Bartlett A N, Hoffbrand A V, Kontoghiorghes G J
Department of Haematology, Royal Free Hospital, School of Medicine, London.
Br J Haematol. 1990 Oct;76(2):301-4. doi: 10.1111/j.1365-2141.1990.tb07888.x.
1,2-Dimethyl-3-hydroxypyrid-4-one (L1) has been given daily for 1-15 months to 13 transfusion dependent iron loaded patients. No significant change occurred in liver, renal or cardiac function, ECG and radionucleotide angiocardiogram, in audiometry tests and in visual function and electrical retinography. No skin rashes, gastrointestinal symptoms and no neurological changes that could be detected clinically were observed. Two of the patients died of their underlying diseases. One patient had severe cardiac abnormalities before receiving L1 and died of congestive heart failure with infections 5 weeks after stopping a 2-month course of L1. The other, a patient with myelodysplasia suffered recurring infections due to progression of the disease. Joint and muscle pains occurred in five patients. In two these disappeared despite continuing the drug; another patient developed swollen ankle joints which gradually resolved on stopping L1 therapy; a patient with underlying osteoarthritis complained of mild pain and stiffness in her knees which remained intermittent both on and off the drug while in the fifth patient peripheral small joint swelling and pain present before starting L1 improved with L1 therapy. One patient, with Blackfan Diamond anaemia, developed a Lw red cell antibody 6 months after commencing L1. This disappeared on stopping the drug and did not reappear. She then developed severe agranulocytosis and thrombocytopenia 6 weeks after recommencing L1 after 3 months discontinuation of the drug. No other patient showed a change in granulocyte or platelet count.
已对13名依赖输血的铁过载患者每日给予1,2 - 二甲基 - 3 - 羟基吡啶 - 4 - 酮（L1），持续1至15个月。肝脏、肾脏或心脏功能、心电图、放射性核素血管造影、听力测试、视觉功能及视网膜电图均无显著变化。未观察到皮疹、胃肠道症状以及临床可检测到的神经变化。两名患者死于基础疾病。一名患者在接受L1之前有严重心脏异常，在停止为期2个月的L1疗程5周后死于充血性心力衰竭伴感染。另一名患有骨髓发育异常的患者因疾病进展反复感染。五名患者出现关节和肌肉疼痛。其中两名患者尽管继续用药疼痛仍消失；另一名患者出现踝关节肿胀，在停止L1治疗后逐渐消退；一名患有原发性骨关节炎的患者抱怨双膝轻度疼痛和僵硬，用药和停药时均为间歇性发作；第五名患者在开始L1治疗前存在的外周小关节肿胀和疼痛经L1治疗后有所改善。一名患有先天性纯红细胞再生障碍性贫血的患者在开始使用L1 6个月后产生了Lw红细胞抗体。停药后该抗体消失且未再次出现。在停药3个月后重新开始使用L1 6周后，她出现了严重的粒细胞缺乏症和血小板减少症。其他患者均未出现粒细胞或血小板计数变化。
