Expert Review of Clinical Immunology最新文献_第5页

Safety profile of efgartigimod from global clinical trials across multiple immunoglobulin G-mediated autoimmune diseases. 依加替莫德在多种免疫球蛋白g介导的自身免疫性疾病的全球临床试验中的安全性

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-05-13 DOI: 10.1080/1744666X.2025.2497840

Kelly G Gwathmey, Catherine M Broome, Matthias Goebeler, Hiroyuki Murai, Zsuzsanna Bata-Csörgo, Adrian C Newland, Jeffrey A Allen, Yoshitaka Miyakawa, Peter Ulrichts, Luc Truyen, Jana Podhorna, Rene Kerstens, Sophie Steeland, Jon Beauchamp, Jeffrey T Guptill, James F Howard

{"title":"Safety profile of efgartigimod from global clinical trials across multiple immunoglobulin G-mediated autoimmune diseases.","authors":"Kelly G Gwathmey, Catherine M Broome, Matthias Goebeler, Hiroyuki Murai, Zsuzsanna Bata-Csörgo, Adrian C Newland, Jeffrey A Allen, Yoshitaka Miyakawa, Peter Ulrichts, Luc Truyen, Jana Podhorna, Rene Kerstens, Sophie Steeland, Jon Beauchamp, Jeffrey T Guptill, James F Howard","doi":"10.1080/1744666X.2025.2497840","DOIUrl":"10.1080/1744666X.2025.2497840","url":null,"abstract":"Background: Efgartigimod is approved in multiple regions for the treatment of gMG, ITP, and CIDP, and is being evaluated in multiple IgG-mediated autoimmune diseases. Here, we report the long-term safety profiles of efgartigimod IV and PH20 SC across different dosing regimens and diseases where efgartigimod has received regulatory approval.Research design and methods: Efgartigimod safety was assessed across dosing regimens and administration routes in Phase 2, placebo-controlled Phase 3, and OLE studies in participants with gMG, ITP, and CIDP. Analyses were performed on all participants who received ≥ 1 dose or partial dose of efgartigimod or placebo. Data from efgartigimod-treated participants were pooled per disease. Event rates were calculated as events per PYFU.Results: Pooled data included 715 participants representing > 850 PYFU. In efgartigimod-treated participants, most TEAEs were mild-to-moderate in severity, with consistently low event rates for TEAE-related treatment discontinuation (range: 0.05-0.47). Severe and serious infection rates were comparable between placebo- and efgartigimod-treated participants. Rates of TEAEs, severe and serious infections, and treatment discontinuation did not increase with prolonged efgartigimod exposure. Efgartigimod did not reduce albumin or increase LDL cholesterol levels.Conclusions: Across clinical trials in IgG-mediated autoimmune diseases, efgartigimod was well tolerated with similar safety profiles regardless of dosing regimen.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"627-638"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143999314","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The therapeutic effect of mRNA vaccines in glioma: a comprehensive review. mRNA疫苗治疗胶质瘤的疗效综述

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-04-22 DOI: 10.1080/1744666X.2025.2494656

Fatemeh Afrashteh, Simin Seyedpour, Nima Rezaei

{"title":"The therapeutic effect of mRNA vaccines in glioma: a comprehensive review.","authors":"Fatemeh Afrashteh, Simin Seyedpour, Nima Rezaei","doi":"10.1080/1744666X.2025.2494656","DOIUrl":"10.1080/1744666X.2025.2494656","url":null,"abstract":"Introduction: Glioma is the most common primary brain tumor, with glioblastoma being the most lethal type due to its heterogeneous and invasive nature of the cancer. Current therapies have low curative success and are limited to surgery, radiotherapy, and chemotherapy. More than 50% of patients become resistant to chemotherapy, and tumor recurrence occurs in most patients following an initial course of therapy. Therefore, developing novel, effective strategies for glioma treatment is essential. Cancer vaccines are novel therapies that demonstrate advantages over conventional methods and, therefore, may be promising options for treating glioma.Areas covered: This article provided a critical review of pre-clinical and clinical studies that explored appropriate tumor antigen candidates for developing mRNA vaccines and discussed their clinical application in glioma patients. Medline database, PubMed, and ClinicalTrials.gov were searched for glioma vaccine studies published before 2025 using related keywords.Expert opinion: mRNA vaccines are promising strategies for treating glioma because they are efficient, cost-beneficial, and have lower side effects than other types such as peptide or DNA-based vaccines.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"603-615"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143974053","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Management of severe asthma in children: current insights and future directions. 儿童严重哮喘的管理：当前的见解和未来的方向。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-04-15 DOI: 10.1080/1744666X.2025.2493698

