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Botulinum toxin treatment improves dysphagia in patients with oculopharyngeal muscular dystrophy and sporadic inclusion body myositis

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OBJECTIVE: 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.

METHODS: 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.

RESULTS: 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.

CONCLUSION: 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.

PROSPECTIVE TRIAL REGISTRATION: EudraCT-number: 2014-002210-23.

Original languageEnglish
JournalJournal of Neurology
Volume269
Issue number8
Pages (from-to)4154-4160
Number of pages7
ISSN0340-5354
DOIs
Publication statusPublished - Aug 2022

Bibliographical note

© 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany.

    Research areas

  • Botulinum Toxins, Type A/adverse effects, Deglutition Disorders/drug therapy, Humans, Muscular Dystrophy, Oculopharyngeal/complications, Myositis, Inclusion Body/complications, Neuromuscular Agents/adverse effects, Prospective Studies, Water

ID: 79737573