Measuring Clinical Treatment Response in Myasthenia Gravis

Abstract

In this article we provide an overview of health-related outcome measurement-to better understand what different outcomes used in myasthenia actually measure-and to provide some guidance when choosing measures based on the clinical context and question. In myasthenia, the most commonly used outcome measures are aimed at assessing the signs and symptoms. In this review, we provide a summary of the most commonly used outcome measures. We discuss instruments that gauge disease overall health impact, such as on disability and quality of life. Finally, we discuss other relevant outcomes such as steroid-sparing effects and the role of surrogate markers.

Keywords: Disability; Minimal important difference; Myasthenia gravis; Outcome measurement; Responsiveness.