James F Howard1, Miriam Freimer2, Fanny O'Brien3, Jing Jing Wang3, Stephen R Collins4, John T Kissel2. 1. Department of Neurology, University of North Carolina, 2200 Physicians Office Building, CB 7025, 170 Manning Drive, Chapel Hill, North Carolina, USA. 2. Department of Neurology, Ohio State University, Columbus, Ohio, USA. 3. Medical Science and Development Operations, Alexion Pharmaceuticals, New Haven, Connecticut, USA. 4. Connexion Healthcare, Newtown, Pennsylvania, USA.
Abstract
INTRODUCTION: A phase 2 study of eculizumab for treating myasthenia gravis (MG) used the quantitative myasthenia gravis score (QMG) and myasthenia gravis activities of daily living profile (MG-ADL) to evaluate baseline disease severity and treatment response. Correlations were then analyzed between these assessments. METHODS: Patients were given eculizumab or placebo during the first 16-week treatment period of the crossover study, with treatment assignments reversed for the second treatment period following a 5-week washout. QMG and MG-ADL scores at baseline and endpoint of each treatment period generated correlation coefficients for baseline status and treatment response during eculizumab therapy. RESULTS:Correlation strength between QMG and MG-ADL scores was higher for treatment response (R = 0.726; 95% confidence interval, 0.264-0.907; P = 0.0036) than for assessing baseline disease status (R = 0.552; 95% confidence interval, -0.022-0.839; P = 0.0495). CONCLUSIONS:MG-ADL may be more sensitive for assessing treatment response than point-in-time disease status. Muscle Nerve 56: 328-330, 2017.
RCT Entities:
INTRODUCTION: A phase 2 study of eculizumab for treating myasthenia gravis (MG) used the quantitative myasthenia gravis score (QMG) and myasthenia gravis activities of daily living profile (MG-ADL) to evaluate baseline disease severity and treatment response. Correlations were then analyzed between these assessments. METHODS:Patients were given eculizumab or placebo during the first 16-week treatment period of the crossover study, with treatment assignments reversed for the second treatment period following a 5-week washout. QMG and MG-ADL scores at baseline and endpoint of each treatment period generated correlation coefficients for baseline status and treatment response during eculizumab therapy. RESULTS: Correlation strength between QMG and MG-ADL scores was higher for treatment response (R = 0.726; 95% confidence interval, 0.264-0.907; P = 0.0036) than for assessing baseline disease status (R = 0.552; 95% confidence interval, -0.022-0.839; P = 0.0495). CONCLUSIONS:MG-ADL may be more sensitive for assessing treatment response than point-in-time disease status. Muscle Nerve 56: 328-330, 2017.
Authors: Michael Schroeter; Benjamin Berger; Franz Blaes; Tim Hagenacker; Sebastian Jander; Julia Kaiser; Petra Kalischewski; De-Hyung Lee; Tobias Ruck; Ulrike Schara; Peter Urban; Andreas Meisel Journal: J Cent Nerv Syst Dis Date: 2021-02-01
Authors: Malin Petersson; Amalia Feresiadou; Daniel Jons; Andreea Ilinca; Fredrik Lundin; Rune Johansson; Anna Budzianowska; Anna-Karin Roos; Viktor Kågström; Martin Gunnarsson; Peter Sundström; Fredrik Piehl; Susanna Brauner Journal: Neurology Date: 2021-08-10 Impact factor: 9.910