 |
|
A Prospective Evaluation of Iron Chelation Therapy in Children With Severe β-ThalassemiaA Six-Year Study
Helen S. Maurer, MD;
John D. Lloyd-Still, MD;
Carol Ingrisano, RN;
Frank Gonzalez-Crussi, MD;
George R. Honig, MD, PhD
Am J Dis Child. 1988;142(3):287-292.
Abstract
• Sixteen patients (age range, 3 to 17 years) with transfusion-dependent β-thalassemia major were studied prospectively, beginning at the onset of chelation therapy with deferoxamine (desferrioxamine). A liver biopsy specimen was obtained from each patient at the start of the study, and periodically thereafter. Liver histologic features, iron content, and iron excretion were assessed during the course of the study. Hepatic iron levels from liver biopsy specimens appeared to correlate well with serum ferritin levels in the younger less heavily iron-loaded patients; however, in patients with higher serum ferritin levels, hepatic iron appeared to reach a saturation level. Fourteen of the 16 patients showed a pattern of marbled fibrosis of the liver in their initial biopsy specimens. Follow-up biopsy specimens from nearly all of the patients showed a substantial reduction in iron concentration, but only two of seven patients showed improvement in the degree of hepatic fibrosis three to five years later. Patients less than 8 years old exhibited a normal pattern of linear growth until approximately the age of 10 years, followed by a progressive decrease to the 30th to 40th percentile. Two patients, aged 18 and 22 years, died of cardiac disease during the study. These findings suggest that chelation therapy in patients with transfusion-dependent thalassemia needs to be initiated at an early age, possibly before 3 years, if significant liver fibrosis and growth impairment are to be effectively prevented.
(AJDC 1988;142:287-292)
Author Affiliations
From the Divisions of Hematology (Drs Maurer and Honig and Ms Ingrisano) and Gastroenterology (Dr Lloyd-Still), Department of Pediatrics and the Department of Pathology (Dr Gonzalez-Crussi), Children's Memorial Hospital, Northwestern University Medical School, Chicago. Dr Maurer is now with the University of Illinois, Chicago.
Footnotes
Accepted for publication Oct 10, 1987.
A preliminary account of this study was presented at a meeting of the American Association for the Study of Liver Disease, Chicago, Nov 6, 1985.
Reprint requests to University of Illinois, Department of Pediatrics (M/C 856), 840 S Wood St, Chicago, IL 60612 (Dr Maurer).
 CiteULike  Connotea  Delicious  Digg  Facebook  Reddit  Technorati  Twitter
What's this?
THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES
Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia
Wanless et al.
Blood 2002;100:1566-1569.
ABSTRACT
| FULL TEXT
Liver disease in transfusion dependent thalassaemia major
Li et al.
Arch. Dis. Child. 2002;86:344-347.
ABSTRACT
| FULL TEXT
Iron-Chelating Therapy and the Treatment of Thalassemia
Olivieri and Brittenham
Blood 1997;89:739-761.
FULL TEXT
|