Maria De Filippo, Riccardo Castagnoli, Ilaria Brambilla, Maddalena Leone, Gian Luigi Marseglia, Amelia Licari

{"title":"Management of severe asthma in children: current insights and future directions.","authors":"Maria De Filippo, Riccardo Castagnoli, Ilaria Brambilla, Maddalena Leone, Gian Luigi Marseglia, Amelia Licari","doi":"10.1080/1744666X.2025.2493698","DOIUrl":"10.1080/1744666X.2025.2493698","url":null,"abstract":"Introduction: Severe pediatric asthma represents a critical challenge in respiratory medicine, affecting a small but significant subset of children with disproportionate morbidity and healthcare burden. Its complexity arises from diverse phenotypes, endotypes, and inflammatory pathways that complicate diagnosis and management. Recent advances in precision medicine, particularly biologic therapies targeting Type 2 inflammation, offer new opportunities for improved outcomes.Areas covered: This review synthesizes current knowledge on severe pediatric asthma, emphasizing the pathophysiology, clinical phenotypes, and therapeutic advancements. It explores the role of biomarkers and endotypes in guiding personalized therapy and critically examines challenges such as non-Type 2 inflammation, barriers to biologic access, and variability in treatment response. A comprehensive literature search was conducted, focusing on biologic therapies, diagnostic innovations, and emerging care models.Expert opinion: Despite progress, challenges remain in achieving standardized treatment response definitions, validating biomarkers, and addressing cost barriers. Early biologic initiation in high-risk children and multidisciplinary care are critical to advancing outcomes. Future efforts should prioritize predictive algorithms, innovative therapies, and equitable access to shift from symptom control to disease prevention, potentially redefining the pediatric asthma care paradigm.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"587-601"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143993727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Innovative surgical approaches for chronic rhinitis: nasal neurectomy mechanisms, techniques, and clinical outcomes. 慢性鼻炎的创新手术方法：鼻神经切除术机制、技术和临床结果。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-05-04 DOI: 10.1080/1744666X.2025.2500610

Zengxiao Zhang, Hongzheng Wei, Chengshuo Wang, Luo Zhang, Yuan Zhang

{"title":"Innovative surgical approaches for chronic rhinitis: nasal neurectomy mechanisms, techniques, and clinical outcomes.","authors":"Zengxiao Zhang, Hongzheng Wei, Chengshuo Wang, Luo Zhang, Yuan Zhang","doi":"10.1080/1744666X.2025.2500610","DOIUrl":"10.1080/1744666X.2025.2500610","url":null,"abstract":"Introduction: Chronic rhinitis (CR) represents a prevalent, persistent inflammatory condition of the nasal mucosa, substantially impacting patients' quality of life. Despite standard pharmacotherapy, many patients with refractory symptoms do not achieve adequate relief, highlighting the need for alternative interventions such as nasal neurectomy.Areas covered: Literature was reviewed on the PubMed, EMBASE, and Web of Science databases published from March 1961 to April 2025. Our review discusses the underlying mechanisms, surgical techniques, and clinical outcomes of different nasal neurectomy approaches, including vidian neurectomy, vidian-branch neurectomy, and anterior ethmoidal neurectomy, as well as recent advancements in endoscopic and minimally invasive methods.Expert opinion: Nasal neurectomy presents a promising alternative for managing refractory CR by directly targeting the neuro-immune pathways that drive symptoms. Understanding the mechanisms, techniques, and clinical outcomes in nasal neurectomy will not only advance our insight into CR pathophysiology but also guide the development of refined, patient-specific surgical strategies for optimal outcomes.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"567-576"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143984756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Vascular endothelial growth factors in airway allergic diseases: pathophysiological functions and therapeutic prospects. 血管内皮生长因子在气道变应性疾病中的病理生理功能及治疗前景。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-04-28 DOI: 10.1080/1744666X.2025.2499597

