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

Maria M. Trujillo-Martin, Pedro Serrano-Aguilar, Fernando Monton-Alvarez, Juan Manuel Ramos-Goni

Abstract


Background: The available 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.




doi: http://dx.doi.org/10.4021/jnr122e


Keywords


Rare diseases; Degenerative ataxia; Treatment effectiveness; Consensus; RAND method; Appropriateness

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Journal of Neurology Research, bimonthly, ISSN 1923-2845 (print), 1923-2853 (online), published by Elmer Press Inc.     
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