Interpretation of patient-reported outcomes

Statistical Methods in Medical Research

Volumn 23, Issue 5, 2014, Pages 460-483

  Cappelleri, Joseph C ^a   Bushmakin, Andrew G ^a  

^a PFIZER INC   (United States)

Author keywords

anchor based approach; distribution based approach; interpretation; mediation analysis; Patient reported outcome

Indexed keywords

ARTICLE; CORRELATION COEFFICIENT; EFFECT SIZE; FIBROMYALGIA IMPACT QUESTIONNAIRE; HUMAN; MULTICENTER STUDY (TOPIC); OUTCOME ASSESSMENT; PAIN ASSESSMENT; PAIN SEVERITY; PATIENT REPORTED OUTCOME; PROBABILITY; RANDOMIZED CONTROLLED TRIAL (TOPIC); RELIABILITY; SELF REPORT; STATISTICAL ANALYSIS; VALIDITY; ANALOGS AND DERIVATIVES; CLINICAL TRIAL (TOPIC); ERECTILE DYSFUNCTION; FEMALE; FIBROMYALGIA; MACULAR DEGENERATION; MALE; PATHOLOGY; QUESTIONNAIRE; SICKNESS IMPACT PROFILE;

4 AMINOBUTYRIC ACID; ANALGESIC AGENT; PIPERAZINE DERIVATIVE; PREGABALIN; PURINE DERIVATIVE; SILDENAFIL; SULFONAMIDE; URINARY TRACT AGENT;

ANALGESICS; CLINICAL TRIALS AS TOPIC; DATA INTERPRETATION, STATISTICAL; ERECTILE DYSFUNCTION; FEMALE; FIBROMYALGIA; GAMMA-AMINOBUTYRIC ACID; HUMANS; MACULAR DEGENERATION; MALE; PATIENT OUTCOME ASSESSMENT; PIPERAZINES; PURINES; QUESTIONNAIRES; SICKNESS IMPACT PROFILE; SULFONAMIDES; UROLOGICAL AGENTS;

EID: 84887957162     PISSN: 09622802     EISSN: 14770334     Source Type: Journal    
DOI: 10.1177/0962280213476377     Document Type: Article

References (64)

