1983年,报道了3,4-二氨基吡啶(3,4-DAP)在Lambert-Eaton肌无力综合征(LEMS)中的首次成功试验。在3,4-DAP中的7项随机研究和3,4-DAPP中的2项随机研究证明了氨苯吡啶(3,4-DAP和3,4-二氨基吡啶磷酸盐[3,4-DAPP])对LEMS的对症治疗的有效性。美国食品和药物管理局于2018年批准3,4-DAPP用于成人LEMS,并于2022年批准用于小儿LEMS。文献中确定了19例小儿LEMS病例。与成人LEMS相比,儿童LEMS的恶性肿瘤发生率低于预期,自主神经失调的发生率也较低.意外发现是2例先前感染后的小儿LEMS。只要成人LEMS患者的日剂量小于每天80毫克,小儿LEMS患者的日剂量小于每天30毫克,阿米非替丁就可以安全使用。对于长期使用,可以用自由量的吡啶斯的明来补充氨法霉素。在19例小儿LEMS患者中,有8例(42%)使用了氨苯吡啶作为对症治疗:6例3,4-DAP和2例3,4-DAPP。3,4-DAP的最常见做法是在四名患者中与吡啶斯的明联合使用。有了3,4-DAP,3例报告活动正常,另外3例报告轻度至中度改善。在两名3,4-DAPP患者中,一个显着改善,一个没有改善。阿米发胺被证明是有效和安全的药物对症治疗,在成人和儿童中没有严重的副作用,只要剂量正确遵守。
In 1983, the first successful trial of 3,4-diaminopyridine (3,4-DAP) in Lambert-Eaton myasthenic syndrome (LEMS) was reported. Efficacy of amifampridine (3,4-DAP and 3,4-diaminopyridine phosphate [3,4-DAPP]) for symptomatic treatment in LEMS was proven by seven randomized studies in 3,4-DAP and two randomized studies in 3,4-DAPP. US Food Drug Administration approved 3,4-DAPP usage for adult LEMS in 2018 and for pediatric LEMS in 2022. Nineteen pediatric LEMS cases were identified in the literature. Compared with adult LEMS, the rate of malignancy is low as expected and the rate of dysautonomia is also low in pediatric LEMS. Unexpected finding is two cases of pediatric LEMS following antecedent infection. Amifampridine can be safely used as long the daily dose is less than 80 mg a day for adult LEMS patients and less than 30 mg a day for pediatric LEMS patients. Amifampridines can be supplemented with a liberal amount of pyridostigmine for long term usage. Amifampridine was used as symptomatic treatment in eight (42%) of 19 pediatric LEMS patients: 3,4-DAP in six and 3,4-DAPP in two patients. The most common practice of 3,4-DAP was a combination with pyridostigmine in four patients. With 3,4-DAP, normal activity was reported in 3 cases and mild to moderate-improvement in other 3 cases. In two patients with 3,4-DAPP, significant improvement in one and no improvement in one. Amifampridines are proven to be effective and safe drugs for the symptomatic treatment without serious side reaction in adults as well as in children as long as the dosage is properly adhered.