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.

Currently available quality of life tools, as well as their validation, and performance characteristics, have been reviewed in the following articles:

• 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. [Abstract 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.]
• 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.PMID: 29651119. [Abstract 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.PMID: 29651119.]
• Rider LG, Werth VP, Huber AM, Alexanderson H, Rao AP, Ruperto N, Herbelin L, Barohn R, Isenberg D, Miller FW. Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), physician global damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI). Arthritis Care Res (Hoboken). 2011 Nov;63 Suppl 11:S118-57. doi: 10.1002/acr.20532. Review. [Abstract Rider LG, Werth VP, Huber AM, Alexanderson H, Rao AP, Ruperto N, Herbelin L, Barohn R, Isenberg D, Miller FW. Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), physician global damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI). Arthritis Care Res (Hoboken). 2011 Nov;63 Suppl 11:S118-57. doi: 10.1002/acr.20532. Review.]
• 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. [Abstract 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.] 
• 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. eCollection 2016. [Abstract 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. eCollection 2016.] 

Health Related Quality of Life using SF-36/CHQ, PedsQL

Description:

• SF-36 for Adults:
  IMACS suggests using the Original SF-36® in adult myositis clinical trials and studies. For use of the Original SF-36®, please contact Quality Metric for licensing information or visit SF-36v2 Health Survey. The SF-36 is also available in the public domain at the RAND Health organization.
  
  
• Child Health Questionnaire (CHQ-PF50) for Children:
  IMACS suggests using the CHQ-PF50® in myositis clinical trials and studies involving pediatric patients. For use of the CHQ-PF50, contact HealthAct . All users will need to register and purchase the CHQ Manual from HealthAct in order to obtain copies of the form for use in their study. The Health Act website provides information regarding terms of use, content, bibliography, translations etc. All translations will be issued only through HealthAct Inc. Some uses may require a license fee (depending on scope, languages, etc), and this will be determined on a case by case basis.

Links to Licensing Requirements for Tool:

• For the SF-36: Available from the RAND Health organization
• For the CHQ-PF50®: contact HealthAct

References:

1. 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 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.]
2. Ponyi A, Borgulya G, Constantin T, Vancsa A, Gergely L, Danko K. Functional outcome and quality of life in adult patients with idiopathic inflammatory myositis. Rheumatology (Oxford). 2004 Sep 20 [Epub ahead of print] [Pub Med Ponyi A, Borgulya G, Constantin T, Vancsa A, Gergely L, Danko K. Functional outcome and quality of life in adult patients with idiopathic inflammatory myositis. Rheumatology (Oxford). 2004 Sep 20 [Epub ahead of print]]  
3. Sultan SM, Ioannou Y, Moss K, Isenberg DA. Outcome in patients with idiopathic inflammatory myositis: morbidity and mortality. Rheumatology (Oxford). 2002 Jan;41(1):22-6. [Pub Med Sultan SM, Ioannou Y, Moss K, Isenberg DA. Outcome in patients with idiopathic inflammatory myositis: morbidity and mortality. Rheumatology (Oxford). 2002 Jan;41(1):22-6.]  
4. 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. [Pub Med 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.]
5. 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 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.]