Development and validation of a patient-reported outcome measure for stroke patients

Health and Quality of Life Outcomes

Yanbo Zhang¹  Jie Yang¹  Yanhong Luo¹ 

[1] Department of Health Statistics, School of Public Health, Shanxi Medical University, 56 South XinJian Road, Taiyuan 030001, Shanxi Province, People’s Republic of China

关键词: Measurement;    Validity;    Reliability;    Confirmatory factor analysis;    Classical test theory;    Item response theory;    Patient-reported outcome;    Stroke;   

Others  :  1207698
DOI  :  10.1186/s12955-015-0246-0

 received in 2014-11-22, accepted in 2015-04-17,  发布年份 2015

【 摘 要 】

Background

Family support and patient satisfaction with treatment are crucial for aiding in the recovery from stroke. However, current validated stroke-specific questionnaires may not adequately capture the impact of these two variables on patients undergoing clinical trials of new drugs. Therefore, the aim of this study was to develop and evaluate a new stroke patient-reported outcome measure (Stroke-PROM) instrument for capturing more comprehensive effects of stroke on patients participating in clinical trials of new drugs.

Methods

A conceptual framework and a pool of items for the preliminary Stroke-PROM were generated by consulting the relevant literature and other questionnaires created in China and other countries, and interviewing 20 patients and 4 experts to ensure that all germane parameters were included. During the first item-selection phase, classical test theory and item response theory were applied to an initial scale completed by 133 patients with stroke. During the item-revaluation phase, classical test theory and item response theory were used again, this time with 475 patients with stroke and 104 healthy participants. During the scale assessment phase, confirmatory factor analysis was applied to the final scale of the Stroke-PROM using the same study population as in the second item-selection phase. Reliability, validity, responsiveness and feasibility of the final scale were tested.

Results

The final scale of Stroke-PROM contained 46 items describing four domains (physiology, psychology, society and treatment). These four domains were subdivided into 10 subdomains. Cronbach’s α coefficients for the four domains ranged from 0.861 to 0.908. Confirmatory factor analysis supported the validity of the final scale, and the model fit index satisfied the criterion. Differences in the Stroke-PROM mean scores were significant between patients with stroke and healthy participants in nine subdomains (P < 0.001), indicating that the scale showed good responsiveness.

Conclusions

The Stroke-PROM is a patient-reported outcome multidimensional questionnaire developed especially for clinical trials of new drugs and is focused on issues of family support and patient satisfaction with treatment. Extensive data analyses supported the validity, reliability and responsiveness of the Stroke-PROM.

【 授权许可】

   
2015 Luo et al.; licensee BioMed Central.

【 图 表 】

Figure 1.

Figure 2.

