Agarwal M B, Gupte S S, Vasandani D, Viswanathan C, Puniyani R R, Ramanathan J, Massil D E, Shah S, Rajyadhyaksha G C, Bhave A A
Department of Medicine and Pathology, LTMG Hospital, Bombay.
J Assoc Physicians India. 1991 Sep;39(9):669-72.
Twenty-four patients with beta thalassaemia major, aged 8-22 years (mean 15.3 +/- 8.1) were given 1-2, dimethyl-3-hydroxypyrid-4-one (L1) orally for a period of three months. The drug was given in the dose of 25 mg/Kg/day for the first week and gradually increased to 100 mg/Kg/day which was continued until 3 months. The mean urinary iron excretion was 5.73 +/- 3.648 mg/day on 25 mg/Kg/day of L1; 15.2 +/- 11.225 mg/day on 50 mg/Kg/day; 24.2 +/- 12.69 mg/day on 75 mg/Kg/day and 36.3 +/- 19.4 mg/day on 100 mg/Kg/day of L1. Serum ferritin estimated by ELISA before and 3 months after L1 therapy in 21 patients showed significant drop in levels, the mean drop being 964.3 +/- 844.4 (P less than 0.001). The only side-effects noted were transient gastrointestinal symptoms in 5 patients and skeletomuscular pain in 3 patients. Both these groups of symptoms were of transient nature. The efficacy of L1 appears to be excellent and equivalent to the standard iron chelation therapy available at present i.e. desferrioxamine. It appears to be free of major toxicity. L1 is also a specific chelator for iron as it does not deplete trace metals. L1 appears to be a cheap and effective oral alternative to desferrioxamine for treating iron overloading.
24例重型β地中海贫血患者，年龄8 - 22岁（平均15.3±8.1岁），口服1,2 - 二甲基 - 3 - 羟基吡啶 - 4 - 酮（L1），疗程3个月。药物给药剂量为第一周25mg/kg/天，然后逐渐增加至100mg/kg/天并持续至3个月。服用25mg/kg/天L1时，平均尿铁排泄量为5.73±3.648mg/天；50mg/kg/天为15.2±11.225mg/天；75mg/kg/天为24.2±12.69mg/天；100mg/kg/天L1时为36.3±19.4mg/天。21例患者在L1治疗前及治疗3个月后通过ELISA法测定血清铁蛋白，结果显示水平显著下降，平均下降964.3±844.4（P＜0.001）。仅观察到5例患者出现短暂的胃肠道症状，3例患者出现骨骼肌肉疼痛这两种副作用。这两组症状均为短暂性。L1的疗效似乎极佳，等同于目前可用的标准铁螯合疗法即去铁胺。它似乎没有重大毒性。L1也是一种铁特异性螯合剂，因为它不会消耗微量金属。L1似乎是一种比去铁胺便宜且有效的口服替代药物，用于治疗铁过载。