1. US Department of Health and Human Services. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims.http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf (2009, accessed 2 April 2012).
2. Fairclough DL. Patient-reported outcomes as endpoints n medical research. Stat Meth Med Res. 2004 ; 13: 115-138
3. Marquis P, Chassany O, Abetz L. A comprehensive strategy for the interpretation of quality-of-life data based on existing methods. Value Health. 2004 ; 7: 93-104
4. Schünemann HJ, Akl EA, Guyatt GH. Interpreting the results of patient reported outcomes in clinical trials: The clinician's perspective. Health Qual Life Outcome. 2006 ; 4: 62-62
5. Revicki D, Erickson PA, Sloan JA, et al. and the Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Interpreting and reporting results based on patient-reported outcomes. Value Health. 2007 ; 10: S116 - S124
6. Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res. 1993 ; 2: 221-226
7. Fayers FM, Machin D Quality of life: The assessment, analysis and interpretation of patient-reported outcomes. 2 nd edn. Chichester, England: John Wiley & Sons Ltd., 2007. Chichester, England: John Wiley & Sons Ltd. ; 2007 :
8. McLeod LD, Coon CD, Martin SA, et al. Interpreting patient-reported outcome results: US FDA guidance and emerging methods. Expert Rev Pharmacoecon Outcome Res. 2011 ; 11: 163-169
9. De Vet HCW, Terwee CB, Mokkink LB, et al Measurement in medicine. Cambridge, United Kingdom: Cambridge University Press ; 2011 :
10. Iacobucci D Mediation analysis. Thousands Oaks, CA: Sage Publications ; 2008 :
11. MacKinnon DP Introduction to statistical mediation analysis. New York, NY: Taylor & Francis Group ; 2008 :
12. Bennett RM, Bushmakin AG, Cappelleri JC, et al. Minimally clinically important difference in the Fibromyalgia Impact Questionnaire (FIQ). J Rheumatol. 2009 ; 36: 1304-1311
13. Cappelleri JC, Bushmakin AG, McDermott A, et al. Measurement properties of the Medical Outcomes Study Sleep Scale in patients with fibromyalgia. Sleep Med. 2009 ; 10: 766-770
14. Russell IJ, Crofford LJ, Leon T, et al. The effects of pregabalin on sleep disturbance symptoms among individuals with fibromyalgia syndrome. Sleep Med. 2009 ; 10: 604-610
15. Cappelleri JC, Bell SS, Althof SE, et al. Comparison between sildenafil-treated subjects with erectile dysfunction and control subjects on the Self-Esteem And Relationship questionnaire. J Sexual Med. 2006 ; 3: 274-282
16. Althof SE, Cappelleri JC, Shpilsky A, et al. Treatment responsiveness of the Self-Esteem And Relationship (SEAR) questionnaire in erectile dysfunction. Urology. 2003 ; 61: 888-893
17. O'Leary MP, Althof SE, Cappelleri JC, et al. on behalf of the US SEAR Study Group. Self-esteem, confidence, and relationship satisfaction in men with erectile dysfunction treated with sildenafil citrate: A multicenter, randomized, parallel-group, double-blind, placebo-controlled study in the United States. J Urol. 2006 ; 175: 1058-1062
18. Cappelleri JC, Althof SE, O'Leary MP, et al. on behalf of the US and International SEAR Study Group. Analysis of single items on the Self-Esteem And Relationship questionnaire in men treated with sildenafil citrate for erectile dysfunction: Results of two double-blind placebo-controlled trials. BJU Int. 2008 ; 101: 861-866
19. Thompson JR, Cappelleri JC, Getter C, et al. Enhanced interpretation of instrument scales using the Rasch model. Drug Inform J. 2007 ; 41: 541-550
20. Cappelleri JC, Rosen RC, Smith MD, et al. Some developments on the International Index of Erectile Function (IIEF). Drug Inform J. 1999 ; 33: 179-190
21. Sechrest L, McKnight P, McKnight K. Calibration of measures for psychotherapy outcome studies. Am Psychol. 1996 ; 51: 1065-1071
22. Lipsey MW, Wilson DB Practical meta-analysis. Thousands Oaks, CA: Sage Publications ; 2001 :
23. Rogers J, Howard KI, Vessey JT. Using significance tests to evaluate equivalence between two experimental groups. Psychol Bull. 1993 ; 113: 553-565
24. Kendall PC, Marrs-Garcia A, Nath SR, et al. Normative comparisons for the evaluation of clinical significance. J Consult Clin Psychol. 1999 ; 7: 285-299
25. Liu J-p Encyclopedia of biopharmaceutical statistics. S-C Chow, ed. 2003 :
26. Steiger JH. Beyond the F test: Effect size confidence intervals and tests of close fit in the analysis of variance and contrast analysis. Psychol Meth. 2004 ; 9: 164-182
27. Westlake WJ. Bioequivalence testing - Response. Biometrics. 1981 ; 37: 591-593
28. Ware JE, Snow KK, Kosinski M SF-36 health survey: Manual and interpretation guide. Lincoln, RI: QualityMetric Incorporated ; 2000 :
29. Ware JE, Kosinski M, Keller SD SF-36 physical and mental health summary scales: A user's manual. Boston, MA: The Health Institute, New England Medical Center ; 1994 :
30. Cappelleri JC, Bell SS, Siegel RL. Interpretation of a self-esteem subscale for erectile dysfunction by cumulative logit model. Drug Inform J. 2007 ; 41: 723-732
31. Hambleton RK, Swaminathan H, Rogers HJ Fundamentals of item response theory. Newbury Park, CA: Sage Publications ; 1991 :
32. Chang CH, Reeve BB. Item response theory and its applications to patient-reported outcomes measurement. Eval Health Profession. 2005 ; 28: 264-282
