【作者】 高凡珠；

【导师】 王永炎； 谢雁鸣；

【作者基本信息】 中国中医科学院 ， 中医临床基础， 2010， 博士

【摘要】 疗效评价是临床评价的主体,对临床疗效的科学诠释是中医药学生存和发展的重要科学问题。中医诊疗过程中,复杂干预贯穿始终,具有辨证论治时空性、治疗手段多样化及干预目的多维度的特点。但是,既往中医临床评价中,多注重单一治疗方法(方案)对临床结局指标的单维度疗效评价,缺乏对中医复杂干预的多维度结局测量,从而降低了中医优势的发挥,降低了实际临床疗效。中医疗效评价应回归临床诊疗真实世界,强调中医复杂干预对患病生命体的整合调节作用,重视临床结局的综合评价。数据包络分析(DEA)是运筹学、管理学和数理经济学交叉研究的一个新领域,用于评价具有相同类型投入和产出部门(决策单元)相对效率的多指标综合评价方法。它在处理多投入、尤其是多产出问题上较其它综合评价方法具有绝对优势,适用于中医复杂干预(多投入)和多指标综合评价(多产出)的特点。本研究在国家863计划的支持依托下,以缺血性中风早期康复为例,首次将DEA应用于中医复杂干预的多指标综合评价中,探索DEA应用于中医疗效评价的可行性和适用性。1中医综合方案的单指标疗效评价研究对象来源于自2008年4月至2009年4月北京中医药大学附属东直门医院、首都医科大学附属北京天坛医院、天津中医药大学第二附属医院、首都医科大学附属安贞医院、河南中医学院附属医院、山东中医药大学第二附属医院、邢台市人民医院、长春中医药大学附属医院及广州中医药大学第二附属医院等9个临床研究中心的早期缺血性中风住院病人342例,按照2：1的比例经中央随机系统分为中医综合治疗组233例,西医对照组109例。中医综合治疗组在内科基础治疗基础上,根据临床实际情况给予中药汤药、中药注射剂、针灸推拿及现代康复治疗；西医对照组在内科基础治疗基础上,给予现代康复治疗。研究中医综合方案对早期缺血性中风神经功能缺损、肢体运动功能、日常生活活动能力、残疾及中医症状积分改善的影响。结果表明：①神经功能缺损(美国国立卫生研究院卒中量表,National Institute of Health Stroke Scale, NIHSS)方面,治疗7天后两组NIHSS积分均明显下降,其中中医综合治疗组改善更显著,两组比较有统计学差异(P<0.05)；至第14天和第21天,两组积分均继续下降,仍以中医综合治疗组改善更显著(P<0.05)。②肢体运动功能(简式Fugl-Meyer评测法,Fugl-Meyer Assessment, FMA)方面,治疗7天后两组FMA积分均明显增高,两组间无显著差异(P>0.05)；至第14天和第21天,两组FMA积分继续增高,两组间仍无显著差异(P>0.05)。③日常生活活动能力(Barthel指数法,Barthel Index, BI)方面,在治疗21天,两组日常生活活动能力BI均明显增高；其中,中医综合治疗组较西医对照组改善更明显,两组比较有统计学差异(P<0.05)。④残疾程度(修订Rankin量表,Modified Rankin Scale, MRS)方面,治疗21天后两组残疾程度MRS积分均明显下降,两组间无显著差异(P>0.05)。⑤中医症状积分方面,治疗21天后两组中医症状积分均较治疗前显著下降,两组间无显著差异(P>0.05)。结论：中医综合方案和西医方案对缺血性中风早期康复神经功能缺损、肢体运动功能、日常生活活动能力、残疾及中医症状均有显著改善作用。其中,中医综合方案在神经功能缺损和日常生活活动能力改善方面疗效较好,优于西医方案；而在肢体运动功能、残疾及中医症状积分方面,中医综合方案较西医方案无显著优势。2中医综合方案的DEA综合评价研究对象、试验分组及治疗方案同1。采用DEA综合评价方法,将入选病例中的每位患者均作为独立的决策单元(Decision Making Unit, DMU),住院总费用作为投入指标,神经功能缺损、肢体运动功能、日常生活活动能力、残疾中医症状改善作为产出指标,使用C2R模型和BC2模型,综合评价中医综合方案的相对有效性。结果表明：①效率值分析结果证实,中医综合治疗组C2R模型和BC2模型的效率均值(θ)分别为0.27和0.35,西医对照组效率均值分别为0.22和0.31,中医综合方案优于西医方案；而且,中医综合方案的规模效率亦优于西医方案；两组的规模收益状况均为递减。②松弛变量分析,中医综合方案在产出1(神经功能缺损),产出2(肢体运动功能),产出4(残疾程度),产出5(中医症状积分)均较西医方案组距离理想状态更近；在产出3(日常生活能力),西医方案距离理想状态更近。结论：采用DEA法综合评价中医综合方案的临床疗效,结果表明中医综合方案优于西医方案；而且,中医综合方案在神经功能缺损、肢体运动功能、残疾及中医症状积分改善上距离理想状态更近,西医方案在日常生活能力方面距离理想状态更近。3不同中医治疗方案的单指标疗效评价研究对象及治疗方案同1。根据住院期间实际干预情况,分为中医综合方案1组(93例)、中医综合方案2组(54例)和中医综合方案3组(46例)。三组病人均在接受内科基础治疗的基础上,中医综合方案1组为中药汤药+中药注射剂+针灸推拿+现代康复,中医综合方案2组为中药注射剂+针灸推拿+现代康复,中医综合方案3组为中药汤药+中药注射剂+针灸推拿。研究不同中医治疗方案对早期缺血性中风神经功能缺损、肢体运动功能、日常生活活动能力、残疾中医症状改善的影响。结果表明：①神经功能缺损方面,在治疗7天时,三组治疗方案在NIHSS改善程度上有显著差异(P＜0.05),其中中药汤药+中药注射剂+针灸推拿+现代康复组(1组)疗效最好；在14和21天时,三组治疗方案对NIHSS的改善无显著差异。②肢体运动功能方面,在治疗7天、14天及21天,三组治疗方案对FMA的改善无显著差异。③日常生活活动能力方面,在治疗21天,三组治疗方案在BI改善程度上有显著差异(P＜0.05),其中中药汤药+中药注射剂+针灸推拿+现代康复组(1组)疗效最好。④残疾程度方面,在治疗21天,三组治疗方案在MRS改善程度上有显著差异(P＜0.05)；其中,中药汤药+中药注射剂+针灸推拿+现代康复组(1组)疗效最好。⑤中医症状积分方面,在治疗21天,三组治疗方案在中医症状改善上有显著差异(P＜0.05)；其中,中药汤药+中药注射剂+针灸推拿+现代康复组(1组)在改善中医症状方面疗效最好。结论：不同中医综合方案的单指标疗效评价,结果表明,中药汤药+中药注射剂+针灸推拿+现代康复在日常生活活动能力、残疾及中医症状改善方面均优于中药注射剂+针灸推拿+现代康复组和中药汤药+中药注射剂+针灸推拿组；在神经功能缺损改善上,治疗7天时,中药汤药+中药注射剂+针灸推拿+现代康复组显示了较好的优势,但在14天和21天,三组疗效无差异；肢体运动功能改善方面,三种中医综合方案无显著差异。4不同中医治疗方案的DEA综合评价研究对象及治疗方案同1,分组情况同3。采用DEA综合评价方法,将入选病例中的每位患者均作为独立的决策单元(DMU),住院总费用作为投入指标,神经功能缺损、肢体运动功能、日常生活活动能力、残疾中医症状改善作为产出指标,应用C2R模型和BC2模型,综合评价不同中医治疗方案的相对有效性。