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Eligibility criteria in clinical trials for cervical dystonia

      Highlights

      • Eligibility measures were reviewed in 134 clinical trials of cervical dystonia.
      • Diagnostic criteria varied across studies and mainly consisted in clinical judgement.
      • Overall eligibility measures were poorly consistent and heterogeneous.
      • Definitions of adequate/insufficient response to botulinum neurotoxins were not consolidated.
      • Consensus on eligibility criteria is necessary for designing future clinical trials.

      Abstract

      Introduction

      Cervical dystonia (CD) is the most common form of adult-onset focal dystonia. Because of a heterogeneous clinical presentation, the diagnosis rests on clinical opinion. During the last decades, several clinical trials have tested safety and efficacy of medical and surgical treatments for CD. We analyzed all the published CD trials and reviewed the strategies adopted for patient enrollment.

      Methods

      The review included clinical trials in patients with CD published in PubMed. Studies were excluded if reviews, meta-analyses, post-hoc analyses on pooled data, or if not reporting a treatment for CD.

      Results

      A total of 174 articles were identified; 134 studies met inclusion criteria. Diagnosis of CD varied among studies and in most cases was based on clinical judgement, using different descriptors such as “cervical dystonia” (37 studies), “idiopathic or isolated CD” (35), “primary CD” (13), and “torticollis” (40). Clinical judgement was supported by a phenomenological description of dystonia in four studies, and by a specific diagnostic strategy in other four. Finally, one study adopted general diagnostic criteria for dystonia. Inclusion and exclusion criteria proved heterogeneous across trials and were defined only in 108 studies, mainly considering age or the phenomenological pattern of muscle involvement.

      Conclusion

      The review showed lack of consolidated diagnostic criteria and non-uniformity of eligibility criteria for CD across clinical trials. There is need to move beyond clinical judgement as diagnostic criterion for selecting participants. New trials assessing specific CD patient subgroups or comparing medical and surgical procedures will need grounds that are more consistent.
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