Chiara Tani, Chiara Cardelli, Margherita Zen, Luca Moroni, Matteo Piga, Fulvia Ceccarelli, Serena Fasano, Ginevra De Marchi, Laura Coladonato, Giacomo Emmi, Mariele Gatto, Francesca Trentin, Giuseppe A Ramirez, Elisabetta Chessa, Gabriele Gallina, Licia Picciariello, Martina Patrone, Maria L Urban, Edoardo Biancalana, Luca Quartuccio, Francesco Ciccia, Fabrizio Conti, Alberto Cauli, Lorenzo Dagna, Andrea Doria, Marta Mosca
{"title":"阿尼洛单抗治疗难治性系统性红斑狼疮:一项真实的多中心研究","authors":"Chiara Tani, Chiara Cardelli, Margherita Zen, Luca Moroni, Matteo Piga, Fulvia Ceccarelli, Serena Fasano, Ginevra De Marchi, Laura Coladonato, Giacomo Emmi, Mariele Gatto, Francesca Trentin, Giuseppe A Ramirez, Elisabetta Chessa, Gabriele Gallina, Licia Picciariello, Martina Patrone, Maria L Urban, Edoardo Biancalana, Luca Quartuccio, Francesco Ciccia, Fabrizio Conti, Alberto Cauli, Lorenzo Dagna, Andrea Doria, Marta Mosca","doi":"10.3899/jrheum.2024-0053","DOIUrl":null,"url":null,"abstract":"<p><strong>Objective: </strong>To report real-world experience on the use of anifrolumab (ANI) in refractory systemic lupus erythematosus (SLE).</p><p><strong>Methods: </strong>The present study is a multicenter, retrospective study involving 9 Italian SLE referral centers participating in a compassionate use program for the use of ANI in adult patients with active SLE in whom all the available treatment choices failed, were not tolerated, or were contraindicated. At baseline and 1, 3, 6, 9, and 12 months of treatment, overall and organ-specific disease activity, flares, daily glucocorticoid (GC) dose, and adverse events were recorded.</p><p><strong>Results: </strong>A total of 26 patients were enrolled. At 4 weeks after starting ANI, a significant decrease in the Systemic Lupus Erythematosus Disease Activity Index 2000 (<i>P</i> = 0.01), Systemic Lupus Erythematosus-Disease Activity Score (<i>P</i> = 0.01), and physician global assessment (<i>P</i> = 0.001) was recorded, and the same trend was maintained over time. A significant reduction in Cutaneous Lupus Erythematosus Disease Area and Severity Index-activity (<i>P</i> < 0.001) and in tender (<i>P</i> = 0.03) and swollen (<i>P</i> = 0.02) joint counts was also recorded. At 3 months of follow-up, 33% of patients already achieved a remission state, whereas 46% were in Lupus Low Disease Activity State (LLDAS); at 6 months, 50% were in remission and 80% were in LLDAS. A significant reduction in the mean GC daily dose was observed, starting from week 4 (<i>P</i> = 0.04). A total of 4 disease flares according to the Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index were recorded (3 mild-moderate and 1 severe). Overall, 4/20 patients with at least 24 weeks of follow-up (20%) were considered nonresponders.</p><p><strong>Conclusion: </strong>This study provides real-world experience on the use of ANI in patients with refractory SLE, confirming its rapid effectiveness and an overall acceptable safety profile.</p>","PeriodicalId":50064,"journal":{"name":"Journal of Rheumatology","volume":null,"pages":null},"PeriodicalIF":3.6000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Anifrolumab in Refractory Systemic Lupus Erythematosus: A Real-World, Multicenter Study.\",\"authors\":\"Chiara Tani, Chiara Cardelli, Margherita Zen, Luca Moroni, Matteo Piga, Fulvia Ceccarelli, Serena Fasano, Ginevra De Marchi, Laura Coladonato, Giacomo Emmi, Mariele Gatto, Francesca Trentin, Giuseppe A Ramirez, Elisabetta Chessa, Gabriele Gallina, Licia Picciariello, Martina Patrone, Maria L Urban, Edoardo Biancalana, Luca Quartuccio, Francesco Ciccia, Fabrizio Conti, Alberto Cauli, Lorenzo Dagna, Andrea Doria, Marta Mosca\",\"doi\":\"10.3899/jrheum.2024-0053\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objective: </strong>To report real-world experience on the use of anifrolumab (ANI) in refractory systemic lupus erythematosus (SLE).</p><p><strong>Methods: </strong>The present study is a multicenter, retrospective study involving 9 Italian SLE referral centers participating in a compassionate use program for the use of ANI in adult patients with active SLE in whom all the available treatment choices failed, were not tolerated, or were contraindicated. At baseline and 1, 3, 6, 9, and 12 months of treatment, overall and organ-specific disease activity, flares, daily glucocorticoid (GC) dose, and adverse events were recorded.