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Pilot Single-Blind Trial of AbobotulinumtoxinA in Oromandibular Dystonia.

ABSTRACT: Oromandibular dystonia (OMD) causes involuntary movements of masticatory and lingual muscles impairing eating, speaking, and swallowing. Treatment options are limited. The objective of this study was to determine the safety and efficacy of abobotulinumtoxinA (aboBoNTA) in OMD. A dose-finding study (phase 1) followed by a single session, prospective, single-blind trial (phase 2) was carried out. OMD subjects were evaluated at baseline, 6 and 12 weeks. Muscles injected were tailored to individual symptoms using EMG guidance, but the aboBoNTA dose for each muscle was pre-specified based on phase 1 results. Evaluations were Global Dystonia Rating Scale (GDS), Unified Dystonia Rating Scale (UDRS), Clinical Global Impression (CGI) improvement and severity, and quality of life (OMDQ-25). Adverse events were monitored. The lowest dosage in phase 1 resulted in adverse effects in two of three patients and thus was used in phase 2. In phase 2, adverse effects were observed in 50% of subjects including dysphagia, voice change, and soft palate weakness. Most were mild. Significant improvement was seen in quality of life (OMDQ-25), speech (BFMq21), and change in GDS, UDRS, CGI severity assessed by the unblinded investigator, but not in blinded video ratings. We conclude that aboBoNTA therapy in this study was associated with improved quality of life and was generally well tolerated in OMD, but occurrence of dysphagia dictated the importance of using low genioglossus dosing. Face to face assessment appears to be more sensitive than video assessment for change in OMD severity. Consideration of the disability in OMD places constraints on traditional placebo-control trial design. Development of novel trial designs is warranted.

SUBMITTER: Scorr LM

PROVIDER: S-EPMC5935649 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature

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Pilot Single-Blind Trial of AbobotulinumtoxinA in Oromandibular Dystonia.

Scorr Laura M LM Silver Michael R MR Hanfelt John J Sperin Elaine E Freeman Alan A Jinnah H A HA Factor Stewart A SA

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 20180401 2

Oromandibular dystonia (OMD) causes involuntary movements of masticatory and lingual muscles impairing eating, speaking, and swallowing. Treatment options are limited. The objective of this study was to determine the safety and efficacy of abobotulinumtoxinA (aboBoNTA) in OMD. A dose-finding study (phase 1) followed by a single session, prospective, single-blind trial (phase 2) was carried out. OMD subjects were evaluated at baseline, 6 and 12 weeks. Muscles injected were tailored to individual ...[more]

PMID: 29542022

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Project description:Background: Oromandibular dystonia manifests with sustained or task-specific contractions of the masticatory, tongue, and/or other muscles in the stomatognathic system. Since its symptoms can vary, it has been difficult to objectively measure disease severity and post-treatment changes. Objective: To develop and validate a comprehensive measurement tool for oromandibular dystonia. Methods: An examiner-rated scale included three subscales for severity, disability, and pain, modified specifically for oromandibular dystonia from the Toronto Western Spasmodic Torticollis Rating Scale-2. To evaluate the severity of each subtype of oromandibular dystonia, four of the six items were selected according to the subtype (jaw closing dystonia, tongue dystonia, jaw opening dystonia, jaw deviation [protrusion] dystonia, and lip dystonia). A patient-administered questionnaire based on clinical features and other relevant aspects associated with oromandibular dystonia was developed, which included five subscales: general, eating, speech, cosmetic, and social/family life. The questionnaire, examiner-rated scale, and four subscales (sleep, annoyance, mood, and psychosocial functioning) of the Cervical Dystonia Impact Profile-58 were combined to construct the oromandibular dystonia rating scale (OMDRS). The reliability and validity of the scale were assessed using clinimetric testing. Results: Six hundred and eighteen patients with oromandibular dystonia (394 women and 224 men; mean age, 51.7 years) were evaluated by the OMDRS. The overall OMDRS showed high-level internal consistency measured by Cronbach's alpha (0.95) with a logical factor structure. Cronbach's alpha for the subscales was satisfactory to excellent, ranging from 0.72 to 0.94. All items revealed acceptable inter-rater reliability (kappa > 0.4, interclass correlation coefficient > 0.6). Repeated ratings of videotapes revealed acceptable intra-rater reliability for all items (kappa > 0.76, interclass correlation coefficient > 0.86). Test-retest reliability showed a significant (p < 0.001) correlation efficiency. The OMDRS showed significant (p < 0.001) convergent and discriminant validity and significant (p < 0.001) sensitivity to changes after botulinum toxin therapy. Conclusion: The OMDRS can be useful for the comprehensive evaluation of disease severity, disability, psychosocial functioning, and impact on the quality of life as well as therapeutic changes in patients with oromandibular dystonia.

Project description:Shoulder pain is a very common presentation in primary care. Evidence of benefit for subacromial corticosteroid injection is inconclusive and confined largely to studies with short follow-up. We plan a large, definitive, primary-care-based trial to determine efficacy and safety in patients with rotator cuff tendinopathy, and conducted a pilot trial to explore feasibility.Six general practitioners (GPs) from Oxfordshire, UK underwent update training in assessing painful shoulders and injecting the subacromial space. Each then recruited patients aged 35 to 74 years from primary care complaining of shoulder pain lasting no more than 6 months. Eligible participants were randomized to receive either methylprednisolone acetate 40 mg with lidocaine 1% (total volume 1 ml), or lidocaine 1% alone (total volume 1 ml), injected into the subacromial space. The participants were blinded to treatment allocation. Feasibility outcomes were rates of recruitment, withdrawal, adherence to the protocol, completeness of follow-up, and success of patient masking. Clinical outcomes were the Oxford Shoulder Score (OSS) at baseline and at 4 and 12 weeks, and responses to three satisfaction questions at 2, 4 and 12 weeks. Outcome data were collected by postal questionnaires.A total of 40 participants were randomized (80% of the target 50 participants) over 26 weeks giving an overall recruitment rate of 1.5 participants per week. Rates of follow-up were maintained to a high level for the full 12 weeks. Four participants requested a 'rescue' corticosteroid injection but no patients withdrew. The trial GPs gave high scores for their confidence that the patient had remained blinded to treatment allocation during the procedure. The OSS at 4 and 12 weeks and the responses to the satisfaction questions are reported.It is feasible to recruit participants with shoulder pain in the primary care setting for a blinded, randomized trial of corticosteroid injection. Online randomization of participants from the practice is also feasible, and postal questionnaires provide an effective means of gathering outcome data in this area of study. The lessons learned from this pilot will usefully inform the design of a large, definitive efficacy trial in primary care.Current Clinical Trials ISRCTN82357435.

Dataset Information

Pilot Single-Blind Trial of AbobotulinumtoxinA in Oromandibular Dystonia.

Publications

Pilot Single-Blind Trial of AbobotulinumtoxinA in Oromandibular Dystonia.

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