Annals of Hematology

, Volume 82, Issue 10, pp 637–640 | Cite as

Growth hormone (GH) deficiency in patients with β-thalassemia major and the efficacy of recombinant GH treatment

  • K. H. Wu
  • F. J. Tsai
  • C. T. Peng
Original Article


Patients with β-thalassemia major still suffer growth retardation. After excluding patients with cortisol deficiency, hypothyroidism, hypogonadism, delayed puberty, malnutrition, severe congestive heart failure, and severely impaired liver function, 29 patients were enrolled in this study. Fifteen (52%) patients exhibited growth retardation and underwent two growth hormone (GH) provocation tests. Eight (53%) of the 15 patients had GH deficiency and were subsequently treated with subcutaneous recombinant human GH (Genotropin, Pharmacia Corporation, Sweden). Growth velocity increased from the pretreatment rate of 3.1±0.4 cm/year to 7.1±1.6 cm/yr (p<0.001) after 1 year and to 6.8±1.3 cm/year (p<0.001) after 2 years. Patients with growth retardation had lower insulin like growth factor-1 (p=0.001) and insulin like growth factor binding protein-3 (p=0.003) levels than those without growth retardation. In patients with β-thalassemia major, growth retardation is a common complication and GH deficiency plays an important role. Thalassemic patients with GH deficiency can safely increase their growth velocity with recombinant human GH for2 years; however, the effect on final height still needs to be determined.


β-thalassemia major Growth hormone (GH) deficiency Recombinant human GH 



We would like to thank Miss Tsai-Chung Li, a teacher at the institute of Chinese Medicine at the China Medical College in Taichung, for her statistical assistance and interpretation.


