Consideration on the study design of clinical research on diabetic retinopathy: from randomized controlled trial to real world study_Chinese Journal of Ocular Fundus Diseases

Authors：

 Sun Xiaodong , Jia Huixun

Department of Ophthalmology, Shanghai General Hospital, Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China;

Corresponding author：

Sun Xiaodong, Email: xdsun@sjtu.edu.cn

Keywords：

Diabetic retinopathy; Randomized controlled trial; Propensity score; Editorial; Real-world study

DOI：

10.3760/cma.j.issn.1005-1015.2019.02.001

Video：

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Diabetic retinopathy (DR), which is a common complication of diabetic and the main cause of blindness, brings not only a heavy economic burden to society, but also seriously threatens to the patients’ quality of life. Clinical researches on the therapies of DR are active at present, but how to perform a good clinical research with scientific design should be considered with high priority. The randomized controlled trial (RCT) is considered to be the gold standard for evidence-based medicine, but RCT is not always perfect. Limitations still exist in certain circumstance and the conclusions from RCTs also need to be interpreted by an objective point of view before clinical practice. Real world study (RWS) bridges the gap between RCT and clinical practice, in which the data can be easily collected without much cost, and results might be obtained within a short period. However, RWS is also faced with the challenge of not having standardized data and being susceptible to confounding bias. The standardized single disease database for DR and propensity score matching method can provide a wide range of data sources and avoid of bias for RWS in DR.

Citation： Sun Xiaodong, Jia Huixun. Consideration on the study design of clinical research on diabetic retinopathy: from randomized controlled trial to real world study. Chinese Journal of Ocular Fundus Diseases, 2019, 35(2): 111-114. doi: 10.3760/cma.j.issn.1005-1015.2019.02.001 Copy

