Skip to main content
PMC home page
Archives of Disease in Childhood logoLink to Archives of Disease in Childhood
. 1994 Aug;71(2):128–132. doi: 10.1136/adc.71.2.128

Relationship of endocrinopathy to iron chelation status in young patients with thalassaemia major.

R G Grundy 1, K A Woods 1, M O Savage 1, J P Evans 1
PMCID: PMC1029942  PMID: 7944532

Abstract

Disturbances of growth and development in patients with thalassaemia receiving hypertransfusion programmes are well recognised and are most likely to be due to iron overload. The extent of endocrine dysfunction was investigated in a group of 18 patients thought to have been treated by acceptable modern standards, 11 of whom could be considered as well chelated. Assessment of growth and puberty showed a wide variation in height SD scores with five patients having significantly short stature. Most patients are progressing through puberty normally with the exception of two boys with marked pubertal delay. The most prominent finding was that growth hormone responses to glucagon stimulation were significantly impaired in all of the patients with iron overload. Basal endocrine assessment showed primary hypothyroidism in two patients aged 16.8 and 12.9 years with plasma thyroxine-concentrations of 86 and 59 nmol/l (normal range 65-165 nmol/l) and plasma thyroid stimulating hormone 10.2 and 30.3 mU/l (normal range 0.5-5 mU/l). One patient had diabetes mellitus. These results show that even when ideal management is sought a significant amount of endocrine damage occurs; surveillance of these patients is thus essential.

