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Disproportionate growth following long-term growth hormone treatment in short children with X-linked
1Sektion für Pädiatrische Nephrologie, Universitäts-Kinderklinik, Heidelberg, Germany.
Insights
Recombinant human growth hormone (rhGH) therapy in children with X-linked hypophosphataemia improved overall height but worsened limb disproportion. Further research is needed to optimize treatment for these growth disorders.
Area of Science:
- Pediatric Endocrinology
- Genetics
- Metabolic Bone Disease
Background:
- X-linked hypophosphataemia (XLH) is a rare genetic disorder causing rickets and short stature.
- Children with XLH often exhibit disproportionate growth, with longer trunks relative to leg length.
- Current management involves phosphate and calcitriol supplementation, but growth remains suboptimal.
Purpose of the Study:
- To evaluate the efficacy and impact of recombinant human growth hormone (rhGH) on growth in prepubertal children with XLH.
- To assess changes in height, sitting height, and leg length following rhGH treatment.
- To monitor biochemical parameters and assess potential side effects.
Main Methods:
- Three prepubertal children with XLH received rhGH (1 IU/kg/week) subcutaneously for 3 years.
- Calcitriol and phosphate supplementation were continued throughout the treatment period.
- Height, sitting height, subischial leg length, and renal phosphate threshold were monitored.
Main Results:
- Growth improved, with height standard deviation score (SDS) increasing by 1.0-1.7 SDS.
- Sitting height showed a significant increase (1.5-2.9 SD), while leg length improved minimally (0.3-0.9 SD).
- Renal phosphate threshold increased slightly, and transient hyperparathyroidism was observed.
Conclusions:
- rhGH treatment effectively enhances growth velocity in children with XLH.
- The treatment appears to exacerbate the pre-existing disproportionate stature characteristic of XLH.
- Careful monitoring is essential to manage growth patterns and biochemical parameters during rhGH therapy.
Unlabelled:
Three short prepubertal children with X-linked hypophosphataemia were treated with 1 IU recombinant human growth hormone (rhGH)/kg per week sc in addition to calcitriol and phosphate supplementation over a period of 3 years. Improvement of height standard deviation score (SDS) ranged from 1.0-1.7 SD based on an increase in sitting height of 1.5-2.9 SD, whereas subischial leg length improved only slightly by 0.3-0.9 SD. In all three patients, renal phosphate threshold concentration increased slightly and transient hyperparathyroidism was noted.
Conclusion:
Treatment of stunted children with X-linked hypophosphataemia is effective in improving growth velocity, but appears to aggravate the pre-existent disproportionate stature of such children.