BioDrugs

, 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

Abstract

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.

Keywords

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

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  1. 1.
    Fessas P, Loukopoulos D. The beta-thalassaemias. Clin Haematol 1974; 3: 411–35Google Scholar
  2. 2.
    Wonke B. Management of beta-thalassaemia major. Curr Paediatr 1994; 4: 38–42CrossRefGoogle Scholar
  3. 3.
    Rebulla P, Modell B. Transfusion requirements and effects in patients with thalassaemia major. Cooley care programme. Lancet 1991; 337: 277–80PubMedCrossRefGoogle Scholar
  4. 4.
    DeSanctis V, Wonke B. Growth in thalassaemia. Farmaci 1994 Suppl.: 1-45Google Scholar
  5. 5.
    Bergeron C, Kovacs K. Pituitary and ultrastructural study. Am Pathol 1978; 93: 292–309Google Scholar
  6. 6.
    De Sanctis V, Atti G, Banin P, al. Growth in thalassaemia major. Acta Med Auxol 1991; 23: 29–36Google Scholar
  7. 7.
    De Sanctis V, Stea S, Savarino L, et al. Growth hormone secretion and bone histomorphometric study in thalassaemic patients with acquired skeletal dysplasia secondary to desferrioxamine. J Pediatr Endocrinol Metab 1998; 11(5): 227–33Google Scholar
  8. 8.
    Vassilopoulou-Sellin R, Oyedejico, Foster PL, et al. Hemoglobin as a direct inhibitor of cartilage growth in vitro. Horm Metabol Res 1989; 21: 11–4CrossRefGoogle Scholar
  9. 9.
    De Virgilis S, Congia S, Frau M, et al. Deferioxamine-induced growth retardation in patients with thalassaemia major. J Pediatr 1988; 113: 661–9CrossRefGoogle Scholar
  10. 10.
    De Sanctis V, Urso L, Galati MC, et al. Growth hormone (GH) treatment in thalassamic patients with short stature, GH insufficiency and different severity of iron overload. Bone Marrow Transplant 1997; 19: 32–3Google Scholar
  11. 11.
    Vullo C, De Sanctis V, Katz M, et al. Endocrine abnormalities in thalassaemia. Ann N Y Acad Sci 1990; 612: 293–310PubMedCrossRefGoogle Scholar
  12. 12.
    Pintor G, Cella SG, Manso P, et al. Impaired growth hormone (GH) response to GH-releasing hormone in thalassaemia major. J Clin Endocrinol Metab 1986; 62; 263–7PubMedCrossRefGoogle Scholar
  13. 13.
    Shehadeh N, Hazani A, Rudolf MCJ, et al. Neurosecretory dysfunction of growth hormone secretion in thalassaemia major. Acta Paediatr Scand 1990; 79: 790–5PubMedCrossRefGoogle Scholar
  14. 14.
    Saenger P, Schwartz E, Markenson AL, et al. Depressed serum somatomedin activity in beta-thalassaemia. J Pediatr 1980; 96: 214–8PubMedCrossRefGoogle Scholar
  15. 15.
    Werther GA, Nathews RN. Burger HG, et al. Lack of response of non-suppressible insulin-like activity in thalassaemia major. J Clin Endocrinol Metab 1981; 53: 806–9PubMedCrossRefGoogle Scholar
  16. 16.
    Herrington AC, Werther GA, Mathews RN, et al. Studies on the possible mechanism for deficiency of nonsuppresible insulinlike activity in thalassaemia major. J Clin Endocrinol Metab 1981; 52: 393–8CrossRefGoogle Scholar
  17. 17.
    De Sanctis V, Pintor C, Gamberini MR, et al. Multicentre study on prevalence of endocrine complications in thalassaemia major. Clin Endocrinol 1995; 42: 581–6CrossRefGoogle Scholar
  18. 18.
    De Luca G, Maggiolini M, Bria M, et al. Valutazione della secrezione dell’ormone della crescita (GH) in soggetti affetti da thalassemia major. Minerva Endocrinol 1993; 18 Suppl.: 62–5PubMedGoogle Scholar
  19. 19.
    Di Martino J, Stoner E, Giardina PJ, et al. Effect of exogenous growth hormone in patients with thalassaemia major [abstract]. Pediatr Res 1985; 80: 900Google Scholar
  20. 20.
    Scacchi M, Danesi L, De Martin M, et al. Treatment with bio-synthetic growth hormone of short thalassaemic patients with impaired growth hormone secretion. Clin Endocrinol 1991; 35: 335–9CrossRefGoogle Scholar
  21. 21.
    Theodoridis C, Karis C, Ladis V, et al. Effects or recombinant human growth hormone on growth of thalassaemic children with G.H.D [abstract]. World Congress of Pediatrics; 1992 Jun 12-15; Rio de Janeiro, Brazil; 55: T914–11Google Scholar
  22. 22.
    Caruso-Nicoletti M, Mancuso M, Spadaro G, et al. Response to growth hormone treatment in thalassaemic patients [abstract]. 10th International Congress of Endocrinology (ICE ’96); 1996 Sep 12-15; San Francisco: 765: P3–43Google Scholar
  23. 23.
    De Sanctis V, Urso L. L’accrescimento staturale nella talassemia. Minerva Pediatr 1997; 47: 121–8Google Scholar
  24. 24.
    Theodoridis C, Papatheodorou A, Karis C, et al. Short and long-term effect of rhGH on growth of short GH deficiency children with beta-thalassaemia [abstract]. 34th Annual Meeting of ESPE, Edinburgh. Horm Res 1995 Jun 25-28; 58: 228Google Scholar
  25. 25.
    Masala A, Alagna, S, Gallisai D, et al. GH secretion after bone marrow transplantation (BMT) in patients with homozygous beta-thalassaemia. J Clin Endocrinol Invest 1992; 15 Suppl. 1: 126Google Scholar
  26. 26.
    Low WK, Kwan EYW, Lim YJ, et al. Growth hormone treatment of short Chinese children with beta-thalassaemia major without GH deficiency. Clin Endocrinol 1995; 42: 359–63CrossRefGoogle Scholar
  27. 27.
    Low LCK. Growth, puberty and endocrine function in beta-thalassaemia major. J Ped Endocrinol Metab 1997; 10: 175–84Google Scholar
  28. 28.
    Frasier SD. Human pituitary growth hormone (hGH) therapy in growth hormone deficiency. Endocr Rev 1983; 4: 155–60PubMedCrossRefGoogle Scholar

Copyright information

© Adis International Limited 1999

Authors and Affiliations

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

Personalised recommendations