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12.	李筱荣, 刘巨平. 认识糖尿病视网膜病变临床研究热点难点, 探索优化未来临床研究方向[J]. 中华眼底病杂志, 2014, 30(2): 121-123. DOI: 10.3760/cma.j.issn.1005-1015.2014.02.001.Li XR, Liu JP. Hot topics and unresolved issues in clinical researches of diabetic retinopathy[J]. Chin J Ocul Fundus Dis, 2014, 30(2): 121-123. DOI: 10.3760/cma.j.issn.1005-1015.2014.02.001.
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16.	D'Agostino RB. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group[J]. Stat Med, 1998, 17(19): 2265-2281. DOI: 10.1002/(ISSN)1097-0258.
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1. American Diabetes Association.10 Microvascular complications and foot care: standards of medical care in diabetes--2018[J]. Diabetes Care, 2018, 41(Suppl 1): S105-118. DOI: 10.2337/dc15-S001.
2. King H, Aubert RE, Herman WH. Global burden of diabetes, 1995-2025: prevalence, numerical estimates, and projections[J]. Diabetes Care, 1998, 21(9): 1414-1431. DOI: 10.2337/diacare.21.9.1414.
3. 国务院. " 十三五”国家科技创新规划[EB/OL]. [2016-07-28]. http://www.most.gov.cn/mostinfo/xinxifenlei/gjkjgh/201608/t20160810_127174.htm.State Council. The 13th five-year plan for national science and technology innovation[EB/OL]. [2016-07-28]. http://www.most.gov.cn/mostinfo/xinxifenlei/gjkjgh/201608/t20160810_127174.htm.
4. 李文生, 黎晓新. 努力学习和实践循证医学, 进一步提高我国常见眼底病的诊疗水平[J]. 中华眼底病杂志, 2010, 26(5): 401-403. DOI: 10.3760/cma.j.issn.1005-1015.2010.05.01.Li WS, Li XX. The study and practice of evidence-based medicine: improving diagnosis and treatment of common ocular fundus diseases in China[J]. Chin J Ocul Fundus Dis, 2010, 26(5): 401-403. DOI: 10.3760/cma.j.issn.1005-1015.2010.05.01.
5. Mitchell P, Bandello F, Schmidt-Erfurth U, et al. The RESTORE study: ranibizumab monotherapy or combined with laser versus laser monotherapy for diabetic macular edema[J]. Ophthalmology, 2011, 118(4): 615-625. DOI: 10.1016/j.ophtha.2011.01.031.
6. Schmidt-Erfurth U, Lang GE, Holz FG. Three-year outcomes of individualized ranibizumab treatment in patients with diabetic macular edema: the RESTORE extension study[J]. Ophthalmology, 2014, 121(5): 1045-1053. DOI: 10.1016/j.ophtha.2013.11.041.
7. Do DV, Nguyen QD, Boyer D, et al. One-year outcomes of the da Vinci Study of VEGF Trap-Eye in eyes with the diabetic macular edema[J]. Ophthalmology, 2012, 119(8): 1658-1665. DOI: 10.1016/j.ophtha.2012.02.010.
8. Diabetic Retinopathy Clinical Research Network, Wells JA, Glassman AR, et al. Aflibercept, bevacizumab, or ranibizumab for diabetic macular edema[J]. N Engl J Med, 2015, 372(13): 1193-1203. DOI: 10.1056/NEJMoa1414264.
9. Wells JA, Glassman AR, Ayala AR, et al. Aflibercept, bevacizumab, or ranibizumab for diabetic macular edema: two-year results from a comparative effectiveness randomizied clinical trail[J]. Ophthalmology, 2016, 123(6): 1351-1359. DOI: 10.1016/j.ophtha.2016.02.022.
10. Diabetic Retinopathy Clinical Research Network, Elman MJ, Aiello LP, et al. Randomized trial evaluating ranibizumab plus prompt or deferred laser or triamcinolone plus prompt laser for diabetic macular edema[J]. Ophthalmology, 2010, 117(6): 1064-1077. DOI: 10.1016/j.ophtha.2010.02.031.
11. Nunome T, Sugimoto M, Kondo M, et al. Short-term results of intravitreal triamcinolone acetonide combined with cataract surgery for diabetic macular edema in Japan: in the era of anti-vascular endothelial growth factor therapy[J]. Ophthalmologica, 2018, 240(2): 73-80. DOI: 10.1159/000487548.
12. 李筱荣, 刘巨平. 认识糖尿病视网膜病变临床研究热点难点, 探索优化未来临床研究方向[J]. 中华眼底病杂志, 2014, 30(2): 121-123. DOI: 10.3760/cma.j.issn.1005-1015.2014.02.001.Li XR, Liu JP. Hot topics and unresolved issues in clinical researches of diabetic retinopathy[J]. Chin J Ocul Fundus Dis, 2014, 30(2): 121-123. DOI: 10.3760/cma.j.issn.1005-1015.2014.02.001.
13. Martens EP, Pestman WR, de Boer A, et al. Systematic differences in treatment effect estimates between propensity score methods and logistic regression[J]. Int J Epidemiol, 2008, 37(5): 1142-1147. DOI: 10.1093/ije/dyn079.
14. Cepeda MS, Boston R, Farrar JT, et al. Comparison of logistic regression versus propensity score when the number of events is low and there are multiple confounders[J]. Am J Epidemiol, 2003, 158(3): 280-287. DOI: 10.1093/aje/kwg115.
15. Han JW, Kamber M. Data mining: concept and techniques[M]. Los Angeles: Morgan Kaufmann Publishers, Inc, 2000.
16. D'Agostino RB. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group[J]. Stat Med, 1998, 17(19): 2265-2281. DOI: 10.1002/(ISSN)1097-0258.
17. Austin PC, Grootendorst P, Anderson GM. A comparison of the ability of different propensity score models to balance measured variables between treated and untreated subjects: a Monte Carlo study[J]. Statist Med, 2007, 26(4): 734-753. DOI: 10.1002/sim.2580.
18. Austin PC. The performance of different propensity-score methods for estimating relative risks[J]. J Clin Epidemiol, 2008, 61(6): 537-545. DOI: 10.1016/j.jclinepi.2007.07.011.

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Consideration on the study design of clinical research on diabetic retinopathy: from randomized controlled trial to real world study

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