结果表明：①效率值分析结果证实,中药注射剂+针灸推拿+现代康复组(2组)的综合评价疗效最优,C2R模型和BC2模型效率值(θ)分别为0.39和0.53；其次是中药汤药+中药注射剂+针灸推拿+现代康复组(1组),效率值分别为0.33和0.43；最差的为中药汤药+中药注射剂+针灸推拿组(3组),效率值分别为0.27和0.34。规模效率中,中药汤药+中药注射剂+针灸推拿+现代康复组(1组)的规模效率最优,其次为中药汤药+中药注射剂+针灸推拿组(3组),中药注射剂+针灸推拿+现代康复组(2组)的规模效率最差；三组的规模收益状况均为递减。②松弛变量分析,中药注射剂+针灸推拿+现代康复组(2组)在产出1(神经功能缺损),产出2(肢体运动功能),产出4(残疾程度),产出5(中医症状积分)均优于其他两个治疗方案,更接近理想状态；中药汤药+中药注射剂+针灸推拿+现代康复组(1组)在产出3(日常生活能力)疗效较好,距离理想状态最近。结论：采用DEA法评价不同中医综合方案的临床疗效,结果表明,中药注射剂+针灸推拿+现代康复方案的综合评价疗效最优,其次是中药汤药+中药注射剂+针灸推拿+现代康复方案,中药汤药+中药注射剂+针灸推拿方案疗效最差。中药注射剂+针灸推拿+现代康复方案在神经功能缺损、肢体运动功能、残疾及中医症状改善上距离理想状态最近；中药汤药+中药注射剂+针灸推拿+现代康复方案在日常生活能力改善上距离理想状态最近。创新点：1.本研究以早期缺血性中风为例,将运筹学数据包络分析法引入到中医复杂干预的多指标疗效综合评价中,具有开创性尝试意义,为中医临床疗效综合评价提供了又一条思路。2.本研究结果表明,DEA用于中医疗效综合评价具有以下优势：①每个患者均作为独立的决策单元,充分体现中医复杂干预及个体化治疗的优势和特点；②将多维度疗效指标同时纳入并综合分析,在分析中忽略了各评价指标量纲的不同质性；③在处理疗效指标间的权重时,将其作为变量进行分析,克服了中医综合评价中“指标赋权”的瓶颈问题；④松弛变量反映不同治疗方案达到最佳疗效的可能性,即实际产出(实际疗效)距离理想产出状态(最佳疗效)有多远,有利于治疗方案的优化。因此,DEA应用于中医综合评价是适合的,将具有重要的研究前景和使用价值。3.中医临床疗效评价应回归临床实际,充分体现中医复杂干预的优势和特点。本研究根据临床科研实际情况将中医综合方案重新划分为不同的方案,并评价各种方案的优势和特点,为中医综合方案的优化奠定一定的基础,在方法学上有一定创新性。更多还原

【Abstract】 Therapeutic effect evaluation is the main of clinical evaluation, the study of methods of Therapeutic effect evaluation is very important, had become a great matter for TCM’s existence and development. Complex is a character of TCM’s diagnosis and intervention, such as the determination of treatment based in pathogenesis obtained through differentiation of symptoms and signs, therapeutic intervention is variegated, and the goals of treatment is multidimensional. Previous TCM clinical evaluation emphasized on single therapy(regime), single outcome index, single dimension, and lacked of the measurement of multi-dimension in TCM complicated intervention, accordingly lowered TCM’S superiority, and covered TCM actual effect, so, therapeutic effect evaluation should return to clinical actuality, reflect the real world of clinical, emphasize TCM’S integrated effect to the patient and comprehensive evaluation.Data envelopment analysis(DEA) is a new field in the cross study of operational research, Management Science and Mathematical Economics, used to evaluate the relative effectiveness in the department have the same kind of input and output. DEA has absolutely superiority to deal with multi-input and multi-output problem, and corresponding to TCM’S characteristic of complex intervention(multi-input) and multi-index comprehensive evaluation.Under the support of 863 program, we utilized DEA to evaluate the relative effectiveness in the patients of chemic stroke early rehabilitation, and research DEA’S possibility and applicability in TCM’s complex intervention.Research contents and results:Study 1 Single index therapeutic effect of TCM’S comprehensive regimenThe hospital patients of chemic stroke early rehabilitation come from Dongzhimen hospital affiliated to Beijing university of traditional Chinese medicine, Beijing Tiantan hospital affiliated to capital medical university, the second affiliated hospital of Tianjin university of traditional Chinese medicine, the first affiliated