护理学报 ›› 2024, Vol. 31 ›› Issue (18): 35-38.doi: 10.16460/j.issn1008-9969.2024.18.035
史晨1, 闫泽霖1, 马嘉骏1, 李新旭2, 欧春泉1
SHI Chen1, YAN Ze-lin1, MA Jia-jun1, LI Xin-xu2, OU Chun-quan1
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。
中图分类号:
[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] | 罗铎麟, 杨春清, 高萍, 胡清文, 徐小群. 2型糖尿病心理痛苦量表的汉化及信效度检验[J]. 护理学报, 2024, 31(19): 24-30. |
[4] | 张亦然, 王康美, 朱盛财, 朱璐, 黄沂. 中文版高血压患者自我管理评估工具的系统评价:基于COSMIN指南[J]. 护理学报, 2024, 31(19): 52-57. |
[5] | 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. |
[6] | 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. |
[7] | . 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. |
[8] | 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. |
[9] | 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. |
[10] | 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. |
[11] | 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. |
[12] | 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. |
[13] | 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. |
[14] | 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. |
[15] | 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. |
|