(TUMC),25.9%是特发性(IDOP)。与CONG和IDOP组相比,TUMC患者胰岛素和血脂水平升高(P < 0.01),腰椎Z评分较低(P < 0.05)。与未接受rhGH治疗的组相比,儿童期接受rhGH治疗的患者脂肪量较低,BMD较高(P < 0.01)。生长完成后接受3年rhGH治疗导致瘦体重显著增加(12.1%),腰椎BMD增加(6.9%),同时脂肪量减少(5.2%)。

结论

儿童期rhGH对向成年期过渡时的代谢状态、BC和BMD具有不可估量的作用。与其他病因的COGHD患者相比,肿瘤相关的COGHD受试者代谢异常、身体成分改变和BMD降低的风险更高。对于持续性COGHD患者,过渡期继续使用rhGH对改善BC和BMD很重要。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9714/8428021/92df9fcf3ff1/EC-21-0274fig1.jpg

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