Expert Opinion on Biological Therapy最新文献_第3页

Current outlook on the use of monoclonal antibody therapies for osteoporosis. 单克隆抗体治疗骨质疏松症的现状展望。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-08 DOI: 10.1080/14712598.2026.2641051

Mohammed Almohaya, Mohammed Almehthel, David Kendler

{"title":"Current outlook on the use of monoclonal antibody therapies for osteoporosis.","authors":"Mohammed Almohaya, Mohammed Almehthel, David Kendler","doi":"10.1080/14712598.2026.2641051","DOIUrl":"10.1080/14712598.2026.2641051","url":null,"abstract":"Introduction: The availability of monoclonal antibody therapies impacting on the basic pathways of bone formation; (romosozumab) and bone resorption (denosumab) has contributed greatly to our ability to manage patients with osteoporosis and fracture risk. Monoclonal antibodies offer the potential to optimize therapeutic efficacy with minimal adverse effects.Areas covered: This review discusses aspects of registration clinical trials as well as experience gained from the clinical use of monoclonal antibodies for osteoporosis. The registration clinical trials prove anti-fracture efficacy with subsequent trials investigating bone mineral density, bone turnover markers, and high-resolution bone imaging. Many of these trials indicate superiority of monoclonal antibody therapy for osteoporosis compared with traditional antiresorbers such as bisphosphonates.Expert opinion: There remain significant care gaps in the management of patients at high risk of fragility fracture. Many patients at very high risk remain undiagnosed and therefore untreated. Monoclonal antibody antiresorptive therapy such as denosumab with high specificity and romosozumab with the ability to rapidly stimulate new bone formation are significant advances. Future therapies may direct 'maintenance' therapies to avoid reversal of beneficial effects on monoclonal antibody treatment discontinuation. Other novel therapies being investigated may provide benefits not only to osteoporosis but other diseases associated with aging.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"243-256"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147347883","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

A review of the clinical efficacy of monoclonal antibody (mAb)-based therapies for relapsed/refractory multiple myeloma (RRMM). 基于单克隆抗体（mAb）治疗复发/难治性多发性骨髓瘤（RRMM）的临床疗效综述

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-04-05 DOI: 10.1080/14712598.2026.2646947

Yuxin Liu, Ji Hyun Lee, Clifton C Mo, Taya Jamal Salman, Shahrier Hossain, Shonali Midha, Omar Nadeem, Taylor Nicholson, Jessica Croteau, Natalie Kazierad, Tarek H Mouhieddine, Poy Theprungsirikul, Jacob P Laubach, Paul G Richardson

{"title":"A review of the clinical efficacy of monoclonal antibody (mAb)-based therapies for relapsed/refractory multiple myeloma (RRMM).","authors":"Yuxin Liu, Ji Hyun Lee, Clifton C Mo, Taya Jamal Salman, Shahrier Hossain, Shonali Midha, Omar Nadeem, Taylor Nicholson, Jessica Croteau, Natalie Kazierad, Tarek H Mouhieddine, Poy Theprungsirikul, Jacob P Laubach, Paul G Richardson","doi":"10.1080/14712598.2026.2646947","DOIUrl":"10.1080/14712598.2026.2646947","url":null,"abstract":"Introduction: Monoclonal antibodies (mAbs) and antibody-drug conjugates (ADCs) are among the backbones of treatment for relapsed/refractory multiple myeloma (RRMM). Beyond current standard-of-care regimens, the roles of mAbs/ADCs are evolving associated with advances in first-line therapy, emerging data on additional regimens, and developments with immunotherapies and other novel agents.Areas covered: We review mechanisms of action, efficacy, real-world effectiveness, and key aspects of the safety profiles of daratumumab, isatuximab, elotuzumab, and belantamab mafodotin in RRMM. We consider efficacy in patient subgroups and the challenges of treatment sequencing and highlight new antigen targets and mAb/ADC therapies under investigation. We searched the published literature with PubMed and congress abstracts using drug names or classes and 'myeloma.'Expert opinion: The widespread use of daratumumab and isatuximab in first-line therapy and evolving roles of CAR T-cell therapies and bispecific antibodies are reshaping RRMM treatment. mAb/ADC-based regimens remain key options in this setting, offering practical, effective, and tolerable approaches for real-world practice. Ongoing research will inform individualized treatment choices and rational sequencing of therapies, with a need for immune-based biomarkers and biologic profiling to enable optimal, integrated use of mAbs, ADCs, CAR T-cell therapies, and bispecific antibodies to further improve outcomes for patients with RRMM.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"285-319"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473171","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