hospital of Henan university of traditional Chinese medicine, the second affiliated hospital of Shandong university of traditional Chinese medicine, the people’s hospital of Xingtai, hospital affiliated to Changchun university of traditional Chinese medicine, the second affiliated hospital of Guangdong university of traditional Chinese medicine, and they are randomly distributed to TCM comprehensive regime group and western medicine group by clinical research central random system according to 2:1 ratio. TCM comprehensive group apply the regime of basic therapy of internal medicine, Chinese herbal medicine, Chinese materia medica injection, acupuncture, massage, rehabilitative treatment based BOBATH Therapy, and the western medicine group apply the regime of basic therapy of internal medicine rehabilitative treatment based BOBATH Therapy. The index including neurologic impairment degree measured by (National Institute of Health Stroke Scale(NIHSS); motor function measured by Fugl-Meyer Assessment(FMA); activities of daily living measured by Barthel Index(BI); disability degree measured by Modified Rankin Scale(MRS); TCM symptom measured by TCM Stroke Sympotom Scale(ZHONGYI) Our object was to evaluate the TCM COMPREHENSIVE REGIMEN’S effect ON neurologic impairment, motor function, activities of daily living, disability degree and TCM symptom.Results:①Improvement of neurologic impairment:at 7 day, all of two group’s NIHSS score obviously decrease(P<0.001), and at 14day,21 day, two group’s score continued decrease., every evaluation point had statistic difference between two group s(P<0.05).②Improvement of motor function:at 7 day, all of two group’s FMA score obviously increase (P<0.001), and 14 days,21 days, two group’s score continued increase. There was no statistic difference at every evaluation point between two groups (P>0.05).③Improvement of activities of daily living:at 21 day, all of two group’s BI score obviously increased(P<0.001), and There was statistic difference between two groups (P<0.05).④Improvement of disability:at 21 day, all of two group’s MRS score obviously increased(P<0.001), but There was no statistic difference between two groups (P>0.05).⑤Improvement of TCM Symptom:at 21 day, all of two group’s ZHONGYI score obviously decreased(P<0.001), but there was no statistic difference between two group (P>0.05).Conclusion:Both TCM comprehensive regimen and western medicine regimen have favourable improvement to neurologic impairment, motor function, activities of daily living, disability, TCM Symptom in ischemic stroke early rehabilitation patient. TCM comprehensive regimen group wais better than western medicine regimen group in neurologic impairment and activities of daily living, but there was no statistic difference between two groups in motor function, disability and TCM Symptom.Study2 DEA evaluation on TCM comprehensive regimenThe study object, groups, therapeutic regimen was same to study 1. Each patient as a decision making unit(DMU), the input index is the cost of hospitalization, and the