【 参考文献 】

• [1]Kaste M. Every day is a world stroke day act now, be a stroke champion and a torchbearer. Stroke. 2010; 41:2449-50.
• [2]Markku K. World stroke day. Stroke. 2011; 42:2715.
• [3]Claiborne Johnston S, Shanthi M, Mathers CD. Global variation in stroke burden and mortality: estimates from monitoring, surveillance, and modeling. Lancet Neurol. 2009; 8:345-54.
• [4]The secondary prevention guidelines of Chinese ischemic stroke and transient ischemic attack. Chin J Neurol. 2010; 43(2):154-60.
• [5]Jones P, Harding G, Wiklund I, Berry P, Leidy N. Improving the process and outcome of care in COPD:development of a standardised assessment tool. Prim Care Respir J. 2009; 18(3):208-15.
• [6]Rinu Susan R, Sarma PS, Pandian JD. Psychosocial problems, quality of life, and functional independence among Indian stroke survivors. Stroke. 2010; 41:2932-7.
• [7]Björn S, Mika N, Per-Olof E, Paul H, Eva Wikström J. Validation of the clinical COPD questionnaire (CCQ) in primary care. Health Qual Life Outcomes. 2009; 7:26. BioMed Central Full Text
• [8]U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH): Guidance for Industry, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Clinical/Medical 2009. [http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm]
• [9]Deshpande PR, Surulivel R, Lakshmi Sudeepthi B, Abdul Nazir CP. Patient-reported outcomes: a new era in clinical research. Perspect Clin Res. 2011; 2(4):136-44.
• [10]Reda AA, Daniel K, Janwillem WH K, Geertjan W, Constant P, van Schayck N. Reliability and validity of the clinical COPD questionniare and chronic respiratory questionnaire. Respir Med. 2010; 104:1675-82.
• [11]Windisch W, Budweiser S, Heinemann F, Pfeiferb M, Rzehak P. The severe respiratory insufficiency questionnaire was valid for COPD patients with severe chronic respiratory failure. J Clin Epidemiol. 2008; 61:848-53.
• [12]The Patient-Reported Outcome and Quality of life Instruments Database.[http://www.proqolid.org/]
• [13]Buck D, Jacoby A, Massey A, Steen N, Sharma A, Ford GA. Development and validation of NEWSQOL, the Newcastle stroke-specific quality of life measure. Cerebrovasc Dis. 2004; 17:143-52.
• [14]Hilari K, Byng S, Lamping DL, Smith SC. Stroke and aphasia quality of life scale-39 (SAQOL-39) evaluation of acceptability, reliability, and validity. Stroke. 2003; 34:1944-50.
• [15]Duncan PW, Dennis W, Sue Min L, Dallas J, Susan E, Louise Jacobs L. The stroke impact scale version 2.0: evaluation of reliability, validity, and sensitivity to change. Stroke. 1999; 30:2131-40.
• [16]Xiaoxv Y, Xiaochen T, Yeqing T, Rui Y, Yunxia W, Shiyi C et al.. Development and validation of a tuberculosis medication adherence scale. Plos One. 2012; 7(12):e50328.
• [17]Richieri R, Boyer L, Reine G, Loundou A, Auquier P, Lançon C et al.. The schizophrenia caregiver quality of life questionnaire (S-CGQoL): development and validation of an instrument to measure quality of life of caregivers of individuals with schizophrenia. Schizophr Res. 2011; 126:192-201.
• [18]Polit DF, Cheryl Tatano B, Owen SV. Is the CVI an acceptable indicator of content validity? appraisal and recommendations. Res Nurs Health. 2007; 30:459-67.
• [19]Wynd CA, Bruce S, Michelle Atkins S. Two quantitative approaches for estimating content validity. West J Nurs Res. 2003; 25(5):508-18.
• [20]Yusoff MSB. The Dundee ready educational environment measure: a confirmatory factor analysis in a sample of Malaysian medical students. Int J Humanities Social Sci. 2012; 2(16):313-21.
• [21]Steven DM. Reliability: on the reproducibility of assessment data. Metric Med Educ. 2004; 38:1006-12.
• [22]Muhamad Saiful Bahri Yusoff, Ahmad Fuad Abdul Rahim, Mohd Jamil Yaacob: The development and validity of the medical student stressor questionnaire(MSSQ). ASEAN Journal of Psychiatry 2010, 11 (1): Jan – June 2010: XX XX.
• [23]tot van Nispen Pannerden SC, Candel MJJM, Zwakhalen SMG, Hamers JPH, Curfs LMG, Berger MPF. An item response theory-based assessment of the pain assessment checklist for seniors with limited ability to communicate (PACSLAC). J Pain. 2009; 10(8):844-53.
