以质量求发展,以服务铸品牌

护理学报 ›› 2024, Vol. 31 ›› Issue (18): 35-38.doi: 10.16460/j.issn1008-9969.2024.18.035

• 循证护理 • 上一篇    下一篇

临床研究中基于患者报告结局的缺失值问题及对策

史晨1, 闫泽霖1, 马嘉骏1, 李新旭2, 欧春泉1   

  1. 1.南方医科大学 公共卫生学院生物统计学系,广东 广州 510515;
    2.博济医药科技股份有限公司 博济数据科技(北京)有限公司,北京 100077
  • 收稿日期:2024-06-15 出版日期:2024-09-25 发布日期:2024-10-08
  • 通讯作者: 欧春泉(1974-),女,湖南宁乡人,博士,教授,博士研究生导师。E-mail: ouchunquan@hotmail.com
  • 作者简介:史晨(1998-),女,安徽阜阳人,本科学历,博士研究生在读。
  • 基金资助:
    广东省基础与应用基础研究基金(2021A1515220065)

Missing data in patient-reported outcomes in clinical studies and its solutions

SHI Chen1, YAN Ze-lin1, MA Jia-jun1, LI Xin-xu2, OU Chun-quan1   

  1. 1. Department of Biostatistics, School of Public Health, Southern Medical University, Guangzhou 510515, China;
    2. Boji Medical Technology Co., Ltd, Boji Data Technology (Beijing) Ltd, Beijing 100077, China
  • Received:2024-06-15 Online:2024-09-25 Published:2024-10-08

摘要: 目的 整合患者报告结局(patient-reported outcomes, PRO)的缺失值问题及对策,为尽可能降低PRO数据缺失所带来的影响提供解决思路和参考。方法 检索国内外相关文献,从数据缺失的原因及其影响、可行的预防措施、对缺失值的处理及规范报道等几个方面归纳PRO的缺失值问题。结果 对缺失数据产生原因及其严重影响的充分认识,严谨的研究设计,规范的实施流程等可降低PRO数据缺失的风险;对无法避免的缺失数据进行合理处理和分析,并透明报告,也能得出可信的结论。结论 随着科学研究的不断发展,PRO数据应被更合理、更有效地利用。

关键词: 患者报告结局, 缺失值, 量表, 临床研究

Abstract: Objective To explore missing data in patient-reported outcomes (PRO) and the solutions, and to provide references for reducing the impact of missing data in PRO as much as possible. Methods Relevant literatures at home and abroad were searched, and missing data in PRO were summarized from the perspective of causes and influences of data missing, feasible preventive measures, handling of missing data, and standardized reporting. Results Adequate understanding of the causes and serious impacts of missing data, rigorous study design, and standardized implementation procedures can reduce the risk of data missing in PRO. Reasonable processing and analysis of unavoidable missing data and transparent reporting were beneficial to drawing credible conclusions. Conclusion With the continuous development of scientific research, PRO data should be used more rationally and effectively.

Key words: patient-reported outcomes, missing data, scale, clinical study

中图分类号: 