An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa. 赞米角素治疗隐性营养不良大疱性表皮松解症的疗效评价。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-18 DOI: 10.1080/14712598.2026.2639393

Aleksandra Lesiak, M Peter Marinkovich

{"title":"An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa.","authors":"Aleksandra Lesiak, M Peter Marinkovich","doi":"10.1080/14712598.2026.2639393","DOIUrl":"10.1080/14712598.2026.2639393","url":null,"abstract":"Introduction: Prademagene zamikeracel is an autologous, genetically corrected epidermal graft therapy designed to address the underlying molecular defect in recessive dystrophic epidermolysis bullosa (RDEB). The product is manufactured from a patient's own keratinocytes, which are modified ex vivo to overexpress a normal copy of COL7A1, the gene coding for type VII collagen (C7), which is essential for anchoring fibril formation and stable dermal-epidermal cohesion. In individuals with biallelic COL7A1 variants, loss of C7 results in severe skin fragility, painful and widespread wounds, debilitating scarring, and a high risk of aggressive cutaneous squamous cell carcinoma.Areas covered: This review outlines the key scientific foundations, translational advances, and clinical trial outcomes that supported regulatory approval of prademagene zamikeracel for use in adult and pediatric patients with RDEB.Expert opinion: The development of prademagene zamikeracel represents a significant advance in regenerative therapy for RDEB, demonstrating that durable restoration of C7 expression and long-term wound improvement can be achieved through skin grafting.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"225-230"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147325165","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

Comparative efficacy and safety of the trastuzumab biosimilar HLX02 versus originator trastuzumab in the neoadjuvant THP-EC regimen for early-stage HER2-positive breast cancer: a real-world study. 曲妥珠单抗生物仿制药HLX02与原曲妥珠单抗在新辅助THP-EC方案中治疗早期her2阳性乳腺癌的疗效和安全性比较：一项现实世界研究

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-26 DOI: 10.1080/14712598.2026.2649512

Mingyu Wang, Xingye Sheng, Qiannan Zhu, Danni Shen, Junhan Li, Xincheng Tao, Jue Wang, Xiaoming Zha

{"title":"Comparative efficacy and safety of the trastuzumab biosimilar HLX02 versus originator trastuzumab in the neoadjuvant THP-EC regimen for early-stage HER2-positive breast cancer: a real-world study.","authors":"Mingyu Wang, Xingye Sheng, Qiannan Zhu, Danni Shen, Junhan Li, Xincheng Tao, Jue Wang, Xiaoming Zha","doi":"10.1080/14712598.2026.2649512","DOIUrl":"10.1080/14712598.2026.2649512","url":null,"abstract":"Objectives: HER2-positive breast cancer is an aggressive subtype. High-cost originator trastuzumab limits treatment access, making biosimilars like HLX02 a valuable alternative.Methods: This retrospective real-world study compared HLX02 (n = 42) with originator trastuzumab (n = 237) within the neoadjuvant THP-EC regimen for early HER2-positive breast cancer. The primary endpoint was total pathological complete response (tpCR).Results: Before propensity score matching (PSM), the tpCR rate was higher with HLX02 (73.81% vs. 43.04%, p < 0.001). After PSM, tpCR rates were comparable (HLX02: 73.81% vs. originator: 72.27%; p = 0.448), with no significant subgroup differences. Safety profiles, including cardiac toxicity, were similar between groups.Conclusion: HLX02 demonstrated equivalent efficacy and safety to the originator trastuzumab in this setting, representing a cost-effective treatment alternative.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"355-362"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485136","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

Nivolumab plus ipilimumab for hepatocellular carcinoma: a game-changer? Nivolumab联合ipilimumab治疗肝细胞癌：改变游戏规则？

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-11 DOI: 10.1080/14712598.2026.2642273

Lorenza Di Marco, Federica Valerio, Yuri Maculan, Bianca Medici, Andrea Spallanzani, Massimo Dominici, Angela Dalia Ricci, Alessandro Rizzo, Massimiliano Salati

