Treatment of growth failure with growth hormone in children with chronic kidney disease: an open-label long-term study
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Published on: April 4, 2025
D Müller-Wiefel1, H Frisch, T Tulassay
1Paediatric Nephrology, University Medical Center, Hamburg, Germany. muellerw@uke.uni-hamburg.de
Insights
Recombinant human growth hormone (GH) significantly improves growth in children with chronic kidney disease (CKD). The treatment was well-tolerated and showed no adverse effects on kidney function over five years.
Area of Science:
- Pediatric Nephrology
- Endocrinology
- Growth Hormone Therapy
Background:
- Children with chronic kidney disease (CKD) often experience growth failure.
- Recombinant human growth hormone (GH) is a potential therapeutic option for improving growth in these patients.
Purpose of the Study:
- To evaluate the long-term efficacy and safety of GH treatment in children with CKD and growth failure.
Main Methods:
- An international, multicenter, open-label study involving 81 children with CKD.
- GH was administered at 0.35 mg/kg/week, with efficacy assessed over 12 months, 24 months, and up to 5 years.
Main Results:
- Significant increases in height velocity (HV) and height standard deviation score (SDS) were observed after 12 and 24 months (p < 0.001).
- The proportion of children achieving normal height SDS increased from 1% at baseline to 43% after 24 months.
- Improvements were consistent across CKD subgroups, with notable gains in Stage 4-5 CKD patients. No detrimental effects on kidney function or bone age were reported.
Conclusions:
- Long-term GH treatment leads to significant growth improvement in children with CKD.
- GH therapy is a safe and effective option for managing growth failure in pediatric CKD patients.
Aims:
To assess long-term efficacy and safety of recombinant human growth hormone (GH) in children with chronic kidney disease (CKD).
Methods:
An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12 months of treatment, extended to 24 months, then to 5 years.
Results:
In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p < 0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p < 0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p < 0.001). A normal height SDS was seen in 1% of children at baseline, 17% after 12 months and 43% after 24 months of treatment. Improvements were similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment.
Conclusions:
In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.
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