Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study

Lee Jones; David F. Garway-Heath; Augusto Azuara-Blanco; David P. Crabb; David F. Garway-Heath; David P. Crabb; Catey Bunce; Gerassimos Lascaratos; Francesca Amalfitano; Nitin Anand; Augusto Azuara-Blanco; Rupert R. Bourne; David C. Broadway; Ian A. Cunliffe; Jeremy P. Diamond; Scott G. Fraser; Tuan A. Ho; Keith R. Martin; Andrew I. McNaught; Anil Negi; Krishna Patel; Richard A. Russell; Ameet Shah; Paul G. Spry; Katsuyoshi Suzuki; Edward T. White; Richard P. Wormald; Wen Xing; Thierry G. Zeyen

doi:10.1016/j.ophtha.2018.09.034

Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study

Lee Jones, David F. Garway-Heath, Augusto Azuara-Blanco, David P. Crabb, David F. Garway-Heath, David P. Crabb, Catey Bunce, Gerassimos Lascaratos, Francesca Amalfitano, Nitin Anand, Augusto Azuara-Blanco, Rupert R. Bourne, David C. Broadway, Ian A. Cunliffe, Jeremy P. Diamond, Scott G. Fraser, Tuan A. Ho, Keith R. Martin, Andrew I. McNaught, Anil NegiKrishna Patel, Richard A. Russell, Ameet Shah, Paul G. Spry, Katsuyoshi Suzuki, Edward T. White, Richard P. Wormald, Wen Xing, Thierry G. Zeyen

Population Health Sciences

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

25 Citations (Scopus)

Abstract

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.

Original language	English
Title of host publication	Ophthalmology
Publisher	Elsevier Inc.
ISBN (Print)	0039-128X (Print)\r0039-128X (Linking)
DOIs	https://doi.org/10.1016/j.ophtha.2018.09.034
Publication status	Published - 28 Sept 2018

Publication series

Name	Ophthalmology

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10.1016/j.ophtha.2018.09.034

http://www.mendeley.com/research/patient-selfreported-outcome-measures-sensitive-enough-used-end-points-clinical-trials-evidence-unit

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Jones, L., Garway-Heath, D. F., Azuara-Blanco, A., Crabb, D. P., Garway-Heath, D. F., Crabb, D. P., Bunce, C., Lascaratos, G., Amalfitano, F., Anand, N., Azuara-Blanco, A., Bourne, R. R., Broadway, D. C., Cunliffe, I. A., Diamond, J. P., Fraser, S. G., Ho, T. A., Martin, K. R., McNaught, A. I., ... Zeyen, T. G. (2018). Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study. In Ophthalmology (Ophthalmology). Elsevier Inc.. https://doi.org/10.1016/j.ophtha.2018.09.034

@inbook{8b4f950503614e1b930fffd17758bf1f,

title = "Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study",

abstract = "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.",

author = "Lee Jones and Garway-Heath, {David F.} and Augusto Azuara-Blanco and Crabb, {David P.} and Garway-Heath, {David F.} and Crabb, {David P.} and Catey Bunce and Gerassimos Lascaratos and Francesca Amalfitano and Nitin Anand and Augusto Azuara-Blanco and Bourne, {Rupert R.} and Broadway, {David C.} and Cunliffe, {Ian A.} and Diamond, {Jeremy P.} and Fraser, {Scott G.} and Ho, {Tuan A.} and Martin, {Keith R.} and McNaught, {Andrew I.} and Anil Negi and Krishna Patel and Russell, {Richard A.} and Ameet Shah and Spry, {Paul G.} and Katsuyoshi Suzuki and White, {Edward T.} and Wormald, {Richard P.} and Wen Xing and Zeyen, {Thierry G.}",

year = "2018",

month = sep,

day = "28",

doi = "10.1016/j.ophtha.2018.09.034",

language = "English",

isbn = "0039-128X (Print)\r0039-128X (Linking)",

series = "Ophthalmology",

publisher = "Elsevier Inc.",

booktitle = "Ophthalmology",

address = "United States",

}

Jones, L, Garway-Heath, DF, Azuara-Blanco, A, Crabb, DP, Garway-Heath, DF, Crabb, DP, Bunce, C, Lascaratos, G, Amalfitano, F, Anand, N, Azuara-Blanco, A, Bourne, RR, Broadway, DC, Cunliffe, IA, Diamond, JP, Fraser, SG, Ho, TA, Martin, KR, McNaught, AI, Negi, A, Patel, K, Russell, RA, Shah, A, Spry, PG, Suzuki, K, White, ET, Wormald, RP, Xing, W & Zeyen, TG 2018, Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study. in Ophthalmology. Ophthalmology, Elsevier Inc. https://doi.org/10.1016/j.ophtha.2018.09.034

Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study. / Jones, Lee; Garway-Heath, David F.; Azuara-Blanco, Augusto et al.
Ophthalmology. Elsevier Inc., 2018. (Ophthalmology).

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

TY - CHAP

T1 - Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study

AU - Jones, Lee

AU - Garway-Heath, David F.

AU - Azuara-Blanco, Augusto

AU - Crabb, David P.

AU - Garway-Heath, David F.

AU - Crabb, David P.

AU - Bunce, Catey

AU - Lascaratos, Gerassimos

AU - Amalfitano, Francesca

AU - Anand, Nitin

AU - Azuara-Blanco, Augusto

AU - Bourne, Rupert R.

AU - Broadway, David C.

AU - Cunliffe, Ian A.

AU - Diamond, Jeremy P.

AU - Fraser, Scott G.

AU - Ho, Tuan A.

AU - Martin, Keith R.

AU - McNaught, Andrew I.

AU - Negi, Anil

AU - Patel, Krishna

AU - Russell, Richard A.

AU - Shah, Ameet

AU - Spry, Paul G.

AU - Suzuki, Katsuyoshi

AU - White, Edward T.

AU - Wormald, Richard P.

AU - Xing, Wen

AU - Zeyen, Thierry G.

PY - 2018/9/28

Y1 - 2018/9/28

N2 - 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.

AB - 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.

U2 - 10.1016/j.ophtha.2018.09.034

DO - 10.1016/j.ophtha.2018.09.034

M3 - Chapter

C2 - 18280526

SN - 0039-128X (Print)\r0039-128X (Linking)

T3 - Ophthalmology

BT - Ophthalmology

PB - Elsevier Inc.

ER -

Are Patient Self-Reported Outcome Measures Sensitive Enough to Be Used as End Points in Clinical Trials?: Evidence from the United Kingdom Glaucoma Treatment Study

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