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. 1974 Apr 6;2(5909):16–20. doi: 10.1136/bmj.2.5909.16

Long-term Chelation Therapy in Thalassaemia Major: Effect on Liver Iron Concentration, Liver Histology, and Clinical Progress

Michael Barry, David M Flynn, Elizabeth A Letsky, R A Risdon
PMCID: PMC1610120  PMID: 4821036

Abstract

A prospective trial of continuous chelation therapy in children with homozygous thalassaemia on a high transfusion regimen was started in April 1966. The effect of treatment on iron concentration in the liver and on hepatic histology was examined in 49 biopsy specimens obtained from nine chelator-treated patients and nine control patients between April 1966 and April 1973.

Chelation therapy was associated with a significant reduction in liver iron concentration, and by the end of the trial the values for the two groups showed no overlap. A similar difference was seen in the amount of hepatic fibrosis, which showed little or no change in the chelator-treated patients but was progressive in the controls.

No major clinical differences were seen, probably because both groups included several older children who were heavily loaded with iron at the start of the trial. Among the younger patients, however, puberty was delayed in four of five controls but in only one of four chelator-treated children. Prepubertal growth rate was significantly greater in the chelator-treated patients than in the controls.

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Selected References

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

  1. Barry M., Sherlock S. Measurement of liver-iron concentration in needle-biopsy specimens. Lancet. 1971 Jan 16;1(7690):100–103. doi: 10.1016/s0140-6736(71)90838-5. [DOI] [PubMed] [Google Scholar]
  2. Berry C. L., Marshall W. C. Iron distribution in the liver of patients with thalassaemia major. Lancet. 1967 May 13;1(7498):1031–1033. doi: 10.1016/s0140-6736(67)91543-7. [DOI] [PubMed] [Google Scholar]
  3. ELLIS J. T., SCHULMAN I., SMITH C. H. Generalized siderosis with fibrosis of liver and pancreas in Cooley's (Mediterranean) anemia; with observations on the pathogenesis of the siderosis and fibrosis. Am J Pathol. 1954 Mar-Apr;30(2):287–309. [PMC free article] [PubMed] [Google Scholar]
  4. FRUMIN A. M., WALDMAN S., MORRIS P. Exogenous hemochromatosis in Mediterranean anemia. Pediatrics. 1952 Mar;9(3):290–294. [PubMed] [Google Scholar]
  5. HOWELL J., WYATT J. P. Development of pigmentary cirrhosis in Cooley's anemia. AMA Arch Pathol. 1953 May;55(5):423–431. [PubMed] [Google Scholar]
  6. Marshall W. A., Tanner J. M. Variations in the pattern of pubertal changes in boys. Arch Dis Child. 1970 Feb;45(239):13–23. doi: 10.1136/adc.45.239.13. [DOI] [PMC free article] [PubMed] [Google Scholar]
  7. SMITH R. S. CHELATING AGENTS IN THE DIAGNOSIS AND TREATMENT OF IRON OVERLOAD IN THALASSEMIA. Ann N Y Acad Sci. 1964 Oct 7;119:776–788. doi: 10.1111/j.1749-6632.1965.tb54079.x. [DOI] [PubMed] [Google Scholar]
  8. WITZLEBEN C. L., WYATT J. P. The effect of long survival on the pathology of thalassaemia major. J Pathol Bacteriol. 1961 Jul;82:1–12. [PubMed] [Google Scholar]
  9. WOLMAN I. J. TRANSFUSION THERAPY IN COOLEY'S ANEMIA: GROWTH AND HEALTH AS RELATED TO LONG-RANGE HEMOGLOBIN LEVELS. A PROGRESS REPORT. Ann N Y Acad Sci. 1964 Oct 7;119:736–747. doi: 10.1111/j.1749-6632.1965.tb54075.x. [DOI] [PubMed] [Google Scholar]
  10. Wolman I. J., Ortolani M. Some clinical features of Cooley's anemia patients as related to transfusion schedules. Ann N Y Acad Sci. 1969 Nov 20;165(1):407–414. doi: 10.1111/j.1749-6632.1969.tb27811.x. [DOI] [PubMed] [Google Scholar]

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