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Open‐label pilot study of oral methylprednisolone for the treatment of patients with friedreich ataxia
Author(s) -
Patel Maya,
Schadt Kimberly,
McCormick Ashley,
Isaacs Charles,
Dong Yi Na,
Lynch David R.
Publication year - 2019
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26610
Subject(s) - ataxia , tolerability , ambulatory , medicine , methylprednisolone , physical therapy , open label , cohort , rating scale , ataxic gait , adverse effect , psychology , psychiatry , developmental psychology
In this study we assessed the effect of methylprednisolone on safety, tolerability, and ability in Friedreich ataxia (FRDA). Methods The study was an open‐label trial of pulse methylprednisolone on 11 participants with FRDA. All participants followed a 28‐day treatment cycle, repeated 7 times. Patients were assessed with the timed 25‐foot walk (T25FW), 1‐minute walk (1MW), the Friedreich Ataxia Rating Scale (FARS), and the 9‐hole peg test (9HPT). Efficacy was tested by comparing baseline and week 26 visits, separated into adult and pediatric groups. Results In comparisons of participants’ baseline and week 26 visits, only the pediatric cohort's 1MW score showed change ( P < 0.05). The T25FW, the primary outcome measure, did not change significantly. Discussion Pediatric participants improved their gait distance in the 1MW, but did not significantly improve in other measures in this overall negative study. Methylprednisolone was generally well tolerated, suggesting that it may be useful for ambulatory children with FRDA if benefit is found with further study.