Summary of findings 11. Erythropoietin versus placebo for speech disorder in Friedreich ataxia and other hereditary ataxias.
| Recombinant human erythropoietin (rhuEPO) for speech disorder in Friedreich ataxia and other hereditary ataxias | ||||||
| Patient or population: people with speech disorder in Friedreich ataxia and other hereditary ataxias Settings: hospital Intervention: rhuEPO | ||||||
| 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 | rhuEPO | |||||
| 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 SARA. Scale from: 0 to 48. Follow‐up: mean 6 months | See comment | See comment | Not calculable | 16 (1 study) | ⊕⊕⊝⊝ low1,2 | Speech subscales were not reported separately to overall SARA scores. No difference between groups |
| 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 SF‐36. Scale from 0 to 100. Higher scores indicate less disability. Follow‐up: mean 6 months | See comment | See comment | Not calculable | 16 (1 study) | ⊕⊕⊝⊝ low3,4 | No difference between groups |
| Adverse effects (during study) | See comment | See comment | Not estimable | 16 (1 study) | See comment | No serious adverse events. 3 participants had sideropenic anaemia (2 rhuEPO group and 1 placebo group) |
| 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). CI: confidence interval; rhuEPO: recombinant human erythropoietin; RR: risk ratio; SARA: Scale for the Assessment and Rating of Ataxia; SF‐36: Short Form 36 Health Survey | ||||||
| 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 was not measured directly but as part of an overall clinical severity scale. 2Speech was measured on a subjective, clinician‐rated, scale.
3Unclear randomisation, allocation concealment and blinding. 4Magnitude of change is not reported.