我国作为拥有帕金森病（Parkinson’s Disease, PD）患者人数最多的国家，承担了全世界近一半数量的PD患者所带来的各方面压力，随着人口老龄化的加剧，这种负担将会愈加严重。长期有效的疾病管理不仅能减轻疾病负担，提高患者生活质量，更能有效利用社会资源，降低疾病成本。深入了解中国PD患者就医和治疗情况，选择更合适的治疗路径，不仅受到卫生政策制定者的关注，而且能通过合理配置卫生资源从而有效满足我国PD患者的需求。在当前中国医疗系统环境下，因PD所产生的负担主要还是由患者家庭所承担，因此，选择效果更突出且成本相对可接受的疗法从一定程度上能够满足患者及其家庭的就医预期，并且帮助他们更好地支配有限的医疗预算。本研究从实际需求出发，以了解中国PD患者的基本情况为基础，从疗法选择入手，从临床疗效和经济成本角度评估药物和脑深部刺激（Deep Brain Stimulation, DBS）疗法的短期效应，并通过建立Markov模型模拟疾病发展过程从而估算疗法的长期成本效用，从疗法选择的角度为疾病管理提供基础依据。论文首先调研了PD患者的基本情况，重点关注就医行为，分析其特点和存在的问题，并通过借鉴学习慢病管理模式，为PD疾病的公共管理提出了建议。基于调研结果，以疗法选择作为切入点，选择PD治疗中最常用的药物治疗和DBS治疗，通过对临床试验患者1年的随访研究证明了DBS在治疗PD短期疗效上的优势；同时通过不同治疗方式的群组间的对比研究发现，DBS手术组的患者生活质量优于药物组，其照顾者的心理负担也小于药物治疗组，且DBS组在其他医疗资源上的消耗更少，但由于参与调研的两组患者病程不匹配，导致经济学的对比存在偏差，需从长期角度做进一步对比。受随访时间限制，选择用模型的方式估算PD疗法的长期成本效用。利用Markov模型来模拟疾病的发展过程，模型参数部分来源于本论文的实际临床研究和调研，部分取自经典文献，借助TreeAge软件，模拟初设条件下DBS和药物疗法在不同时间点所产生的成本、效用以及两者的增量成本效果比。模拟结果证明，在整个生命周期内，DBS相对药物治疗的增量成本效果比ICER在可接受范围内，时间越长，两者的ICER越小。患者的发病年龄，选择DBS手术的时间以及手术的治疗成本等因素会较大的影响模型结果。
As a country with the largest number of Patients with Parkinson’s Disease (PD), China is bearing a heavy disease burden from almost half the number of PD patients in the world. Along with the increasingly aging population, the burden will become more serious. Long-term effective disease management will not only reduce the disease burden and improve patients’ quality of life, but make more effective use of social resources thus reducing disease costs. Futher understanding of Chinese PD patients’ activities on how to find solution and the choice of different therapies is important to design a more appropriate intervention, and this is not only to provide information for the health policy makers, but also to meet the demand of the PD patients who need to get efficient resources. In the current medical environment in China, the load of PD treatment was almost transfered to patients’ families, and to them, choosing an efficient treatment with an affordable cost could meet the needs of patients and their expectations of therapy, and help them better allocate the limited health budget.By surveying the basic data of Chinese PD patients, this research focused on the medical treatment decisions of patients during their seeking medical help. Typically, most of patients finally chose medicine for PD and some found deep brain stimulation (DBS) therapy. This paper evaluated the short-term effects of medicine treatment (MT) and DBS therapy from the perspective of clinical effects and economic costs. And by establishing a Markov model to simulate disease progress, this paper also estimated the long-term cost-effectiveness of MT and DBS therapy. All these results provided fundamental data for treatment decisions during disease management.In this thesis, we first investigated the basic data of Chinese PD patients through an Internet survey. Main attention was forcused on the healthcare seeking behaviors of PD patients or their family members. By data analysis, some characteristics and exising problems were found. On this point, we proposed some strategic recommendations for the public disease management of PD.From surveying data, one key point we fully understood was patients’ decision on treatments. Further comparative studis of differerent treatments were conducted. The candidate treatments were classic antiparkinsonism medicine treatment and the DBS therapy. A one-year follow-up study proved the advantages of DBS on the short-term clinical effect in the treatment of PD. Another comparison from economic perspective found that those PD patients who received DBS therapy got higher qualities of life and eased the psychological buren of their caregivers compared with MT group. The medical resource consumption in DBS group was also significantly lower than that of MT group. But the deviation of the short-term cost-effectiveness results among these two groups suggested a need for further analysis in an enough long time horizon.Limited by the follow-up time, a Markov model simulating the progress of PD was used to estimate long-term cost-effectiveness between MT and DBS therapy. The model parameters partly came from the previous results of this thesis and the rest were selected through literature reviewing. With the help of TreeAge software, an initial condition was set to simulate the accumulated costs and utilities under the treatment of these two candidates at different cycle periods. The simulation results showed that in the whole life-long time horizon, patient would get more QALYs and spend more money if she/he chose DBS therapy. The incremental cost-effectiveness ratio (ICER) between DBS and MT was in the acceptable range in the life-long horizon. The onset age of PD patients, time of choosing DBS therapy and the treatment costs concerning DBS surgery could greatly affect the model results.