{"title":"Nivolumab plus ipilimumab for hepatocellular carcinoma: a game-changer?","authors":"Lorenza Di Marco, Federica Valerio, Yuri Maculan, Bianca Medici, Andrea Spallanzani, Massimo Dominici, Angela Dalia Ricci, Alessandro Rizzo, Massimiliano Salati","doi":"10.1080/14712598.2026.2642273","DOIUrl":"10.1080/14712598.2026.2642273","url":null,"abstract":"Introduction: Hepatocellular carcinoma (HCC) remains a leading cause of cancer-related mortality worldwide, largely arising in the setting of chronic liver disease and cirrhosis. Given the immunosuppressive hepatic tumor microenvironment, optimizing immunotherapeutic strategies is critical to improve patient outcomes.Areas covered: This review examines the biological rationale and clinical evidence supporting dual immune checkpoint inhibition with nivolumab plus ipilimumab in advanced HCC. We discuss mechanisms of immune tolerance in cirrhosis and tumor progression, including regulatory T cells, myeloid-derived suppressor cells, hypoxia, metabolic reprogramming, and T-cell exhaustion. Key clinical data from early-phase studies, such as CheckMate 040 and the phase III CheckMate 9DW, are analyzed, focusing on overall survival, response rates, and durability of response compared with tyrosine kinase inhibitors. A structured literature search of PubMed, Embase, and major oncology congress proceedings was conducted to identify relevant preclinical and clinical studies.Expert opinion: Dual checkpoint blockade represents an effective first-line option for selected patients with advanced HCC, offering meaningful survival benefit at the cost of increased immune-related toxicity. Future progress depends on improved patient selection, biomarker development, and rational combination strategies.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"257-267"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147364524","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

An evaluation of axatilimab for the treatment of chronic graft-versus-host disease. 阿替利单抗治疗慢性移植物抗宿主病的评价。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-26 DOI: 10.1080/14712598.2026.2649515

Saurabh Chhabra, Sriram Banigallapati

{"title":"An evaluation of axatilimab for the treatment of chronic graft-versus-host disease.","authors":"Saurabh Chhabra, Sriram Banigallapati","doi":"10.1080/14712598.2026.2649515","DOIUrl":"10.1080/14712598.2026.2649515","url":null,"abstract":"Introduction: Chronic graft-versus-host disease (cGVHD), a frequent, debilitating autoimmune-like syndrome affecting allogeneic transplant recipients begins with inflammatory response to per-transplant tissue injury which evolves into chronic inflammation, T- and B-cell dysregulation, and aberrant tissue repair and fibrotic reaction. Monocytes and macrophages contribute to multiorgan inflammation and fibrosis that are hallmarks of cGVHD.Areas covered: Axatilimab is a high-affinity anti-CSF-1 R humanized immunoglobulin G4 monoclonal antibody that blocks ligand binding to CSF-1 R and downregulates development and differentiation of pathogenic monocyte-derived macrophages. A phase 1/2 study, and subsequently a phase 2 randomized trial that evaluated axatilimab in patient with refractory cGVHD, reported an ORR of 50-74%. Toxicity was dose-dependent. Based on the Phase 2 results, the lowest dosage, 0.3 mg/kg every 2 weeks, was identified as appropriate for the indication. Here, we examine clinical development of axatilimab leading up to its approval by the Food and Drug Administration for treatment of cGVHD after failure of at least two prior therapies.Expert opinion: Axatilimab has demonstrated safety, tolerability, and efficacy in clinical trials; however, many questions remain unanswered including long-term safety data, efficacy when compared to other cGVHD therapies, risks and benefits of axatilimab in combination, and role in earlier lines of cGVHD treatment.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"231-241"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485018","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

Extracellular RNAs in pancreatic cancer: from pathogenesis to precision medicine. 胰腺癌的细胞外rna：从发病机制到精准医学。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-03-20 DOI: 10.1080/14712598.2026.2648644

