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Growth hormone treatment in short children with beta-thalassemia major
G Katzos1, E Papakostantinou-Athanasiadou, M Athanasiou-Metaxa
1Department of Pediatrics, Aristotle University, Hippokration General Hospital, Thessaloniki, Greece.
Insights
Recombinant human growth hormone (rhGH) significantly increased growth velocity and insulin-like growth factor I (IGF-I) in children with beta-thalassemia major. Further studies are needed to determine if long-term rhGH treatment impacts final height.
Area of Science:
- Pediatrics
- Endocrinology
- Hematology
Background:
- Beta-thalassemia major is a genetic blood disorder often associated with growth impairment.
- Children with beta-thalassemia major may have normal growth hormone (GH) response but low insulin-like growth factor I (IGF-I) levels.
- Growth hormone (GH) therapy is a potential intervention for growth deficits in these patients.
Purpose of the Study:
- To evaluate the effect of one year of recombinant human growth hormone (rhGH) treatment on growth rate and bone age in prepubertal children with beta-thalassemia major.
- To assess changes in insulin-like growth factor I (IGF-I) levels during rhGH therapy.
Main Methods:
- Ten short prepubertal children with beta-thalassemia major and normal GH response were treated with rhGH (28 IU/m2/week) subcutaneously for 12 months.
- Growth velocity and bone age were measured before and after treatment.
- Serum IGF-I levels were monitored at 3, 6, and 12 months post-treatment.
Main Results:
- Growth velocity increased significantly from 4.22 cm/yr to 7.61 cm/yr after one year of rhGH treatment.
- Mean bone age advanced from 8.20 years to 9.55 years, proportionally to chronological age.
- Serum IGF-I levels, initially low, rose significantly during treatment, indicating a positive response to rhGH.
Conclusions:
- One-year supraphysiological rhGH treatment significantly increases growth velocity and IGF-I levels in children with beta-thalassemia major and normal GH reserve.
- The accelerated bone maturation suggests GH influences skeletal development.
- Long-term effects of rhGH on final height in this population require further investigation.
Abstract:
The effect of one year recombinant human growth hormone (rhGH) treatment on growth rate and bone age was studied in ten short prepubertal children with beta-thalassemia major (age range 7.10-12.03 yr) with normal GH response to provocative stimuli. rhGH was given subcutaneously every day in a dose of 28 IU/m2/week. In the 10 children who completed 12 months of treatment the growth velocity increased from 4.22+/-0.81 cm/yr (-1.38+/-0.80 SDS for CA) to 7.61+/-1.16 cm/yr (+2.27+/-1.64 SDS for CA). IGF-I was low before treatment, 138.3 +/-38.9 ng/ml, and rose significantly to 232.2+/-122.1, 243.2+/-98.4 and 227.5+/-86.2 at 3, 6 and 12 months post-treatment, respectively (p<0.01). Bone maturation was accelerated in proportion to the increase in chronological age. The mean pre-treatment bone age in the ten children was 8.20+/-1.97 and increased to 9.55+/-1.80 yr after one year of treatment. Our data demonstrate that GH treatment of thalassemic children with normal GH reserve and low serum IGF-I concentrations with supraphysiological doses of rhGH for one year can cause a significant increase in serum IGF-I levels and growth velocity, but it remains to be elucidated whether long-term administration will affect the final height.