33. De Ayala RJ The theory and practice of item response theory. New York, NY: Guilford Press ; 2009 :
34. Lall R, Campbell MJ, Walters SJ, et al. A review of ordinal regression models applied on health-related quality of life assessments. Stat Meth Med Res. 2002 ; 11: 49-67
35. Arostegui I, Núñez-Antón V, Quintana JM. Statistical approaches to analyse patient-reported outcomes as response variables: An application to health-related quality of life. Stat Meth Med Res. 2012 ; 21: 189-214
36. O'Connell AA Logistic regression models for ordinal response variables. Thousands Oaks, CA: Sage Publications ; 2005 :
37. Hosmer DW, Lemeshow S Applied logistic regression. 2 nd edn. Hoboken, NJ: John Wiley & Sons, 2000. Hoboken, NJ: John Wiley & Sons ; 2000 :
38. Agresti A Categorical data analysis. 2 nd edn. Hoboken, New Jersey: John Wiley & Sons, 2002. Hoboken, New Jersey: John Wiley & Sons ; 2002 :
39. Kleinbaum D, Klein M Logistic regression. 3 rd edn. New York, NY: Springer, 2010. New York, NY: Springer ; 2010 :
40. Luo X, Cappelleri JC, Cella D, et al. Using the Rasch model to validate and enhance the interpretation of the Functional Assessment of Cancer Therapy-Kidney Symptom Index-Disease Related Symptoms. Value Health. 2009 ; 12: 580-586
41. Bond TG, Fox CM Applying the Rasch model; Fundamental measurement in the human sciences. 2 nd edn. Mahwah, NJ: Lawrence Erlbaum, 2007. Mahwah, NJ: Lawrence Erlbaum ; 2007 :
42. King MT. A point of minimal important difference (MID): a critique of terminology and methods. Expert Rev Pharmacoecon Outcome Res. 2011 ; 11: 171-184
43. Guyatt GH, Osoba D, Wu A, et al. and the Clinical Significance Consensus Meeting Group. Methods to explain the clinical significance of health status measures. Mayo Clin Proceed. 2003 ; 77: 371-383
44. Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol. 2003 ; 56: 395-440
45. Hays RD, Farivar SS, Liu H. Approaches and recommendations for estimating minimally important difference for health-related quality of life measures. J Chronic Obstr Pulmon Dis. 2005 ; 2: 63-67
46. Kazis LE, Anderson JJ, Meenan RF. Effect sizes for interpreting changes in health status. Med Care. 1989 ; 27: S178 - S189
47. Cohen J Statistical power analysis for the behavioral science. 2 nd edn. Hillsdale, NJ: Lawrence Erlbaum, 1988. Hillsdale, NJ: Lawrence Erlbaum ; 1988 :
48. Donaldson GW. Ridit scores for analysis and interpretation of ordinal pain data. Eur J Pain. 1998 ; 2: 221-227
49. Acion L, Peterson JL, Temple S, et al. Probabilistic index: an intuitive non-parametric approach to measuring the size of treatment effects. Stat Med. 2006 ; 25: 591-602
50. Uryniak T, Chan ISF, Fedorov VV, et al. Responder analyses - a PhRMA position paper. Stat Biopharm Res. 2011 ; 3: 476-487
51. Farrar JT, Dworkin RH, Max MB. Use of the cumulative proportion of responders analysis graph to present pain data over a range of cut-off points: making clinical trial data more understandable. J Pain Symptom Manage. 2006 ; 31: 369-377
52. Cappelleri JC, Bushmakin AG, Zlateva G, et al. Pain responder analysis: use of area under the curve to enhance interpretation of clinical trial results. Pain Practice. 2009 ; 9: 348-353
53. Bushmakin AG, Cappelleri JC, Zlateva G, et al. Applying area-under-the-curve analysis to enhance interpretation of response profiles: An application to sleep quality scores in patients with fibromyalgia. Qual Life Res. 2011 ; 20: 491-498
54. Bushmakin AG, Cappelleri JC. A note on cumulative distribution functions for patient-reported outcomes. MAPI Patient-Reported Outcomes Newsletter. 2011 ; 45: 11-12
55. Copay AG, Subach BR, Glassman SD, et al. Understanding the minimum clinically important difference: A review of concepts and methods. Spine J. 2007 ; 7: 541-546
56. De Vet HCQ, Ostello RWJG, Terwee CB, et al. Minimally important change determined by a visual method integrating an anchor-based and a distribution-based approach. Qual Life Res. 2007 ; 16: 131-142
57. Lewis JA. In defence of the dichotomy. Pharmaceut Stat. 2004 ; 3: 77-79
58. Senn S. Disappointing dichotomies. Pharmaceut Stat. 2003 ; 2: 239-240
59. Kunz M. On responder analyses when a continuous variable is dichotomized and measurement error is present. Biometr J. 2011 ; 53: 137-155
60. Lachin JM. Worst-rank score analysis with informatively missing observations in clinical trials. Control Clin Trials. 1999 ; 20: 408-422
61. Althof SE, Berner MM, Goldstein I, et al. Interrelationship of sildenafil treatment effects on the physiological and psychosocial aspects of erectile dysfunction of mixed or organic etiology. J Sex Med. 2010 ; 7: 3170-3178
62. Claes HIM, Goldstein I, Althof SE, et al. Understanding the effects of sildenafil treatment on erection maintenance and erection hardness. J Sex Med. 2010 ; 7: 2184-2191
63. Fairclough DL Design and analysis of quality of life studies in clinical trials. 2 nd edn. Boca Raton, FL: Chapman & Hall/CRC Press, 2010. Boca Raton, FL: Chapman & Hall/CRC Press ; 2010 :
64. The prevention and treatment of missing data in clinical trials. Washington, DC: The National Academies Press ; 2010 :