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The online version of this article (doi:10.1007/s11136-015-1206-1) contains supplementary material, which is available to authorized users.
Patient-reported outcome measures (PROMs) are designed to assess patients’ perceived health states or health-related quality of life. However, PROMs are susceptible to missing data, which can affect the validity of conclusions from randomised controlled trials (RCTs). This review aims to assess current practice in the handling, analysis and reporting of missing PROMs outcome data in RCTs compared to contemporary methodology and guidance.
This structured review of the literature includes RCTs with a minimum of 50 participants per arm. Studies using the EQ-5D-3L, EORTC QLQ-C30, SF-12 and SF-36 were included if published in 2013; those using the less commonly implemented HUI, OHS, OKS and PDQ were included if published between 2009 and 2013.
The review included 237 records (4–76 per relevant PROM). Complete case analysis and single imputation were commonly used in 33 and 15 % of publications, respectively. Multiple imputation was reported for 9 % of the PROMs reviewed. The majority of publications (93 %) failed to describe the assumed missing data mechanism, while low numbers of papers reported methods to minimise missing data (23 %), performed sensitivity analyses (22 %) or discussed the potential influence of missing data on results (16 %).
Considerable discrepancy exists between approved methodology and current practice in handling, analysis and reporting of missing PROMs outcome data in RCTs. Greater awareness is needed for the potential biases introduced by inappropriate handling of missing data, as well as the importance of sensitivity analysis and clear reporting to enable appropriate assessments of treatment effects and conclusions from RCTs.
Supplementary material 1 (DOCX 11 kb)11136_2015_1206_MOESM1_ESM.docx
Reeve, B. B., Wyrwich, K. W., Wu, A. W., Velikova, G., Terwee, C. B., Snyder, C. F., et al. (2013). ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research. Quality of Life Research, 22(8), 1889–1905. CrossRefPubMed
Little, R. J. A., & Rubin, D. B. (1987). Statistical analysis with missing data. New York: Wiley
Li, T., Hutfless, S., Scharfstein, D. O., Daniels, M. J., Hogan, J. W., Little, R. J., et al. (2014). Standards should be applied in the prevention and handling of missing data for patient-centered outcomes research: A systematic review and expert consensus. Journal of Clinical Epidemiology, 67(1), 15–32. CrossRefPubMedPubMedCentral
Peyre, H., Leplege, A., & Coste, J. (2011). 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. Quality of Life Research, 20(2), 287–300. CrossRefPubMed
EuroQol, G. (1990). EuroQol—A new facility for the measurement of health-related quality of life. Health Policy, 16(3), 199–208. CrossRef
Dawson, J., Fitzpatrick, R., Carr, A., & Murray, D. (1996). Questionnaire on the perceptions of patients about total hip replacement. Journal of Bone & Joint Surgery—British, 78(2), 185–190.
Murray, D. W., Fitzpatrick, R., Rogers, K., Pandit, H., Beard, D. J., Carr, A. J., & Dawson, J. (2007). The use of the Oxford hip and knee scores. Journal of Bone and Joint Surgery. British Volume, 89(8), 1010–1014. CrossRef
Dawson, J., Fitzpatrick, R., Murray, D., & Carr, A. (1998). Questionnaire on the perceptions of patients about total knee replacement. Journal of Bone & Joint Surgery—British, 80(1), 63–69. CrossRef
Aaronson, N., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N., et al. (1993). The European Organization for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute, 85(5), 365–376. CrossRefPubMed
Torrance, N., Lawson, K. D., Afolabi, E., Bennett, M. I., Serpell, M. G., Dunn, K. M., & Smith, B. H. (2014). Estimating the burden of disease in chronic pain with and without neuropathic characteristics: does the choice between the EQ-5D and SF-6D matter? Pain, 155(10), 1996–2004. CrossRefPubMedPubMedCentral
Stark, D., Nankivell, M., Pujade-Lauraine, E., Kristensen, G., Elit, L., Stockler, M., et al. (2013). Standard chemotherapy with or without bevacizumab in advanced ovarian cancer: Quality-of-life outcomes from the International Collaboration on Ovarian Neoplasms (ICON7) phase 3 randomised trial. The Lancet Oncology, 14(3), 236–243. CrossRefPubMedPubMedCentral
Nilsdotter, A., & Bremander, A. (2011). Measures of hip function and symptoms: Harris Hip Score (HHS), Hip Disability and Osteoarthritis Outcome Score (HOOS), Oxford Hip Score (OHS), Lequesne Index of Severity for Osteoarthritis of the Hip (LISOH), and American Academy of Orthopedic Surgeons (AAOS) Hip and Knee Questionnaire. Arthritis Care & Research (Hoboken), 63(Suppl 11), S200–S207. CrossRef
Collins, N. J., Misra, D., Felson, D. T., Crossley, K. M., & Roos, E. M. (2011). Measures of knee function: International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Form, Knee Injury and Osteoarthritis Outcome Score (KOOS), Knee Injury and Osteoarthritis Outcome Score Physical Function Short Form (KOOS-PS), Knee Outcome Survey Activities of Daily Living Scale (KOS-ADL), Lysholm Knee Scoring Scale, Oxford Knee Score (OKS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Activity Rating Scale (ARS), and Tegner Activity Score (TAS). Arthritis Care Res (Hoboken), 63(Suppl 11), S208–S228. CrossRef
- The current practice of handling and reporting missing outcome data in eight widely used PROMs in RCT publications: a review of the current literature
Alastair M. Gray
- Springer International Publishing
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