POS1022 EVALUATION OF PATIENT-REPORTED OUTCOMES FOR PATIENTS WITH MYOSITIS-ASSOCIATED INTERSTITIAL LUNG DISEASE: A JAPANESE PROSPECTIVE MULTICENTRE REGISTRY STUDY

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Background: Idiopathic inflammatory myopathies (IIMs) are a heterogeneous disease characterized by autoimmune myositis and coexistence of extramuscular manifestations such as arthritis, skin rash, interstitial lung disease (ILD), myocarditis, and gastrointestinal involvement [1]. ILD is a leading cause of mortality for patients with IIMs, and the screening and management of ILD are crucial in daily practice [2]. Patient-reported outcome (PRO) provides reports from patients about their own health, quality of life, or functional status associated with the health care or treatment they have received [3]. In general, PRO closely correlates with disease activity and is significantly associated with treatment outcome. Thus, the measurement of PRO is useful for the evaluation of disease status and treatment response in daily practice as well as in clinical trials. However, PROs which reflect disease activity and severity have not been established yet for patients with IIM-associated ILD (myositis-ILD). Objectives: We investigated correlations between PROs at baseline which are validated for idiopathic interstitial pneumonia, patient global assessment with VAS (PGA) for IIMs or EuroQol 5 dimensions 5-level (EQ-5D-5L) and clinical parameters including physician assessment for disease activity, severity of ILD, and recognized prognostic factors. Methods: We prospectively enrolled incident cases with myositis-ILD in our multicentre registry cohort of Japanese patients with myositis-ILD, JAMI, from October 2021 to December 2023. Out of this database, we obtained epidemiological and clinical characteristics data: age, gender, dust exposure, smoking, disease duration at diagnosis of IIM, subclassification of IIM based on clinical diagnosis, disease behavior of ILD, fever, skin rash, muscle weakness, arthralgia/arthritis, and Raynaud's phenomenon; manual muscle testing 8 (MMT-8), biomarkers: creatin kinase (CK), aldolase, C-reactive protein (CRP), erythrocyte sedimentation rate, ferritin, Krebs von den Lungen-6, and surfactant protein D; myositis specific autoantibody: anti-synthetase and anti-melanoma differentiation-associated gene 5 (MDA5) measured by ELISA; blood gas analysis: alveolar-arterial oxygen difference and PaO 2 /FiO 2 (P/F) ratio; pulmonary function test (PFT): percentage of predicted forced vital capacity (%FVC), percent predicted diffusing capacity of the lung for carbon monoxide, and the ratio of residual volume to total lung capacity; and 6-minute walk distance (6MWD) and lowest saturation of percutaneous oxygen. In terms of PRO, modified medical research council dyspnea scale (mMRC), chronic obstructive pulmonary disease assessment test (CAT), cough visual analog scale (VAS), King's brief ILD (K-BILD), PGA, and EQ-5D-5L were employed in this study. We evaluated correlations between individual PROs and physician assessment for disease activity or serum biomarkers and pulmonary-relevant variables which are associated with poor outcomes using Spearman's correlation. SPSS version 29.0.2.0 (IBM, New York, US) was used for all-analysis. Results: We enrolled 316 patients who responded to PROs with the median age (standard deviation) of 59 (14) years and 69% female. The median disease duration at diagnosis was 1 month. The diagnosis of IIMs was dermatomyositis (DM) in 100 patients, amyopathic DM in 70, anti-synthetase syndrome in 82, polymyositis in 40, immune-mediated necrotizing myopathy in 2, overlap myositis in 6. Disease behavior of ILD was acute/subacute in 158 patients, chronic in 98, and unclassifiable in 44. Anti-synthetase-positive, anti-MDA5-positive, and double autoantibodies-negative were revealed in 167, 70, and 46 patients, respectively. mMRC, CAT, and K-BILD significantly (P<0.001), but weakly correlated with physician lung assessment, P/F ratio, and %FVC (Figure 1A). On the other hand, cough VAS did not closely correlate