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Long-term treatment with growth hormone in short children with nephropathic cystinosis
1Renal Units, the Department of Pediatrics, University Hospital of Heidelberg, 69120 Heidelberg, Germany.
Insights
Growth hormone (GH) treatment effectively improves height in children with nephropathic cystinosis. Early intervention is recommended for optimal growth outcomes in this condition.
Area of Science:
- Pediatric Endocrinology
- Nephrology
- Genetics
Background:
- Nephropathic cystinosis is a rare genetic disorder leading to progressive multi-organ damage, particularly affecting the kidneys.
- Growth retardation is a significant complication in children with nephropathic cystinosis, impacting their overall health and development.
Purpose of the Study:
- To evaluate the efficacy and safety of growth hormone (GH) therapy in children with nephropathic cystinosis experiencing severe growth failure.
- To assess GH treatment outcomes during both conservative management and renal replacement therapy (dialysis or transplantation).
Main Methods:
- An open-labeled prospective trial was conducted with a 1-year run-in period.
- Seventy-four children with cystinosis, aged 3.0 to 18 years, received GH treatment for a mean of 3.1 years.
- Patients were stratified into conservative treatment, dialysis, and renal transplant groups.
Main Results:
- GH treatment significantly increased height velocity in all groups, with the most pronounced effect in the conservative treatment group (doubled height velocity).
- Height SDS improved by +1.6 within 3 years in prepubertal patients on conservative treatment, with sustained growth.
- GH treatment demonstrated a favorable safety profile with no major adverse events observed.
Conclusions:
- Long-term growth hormone therapy is a safe and effective treatment for growth retardation in young children with nephropathic cystinosis.
- Early initiation of GH treatment is crucial, especially when nutrition and cysteamine therapy fail to prevent growth deficits.
Objective:
The objective was to assess the efficacy and safety of growth hormone (GH) treatment in severely growth retarded children with nephropathic cystinosis during conservative treatment and during renal replacement therapy.
Study Design:
The design was an open-labeled prospective trial with a run-in period of 1 year.
Results:
A total of 74 children with cystinosis (age 3.0 to 18 years) were treated with GH over a mean period of 3.1 years (range 1 to 10 years); 52 patients were receiving conservative treatment (mean age 7.1 years), 7 were receiving dialysis (12.5 years), and 15 had received a renal transplant (14.8 years). The mean standardized height (SD score) was -4.0 in the conservative treatment group, -4.4 in the dialysis group, and -4.9 in the renal transplant group. During the first treatment year, height velocity doubled in the conservative treatment group, increased by 80% in the dialysis group, and increased by 45% in renal transplant group. Within 3 years the height SD score increased by +1.6 (P <.001) in prepubertal patients receiving conservative treatment, and percentile parallel growth was maintained thereafter. These effects of GH were less expressed in peripubertal patients receiving renal replacement therapy. No major side effects were observed.
Conclusion:
Long-term GH treatment is safe and effective in young children with nephropathic cystinosis. GH treatment should be started early in the course of the disease if adequate nutrition and cysteamine treatment do not prevent growth retardation.