生长和rhGH在先天性慢性肾病儿童中的应用:一项多中心的当代研究

IF 2.6 3区 医学 Q3 ENDOCRINOLOGY & METABOLISM Hormone Research in Paediatrics Pub Date : 2025-01-16 DOI:10.1159/000543435
Séverine Dubois, Charlotte Duneton, Rémi Henri Salomon, Tim Ulinski, Priscilla Boizeau, Jean-Claude Carel, Dominique Simon
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引用次数: 0

摘要

生长迟缓在慢性肾脏疾病(CKD)患儿中很常见,反映了CKD的严重程度。重组人生长激素(rhGH)治疗于1995年被批准用于CKD。我们在这里描述了三个儿科肾脏病科先天性CKD患儿的治疗模式和生长结果。方法:我们纳入了2015年至2020年间接受肾移植(KT)的患者,年龄在3至18岁之间。在四个时间点收集数据:CKD诊断,开始rhGH,开始透析和移植。结果:87例患者中42例(48%)接受rhGH治疗。治疗开始时的中位身高为-2.0 SDS,中位身高增加为+0.7 SD (p)。结论:约一半的CKD患者接受了rhGH治疗,出现了显著的身高增加。未经治疗的患者在身高方面没有受到不利影响。这些数据强调了CKD患者在需要时仔细监测生长和rhGH治疗的重要性。
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Growth and rhGH use in children with congenital chronic kidney disease: a multicentric contemporary study.

Introduction: Growth retardation is common in children with chronic kidney disease (CKD) and reflects CKD severity. Recombinant human growth hormone (rhGH) treatment was approved for CKD in 1995. We describe here treatment patterns and growth outcomes in children with congenital CKD in three pediatric nephrology departments.

Methods: We included patients with kidney transplantation (KT) performed between 2015 and 2020 at an age of 3 to 18 years. Data were collected at four timepoints: CKD diagnosis, initiation of rhGH, initiation of dialysis, and transplantation.

Results: Among 87 patients, 42 (48%) received rhGH. The median height at treatment initiation was -2.0 SDS, with a median height gain of +0.7 SD (p<0.0001) in 1.7 years. Growth outcomes were negatively associated with older age and CKD stage 5. The 45 rhGH-untreated patients lost 0.6 SD (p=0.02) from diagnosis to transplantation but maintained their height in the normal range. At transplantation, 26% of rhGH-treated and 9% of rhGH-untreated patients had a height SDS below -2 SDS. rhGH was initiated by nephrologists in 52% of cases and endocrinologists in 48%. Deviations from marketing authorization criteria were observed in 68% of cases: endocrinologists typically prescribed rhGH for children under 2 years, while nephrologists prescribed it for patients with a height above -2 SDS.

Conclusion: About half of CKD patients received rhGH treatment, resulting in significant height gain. Untreated patients were not adversely affected in terms of height. These data highlight the importance of careful monitoring of growth and rhGH treatment if needed in patients with CKD.

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来源期刊
Hormone Research in Paediatrics
Hormone Research in Paediatrics ENDOCRINOLOGY & METABOLISM-PEDIATRICS
CiteScore
4.90
自引率
6.20%
发文量
88
审稿时长
4-8 weeks
期刊介绍: The mission of ''Hormone Research in Paediatrics'' is to improve the care of children with endocrine disorders by promoting basic and clinical knowledge. The journal facilitates the dissemination of information through original papers, mini reviews, clinical guidelines and papers on novel insights from clinical practice. Periodic editorials from outstanding paediatric endocrinologists address the main published novelties by critically reviewing the major strengths and weaknesses of the studies.
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