Appropriateness of Treatments for Patients With Degenerative Ataxias: Recommendations by a Panel of Experts

Abstract

Background: The a vailable scientific evidence regarding treatment of degenerative ataxias is scarce. Appropriateness of therapy options for degenerative ataxias were evaluated by using the RAND/UCLA Method. Methods: After a systematic literature review, a list of clinical scenarios were developed to simulate situations most likely to arise in clinical practice. An 8-members expert panel rated, in a two rounds process, the appropriateness of each clinical scenario-treatment combination or indication. Analysis used the ratings to categorize each indication as appropriate, of uncertain appropriateness or inappropriate. Results: Final rankings for the indications were as follow: 26/154 (18.3%) appropriate, 36/154 (25.4%) uncertain and 92/154 (56.3%) inappropriate. The agreement rate was 66.2%. For patients with Friedreich ataxia, physostigmine, 5-hydroxytryptophan and amantadine were rated inappropriate while L -carnitine was rated appropriate only for asymptomatic patients or for patients with gait ataxia. Panelists recommended idebenone therapy for Friedreich ataxia complicated by cardiomyopathy . Therapy with 4-aminopyridine was rated inappropriate for episodic ataxia type 1 but it was rated appropriate for type 2. In the treatment of other ataxias, such as autosomal dominant ataxias and the autosomal recessive ataxias not Friedreich, physostigmine, acetazolamide and L - carnitine were rated inappropriate while amantadine was inappropriate only in patients without gait ataxia. All other combinations were considered uncertain. Conclusions: Within the limits of expert opinion, these guidelines provide direction for some common clinical uncertainties in the treatment of degenerative ataxias. J Neurol Res. 2012;2(4):152-158 doi: https://doi.org/10.4021/jnr122e