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Patient reported outcomes are an important component of the overall assessment of subjects in clinical studies. IMACS has agreed to incorporate patient-reported outcomes in all myositis trials and clinical studies. This includes a generic health-related quality of life measure that is age appropriate (adult vs. pediatric. Currently an OMERACT working group is dedicated to developing and validating new patient-reported outcome measures for adult myositis.

PROMs in Adult Idiopathic Inflammatory Myopathies

While there have been many PROMs used in adult IIM research, a short list provided here comprises the most validated measures. These include the SF-36, HAQ-DI, Patient Global Assessment, and PROMIS® measures, specifically the PROMIS short forms Fatigue 7a, Pain Interference 6a, Physical Function 8b and 20a, as well as PROMIS 57, a long form. Each individual PROM is briefly discussed below:

PROMIS® Measures

  • Description: PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health in adults. It can be used with the general population and with individuals living with chronic conditions such as IIM. Advantages of PROMIS measures include (i) they are translated into multiple languages (ii) English/Spanish language versions are free to use in academic settings (with additional translations also available by request), (iii) they provide results that are interpretable relative to the general population (i.e. T-scores). 
  • Several PROMIS® measures have been used in adult IIM research, including the PROMIS-29 and PROMIS Physical Function 20. However, the IMACS project on PROs (OMERACT Myositis Working Group) has extensively studied PROMIS Fatigue 7a, PROMIS Pain Interference 6a, PROMIS Physical Function 8b including assessing content validity, construct validity, responsiveness, and minimal clinical difference. PROMIS 57 has also been studied in adult IIM.  
  • The PROMIS tools are available at Health Measures.
  • Download:

The coding of several PROMIS tools, including PROMIS Physical Function Short Form 8b, PROMIS Pain Interference Short Form 6a, PROMIS Fatigue Short Form 7a, and PROMIS Profile-57 v2.0, will be made publicly available in the NIH Common Data Elements (CDE) Repository – coming soon.

SF-36

HAQ-DI

Patient Global Activity (PtGA) Assessment

References:

  1. Austenfeld EM, Sabbagh SE, Liegl M, Yan K, Do V, Fuller J, Rouster-Stevens K, Rider LG, Schiffenbauer A. PROMIS displays strong construct validity in pediatric and adult patients with idiopathic inflammatory myopathies. Rheumatology (Oxford). 2026 Jan 8;65(1):keaf502. doi: 10.1093/rheumatology/keaf502. [Abstract]
  2. Razok A, Taylor J, Ritz E, Wipfler K, Michaud K, Saygin D. Short Form 36 (SF-36) health survey questionnaire in health-related quality of life assessment in patients with inflammatory myopathies. Clin Exp Rheumatol. 2026 Feb;44(2):234-239. doi: 10.55563/clinexprheumatol/sbc4v3. Epub 2025 Jul 29. [Abstract]
  3. Romich E, Saygin D, DiRenzo D, Mecoli CA, de Groot I, Lodin K, Regardt M, Sarver C, Kim JY, Park JK, Beer K, Needham M, Alexanderson H, Christopher-Stine L, de Visser M, Raaphorst J; OMERACT Myositis Working Group. 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. Semin Arthritis Rheum. 2024 Oct;68:152534. doi: 10.1016/j.semarthrit.2024.152534. Epub 2024 Aug 10. [Abstract]
  4. Keret S, Saygin D, Moghadam-Kia S, Ren D, Oddis CV, Aggarwal R. Discordance between patient- and physician-reported disease activity in adult idiopathic inflammatory myopathy. Rheumatology (Oxford). 2023 Dec 1;62(12):3957-3961. doi: 10.1093/rheumatology/kead316. [Abstract]
  5. DiRenzo D, Saygin D, de Groot I, Bingham Iii CO, Lundberg IE, Needham M, Park JK, Regardt M, Sarver C, Song YW, Maxwell L, Beaton D, de Visser M, Christopher-Stine L, Mecoli CA, Alexanderson H. Reliability and validity of PROMIS physical function, pain interference, and fatigue as patient reported outcome measures in adult idiopathic inflammatory myopathies: International study from the OMERACT myositis working group. Semin Arthritis Rheum. 2023 Feb;58:152111. doi: 10.1016/j.semarthrit.2022.152111. Epub 2022 Nov 3. [Abstract]
  6. Saygin D, Oddis CV, Dzanko S, Koontz D, Moghadam-Kia S, Ardalan K, Coles TM, Aggarwal R. Utility of patient-reported outcomes measurement information system (PROMIS) physical function form in inflammatory myopathy. Semin Arthritis Rheum. 2021 Jun;51(3):539-546. doi: 10.1016/j.semarthrit.2021.03.018. Epub 2021 Apr 15. [Abstract]
  7. Esfandiary T, Park JK, Alexanderson H, Regardt M, Needham M, de Groot I, Sarver C, Lundberg IE, de Visser M, Song YW, DiRenzo D, Bingham CO 3rd, Christopher-Stine L, Mecoli CA. Assessing the content validity of patient-reported outcome measures in adult myositis: A report from the OMERACT myositis working group. Semin Arthritis Rheum. 2020 Oct;50(5):943-948. doi: 10.1016/j.semarthrit.2020.06.006. Epub 2020 Jun 17. [Abstract]
  8. DiRenzo D, Bingham CO 3rd, Mecoli CA. Patient-Reported Outcomes in Adult Idiopathic Inflammatory Myopathies. Curr Rheumatol Rep. 2019 Nov 19;21(11):62. doi: 10.1007/s11926-019-0862-5. [Abstract]
  9. Rider LG, Aggarwal R, Machado PM, Hogrel JY, Reed AM, Christopher-Stine L, Ruperto N. Update on outcome assessment in myositis. Nat Rev Rheumatol. 2018 May;14(5):303-318. doi: 10.1038/nrrheum.2018.33. Epub 2018 Apr 12. [Abstract]
  10. Benveniste O, Rider LG; ENMC Myositis Outcomes Study Group. 213th ENMC International Workshop: Outcome measures and clinical trial readiness in idiopathic inflammatory myopathies, Heemskerk, The Netherlands, 18-20 September 2015. Neuromuscul Disord. 2016 Aug;26(8):523-34. doi: 10.1016/j.nmd.2016.05.014. Epub 2016 May 27. [Abstract]
  11. Leclair V, Regardt M, Wojcik S, Hudson M; Canadian Inflammatory Myopathy Study (CIMS). Health-Related Quality of Life (HRQoL) in Idiopathic Inflammatory Myopathy: A Systematic Review. PLoS One. 2016 Aug 9;11(8):e0160753. doi: 10.1371/journal.pone.0160753. [Abstract]
  12. Ponyi A, Borgulya G, Constantin T, Váncsa A, Gergely L, Dankó K. Functional outcome and quality of life in adult patients with idiopathic inflammatory myositis. Rheumatology (Oxford). 2005 Jan;44(1):83-8. doi: 10.1093/rheumatology/keh404. Epub 2004 Sep 20. [Abstract]

PROMs in Juvenile Idiopathic Inflammatory Myopathies

PROMIS® Pediatric Self-Report and Parent-Proxy Measures

  • Description: PROMIS measures are available to assess a wide variety of physical, mental, and social health domains in pediatric patients with chronic illness as well as in the general pediatric population.  PROMIS measures are available as fixed short forms, as well as computerized adaptive testing item banks, with the option to create custom fixed short forms as well. Pediatric self-report measures are valid for use in patients ages 8 years and older, while parent-proxy report measures are available for patients 5 years and older.  Scores generated by PROMIS measures are normalized to reference populations, with T-scores allowing interpretation of scores for individual patients against the reference population.
  • PROMIS measures assessing global health, pain interference, and physical function (mobility, upper extremity function) have been used in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) juvenile dermatomyositis registry, with interpretation of scores in relation to nationally representative percentiles.  Additional PROMIS measures that have been utilized in research with juvenile idiopathic inflammatory myopathy patients include PROMIS Fatigue, Physical Activity, Anxiety, Depressive Symptoms, Psychological Stress Experiences, Life Satisfaction, and Positive Affect and the PROMIS-49 Profile.
  • Download: The PROMIS measures are available at Health Measures.

