Development of the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA): A De Novo IGA of Cutaneous Manifestations of Dermatomyositis.
Clinical outcome assessment
Cutaneous manifestations
Dermatomyositis
Investigator global assessment
Journal
Dermatology and therapy
ISSN: 2193-8210
Titre abrégé: Dermatol Ther (Heidelb)
Pays: Switzerland
ID NLM: 101590450
Informations de publication
Date de publication:
08 Jul 2024
08 Jul 2024
Historique:
received:
14
05
2024
accepted:
21
06
2024
medline:
8
7
2024
pubmed:
8
7
2024
entrez:
8
7
2024
Statut:
aheadofprint
Résumé
Dermatomyositis (DM) is a rare systemic autoimmune disease characterized by a distinctive debilitating skin rash and skeletal muscle weakness. It is unclear if existing clinical outcome assessment (COA) measures include the concepts of priority to patients and those necessary to fully capture improvements in the active cutaneous manifestations of DM. This study aimed to develop the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA), a de novo IGA, for use in clinical trials of adult DM. Eight DM clinical experts participated in 60-min qualitative interviews consisting of concept elicitation and cognitive debriefing methodologies. Concept elicitation comprised open-ended questions with follow-up probes to explore clinicians' experiences of treating patients with DM, the impact of symptoms on patients' quality of life, and the severity levels of disease characteristics to explore DM progression. Cognitive debriefing required the clinical experts to perform a review of the CDM-IGA, designed to assess the severity of cutaneous disease activity of DM. After the interviews, a consensus meeting with three clinical experts was held to agree on any outstanding issues relating to the CDM-IGA. The CDM-IGA was iteratively developed using the opinions of nine clinical experts. Feedback provided by all clinicians agreed that erythema was the main active cutaneous manifestation of DM and should be the primary characteristic on the CDM-IGA, split by erythema color and extent. To determine cutaneous disease severity, experts suggested adding a metric called secondary changes, which combined erosion/ulceration and lichenification, which could modify the patient's final score. Three clinical experts suggested that a photo-guide to support assessments of erythema across different skin tones could be beneficial. A novel CDM-IGA was developed for use with adult patients with DM in clinical trials, based on an iterative development process that combined qualitative feedback from clinical experts of DM and importantly adult patients living with DM.
Identifiants
pubmed: 38976170
doi: 10.1007/s13555-024-01220-1
pii: 10.1007/s13555-024-01220-1
doi:
Types de publication
Journal Article
Langues
eng
Informations de copyright
© 2024. The Author(s).
Références
Cobos GA, Femia A, Vleugels RA. Dermatomyositis: an update on diagnosis and treatment. Am J Clin Dermatol. 2020;21(3):339–53.
doi: 10.1007/s40257-020-00502-6
pubmed: 32096127
Isak V, Jorizzo JL. Recent developments on treatment strategies and the prognosis of dermatomyositis: a review. J Dermatolog Treat. 2018;29(5):450–9.
doi: 10.1080/09546634.2017.1403549
pubmed: 29160756
Patil A, Lu J, Kassir M, Babaei M, Goldust M. Adult and juvenile dermatomyositis treatment. J Cosmet Dermatol. 2023;22(2):395–401.
doi: 10.1111/jocd.15363
pubmed: 36065712
Mayo Clinic. Dermatomyositis. https://www.mayoclinic.org/diseases-conditions/dermatomyositis/symptoms-causes/syc-20353188 Accessed Jun 2023.
National Organization for Rare Disorders. Dermatomyositis. https://rarediseases.org/rare-diseases/dermatomyositis/ Accessed Jun 2023.
Hu T, Vinik O. Dermatomyositis and malignancy. Can Fam Physician. 2019;65(6):409–11.
pubmed: 31189628
pmcid: 6738379
Olazagasti JM, Baez PJ, Wetter DA, Ernste FC. Cancer risk in dermatomyositis: a meta-analysis of cohort studies. Am J Clin Dermatol. 2015;16(2):89–98.
doi: 10.1007/s40257-015-0120-1
pubmed: 25721032
Buchbinder R, Forbes A, Hall S, Dennett X, Giles G. Incidence of malignant disease in biopsy-proven inflammatory myopathy. A population-based cohort study. Ann Intern Med. 2001;134(12):1087–95.
doi: 10.7326/0003-4819-134-12-200106190-00008
pubmed: 11412048
Okogbaa J, Batiste L. Dermatomyositis: an acute flare and current treatments. Clin Med Insights Case Rep. 2019;12:1179547619855370.
doi: 10.1177/1179547619855370
pubmed: 31244526
pmcid: 6582284
Livermore P, Gray S, Mulligan K, Stinson JN, Wedderburn LR, Gibson F. Being on the juvenile dermatomyositis rollercoaster: a qualitative study. Pediatr Rheumatol. 2019;17(1):1–9.
doi: 10.1186/s12969-019-0332-7
Chandra T, Aggarwal R. Clinical trials and novel therapeutics in dermatomyositis. Expert Opin Emerg Drugs. 2020;25(3):213–28.
