Highlights
- •The Friedreich Ataxia Impact Scale (FAIS) is a patient reported outcome measure developed for FRDA.
- •The relationship between the FAIS and clinical characteristics of FRDA was assessed.
- •The responsiveness of the FAIS to change over one and two years was determined.
- •The FAIS is a valuable tool for measuring health status in FRDA.
- •The FAIS has limited responsiveness and its use in intervention studies is uncertain.
Abstract
Background
Quality of life in Friedreich ataxia (FRDA) has been explored using various generic
health status measurement tools, most commonly the Short Form Health Survey Version
2 (SF-36v2). The tool did not address many specific issues related to disease impact
in people with FRDA. The Friedreich Ataxia Impact Scale (FAIS) was developed to examine
clinically relevant areas in FRDA. The aims of the current study were to assess the
relationship between the FAIS and clinical characteristics of FRDA, as well as to
determine the responsiveness of the FAIS to change over one and two years.
Methods
One hundred and four individuals with FRDA, homozygous for the GAA expansion in intron
1 of FXN, completed the FAIS at baseline. Seventy individuals completed the FAIS again 12 months later and 49 completed the FAIS at 24 months. Clinical parameters and neurologic scales (Friedreich Ataxia Rating Scale
(FARS)) were also recorded.
Results
The total FARS score, onset age and disease duration correlated significantly with
FAIS subscales measuring symptoms and physical functioning. The physical and mental
summary measures of the SF-36 V2 also correlated well with the FAIS subscales. Speech
was the only subscale that demonstrated significant change over one and two years.
Conclusions
The FAIS provides valuable insight into the perspective of individuals with FRDA on
their health status, and is an important measure of morbidity. It has, however, limited
responsiveness to change and its use in intervention studies is questionable.
Keywords
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Article info
Publication history
Published online: March 25, 2015
Accepted:
March 13,
2015
Received in revised form:
February 16,
2015
Received:
November 18,
2014
Identification
Copyright
© 2015 Elsevier B.V. Published by Elsevier Inc. All rights reserved.
