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Growth and Recombinant Human Growth Hormone Use in Children with Congenital Chronic Kidney Disease: A Multicentric
Séverine Dubois1, Charlotte Duneton2, Rémi Salomon3
1Department 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.
Insights
Recombinant human growth hormone (rhGH) improved height in children with chronic kidney disease (CKD). However, untreated patients maintained normal height, suggesting careful monitoring is key for rhGH therapy in pediatric CKD.
Area of Science:
- Pediatric Nephrology
- Endocrinology
- Growth Disorders
Background:
- Growth retardation is a significant complication in pediatric chronic kidney disease (CKD), often correlating with disease severity.
- Recombinant human growth hormone (rhGH) has been an approved treatment for CKD-related growth issues since 1995.
Purpose of the Study:
- To analyze treatment patterns and growth outcomes of rhGH in children with congenital CKD.
- To evaluate the effectiveness and utilization of rhGH in pediatric nephrology settings.
Main Methods:
- Retrospective analysis of 87 children (3-18 years) undergoing kidney transplantation (2015-2020).
- Data collected at CKD diagnosis, rhGH initiation, dialysis initiation, and transplantation.
- Comparison of growth outcomes between rhGH-treated and untreated groups.
Main Results:
- 48% of patients received rhGH, achieving a median height gain of +0.7 SDS.
- Untreated patients maintained height SDS, while some rhGH-treated patients remained below -2 SDS at transplantation.
- Off-label rhGH use was common, with deviations from marketing authorization criteria in 68% of cases.
Conclusions:
- rhGH treatment led to significant height gain in approximately half of pediatric CKD patients.
- Growth outcomes in untreated patients were not negatively impacted, highlighting the need for judicious rhGH use.
- Close monitoring of growth and appropriate rhGH application are crucial for managing children with CKD.
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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