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.
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References
Fessas P, Loukopoulos D. The beta-thalassaemias. Clin Haematol 1974; 3: 411–35
Wonke B. Management of beta-thalassaemia major. Curr Paediatr 1994; 4: 38–42
Rebulla P, Modell B. Transfusion requirements and effects in patients with thalassaemia major. Cooley care programme. Lancet 1991; 337: 277–80
DeSanctis V, Wonke B. Growth in thalassaemia. Farmaci 1994 Suppl.: 1-45
Bergeron C, Kovacs K. Pituitary and ultrastructural study. Am Pathol 1978; 93: 292–309
De Sanctis V, Atti G, Banin P, al. Growth in thalassaemia major. Acta Med Auxol 1991; 23: 29–36
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): 227–33
Vassilopoulou-Sellin R, Oyedejico, Foster PL, et al. Hemoglobin as a direct inhibitor of cartilage growth in vitro. Horm Metabol Res 1989; 21: 11–4
De Virgilis S, Congia S, Frau M, et al. Deferioxamine-induced growth retardation in patients with thalassaemia major. J Pediatr 1988; 113: 661–9
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: 32–3
Vullo C, De Sanctis V, Katz M, et al. Endocrine abnormalities in thalassaemia. Ann N Y Acad Sci 1990; 612: 293–310
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; 263–7
Shehadeh N, Hazani A, Rudolf MCJ, et al. Neurosecretory dysfunction of growth hormone secretion in thalassaemia major. Acta Paediatr Scand 1990; 79: 790–5
Saenger P, Schwartz E, Markenson AL, et al. Depressed serum somatomedin activity in beta-thalassaemia. J Pediatr 1980; 96: 214–8
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: 806–9
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: 393–8
De Sanctis V, Pintor C, Gamberini MR, et al. Multicentre study on prevalence of endocrine complications in thalassaemia major. Clin Endocrinol 1995; 42: 581–6
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.: 62–5
Di Martino J, Stoner E, Giardina PJ, et al. Effect of exogenous growth hormone in patients with thalassaemia major [abstract]. Pediatr Res 1985; 80: 900
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: 335–9
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: T914–11
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: P3–43
De Sanctis V, Urso L. L’accrescimento staturale nella talassemia. Minerva Pediatr 1997; 47: 121–8
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: 228
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: 126
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: 359–63
Low LCK. Growth, puberty and endocrine function in beta-thalassaemia major. J Ped Endocrinol Metab 1997; 10: 175–84
Frasier SD. Human pituitary growth hormone (hGH) therapy in growth hormone deficiency. Endocr Rev 1983; 4: 155–60
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De Sanctis, V., Urso, L. Clinical Experience with Growth Hormone Treatment in Patients with β-Thalassaemia Major. BioDrugs 11, 79–85 (1999). https://doi.org/10.2165/00063030-199911020-00002
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DOI: https://doi.org/10.2165/00063030-199911020-00002