Dataset Information

Tetrabenazine in treatment of hyperkinetic movement disorders: an observational study.

ABSTRACT:

Background

Tetrabenazine (TBZ) is commonly used in hyperkinetic movement disorders. In this retrospective study, we aimed to assess the TBZ effectiveness and adverse events (AEs) in Huntington disease (HD), vascular chorea, tics, dystonia, tardive oromandibular (OM) dyskinesia and other tardive syndromes (TS).

Methods

Qualitative analysis of clinical response was used to estimate TBZ effectiveness. TBZ-associated AE frequency and subsequent discontinuation rate were used to estimate tolerability; the tolerability profile was measured through the TBZ minimal dose and exposure time required to elicit AEs.

Results

Of 108 included patients, 87% had a clinically meaningful improvement sustained over a period of 40 months. TBZ-responder rate ranged from 100% in HD to 62.5% and 77.1% in tic disorders and OM dyskinesia, respectively (p < 0.001). TBZ-associated AE frequency ranged from 40.9% in other TS and 41.7% in vascular chorea and HD, to 60% in OM dyskinesia (p < 0.001). The most common AEs were Parkinsonism (51.8%) and psychiatric disorders (25%). The 'other AEs' category (mainly somnolence) presented the shortest minimal exposure time (3 months). AE-eliciting dose differed from 18.8 mg and 25 mg in tics and tardive disorders, to 75 mg in HD (p = 0.003). Patients with AEs were tendentiously older at TBZ initiation (p = 0.022).

Conclusions

TBZ proved an effective and relatively well tolerated treatment in hyperkinetic disorders, with excellent results in HD. AEs were more common in OM dyskinesia, which may be related to higher age at TBZ initiation. TBZ-associated somnolence and Parkinsonism were more frequent during the titration and maintenance periods, respectively.

SUBMITTER: Miguel R

PROVIDER: S-EPMC5367646 | biostudies-literature | 2017 Feb

REPOSITORIES: biostudies-literature

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Publications

Tetrabenazine in treatment of hyperkinetic movement disorders: an observational study.

Miguel Rita R Mendonça Marcelo D MD Barbosa Raquel R Ladeira Filipa F Lampreia Tânia T Vale José J Bugalho Paulo P

Therapeutic advances in neurological disorders 20161121 2

<h4>Background</h4>Tetrabenazine (TBZ) is commonly used in hyperkinetic movement disorders. In this retrospective study, we aimed to assess the TBZ effectiveness and adverse events (AEs) in Huntington disease (HD), vascular chorea, tics, dystonia, tardive oromandibular (OM) dyskinesia and other tardive syndromes (TS).<h4>Methods</h4>Qualitative analysis of clinical response was used to estimate TBZ effectiveness. TBZ-associated AE frequency and subsequent discontinuation rate were used to estima ...[more]

PMID: 28382107

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Project description:ObjectiveThis proof-of-concept feasibility trial examined the potential of the Cognitive Orientation to daily Occupational Performance Approach (CO-OP) to augment deep brain stimulation (DBS) outcomes in childhood-onset hyperkinetic movement disorders (HMD) including dystonia and dyskinetic cerebral palsy.MethodsThis is a single case experimental design using multiple baseline as n-of-1 trial comprising 10 intervention sessions, with replications across participants (n = 10). Treatment focused on 3 participant-selected goals. Transfer was assessed on 2 additional untreated goals. Individuals enrolled were 6-21 years of age and had DBS in situ and sufficient manual ability. Primary outcome was functional performance change on the Performance Quality Rating Scale-Individualized (PQRS-i) measured before, during, and posttreatment, and at 3-month follow-up. Assessors of outcome were blinded to time of assessment, number of intervention session, and treatment allocation. To measure effect size, a nonoverlapping index, Tau-U, was used. Feasibility measures were captured.ResultsOne participant withdrew before baseline assessment. Effect sizes of at least 0.66 were seen at both posttreatment and follow-up with all participants showing improvements in at least one trained goal in PQRS-i. Six participants improved on all 3 goals and 2 improved on 2 trained goals. Two children showed deterioration in one trained goal each. Transfer to untrained goals was observed in 3 participants for a total of 5 goals. CO-OP was feasible and acceptable to all participants.ConclusionA cognitive-based, task-oriented approach to support performance of personally relevant functional skills enabling participation is acceptable in childhood-onset HMD post-DBS. Further, preliminary efficacy to improve outcomes and proof of concept with CO-OP has been established in this population.Classification of evidenceThis study provides Class IV evidence that for children with HMD who had undergone DBS, CO-OP improves performance of personally relevant functional skills.

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Dataset Information

Tetrabenazine in treatment of hyperkinetic movement disorders: an observational study.

Background

Methods

Results

Conclusions

Publications

Tetrabenazine in treatment of hyperkinetic movement disorders: an observational study.

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