Abstract
Background Making comprehensive management of myasthenia gravis (MG) is a challenge in clinical practice due to heterogeneity and multiple comorbidities among patients. Aim To develop an end-to-end instrument for individualized assessment of MG in the perspective of Chinese medicine (TCM) with the application of multidisciplinary quantification approaches. Methods A self-administrated questionnaire was developed integrating typical symptoms of MG and spleen-kidney deficiency syndrome on basis of the conceptual framework of TCM. With data collected in a multicenter cross-sectional study, confirmatory factor analysis together with multidimensional item response theory (MIRT) was used for evaluating the psychometric property of the questionnaire. A computerized adaptive test was developed based on the MIRT model, and scores of syndrome factors were calculated in simulation. A logistics regression model was also estimated for evaluating the consistency between the quantitative result and the clinical diagnosis of syndrome from clinical practitioners. Result With 337 patients enrolled and assessed, the 14-item questionnaire was evaluated to be with adequate validity and reliability (Cronbach's alpha indices = 0.87, AIC = 195.827, BIC = 348.631, CFI = 0.921, RMR = 0.006, GFI = 0.954, RMSEA = 0.048, and χ2/df = 1.782). With adequate factor loadings of symptoms on related syndrome factor, the instrument was evaluated with preliminary interpretation and was suitable for evaluating patients with moderate severity of the spleen and kidney deficiency syndrome. Conclusion Setting typical symptoms of MG together with systemic discomforts in a computerized adaptive test on the basis of MIRT, this study proposed an innovative research paradigm for quantifying individual condition in the perspective of TCM with application of interdisciplinary approaches.
Highlights
Making comprehensive management of myasthenia gravis (MG) is a challenge in clinical practice due to heterogeneity and multiple comorbidities among patients
Restrictive respiratory failure caused by severe weakness of respiratory muscle could even lead to emergency known as MG crisis in 15% of MG patients [2]. e heterogeneity of clinical appearances of MG ranging from mild ocular deficits to severe widespread weakness posed a challenge for clinical assessment of MG patients [3]
With the purpose of quantifying abstract concepts in the perspective of Chinese medicine (TCM) theory, many innovative research paradigms were proposed with application of interdisciplinary methods including structural equation modeling (SEM) and multidimensional item response theory (MIRT) [21, 22]
Summary
As an autoimmune neuromuscular disease, myasthenia gravis (MG) was reported to be mediated by autoantibodies targeting components of the neuromuscular junction [1]. Ese instruments served as practical tools for the management of MG offering quantitative scores as references for clinical diagnosis and decision of treatment Shortcomings of these scales are obvious among which the lengthy setting of scalesalways resulted in reduction of compliance of patients during assessment. With the purpose of quantifying abstract concepts in TCM theory, many innovative research paradigms were proposed with application of interdisciplinary methods including structural equation modeling (SEM) and multidimensional item response theory (MIRT) [21, 22] In these studies, mathematical models were estimated bridging the gap between observable symptoms or signs and syndromes which were regarded as the latent trait of patients.In this way, individual condition could be quantified within a interpretable conceptual framework. Aiming at providing a flexible approach to support clinical management of MG patients in the perspective of TCM, this article proposed an innovative strategy for quantifying TCM syndrome of MG with development of a computerized adaptive test (CAT) on the basis of MIRT
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