Yu Zhang, Jingyun Li, Luo Zhang, Yuan Zhang

{"title":"Vascular endothelial growth factors in airway allergic diseases: pathophysiological functions and therapeutic prospects.","authors":"Yu Zhang, Jingyun Li, Luo Zhang, Yuan Zhang","doi":"10.1080/1744666X.2025.2499597","DOIUrl":"10.1080/1744666X.2025.2499597","url":null,"abstract":"Introduction: Vascular endothelial growth factors (VEGFs) play a crucial role in regulating physiological angiogenesis and homeostasis during growth and development. Recent advancements in our knowledge of VEGFs have revealed their complex role in coordinating vascular homeostasis and pathological role in various airway allergic reactions and structural remodeling, especially in allergic asthma and allergic rhinitis (AR), which has become more apparent.Areas covered: After an extensive search of PubMed and Web of Science databases, our review covered articles published from 1989 to 2024. The purpose of this review was to review previous studies on VEGFs involved in inflammatory progression and tissue remodeling in airway allergic diseases, to summarize the relevant pathways. This article further reviews that VEGFs and their receptors can also be potential targets for treating airway allergic diseases.Expert opinion: The prevalence of airway allergic diseases is increasing, which has caused a serious economic burden. VEGFs and their receptors have been recognized as potential targets for therapeutic interventions, which have been effectively applied in the treatment of tumors and other diseases. Fully elucidating the involvement of VEGFs in the disease process will help us understand their mechanisms of action and develop targeted therapies for allergic diseases.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"577-586"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957529","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Predicting worsening risk in MGFA class I, II and III myasthenia gravis patients: development and validation of a predictive nomogram. 预测MGFA I、II和III级重症肌无力患者恶化的风险：一种预测图的开发和验证

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-05-01 Epub Date: 2025-04-30 DOI: 10.1080/1744666X.2025.2494653

Seoyeong Pang, Yanyuan Du, Siyang Peng, Linghao Meng, Anni Xiong, Wenzeng Zhu

{"title":"Predicting worsening risk in MGFA class I, II and III myasthenia gravis patients: development and validation of a predictive nomogram.","authors":"Seoyeong Pang, Yanyuan Du, Siyang Peng, Linghao Meng, Anni Xiong, Wenzeng Zhu","doi":"10.1080/1744666X.2025.2494653","DOIUrl":"10.1080/1744666X.2025.2494653","url":null,"abstract":"Background: Myasthenia gravis (MG), a neuromuscular junction autoimmune disorder, causes skeletal muscle weakness. MG worsening frequently occurs during the disease course, severely impairing quality of life and elevating myasthenic crisis risk. Existing predictive models remain scarce. This study developed a predictive model for MG worsening to facilitate early risk stratification and personalized care.Research design & methods: Retrospective analysis included 437 the Myasthenia Gravis Foundation of America (MGFA) class I - III myasthenia gravis patients from December 2019 to September 2024. Sociodemographic, clinical variables and worsening status were analyzed. Predictors were identified via univariate analysis, the Least Absolute Shrinkage and Selection Operator (LASSO) regression, and multivariate logistic regression. Model performance was assessed using receiver operating characteristic (ROC) analysis, calibration curves, and decision curve analysis.Results: Patients were randomized into training (n = 305) and validation (n = 132) cohorts. Worsening rates were comparable (26.52% vs. 31.15%, p = 0.331). Six predictors emerged: age, MGFA classification, thymectomy history, chills, fatigue, and emotional disturbances (ED). The nomogram demonstrated strong discrimination (AUC: 0.82 training, 0.83 validation) and calibration (Hosmer-Lemeshow p > 0.05). Decision curve analysis confirmed clinical utility at 10-70% probability thresholds.Conclusion: This nomogram integrates accessible clinical variables to stratify MG worsening risk, enabling early intervention. Validation through multicenter prospective studies is warranted to optimize generalizability.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"639-649"},"PeriodicalIF":3.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143960550","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Differential risk of autoimmune disorders in non-traumatic osteonecrosis: clue to pathogenesis. 非外伤性骨坏死中自身免疫性疾病的不同风险：发病机制的线索。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-04-01 Epub Date: 2025-03-05 DOI: 10.1080/1744666X.2025.2475982

