Department of Neurology, Copenhagen Neuromuscular Center and Department of Neurology, Rigshospitalet and Copenhagen University, Copenhagen, Denmark.
Department of Neurology, Bispebjerg Hospital, Copenhagen, Denmark.
J Neurol. 2022 Aug;269(8):4154-4160. doi: 10.1007/s00415-022-11028-8. Epub 2022 Mar 4.
Dysphagia can be troublesome in sporadic inclusion body myositis (sIBM) and oculopharyngeal muscular dystrophy (OPMD), but no established treatment exists. Cricopharyngeal muscle botulinum toxin injection has at case level been reported to be effective. We evaluated safety and efficacy of botulinum toxin injections in the cricopharyngeal muscle in patients with dysphagia due to sIBM or OPMD.
Participants were included from our outpatient clinic. Cricopharyngeal constriction was confirmed by laryngoscopy. After EMG confirmation of needle placement in the cricopharyngeal muscle, botulinum toxin A was injected in awake patients. An individualized dose of 5-10 units of botulinum toxin A was applied initially and titrated up a maximum of 3 times. Outcome measures were change in dysphagia questionnaire, timed cold-water swallow test and subjective dysphagia status (worse, unchanged, improved). Due to the need for individualized dosing and a limited number of available patients, an uncontrolled, un-blinded design was used.
Thirteen patients, 3 with OPMD, received at least 1 injection. In the dysphagia questionnaire, all but 2 subjects, none with subjective worsening, improved (p < 0.001). Subjectively, seven felt an improvement, 4 no change and 2 a worsening. No overall change was seen the timed cold-water swallow test. No serious adverse events were observed.
Botulinum toxin injection of the cricopharyngeal muscle in patients with OPMD and sIBM had a beneficial effect on dysphagia in most of the treated patients. Two of 13 patients experienced a temporary worsening not reflected in dysphagia score. Limitations are the un-blinded and un-randomized design and subjective assessments methods.
EudraCT-number: 2014-002210-23.
吞咽困难在散发型包涵体肌炎（sIBM）和口咽型肌营养不良症（OPMD）中较为棘手，但目前尚无既定的治疗方法。已有个案报道称，甲状舌骨肌肉毒毒素注射治疗有效。我们评估了肉毒毒素注射治疗 sIBM 或 OPMD 相关吞咽困难患者甲状舌骨肌的安全性和有效性。
参与者从我们的门诊中选取。通过喉镜检查确认环咽肌收缩。在肌电图确认针尖位于甲状舌骨肌后，在清醒患者中注射肉毒毒素 A。初始剂量为 5-10 个单位，最多可滴定 3 次。评估指标为吞咽困难问卷、冷饮水吞咽时间测试和主观吞咽困难状况（恶化、不变、改善）的变化。由于需要个体化剂量和有限的患者人数，因此采用了非对照、非盲设计。
13 例患者（3 例 OPMD）至少接受了 1 次注射。在吞咽困难问卷中，除 2 例患者外，其余患者均有所改善（p<0.001），但无主观恶化。主观上，7 例患者感觉有所改善，4 例无变化，2 例感觉恶化。冷饮水吞咽时间测试未见总体变化。未观察到严重不良事件。
肉毒毒素注射治疗 OPMD 和 sIBM 患者的环咽肌对大多数接受治疗的患者的吞咽困难有有益影响。13 例患者中有 2 例出现暂时性恶化，但未反映在吞咽困难评分中。局限性在于非盲法和非随机设计以及主观评估方法。
EudraCT 编号：2014-002210-23。