    The coding of several PROMIS tools, including PROMIS-48 Profile, will be made publicly available in the NIH Common Data Elements (CDE) Repository. – coming soon

Child Health Questionnaire (CHQ-PF50) for Children

  • Description: PRINTO has suggested using the CHQ-PF50® in myositis clinical trials and studies involving pediatric patients. All users will need to register and purchase the CHQ Manual from IQVIA COA Marketplace and to obtain copies of the form for use in their study. The HealthActCHQ website provides information regarding terms of use, content, bibliography, translations etc. All translations will be issued only through IQVIA COA Marketplace. Some users may require a license fee (depending on scope, languages, etc.).

Childhood Health Assessment Questionnaire (CHAQ)

PedsQL Generic Core Scales, Rheumatology Module, and Family Impact Module

  • Description: PedsQL is a system of pediatric self-report and parent-proxy report measures that can be used to assess multidimensional health-related quality of life (HRQoL) and disease impact constructs. PedsQL has undergone partial validation in patients with JIIMs and been used descriptively in JIIM HRQoL studies as well as in at least one study of family impact of juvenile dermatomyositis.
  • Information on licensing, access, and fee structure is available at PedsQL.org.

References:

  1. Austenfeld EM, Sabbagh SE, Liegl M, Yan K, Do V, Fuller J, Rouster-Stevens K, Rider LG, Schiffenbauer A. PROMIS displays strong construct validity in pediatric and adult patients with idiopathic inflammatory myopathies. Rheumatology (Oxford). 2026 Jan 8;65(1):keaf502. doi: 10.1093/rheumatology/keaf502. [Abstract]
  2. Ardalan K, Marques MC, Cella D, Curran ML, Gray EL, Lee J, Fahey KJ, Wolfe ML, Pachman LM, Chang RW. Psychometric properties of patient-reported outcomes measurement information system (PROMIS) fixed short forms in Juvenile Myositis. Semin Arthritis Rheum. 2025 Apr;71:152649. doi: 10.1016/j.semarthrit.2025.152649. Epub 2025 Feb 1. [Abstract]
  3. Patel RN, Esparza VG, Lai JS, Gray EL, Reeve BB, Chang RW, Cella D, Ardalan K. Comparison of Patient-Reported Outcomes Measurement Information System Computerized Adaptive Testing Versus Fixed Short Forms in Juvenile Myositis. Arthritis Care Res (Hoboken). 2023 Feb;75(2):381-390. doi: 10.1002/acr.24760. Epub 2022 Aug 31. [Abstract]
  4. Neely J, Ardalan K, Huber A, Kim S; Childhood Arthritis and Rheumatology Research Alliance Investigators. Baseline characteristics of children with juvenile dermatomyositis enrolled in the first year of the new Childhood Arthritis and Rheumatology Research Alliance registry. Pediatr Rheumatol Online J. 2022 Jul 19;20(1):50. doi: 10.1186/s12969-022-00709-3. [Abstract]
  5. Carle AC, Bevans KB, Tucker CA, Forrest CB. Using nationally representative percentiles to interpret PROMIS pediatric measures. Qual Life Res. 2021 Apr;30(4):997-1004. doi: 10.1007/s11136-020-02700-5. Epub 2020 Nov 17. [Abstract]
  6. Craig J, Feldman BM, Spiegel L, Dover S. Comparing the Measurement Properties and Preferability of Patient-reported Outcome Measures in Pediatric Rheumatology: PROMIS vs CHAQ. J Rheumatol. 2021 Jul;48(7):1065-1072. doi: 10.3899/jrheum.200943. Epub 2020 Dec 1. [Abstract]
