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

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机构: [1]The First Clinical College, Guangzhou University of Chinese Medicine,Guangzhou, China [2]Lingnan Medical Research Center of GuangzhouUniversity of Chinese Medicine, Guangzhou, China [3]The Second ClinicalCollege, Guangzhou University of Chinese Medicine, Guangzhou, China [4]School of Basic Medical Science, Guangzhou University of Chinese Medicine,Guangzhou, China [5]Department of Gastroenterology, The First AffiliatedHospital of Guangzhou University of Chinese Medicine, Guangzhou, China [6]Baiyun Hospital of the First Affiliated Hospital of Guangzhou University ofChinese Medicine, Guangzhou, China
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关键词: Modified Buzhong Yiqi decoction Myasthenia gravis N-of-1 trials Traditional Chinese medicine Gut microbiota

摘要:
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-gamma, TGF-beta) 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.

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出版当年[2021]版:
大类 | 4 区 医学
小类 | 4 区 医学:研究与实验
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大类 | 4 区 医学
小类 | 4 区 医学:研究与实验
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出版当年[2020]版:
Q4 MEDICINE, RESEARCH & EXPERIMENTAL
最新[2023]版:
Q3 MEDICINE, RESEARCH & EXPERIMENTAL

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第一作者机构: [1]The First Clinical College, Guangzhou University of Chinese Medicine,Guangzhou, China [2]Lingnan Medical Research Center of GuangzhouUniversity of Chinese Medicine, Guangzhou, China
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通讯机构: [2]Lingnan Medical Research Center of GuangzhouUniversity of Chinese Medicine, Guangzhou, China [5]Department of Gastroenterology, The First AffiliatedHospital of Guangzhou University of Chinese Medicine, Guangzhou, China [6]Baiyun Hospital of the First Affiliated Hospital of Guangzhou University ofChinese Medicine, Guangzhou, China
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