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Recombinant human growth hormone treatment in children with thalassemia major
1Department of Pediatrics, Faculty of Medicine, Ankara University, Turkey.
Insights
Recombinant human growth hormone (rhGH) effectively treats growth retardation in prepubertal children with thalassemia. This study shows rhGH significantly increases growth velocity in these patients.
Area of Science:
- Pediatric Endocrinology
- Hematology
- Growth Hormone Therapy
Background:
- Thalassemia is associated with growth retardation in prepubertal children.
- Evaluating growth hormone (GH) reserve is crucial for managing short stature in these patients.
Purpose of the Study:
- To assess growth hormone reserve in prepubertal thalassemic children.
- To determine the efficacy of recombinant human growth hormone (rhGH) treatment for growth retardation.
Main Methods:
- A randomized controlled trial involving 20 prepubertal thalassemic children with short stature.
- Patients were divided into two groups: one receiving rhGH (Genotropin, 0.7 IU/kg/week for 12 months) and a control group.
- Growth velocity and height standard deviation scores were monitored over 12 months.
Main Results:
- A significant increase in growth velocity was observed in the rhGH-treated group (from 2.47 cm/year to 6.27 cm/year, P=0.005).
- The rhGH group showed significantly greater height velocity compared to the control group (6.27 cm/year vs 3.99 cm/year, P=0.025).
- Significant differences in height velocity improvement and standard deviation scores were noted between the groups.
Conclusions:
- Recombinant human growth hormone (rhGH) is a safe and effective treatment for growth retardation in prepubertal children with thalassemia.
- rhGH therapy can significantly improve growth outcomes in this patient population.
Background:
To evaluate the growth hormone reserve and the growth hormone response to recombinant human growth hormone (GH) in prepubertal thalassemic children with growth retardation.
Methods:
Twenty thalassemic patients with short stature and delayed bone age were studied. Patients were randomized into GH-treated (n = 10) and non-GH treated (control; n = 10) groups. The GH-treated group received recombinant human (rh)-GH (Genotropin) at the dose of 0.7 IU/kg per week for 12 months.
Results:
There was a significant discordance between GH response to pharmacologic stimuli and physiological secretion of GH/GHRH testing. Following the administration of rhGH, growth velocity increased from 2.47 +/- 0.48 cm/year to 6.27 +/- 0.76 cm/year (P = 0.005), whereas there was not a similar change in the non-GH-treated group. The height velocities of the two groups during the 1 year follow-up period were significantly different (6.27 +/- 0.76 vs 3.99 +/- 0.34 cm/year; P = 0.025). There were significant differences between the height velocity improvements and height velocity standard deviation scores of the two groups as well.
Conclusion:
The present study has demonstrated that rhGH is a safe and efficacious mode of treatment in thalassemic children.
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