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Patient Questionnaires in Rheumatology Treatment Decisions

This article discusses the importance of standardized patient questionnaires in gathering quantitative data for rheumatology treatment decisions. It highlights the limitations of relying solely on other sources of information and emphasizes the need for structured self-report questionnaires. The article also explores the different measures used to assess patients with rheumatoid arthritis (RA) and the benefits and limitations of formal joint counts and radiographs in diagnosis and management.

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Patient Questionnaires in Rheumatology Treatment Decisions

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  1. Patient questionnaires: Standardized quantitative “scientific” data from a patient history, the primary source of rheumatology treatment decisions Theodore Pincus MD Clinical Professor of Medicine New York University tedpincus@gmail.com

  2. Disclosures Theodore Pincus, MD Sources of Funding for Research: Amgen Inc.; Bristol-Myers Squibb Company Consulting Agreements: Abbott Laboratories; Amgen Inc.; Bristol-Myers Squibb Company; UCB Speakers’ Bureau/Honorarium Agreements: Abbott Laboratories; Wyeth Pharmaceuticals, Genentech Financial Interests/Stock Ownership: None Discussion of Off-Label, Investigational, or Experimental Drug Use: None

  3. Many, if not most, doctors have extensive information about their patients with a few mouse clicks concerning: Scheduling Billing Laboratory tests Medications BUT NOT: Is the patient better, worse, or the same? With which treatments? Why not ask the patient in a structured, “scientific” format, ie, self-report questionnaire?

  4. Why measurement? This wine is expensive – $60 or $6,000 The patient has a fever – 101º or 106ºF, 38º or 40ºC The blood pressure is high – 150/95 or 250/125 The patient is “doing well” – What is the DAS28, CDAI or RAPID3

  5. Complexities in quantitative assessment of patients with RA and rheumatic diseases Laboratory tests are limited in diagnosis and treatment decisions Treat radiograph before damage No single ‘Gold Standard’ measure, eg, blood pressure, cholesterol, glucose, for diagnosis and management in all individual patients Therefore, need indices of 3–7 measures

  6. American College of Rheumatology (ACR) Core Data Set & Disease Activity Score (DAS) 3 Physician/Assessor measures 1. Tender joint count (also in DAS) 2. Swollen joint count (also in DAS) Assessor Global status 3 Patient self-report measures 4. Physical Function - HAQ, HAQ II, MDHAQ 5. Pain 6. Patient Global status (also in DAS) 1 Laboratory Measure 7. Acute phase reactant –ESR, CRP–also in DAS (8. Radiograph – longer than 1 year) Felson et al, Arth Rheum 36:729, 1993. van Riel, Br J Rheumatol 31:793, 1994.

  7. Types of measures to assess patients with RA • Joint counts • Radiographs • Laboratory tests • Patient questionnaires • Global estimates

  8. Formal Joint Counts in Management of Patients With RA • Most specific measure to assess RA • Most important measure in clinical trials – 20, 50, 70% required for ACR improvement criteria • Widely-accepted by rheumatologists and FDA as “best” measures • 28-joint count as useful as 68–70 joint count

  9. Changes in ACR Core Data Set Measures Over 12 Months: Leflunomide (LEF) vs Methotrexate (MTX) vs Placebo (PBO) Measure: LEF PBO MTX Effect Relative Size Efficiency Tender Jts -7.7 -3.0 -6.6 -0.59 1.00 Swollen Jts -5.7 -2.9 -5.4 -0.44 0.56 MD Global -2.8 -1.0 -2.4 -0.68 1.33 ESR -6.3 +2.6 -6.5 -0.41 0.48 FN- HAQ -0.45 +0.03 -0.26 -0.80 1.84 FN-MHAQ -0.29 +0.07 -0.15 -0.69 1.37 Pain -2.2 -0.4 -1.7 -0.65 1.21 Pt Global -2.1 +0.1 -1.5 -0.81 1.88 Strand V, et al. Arch Intl Med. 1999; 159:2542-2550; Tugwell P, et al. Arthritis Rheum. 2000; 43:506-514.

  10. Question for Rheumatologists For patients with RA under your care (not including patients in clinical trials), how often do you perform formal tender and swollen joint counts? Never 13% 1–24% of visits 32% 25–49% of visits 11% 50–74% of visits 14% 75–99% of visits 16% Always 14%

  11. Time to Score RA Measures - Seconds Pincus et al 2009; Arthritis Care Res. in press

  12. Some Limitations of Formal Joint Counts • Relative efficiencies similar or lower than global and patient measures in clinical trials • May improve over 5 years while joint damage and functional disability may progress • Poorly reproducible • Not performed at most visits in usual care

  13. The most specific measure for diagnosis is not necessarily the most significant measure for prognosis and management.

