Measuring functional performance in patients with COPD: a discussion of patient-reported outcome measures

Abstract

Objective:

This study was designed to explore antihypertensive efficacy and safety of a combination of amlodipine (CCB) and valsartan (ARB), in essential hypertensive patients not adequately controlled by amlodipine monotherapy.     

患者报告结局在罕见病临床研究中的应用探讨

患者报告结局(patient-reported outcome,PRO)是指直接来自于患者对自身健康状况、功能状态以及治疗感受的报告,近年来被广泛应用于慢性病生存质量评价、临床研究、不良事件监测以及药物注册审批等。罕见病是一类严重影响患者身体功能、精神和生活质量的疾病。由于缺乏对疾病自然史的了解以及敏感的生物标志物,患者的意见和感受往往是罕见病用药临床决策及干预措施疗效评价的关键及最终目标。目前,国内关于PRO的研究刚刚起步,鲜少涉足罕见病领域。该文就国际上PRO的定义、政策历程及其在罕见病领域的应用现状作一综述,并进一步探讨PRO在罕见病领域应用存在的挑战,展望PRO在我国的应用前景。     

A case study of a patient with MS followed for 15?years using patient-reported outcome measures

Patient-reported outcome (PRO) instruments have grown in popularity because they provide unique information to help evaluate healthcare services from the patient??s perspective. The objective of this case study is to present the utility of both qualitative and quantitative self-reported data collected over time in describing disease progression, managing treatment, and assessing quality of life in patients with multiple sclerosis (MS). A 62-year-old female with relapsing-remitting MS was followed longitudinally for 15?years by the same neurologist and physiatrist who used magnetic resonance images to monitor her lesions and the neurologist-administered EDSS instrument to monitor her disability. Concurrently, the patient kept track of her symptoms, feelings, moods, treatments, side effects and outcomes of care through diaries and used the comprehensive LIFEware^SM System self-reported, symptom-oriented questionnaires (assessments) to track her functional health status and quality of life. Medical records, EDSS scores, LIFEware^? self-reported measures along with diary entries for the period were reviewed. Self-reported data, as well as clinician assessed data, are both key to comprehensively treating a patient with MS. By incorporating PRO measures into practice, a physician can address significant self-reported symptoms, minimize limitations, maintain function, enhance quality of life and provide more holistic disease management treatment.     

慢性心力衰竭患者报告结局量表的信度 效度和可行性评价

目的对慢性心力衰竭患者报告结局(PRO)量表进行信度、效度和可行性的评价。方法参考国外的慢性心血管疾病评价工具,编制慢性心力衰竭PRO量表。用该量表对太原不同区的8所医院进行大规模调查,利用大规模调查的信息数据再一次进行条目筛选,形成终量表,并对终量表的信度、效度、可行性进行分析。结果慢性心力衰竭PRO量表共57个条目,对360例患者和100名健康人有效数据进行分析。结果显示:该心力衰竭PRO量表具有较好的信度、效度及临床可行性。总量表的克朗巴赫系数为0.913,生理领域0.903,心理领域0.941,社会领域0.827,治疗领域0.839;内容效度、维度相关性、构念效度、反应度结果显示该表具有良好的效度;此次调查的回收率为98.9%,有效率为98.9%。结论我们研制出的基于慢性心力衰竭患者报告的临床结局评价量表具有较好的信度、效度和可行性,可以作为慢性心力衰竭疾病临床疗效评价研究的工具。     

Measuring symptom response to pharmacological interventions in patients with COPD: a review of instruments used in clinical trials

OBJECTIVES: To identify and evaluate the instruments used to measure the effect of pharmacological intervention on symptoms of chronic obstructive pulmonary disease (COPD) in clinical trials. DESIGN: An extensive literature search was conducted for articles published in English in a peer-reviewed journal from 1995 to March 2002 which described a randomised controlled clinical trial measuring symptoms of COPD in response to pharmacological interventions. PATIENTS: Patients with any severity of COPD. INTERVENTIONS: Any pharmacological intervention for treatment of COPD. MEASUREMENTS AND RESULTS: A total of 43 eligible articles were identified. The individual symptoms most frequently measured were dyspnoea/breathlessness, chest tightness or discomfort and exacerbations. There was considerable variability in the methods, terminology and symptom measurement instruments used. The most widely used instruments for measuring dyspnoea were the Borg scale, the Baseline Dyspnoea Index and the Transitional Dyspnoea Index. None of the instruments used had published evidence of rigorous psychometric testing. CONCLUSIONS: Numerous methods have been employed to assess the symptoms of COPD in clinical trials, making it difficult to compare the results of different trials. No single measurement instrument predominates, and none of the measures identified in the review have undergone rigorous psychometric testing in this patient population. There is a clear need for a fully developed and validated tool for measuring the effects of therapeutic interventions on symptoms in COPD in clinical trials.     

Measuring symptom response to pharmacological interventions in patients with COPD: a review of instruments used in clinical trials

SUMMARY

Objectives: To identify and evaluate the instruments used to measure the effect of pharmacological intervention on symptoms of chronic obstructive pulmonary disease (COPD) in clinical trials.

