ESPE Yearbook of Paediatric Endocrinology

Horm Res Paediatr. 2025 Jan 16:1-9. PMID: 39820089. doi: 10.1159/000543435. [email protected]

Brief summary: This multicenter cohort study in France evaluated recombinant human growth hormone (rhGH) treatment patterns and growth outcomes in 87 patients with congenital chronic kidney disease (CKD) who underwent kidney transplantation between the ages 3 to 18 years. Of these, 48% received GH therapy, prescribed either by nephrologists (52%) or endocrinologists (48%).

The median age at GH treatment initiation was 7.4 years (range: 3.4–10.7), at median height –2 SDS. The starting rhGH dose was 0.044 mg/kg/day, and the median dose at follow-up was 0.036 mg/kg/day. Two-thirds of the prescriptions deviated from the French authorization criteria, which include the following: height ≤ –2 SDS or growth velocity < 2 cm/year, age > 2 years, GFR < 60 mL/min/1.73 m², bone age < 11 years in girls and < 13 years in boys, at least 1 year of nephrological management, and absence of active malignancy. Endocrinologists typically prescribed rhGH for patients under 2 years of age, whereas nephrologists did so for those with height above –2 SDS. The median height gain was +0.7 SDS over 1.7 years. Growth response was negatively associated with older age at GH initiation and being on dialysis at treatment start. 52% of patients did not receive rhGH treatment. From CKD diagnosis to kidney transplantation, untreated patients experienced a median height loss of −0.6 SDS, and half of them had height SDS < −2 at transplantation. Reasons for not receiving rhGH therapy included spontaneous catch-up growth, active malignancy or near-final height.

Comment: This is the first French multicenter cohort study since the approval of rhGH treatment for CKD in 1995. It raises important questions about the criteria for rhGH prescription in these patients, which are still not universally defined^1,2. For example, the European consensus recommends starting GH therapy in patients with height between the 3rd and 10th percentile and growth velocity <25th percentile². This study also highlights the need to clarify the optimal methods for monitoring therapy. The authors note that the mean height gain in SDS was lower than that reported in the literature, likely attributable at least in part to GH dosing. Indeed, although treatment was initiated at appropriate doses, follow-up adjustments were often not made in response to either weight gain or IGF-1 levels. Moreover, conclusions regarding untreated patients are limited by the lack of data on target height, pubertal stage, and bone age. Study limitations include missing information on pubertal status and target height for a significant proportion of patients, a relatively small cohort size, and a short follow-up duration. Further studies with larger sample sizes and prolonged follow-up extending through final height attainment are warranted. As the authors rightly emphasize, improved collaboration between nephrologists and endocrinologists is essential to optimize the initiation and monitoring of rhGH therapy in children with CKD.

References: 1. Mahan JD, Warady BA; Consensus Committee. Assessment and treatment of short stature in pediatric patients with chronic kidney disease: a consensus statement. PediatrNephrol. 2006;21(7):917–30.2. Drube J, Wan M, Bonthuis M, Wühl E, Bacchetta J, Santos F, et al. Clinical practice recommendations for growth hormone treatment in children with chronic kidney disease. Nat Rev Nephrol. 2019;15(9):577–89.