  14. Radiographs in Diagnosis and Management of Patients With RA • Excellent quantitative scoring systems - Sharp, van der Heijde, Larsen, Genant • Erosions are closest to pathognomonic sign in RA • Reflect cumulative damage of disease

  15. 9- to 10-Year Survival According to Quantitative Markers in Three Chronic Diseases Rheumatoid Arthritis – Activities of Daily Living Rheumatoid Arthritis – Formal Education Level A B 100 100 >12 Years >90% 80 81%–90% 80 9–12 Years % Active “With Ease” 60 60 £8 Years Survival (%) Survival (%) 40 40 71%–80% 20 20 £70% (Data from Pincus et al, 1987) (Data from Pincus et al, 1987) Months Months 0 20 40 60 80 100 0 20 40 60 80 100 Hodgkin Disease – Anatomic Stage Coronary Artery Disease – No. of Involved Vessels C D 100 100 Stage I 80 80 1 Artery Stage II 60 60 Stage III All Stages, All Causes Survival (%) Survival (%) 2 Arteries Stage IV 40 40 3 Arteries 20 20 LCA (Data from Kaplan, 1972) (Data from Proudfit et al, 1978) Years Years 0 2 4 6 8 10 0 2 4 6 8 10

  16. TEMPO Trial: Year 2 Radiograph:Change in Total Sharp Score from Baseline to Year 2 3.34 (CI 1.18, 5.50) 1.10* (CI 0.13, 2.07) * p < 0.05, E vs MTX † p < 0.05, Combination vs MTX ‡ p < 0.05, Combination vs E -0.56†‡(CI –1.05, -0.06)

  17. Yazıcı Y, Yazıcı H, Arthritis Rheum 2006;54(supl)

  18. 2 Year Change in Total Sharp/van der Heijde X-ray score (0–448): TEMPO probability plot TEMPO=Trial of Etanercept and MTX with radiographic Patient Outcomes. van der Heijde, et al. Arthritis Rheum 2006;54:1063–74. 19

  19. Strongly and Weakly Related Measures to Assess RA Radiographs ESR, CRP Shared epitope Rheumatoid factor Joint deformity Duration of disease Functional disability Pain Patient global estimate Socioeconomic status Joint tenderness Age Pincus T, Sokka T: Best Pract Res Clin Rheumatol 17:753-781, 2003.

  20. Predicting Mortality in RA: Most Baseline Measures Are Worse in Patients Who Will Die Over a 5-Year Period Mean Baseline Values P Value Alive Dead Age (years) 55.1 65.5 < 0.001 ARA functional class 2.2 2.6 < 0.001 1.1 2.1 < 0.001 Number of comorbidities 10.8 16.8 < 0.001 Walking time 33.8 48.3 0.004 ESR 1.98 2.32 0.005 mHAQ score 2.41 2.55 0.007 Learned helplessness 2.6 3.0 0.01 Global self-report 0.2 0.5 0.02 Number of extra-articular features 9.1 12.7 0.03 Duration of disease 10.8 9.4 0.03 Years of education 12.8 15.9 0.04 Joint count 1.2 1.4 0.20 Radiograph score 2.7 2.9 0.28 RF titer 5.40 5.19 0.68 Pain Callahan LF, et al. Arthritis Care Res. 1997;10:381–394.

  21. RA Cohort #2-Cox Proportional Hazards Model Analyses Including Demographic, Functional, Self-Report, Joint Count, X-ray, Laboratory and Disease Variables in 206 patients Univariate Stepwise Model RR (95% CL) RR (95% CL) P Value P Value 1.07 <0.001 1.06 <0.001 Age 1.63 <0.001 1.40 0.02 Comorbidity 2.00 0.003 1.76 0.02 MHAQ ADL Score 1.04 0.02 -- -- Disease duration 0.89 0.007 -- -- Education 1.01 0.005 -- -- ESR 1.02 0.10 -- -- Joint count 1.03 0.04 -- -- Walking time 1.40 0.17 -- -- X-ray Arthritis Care Res 10:381,1997

  22. MRI can better identify early bone erosions than X-ray

  23. Some Problems With Radiographs in RA • Quantitative score tedious to perform • Treatment initiated prior to erosions – MRI, ultrasound more sensitive • Radiographic damage has poor prognostic value for work disability, death and even joint replacement • Treatment prior to erosions