output index is the improvement of neurologic impairment, motor function, activities of daily living, disability degree and TCM symptom. The object was to evaluate the relative effectiveness, the DEA model are C2R and BC2.Results:①TCM comprehensive group’s average efficiency value was 0.27 and 0.35, and the western medicine group’s average efficiency value was 0.22 and 0.31, the former was superior to the latter. TCM comprehensive group’s average scale efficiency value was also superior to the western medicine group.②Slack variance analysis:TCM comprehensive group is relatively approaching to ideal condition in the improvement of neurologic impairment, motor function, disability degree and TCM symptom, but western medicine group was relative approaching to ideal condition in the improvement of activities of daily living.Conclusion:DEA evaluation on TCM comprehensive regimen and western medicine group, TCM comprehensive group is better than western medicine group, and TCM comprehensive group was relatively approaching to ideal condition in the improvement of neurologic impairment, motor function, disability degree and TCM symptom, but western medicine group was relative approaching to ideal condition in the improvement of activities of daily living. Study3 Single index therapeutic effect of different TCM’S comprehensive regimenThe research object is the patients in TCM’S comprehensive group. Based on the clinical actuality, we divided TCM’S comprehensive group into three groups:under the basic treatment of internal medicine, group 1(93 patients) apply the regimen of, Chinese herbal medicine, Chinese materia medica injection, acupuncture, massage, rehabilitative treatment based BOBATH Therapy, group 2 (54 patients) was Chinese materia medica injection, acupuncture, massage, rehabilitative treatment based BOBATH Therapy, and group 3(46 patients) was Chinese herbal medicine, Chinese materia medica injection, acupuncture, massage.Object:To evaluate the therapeutic effect of different TCM’S comprehensive regimen on neurologic impairment, motor function, activities of daily living, disability degree and TCM symptom.Results:①Improvement of neurologic impairment:at 7 day evaluation point, all of group 1’s NIHSS score obviously decrease compared to the other two groups(P<0.05), but at 14day,21 day, there is no statistic difference between three group (P>0.05).②Improvement of motor function:There was no statistic difference at every evaluation point between three group (P>0.05).③Improvement of activities of daily living:at 21 day, There was statistic difference between three group (P<0.05), group 1 was the best.④Improvement of disability:at 21 day, There was statistic difference between three group (P<0.05), group 1 was the best.