Cochrane · systematic review · CD008953
Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes
Vogel, AP, Folker, J, Poole, ML · Cochrane Database of Systematic Reviews · 2014 · Cochrane Library
NeuromuscularAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
76
current version
GRADE rated
14
No studies
0
Included studies
NR
No studies 0 · 0%Ungraded 62 · 81.6%Very low 7 · 9.2%Low 5 · 6.6%Moderate 2 · 2.6%High 0 · 0%
Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.
Questions and results
Hydroxytryptophan versus placebo for speech disorder resulting from hereditary ataxias
Population: people with speech disorder resulting from hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (within 1 month) percentage change (improvement) in overall speech production Hydroxytryptophan vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production Wessel 1995: Mean syllable duration during rapid‐syllable repetition task. Scale from: 1 to 500. Shorter durations are better. Hydroxytryptophan vs placebo | no estimate extracted | 4 participants | Very low Downgraded: risk of bias |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments Hydroxytryptophan vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores Hydroxytryptophan vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during the study) Hydroxytryptophan vs placebo | no estimate extracted | 2 studies · 65 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example demands on caregivers, frequency of tests and restrictions on lifestyle) Hydroxytryptophan vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Hydroxytryptophan vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Thyrotropin‐releasing hormone tartrate versus placebo for speech disorder resulting from hereditary ataxia
Population: people with speech disorder resulting from hereditary ataxia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (1 week) change in isolated movement, objective and subjective measures of speech production Inherited Ataxias Clinical Rating Scale (IACRS) Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 2 studies · 245 participants | Very low |
Short‐term (1 week) change in quality of life scores related to communication as measured by validated communication assessments Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 2 studies · 320 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example demands on caregivers, frequency of tests, restrictions on lifestyle) Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Thyrotropin‐releasing hormone tartrate vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Varenicline versus placebo for speech disorder resulting from hereditary ataxia
Population: people with speech disorder resulting from hereditary ataxia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Varenicline vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (1 week) change in isolated movement, objective and subjective measures of speech production SARA. Scale from: 0 to 6. Higher scores indicate more severe speech disorder. Varenicline vs placebo | no estimate extracted | 1 study · 13 participants | Very low Downgraded: imprecision |
Short‐term (1 week) change in quality of life scores related to communication as measured by validated communication assessments Varenicline vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores SF‐36 Varenicline vs placebo | no estimate extracted | 1 study · 13 participants | Very low Downgraded: imprecision |
Adverse effects (during study) Varenicline vs placebo | no estimate extracted | 1 study · 18 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Varenicline vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Varenicline vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Riluzole versus placebo for speech disorder resulting from hereditary ataxias
Population: people with speech disorder resulting from hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Riluzole vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
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 speech disorder. Riluzole vs placebo | no estimate extracted | 1 study · 38 participants | Low |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments Riluzole vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores Riluzole vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Riluzole vs placebo | no estimate extracted | 1 study · 38 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Riluzole vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Riluzole vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Idebenone versus placebo for speech disorder resulting from hereditary ataxias
Population: people with speech disorder resulting from hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Idebenone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production ICARS, FARS Idebenone vs placebo | no estimate extracted | 2 studies · 117 participants | Very low |
Longer‐term (minimum 1 month) change in generic quality of life scores Idebenone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Idebenone vs placebo | no estimate extracted | 2 studies · 118 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Idebenone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Idebenone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Physiotherapy and occupational therapy versus placebo for speech disorder resulting from hereditary ataxias
Population: people with speech disorder resulting from hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Physiotherapy and occupational therapy vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (1 week) change in isolated movement, objective and subjective measures of speech production SARA. Scale from: 0 to 6. Higher scores indicate more severe speech disorder. Physiotherapy and occupational therapy vs placebo | no estimate extracted | 1 study · 42 participants | Very low |
Short‐term (1 week) change in quality of life scores related to communication as measured by validated communication assessments Functional Independence Measure (total). Scale from: 0 to 126. Higher scores indicate less disability Physiotherapy and occupational therapy vs placebo | no estimate extracted | 1 study · 42 participants | Very low |
Longer‐term (minimum 1 month) change in generic quality of life scores Physiotherapy and occupational therapy vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Physiotherapy and occupational therapy vs placebo | no estimate extracted | 1 study · 42 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Physiotherapy and occupational therapy vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Physiotherapy and occupational therapy vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Betamethasone versus placebo for speech disorder resulting from hereditary ataxias
Population: people with speech disorder resulting from hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Betamethasone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
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. Betamethasone vs placebo | no estimate extracted | 13 participants | Moderate |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments Betamethasone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores Child Health Questionnaires Betamethasone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Betamethasone vs placebo | no estimate extracted | 13 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Betamethasone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Betamethasone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
High‐dose versus low‐dose coenzyme Q10 and vitamin E for speech disorder resulting from hereditary ataxia
Population: people with speech disorder resulting from hereditary ataxia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production Cooper 2008 reported 3 speech measures: ICARS speech subscale. Scale from: 0 to 8. Higher scores indicate greater clinical severity Syl... High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 1 study · 43 participants | Low |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 1 study · 50 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes High‐dose vs low‐dose coenzyme Q10 and vitamin E | no estimate extracted | 0 studies · 0 participants | Ungraded |
Buspirone versus placebo for speech disorder in Friedreich ataxia and other hereditary ataxias
Population: people with speech disorder in Friedreich ataxia and other hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Buspirone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production ICARS. Buspirone vs placebo | no estimate extracted | 19 participants | Low |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments Buspirone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores Buspirone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Adverse effects (during study) Buspirone vs placebo | no estimate extracted | 19 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Buspirone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Buspirone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
α‐tocopheryl quinone versus placebo for speech disorder resulting from hereditary ataxia
Population: people with speech disorder resulting from hereditary ataxia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production α‐tocopheryl quinone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production α‐tocopheryl quinone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments α‐tocopheryl quinone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores SF‐36 scale from: 0 to 100. Higher score indicate less disability. α‐tocopheryl quinone vs placebo | no estimate extracted | 1 study · 19 participants | Moderate |
Adverse effects (during study) α‐tocopheryl quinone vs placebo | no estimate extracted | 1 study · 31 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) α‐tocopheryl quinone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes α‐tocopheryl quinone vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Erythropoietin versus placebo for speech disorder in Friedreich ataxia and other hereditary ataxias
Population: people with speech disorder in Friedreich ataxia and other hereditary ataxias
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Short‐term (1 week) percentage change (improvement) in overall speech production Erythropoietin vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production SARA. Scale from: 0 to 48. Erythropoietin vs placebo | no estimate extracted | 1 study · 16 participants | Low |
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments Erythropoietin vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Longer‐term (minimum 1 month) change in generic quality of life scores SF‐36. Scale from 0 to 100. Higher scores indicate less disability. Erythropoietin vs placebo | no estimate extracted | 1 study · 16 participants | Low Downgraded: risk of bias |
Adverse effects (during study) Erythropoietin vs placebo | no estimate extracted | 1 study · 16 participants | Ungraded |
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) Erythropoietin vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |
Economic outcomes Erythropoietin vs placebo | no estimate extracted | 0 studies · 0 participants | Ungraded |