</p><p><strong>Results: </strong>A total of 26 patients were enrolled. At 4 weeks after starting ANI, a significant decrease in the Systemic Lupus Erythematosus Disease Activity Index 2000 (<i>P</i> = 0.01), Systemic Lupus Erythematosus-Disease Activity Score (<i>P</i> = 0.01), and physician global assessment (<i>P</i> = 0.001) was recorded, and the same trend was maintained over time. A significant reduction in Cutaneous Lupus Erythematosus Disease Area and Severity Index-activity (<i>P</i> < 0.001) and in tender (<i>P</i> = 0.03) and swollen (<i>P</i> = 0.02) joint counts was also recorded. At 3 months of follow-up, 33% of patients already achieved a remission state, whereas 46% were in Lupus Low Disease Activity State (LLDAS); at 6 months, 50% were in remission and 80% were in LLDAS. A significant reduction in the mean GC daily dose was observed, starting from week 4 (<i>P</i> = 0.04). A total of 4 disease flares according to the Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index were recorded (3 mild-moderate and 1 severe). Overall, 4/20 patients with at least 24 weeks of follow-up (20%) were considered nonresponders.</p><p><strong>Conclusion: </strong>This study provides real-world experience on the use of ANI in patients with refractory SLE, confirming its rapid effectiveness and an overall acceptable safety profile.</p>\",\"PeriodicalId\":50064,\"journal\":{\"name\":\"Journal of Rheumatology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":3.6000,\"publicationDate\":\"2024-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Rheumatology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.3899/jrheum.2024-0053\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"RHEUMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Rheumatology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3899/jrheum.2024-0053","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
目的:报告在难治性系统性红斑狼疮中使用ANI的实际经验:报告在难治性系统性红斑狼疮中使用ANI的实际经验:多中心回顾性研究:9个意大利系统性红斑狼疮转诊中心参与了一项同情性用药计划,在所有可用治疗方案均无效、不能耐受或有禁忌症的活动性成年系统性红斑狼疮患者中使用ANI:共有26名患者入组。在开始 ANI 治疗 4 周后,SLEDAI-2K(p=0.005)、SLEDAS(p=0.005)和 PGA(p=0.001)均显著下降,并随着时间的推移保持同样的趋势。CLASI活动明显减少(p结论:这项研究提供了在难治性系统性红斑狼疮患者中使用ANI的实际经验,证实了ANI的快速有效性和总体可接受的安全性。
Anifrolumab in Refractory Systemic Lupus Erythematosus: A Real-World, Multicenter Study.
Objective: To report real-world experience on the use of anifrolumab (ANI) in refractory systemic lupus erythematosus (SLE).
Methods: The present study is a multicenter, retrospective study involving 9 Italian SLE referral centers participating in a compassionate use program for the use of ANI in adult patients with active SLE in whom all the available treatment choices failed, were not tolerated, or were contraindicated. At baseline and 1, 3, 6, 9, and 12 months of treatment, overall and organ-specific disease activity, flares, daily glucocorticoid (GC) dose, and adverse events were recorded.
Results: A total of 26 patients were enrolled. At 4 weeks after starting ANI, a significant decrease in the Systemic Lupus Erythematosus Disease Activity Index 2000 (P = 0.01), Systemic Lupus Erythematosus-Disease Activity Score (P = 0.01), and physician global assessment (P = 0.001) was recorded, and the same trend was maintained over time. A significant reduction in Cutaneous Lupus Erythematosus Disease Area and Severity Index-activity (P < 0.001) and in tender (P = 0.03) and swollen (P = 0.02) joint counts was also recorded. At 3 months of follow-up, 33% of patients already achieved a remission state, whereas 46% were in Lupus Low Disease Activity State (LLDAS); at 6 months, 50% were in remission and 80% were in LLDAS. A significant reduction in the mean GC daily dose was observed, starting from week 4 (P = 0.04). A total of 4 disease flares according to the Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index were recorded (3 mild-moderate and 1 severe). Overall, 4/20 patients with at least 24 weeks of follow-up (20%) were considered nonresponders.
Conclusion: This study provides real-world experience on the use of ANI in patients with refractory SLE, confirming its rapid effectiveness and an overall acceptable safety profile.
期刊介绍:
The Journal of Rheumatology is a monthly international serial edited by Earl D. Silverman. The Journal features research articles on clinical subjects from scientists working in rheumatology and related fields, as well as proceedings of meetings as supplements to regular issues. Highlights of our 41 years serving Rheumatology include: groundbreaking and provocative editorials such as "Inverting the Pyramid," renowned Pediatric Rheumatology, proceedings of OMERACT and the Canadian Rheumatology Association, Cochrane Musculoskeletal Reviews, and supplements on emerging therapies.