PURPOSE The United Kingdom Glaucoma Treatment Study (UKGTS) demonstrated the effectiveness of an intraocular pressure-lowering drug in patients with glaucoma using visual field progression as a primary outcome. The present study tested the hypothesis that responses on patient-reported outcome measures (PROMs; secondary outcome measure) differ between patients receiving a topical prostaglandin analog (latanoprost) or placebo eye drops in UKGTS. DESIGN Multicenter, randomized, triple-masked, placebo-controlled trial. PARTICIPANTS Newly diagnosed glaucoma patients in the UKGTS with baseline and exit PROMs (n = 182 and n = 168 patients from the treatment and placebo groups, respectively). METHODS In the UKGTS (trial registration number, ISRCTN96423140), patients with open-angle glaucoma were allocated to receive latanoprost (treatment) or placebo; the observation period was 24 months. Patients completed general health PROMs (European Quality of Life in 5 Dimensions [EQ-5D] and 36-item Short Form [SF-36]) and PROMs specific to glaucoma (15-item Glaucoma Quality of Life [GQL-15] and 9-item Glaucoma Activity Limitation [GAL-9]) at baseline and exit from the trial. Percentage changes between measurement on PROMs were calculated for each patient and compared between treatment arms. In addition, differences between stable patients (n = 272) and those with glaucomatous progression (n = 78), as determined by visual field change (primary outcome), were assessed. MAIN OUTCOME MEASURE PROMs on health-related and vision-related quality of life. RESULTS Average percentage change on PROMs was similar for patients in both arms of the trial, with no statistically significant differences between treatment and placebo groups (EQ-5D, P = 0.98; EQ-5D visual analog scale, P = 0.88; SF-36, P = 0.94, GQL-15, P = 0.66; GAL-9, P = 0.87). There were statistically significant differences between stable and progressing patients on glaucoma-specific PROMs (GQL-15, P = 0.02; GAL-9, P = 0.02), but not on general health PROMs (EQ-5D, P = 0.62; EQ-5D visual analog scale, P = 0.23; SF-36, P = 0.65). CONCLUSIONS Average change in PROMs on health-related and vision-related quality of life was similar for the treatment and placebo groups in the UKGTS. The PROMs used may not be sensitive enough to function as primary end points in clinical trials when participants have newly diagnosed early-stage glaucoma.

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患者自我报告的结果是否足够敏感，可以用作临床试验的终点？：来自英国青光眼治疗研究的证据。

目的英国青光眼治疗研究（UKGTS）以视野进展为主要结果证明了一种降低眼压的药物在青光眼患者中的有效性。本研究检验了以下假设：在UKGTS中接受局部前列腺素类似物（拉坦前列素）或安慰剂滴眼液的患者之间，对患者报告的结局指标（PROM；次要结局指标）的反应有所不同。设计多中心，随机，三重屏蔽，安慰剂对照试验。参与者UKGTS中新诊断的青光眼患者具有基线和退出PROM（治疗组和安慰剂组分别为182例和168例）。方法在UKGTS（试验注册号ISRCTN96423140）中，将开角型青光眼患者分配接受拉坦前列素（治疗）或安慰剂。观察期为24个月。患者完成了一般健康PROMs（5个维度的欧洲生活质量[EQ-5D]和36个项目的简短形式[SF-36]）和青光眼特有的PROMs（15个项目的青光眼生活质量[GQL-15]和9个-基线青光眼活动受限[GAL-9]），并退出试验。计算每个患者在PROMs测量之间的百分比变化，并在治疗组之间进行比较。此外，评估了稳定患者（n = 272）和青光眼进展患者（n = 78）之间的差异，这是通过视野变化（主要结局）确定的。关于健康和视力的生活质量的主要成果衡量方案。结果试验两部分患者的PROM的平均百分比变化相似，治疗组与安慰剂组之间无统计学差异（EQ-5D，P = 0.98； EQ-5D视觉模拟量表，P = 0.88； SF-36，P = 0.94，GQL-15，P = 0.66； GAL-9， P = 0.87）。稳定期和进展期患者在青光眼特异性PROMs（GQL-15，P = 0.02; GAL-9，P = 0.02）上有统计学差异，但在一般健康PROMs（EQ-5D，P = 0.62; EQ- 5D视觉模拟量表，P = 0.23； SF-36，P = 0.65）。结论UKGTS的治疗组和安慰剂组在健康相关和视觉相关的生活质量方面，PROM的平均变化相似。当参与者新诊断出早期青光眼时，所使用的PROM可能不够灵敏，无法在临床试验中用作主要终点。P = 0.66；GAL-9，P = 0.87）。在稳定期和进展期患者中，青光眼特异性PROM（GQL-15，P = 0.02； GAL-9，P = 0.02）在统计学上有显着差异，但在一般健康PROM（EQ-5D，P = 0.62; EQ- 5D视觉模拟量表，P = 0.23； SF-36，P = 0.65）。结论UKGTS的治疗组和安慰剂组在健康相关和视觉相关的生活质量方面，PROM的平均变化相似。当参与者新诊断出早期青光眼时，所使用的PROM可能不够灵敏，无法在临床试验中用作主要终点。P = 0.66；GAL-9，P = 0.87）。在稳定期和进展期患者中，青光眼特异性PROM（GQL-15，P = 0.02； GAL-9，P = 0.02）在统计学上有显着差异，但在一般健康PROM（EQ-5D，P = 0.62; EQ- 5D视觉模拟量表，P = 0.23； SF-36，P = 0.65）。结论UKGTS的治疗组和安慰剂组在健康相关和视觉相关的生活质量方面，PROM的平均变化相似。当参与者新诊断出早期青光眼时，所使用的PROM可能不够灵敏，无法在临床试验中用作主要终点。23; SF-36，P = 0.65）。结论UKGTS的治疗组和安慰剂组在健康相关和视觉相关的生活质量方面，PROM的平均变化相似。当参与者新诊断出早期青光眼时，所使用的PROM可能不够灵敏，无法在临床试验中用作主要终点。23; SF-36，P = 0.65）。结论UKGTS的治疗组和安慰剂组在健康相关和视觉相关的生活质量方面，PROM的平均变化相似。当参与者新诊断出早期青光眼时，所使用的PROM可能不够灵敏，无法在临床试验中用作主要终点。