Edward Y Cheng, Alireza Mirzaei

{"title":"Differential risk of autoimmune disorders in non-traumatic osteonecrosis: clue to pathogenesis.","authors":"Edward Y Cheng, Alireza Mirzaei","doi":"10.1080/1744666X.2025.2475982","DOIUrl":"10.1080/1744666X.2025.2475982","url":null,"abstract":"Introduction: Non-traumatic osteonecrosis is a frequent complication in patients with autoimmune disorders, though its prevalence varies markedly depending upon the type of disorder. Understanding the causes of this difference can help uncover the underlying pathophysiology of osteonecrosis and guide the development of effective preventive and therapeutic strategies.Areas covered: In this perspective study, we reviewed available databases, including PubMed, Cochrane Library, Scopus, and Web of Science, to explore why the risk of osteonecrosis varies among different autoimmune disorders. Is this variation primarily due to the disease's pathophysiology, the use of medications such as corticosteroids, or a combination of both? If both factors are involved, what is the extent of each contribution in this context?Expert opinion: Non-traumatic osteonecrosis is often induced by an interaction between disease pathophysiology and corticosteroid use. In patients with different autoimmune disorders but an identical history of corticosteroid use, the risk of osteonecrosis is influenced by how the underlying pathophysiology compromises bone health. In autoimmune disorders with multiple adverse effects on bone, such as SLE (systemic lupus erythematosus), there is a much higher risk of osteonecrosis compared to disorders with minimal impact on bone health, such as celiac disease and MS (multiple sclerosis).","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"413-424"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143540798","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Diagnostic methods and strategies for autoimmune bullous diseases. 自身免疫性大疱性疾病的诊断方法和策略。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-04-01 Epub Date: 2025-02-17 DOI: 10.1080/1744666X.2025.2465405

Hua Qian, Norito Ishii, Hiroshi Koga, Kwesi Teye, Atsunari Tsuchisaka, Takekuni Nakama, Chiharu Tateishi, Mako Mine, Daisuke Tsuruta, Yoshiaki Hirako, Xiaoguang Li, Takashi Hashimoto

{"title":"Diagnostic methods and strategies for autoimmune bullous diseases.","authors":"Hua Qian, Norito Ishii, Hiroshi Koga, Kwesi Teye, Atsunari Tsuchisaka, Takekuni Nakama, Chiharu Tateishi, Mako Mine, Daisuke Tsuruta, Yoshiaki Hirako, Xiaoguang Li, Takashi Hashimoto","doi":"10.1080/1744666X.2025.2465405","DOIUrl":"10.1080/1744666X.2025.2465405","url":null,"abstract":"Introduction: Autoimmune bullous disease (AIBD), showing blistering lesions on the skin and/or mucous membranes, is characterized by autoantibodies against various structural molecules present in keratinocyte cell surfaces and epidermal basement membrane zone. In addition to the clinical and pathological features, identification of specific autoantibodies is essential for AIBD diagnosis, and therefore corresponding methods should be well summarized and popularized.Area covered: Currently, direct immunofluorescence using biopsy tissue specimen and indirect immunofluorescence using normal human skin, 1 M NaCl-split skin and rat bladder are primarily used to identify tissue-bound and circulating autoantibodies, respectively. Immunoblotting and enzyme-linked immunosorbent assay (ELISA) methods have been developed for detection of autoantibodies against AIBD autoantigens, including desmoglein 1, desmoglein 3, BP180, BP230, type VII collagen, laminin (LM) 332, integrin α6β4, p200 (LMγ1/LMβ4) and human serum albumin. In this review, we summarized the detailed laboratory protocols for AIBD diagnosis methods used in our three institutes (Kurume University and Osaka Metropolitan University in Japan, and Daqing Oilfield General Hospital in China) before 9 October 2024.Expert opinion: This review will benefit both clinical practitioners and basic researchers on AIBD. In the future, simpler and easier AIBD diagnostic algorithms using a smaller number of tests should be established.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"451-460"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143390496","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Guidance for stem cell therapy for juvenile systemic sclerosis patients. 青少年系统性硬化症患者干细胞治疗指南。

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-04-01 Epub Date: 2025-03-24 DOI: 10.1080/1744666X.2025.2474216

Ivan Foeldvari, Kathryn S Torok, Juliana Silva, Christopher P Denton, Jörg Henes, Paulina Horvei, Franziska Rosser, Catherine H Orteu, Suzanne C Li, Clare E Pain, Tamás Constantin, Patricia Costa-Reis, Megan L Curran, Maurizio Cutolo, Bernd Hinrichs, Kim Fligelstone, Susan Maillard, Pia Moinzadeh, Clarissa Pilkington, Linda Schraven, Vanessa Smith

