The Preparation And Preliminary Evaluation Of The Clinical Outcome Evaluation Scale Based On The Report Of Patients With Motor Neuron Disease

BACKGROUNDPeople think much of the function of Clinician-Reported outcomes,Physiological outcomes and Caregiver-Reported outcomes in Patient Outcomes Assessment Sources in the past.But with the change of medicine pattern,the importance of Patient-reported Outcomes(PRO)is waken up to people gradually.Scale is used to assess the items of PRO,which is accepted by international medical experts and it is applicable extensively.PRO instruments provide patients’ information for doctors,which is not found by chemical and physical methods.The development and assessment about PRO instruments are accounted in the Guidance for Industry Patient-reported Outcome Measure:Use in Medical Product Development to support Labeling Claims drafted by U.S.Food and Drug Administration.It is an important problem for us to develop Chinese characteristic PRO instruments.The etiology and pathogenesis of motor neuron disease has not been studied clearly,so as the targeted treatment,and motor neuron disease patients suffer from long-term disease,live better and more comfortable is the pursuit of the vast majority of MND patients.However,the current evaluation of motor neuron disease commonly used tools are mainly introduced abroad,and the lack of awareness of the patient’s content,so the preparation of suitable for motor neuron disease in this particular group of patient-based clinical measurement tool is of great significance.ObjectiveTo develop and assess the "self-evaluating instrument based on Patient-Reported Outcomes for motor neuron disease patients"ThinkingAccording to the research target of PRO,researchers defined the concepts what to measure and operationalized the conception of motor neuron disease patients,reported outcomes.The indexes to be measured for the scale came into being.Then we optimized the indexes and developed the instrument’s conceptual framework.After amending the questions again and agin,we confirmed the item pool.With the heading and the direction accomplished,the research group developed the draft of the scale.Patients received investigation.With data collection,we analyzed the appropriate patients for the instrument and assessed the measurement.At last,researchers confirmed the "self-evaluating instrument based on Patient-Reported Outcomes for motor neuron disease patients".Methods1 Construct theoretical framework and Form Item-Bank:According to the Specific instructions of the PRO research issued by the US FDA,comply with the principle and requirement of Scale.Form a research team,based on literature and analysis of 192 clinical data,through group discussion,build a scientific rigorous theoretical framework of scale,and make clear definition of the dimensionality of scale.From 192 clinical cases about motor neuron disease in past to choose symptoms which frequency appears>5%and significantly changed after treatment,in conjunction with the patient interview,through group discussion and refining,confirmed the item-banks of PRO scale.2 Item selection:According to the built theoretical framework and measurable symptoms indexes to design experts questionnaire,consulting experts who have long-term clinical study in brain department by Delphi method.After the questionnaire,analyzing questionnaire through the mean,sum of grade score and coefficient of variation,and according to experts’approval ratings and advices,in conjunction with discussion of research team,combining,increasing or decreasing items.3 Form the draft scale:After items selection,the scale item-banks which are consisted of 4 dimensionality included 16 items(10 items of the core symptoms dimensionality,2 items of physical dimensionality,2 items of psychological dimensionality,2 items of social dimensionality)was formed.After the discussion of research group,adopt Likert-t to design score by five,identified foreword,developed derailed instruction,completed the format of scale,the draft scale formed.4 Clinical validation of the draft scale:From February 2017 to March 2017,we collected clinical dates from the Brain department of Yiling hospital and the out-patient of Dongzhimen hospital which affiliated to Beijing University of TCM for clinical validation of the draft scale.After that,developing the database,analyzing the dates by SPSS 20.0 software,studying the application of the crowd of the scale and evaluated the scale reliability,validity.Results1 Item-Bank selection:58 items were selected from literatures and clinical cases and patient interview,included 14 items of the core symptoms dimensionality,34 items of physical dimensionality,7 items of psychological dimensionality,3 items of social dimensionality.After group discussion,preliminarily determine 40 symptoms can be used for clinical efficacy evaluation about motor neuron disease.2 Items selection and optimization:Deleted items which the sum of grade score<85%,after expert questionnaire,the selection of experts on the importance of the entry and the operability of the higher evaluation of the items.The scale draft which consisted of 4 dimensionality include 16 items was formed.3 Clinical validation,of the draft scale:we examined the reliability,validity and responsibility of the scale with the authentic data,20 cases.The statistical results show that the split-half reliability coefficients were 0.647;and the Cronbach’s a coefficients were 0.801 respectively for the total scale.The results suggested that the scale has perfect internal consistency.Studied construct validity of the scale by confirmatory factor analysis,results showed the comparative for index(CFI)was 0.567,showed that internal structure of the scale can be fit in theoretical assumption,so the scale has good construct validity.ConclusionsIn this study,we introduced the method of PRO which were internationally recongnized into clinical study of motor neuron disease,and based on the theory of TCM,combined with the clinical characteristics of motor neuron disease,formulated motor neuron disease Pro Scale.The scale has good reliability,validity,and feasibility,has ability to show changes.of self-conscious symptoms of motor neuron disease truly.This will enrich and perfect the methods of clinical efficacy evaluation of motor neuron disease appraisal,and provide reference for evaluation of efficiency of TCM research about the rare disease.