Responsiveness and Meaningful Thresholds of PROMIS Pain Interference, Fatigue, and Physical Function Forms in Adults with Idiopathic Inflammatory Myopathies: Report from the OMERACT Myositis Working Group

Overview

Journal Semin Arthritis Rheum

Specialty Rheumatology

Date 2023 Dec 23

PMID 38141522

Authors

D Saygin

D DiRenzo

J Raaphorst

I de Groot

C O Bingham

I E Lundberg

M Regardt

C Sarver

M de Visser

L J Maxwell

D Beaton

J Y Kim

M Needham

H Alexanderson

L Christopher-Stine

C A Mecoli

J K Park

Affiliations

Soon will be listed here.

Abstract

Background: A series of qualitative studies conducted by the OMERACT Myositis Working Group identified pain interference, fatigue, and physical function as highly important life impact domains for adults with idiopathic inflammatory myositis (IIM). In this study, our goal was to assess the responsiveness and minimal important difference of PROMIS pain interference (6a), fatigue (7a), and physical function (8b).

Methods: Adults with IIM from USA, Netherlands, Korea, Sweden, and Australia with two "clinical" visits were enrolled in this prospective study. Anchor questions on a Likert scale were collected at baseline, and manual muscle testing (MMT), physician and patient reported global disease activity, and PROMIS instruments were collected at both visits. Responsiveness was assessed with i) ANOVA, ii) paired t-test, effect size and standardized response mean, and iii) Pearson correlation. Minimal important difference (MID), minimal important change (MIC) and minimal detectable change (MDC) values were calculated.

Results: 114 patients with IIM (median age 60, 60 % female) completed both visits. Changes in PROMIS instruments were significantly different among anchor categories. Patients who reported improvement had a significant improvement in their PROMIS scores with at least medium effect size, while patients who reported worsening and stability did not show a significant change with weak effect size. PROMIS instruments had weak to moderate correlations with MMT, patient and physician global disease activity. MID was approximately 2-3 points for Pain Interference and 3-4 points for Fatigue and Physical Function forms based on the method used. MIC was approximately 4-5 for improvement of all the instruments, while MDC was 1.7-2 points for Pain Interference and Physical Function and 3.2-3.9 for Fatigue.

Conclusion: This study provides evidence towards the responsiveness of the PROMIS instruments in a large international prospective cohort of adults with IIM supporting their use as PROMs in adult myositis.

Citing Articles

Evaluating Meaningful Changes in Patient-Reported Outcome Measurement Information System-Fatigue Scores from Three Phase 3 Clinical Trials of Sarilumab for Patients With Rheumatoid Arthritis.

Bingham C, Molina E, Praestgaard A, Fiore S, Cella D ACR Open Rheumatol. 2024; 7(1):e11763.

PMID: 39545383 PMC: 11694253. DOI: 10.1002/acr2.11763.

Construct validity of PROMIS pain interference, fatigue, and physical function as patient-reported outcomes in adults with idiopathic inflammatory myopathies: An international study from the OMERACT myositis working group.

Romich E, Saygin D, DiRenzo D, Mecoli C, de Groot I, Lodin K Semin Arthritis Rheum. 2024; 68:152534.

PMID: 39159576 PMC: 11404521. DOI: 10.1016/j.semarthrit.2024.152534.

References

Terwee C, Peipert J, Chapman R, Lai J, Terluin B, Cella D . Minimal important change (MIC): a conceptual clarification and systematic review of MIC estimates of PROMIS measures. Qual Life Res. 2021; 30(10):2729-2754. PMC: 8481206. DOI: 10.1007/s11136-021-02925-y. View

Revicki D, Hays R, Cella D, Sloan J . Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008; 61(2):102-9. DOI: 10.1016/j.jclinepi.2007.03.012. View

Leclair V, Lundberg I . New Myositis Classification Criteria-What We Have Learned Since Bohan and Peter. Curr Rheumatol Rep. 2018; 20(4):18. PMC: 5857275. DOI: 10.1007/s11926-018-0726-4. View

Jaeschke R, Singer J, Guyatt G . Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989; 10(4):407-15. DOI: 10.1016/0197-2456(89)90005-6. View

Lydick E, Epstein R . Interpretation of quality of life changes. Qual Life Res. 1993; 2(3):221-6. DOI: 10.1007/BF00435226. View

DiRenzo D, Bingham 3rd C, Mecoli C . Patient-Reported Outcomes in Adult Idiopathic Inflammatory Myopathies. Curr Rheumatol Rep. 2019; 21(11):62. PMC: 7050440. DOI: 10.1007/s11926-019-0862-5. View