Design: An extensive literature search was conducted for articles published in English in a peer-reviewed journal from 1995 to March 2002 which described a randomised controlled clinical trial measuring symptoms of COPD in response to pharmacological interventions.

Patients: Patients with any severity of COPD.

Interventions: Any pharmacological intervention for treatment of COPD.

Measurements and results: A total of 43 eligible articles were identified. The individual symptoms most frequently measured were dyspnoea/breathlessness, chest tightness or discomfort and exacerbations. There was considerable variability in the methods, terminology and symptom measurement instruments used. The most widely used instruments for measuring dyspnoea were the Borg scale, the Baseline Dyspnoea Index and the Transitional Dyspnoea Index. None of the instruments used had published evidence of rigorous psychometric testing.

Conclusions: Numerous methods have been employed to assess the symptoms of COPD in clinical trials, making it difficult to compare the results of different trials. No single measurement instrument predominates, and none of the measures identified in the review have undergone rigorous psychometric testing in this patient population. There is a clear need for a fully developed and validated tool for measuring the effects of therapeutic interventions on symptoms in COPD in clinical trials.     

基于关节炎患者报告结局量表的条目筛选

目的研制关节炎患者报告结局量表,为临床疗效评价提供工具。方法依照美国食品药品管理局(FDA)规定的量表制作流程,建立条目池,编制初量表,对太原市3家医院的322个调查对象实行多中心临床调查,回收后利用项目反应理论、离散趋势法、因子分析法、相关系数法、克朗巴赫系数法、重测信度法6种方法联合筛选条目。结果综合考虑分析结果,最终形成包含生理、心理、社会、治疗4个领域的量表,涵盖了躯体症状、自理能力、抑郁、焦虑、自信、个人关系、社会支持、社会活动、依从性、满意度、药物不良反应11个方面,共45个条目。结论最终量表全面反映了关节炎患者生命质量的内涵,对临床疗效评价具有指导意义。     

The state of patient-reported outcomes in atrial fibrillation : a review of current measures

Atrial fibrillation (AF) is the most common cardiac arrhythmia, affecting approximately 2.2 million people in the US. The presentation of AF ranges from asymptomatic to severely symptomatic. When symptomatic, AF has been shown to have an adverse impact on health-related quality of life (HR-QOL) and to result in increased healthcare costs. The objective of this analysis was to review the current AF literature on patient-reported outcomes (PROs) in order to evaluate the impact of AF on PROs and the applicability of current PRO measures in assessing AF outcomes.HR-QOL and symptoms were the most frequently assessed PROs; however, the sensitivity of the majority of the questionnaires for detecting subtle change is not known. For highly symptomatic patients, interventional procedures reduce symptoms and improve HR-QOL; however, this is a small cohort of patients with AF. For the most part, PROs are equivalent between pharmacological treatments or are not known for the large percentage of patients treated pharmacologically with antiarrhythmic or rate-controlling drugs.PRO assessment in AF patients is an area that needs continued development. AF-specific PRO measures are needed to assess the full range of patient symptoms and treatment outcomes. The impact of paroxysmal AF versus permanent AF is not well delineated, and sex and nationality differences are not known. In addition, the impact of AF on daily activities and HR-QOL is not clearly described.     

Hospital referral of older patients to community pharmacy: outcome measures in a feasibility study

International Journal of Clinical Pharmacy - Background Post-discharge medication use reviews in English community pharmacy aim to improve medicine support to recently discharged patients. However,...     

Efficacy of sublingual specific immunotherapy in patients with respiratory allergy to Alternaria alternata: a randomised,assessor-blinded,patient-reported outcome,controlled 3-year trial

Abstract

Background:

Alternaria alternata (AA) sensitisation is a common cause of respiratory allergies such as rhinitis and asthma. So far there are no controlled double-blind trials evaluating the efficacy and safety of specific sublingual immunotherapy (SLIT) in AA allergies.     

罗氟司特治疗慢性阻塞性肺疾病患者的安全性和耐受性Meta分析

目的初步系统评价罗氟司特治疗慢性阻塞性肺疾病（COPD）患者的安全性和耐受性。方法计算机检索PubMed、ISI、ScineceDirect、OVID、http：／／clinicahrials．gov、中国生物医学文献数据库（CBM）、万方数据数字化期刊全文数据库、中国期刊全文数据库（CJFD）。纳入罗氟司特治疗COPD的临床随机对照试验（RCT）,收集罗氟司特治疗COPD患者的安全性和耐受性的临床资料。应用RevMan5．0．18软件进行Meta分析。结果共纳入6个RCT,研究地点均在国外,研究质量均较高,各试验间具有基线可比性。Meta分析结果显示：与安慰剂组对比,罗氟司特治疗COPD患者时主要不良反应为：腹泻、恶心、头痛和体质量下降,具有统计学意义（P〈0．01）。结论罗氟司特的不良反应轻微,持续时间短,多不需停、减药。需进一步研究使选择性磷酸二酯酶-4（PDE-4）抑制剂在保留或提高疗效的基础上减少药物不良反应。     

