Long-term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion dependent thalassaemia: Indian trial.

Summary. From August 1989 to May 1991. 52 patients with transfusion dependent thalassaemia major received L1 (1.2-dimethyl-3-hydroxypyrid-4-one), the oral iron chelator, for a period of 3–21 months (mean±SD: 14.2±6.8). Mean (±SD) urinary iron excretion varied from 6.2±4.6 mg/d on 25 mg/kg/d of L1 to 42.3±37.1 mg/d on 100 mg/kg/d of L1. Mean (±SD) drop in S ferritin was 1465±990 μg/l after 5.0±0.8 months to 3641.2 ±2299.3 μg/l after 20.1 ±0.9 months of therapy. There was no evidence of neutropenia, thrombocytopenia, ear or eye toxicity. L1-related arthralgia, which was reversible on dose reduction or stoppage, was seen in 20 patients (38.5%), while minor gastrointestinal (GI) tract symptoms occurred in seven (3.5%) cases. We conclude that although L1 is an effective iron chelator, further studies are required to understand the mechanism of L1 related arthralgia and also to find a safer but effective dose on which incidence of L1 related arthralgia is minimal.