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Long-term treatment with growth hormone in short children with nephropathic cystinosis

E Wühl1, D Haffner, G Offner

  • 1Renal Units, the Department of Pediatrics, University Hospital of Heidelberg, 69120 Heidelberg, Germany.

The Journal of Pediatrics
|June 8, 2001
PubMed

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.
Abstract

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