Agarwal M B, Gupte S S, Viswanathan C, Vasandani D, Ramanathan J, Desai N, Puniyani R R, Chhablani A T
Department of Haematology, L.T.M.G. Hospital, Bombay, India.
Br J Haematol. 1992 Oct;82(2):460-6. doi: 10.1111/j.1365-2141.1992.tb06445.x.
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 micrograms/l after 5.0 +/- 0.8 months to 3641.2 +/- 2299.3 micrograms/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.
1989年8月至1991年5月，52例依赖输血的重型地中海贫血患者接受了口服铁螯合剂L1（1,2 - 二甲基 - 3 - 羟基吡啶 - 4 - 酮）治疗，为期3 - 21个月（平均±标准差：14.2±6.8）。L1剂量为25mg/kg/d时，平均（±标准差）尿铁排泄量为6.2±4.6mg/d；L1剂量为100mg/kg/d时，平均（±标准差）尿铁排泄量为42.3±37.1mg/d。治疗5.0±0.8个月后，血清铁蛋白平均（±标准差）下降1465±990μg/L；治疗20.1±0.9个月后，血清铁蛋白降至3641.2±2299.3μg/L。未发现中性粒细胞减少、血小板减少、耳或眼毒性的证据。20例患者（38.5%）出现与L1相关的关节痛，减少剂量或停药后可逆转；7例（3.5%）出现轻微胃肠道症状。我们得出结论，虽然L1是一种有效的铁螯合剂，但需要进一步研究以了解L1相关关节痛的机制，并找到一种更安全但有效的剂量，使L1相关关节痛的发生率降至最低。