Mohammad Rezazadeh, Ava Aghakhani, Farbod Bahreini, Nima Rezaei

{"title":"Extracellular RNAs in pancreatic cancer: from pathogenesis to precision medicine.","authors":"Mohammad Rezazadeh, Ava Aghakhani, Farbod Bahreini, Nima Rezaei","doi":"10.1080/14712598.2026.2648644","DOIUrl":"10.1080/14712598.2026.2648644","url":null,"abstract":"Introduction: Pancreatic cancer is highly aggressive with poor prognosis and limited therapies. Extracellular RNAs (exRNAs), including microRNAs (miRNAs), long noncoding RNAs (lncRNAs), and circular RNAs (circRNAs), play key roles in its progression. Transported through vesicles or freely circulating, they regulate proliferation, epithelial-mesenchymal transition, angiogenesis, immune evasion, and metastasis. exRNAs contribute to chemoresistance, immune suppression, tumor growth, and intercellular communication, shaping pancreatic cancer's aggressive phenotype and overall tumor microenvironment.Areas covered: Recent research highlights exRNAs as prognostic biomarkers and therapeutic targets. Modulating exRNA expression or inhibiting their function may offer new strategies to overcome resistance to existing therapies. This review summarizes current knowledge on the relevance of exRNAs and their potential applications in understanding pathogenesis and improving treatment. A deeper insight into exRNA-mediated signaling networks may help develop innovative therapeutic approaches for more effective pancreatic cancer management in the future.Expert opinion: Pancreatic cancer, especially pancreatic ductal adenocarcinoma, has poor prognosis due to late diagnosis, aggressiveness, and treatment resistance. exRNAs, including circRNAs, miRNAs, and lncRNAs, regulate tumor progression and are promising biomarkers and therapeutic targets. Despite challenges with stability, delivery, and patient variability, exRNAs show considerable potential for early detection, personalized therapy, genome-editing strategies, and combination treatments with chemotherapy or immunotherapy.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"339-353"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485158","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 role of monoclonal antibodies in the treatment of lupus nephritis. 单克隆抗体在狼疮性肾炎治疗中的作用。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-03-01 Epub Date: 2026-04-10 DOI: 10.1080/14712598.2026.2649524

Uxía Couto-Lareo, Blanca Viejo-Sosa, Mònica Angerri-Nadal, Roni Meidan, David Isenberg

{"title":"The role of monoclonal antibodies in the treatment of lupus nephritis.","authors":"Uxía Couto-Lareo, Blanca Viejo-Sosa, Mònica Angerri-Nadal, Roni Meidan, David Isenberg","doi":"10.1080/14712598.2026.2649524","DOIUrl":"10.1080/14712598.2026.2649524","url":null,"abstract":"Introduction: Systemic lupus erythematosus is a heterogeneous autoimmune disease in which lupus nephritis represents one of the most severe and prognostically relevant manifestations, accounting for substantial morbidity and mortality. Despite therapeutic advances, long-term renal outcomes remain suboptimal, prompting the need to reassess current management strategies.Areas covered: This review discusses recent advances in the classification and treatment of lupus nephritis, with particular emphasis on evolving conceptual frameworks and emerging therapies. We summarize updates in international guidelines, including the 2025 EULAR recommendations, which incorporate biologic and targeted agents such as obinutuzumab and voclosporin into standard treatment regimens and promote combination strategies to improve efficacy while limiting cumulative toxicity. In addition, we review data from recent clinical trials evaluating novel agents, including telitacicept, baricitinib, and dapirulizumab, as well as early-stage and experimental approaches such as CAR T-cell therapies. The review is based on a narrative analysis of recent clinical trials and guideline updates.Expert opinion: The therapeutic landscape of lupus nephritis is rapidly evolving, with promising new agents expanding available options; however, high costs, limited access, and insufficient long-term data currently restrict their widespread first-line use. Future head-to-head trials and real-world studies are essential to refine treatment algorithms and improve durable renal outcomes.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"321-337"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147627355","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 role of targeted therapies in blastic plasmacytoid dendritic cell neoplasm. 靶向治疗在母细胞浆细胞样树突状细胞肿瘤中的作用。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-01-01 Epub Date: 2025-12-31 DOI: 10.1080/14712598.2025.2610291

Roberta S Azevedo, Zohra Nooruddin, Sumeet Bhatia, Laura Finn, Carter Davis, Leonard C Alsfeld, Suki Subbiah, Maxim Norkin, Matthew Ulrickson, Adolfo De La Fuente, Melissa L Larson, Tulin Budak-Alpdogan, Naveen Pemmaraju

