Browsing by Author "Nowak, Richard J."

Now showing 1 - 2 of 2
  • Thumbnail Image
    Item
    Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study
    (Wolters Kluwer, 2022-01-25) Nowak, Richard J.; Coffey, Christopher S.; Goldstein, Jonathan M.; Dimachkie, Mazen M.; Benatar, Michael; Kissel, John T.; Wolfe, Gil I.; Burns, Ted M.; Freimer, Miriam L.; Nations, Sharon; Granit, Volkan; Smith, A. Gordon; Richman, David P.; Ciafaloni, Emma; Al-Lozi, Muhammad T.; Sams, Laura Ann; Quan, Dianna; Ubogu, Eroboghene; Pearson, Brenda; Sharma, Aditi; Yankey, Jon W.; Uribe, Liz; Shy, Michael; Amato, Anthony A.; Conwit, Robin; O'Connor, Kevin C.; Hafler, David A.; Cudkowicz, Merit E.; Barohn, Richard J.; NeuroNEXT NN103 BeatMG Study Team; Neurology, School of Medicine
    Objective: To determine whether rituximab is safe and potentially beneficial, warranting further investigation in an efficacy trial for acetylcholine receptor antibody-positive generalized MG (AChR-Ab+ gMG). Methods: The B-Cell Targeted Treatment in MG (BeatMG) study was a randomized, double-blind, placebo-controlled, multicenter phase-2 trial that utilized a futility design. Individuals 21-90 years of age, with AChR-Ab+ gMG (MG Foundation of America Class II-IV) and receiving prednisone ≥15 mg/day were eligible. The primary outcome was a measure of steroid-sparing effect, defined as the proportion achieving ≥75% reduction in mean daily prednisone dose in the 4-weeks prior to week 52 and with clinical improvement or no significant worsening as compared to the 4-week period prior to randomization. The co-primary outcome was safety. Secondary outcomes included MG-specific clinical assessments. Fifty-two individuals were randomized (1:1) to either a two-cycle rituximab/placebo regimen, with follow-up through 52-weeks. Results: Of the 52 participants included, mean (±SD) age at enrollment was 55.1 (±17.1) years; 23 (44.2%) were female, and 31 (59.6%) were MGFA Class II. The mean (±SD) baseline prednisone dose was 22.1 (±9.7) mg/day. The primary steroid-sparing outcome was achieved in 60% of those on rituximab vs. 56% on placebo. The study reached its futility endpoint (p=0.03) suggesting that the pre-defined clinically meaningful improvement of 30% due to rituximab over placebo was unlikely to be achieved in a subsequent, larger trial. No safety issues identified. Conclusions: While rituximab was safe and well-tolerated, these results suggest that there is a low probability of observing the defined clinically meaningful steroid-sparing effect over a 12-month period in a phase-3 trial of mild-moderately symptomatic AChR-Ab+ gMG. Classification of evidence: This study provides Class I evidence that for mild-to-moderate AChR-Ab+ gMG, compared with placebo, rituximab is safe but unlikely to reduce steroid use by an absolute difference of at least 30% at 1 year.
  • Thumbnail Image
    Item
    Telemedicine visits in myasthenia gravis: Expert guidance and the Myasthenia Gravis Core Exam (MG-CE)
    (Wiley, 2021) Guidon, Amanda C.; Muppidi, Srikanth; Nowak, Richard J.; Guptill, Jeffrey T.; Hehir, Michael K.; Ruzhansky, Katherine; Burton, Leeann B.; Post, David; Cutter, Gary; Conwit, Robin; Mejia, Nicte I.; Kaminski, Henry J.; Howard, James F., Jr.; Neurology, School of Medicine
    Introduction/aims: Telemedicine may be particularly well-suited for myasthenia gravis (MG) due to the disorder's need for specialized care, its hallmark fluctuating muscle weakness, and the potential for increased risk of virus exposure among patients with MG during the coronavirus disease 2019 (COVID-19) pandemic during in-person clinical visits. A disease-specific telemedicine physical examination to reflect myasthenic weakness does not currently exist. Methods: This paper outlines step-by-step guidance on the fundamentals of a telemedicine assessment for MG. The Myasthenia Gravis Core Exam (MG-CE) is introduced as a MG-specific, telemedicine, physical examination, which contains eight components (ptosis, diplopia, facial strength, bulbar strength, dysarthria, single breath count, arm strength, and sit to stand) and takes approximately 10 minutes to complete. Results: Pre-visit preparation, remote ascertainment of patient-reported outcome scales and visit documentation are also addressed. Discussion: Additional knowledge gaps in telemedicine specific to MG care are identified for future investigation.