Hepatic iron concentration combined with long-term monitoring of serum ferritin to predict complications of iron overload in thalassaemia major

Clinical complications of transfusional iron overload are still common in p atients with thalassaemia major (TM) and it is not clear how best to monito r body iron stores during long-term follow-up to anticipate tissue damage. In this study, we have reviewed a group of 32 patients who underwent liver biopsy between 1984 and 1986. We developed a method of assessing the trend in serum ferritin (TSF) during long-term monitoring and compared this with mean serum ferritin (MSF) and initial liver iron (LI) concentration to dete rmine whether, individually or in combination, they were accurate in predic ting clinical outcome. LI levels were low (< 7 mg/g), medium (7-15 mg/g) an d high (> 15 mg/g dry weight) in 15, 7 and 10 patients respectively. MSF wa s low (< 1500 mu g/l), medium (1500-2500 mu g/l) and high (> 2500 mu g/l) i n 10, 14 and 8 patients. TSF was low, medium and high risk in 9, 9 and 11 o ut of 29 evaluable patients. During a median follow-up of 13.6 years (range 2.3-14.8 years) after biopsy, nine patients died and an additional three p atients developed heart failure. Hypothyroidism developed in five, hypopara thyroidism in four, and diabetes mellitus in seven patients. Cirrhosis deve loped in four of 10 evaluable patients. The clinical end-point of death or cardiac failure was significantly associated with increasing iron load usin g all three means of assessment. Although numbers were insufficient for sta tistical analysis, MSF or TSF were more closely associated with complicatio ns of iron overload than LI. There was no clear additional value in combini ng LI with MSF or TSF. The data show that quantitation of liver iron from a single liver biopsy has little value in long-term monitoring of iron store s. Most complications can be avoided if ferritin levels can be brought down to < 1500 mu g/l.