Team: HAQ, aka “Ask the PROs”
Region: The Whole Patient
Authors: RheumMadness Leadership Team. Meridith Balbach, medical student, Vanderbilt University Medical Center; Courtney Bair, medical student, Duke University School of Medicine; Ben Lueck, medical student, Duke University School of Medicine; Lauren He, MD, chief internal medicine resident, University of Chicago; Ben Kellogg, MD, internal medicine resident, Duke University School of Medicine; Sabahat Usmani, MD, internal medicine resident, Weiss Memorial Hospital; Guy Katz, MD, rheumatology fellow, Massachusetts General Hospital; Michael Macklin, MD, rheumatology fellow, University of Chicago; Iman Qaiser, MD, rheumatology fellow, LSU Health Shreveport; Akrithi Udupa Garren, MD, assistant professor of medicine, Medstar / Georgetown Washington Hospital Center; David Leverenz, MD, assistant professor of medicine, Duke University School of Medicine.
Field goals. Rebounds. Assists. Steals. Turnovers. Basketball stats are a celebrated part of the game, arming fans with cold hard data to quantify the performance of a team or player. One must be wary, however, of measures that don’t translate into wins—not only in basketball, but in rheumatology, too.
In the late 20th century, rheumatology coaches were grappling with finding measures that represented clinically meaningful buckets. Historically they had turned to outcomes such as the presence of synovitis, grip strength, morning stiffness; labs such as ESR; and radiographic changes to assess response to treatment.1 In 1980, James Fries et al. entered the endpoints arena with an entirely new type of play—patient-reported outcomes (PROs).2
In “Measurement of Patient Outcome in Arthritis,” the authors recognize and address the need for clinically meaningful measures in the context of chronic disease—specifically, rheumatoid arthritis. Moving beyond pre-existing scales that often lacked reliability, validity, and feasibility, the authors sought to develop and validate a systematic PRO, coined as the Health Assessment Questionnaire (HAQ).3-5 To do so, they administered self-evaluated and interview questionnaires in an initial cohort of 20 patients to assess dimensions of death, discomfort, disability, drug toxicity, and dollar cost, finding good reliability. Subsequent correlation of evaluator and self-administered HAQ assessing standardized task performance in 25 patients demonstrated validity.
The resulting validated instrument, now known as the HAQ disability index (HAQ-DI) or legacy HAQ, transformed the way rheumatologists view the (assessment) lineup—moving the patient from the role of oft-overlooked benchwarmer to coveted starter.
Impact on Rheumatology
Coach Fries et al. revolutionized the recruitment landscape, demonstrating principles of good PRO development and evolution.6 The HAQ, translated into 60+ languages and now cited more than 5000 times, spawned the development of derived PROs including the modified HAQ (MHAQ), simplified 10-item HAQ-II, and multidimensional HAQ (MDHAQ).7 Alongside its friendly rival (also published in 1980), the Arthritis Impact Measurement Scales (AIMS), it spurred exponential growth of additional PROs in rheumatology—as evidenced by dedicated Arthritis Care & Research special editions in 1992, 2003, and 2011—the last of which reported greater than 250 PROs.1,8,9 No longer limited to rheumatoid arthritis, generic and disease-specific instruments cover a diversity of pathologies from systemic lupus erythematosus with the LupusQoL to systemic sclerosis with the scleroderma HAQ (SHAQ).10,11
The HAQ provided evidence that well-crafted patient-reported outcomes might outperform “the so-called hard measures.”1 Like an energized fanbase doing the wave, the rippling effects of this paradigm shift are apparent. Consider OMERACT (initially coined as “Outcome Measures in Rheumatoid Arthritis Clinical Trials”, now broadened to “Outcomes Measures in Rheumatology”), an international effort to achieve endpoint consensus. Recognizing the importance of patient experience, they included three PROs (pain, patient global assessment, and functional capacity) amongst their Core Set of variables to be collected in all rheumatoid arthritis clinical trials.12 This recommendation has since been expanded to include additional PROs and disease-specific criteria for ANCA-associated vasculitis, idiopathic inflammatory myopathies, and more.13,14
In the post-game interview, Fries reflected that “people almost didn’t notice that their thinking had shifted from a narrower medical model to a broader psychosocial model of health and illness.”1 Indeed, the PROs inspired rheumatologists and other chronic disease specialists to become more patient-centered not only in their endpoints, but entire practice— fundamentally changing the game.
