Expert Opinion on Biological Therapy最新文献_第8页

An evaluation of exagamglogene autotemcel for the treatment of sickle cell disease and transfusion-dependent beta-thalassaemia. 对 exagamglogene autotemcel 治疗镰状细胞病和输血依赖型β-地中海贫血症的评估。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-09-01 Epub Date: 2024-09-02 DOI: 10.1080/14712598.2024.2399134

Rupert Handgretinger, Markus Mezger

{"title":"An evaluation of exagamglogene autotemcel for the treatment of sickle cell disease and transfusion-dependent beta-thalassaemia.","authors":"Rupert Handgretinger, Markus Mezger","doi":"10.1080/14712598.2024.2399134","DOIUrl":"10.1080/14712598.2024.2399134","url":null,"abstract":"Introduction: Sickle cell disease is the most common hereditary hemoglobinopathy followed by beta-thalassemia. Until recently, allogeneic stem cell transplantation was the only curative approach. Based on the Crispr-Cas9-technology enabling targeting specific genes of interest, fetal hemoglobin which is normally shut-off after birth can be switched on and sufficient levels can alleviate symptoms in sickle cell disease and avoid transfusions in beta-thalassemia. Two first-in-human clinical studies in sickle cell disease and beta-thalassemia aiming to increase the level of fetal hemoglobin by using Crispr-Cas9 to modify autologous hematopoietic stem cells in patients aged 12-35 years have proved safety and efficacy and have shown promising clinical outcomes.Areas covered: The paper summarizes the outcome of the results of the two recently published clinical studies and compares them with the other available curative approaches.Expert opinion: Based on the currently available safety and efficacy data of the two published clinical results on gene therapy with Crispr-Cas9 modified autologous stem cells (exagamglogene autotemcel), it can be anticipated that this approach will add significantly to the therapeutic options for patients with sickle cell disease and beta-thalassemia and can be considered for all patients above 12 years of age independent of a suitable allogeneic stem cell donor.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"883-888"},"PeriodicalIF":3.6,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105777","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

Correction. 更正。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-08-02 DOI: 10.1080/14712598.2024.2388405

引用次数: 0

Review of emerging biological therapies for recurrent and advanced metastatic cervical cancer. 复发性和晚期转移性宫颈癌的新兴生物疗法综述。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-06-29 DOI: 10.1080/14712598.2024.2373320

Virginia E Fontenot, Alex Francoeur, Krishnansu S Tewari

引用次数: 0

Long-acting delivery and therapies for neovascular age-related macular degeneration. 新生血管性老年黄斑变性的长效给药和疗法。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-22 DOI: 10.1080/14712598.2024.2374869

Lucas W Rowe, Thomas A Ciulla

{"title":"Long-acting delivery and therapies for neovascular age-related macular degeneration.","authors":"Lucas W Rowe, Thomas A Ciulla","doi":"10.1080/14712598.2024.2374869","DOIUrl":"10.1080/14712598.2024.2374869","url":null,"abstract":"Introduction: Neovascular age-related macular degeneration (nAMD) represents a leading cause of severe visual impairment in individuals over 50 years of age in developed nations. Intravitreal anti-vascular endothelial growth factor (VEGF) injections have become the standard of care for treating nAMD; however, monthly or bimonthly dosing represents significant time and cost burden due to the disease's chronic nature and limited medication half-life.Areas covered: This review summarizes innovative therapeutics and delivery methods for nAMD. Emerging methods for extended drug delivery include high molar concentration anti-VEGF drugs, intravitreal sustained-release polymers and devices, reservoirs for intravitreal delivery, suprachoroidal delivery of small molecular suspensions and gene therapy biofactories. In addition to VEGF-A, therapies targeting inhibition of VEGF-C and D, the angiopoetin-2 (Ang-2)/Tie-2 pathway, tyrosine kinases, and integrins are reviewed.Expert opinion: The evolving therapeutic landscape of nAMD is rapidly expanding our toolkit for effective and durable treatment. Recent FDA approvals of faricimab (Vabysmo) and high-dose aflibercept (Eylea HD) for nAMD with potential extension of injection intervals up to four months have been promising developments for patients and providers alike. Further research and innovation, including novel delivery techniques and pharmacologic targets, is necessary to validate the efficacy of developing therapeutics and characterize real-world outcomes, demonstrating promise in expanding treatment durability.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"799-814"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141491494","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 nail in psoriatic arthritis: new insights into prognosis and treatment. 银屑病关节炎的指甲：预后和治疗的新见解。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-25 DOI: 10.1080/14712598.2024.2385020

Rubén Queiro, Sara Alonso, Jose A Pinto-Tasende

引用次数: 0

The chimera of reaching a universal consensus on platelet-rich plasma treatment for knee osteoarthritis: a review of recent consensus statements and expert opinion. 就富血小板血浆治疗膝骨关节炎达成普遍共识的 "锲机"：近期共识声明和专家意见综述。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-08-02 DOI: 10.1080/14712598.2024.2383865