with physician lung assessment and PFT variables. PGA weakly (P<0.001) correlated with physician global assessment, MMT-8, and serum levels of CRP and ferritin, but less significantly (P<0.05) with physician lung assessment and serum CK levels. EQ-5D-5L weakly (P<0.001) correlated with physician global assessment, physician lung assessment, MMT-8, serum levels of CRP and ferritin, P/F ratio, and %FVC. Among PROs, CAT moderately (P<0.001) correlated with other PROs except for mMRC (Figure 1B). PGA had weak correlations with mMRC or cough VAS. Conclusion: Existing PROs did not strongly correlate with severity of ILD or prognostic biomarkers for myositis-ILD patients who have unique clinical phenotypes and courses. It is considered necessary to develop a novel PRO specifically for myositis-ILD patients. REFERENCES: [1] Lundberg IE, Fujimoto M, Vencovsky J, Aggarwal R, Holmqvist M, Christopher-Stine L, et al. Idiopathic inflammatory myopathies. Nat Rev Dis Primers. 2021;7(1):86. [2] Gono T, Kuwana M. New paradigm in the treatment of myositis-associated interstitial lung disease. Expert Rev Respir Med. 2023;17(5):397-411. [3] Weldring T, Smith SM. Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs). Health Serv Insights. 2013;6:61-8. Figure 1Correlations between each PRO and myositis disease activity indicators, serum biomarkers or pulmonary-relevant variables and between individual PROs. Correlations between each PRO and myositis disease activity indicators, serum biomarkers or pulmonary-relevant variables (A) and between the PROs (B) were estimated using Spearman's correlation. *P<0.05, **P<0.01, ***P<0.001. The magnitude of correlation: negligible, 0.0 - 0.09; weak, 0.1 - 0.39, moderate, 0.4 - 0.69; strong, 0.7 - 0.89; very strong, 0.9 - 1.0. Acknowledgements: This study was supported by Japan Agency for Medical Research and Development (23ek0109531h0003). Disclosure of Interests: Takahisa Gono Boehringer-Ingelheim, Kenichi Masui Aspen Japan K.K., Sandoz K.K., Terumo Corporation, Maruishi Pharmaceutical Co., Ltd, Mundipharma K.K., Masimo Japan Corporation, Covidien Japan Inc., Pfizer Japan Inc., and MSD K.K., Mundipharma K.K., Terumo Corporation, and Nihon Kohden Corporation, Toshimasa Shimizu AbbVie, ONO, Astellas, Takafumi Suda: None declared, Shinji Sato Eisai Pharma, Abbvie Inc., GlaxoSmithKline, Eli lilly, Pfizer, Astellas Pharma Inc., Nippon Boehringer Ingelheim Co., Ltd., Eisai Pharma, Tomoaki Higuchi Chugai Pharmaceutical Co., Ltd., Pfizer Japan Inc., Boehringer Ingelheim Japan, Inc., Asahi Kasei Corp., Gilead Sciences Inc., Eli Lilly Japan K.K., Janssen Pharmaceutical K.K., RIBOMIC Inc., Ayumi Pharmaceutical Corp., Asahi Kasei Corp., Taisho Pharmaceutical Co., Ltd., Mochida Pharmaceutical Co., Ltd., Chugai Pharmaceutical Co., Ltd., AbbVie Japan GK, Takeshi Johkoh: None declared, Tomoaki Hoshino: None declared, Naoki Shimada: None declared, Yasuhiro Kondoh Asahi Kasei Pharma Corp, Bristol Myers Squibb, Boehringer Ingelheim, Eisai Co, Ltd, Janssen Pharmaceutical K.K., KYORIN Pharmaceutical Co, Ltd, Mitsubishi Tanabe Pharma, NIPPON SHINYAKU CO., LTD, Novartis Pharma KK, Shionogi Co, Ltd., Teijin Pharma Ltd., Asahi Kasei Pharma Corp, Boehringer Ingelheim, Chugai Pharmaceutical Co., Ltd., Healios K.K., Janssen Pharmaceutical KK, Shionogi Co, Ltd., Taiho Pharmaceutical Co., Yoshinori Tanino: None declared, Tohru Takeuchi: None declared, Yoshiyuki Abe: None declared, Shinsuke Yasuda Abbvie, Asahi Kasei Pharma, Chugai Pharmaceutical, Eisai, Eli Lilly, GlaxoSmithKline, Mitsubishi Tanabe Pharma, and Ono pharmaceutical., Eisai, ImmunoForge, Novartis and Otsuka seiyaku., Asahi Kasei Pharma, Ayumi Pharmaceutical Corporation, Chugai Pharmaceutical, Nippon Boehringer Ingelheim Co., Ltd, and Taisho Pharmaceutical Co., Ltd, Toru Arai Boehringer Ingelheim, Shionogi, AstraZeneka, Sekisui Medical, Sekisui Medical, Sysmex, Yasushi Inoue: None declared, Keisuke Tomii: None declared, Kunihiro Yamaoka: None declared, Kaneshige Sasaki: None declared, Taio Naniwa: None declared, Akio Morinobu: None declared, O

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