, Volume 11, Issue 2, pp 79–85 | Cite as

Clinical Experience with Growth Hormone Treatment in Patients with β-Thalassaemia Major

  • Vincenzo De Sanctis
  • Luigina Urso
Disease Management


The aetiology of the growth retardation occurring in patients with β-thalassaemia major is considered to be multifactorial. Although growth hormone (GH) secretion appears to be normal in many thalassaemic patients with short stature, there is evidence indicating impaired GH secretion in approximately 3% of patients. The response to recombinant human GH treatment (rHGH) is not predictable, based on either the parameters known to affect the response to treatment or the type of defect in the GH-insulin-like growth factor (IGF-1) axis. The wide variation in growth velocity observed during rHGH treatment suggests that treatment with the usual dose of rHGH (0.6 U/kg/week) cannot be considered effective in all patients, although rHGH has been successful in some patients. Therefore further studies are required in order to evaluate the effects of supraphysiological doses of rHGH on growth. Since these patients are prone to develop abnormal glucose homeostasis, oral glucose tolerance tests must be performed periodically during rHGH treatment.


Growth Hormone Adis International Limited Iron Overload Growth Hormone Secretion Thalassaemia Major 
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Copyright information

© Adis International Limited 1999

Authors and Affiliations

  • Vincenzo De Sanctis
    • 1
  • Luigina Urso
    • 1
  1. 1.Department of Paediatrics and Adolescent MedicineFerraraItaly

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