{"title":"The role of targeted therapies in blastic plasmacytoid dendritic cell neoplasm.","authors":"Roberta S Azevedo, Zohra Nooruddin, Sumeet Bhatia, Laura Finn, Carter Davis, Leonard C Alsfeld, Suki Subbiah, Maxim Norkin, Matthew Ulrickson, Adolfo De La Fuente, Melissa L Larson, Tulin Budak-Alpdogan, Naveen Pemmaraju","doi":"10.1080/14712598.2025.2610291","DOIUrl":"10.1080/14712598.2025.2610291","url":null,"abstract":"Introduction: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an ultra-rare disease that arises from malignant precursor dendritic cells. Historically, there was no uniform standard-of-care chemotherapy. In this setting, outcomes were substandard, especially for patients not eligible for intensive chemotherapy, clinical trials, and/or hematopoietic stem cell transplantation, which constituted the majority of patients. The development of targeted therapy, namely the anti-CD123 agent tagraxofusp, marked the beginning of a new era in the treatment of BPDCN.Areas covered: This review summarizes current targeted therapies in BPDCN, highlights recent therapeutic strategies and emerging approaches with the potential to improve patient outcomes. We also discuss key barriers to clinical implementation, including adverse events associated with anti-CD123-directed therapies and limitations related to treatment cost and accessibility.Expert opinion: The approval of tagraxofusp established a foundation for targeted therapy in BPDCN and demonstrated high response rates, leading to the development of additional antibody - drug conjugates and cellular therapies targeting CD123. Other promising investigational targeted approaches may include BCL2 inhibitors, proteasome inhibitors, and therapies directed against surface antigens such as CD38 and CD303, as well as bromodomain and extraterminal (BET) inhibitors. Collectively, these strategies represent important advances that may significantly improve the historically poor outcomes associated with BPDCN.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"15-24"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145827269","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

Biologic treatment patterns and challenges in defining difficult-to-treat disease in children with polyarticular juvenile idiopathic arthritis: a real-world study. 多关节幼年特发性关节炎儿童的生物治疗模式和定义难治性疾病的挑战：一项真实世界的研究。

IF 4 3区医学

Expert Opinion on Biological Therapy Pub Date : 2026-01-01 Epub Date: 2026-02-22 DOI: 10.1080/14712598.2026.2635527

Sıla Atamyıldız Uçar, Eray Tunce, Neslihan Kara Çanlıoğlu, Betül Sözeri

{"title":"Biologic treatment patterns and challenges in defining difficult-to-treat disease in children with polyarticular juvenile idiopathic arthritis: a real-world study.","authors":"Sıla Atamyıldız Uçar, Eray Tunce, Neslihan Kara Çanlıoğlu, Betül Sözeri","doi":"10.1080/14712598.2026.2635527","DOIUrl":"10.1080/14712598.2026.2635527","url":null,"abstract":"Background: To evaluate the clinical characteristics, and treatment patterns of difficult-to-treat (D2T) disease in children with polyarticular juvenile idiopathic arthritis (pJIA) treated with biologic disease-modifying antirheumatic drugs (bDMARDs).Research design and methods: This single-center, cross-sectional study included children with RF-positive or RF-negative pJIA who received bDMARD. D2T defined according to EULAR criteria, as inadequate response to at least two bDMARDs with different mechanisms of action. Disease activity was assessed using the JADAS-27. Treatment patterns, switches, and factors associated with D2T were analyzed.Results: A total of 59 pJIA patients (79.9% female) were included, with a median follow-up of 60 months. Ten patients (16.9%) fulfilled the criteria for D2T disease after a follow-up duration of 36 months. At diagnosis, D2T patients had significantly higher inflammatory markers than non-D2T patients, with higher median CRP (45 vs. 12 mg/L, p = 0.002) and ESR (77 vs. 38 mm/h, p = 0.003) values. In univariate analyses, higher CRP, ESR, temporomandibular joint involvement, and higher JADAS-27 at bDMARD initiation were associated with D2T status (p < 0.05).Conclusions: D2T course was associated with temporomandibular joint involvement, elevated inflammatory markers, and higher disease activity at the initiation of the first biologic therapy.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"189-198"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146776173","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