• [24]Guidance for industry patient-reported outcome measures: Use in medical product development to support labeling claims. Clin/Med. 2009; 12:1-39.
• [25]van der Molen T, Brigitte WM W, Siebrig S, ten Hacken NHT, Postma DS, Juniper EF. Development, validity and responsiveness of the Clinical COPD Questionnaire. Health Qual Life Outcomes. 2003; 1:13. BioMed Central Full Text
• [26]van Swieten JC, Koudstaal PJ, Visser MC, Schouten HJ, van Gijn J. Interobserver agreement for the assessment of handicap in stroke patients. Stroke. 1988; 19:604-7.
• [27]Lyden PD, Mei L, Levine SR, Brott TG, Broderick J. A modified national institutes of health stroke scale for use in stroke clinical trials. Stroke. 2001; 32:1310-7.
• [28]Cote R, Hachinski VC, Shurvell BL, Norris JW, Wolfson C. The Canadian neurological scale: a preliminary study in acute stroke. Stroke. 1986; 17:731-7.
• [29]Williams LS, Weinberger M, Harris LE, Clark DO, Biller J´. Development of a stroke-specific quality of life scale. Stroke. 1999; 30:1362-9.
• [30]Wang Y, Zhao H, Liu Z, Liu B. Reliability, validity and response of patient reported outcome scale in stroke patients with spastic paralysis. Chinese General Practice. 2009; 12:1168-70.
• [31]Buck D, Jacoby A, Massey A, Ford G. Evaluation of measures used to assess quality of life after stroke. Stroke. 2000; 31:2004-10.
• [32]Watanabe Y, Araki S, Kurihara M. Health-related quality of life of stroke patients’ families during the patients’ hospitalization: a pilot study in Japan. Int J Rehabil Res. 2003; 26(1):43-5.
• [33]Palmer S, Glass TA. Family function and stroke recovery: a review. Rehabil Psychol. 2003; 48(4):255-65.
• [34]Cameron JI, Gary N, Gignac MAM, Mark B, Grace W, Theresa G et al.. Randomized clinical trial of the timing it right stroke family support program: research protocol. Health Serv Res. 2014; 14:18. BioMed Central Full Text
• [35]Clark MS, Sally R, Adrian W. A randomized controlled trial of an education and counselling intervention for families after stroke. Clin Rehabil. 2003; 17:703-12.
• [36]Clark PC, Dunbar SB, Shields CG, Viswanathan B, Aycock DM, Wolf SL. Influence of stroke survivor characteristics and family conflict surrounding recovery on Caregivers’ mental and physical health. Nurs Res. 2004; 53(6):406-13.
• [37]Lawrence M, Kinn S. Needs, priorities, and desired rehabilitation outcomes of family members of young adults who have had a stroke: findings from a phenomenological study. Disabil Rehabil. 2013; 35(7):586-95.
• [38]Anne V-M, Marcel P, Jan Willem G, Berlekom SBV, Trudi Van Den B, Eline L. Rehabilitation of stroke patients needs a family-centred approach. Disabil Rehabil. 2006; 28(24):1557-61.
• [39]Han B, Haley WE. Family caregiving for patients with stroke review and analysis. Stroke. 1999; 30:1478-85.
• [40]Francesco M, Harin Padma N, Andrew M, Brock GB, Gregory B, Sanjeev A et al.. Tadalafil in the treatment of erectile dysfunction following bilateral nerve sparing radical retropubic prostatectomy: a randomized, double-blind, placebo controlled trial. J Urol. 2004; 172:1036-41.
• [41]Dworkin RH, Turk DC, Wyrwich KW, Beaton D, Cleeland CS, Farrar JT et al.. Interpreting the clinical importance of treatment outcomes in chronic pain clinical trials: IMMPACT recommendations. J Pain. 2008; 9(2):105-21.
• [42]Burgio KL, Locher JL, Goode PS, Michael Hardin J, Joan McDowell B, Marianne D et al.. Behavioral vs drug treatment for urge urinary incontinence in older women a randomized controlled trial. JAMA. 1998; 280(23):1995.
• [43]Linda A, Krithika R, Polyxane M, Carolyn B, Rod B, Robin C. Development and validation of the Impact of Dry Eye on Everyday Life (IDEEL) questionnaire, a patient-reported outcomes (PRO) measure for the assessment of the burden of dry eye on patients. Health Qual Life Outcomes. 2011; 9:111. BioMed Central Full Text
• [44]Hagman BT, Kuerbis AN, Morgenstern J, Bux DA, Parsons JT, Heidinger BE. An item response theory (IRT) analysis of the short inventory of problems-alcohol and drugs (SIP-AD) among non-treatment seeking men-who-have-sex-with-men: evidence for a shortened 10-item SIP-AD. Addict Behav. 2009; 34:948-54.