Log In

Forgot Password?
Create New Account

Loading... please wait

Abstract Details

Trial Design and Impact on Study Outcome: Post-hoc Analysis of the BeatMG Study Based on RINOMAX Trial Criteria
Neuromuscular and Clinical Neurophysiology (EMG)
P4 - Poster Session 4 (11:45 AM-12:45 PM)
11-004
While BeatMG study showed no definitive benefit of rituximab, the RINOMAX trial demonstrated efficacy. Diverging conclusions have led to further questions as to the role of rituximab in the treatment paradigm for AChR-Ab+ MG. Some key differences in trial design between BeatMG and RINOMAX may have impacted the study findings.
To perform a post-hoc analysis of the B-cell Targeted Treatment in Myasthenia Gravis (BeatMG, NCT02110706) study with select inclusion-exclusion criteria as of the Rituximab in Patients with New-Onset Generalized MG (RINOMAX, NCT02950155) trial.
We applied select RINOMAX eligibility criteria, including baseline quantitative MG (QMG) score of ≥ 6, no other IST, baseline daily prednisone dose of ≤ 40 mg, and no history of thymectomy to the BeatMG cohort to reduce the influence of high dose of prednisone, concomitant IST use, and thymectomy. Both changes in raw numbers and percentage changes in the disease severity scores from baseline were considered. Intention to treat analysis was performed with the last observation carried forward.
Ten patients in the placebo arm (56.1 ± 16.1 years, five women) and ten patients in the rituximab arm (55.9 ± 16.4 years, four women) fulfilled the modified inclusion criteria for this analysis. Patients in the rituximab arm had more severe disease. Patients in the rituximab group showed significant changes in QMG scores from baseline [for raw values (p < 0.001) and percentage changes (p 0.02)]. Prednisone dose was successfully reduced in both placebo and rituximab groups. Rituximab was successful in lowering the AChR autoantibody titer.

Clinical trial design may impact the assessment of an intervention and should be considered when comparing studies. While this post-hoc analysis showed some trends of beneficial effect of rituximab in a subgroup of patients with AChR-Ab+ MG, it highlights the need to better understand the role of B cell depletion therapy in MG.

Authors/Disclosures
Bhaskar Roy, MD (Yale University)
PRESENTER
Dr. Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda Pharmaceuticals. Dr. Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Roy has stock in Cabaletta bio. . The institution of Dr. Roy has received research support from Martin Shubik Fund for IBM at Yale University. The institution of Dr. Roy has received research support from Abcuro Pharmaceuticals.
Richard J. Nowak, MD (Yale University School of Medicine) Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Viela Bio. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. The institution of Dr. Nowak has received research support from Ra Pharma. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Momenta . The institution of Dr. Nowak has received research support from Immunovant . Dr. Nowak has received research support from argenx. Dr. Nowak has received research support from Viela Bio . Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.