Minimal clinically important difference in myasthenia gravis: outcomes from a randomized trial.

Abstract:

INTRODUCTION:The minimal clinically important difference (MCID) is the smallest outcome change that has clinical significance. Its use has not been established in the study of myasthenia gravis (MG). METHODS:Patients from a published intravenous immunoglobulin (IVIg) vs. placebo study were studied. One anchor-based and 3 distribution-based techniques were used to identify quantitative myasthenia gravis score (QMGS), repetitive nerve stimulation (RNS), and single-fiber electromyography (SFEMG) MCID cut-offs. Patients with a change-score exceeding MCID cut-offs were compared. RESULTS:MCID cut-offs were below a QMGS change of 3.0. Anchor-based and 1 × SEM cut-offs showed 58.3% vs. 30.7% responders (P = 0.017), ½ SD 54.2% vs. 19.2% responders (P = 0.018), and effect size 0.519 vs. 0.164 (P = 0.011) in IVIg vs. placebo. Anchor-based (P = 0.73) and effect-size (P = 0.41) MCID cut-offs did not show a difference between IVIg and placebo. MCID methods did not produce meaningful RNS cut-offs. CONCLUSIONS:QMGS MCID values provide clinically relevant information and are recommended in MG trials. MCID analysis shows that improvement in MG patients treated with IVIg reflects clinically meaningful changes.

journal_name

Muscle Nerve

journal_title

Muscle & nerve

authors

Katzberg HD,Barnett C,Merkies IS,Bril V

doi

10.1002/mus.23988

subject

Has Abstract

pub_date

2014-05-01 00:00:00

pages

661-5

issue

5

eissn

0148-639X

issn

1097-4598

journal_volume

49

pub_type

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