Oh S J
Distinguished Professor of Neurology Emeritus, Department of Neurology, University of Alabama at Birmingham, Alabama, USA.
Drugs Today (Barc). 2020 Oct;56(10):623-641. doi: 10.1358/dot.2020.56.10.3137144.
Lambert-Eaton myasthenic syndrome (LEMS) is a presynaptic autoimmune disabling neuromuscular disease caused by antibodies against presynaptic voltage-gated calcium channels. It reduces the quantal release of acetylcholine (Ach), causing muscle weakness, reduced or absent reflex and dysautonomia. About half of LEMS patients have associated small cell lung cancer. For symptomatic treatment, amifampridine (3,4-diaminopyridine [3,4-DAP]) is ideal because it increases the release of Ach at the presynaptic membrane. Since the first use of 3,4-DAP in LEMS patients in the 1980s, 136 LEMS patients were treated with amifampridines in the open-label studies and 208 patients in the eight randomized studies. These studies showed that amifampridine is the most effective drug for symptomatic treatment in LEMS. Now, 3,4-DAPP (3,4-DAP phosphate) is approved for adult LEMS patients and 3,4-DAP for pediatric patients. The recommended dose is 80 mg a day, divided 3 or 4 times a day. Side effects are usually mild, and the most frequently reported are paresthesia.
兰伯特-伊顿肌无力综合征（LEMS）是一种由抗突触前电压门控钙通道抗体引起的突触前自身免疫性致残性神经肌肉疾病。它减少了乙酰胆碱（Ach）的量子释放，导致肌肉无力、反射减弱或消失以及自主神经功能障碍。约一半的LEMS患者伴有小细胞肺癌。对于症状性治疗，氨吡啶（3,4-二氨基吡啶[3,4-DAP]）是理想药物，因为它可增加突触前膜Ach的释放。自20世纪80年代首次在LEMS患者中使用3,4-DAP以来，在开放标签研究中有136例LEMS患者接受了氨吡啶治疗，在八项随机研究中有208例患者接受了治疗。这些研究表明，氨吡啶是LEMS症状性治疗中最有效的药物。目前，3,4-DAPP（3,4-二氨基吡啶磷酸盐）已被批准用于成年LEMS患者，3,4-DAP用于儿科患者。推荐剂量为每日80毫克，分3或4次服用。副作用通常较轻，最常报告的是感觉异常。