  24. Laboratory Tests in Diagnosis and Management of Patients With RA • Most important measure in most clinical situations, e.g., cholesterol, hemoglobin, creatinine, glucose, etc. • Many tests may be of value – CBC, ESR, CRP, RF, anti-CCP • No work for the rheumatologist

  25. "the erythrocyte sedimentation rate is increased in nearly all patients with active RA” Lipsky PE. Rheumatoid arthritis. In: Fauci AS, Langford CA, eds. Harrison's Medicine. New York: McGraw-Hill,2006:85. “at least 5% of patients with clinically active disease may have a normal ESR” Chatham WW, Blackburn WD, Jr. Laboratory findings in rheumatoid arthritis. In: Koopman WJ, Moreland LW, editors. Arthritis and allied conditions: a textbook of rheumatology. Philadelphia, PA: Lippincott, Williams & Wilkins, 2005:1207 Textbook statements concerning ESR in RA

  26. Traditional approaches to clinical expertise: EMINENCE BASED MEDICINE - making the same mistakes with increasing confidence over an impressive number of years ELOQUENCE BASED MEDICINE - a year-roundsuntan and brilliant oratory may overcome absence of any supporting data ELEGANCE BASED MEDICINE - where the sartorialsplendor of a silk-suited sycophant substitutes for substance The modern alternative? EVIDENCE BASED MEDICINE - the best approach to clinical data - requires information from clinical observational data in addition to clinical trials Pincus and Tugwell J Rheumatol 2006

  27. ESR Values in Patients With RA Wolfe F, Michaud K, J Rheumatol. 1994;21:1227–1237. Wichita KS, USA Similar results have seen reported from: Nashville, TN USA Jyvaskyla, Finland Oslo, Norway Nancy, France Gronigen, the Netherlands Belfast, Ireland

  28. Mean ESR (mm/Hr) 4 Locations – 1996: Smedstad LM, Moum T, Guillemin F,Kvien TK, Finch MB, Suurmeijer TPBM, Van Den Heuvel WJA Br J Rheumatol 1996; 35:746-51

  29. ESR and CRP at 1st visit in US and Finland – 1980-2005 Sokka and Pincus – J Rheumatol 2009

  30. Mean/median baseline ESR in RA patients in 23 studies, by first year of recruitment Abelson B, Sokka T, Pincus T. J Rheumatol 2009

  31. Meta-analysis: Anti-cyclic citrullinated peptide (CCP) antibody and rheumatoid factor (RF) Nishimura K et al. Annals of Internal Medicine 146:797-808, 2007

  32. Meta-analysis: Anti-cyclic citrullinated peptide (CCP) antibody and rheumatoid factor (RF) Nishimura K et al. Annals of Internal Medicine 146:797-808, 2007

  33. RA Cohort #2-Cox Proportional Hazards Model Analyses Including Demographic, Functional, Self-Report, Joint Count, X-ray, Laboratory and Disease Variables in 206 patients 1985-1990 Univariate Stepwise Model RR (95% CL) RR (95% CL) P Value P Value 1.07 <0.001 1.06 <0.001 Age 1.63 <0.001 1.40 0.02 Comorbidity 2.00 0.003 1.76 0.02 MHAQ ADL Score 1.04 0.02 -- -- Disease duration 0.89 0.007 -- -- Education 1.01 0.005 -- -- ESR 1.02 0.10 -- -- Joint count 1.03 0.04 -- -- Walking time 1.40 0.17 -- -- X-ray Arthritis Care Res 10:381,1997

  34. 5-Year Survival in 206 Patients With RA: Cohort #2 – 1985-1990 Rheumatoid Factor MHAQ Score 100 100 80 80 60 60 Survival (%) Survival (%) 0.00 (12) Absent (29) 40 40 0.01–0.99 (91) Present (175) 1.00–1.99 (86) 20 20 >2.00 (21) 0 0 0 12 24 36 48 60 0 12 24 36 48 60 Months After Baseline Months After Baseline Arthritis Care Res 10:381,1997

  35. IgM rheumatoid factor binding IgG

  36. Multi-Dimensional Health Assessment Questionnaire (MDHAQ) Page 1

  37. RF positive - 69% (1) Anti-CCP positive - 67% (1) ESR >28 mm/Hr - 57% (2,3) CRP >10 - 58% (2) % of RA patients with abnormal measures at presentation: evidence, not eminence-based 1- Nishimura et al, Ann Int Med 146:797-808, 2007 2 - Wolfe and Michaud, J Rheumatol 21:1227–1237, 1994 3 - Sokka and Pincus, J Rheumatol 36:1387--1390,2009