  • R47
[1] 国家药品监督管理局药品审评中心.患者报告结局在药物临床研发中应用的指导原则(试行)[EB/OL].[2024-03-25].https://www.cde.org.cn/zdyz/domesticinfopage?zdyzIdCODE=8a900c1173e7aaf72ea3724796cebbeb.
[2] 周慧, 姚弥, 陈杰, 等. 《临床试验方案纳入患者报告结局的指南-扩展声明》解读[J]. 中国新药杂志, 2021,30(14):1296-1301.
[3] Palmer MJ, Mercieca-Bebber R, King M, et al.A systematic review and development of a classification framework for factors associated with missing patient-reported outcome data[J]. Clin Trials,2018,15(1):95-106.DOI:10.1177/1740774517741113.
[4] Rombach I,Gray AM, Jenkinson C, et al.Multiple imputation for patient reported outcome measures in randomised controlled trials: advantages and disadvantages of imputing at the item, subscale or composite score level[J]. BMC Med Res Methodol,2018,18(1):87.DOI:10.1186/s12874-018-0542-6.
[5] 念家云. 疏肝健脾颗粒治疗乳腺癌癌因性疲乏的临床研究[D]. 北京:北京中医药大学, 2017.
[6] 李平. 探讨甲亢患者人格特征、中医体质关系并观察其饮食护理的效果[D]. 哈尔滨:黑龙江中医药大学, 2022.DOI:10.27127/d.cnki.ghlzu.2021.000474.
[7] Carpenter JR, Smuk M.Missing data: a statistical framework for practice[J]. Biom J, 2021,63(5):915-947.DOI:10.1002/bimj.202000196.
[8] Little RJ, D’Agostino R, Cohen ML, et al. The prevention and treatment of missing data in clinical trials[J]. N Engl J Med,2012,367(14):1355-1360.DOI:10.1056/NEJMsr1203730.
[9] Stang A.Nonresponse research--an underdeveloped field in epidemiology[J]. Eur J Epidemiol, 2003,18(10):929-931.DOI:10.1023/a:1025877501423.
[10] Zhao Y.Statistical inference for missing data mechanisms[J]. Stat Med, 2020,39(28):4325-4333.DOI:10.1002/sim.8727.
[11] Mercieca-Bebber R, Palmer MJ, Brundage M, et al.Design, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic review[J]. BMJ Open, 2016,6(6):e10938.DOI:10.1136/bmjopen-2015-010938.
[12] Bell ML, Floden L, Rabe BA, et al.Analytical approaches and estimands to take account of missing patient-reported data in longitudinal studies[J]. Patient Relat Outcome Meas, 2019(10):129-140.DOI:10.2147/PROM.S178963.
[13] Bell ML, Fairclough DL.Practical and statistical issues in missing data for longitudinal patient-reported outcomes[J]. Stat Methods Med Res, 2014,23(5):440-459.DOI:10.1177/0962280213476378.
[14] Li T, Hutfless S, Scharfstein DO, et al.Standards should be applied in the prevention and handling of missing data for patient-centered outcomes research: a systematic review and expert consensus[J]. J Clin Epidemiol, 2014,67(1):15-32.DOI:10.1016/j.jclinepi.2013.08.013.
[15] Wehrlen L, Krumlauf M, Ness E, et al.Systematic collection of patient reported outcome research data: a checklist for clinical research professionals[J]. Contemp Clin Trials, 2016(48):21-29.DOI:10.1016/j.cct.2016.03.005.
[16] Crossnohere NL, Anderson N, Baumhauer J, et al.A framework for implementing patient-reported outcomes in clinical care:the PROTEUS-practice guide[J].Nature Medicine, 2024,30(6):1519-1520.DOI:10.1038/s41591-024-02909-8.
[17] Peyre H, Leplège A,Coste J.Missing data methods for dealing with missing items in quality of life questionnaires. A comparison by simulation of personal mean score, full information maximum likelihood, multiple imputation, and hot deck techniques applied to the SF-36 in the French 2003 decennial health survey[J]. Qual Life Res, 2011,20(2):287-300.DOI:10.1007/s11136-010-9740-3.
[18] Blough DK, Ramsey S, Sullivan SD, et al.The impact of using different imputation methods for missing quality of life scores on the estimation of the cost-effectiveness of lung-volume-reduction surgery[J]. Health Econ, 2009,18(1):91-101.DOI:10.1002/hec.1347.
[19] Eekhout I, de Vet HC, Twisk JW, et al. Missing data in a multi-item instrument were best handled by multiple imputation at the item score level[J]. J Clin Epidemiol, 2014,67(3):335-342.DOI:10.1016/j.jclinepi.2013.09.009.
[20] Simons CL, Rivero-Arias O, Yu LM, et al.Multiple imputation to deal with missing EQ-5D-3L data: should we impute individual domains or the actual index?[J]. Qual Life Res,2015,24(4):805-815.DOI:10.1007/s11136-014-0837-y.