Combination therapy in COPD: different response of COPD stages and predictivity of functional parameters

BACKGROUND: Inhaled corticosteroids reduce exacerbations in patients with chronic obstructive pulmonary disease (COPD) but they do not affect disease progression. FEV1, as single parameter, showed limits in describing the heterogeneity of COPD population. Combination therapy, with long-acting Beta2-agonist and corticosteroid, showed a more beneficial effect on lung function, exacerbations, and health status than single inhaled drug. The aim of this study was to assess, in stable COPD, which stage (mild, moderate, severe) shows the best response after 12 weeks inhaled treatment, and which starting functional parameters show a correlation with the response. METHODS: 170 stable COPD patients (38 mild, 66 moderate, 66 severe) were enrolled. Patients received salmeterol/fluticasone 50/500 microg Metered Dose Inhaler (MDI) bid for 12 weeks. Pulmonary function tests and clinical data were performed. Results were subdivided, on functional and clinical data, in "responders (R)" and "no-responders (NR)". RESULTS: A FEV1 improvement (+ 12% and 200 ml) was achieved in 21 mild, 28 moderate and 17 severe COPD patients, respectively 55.3%, 45.9%, and 30.9% of each group. Statistical analysis of starting functional parameters showed a correlation with the therapeutic response for FEV1/FVC, MEF50 and DLCO/VA% (p < 0.05). CONCLUSIONS: Salmeterol/fluticasone improves FEV1% in mild and moderate more than in severe COPD patients. The study confirmed the difference in response between early and advanced stage. Starting FEV1/FVC and MEF50 were significant predictors in mild and moderate stages, and starting DLCO/VA% resulted a significant predictor in moderate and severe stages.     

Inpatient hospitalization in addiction treatment for patients with a history of suicide attempt: a case of support for treatment performance measures

This study attempts to validate substance use disorder (SUD) treatment performance measures (PM) in a naturalistic treatment setting. Despite its significance in healthcare systems and in SUD populations, suicidality is one patient characteristic that remains unexplored in the context of SUD PMs. The current study focused on the extent to which the care processes encouraged by SUD PMs were associated with improved outcomes in patients with a prior suicide attempt as compared to those without. We abstracted Addiction Severity Index and health services data from the VA medical record for 381 veterans who initiated outpatient SUD treatment and completed baseline intake measures at a Midwestern VA hospital. Cox proportional hazard regressions examined how baseline characteristics, prior suicide attempts, and PM status predicted the time until hospitalization for psychiatric or substance use problems. Prior suicide attempts significantly interacted with treatment engagement, and hospitalization risk was significantly higher among individuals with a prior suicide attempt who did not meet PMs. This study provides initial observational evidence that past suicide attempts may be a factor that should be considered when defining performance standards that influence the processes of SUD treatment. Future research on PMs should take into account the differences on indicators of high risk and poor treatment outcomes.     

Oxidative stress in patients with COPD

The lung is the organ with the highest exposure to ambient air in the entire human architecture. Due to its large surface area and blood supply, the lung is susceptible to oxidative injury in the form of myriads of reactive oxygen species (ROS) and free radicals. In order to provide defense against the oxidative burden, the lungs produce various endogenous agents called antioxidants. The antioxidant species help the lungs ward off the deleterious consequences of a wide variety of oxidants/ROS, either of endogenous or environmental origin. Several mechanisms are related to the potential connection between COPD and oxidative stress. One of the most important actions of the oxidative stress is the influence of the molecular mechanisms involved in the expression proinflammatory genes. There is plenty of evidence supporting an imbalance between oxidants and antioxidants in the lung and systemic circulation of smokers and COPD patients. Detection of the oxidative burden and evaluation of their progression and phenotypes by oxidative stress biomarkers have proven challenging and difficult. Both invasive and non-invasive techniques have provided different biomarkers which contribute to the oxidative burden of the airways. An effective wide-spectrum antioxidant therapy with bioavailability is urgently needed to control the local and systemic oxidative burst in COPD. In that direction, several antioxidant agents have been evaluated as potential candidates for the management of COPD. However, despite some encouraging results, clinical trials so far have failed to elaborately define the type of antioxidant, the regimen and the time period of treatment that may improve clinically meaningful outcomes in patients with COPD.     

呼吸功能训练对老年稳定期COPD病人生活质量的影响

目的探讨呼吸功能训练对老年稳定期COPD病人生活质量的影响。方法100例老年稳定期COPD病人随机分为观察组与对照组各50例,对照组患者按慢性阻塞性肺疾病护理常规进行护理,观察组在常规护理的基础上进行积极的呼吸功能训练。结果干预后观察组的生存质量（日常生活、社会活动）显著优于对照组（P〈0．05）;观察组干预前后生存质量比较,差异有显著性（P〈0．05）。干预后观察组患者的FEVl、FEVl／FVC、FEVl／预计值明显提高（P〈0．05）。结论呼吸功能训练能提高老年稳定期COPD病人的生活质量,同时改善肺功能,值得临床推广应用。