Summary of findings 7. Betamethasone versus placebo for speech disorder resulting from hereditary ataxias.
| Betamethasone (BETA) for speech disorder resulting from hereditary ataxias | ||||||
| Patient or population: people with speech disorder resulting from hereditary ataxias Settings: university Intervention: betamethasone | ||||||
| Outcomes | Illustrative comparative risks* (95% CI) | Relative effect (95% CI) | No of participants (studies) | Quality of the evidence (GRADE) | Comments | |
| Assumed risk | Corresponding risk | |||||
| Placebo | BETA | |||||
| Short‐term (1 week) percentage change (improvement) in overall speech production | See comment | See comment | Not estimable | 0 (0) | See comment | Not an outcome in this study |
| Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production ICARS. Scale from: 0 to 8. Higher scores indicate more severe disorder. Follow‐up: mean 30 days | The median short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production in the control groups was a reduction of 0.5 | The median short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production in the intervention groups was a 0.1 greater reduction (0.5 lower to 2.5 lower))2 | Not estimable | 13 (1 cross‐over study) | ⊕⊕⊕⊝ moderate1 | Statistically significant difference between groups (P value = 0.02) Changes were reported as medians |
| Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments | See comment | See comment | Not estimable | 0 (0) | See comment | Not an outcome in this study |
| Longer‐term (minimum 1 month) change in generic quality of life scores Child Health Questionnaires Follow‐up: mean 30 days | See comment | See comment | Not calculable | 0 (0) | See comment | Data not presented. No difference reported between groups |
| Adverse effects (during study) | See comment | See comment | Not estimable | 13 (1 cross‐over study) | See comment | Mild adverse effects included asthenia (1 participant), mood swings (1 participant), moon face (8 participants), increased body weight (12 participants) |
| Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) | See comment | See comment | Not estimable | 0 (0) | See comment | Not an outcome in this study |
| Economic outcomes | See comment | See comment | Not estimable | 0 (0) | See comment | Not an outcome in this study |
| *The basis for the assumed risk (e.g. the median control group risk across studies) is provided in footnotes. The corresponding risk (and its 95% CI) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). BETA: betamethasone; CI: confidence interval;ICARS: International Cooperative Ataxia Rating Scale; RR: risk ratio | ||||||
| GRADE Working Group grades of evidence High quality: Further research is very unlikely to change our confidence in the estimate of effect. Moderate quality: Further research is likely to have an important impact on our confidence in the estimate of effect and may change the estimate. Low quality: Further research is very likely to have an important impact on our confidence in the estimate of effect and is likely to change the estimate. Very low quality: We are very uncertain about the estimate. | ||||||
1Speech disorder was measured on a subjective, clinician‐derived severity rating scale.
2The CI was not calculable in Zannolli 2012 as the variance of change was not reported.