Full text

PDF
128

Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Barry M., Flynn D. M., Letsky E. A., Risdon R. A. Long-term chelation therapy in thalassaemia major: effect on liver iron concentration, liver histology, and clinical progress. Br Med J. 1974 Apr 6;2(5909):16–20. doi: 10.1136/bmj.2.5909.16. [DOI] [PMC free article] [PubMed] [Google Scholar]
  2. Bronspiegel-Weintrob N., Olivieri N. F., Tyler B., Andrews D. F., Freedman M. H., Holland F. J. Effect of age at the start of iron chelation therapy on gonadal function in beta-thalassemia major. N Engl J Med. 1990 Sep 13;323(11):713–719. doi: 10.1056/NEJM199009133231104. [DOI] [PubMed] [Google Scholar]
  3. Canale V. C., Steinherz P., New M., Erlandson M. Endocrine function in thalassemia major. Ann N Y Acad Sci. 1974;232(0):333–345. doi: 10.1111/j.1749-6632.1974.tb20597.x. [DOI] [PubMed] [Google Scholar]
  4. De Sanctis V., Zurlo M. G., Senesi E., Boffa C., Cavallo L., Di Gregorio F. Insulin dependent diabetes in thalassaemia. Arch Dis Child. 1988 Jan;63(1):58–62. doi: 10.1136/adc.63.1.58. [DOI] [PMC free article] [PubMed] [Google Scholar]
  5. Fargion S., Taddei M. T., Gabutti V., Piga A., Di Palma A., Capra L., Fontanelli G., Avanzini A. Early iron overload in beta-thalassaemia major: when to start chelation therapy? Arch Dis Child. 1982 Dec;57(12):929–933. doi: 10.1136/adc.57.12.929. [DOI] [PMC free article] [PubMed] [Google Scholar]
  6. Flynn D. M., Fairney A., Jackson D., Clayton B. E. Hormonal changes in thalassaemia major. Arch Dis Child. 1976 Nov;51(11):828–836. doi: 10.1136/adc.51.11.828. [DOI] [PMC free article] [PubMed] [Google Scholar]
  7. Leger J., Girot R., Crosnier H., Postel-Vinay M. C., Rappaport R. Normal growth hormone (GH) response to GH-releasing hormone in children with thalassemia major before puberty: a possible age-related effect. J Clin Endocrinol Metab. 1989 Aug;69(2):453–456. doi: 10.1210/jcem-69-2-453. [DOI] [PubMed] [Google Scholar]
  8. Lucarelli G., Galimberti M., Polchi P., Angelucci E., Baronciani D., Giardini C., Politi P., Durazzi S. M., Muretto P., Albertini F. Bone marrow transplantation in patients with thalassemia. N Engl J Med. 1990 Feb 15;322(7):417–421. doi: 10.1056/NEJM199002153220701. [DOI] [PubMed] [Google Scholar]
  9. Masala A., Meloni T., Gallisai D., Alagna S., Rovasio P. P., Rassu S., Milia A. F. Endocrine functioning in multitransfused prepubertal patients with homozygous beta-thalassemia. J Clin Endocrinol Metab. 1984 Apr;58(4):667–670. doi: 10.1210/jcem-58-4-667. [DOI] [PubMed] [Google Scholar]
  10. McIntosh N. Endocrinopathy in thalassaemia major. Arch Dis Child. 1976 Mar;51(3):195–201. doi: 10.1136/adc.51.3.195. [DOI] [PMC free article] [PubMed] [Google Scholar]
  11. Modell B., Letsky E. A., Flynn D. M., Peto R., Weatherall D. J. Survival and desferrioxamine in thalassaemia major. Br Med J (Clin Res Ed) 1982 Apr 10;284(6322):1081–1084. doi: 10.1136/bmj.284.6322.1081. [DOI] [PMC free article] [PubMed] [Google Scholar]
  12. Pintor C., Cella S. G., Manso P., Corda R., Dessi C., Locatelli V., Müller E. E. Impaired growth hormone (GH) response to GH-releasing hormone in thalassemia major. J Clin Endocrinol Metab. 1986 Feb;62(2):263–267. doi: 10.1210/jcem-62-2-263. [DOI] [PubMed] [Google Scholar]
  13. Postel-Vinay M. C., Girot R., Leger J., Hocquette J. F., McKelvie P., Amar-Costesec A., Rappaport R. No evidence for a defect in growth hormone binding to liver membranes in thalassemia major. J Clin Endocrinol Metab. 1989 Jan;68(1):94–98. doi: 10.1210/jcem-68-1-94. [DOI] [PubMed] [Google Scholar]
  14. Sabato A. R., de Sanctis V., Atti G., Capra L., Bagni B., Vullo C. Primary hypothyroidism and the low T3 syndrome in thalassaemia major. Arch Dis Child. 1983 Feb;58(2):120–127. doi: 10.1136/adc.58.2.120. [DOI] [PMC free article] [PubMed] [Google Scholar]
  15. Saenger P., Schwartz E., Markenson A. L., Graziano J. H., Levine L. S., New M. I., Hilgartner M. W. Depressed serum somatomedin activity in beta-thalassemia. J Pediatr. 1980 Feb;96(2):214–218. doi: 10.1016/s0022-3476(80)80805-5. [DOI] [PubMed] [Google Scholar]
  16. Scacchi M., Danesi L., De Martin M., Dubini A., Forni L., Masala A., Gallisai D., Burrai C., Terzoli S., Boffa C. Treatment with biosynthetic growth hormone of short thalassaemic patients with impaired growth hormone secretion. Clin Endocrinol (Oxf) 1991 Oct;35(4):335–339. doi: 10.1111/j.1365-2265.1991.tb03545.x. [DOI] [PubMed] [Google Scholar]
  17. Shehadeh N., Hazani A., Rudolf M. C., Peleg I., Benderly A., Hochberg Z. Neurosecretory dysfunction of growth hormone secretion in thalassemia major. Acta Paediatr Scand. 1990 Aug-Sep;79(8-9):790–795. doi: 10.1111/j.1651-2227.1990.tb11556.x. [DOI] [PubMed] [Google Scholar]
  18. Tanner J. M., Goldstein H., Whitehouse R. H. Standards for children's height at ages 2-9 years allowing for heights of parents. Arch Dis Child. 1970 Dec;45(244):755–762. doi: 10.1136/adc.45.244.755. [DOI] [PMC free article] [PubMed] [Google Scholar]
  19. Vullo C., Di Palma A. Compliance with therapy in Cooley's anaemia. Prog Clin Biol Res. 1989;309:43–49. [PubMed] [Google Scholar]

Articles from Archives of Disease in Childhood are provided here courtesy of BMJ Publishing Group

RESOURCES