  7. Kountz-Edwards S, Aoki C, Gannon C, Gomez R, Cordova M, Packman W. The family impact of caring for a child with juvenile dermatomyositis. Chronic Illn. 2017 Dec;13(4):262-274. doi: 10.1177/1742395317690034. Epub 2017 Jan 30. [Abstract]
  8. Butbul Aviel Y, Stremler R, Benseler SM, Cameron B, Laxer RM, Ota S, Schneider R, Spiegel L, Stinson JN, Tse SM, Feldman BM. Sleep and fatigue and the relationship to pain, disease activity and quality of life in juvenile idiopathic arthritis and juvenile dermatomyositis. Rheumatology (Oxford). 2011 Nov;50(11):2051-60. doi: 10.1093/rheumatology/ker256. Epub 2011 Aug 25. [Abstract]
  9. Apaz MT, Saad-Magalhães C, Pistorio A, Ravelli A, de Oliveira Sato J, Marcantoni MB, Meiorin S, Filocamo G, Pilkington C, Maillard S, Al-Mayouf S, Prahalad S, Fasth A, Joos R, Schikler K, Mozolova D, Landgraf JM, Martini A, Ruperto N; Paediatric Rheumatology International Trials Organisation.Health-related quality of life of patients with juvenile dermatomyositis: results from the Pediatric Rheumatology International Trials Organisation multinational quality of life cohort study. Arthritis Rheum. 2009 Apr 15;61(4):509-17. doi: 10.1002/art.24343. [Abstract]
  10. Ruperto N, Ravelli A, Pistorio A, Ferriani V, Calvo I, Ganser G, Brunner J, Dannecker G, Silva CA, Stanevicha V, Cate RT, van Suijlekom-Smit LW, Voygioyka O, Fischbach M, Foeldvari I, Hilario O, Modesto C, Saurenmann RK, Sauvain MJ, Scheibel I, Sommelet D, Tambic-Bukovac L, Barcellona R, Brik R, Ehl S, Jovanovic M, Rovensky J, Bagnasco F, Lovell DJ, Martini A; Paediatric Rheumatology International Trials Organisation (PRINTO); Pediatric Rheumatology Collaborative Study Group (PRCSG). The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective validation study. Arthritis Rheum. 2008 Jan 15;59(1):4-13. doi: 10.1002/art.23248. [Abstract]
  11. Varni JW, Seid M, Smith Knight T, Burwinkle T, Brown J, Szer IS. The PedsQL in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory Generic Core Scales and Rheumatology Module. Arthritis Rheum. 2002 Mar;46(3):714-25. doi: 10.1002/art.10095. [Abstract]
  12. Huber AM, Hicks JE, Lachenbruch PA, Perez MD, Zemel LS, Rennebohm RM, Wallace CA, Lindsley CB, Passo MH, Ballinger SH, Bowyer SL, Reed AM, White PH, Katona IM, Miller FW, Rider LG, Feldman BM; Juvenile Dermatomyositis Disease Activity Collaborative Study Group. Validation of the Childhood Health Assessment Questionnaire in the juvenile idiopathic myopathies. Juvenile Dermatomyositis Disease Activity Collaborative Study Group. J Rheumatol. 2001 May;28(5):1106-11. PMID: 11361197. [Abstract]
  13. Ruperto N, Ravelli A, Pistorio A, Malattia C, Cavuto S, Gado-West L, Tortorelli A, Landgraf JM, Singh G, Martini A; Paediatric Rheumatology International Trials Organisation. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clin Exp Rheumatol. 2001 Jul-Aug;19(4 Suppl 23):S1-9. [Abstract]
  14. Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum. 1994 Dec;37(12):1761-9. doi: 10.1002/art.1780371209. [Abstract]