| Objective:To explore the adaptability of the Revised ALS functional rating scale(ALSFRS-R)in the Chinese population;verify the feasibility and application of the Patient-reported Outcomes of Motor Neuron Disease(MND-PRO)in the Chinese population based on the item response theory(IRT);explore the relationship between neurofilament light chain(NfL)level,Traditional Chinese Medicine syndrome differentiation and disease evolution in patients with motor neuron disease(MND)based on the MND-PRO scaleMethods:Collected 61 domestic MND patients’ ALSFRS-R,used the propensity score matching to match the foregin MND patients from the Pooled Resource Open-Access ALS Clinical Trials(PRO-ACT),and then used the IRT to compare these two groups’ performance on the ALSFRS-R to analyze the differences between the Caucasian population and Chinese population,to verify whether the ALSFRS-R is suitable for Chinese culture,language and lifestyle background.Used the IRT to quantitatively analyze the items and options of the MND-PRO scale which debeloped by the team,and further collected the ALSFRS-R of 79 MND patients to verify the effectiveness of the MND-PRO scale and the severity of the disease evaluation ability.Based on the neurofibrillary light chain(NfL)of 32 MND patients and the information of the diagnosis of TCM syndrome,used the Different Item Function to explre the different subgroup’s evolution model and the application in the clinical practice.Results:The propensity score was successfully 1:1 matched with 58 patients on the motor neuron disease registration platform and 58 patients on the PRO-ACT platform(based on patient’s age,gender and the location of onset),and the 12 items of the ALSFRS-R scale of the Chinese population were found through the IRT.The discrimination is(12.71,1.27,2.63,0.86,1.42,3.68,6.84,1.65,0.96,2.64,0.81,3.56),and the item difficulty is(6.50,1.30,5.24,0.72,0.39,3.37,2.20,0.72,0.47,1.66,1.27,3.03);the item distinction of the 12 items of the ALSFRS-R scale of the Caucasian population on the PRO-ACT platform is(2,2.29,9.21,0.41,0.32,2.22,7,1.47,0.57,1.65,3.03,1.01),the difficulty of the item is(3.13,3.05,9.60,2.36,0.85,1.34,11.60,0.29,1.12,2.51,1.68,1.88),it is found that the differences between the medullary dimension and respiratory dimension of Chinese,Caucasian populations,the DIF analysis found that the Chinese and Caucasian population in the "item 4 writing","item 6 wash" and"item 12 respiratory insufficiency" have significant differences(0.001,0.003,0.002),indicating ALSFRS-R should be adjusted according to Chinese background.Quantitative analysis of the MND-PRO scale included 79 Chinese MND patients.The IRT found that the 20 items of the MND-PRO scale had the item discrimination of(3.61,3.71,1.33,3.75,-0.06,1.17,3.06,0.45,0.55,0.4,1.21,0.97,0.94,0.22,3,0.75,1.88,2.95,0.42,0.99),the item difficulty is(0.24,1.05,-3.04,-1.24,0.64,-0.11,1.27,-1.58,-1.23,-1.48,-2.10,-1.09,-1.69,-1.87,4.80,-2.59,-0.69,-2.46,-0.87,-2.66).The analysis showed that the Pearson correlation between the MND-PRO scale and ALSFRS-R is-0.69,indicating that the MND-PRO scale is feasible and has the ability to predict and evaluate disease.32 patients with serum collected according to the median NfL(58.32 pg/ml)grouping,the DIF of the MND-PRO scale is shown in the MH test P of "item8 speech","item9 drinking choking","itemlO dysphagia","iteml6 treatment craving" were(0.00001,0.00006,0.00002,0.00003).Used the DIF to analyze patients with different TCM syndromes,there is no difference between patients with syndromes of excessive and deficiency in the early stage,but as the severity of the disease increases,the rate of disease progression of patients with excessive syndromes is faster than deficiency syndromes;The function of patients with wind is not obvious in the early stage of the disease,but with the progress of the severity of the disease,the disease of the yin deficiency is more advanced and lost more function in the later stage of the disease.Conclusions:This research found that in the application of the ALSFRS-R scale to the Chinese population,DIF performance between the Caucasian populations were observed in the sports dimension,and the Chinese population had their own disease characteristics and description habits in the medullary dimension and respiratory dimension.Therefore,ALSFRS-R is not well applicable to the Chinese population,and the scale needs to be further adjusted and scientifically evaluated,or a new evaluation scale suitable for Chinese MND patients needs to be developed.This research further explored the applicability of the MND-PRO developed by our team among Chinese patients with MND.Quantitative analysis based on theIRT proved that the MND-PRO scale model is set up reasonably.Except for "item 5 muscle beat",the remaining items can provide appropriate information on the severity of different disease stage,and in different ages,genders,and onset also showed good consistency;and the strong correlation between the MND-PRO scale and the ALSFRS-R also proved the credibility of MND-PRO.These results together indicate that MND-PRO can be used as an evaluation tool in clinical practice and scientific research of MND,especially for the Chinese population.This study also confirmed that patients with different NfL expressions have different evolution model on the MND-PRO scale,and are not only related to neurological deficits,but also related to psychology and social states.It also proved that the scale can reflect the prognosis of the disease.There are differences in disease evolution in MND patients with different TCM syndrome types.The syndrome differentiation between deficiency and excessive and Yin-Yang syndrome in internal wind have different disease manifestations,evolution model,and prognosis.This research provided the basis and showed an important basis for optimizing clinical intervention programs and improving the effect of syndrome differentiation and treatment for patients with different syndrome characteristics in the future. |
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