{"title":"Guidance for stem cell therapy for juvenile systemic sclerosis patients.","authors":"Ivan Foeldvari, Kathryn S Torok, Juliana Silva, Christopher P Denton, Jörg Henes, Paulina Horvei, Franziska Rosser, Catherine H Orteu, Suzanne C Li, Clare E Pain, Tamás Constantin, Patricia Costa-Reis, Megan L Curran, Maurizio Cutolo, Bernd Hinrichs, Kim Fligelstone, Susan Maillard, Pia Moinzadeh, Clarissa Pilkington, Linda Schraven, Vanessa Smith","doi":"10.1080/1744666X.2025.2474216","DOIUrl":"10.1080/1744666X.2025.2474216","url":null,"abstract":"Introduction: Autologous stem cell transplantation (ASCT) and cellular therapies (CTs) are emerging therapeutic options for both adult and juvenile-onset systemic sclerosis (jSSc) patients. However, most efficacy data are derived from adult studies, and it remains unclear whether adult stem cell transplant criteria are fully applicable to pediatric patients with jSSc. Given pediatric patients' unique potential for recovery and tissue remodeling, the stringent criteria used in adults need adaptation for children.Areas covered: We reviewed the current data on indications, patient selection, and outcomes of ASCT and CTs in both adult and pediatric patients with systemic sclerosis. At a multidisciplinary expert workshop held in Hamburg, Germany, in December 2023, we developed consensus guidance on when to consider ASCT and cellular therapies in jSSc.Expert opinion: HSCT and CT hold promise as treatment options for jSSc. Our proposed guidance aims to standardize inclusion criteria globally, enhancing comparability of outcomes across future procedures. Establishing consistent inclusion/exclusion criteria and transplant protocols will enable better data collection, interpretation, and ultimately improve outcomes and care for jSSc patients.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"435-450"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143582263","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Usability, efficacy, and safety of candidate tocilizumab biosimilar CT-P47 self-administration via auto-injector and pre-filled syringe in patients with rheumatoid arthritis: a single-arm, open-label, phase 3 study. 候选tocilizumab生物类似药CT-P47在类风湿性关节炎患者中通过自动注射器和预填充注射器自我给药的可用性、有效性和安全性：一项单臂、开放标签、3期研究

IF 3.9 3区医学

Expert Review of Clinical Immunology Pub Date : 2025-04-01 Epub Date: 2025-01-15 DOI: 10.1080/1744666X.2025.2451215

Gerd Burmester, Piotr Adrian Klimiuk, Jakub Trefler, Janusz Jaworski, SungHyun Kim, YunJu Bae, DaBee Jeon, HyunSeung Lee, JiYoung Jang, ChanKyoung Hwang, HoJae Lee, Josef S Smolen

{"title":"Usability, efficacy, and safety of candidate tocilizumab biosimilar CT-P47 self-administration via auto-injector and pre-filled syringe in patients with rheumatoid arthritis: a single-arm, open-label, phase 3 study.","authors":"Gerd Burmester, Piotr Adrian Klimiuk, Jakub Trefler, Janusz Jaworski, SungHyun Kim, YunJu Bae, DaBee Jeon, HyunSeung Lee, JiYoung Jang, ChanKyoung Hwang, HoJae Lee, Josef S Smolen","doi":"10.1080/1744666X.2025.2451215","DOIUrl":"10.1080/1744666X.2025.2451215","url":null,"abstract":"Background: CT-P47 is a candidate tocilizumab biosimilar that is currently in clinical development. We assessed the usability of CT-P47 self-administration via auto-injector (AI) in patients with rheumatoid arthritis (RA).Research design and methods: This was a 12-week, single-arm, open-label, multiple-dose, Phase 3 study. Patients self-injected CT-P47 (162 mg/0.9 mL) via AI at Weeks 0 and 2, and then every other week via pre-filled syringe (PFS) from Week 4 through Week 10. The primary endpoint was POST-Self-Injection Assessment Questionnaire (SIAQ) at Week 2. Efficacy, safety, and immunogenicity were also assessed.Results: Thirty-three patients were enrolled. Mean scores for all POST-SIAQ domains at Week 2 exceeded 8, except for 'self-confidence' (7.11) and 'satisfaction with self-injection' (7.98), indicating positive patient experiences with CT-P47 AI. Furthermore, an observer-completed checklist found that all patients successfully followed the required steps for self-injection. Efficacy, assessed by Disease Activity Score in 28 joints and its components, showed improvements from baseline to Week 12. No new safety signals were observed; the most common adverse events were leukopenia, neutropenia, and injection-site reaction, each occurring in 3 (9.1%) patients.Conclusions: CT-P47 self-administered using an AI showed successful usability in patients with moderate-to-severe RA.Trial registration: ClinicalTrials.gov identifier: NCT05725434.","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":"521-529"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970300","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0