背景:为了报告我们对7名患者(4名男性和3名女性)的经验,受非副肿瘤性Lambert-Eaton肌无力综合征影响,用3,4-二氨基吡啶磷酸酯(3,4-DAPP)单独或与其他免疫抑制剂或类固醇组合处理。
方法:已在特定时间点对患者进行了评估(即,基线和最后5年随访),神经系统检查,针对突触前电压门控Cav2.1(P/Q型)钙离子通道(VGCC)剂量的自身抗体,神经生理学评估侧重于最大自愿努力后复合肌肉动作电位(cMAP)的振幅增加,定量重症肌无力(QMG)和日常生活活动量表,和自主神经系统受累评估。
结果:在最近5年的随访中,7例患者中有5例表现出持续的临床改善;其中2例在最大剂量下仅服用3,4-DAPP时有所改善,而其余的人接受了合并用药,如泼尼松和硫唑嘌呤。然而,临床改善无统计学意义.没有一个患者报告严重的不良事件,除了一个,抱怨短暂的下巴和口周感觉异常。QMG与药物治疗类型之间存在显着关联(P=.028)。的确,我们观察到3例接受3,4-DAPP和泼尼松治疗的受试者的临床状况均有所改善。
结论:在这项研究中,我们确认3,4-DAPP治疗对肌肉力量的疗效,但是在自主神经系统受累和深肌腱反射重新出现方面已经证明了药物有效性的次要证据,一部分来自接受与泼尼松相关的3,4-DAPP的患者。
BACKGROUND: To report our experience on 7 patients (4 males and 3 females), affected by nonparaneoplastic Lambert-Eaton myasthenic syndrome, treated with 3,4-diaminopyridine phosphate (3,4-DAPP) either alone or in combination with other immunosuppressants or steroids.
METHODS: Patients have been evaluated at specific timepoints (ie, baseline and last 5 year follow-up), with neurological examination, autoantibodies against presynaptic voltage-gated Cav2.1 (P/Q type) calcium ion channel (VGCC) dosage, neurophysiological evaluation focusing on the increased amplitude of the compound muscle action potential (cMAP) after maximum voluntary effort, quantitative myasthenia gravis (QMG) and activities of daily living scales, and autonomic nervous system involvement evaluation.
RESULTS: Five out of 7 patients presented a clinical improvement persisting at last 5-year follow-up; 2 out of them improved taking only 3,4-DAPP at the maximal dosage, whereas the remaining received concomitant medications, such as prednisone and azathioprine. However, the clinical amelioration was not statistically significant. No one of the patients reported severe adverse events, except one, complaining of transient chin and perioral paresthesias. A significant association between QMG and the type of pharmacological drugs therapy (P = .028) emerged. Indeed, we observed an improvement of the clinical condition in all 3 subjects treated with 3,4-DAPP and prednisone.
CONCLUSIONS: In this study, we confirm 3,4-DAPP treatment efficacy on muscle strength, but minor evidence of drug effectiveness have been demonstrated on the autonomic nervous system involvement and on the deep tendon reflexes reappearance, a part from patients who received 3,4-DAPP associated to prednisone.