⑤Improvement of TCM Symptom:at 21 day, There was statistic difference between three group (P<0.05), group 1 is the best.Conclusion:Single index therapeutic effect of different TCM’S comprehensive regimen, the group 1 (regimen of, Chinese herbal medicine, Chinese materia medica injection, acupuncture, massage, rehabilitative treatment based BOBATH Therapy) is the best in improvement of activities of daily living, disability, TCM Symptom. And at 7 day evaluation point, group 1’s NIHSS score obviously decreased compared to the other two groups but at 14day,21 day, there was no statistic difference between three groups. There was no statistic difference at every evaluation point between three groups in the improvement of motor function.Study 4 DEA evaluation on different TCM comprehensive regimensThe study object, groups, therapeutic regimen is same to study 3.Each patient as a decision making unit(DMU), the input index is the cost of hospitalization, and the output index is the improvement of neurologic impairment, motor function, activities of daily living, disability degree and TCM symptom. The object was to evaluate the relative effectiveness, the DEA model are C2R and BC2.Result:①groupl’s average efficiency value was 0.33 and 0.43, group2 is 0.39 and 0.53, group3 was 0.27 and 0.34, group2’s relative effectiveness is the highest. But group1’s scale efficiency was the highest, the next was group3, the next is group2.②slack variance analysis:group2 was relatively approaching to ideal condition in the improvement of neurologic impairment, motor function, disability degree and TCM symptom, but group 1 was relative approaching to ideal condition in the improvement of activities of daily living.Conclusion:DEA evaluation on different TCM comprehensive regimen, group 1 was the best, the next is group2, group3 was the worst. Slack variance analysis indicated group2 was relatively approaching to ideal condition in the improvement of neurologic impairment, motor function, disability degree and TCM symptom, but group 1 was relative approaching to ideal condition in the improvement of activities of daily living.Innovation1. We applied DEA on TCM complex intervention and multi-index comprehensive evaluation, and compared to mono-index evaluation, we research the index system and adapted mathematic model in TCM Therapeutic effect evaluation firstly.2. The results indicate that DEA has some superiority in TCM Therapeutic effect comprehensive evaluation.①Each patient as a decision making unit(DMU), sufficiently indicated the superiority and characteristic.②DEA can envelope multi-dimension index into analysis, ignore index’s nonhomogeneity.③It is not necessary to determine the weight of the index in advance, and overcome the deficiency in index weight’s determination.④Slack variable can reflect possibility of different regimens reaching the best therapeutic effectness, it is profit to optimize the therapeutic regimen. So, DEA is a suitable way to evaluate TCM comprehensive effect, and should have a good perspective.3. In order to increase the evidence level, TCM clinical evaluation should return to clinical actuality. Based actual clinical science research, this study classified TCM regimen into three regimen, and evaluated their superiority and characteristic, this method may supply a way for TCM regimen’s Optimization.更多还原