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Patient-reported outcome measures should not be the primary outcome in glaucoma clinical trials of disease modification
British Journal of Ophthalmology ( IF 3.7 ) Pub Date : 2023-01-01 , DOI: 10.1136/bjo-2021-320722
Alessandro Rabiolo 1 , Keith Barton 1 , Andrew Ian McNaught 2
Affiliation  

In the 19th century, Sir William Osler, one of the fathers of modern medicine, stated that ‘ The good physician treats the disease; the great physician treats the patient who has the disease’, indicating that, as clinicians, we should pursue a patient-centred, rather than disease-centred approach. In the attempt to provide more patient-centred holistic care, there has been increasing interest in patient-reported outcomes (PROs) and patient-reported outcomes measures (PROMs), which are any self-reported health status directly reported by patients and the tools (usually questionnaires) used to measure them, respectively. A variety of validated PROMs, either with generic or disease-specific constructs, are available in ophthalmology to measure the effect of a given eye condition on the overall health function and disease-specific features (eg, symptoms, treatment side effects, coping), respectively. The widespread use of PROMs across all fields of medicine has caused a paradigm shift in clinical research, with patients’ perception becoming a major determinant of therapeutic interventions’ outcomes. As a result, PROMs are increasingly used as endpoints in clinical trials across all medical fields, including, recently, glaucoma. The choice of primary outcome is critical in randomised controlled trials (RCTs), and determines the sample size. A poorly chosen primary outcome may invalidate the results of otherwise well-designed and rigorously conducted trials, producing unreliable results and wasting resources.1 The primary outcome is the outcome measure that has the greatest importance according to the various stakeholders (investigators, patients, policymakers, funding bodies, pharmaceutical companies).1 Preventing visual disability from glaucoma and preserving the visual-related quality of life (VR QoL) are arguably the ultimate therapeutic goals in glaucoma management. Glaucoma is an initially asymptomatic and usually slowly progressing disease, so attaining such a hard endpoint as symptomatic visual disability is (fortunately) infrequent, and besides, nearly always requires a long period of time, perhaps measured in …

中文翻译:

患者报告的结果测量不应作为青光眼疾病修正临床试验的主要结果

在 19 世纪,现代医学之父之一威廉·奥斯勒爵士说:“良医治病;大医治病”,这表明作为临床医生,我们应该追求以患者为中心,而不是以疾病为中心。为了提供更多以患者为中心的整体护理,人们越来越关注患者报告的结果 (PRO) 和患者报告的结果测量 (PROM),它们是患者直接报告的任何自我报告的健康状况和工具(通常是问卷)分别用来衡量他们。各种经过验证的 PROM,无论是具有通用结构还是特定于疾病的结构,都可用于眼科,以测量给定眼睛状况对整体健康功能和特定疾病特征的影响(例如,症状、治疗副作用、应对)。PROM 在所有医学领域的广泛使用引起了临床研究的范式转变,患者的感知成为治疗干预结果的主要决定因素。因此,PROM 越来越多地用作所有医学领域临床试验的终点,包括最近的青光眼。主要结果的选择在随机对照试验 (RCT) 中至关重要,它决定了样本量。主要结果选择不当可能会使设计良好和严格执行的试验结果无效,产生不可靠的结果并浪费资源。 , 资助机构, 制药公司)1 青光眼最初是无症状的,通常进展缓慢,因此(幸运的是)很少会出现有症状的视力障碍这样的硬终点,此外,几乎总是需要很长一段时间,也许以……衡量
更新日期:2022-12-15
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