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Pour comprendre cette publication qui est un point d('information sur les essais Phase II :QMG and MG-ADL correlations: Study of eculizumab treatment of myasthenia gravis
James F. Howard Jr MD1,*, Miriam Freimer MD2, Fanny O'brien PhD3, Jing Jing Wang MD, MS3, Stephen R. Collins MS4, John T. Kissel MD2 andon behalf of the MG (Phase2) Study Group
Version of Record online: 25 FEB 2017
Alexion Pharmaceuticals provided funding for the original study and was responsible for the design and conduct of the study. Alexion Pharmaceuticals also provided funding for the preparation, review, and approval of the manuscript along with collection, management, analysis, and interpretation of the data.
- Conflicts of Interest:
J.F. Howard is a consultant to Alexion Pharmaceuticals for which he receives no remuneration. J.J. Wang and F. O'Brien are employees of Alexion Pharmaceuticals. Grant support was provided to the authors to conduct the clinical trial, and the authors received no financial compensation for their activities in the study. J.F. Howard, M. Freimer, J.J. Wang, and J.T. Kissel were responsible for the study design, analysis and interpretation of the study data, critical review of the manuscript, and approval of the final version for submission. F. O'Brien provided statistical analysis. S.R. Collins drafted and revised the work critically for important intellectual content.
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.
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.
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).
MG-ADL may be more sensitive for assessing treatment response than point-in-time disease status.
- l'eculizumab est connu sous le nom commercial SOLIRIS ...
- QMG (Quantitative Myasthenia Gravis) test est le nom du score myasthénique mis au point par MGFA (fondation américaine de la myasthenie)
http://myasthenia.org/LinkClick.aspx?fi ... &tabid=125
- MG-ADL (Myasthenia gravis activities of daily living) Profile est un questionnaire en 8 points pour évaluer le profil "MG" journalier
https://www.researchgate.net/publicatio ... ng_profile