[21] Fielding S, Fayers P, Ramsay CR.Analysing randomised controlled trials with missing data: choice of approach affects conclusions[J]. Contemp Clin Trials, 2012,33(3):461-469.DOI:10.1016/j.cct.2011.12.002.
[22] Little RJ, Rubin DB.Statistical analysis with missing data[M]. Hoboken: John Wiley & Sons, 2019.
[23] Donneau AF, Mauer M, Lambert P, et al.Simulation-based study comparing multiple imputation methods for non-monotone missing ordinal data in longitudinal settings[J]. J Biopharm Stat,2015,25(3):570-601.DOI:10.1080/10543406.2014.920864.
[24] Hughes RA, Heron J, Sterne J, et al.Accounting for missing data in statistical analyses: multiple imputation is not always the answer[J]. Int J Epidemiol, 2019,48(4):1294-1304.DOI:10.1093/ije/dyz032.
[25] Kenward MG, Lesaffre E, Molenberghs G.An application of maximum likelihood and generalized estimating equations to the analysis of ordinal data from a longitudinal study with cases missing at random[J]. Biometrics, 1994,50(4):945-953.
[26] Doidge JC.Responsiveness-informed multiple imputation and inverse probability-weighting in cohort studies with missing data that are non-monotone or not missing at random[J]. Stat Methods Med Res, 2018,27(2):352-363.DOI:10.1177/0962280216628902.
[27] Molenberghs G, Thijs H,Jansen I,et al.Analyzing incomplete longitudinal clinical trial data[J]. Biostatistics, 2004,5(3):445-464.DOI:10.1093/biostatistics/5.3.445.
[28] Khan T, Khalid M, Dunford B, et al.Incomplete reporting of patient-reported outcomes in multiple sclerosis: a meta-epidemiological study of randomized controlled trials[J]. Mult Scler Relat Disord, 2022(63):103819. DOI:10.1016/j.msard.2022.103819.
[29] Fayers PM, Machin D.Quality of life: the assessment, analysis and interpretation of patient-reported outcomes[M]. Hoboken: John wiley & sons, 2013.
[30] Calvert M, Blazeby J, Altman DG, et al.Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension[J]. JAMA, 2013,309(8):814-822.DOI:10.1001/jama.2013.879.
[31] Friedlander M, Mercieca-Bebber RL, King MT.Patient-reported outcomes (PRO) in ovarian cancer clinical trials-lost opportunities and lessons learned[J]. Ann Oncol, 2016,27(Suppl 1):i66-i71.DOI:10.1093/annonc/mdw080.
[32] Krepper D, Giesinger JM, Dirven L, et al.Information about missing patient-reported outcome data in breast cancer trials is frequently not documented: a scoping review[J]. J Clin Epidemiol, 2023(162):1-9.DOI:https://doi.org/10.1016/j.jclinepi.2023.07.012.
[1] 丁心舒, 孙乐菲, 高伟, 鲁琦, 闫畅, 刘德山. 矛盾年龄歧视量表在社区老年人中的信效度检验[J]. 护理学报, 2024, 31(7): 12-16.
[2] 张国春, 李海云, 褚梁梁, 程雪, 王君芝. 产科护士围产期姑息照护知信行量表的编制及信效度检验[J]. 护理学报, 2024, 31(2): 6-11.
[3] 王晓宁, 缪群芳, 李培清, 辛思莹, 黄姚姚. 自杀公众污名量表的汉化及在中国公众人群中的信效度检验[J]. 护理学报, 2024, 31(17): 1-7.
[4] 朱亚晋, 王朝辉, 代明珠, 王琳, 孙莹莹. 临床护士文化能力量表的汉化及信效度检验[J]. 护理学报, 2024, 31(17): 46-50.
[5] . 静脉血栓栓塞症机械预防护理实践评价量表的研制与信效度检验[J]. 护理学报, 2024, 31(12): 7-12.
[6] 官美君, 田黇, 彭继蓉, 苏小秋. 14种HPV疫苗犹豫量表的评价分析[J]. 护理学报, 2023, 30(20): 31-36.
[7] 张轶, 王艾红, 陈静. 中文版宿命观量表在恶性血液病患者中的信效度检验[J]. 护理学报, 2023, 30(20): 54-57.
[8] 张馥丽, 王丹妮, 叶红, 蔡姣芝. 胃癌患者报告结局评估工具的研究进展[J]. 护理学报, 2023, 30(19): 41-44.
[9] 华小倩, 黎巧玲, 党兆, 胡宏美, 李春莉, 皮晨曦. 脑卒中照顾者对卒中复发应急能力测评量表的构建[J]. 护理学报, 2023, 30(19): 62-67.
[10] 侯爱琳, 宁宁, 陈佳丽, 李佩芳, 张林, 谢艳, 朱红彦, 屈俊宏. 膝关节骨性关节炎患者日常生活困难评估量表的汉化及信效度检验[J]. 护理学报, 2023, 30(18): 6-10.
[11] 花芸, 王雯沁, 刘志梅, 冯国和, 杜沫晗. 癌症患者PICC置管支持性照护需求量表的编制及信效度检验[J]. 护理学报, 2023, 30(15): 23-28.
[12] 张颖, 刘雪琴, 刘晓辉, 聂亚杰, 章晓菲, 赵洁, 代楠, 王肖阳. 血液透析疲乏患者报告结局评估工具临床应用的研究进展[J]. 护理学报, 2023, 30(13): 38-41.
[13] 雷丹, 卞薇. 角膜疾病患者报告结局评估工具的研究进展[J]. 护理学报, 2023, 30(12): 40-44.
[14] 许汇娟, 刘颖, 姚嘉丽, 姚晶晶, 陈敏, 邱甜甜, 徐丹. 3种量表预测急性髓系白血病患者有症状性PICC相关血栓形成风险效果的比较[J]. 护理学报, 2023, 30(11): 59-64.
[15] 张佳惠, 庞书勤, 洪雪珮, 郑智慧, 李绵利, 黄钰峰, 王炜骏. 老年前期人群退休规划过程量表的汉化及信效度检验[J]. 护理学报, 2023, 30(10): 1-6.
Viewed
Full text


Abstract

Cited

  Shared   
No Suggested Reading articles found!