Dubois Séverine, Duneton Charlotte, Salomon Rémi, Ulinski Tim, Boizeau Priscilla, Carel Jean-Claude, Simon Dominique
Department of Pediatric Endocrinology and Diabetes and Reference Center for Growth and Development Endocrine Diseases, Assistance Publique-Hôpitaux de Paris, Robert-Debré University Hospital, Paris, France,
Department of Pediatric Nephrology, Robert-Debré Hospital, Reference Center for Nephrotic Syndrome in Children and Adults, AP-HP, Paris, France.
Horm Res Paediatr. 2025 Jan 16:1-9. doi: 10.1159/000543435.
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.
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.
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.
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.
生长发育迟缓在慢性肾脏病（CKD）患儿中很常见，且反映了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治疗。我们描述了三个儿科肾脏病科室中先天性CKD患儿的治疗模式和生长结局。
我们纳入了2015年至2020年间在3至18岁时接受肾移植的患者。在四个时间点收集数据：CKD诊断、rhGH开始使用、透析开始和移植。
87例患者中，42例（48%）接受了rhGH治疗。治疗开始时的中位身高标准差为-2.0，在1.7年中身高增益中位数为+0.7标准差（p<0.0001）。生长结局与年龄较大和CKD 5期呈负相关。45例未接受rhGH治疗的患者从诊断到移植身高下降了0.6标准差（p=0.02），但仍保持在正常范围内。移植时，26%接受rhGH治疗的患者和9%未接受rhGH治疗的患者身高标准差低于-2标准差。68%的病例观察到偏离上市许可标准的情况：内分泌科医生通常为2岁以下儿童开具rhGH，而肾内科医生为身高高于-2标准差的患者开具。
约一半的CKD患者接受了rhGH治疗，身高显著增加。未治疗的患者在身高方面未受到不利影响。这些数据强调了对CKD患者生长情况进行仔细监测以及在必要时进行rhGH治疗的重要性。
