Publications by authors named "M Pasnoor"
Article Synopsis
- The study aimed to assess the safety and effectiveness of caprylate/chromatography-purified intravenous immunoglobulin (IGIV-C) in patients with generalized myasthenia gravis (MG) compared to a placebo.
- A total of 62 patients participated in a randomized trial, where IGIV-C showed a numerical improvement in certain outcomes like activities of daily living, although most primary and secondary outcomes did not reach statistical significance.
- While IGIV-C was generally well-tolerated, the small size of the study suggests the need for further research to better understand its potential as a maintenance therapy for MG.
Amyotrophic lateral sclerosis (ALS) patients lack effective treatments to maintain motor and neuromuscular function. This study aimed to evaluate the effect of a home-based exercise program on muscle strength, ALS scores, and transcriptome in ALS patients, Clinical Trials.gov #NCT03201991 (28/06/2017).
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- * Most MG patients have acetylcholine receptor antibodies, but other notable antibodies like MuSK and LRP4 have also been identified, with some being available for testing.
- * The Myasthenia Gravis Foundation of America (MGFA) has established a disease classification system, and a range of therapies for managing MG are being researched and developed, guided by international recommendations.
Article Synopsis
- The ADAPT+ study was conducted to evaluate the long-term safety and effectiveness of the drug efgartigimod in adults with generalized myasthenia gravis (gMG) over a period of up to 3 years.
- The study involved 151 participants from the earlier phase 3 ADAPT study, with a significant portion (76.6%) being AChR-Ab positive, and it measured adverse events as well as improvements in disease severity using specific scoring systems (MG-ADL and QMG).
- Results showed that 84.8% of participants experienced at least one side effect, with headache and COVID-19 being the most common, while many participants achieved clinically meaningful improvements in their symptoms shortly after
Background: Response adaptive randomization is popular in adaptive trial designs, but the literature detailing its execution is lacking. These designs are desirable for patients/stakeholders, particularly in comparative effectiveness research, due to the potential benefits including improving participant buy-in by providing more participants with better treatment during the trial. Frequentist approaches have often been used, but adaptive designs naturally fit the Bayesian methodology; it was developed to deal with data as they come in by updating prior information.
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