Chances in the Tournament
In our view, the first-round opponent of the PROs may be their toughest, with the LUMINA study drawing attention to socioeconomic-demographic disparities in SLE outcomes. However, the Blue Ribbon Panel might recognize that many of the salient findings of LUMINA rely on use of the Illness Behavior Questionnaire and Rheumatology Attitudes Index—that is, PROs.
After conquering the “Whole Patient” bracket, the PROs will conquer the winner of the “Origin Story” region. While RheumMadness fans may enjoy rooting for a Cinderella team, they must remember that such success is few and far between— an early case report of rheumatology disease is fascinating, but it’s best to bet on a #1 seed. From there, the success of the PROs largely depends on whether the Blue Ribbon Panel is more focused on impressive clinical trial data or paradigm shifts within the field. Given the theme of this year’s competition, the latter may be true, allowing this team to truly live up to its name.
The most important and transformational article ever written in the field of rheumatology? Just ask the PROs!
Next scouting report: Cortisone
Back to the full list of scouting reports.
See the Q&A on theMednet.org for The Whole Patient region: What target do you utilize in clinical practice for defining disease remission in RA?
- Callahan LF. The History of Patient-Reported Outcomes in Rheumatology. Rheum Dis Clin North Am. May 2016;42(2):205-17. doi:10.1016/j.rdc.2016.01.012
- Fries JF, Spitz P, Kraines RG, Holman HR. Measurement of patient outcome in arthritis. Arthritis Rheum. Feb 1980;23(2):137-45. doi:10.1002/art.1780230202
- McCloy L, Jongbloed L. Robinson Bashall Functional Assessment for arthritis patients: reliability and validity. Arch Phys Med Rehabil. Aug 1987;68(8):486-9.
- Lee P, Jasani MK, Dick WC, Buchanan WW. Evaluation of a functional index in rheumatoid arthritis. Scand J Rheumatol. 1973;2(2):71-7. doi:10.3109/03009747309098820
- Convery FR, Minteer MA, Amiel D, Connett KL. Polyarticular disability: a functional assessment. Arch Phys Med Rehabil. Nov 1977;58(11):494-9.
- Deshpande PR, Rajan S, Sudeepthi BL, Abdul Nazir CP. Patient-reported outcomes: A new era in clinical research. Perspect Clin Res. Oct 2011;2(4):137-44. doi:10.4103/2229-3485.86879
- Maska L, Anderson J, Michaud K. Measures of functional status and quality of life in rheumatoid arthritis: Health Assessment Questionnaire Disability Index (HAQ), Modified Health Assessment Questionnaire (MHAQ), Multidimensional Health Assessment Questionnaire (MDHAQ), Health Assessment Questionnaire II (HAQ-II), Improved Health Assessment Questionnaire (Improved HAQ), and Rheumatoid Arthritis Quality of Life (RAQoL). Arthritis Care Res (Hoboken). Nov 2011;63 Suppl 11:S4-13. doi:10.1002/acr.20620
- Katz PP. Introduction to special issue: patient outcomes in rheumatology, 2011. Arthritis Care Res (Hoboken). Nov 2011;63 Suppl 11:S1-3. doi:10.1002/acr.20585
- Meenan RF, Gertman PM, Mason JH. Measuring health status in arthritis. The arthritis impact measurement scales. Arthritis Rheum. Feb 1980;23(2):146-52. doi:10.1002/art.1780230203
- McElhone K, Abbott J, Shelmerdine J, et al. Development and validation of a disease-specific health-related quality of life measure, the LupusQol, for adults with systemic lupus erythematosus. Arthritis Rheum. Aug 15 2007;57(6):972-9. doi:10.1002/art.22881
- Steen VD, Medsger TA. The value of the Health Assessment Questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis patients over time. Arthritis Rheum. Nov 1997;40(11):1984-91. doi:10.1002/art.1780401110
- Gossec L, Dougados M, Dixon W. Patient-reported outcomes as end points in clinical trials in rheumatoid arthritis. RMD Open. 2015;1(1):e000019. doi:10.1136/rmdopen-2014-000019
- Merkel PA, Aydin SZ, Boers M, et al. The OMERACT core set of outcome measures for use in clinical trials of ANCA-associated vasculitis. J Rheumatol. Jul 2011;38(7):1480-6. doi:10.3899/jrheum.110276
- Regardt M, Basharat P, Christopher-Stine L, et al. Patients’ Experience of Myositis and Further Validation of a Myositis-specific Patient Reported Outcome Measure – Establishing Core Domains and Expanding Patient Input on Clinical Assessment in Myositis. Report from OMERACT 12. J Rheumatol. Dec 2015;42(12):2492-5. doi:10.3899/jrheum.141243