Kastriot Kastrati, Thomas Karonitsch, Hanien Rajab, Roman Reindl-Schwaighofer, Farsad Eskandary, Sahra Pajenda, Daniel Mrak, Peter Maximilian Heil, Hans Peter Kiener, Michael Bonelli, Kurt Derfler, Daniel Aletaha, Josef S Smolen, Helga Radner
{"title":"免疫吸附作为一种治疗难治性特发性炎性肌病的新方法——一项回顾性观察研究","authors":"Kastriot Kastrati, Thomas Karonitsch, Hanien Rajab, Roman Reindl-Schwaighofer, Farsad Eskandary, Sahra Pajenda, Daniel Mrak, Peter Maximilian Heil, Hans Peter Kiener, Michael Bonelli, Kurt Derfler, Daniel Aletaha, Josef S Smolen, Helga Radner","doi":"10.1093/rheumatology/keaf289","DOIUrl":null,"url":null,"abstract":"Objective Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterised by muscle inflammation, high creatine kinase (CK) levels, and disability. Despite immunomodulating therapies, patients often experience progressive disease, resulting in refractory conditions or dependence on glucocorticoids (GC). This study addresses the efficacy and safety of immunoadsorption (IAS) in refractory IIM. Methods A retrospective, monocentric study was conducted on patients with highly active IIM subjected to IAS between January 2000 and September 2021. Inclusion criteria were adult patients with confirmed IIM unresponsive to standard therapies, including GC and at least one disease-modifying antirheumatic drug. Primary end point was defined as minor improvement, requiring a ≥ 20% reduction in both daily GC-dosage and CK-levels at week 12, alongside patient comparative self-assessment (PCSA) rated as “no change” or “better.” Secondary endpoints included moderate (≥ 40%) and major (≥ 60%) improvements at weeks 4, 8, and 12. Relapse rates were assessed over a 3-month period following the last IAS procedure. Results The study included 23 refractory IIM patients treated with IAS. Primary end point was reached by 52.2% (n = 12) patients. Moderate improvement was observed in 8.7% (n = 2), 26.1% (n = 6), and 34.8% (n = 8) and major improvement in 8.7% (n = 2), 17.4% (n = 4), and 30.4% (n = 7) at weeks 4, 8, and 12, respectively. No unexpected adverse events were reported, with low relapse rates within 3 months post-IAS (18%). Conclusion IAS may be an effective adjunctive therapy in patients refractory to GC and conventional treatments, leading to rapid CK level reductions, decreased GC usage, and improvements in PCSA and muscle function.","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":"14 1","pages":""},"PeriodicalIF":4.7000,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Immunoadsorption as a novel therapy for refractory idiopathic inflammatory myopathies—a retrospective observational study\",\"authors\":\"Kastriot Kastrati, Thomas Karonitsch, Hanien Rajab, Roman Reindl-Schwaighofer, Farsad Eskandary, Sahra Pajenda, Daniel Mrak, Peter Maximilian Heil, Hans Peter Kiener, Michael Bonelli, Kurt Derfler, Daniel Aletaha, Josef S Smolen, Helga Radner\",\"doi\":\"10.1093/rheumatology/keaf289\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Objective Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterised by muscle inflammation, high creatine kinase (CK) levels, and disability. Despite immunomodulating therapies, patients often experience progressive disease, resulting in refractory conditions or dependence on glucocorticoids (GC). This study addresses the efficacy and safety of immunoadsorption (IAS) in refractory IIM. Methods A retrospective, monocentric study was conducted on patients with highly active IIM subjected to IAS between January 2000 and September 2021. Inclusion criteria were adult patients with confirmed IIM unresponsive to standard therapies, including GC and at least one disease-modifying antirheumatic drug. Primary end point was defined as minor improvement, requiring a ≥ 20% reduction in both daily GC-dosage and CK-levels at week 12, alongside patient comparative self-assessment (PCSA) rated as “no change” or “better.” Secondary endpoints included moderate (≥ 40%) and major (≥ 60%) improvements at weeks 4, 8, and 12. Relapse rates were assessed over a 3-month period following the last IAS procedure. Results The study included 23 refractory IIM patients treated with IAS. Primary end point was reached by 52.2% (n = 12) patients. Moderate improvement was observed in 8.7% (n = 2), 26.1% (n = 6), and 34.8% (n = 8) and major improvement in 8.7% (n = 2), 17.4% (n = 4), and 30.4% (n = 7) at weeks 4, 8, and 12, respectively. No unexpected adverse events were reported, with low relapse rates within 3 months post-IAS (18%). Conclusion IAS may be an effective adjunctive therapy in patients refractory to GC and conventional treatments, leading to rapid CK level reductions, decreased GC usage, and improvements in PCSA and muscle function.\",\"PeriodicalId\":21255,\"journal\":{\"name\":\"Rheumatology\",\"volume\":\"14 1\",\"pages\":\"\"},\"PeriodicalIF\":4.7000,\"publicationDate\":\"2025-05-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Rheumatology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1093/rheumatology/keaf289\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"RHEUMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Rheumatology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1093/rheumatology/keaf289","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
Immunoadsorption as a novel therapy for refractory idiopathic inflammatory myopathies—a retrospective observational study
Objective Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterised by muscle inflammation, high creatine kinase (CK) levels, and disability. Despite immunomodulating therapies, patients often experience progressive disease, resulting in refractory conditions or dependence on glucocorticoids (GC). This study addresses the efficacy and safety of immunoadsorption (IAS) in refractory IIM. Methods A retrospective, monocentric study was conducted on patients with highly active IIM subjected to IAS between January 2000 and September 2021. Inclusion criteria were adult patients with confirmed IIM unresponsive to standard therapies, including GC and at least one disease-modifying antirheumatic drug. Primary end point was defined as minor improvement, requiring a ≥ 20% reduction in both daily GC-dosage and CK-levels at week 12, alongside patient comparative self-assessment (PCSA) rated as “no change” or “better.” Secondary endpoints included moderate (≥ 40%) and major (≥ 60%) improvements at weeks 4, 8, and 12. Relapse rates were assessed over a 3-month period following the last IAS procedure. Results The study included 23 refractory IIM patients treated with IAS. Primary end point was reached by 52.2% (n = 12) patients. Moderate improvement was observed in 8.7% (n = 2), 26.1% (n = 6), and 34.8% (n = 8) and major improvement in 8.7% (n = 2), 17.4% (n = 4), and 30.4% (n = 7) at weeks 4, 8, and 12, respectively. No unexpected adverse events were reported, with low relapse rates within 3 months post-IAS (18%). Conclusion IAS may be an effective adjunctive therapy in patients refractory to GC and conventional treatments, leading to rapid CK level reductions, decreased GC usage, and improvements in PCSA and muscle function.
期刊介绍:
Rheumatology strives to support research and discovery by publishing the highest quality original scientific papers with a focus on basic, clinical and translational research. The journal’s subject areas cover a wide range of paediatric and adult rheumatological conditions from an international perspective. It is an official journal of the British Society for Rheumatology, published by Oxford University Press.
Rheumatology publishes original articles, reviews, editorials, guidelines, concise reports, meta-analyses, original case reports, clinical vignettes, letters and matters arising from published material. The journal takes pride in serving the global rheumatology community, with a focus on high societal impact in the form of podcasts, videos and extended social media presence, and utilizing metrics such as Altmetric. Keep up to date by following the journal on Twitter @RheumJnl.