  38. Some Problems With Laboratory Tests in Diagnosis and Management of RA • ESR & CRP - normal in 40% at presentation • Anti-CCP & RF - negative in 20–50% of patients • Treatment decisions are based primarily on clinical criteria • Lab tests have good prognostic value for radiographic damage but poor prognostic value for work disability or death CRP = C-reactive protein; CCP = cyclic citrullinated protein

  39. Patient self-report questionnaires • HAQ and RAPID3 score as informative as ACR20/50/70 or DAS in clinical trials • Significant correlation with joint count, ESR, X-ray – individual measures and indices • Predict work disability, costs, TJR, and premature death more significantly than traditional measures • Quantitative measures to save time for patient and MD to focus on major patient matters

  40. 9-10 Year Survival According to Quantitative Markers in Three Chronic Diseases A B Formal Education Level Activities of Daily Living 100 >12 Years 100 >90% 80 80 81–90% 9–12 Years 60 60 Survival (%) Survival (%) £ 8 Years 40 40 71–80% 20 20 £70% (Data from Pincus et al, 1987) (Data from Pincus et al, 1987) Months 0 20 40 60 80 100 Months 0 20 40 60 80 100 D Coronary Artery Disease - # of Involved Vessels 100 80 1 Artery 60 Survival (%) 2 Arteries 40 3 Arteries LCA 20 (Data from Proudfit et al, 1978) Years 0 2 4 6 8 10 Rheumatoid Arthritis - Rheumatoid Arthritis - % Active “With Ease” C Hodgkin’s Disease - Anatomic Stage 100 Stage I 80 Stage II 60 All Stages, All Causes Survival (%) Stage III 40 Stage IV 20 (Data from Kaplan, 1972) Years 0 2 4 6 8 10

  41. 5-Year Survival in 206 Patients With RA: Cohort #2 – 1985-1990 Rheumatoid Factor MHAQ Score 100 100 80 80 60 60 Survival (%) Survival (%) 0.00 (12) Absent (29) 40 40 0.01–0.99 (91) Present (175) 1.00–1.99 (86) 20 20 >2.00 (21) 0 0 0 12 24 36 48 60 0 12 24 36 48 60 Months After Baseline Months After Baseline Arthritis Care Res 10:381,1997

  42. Significant in multivariate analyses Significant in univariate analyses Not Significant Significance of 8 variables as predictors of mortality in 53 RA cohorts 4% 6% 34% 17% 32% 23% 50% 39% 22% 30% 21% 39% 32% 46% 50% 28% 37% 72% 65% 45% 44% 31% 22% 11% Physicalfunction (N=18) Co- morbidities (N=23) Rheum-atoid factor (N=29) Extra- articular disease (N=18) ESR (N=19) Socio- economic status (N=13) Jointcount (N=18) Hand radio- graph (N=18) Sokka T, Abelson B, Pincus T. Clin Exp Rheumatol 26(suppl):S35-61, 2008

  43. Prediction of premature mortality according to blood pressure and cholesterol convertedhypertension and hypercholesterolemiafrom optionaltreatments tomajor public health campaigns.

  44. Imagine doctors saying that they do not measure blood pressure or cholesterol because “it takes too much time” or “the staff will not cooperate,” as suggested for why they do not measure physical function.

  45. The MDHAQ in Clinical Rheumatology In rheumatoid arthritis, the MDHAQ distinguishes MTX or LEF from placebo in a clinical trial as effectively as a joint count or the ACR 20 In osteoarthritis, the MDHAQ distinguishes NSAID from acetaminophen as effectively as the WOMAC Infibromyalgia, the MDHAQ distinguishes patients from those with rheumatoid arthritis as effectively as an ESR

  46. Physical function/activities of daily living (ADL) in prognosis of non-Rheumatic Diseases In congestive heart failure, ADL predict 36-month mortality as ejection fraction Konstam, Am J Cardiology 78:890, 1996 In AIDS, ADL predict 36-month mortality as CD4/CD8 ratios, clinical AIDS prognostic staging (CAPS), severity classification for AIDS hospitalizations (SCAH) Justice, J Clin Epidemiology 49:193, 1996 In hospitalized elder patients, ADL predict 1-year mortality beyond physiologic data and comorbidities Covinsky, J Gen Intern Med 12:203, 1997

  47. Need for translation Cultural and linguistic issues Possibility of ‘gaming’ by patient, health professional to provide desired responses Not specific to any disease Some limitations of patient self-report questionnaires

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