Growth and Recombinant Human Growth Hormone Use in Children with Congenital Chronic Kidney Disease: A Multicentric Contemporary Study.

Séverine Dubois,Charlotte Duneton, Rémi Salomon, Tim Ulinski,Priscilla Boizeau, Jean-Claude Carel,Dominique Simon

Hormone research in paediatrics（2025）SCI 3区SCI 4区

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Abstract

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 treatment patterns and growth outcomes in children with congenital CKD in three pediatric nephrology departments. METHODS:We included patients with kidney transplantation performed between 2015 and 2020 at an age of 3-18 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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【要点】：本研究探讨了先天性慢性肾病儿童使用重组人生长激素（rhGH）治疗对生长发育的影响，发现rhGH治疗可显著增加身高，但需谨慎监测与适应症。

【方法】：通过回顾性分析2015至2020年间接受肾移植的3-18岁先天性CKD患者，收集患者在CKD诊断、rhGH治疗开始、透析开始及移植四个时间点的数据。

【实验】：共纳入87名患者，其中42名（48%）接受rhGH治疗。治疗开始时平均身高为-2.0 SDS，1.7年内平均身高增长+0.7 SD（p < 0.0001）。未接受rhGH治疗的患者在诊断到移植期间身高下降0.6 SD（p = 0.02），但维持正常身高范围内。移植时，26%的rhGH治疗患者和9%的未治疗患者身高SDS低于-2 SDS。rhGH治疗由肾脏病专家和内分泌专家分别负责52%和48%的病例，且有68%的病例未遵循市场批准标准。