Effectiveness and safety of treatments for degenerative ataxias: A systematic review

  title={Effectiveness and safety of treatments for degenerative ataxias: A systematic review},
  author={M. M. Trujillo-Mart{\'i}n and Pedro Serrano-Aguilar and Fernando Mont{\'o}n-{\'A}lvarez and Romen Carrillo-Fumero},
  journal={Movement Disorders},
The aim of this study was to determine the effectiveness and safety of available treatment alternatives for degenerative ataxias (DA). We systematically reviewed studies that assess pharmacological, rehabilitative, or psychological treatments in patients with DA. Studies were included if they fulfilled prespecified criteria. All included clinical trials were scored for methodological quality. Main outcome measures were clinical status of neurological disorder, adverse events, and patient‐based… 
Exercise and Physical Therapy Interventions for Children with Ataxia: A Systematic Review
The studies investigating the effectiveness of exercise and physical therapy interventions for children with ataxia reported promising results but were of low methodological quality (no RCTs), used small sample sizes and were heterogeneous in terms of interventions, participants and outcomes.
Placebo response in degenerative cerebellar ataxias: a descriptive review of randomized, placebo-controlled trials
The placebo response in patients with CAs on various objective ataxia scales is demonstrated and it is emphasized that the placebo response should be considered when designing, analyzing, and interpreting clinical trials and in clinical practice in CA patients.
Appropriateness of Treatments for Patients With Degenerative Ataxias: Recommendations by a Panel of Experts
Within the limits of expert opinion, these guidelines provide direction for some common clinical uncertainties in the treatment of degenerative ataxias.
The assessment and treatment of postural disorders in cerebellar ataxia: a systematic review.
Therapeutic Interventions in the Primary Hereditary Ataxias
Treatment of the core clinical feature of these diseases—ataxia—is predominantly rehabilitative, and the value of good physical therapy far exceeds any potential benefit from medications that a physician might prescribe to improve balance and coordination.
Investigating the Clinical Significance and Research Discrepancies of Balance Training in Degenerative Cerebellar Disease
Most studies showed statistical and clinically significant ataxia severity improvements in subjects who performed balance training, and the amount of balance challenge and frequency of training were important factors in determining the extent of training benefit.
General Management of Cerebellar Disorders: An Overview
  • D. Timmann
  • Psychology, Biology
    Handbook of the Cerebellum and Cerebellar Disorders
  • 2020
This chapter reviews the state of the art in medical intervention and rehabilitation, focusing on presenting new results on motor rehabilitation in cerebellar disease, and current studies in the area of motor learning – in combination with modern imaging techniques – in cere Bellar disease are described.
Home Aerobic Training for Cerebellar Degenerative Diseases: a Randomized Controlled Trial
Balance training has shown some benefits in cerebellar ataxia whereas the effects of aerobic training are relatively unknown. To determine whether a phase III trial comparing home aerobic to balance
Physical activity and exercise interventions for people with rare neurological disorders: a scoping review of systematic reviews
Novel approaches to implementing common interventions and modalities are needed to increase accessibility and engagement in physical activity irrespective of disease type to achieve consensus on outcome measures that reflect areas of importance and relevance to people with RNCs.
Phase I randomized single-blinded controlled study investigating the potential benefit of aerobic exercise in degenerative cerebellar disease
Improvements in ataxia, balance, and gait are promising in people with cerebellar degeneration after aerobic training, and the treatment was considered technically feasible if participants achieved target training frequency, duration, and intensity.


Double-Blind, Crossover, Placebo-Controlled Clinical Trial with L-Acetylcarnitine in Patients with Degenerative Cerebellar Ataxia
Whether treatment with L-acetylcarnitine may improve some clinical conditions of patients with ataxia and a statistically significant improvement of some symptoms and a slow progression of the disease in both groups of patients is observed.
Double-blind crossover study with levorotatory form of hydroxytryptophan in patients with degenerative cerebellar diseases.
Long-term treatment with a high dose of the levorotatory form of hydroxytryptophan does not improve the conditions of patients with ataxia and had no significant effect on cerebellar symptoms.
How is disease progress in Friedreich’s ataxia best measured? A study of four rating scales
The Friedreich Ataxia Rating Scale (FARS) is the best to use in clinical trials of FRDA based on effect size, and power calculations that show that fewer participants are required to demonstrate the same effect of an intervention.
Eosinophilia-myalgia syndrome: selective cognitive impairment, longitudinal effects, and neuroimaging findings
The neurocognitive and neuroimaging findings contribute to the evidence which indicates that the neural substrate of EMS is white matter damage.
Antioxidant treatment of patients with Friedreich ataxia: four-year follow-up.
This therapy resulted in sustained improvement in mitochondrial energy synthesis that was associated with a slowing of the progression of certain clinical features and a significant improvement in cardiac function.
Levorotatory form of 5-hydroxytryptophan in Friedreich's ataxia. Results of a double-blind drug-placebo cooperative study.
It is demonstrated that the levorotatory form of 5-hydroxytryptophan is able to modify significantly the cerebellar symptoms in patients with Friedreich's ataxia, however, the effect is only partial and not clinically major.
Friedreich's ataxia: idebenone treatment in early stage patients.
Idebenone treatment at early stages of the disease seems to reduce the progression of cerebellar manifestations and further blind trials with a greater number of patients and higher doses are needed to fully assess the therapeutic potential of idebenone in Friedreich's ataxia.
Neurocognitive dysfunction in the eosinophilia‐myalgia syndrome
The hypothesis that the eosinophilia-myalgia syndrome is associated with an encephalopathy in addition to its previously recognized peripheral neuropathy and other rheumatologic manifestations is supported.
Double‐blind, triple‐crossover trial of low doses of oral physostigmine in inherited ataxias
A randomized, double-blind, triple-crossover trial of physostigmine and inert placebo taken orally in low doses for four sequential 3-month periods found the drug was more effective than the placebo.