doi: 10.1080/14728214.2020.1787985
pubmed: 32597690
Bogdanov I, Kazandjieva J, Darlenski R, Tsankov N. Dermatomyositis: current concepts. Clin Dermatol. 2018;36(4):450–8.
doi: 10.1016/j.clindermatol.2018.04.003
pubmed: 30047429
Miller FW, Rider LG, Chung YL, et al. Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology. 2001;40(11):1262–73.
doi: 10.1093/rheumatology/40.11.1262
pubmed: 11709610
Rider LG, Werth VP, Huber AM, et al. 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;63(Suppl 11):S118–57.
pubmed: 22588740
Rider LG, Aggarwal R, Machado PM, et al. Update on outcome assessment in myositis. Nat Rev Rheumatol. 2018;14(5):303–18.
doi: 10.1038/nrrheum.2018.33
pubmed: 29651119
pmcid: 6702032
Aggarwal R, Rider LG, Ruperto N, et al. 2016 American College of Rheumatology/European League Against Rheumatism criteria for minimal, moderate, and major clinical response in adult dermatomyositis and polymyositis: an International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative. Ann Rheum Dis. 2017;76(5):792–801.
doi: 10.1136/annrheumdis-2017-211400
pubmed: 28385805
Anyanwu CO, Fiorentino DF, Chung L, et al. Validation of the cutaneous dermatomyositis disease area and severity index: characterizing disease severity and assessing responsiveness to clinical change. Br J Dermatol. 2015;173(4):969–74.
doi: 10.1111/bjd.13915
pubmed: 25994337
pmcid: 4878996
Tiao J, Feng R, Bird S, et al. The reliability of the cutaneous dermatomyositis disease area and severity index (CDASI) among dermatologists, rheumatologists and neurologists. Br J Dermatol. 2017;176(2):423–30.
doi: 10.1111/bjd.15140
pubmed: 28004387
Food and Drug Administration (FDA). Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. FDA; 2009.
Food and Drug Administration (FDA). Patient-focused drug development: collecting comprehensive and representative input. Guidance for industry, Food and Drug Administration staff, and other stakeholders. FDA; 2020.
Food and Drug Administration (FDA). Patient-focused drug development: selecting, developing, or modifying fit-for-purpose clinical outcome assessments. Guidance for industry (DRAFT), Food and Drug Administration staff, and other stakeholders. FDA; 2022.
Food and Drug Administration (FDA). Patient-focused drug development: methods to identify what is important to patients. Guidance for industry, Food and Drug Administration staff, and other stakeholders. FDA; 2022.
Food and Drug Administration (FDA). Patient-focused drug development: incorporating clinical outcome assessments into endpoints for regulatory decision-making. Guidance for industry (DRAFT). FDA; 2023.
Food and Drug Administration (FDA). Patient-focused drug development: select, develop or modify fit-for-purpose clinical outcome assessments. Guidance 3 discussion document. FDA; 2018.
Adamantios D. Guidelines for choosing between multi-item and single-item scales for construct measurement: a predictive validity perspective. J Acad Mark Sci. 2012;40:434–49.
doi: 10.1007/s11747-011-0300-3
Acne Vulgaris: Establishing Effectiveness of Drugs Intended for Treatment Guidance for Industry. Food and Drug Administration, Center for Drug Evaluation and Research (CDER). 2018. Available from: https://www.fda.gov/media/71152/download .
Perez-Chada LM, Salame NF, Ford AR, et al. Investigator and patient global assessment measures for psoriasis clinical trials: a systematic review on measurement properties from the International Dermatology Outcome Measures (IDEOM) initiative. Am J Clin Dermatol. 2020;21(3):323–38.
doi: 10.1007/s40257-019-00496-w
pubmed: 31950353
Langley RG, Feldman SR, Nyirady J, van de Kerkhof P, Papavassilis C. The 5-point investigator’s global assessment (IGA) scale: a modified tool for evaluating plaque psoriasis severity in clinical trials. J Dermatolog Treat. 2015;26(1):23–31.
doi: 10.3109/09546634.2013.865009
pubmed: 24354461
Aggarwal R, Charles-Schoeman C, Schessl J, Dimachkie MM, Beckmann I, Levine T. Prospective, double-blind, randomized, placebo-controlled phase III study evaluating efficacy and safety of octagam 10% in patients with dermatomyositis (“ProDERM study”). Medicine (Baltimore). 2021;100(1):e23677.
doi: 10.1097/MD.0000000000023677
pubmed: 33429735
Werth VP, Hejazi E, Pena SM, et al. Safety and efficacy of lenabasum, a cannabinoid receptor type 2 agonist, in patients with dermatomyositis with refractory skin disease: a randomized clinical trial. J Invest Dermatol. 2022;142(10):2651-9.e1.
doi: 10.1016/j.jid.2022.03.029
pubmed: 35490744
pmcid: 10226779
Jensen E, Jones N, Rabe M, et al. The chance that two people chosen at random are of different race or ethnicity groups has increased since 2010. United States Census Bureau. 2021.