Regulatory Affairs, Centers for Disease Control and Prevention, Atlanta, Georgia.
Enteric Diseases Epidemiology Branch, Division of Foodborne, Waterborne, and Environmental Diseases (DFWED), Centers for Disease Control and Prevention, Atlanta, Georgia.
Clin Infect Dis. 2017 Dec 27;66(suppl_1):S57-S64. doi: 10.1093/cid/cix816.
Botulism is a rare, life-threatening paralytic illness. Equine-derived heptavalent botulinum antitoxin (HBAT), the only currently available treatment for noninfant botulism in the United States, was licensed in 2013. No reports have systematically examined safety and clinical benefit of HBAT among botulism patients.
From March 2010 through March 2013, we collected data prospectively and through medical record reviews of patients with confirmed or suspected botulism who were treated with HBAT under an expanded-access Investigational New Drug program.
Among 249 HBAT-treated patients, 1 (<1%) child experienced an HBAT-related serious adverse event (hemodynamic instability characterized by bradycardia, tachycardia, and asystole); 22 (9%) patients experienced 38 nonserious adverse events reported by physicians to be HBAT related. Twelve (5%) deaths occurred; all were determined to be likely unrelated to HBAT. Among 104 (42%) patients with confirmed botulism, those treated early (≤2 days) spent fewer days in the hospital (median, 15 vs 25 days; P < .01) and intensive care (10 vs 17 days; P = .04) than those treated later. Improvements in any botulism sign/symptom were detected a median of 2.4 days and in muscle strength a median of 4.8 days after HBAT.
HBAT was safe and provided clinical benefit in treated patients. HBAT administration within 2 days of symptom onset was associated with shorter hospital and intensive care stays. These results highlight the importance of maintaining clinical suspicion for botulism among patients presenting with paralytic illness to facilitate early HBAT treatment before laboratory confirmation might be available. Clinical consultation and, if indicated, HBAT release, are available to clinicians 24/7 through their state health department in conjunction with CDC.
肉毒中毒是一种罕见的、危及生命的麻痹性疾病。马源七价肉毒毒素抗毒素（HBAT）是美国目前唯一可用于治疗非婴儿肉毒中毒的药物，于 2013 年获得许可。目前尚无报告系统地评估 HBAT 在肉毒中毒患者中的安全性和临床获益。
2010 年 3 月至 2013 年 3 月，我们通过扩大准入调查性新药计划，前瞻性收集和通过病历回顾，收集了接受 HBAT 治疗的确诊或疑似肉毒中毒患者的数据。
在 249 例接受 HBAT 治疗的患者中，1 例（<1%）儿童发生 1 例与 HBAT 相关的严重不良事件（表现为心动过缓、心动过速和心搏停止的血流动力学不稳定）；22 例（9%）患者发生 38 例医生报告与 HBAT 相关的非严重不良事件。12 例（5%）死亡；均被认为与 HBAT 无关。在 104 例（42%）确诊的肉毒中毒患者中，早期（≤2 天）接受治疗的患者在医院（中位数 15 天 vs 25 天；P <.01）和重症监护病房（10 天 vs 17 天；P =.04）的住院时间更短。HBAT 治疗后，中位 2.4 天出现任何肉毒中毒体征/症状改善，中位 4.8 天出现肌肉力量改善。
HBAT 是安全的，对治疗患者具有临床益处。在症状出现后 2 天内给予 HBAT 治疗与较短的住院时间和重症监护病房住院时间相关。这些结果突出表明，在出现麻痹性疾病的患者中，保持对肉毒中毒的临床怀疑很重要，以便在可能获得实验室确认之前及时进行 HBAT 治疗。临床咨询和，如果需要，HBAT 释放，通过州卫生部门与疾病预防控制中心合作，24/7 为临床医生提供。