Lundberg I, Tjarnlund A, Bottai M, Werth V, Pilkington C, de Visser M . 2017 European League Against Rheumatism/American College of Rheumatology Classification Criteria for Adult and Juvenile Idiopathic Inflammatory Myopathies and Their Major Subgroups. Arthritis Rheumatol. 2017; 69(12):2271-2282. PMC: 5846474. DOI: 10.1002/art.40320. View

Lundberg I, Fujimoto M, Vencovsky J, Aggarwal R, Holmqvist M, Christopher-Stine L . Idiopathic inflammatory myopathies. Nat Rev Dis Primers. 2021; 7(1):86. DOI: 10.1038/s41572-021-00321-x. View

Norman G, Stratford P, Regehr G . Methodological problems in the retrospective computation of responsiveness to change: the lesson of Cronbach. J Clin Epidemiol. 1997; 50(8):869-79. DOI: 10.1016/s0895-4356(97)00097-8. View

10.

Leclair V, Tsui H, Hudson M . Pain in autoimmune inflammatory myopathies: a scoping review. RMD Open. 2023; 9(1). PMC: 9843184. DOI: 10.1136/rmdopen-2022-002591. View

11.

Kamperman R, Bogaards J, Evers S, Walter H, de Visser M, de Borgie C . Treatment with add-on IVIg in Myositis Early In the diSease course May be sUperior to Steroids alone for reaching CLinical improvEment (TIME IS MUSCLE): study protocol of a phase-2 double-blind placebo-controlled randomised trial. BMJ Open. 2023; 13(7):e067435. PMC: 10335467. DOI: 10.1136/bmjopen-2022-067435. View

12.

Mecoli C, Park J, Alexanderson H, Regardt M, Needham M, de Groot I . Perceptions of Patients, Caregivers, and Healthcare Providers of Idiopathic Inflammatory Myopathies: An International OMERACT Study. J Rheumatol. 2018; 46(1):106-111. PMC: 7497902. DOI: 10.3899/jrheum.180353. View

13.

Esfandiary T, Park J, Alexanderson H, Regardt M, Needham M, de Groot I . Assessing the content validity of patient-reported outcome measures in adult myositis: A report from the OMERACT myositis working group. Semin Arthritis Rheum. 2020; 50(5):943-948. PMC: 7646936. DOI: 10.1016/j.semarthrit.2020.06.006. View

14.

de Vet H, Ostelo R, Terwee C, van der Roer N, Knol D, Beckerman H . Minimally important change determined by a visual method integrating an anchor-based and a distribution-based approach. Qual Life Res. 2006; 16(1):131-42. PMC: 2778628. DOI: 10.1007/s11136-006-9109-9. View

15.

Akoglu H . User's guide to correlation coefficients. Turk J Emerg Med. 2018; 18(3):91-93. PMC: 6107969. DOI: 10.1016/j.tjem.2018.08.001. View

16.

Limaye V, Lester S, Blumbergs P, Roberts-Thomson P . Idiopathic inflammatory myositis is associated with a high incidence of hypertension and diabetes mellitus. Int J Rheum Dis. 2010; 13(2):132-7. DOI: 10.1111/j.1756-185X.2010.01470.x. View

17.

Bohan A, Peter J . Polymyositis and dermatomyositis (first of two parts). N Engl J Med. 1975; 292(7):344-7. DOI: 10.1056/NEJM197502132920706. View

18.

Alexanderson H, Del Grande M, Bingham 3rd C, Orbai A, Sarver C, Clegg-Smith K . Patient-reported outcomes and adult patients' disease experience in the idiopathic inflammatory myopathies. report from the OMERACT 11 Myositis Special Interest Group. J Rheumatol. 2014; 41(3):581-92. PMC: 6592050. DOI: 10.3899/jrheum.131247. View

19.

Hendrikx J, Fransen J, Kievit W, van Riel P . Individual patient monitoring in daily clinical practice: a critical evaluation of minimal important change. Qual Life Res. 2014; 24(3):607-16. DOI: 10.1007/s11136-014-0809-2. View

20.

Seamon B, Kautz S, Bowden M, Velozo C . Revisiting the Concept of Minimal Detectable Change for Patient-Reported Outcome Measures. Phys Ther. 2022; 102(8). PMC: 9361333. DOI: 10.1093/ptj/pzac068. View