A controlled, open-label and randomized study was conducted to evaluate the safety and efficacy of the oral
iron chelator deferiprone (L1) in
thalassemia major patients from Hong Kong. Forty-nine patients were recruited in total (median age: 20 years; range: 8 to 40 years). The division of the patients was determined based on liver
iron content and put into either the poorly-chelated (Group I) or well-chelated (Group II) groups. In Group I, 20 patients received combined
therapy of L1 daily plus
desferrioxamine (DFO), in a reduced frequency of twice weekly, while the control group consisted of 16 patients who were treated with DFO alone. In Group II, six patients received L1 only, while the control group consisted of seven patients treated with DFO alone. Only patients who participated for longer than 6 months were analyzed for efficacy (n = 44). The median study period was 18 months. Transient and mild gastrointestinal upset (31%),
joint pain (15%) and liver
enzyme elevation (23%) were the most common side effects noted for L1. No case of
neutropenia was observed in this study. Serum
ferritin (SF) levels showed significant decline in the poorly-chelated patients using combined
therapy (L1 and reduced frequency DFO) as compared to those on DFO alone. However, their pre- and post-study liver
iron content was not significantly different. Evaluation of the well-chelated group demonstrated no significant change in SF or liver
iron content in both the study and control arms. We conclude that the short-term use of L1, with or without DFO, was safe and efficacious in our Chinese patient cohort. The long-term efficacy of reducing
iron overload by treatment regimens including L1 requires further study.