Elizaveta Kon, Giuseppe Anzillotti, Pietro Conte, Luca Ruosi, Brian Cole, Jason Dragoo, Ken Zaslav, Rachel Frank, Laura De Girolamo, Bert Mandelbaum, Scott Rodeo, Maurilio Marcacci, Giuseppe Filardo, Berardo Di Matteo

{"title":"The chimera of reaching a universal consensus on platelet-rich plasma treatment for knee osteoarthritis: a review of recent consensus statements and expert opinion.","authors":"Elizaveta Kon, Giuseppe Anzillotti, Pietro Conte, Luca Ruosi, Brian Cole, Jason Dragoo, Ken Zaslav, Rachel Frank, Laura De Girolamo, Bert Mandelbaum, Scott Rodeo, Maurilio Marcacci, Giuseppe Filardo, Berardo Di Matteo","doi":"10.1080/14712598.2024.2383865","DOIUrl":"10.1080/14712598.2024.2383865","url":null,"abstract":"Introduction: Osteoarthritis (OA) is a prevalent cause of disability worldwide, affecting millions and posing significant socioeconomic burdens. Various conservative measures like hyaluronic acid (HA) and platelet-rich plasma (PRP) injections aim to manage OA symptoms and delay surgical interventions. Despite the increasing utilization of PRP, consensus on its efficacy remains elusive, reflecting the evolving landscape of OA management.Areas covered: This study reviews guidelines and recommendations on intra-articular PRP injections for OA globally, highlighting divergent perspectives among different medical societies. A comprehensive literature search identified 19 relevant guidelines, indicating a temporal and geographic evolution in attitudes toward PRP use. While some guidelines endorse PRP for mild-to-moderate OA, others express caution due to concerns about product standardization and clinical evidence heterogeneity.Expert opinion: The lack of universal consensus on PRP for OA underscores the complex interplay between clinical evidence, practice patterns, and evolving perspectives. Recent shifts toward endorsing PRP may reflect advancements in preparation techniques and personalized medicine approaches. However, challenges persist, including patient selection and product standardization. Efforts to develop consensus and refine PRP classification systems are essential for guiding clinical practice and advancing OA management.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"827-833"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787661","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

Targeting gut and intratumoral microbiota: a novel strategy to improve therapy resistance in cancer with a focus on urologic tumors. 针对肠道和肿瘤内微生物群：改善癌症耐药性的新策略，重点关注泌尿系统肿瘤。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-06-24 DOI: 10.1080/14712598.2024.2371543

Bingquan Wu, Chao Quan, Yunbo He, Juliet Matsika, Jinliang Huang, Bolong Liu, Jinbo Chen

{"title":"Targeting gut and intratumoral microbiota: a novel strategy to improve therapy resistance in cancer with a focus on urologic tumors.","authors":"Bingquan Wu, Chao Quan, Yunbo He, Juliet Matsika, Jinliang Huang, Bolong Liu, Jinbo Chen","doi":"10.1080/14712598.2024.2371543","DOIUrl":"10.1080/14712598.2024.2371543","url":null,"abstract":"Introduction: Growing attention has been drawn to urologic tumors due to their rising incidence and suboptimal clinical treatment outcomes. Cancer therapy resistance poses a significant challenge in clinical oncology, limiting the efficacy of conventional treatments and contributing to disease progression. Recent research has unveiled a complex interplay between the host microbiota and cancer cells, highlighting the role of the microbiota in modulating therapeutic responses.Areas covered: We used the PubMed and Web of Science search engines to identify key publications in the fields of tumor progression and urologic tumor treatment, specifically focusing on the role of the microbiota. In this review, we summarize the current literature on how microbiota influence the tumor microenvironment and anti-tumor immunity, as well as their impact on treatments for urinary system malignancies, highlighting promising future applications.Expert opinion: We explore how the composition and function of the gut microbiota influence the tumor microenvironment and immune response, ultimately impacting treatment outcomes. Additionally, we discuss emerging strategies targeting the microbiota to enhance therapeutic efficacy and overcome resistance. The application of antibiotics, fecal microbiota transplantation, and oncolytic bacteria has improved tumor treatment outcomes, which provides a novel insight into developing therapeutic strategies for urologic cancer.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"747-759"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442390","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 potential for medical therapies to address fistulizing Crohn's disease: a state-of-the-art review. 治疗瘘管化克罗恩病的药物疗法潜力：最新综述。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-24 DOI: 10.1080/14712598.2024.2383882

