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Open Access 01-12-2022 | Myasthenia Gravis | Study protocol

Effectiveness of modified Buzhong Yiqi decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trials

Authors: Senhui Weng, Jinghao Li, Benshu Chen, Long He, Zhuotai Zhong, Linwen Huang, Shijing Zhang, Fengbin Liu, Qilong Jiang

Published in: Trials | Issue 1/2022

Abstract

Background

Myasthenia gravis (MG) is an acquired autoimmune disease with high heterogeneity. The disease is chronic, relapsing repeatedly and progressive with acute exacerbation occasionally. Although the treatment of MG has developed, it is still unsatisfactory and has some unexpected side effects. Traditional Chinese medicine (TCM) has shown great potential in MG treatment, including relief of muscle weakness syndrome, improvement of patient’s quality of life, and reduction of side effects of western medicine. The purpose of this study is to evaluate the effectiveness of modified Buzhong Yiqi decoction (MBYD) as an add-on therapy for MG through a small series of N-of-1 trials.

Methods

Single-centre, randomized, double-blind, 3 crossover N-of-1 trials will be conducted to enroll patients with MG diagnosed as spleen-stomach deficiency syndrome or spleen-kidney deficiency syndrome in TCM. Each N-of-1 trial has 3 cycles of two 4-week periods containing the MBYD period and placebo period. The wash-out interval of 1 week is prior to switching each period. Primary outcome: quantitative myasthenia gravis (QMG). Secondary outcomes: the following scales: myasthenia gravis composite (MGC), myasthenia gravis activities of daily living profile (MG-ADL), myasthenia gravis quality of life (MG-QOL); the level of CD4+FoxP3+Treg cells and cytokines (IL-4, IL-17A, INF-γ, TGF-β) in the peripheral blood; the alterations of the composition of gut microbiota; reduction of the side effects of western medicine.

Discussion

Used by WinBUGS software, we will conduct a hierarchical Bayesian statistical method to analyze the efficacy of MBYD in treating MG in individuals and populations. Some confounding variables such as TCM syndrome type and potential carryover effect of TCM will be introduced into the hierarchical Bayesian statistical method to improve the sensitivity and applicability of the trials, and the use of prior available information within the analysis may improve the sensitivity of the results of a series of N-of-1 trials, from both the individual and population level to study the efficacy of TCM syndrome differentiation. We assumed that this study would reveal that MBYD is effective for MG and provide robust evidence of the efficacy of TCM to treat MG.

Trial registration

Chinese Clinical Trial Register, ID: ChiCTR2000040477, registration on 29 November 2020.

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Title: Effectiveness of modified Buzhong Yiqi decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trials
Authors: Senhui Weng
Jinghao Li
Benshu Chen
Long He
Zhuotai Zhong
Linwen Huang
Shijing Zhang
Fengbin Liu
Qilong Jiang
Publication date: 01-12-2022
Publisher: BioMed Central
Keywords: Myasthenia Gravis
Respiratory Microbiota
Published in: Trials / Issue 1/2022
Electronic ISSN: 1745-6215
DOI: https://doi.org/10.1186/s13063-022-06287-9

2024 ASCO Annual Meeting

Springer Medicine

Effectiveness of modified Buzhong Yiqi decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trials

Abstract

Background

Methods

Discussion

Trial registration

2024 ASCO Annual Meeting

Springer Medicine

Abstract

Background

Methods

Discussion

Trial registration

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