Pharmacological treatments for Friedreich ataxia.

What was studied

This Cochrane review looked at drugs for Friedreich ataxia, or FRDA. FRDA is a rare inherited nerve disease. It starts with unsteadiness in standing and walking. It slowly leads to wheelchair use. The review checked effects after 12 months of treatment. It included randomized and quasi-randomized trials. Vitamins counted as drugs. Outcomes were ataxia scores, heart wall thickness, daily living, hand skill and exercise tests. Adverse events were also checked.

What they found

7 studies gave data on the ataxia scale. They had 513 participants. Drugs probably made little or no difference after 12 months. The score difference was 0.02. Evidence was very uncertain for heart wall thickness and daily living. Drugs probably improved hand skill. Evidence was very uncertain for exercise testing and drug-related adverse events. Events leading to stopping the drug or death may differ little from placebo.

What it means for patients

The authors say drugs probably make little or no difference on the ataxia scale. They call the probable gain in hand skill unexpected. They say serious events may be no more common with treatment.

Why it is not certain yet

Certainty of evidence ranged from very low to moderate. The authors downgraded outcomes for imprecision, inconsistency and suspected publication bias. Only 104 participants were in the analysis of drug-related adverse events. The studies may not have found all rare and serious events. Some studies were controlled by drug companies.

Source

Pharmacological treatments for Friedreich ataxia.

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