Mohammad Shehab, Davide De Marco, Peter L Lakatos, Talat Bessissow

{"title":"The potential for medical therapies to address fistulizing Crohn's disease: a state-of-the-art review.","authors":"Mohammad Shehab, Davide De Marco, Peter L Lakatos, Talat Bessissow","doi":"10.1080/14712598.2024.2383882","DOIUrl":"10.1080/14712598.2024.2383882","url":null,"abstract":"Introduction: Crohn's disease (CD) is a chronic, relapsing immune mediated disease, which is one of the two major types of inflammatory bowel disease (IBD). Fistulizing CD poses a significant clinical challenge for physicians. Effective management of CD requires a multidisciplinary approach, involving a gastroenterologist and a GI surgeon while tailoring treatment to each patient's unique risk factors, clinical representations, and preferences.Areas covered: This comprehensive review explores the intricacies of fistulizing CD including its manifestations, types, impact on quality of life, management strategies, and novel therapies under investigation.Expert opinion: Antibiotics are often used as first-line therapy to treat symptoms. Biologics that selectively target TNF-α, such infliximab (IFX), have shown high efficacy in randomized controlled trials. However, more than 50% of patients lose response to IFX, prompting them to explore alternative strategies. Current options include adalimumab and certolizumab pegol combination therapies, as well as small-molecule drugs targeting Janus kinases such as Upadacitinib. Furthermore, a promising treatment for complex fistulas is mesenchymal stem cells such as Darvadstrocel (Alofisel), an allogeneic stem cell-based therapy. However, surgical interventions are necessary for complex cases or intra-abdominal complications. Setons and LIFT procedures are the most common surgical options.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"733-746"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141751466","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

Optimizing withdrawal strategies for anti-TNF-α therapies in rheumatoid arthritis. 优化类风湿性关节炎抗肿瘤坏死因子-α疗法的停药策略。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-25 DOI: 10.1080/14712598.2024.2384000

Aliki I Venetsanopoulou, Paraskevi V Voulgari, Alexandros A Drosos

{"title":"Optimizing withdrawal strategies for anti-TNF-α therapies in rheumatoid arthritis.","authors":"Aliki I Venetsanopoulou, Paraskevi V Voulgari, Alexandros A Drosos","doi":"10.1080/14712598.2024.2384000","DOIUrl":"10.1080/14712598.2024.2384000","url":null,"abstract":"Introduction: Rheumatoid arthritis (RA) is a chronic autoimmune disease that significantly impacts patients' quality of life. While treatment options have expanded over the years, including the introduction of tumor necrosis factor-alpha (TNFα) inhibitors (TNFi), optimizing withdrawal strategies for these agents remains a challenge.Areas covered: This review examines the current evidence on TNFi withdrawal strategies in RA, focusing on factors influencing withdrawal decisions such as disease activity monitoring, treatment response, patient characteristics, and biomarkers. A comprehensive literature search was conducted, including randomized controlled trials, observational studies, and expert guidelines. The pathophysiology of RA, current pharmacological agents, and the treat-to-target strategy are discussed to provide a holistic understanding of RA management.Expert opinion: Withdrawal strategies could be suitable for certain patients, keeping in mind that several factors influence withdrawal decisions, including treatment response, disease activity and monitoring, and patient characteristics. The decision to withdraw TNFi must balance the benefits against the potential risks of disease flare and long-term treatment-related adverse effects. Combining DMARDs and TNFi early improves outcomes, supporting tapering strategies for cost-effectiveness and flare prevention. Future directions, including precision medicine approaches, patient-centered care models, and health economics analyses, are proposed to further optimize RA management and improve patient outcomes.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"815-825"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141758042","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

Intraparenchymal delivery of adeno-associated virus vectors for the gene therapy of neurological diseases. 将腺相关病毒载体用于神经系统疾病的基因治疗。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-31 DOI: 10.1080/14712598.2024.2386339

Shinichi Kumagai, Takeshi Nakajima, Shin-Ichi Muramatsu

{"title":"Intraparenchymal delivery of adeno-associated virus vectors for the gene therapy of neurological diseases.","authors":"Shinichi Kumagai, Takeshi Nakajima, Shin-Ichi Muramatsu","doi":"10.1080/14712598.2024.2386339","DOIUrl":"10.1080/14712598.2024.2386339","url":null,"abstract":"Introduction: In gene therapy with adeno-associated virus (AAV) vectors for diseases of the central nervous system, the vectors can be administered into blood vessels, cerebrospinal fluid space, or the brain parenchyma. When gene transfer to a large area of the brain is required, the first two methods are used, but for diseases in which local gene transfer is expected to be effective, vectors are administered directly into the brain parenchyma.Areas covered: Strategies for intraparenchymal vector delivery in gene therapy for Parkinson's disease, aromatic l-amino acid decarboxylase (AADC) deficiency, and epilepsy are reviewed.Expert opinion: Stereotactic intraparenchymal injection of AAV vectors allows precise gene delivery to the target site. Although more surgically invasive than intravascular or intrathecal administration, intraparenchymal vector delivery has the advantage of a lower vector dose, and preexisting neutralizing antibodies have little effect on the transduction efficacy. This approach improves motor function in AADC deficiency and led to regulatory approval of an AAV vector for the disease in the EU. Although further validation through clinical studies is needed, direct infusion of viral vectors into the brain parenchyma is expected to be a novel treatment for Parkinson's disease and drug-resistant epilepsy.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"773-785"},"PeriodicalIF":3.6,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787659","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