Placebo response in degenerative cerebellar ataxias: a descriptive review of randomized, placebo-controlled trials

Ji Hyun Choi, Chaewon Shin, Han Joon Kim, Beomseok Jeon

Research output: Contribution to journalReview articlepeer-review

Abstract

Placebo response in degenerative cerebellar ataxias (CAs) has never been studied despite the large number of randomized controlled trials (RCTs) that have been conducted. In this descriptive review, we aimed to examine the placebo response in patients with CAs. We performed a literature search on PubMed for RCTs on CAs that were published from 1977 to January 2020 and collected data on the changes from the baseline to the endpoint on various objective ataxia-associated clinical rating scales. We reviewed 56 clinical trials, finally including 35 parallel-group studies and excluding 21 cross-over studies. The included studies were categorized as follows: (1) studies showing significant improvements in one or more ataxia scales in the placebo groups (n = 3); (2) studies reporting individual placebo responders with improvements in one or more ataxia scales in the placebo groups (n = 5)—the overall proportion of placebo responders was 31.9%; (3) studies showing mean changes in the direction of improvement in at least one ataxia scale in the placebo groups, though not statistically significant (n = 19); (4) studies showing no placebo response in any of the ataxia scales in the placebo groups (n = 4); (5) studies where data on the placebo groups were unavailable (n = 9). This review demonstrated the placebo response in patients with CAs on various objective ataxia scales. Our study emphasizes that the placebo response should be considered when designing, analyzing, and interpreting clinical trials and in clinical practice in CA patients.

Original languageEnglish
Pages (from-to)62-71
Number of pages10
JournalJournal of Neurology
Volume269
Issue number1
DOIs
StatePublished - Jan 2022

Keywords

  • Cerebellar ataxia
  • Degenerative
  • Placebo response
  • Randomized controlled trials

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