  1. 1.
    Olivieri NF, Nathan DG, MacMillan JH, Wayne AS, Liu PP, McGee A, Martin M, Koren G, Cohen AR (1994) Survival in medically treated patients with homozygous thalassemia major. N Engl J Med 331:574–578CrossRefPubMedGoogle Scholar
  2. 2.
    Caruso Nicoletti M, Di Bella D, Pizzarelli G, Leonardi C, Sciuto C, Coco M, Di Gregorio F (1998) Growth failure and bone lesions due to desferrioxamine in thalassaemic patients. J Pediatr Endocrinol Metab 11(3):957–960PubMedGoogle Scholar
  3. 3.
    De Sanctis V, Pinamonti A, Di Palma A, Sprocati M, Atti G, Gamberini MR, Vullo C (1996) Growth and development in thalassaemia major patients with severe bone lesions due to desferrioxamine. Eur J Pediatr 155:368–372CrossRefPubMedGoogle Scholar
  4. 4.
    Fuchs GJ, Tienboon P, Khaled MA, Nimsakul S, Linpisarn S, Faruque AS, Yutrabootr Y, Dewier M, Suskind RM (1997) Nutritional support and growth in thalassaemia major. Arch Dis Child 76:509–512PubMedGoogle Scholar
  5. 5.
    Fuchs GJ, Tienboon P, Linpisarn S, Nimsakul S, Leelapat P, Tovanabutra S, Tubtong V, DeWier M, Suskind RM (1996) Nutritional factors and thalassaemia major. Arch Dis Child 74:224–227PubMedGoogle Scholar
  6. 6.
    Jain M, Sinha Rs, Chellani H, Anad NK (1995) Assessment of thyroid function and its role in body growth in thalassemia major. Indian-Pediatr 32:312–319Google Scholar
  7. 7.
    Roth C, Pekrun A, Bartz M, Jarry H, Eber S, Lakomek M, Schroter W (1997) Short stature and failure of pubertal development in thalassaemia major: evidence for hypothalamic neurosecretory dysfunction of growth hormone secretion and defective pituitary gonadotropin secretion. Eur J Pediatr 156:777–783PubMedGoogle Scholar
  8. 8.
    Soliman AT, El Banna N, Ansari BM (1998) GH response to provocation and circulating IGF-1 and IGF-binding protein-3 concentrations, the IGF-1 generation test and clinical response to GH therapy in children with beta-thalassaemia. Eur J Endocrinol 138:394–400PubMedGoogle Scholar
  9. 9.
    Low LC, Postel Vinay MC, Kwan EY, Cheung-PT (1998) Serum growth hormone(GH) binding protein, IGF-1 and IGFBP-3 in patients with beta-thalassaemia major and the effect of GH treatment. Clin Endocrinol Oxf 48:641–646CrossRefPubMedGoogle Scholar
  10. 10.
    Low LC, Kwan EY, Lim YJ, Lee AC, Tam CF, Lam KS (1995) Growth hormone treatment of short Chinese children with β-thalassaemia major without GH deficiency. Clin Endocrinol Oxf 42:359–363PubMedGoogle Scholar
  11. 11.
    Cavallo L, Gurrado R, Zecchino C, Manolo F, De Sanctis V, Cisternino M, Caruso-Nicoletti M, Galati M (1998) Short-term therapy with recombinant growth hormone in polytransfused thalassaemia major patients with growth deficiency. J Pediatr Endocrinol Metab 11(3):845–849PubMedGoogle Scholar
  12. 12.
    Herington AC, Werther GA, Mathews RN, Burger HG (1981) Studies on the possible mechanism for deficiency of non-suppressible insulin-like activity in thalassemia major. J Clin Endocrinol Metab 52:393–398PubMedGoogle Scholar
  13. 13.
    Saenger P, Schwartz E, Markenson AL, Graziano JH, Levine LS, New MI, Hilgartner MW (1980) Depressed serum somatomedin activity in beta-thalassemia. J Pediatr 96:214–218PubMedGoogle Scholar
  14. 14.
    Chatterjee R, Katz M, Cox T, Bantock H (1993) Evaluation of growth hormone in thalassaemic boys with failed puberty: spontaneous versus provocative test. Eur J Pediatr 152:721–726PubMedGoogle Scholar
  15. 15.
    Pintor C, Cella SG, Manso P, Corda R, Dessi C, Locatelli V, Muller EE (1986) Impaired growth hormone (GH) response to GH-releasing hormone in thalassemia major. J Clin Endocrinol Metab 62:263–267PubMedGoogle Scholar
  16. 16.
    Arcasoy A, Ocal G, Kemahli S, Berberoglu M, Yildirmak Y, Canatan D, Akcurin S, Akar N, Uysal Z, Adiyaman P, Cetinkaya E (1999) Recombinant human growth hormone treatment in children with thalassemia major. Pediatr Int 41:655–661CrossRefPubMedGoogle Scholar
  17. 17.
    Katzos G, Papakostantinou-Athanasiadou E, Athanasiou-Metaxa M, Harsoulis F (2000) Growth hormone treatment in short children with beta-thalassemia major. J Pediatr Endocrinol Metab 13:163–170PubMedGoogle Scholar
  18. 18.
    Kwan EY, Tam SC, Cheung PT, Low LC (2000) The effect of 3 years of recombinant growth hormone therapy on glucose metabolism in short Chinese children with beta-thalassemia major. J Pediatr Endocrinol Metab 13:545–552PubMedGoogle Scholar
  19. 19.
    Cavallo L, Acquafredda A, Zecchino C, De Sanctis V, Cistemino M, Caruso Nicoletti M, Galati M, Massolo F (2001) Recombinant growth hormone treatment in short patients with thalassemia major: results after 24 and 36 months. J Pediatr Endocrinol Metab 14:1133–1137PubMedGoogle Scholar
  20. 20.
    Walter Chen, Jasson Chiang, Po Chao Huang (1999) Revised growth charts, Taiwan, 1997. M Taiwan J Med 4:256–263Google Scholar
  21. 21.
    Greulich WW, Pyle SI (1970) Radiographic Atlas of Skeletal Development of the Hand and Wrist. Stanford University, New YorkGoogle Scholar
  22. 22.
    Wonke B, Hoffbrand AV, Bouloux P, Jensen C, Telfer P (1998) New approaches to the management of hepatitis and endocrine disorders in Cooly's anemia. Ann N Y Acad Sci 850:232–241PubMedGoogle Scholar
  23. 23.
    Blum WF, Ranke MB, Kietzman K, Gauggel E, Zeisel HJ, Bierich JR (1990) A specific radioimmunoassay for the growth hormone (GH)-dependent somatomedin binding protein: its use for diagnosis GH deficiency. J Clin Endocrinol Metab 70:1292–1298PubMedGoogle Scholar
  24. 24.
    Hasegawa Y, Hasegawa T, Aso T, Kotoh S, Tsuchiya Y, Nose O, Ohyama Y, Araki K, Tanaka T, Saisyo S (1992) Usefulness and limitation of measurement of insulin-like growth factor binding protein-3 (IGFBP-3) for diagnosis of growth hormone deficiency. Endocrinol Jpn 39:585–291PubMedGoogle Scholar

Copyright information

© Springer-Verlag 2003

Authors and Affiliations

  1. 1.Department of PediatricsChina Medical College HospitalTaichungTaiwan

Personalised recommendations