Expert Opinion on Biological Therapy最新文献_第7页

Gene therapy for Leber hereditary optic neuropathy. 治疗 Leber 遗传性视神经病变的基因疗法。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-28 DOI: 10.1080/14712598.2024.2359015

Marco Battista, Valerio Carelli, Leonardo Bottazzi, Francesco Bandello, Maria Lucia Cascavilla, Piero Barboni

{"title":"Gene therapy for Leber hereditary optic neuropathy.","authors":"Marco Battista, Valerio Carelli, Leonardo Bottazzi, Francesco Bandello, Maria Lucia Cascavilla, Piero Barboni","doi":"10.1080/14712598.2024.2359015","DOIUrl":"https://doi.org/10.1080/14712598.2024.2359015","url":null,"abstract":"Introduction: Leber hereditary optic neuropathy (LHON) is among the most frequent inherited mitochondrial disease, causing a severe visual impairment, mostly in young-adult males. The causative mtDNA variants (the three common are m.11778 G>A/MT-ND4, m.3460 G>A/MT-ND1, and m.14484T>C/MT-ND6) by affecting complex I impair oxidative phosphorylation in retinal ganglion cells, ultimately leading to irreversible cell death and consequent functional loss. The gene therapy based on allotopic expression of a wild-type transgene carried by adeno-associated viral vectors (AVV-based) appears a promising approach in mitochondrial disease and its efficacy has been explored in several large clinical trials.Areas covered: The review work employed basic concepts in mitochondrial diseases, LHON, and gene therapy procedures. Reports from completed trials in LHON (i.e. RESCUE) were reviewed and critically compared.Expert opinion: New challenges, as the improvement of the contralateral untreated eye or the apparently better outcome in patients treated in later stages (6-12 months), were highlighted by the latest gene therapy trials. A better understanding of the pathogenetic mechanisms of the disease together with combined therapy (medical and gene therapy) and optimization in genetic correction approaches could improve the visual outcome of treated eyes.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467181","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 proctitis in ulcerative colitis and the place of biological therapies. 溃疡性结肠炎直肠炎的治疗与生物疗法的地位。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-17 DOI: 10.1080/14712598.2024.2369189

Diletta De Deo, Arianna Dal Buono, Roberto Gabbiadini, Paola Spaggiari, Anita Busacca, Benedetta Masoni, Silvia Ferretti, Cristina Bezzio, Alessandro Armuzzi

{"title":"Management of proctitis in ulcerative colitis and the place of biological therapies.","authors":"Diletta De Deo, Arianna Dal Buono, Roberto Gabbiadini, Paola Spaggiari, Anita Busacca, Benedetta Masoni, Silvia Ferretti, Cristina Bezzio, Alessandro Armuzzi","doi":"10.1080/14712598.2024.2369189","DOIUrl":"10.1080/14712598.2024.2369189","url":null,"abstract":"Introduction: Approximately 20-30% of the patients with ulcerative colitis (UC) may present with isolated proctitis. Ulcerative proctitis (UP) is a challenging condition to manage due to its significant burden in terms of disabling symptoms.Areas covered: PubMed was searched up to March 2024 to identify relevant studies on UP. A comprehensive summary and critical appraisal of the available data on UP are provided, highlighting emerging treatments and areas for future research.Expert opinion: Patients with UP are often undertreated, and the disease burden is often underestimated in clinical practice. Treat-to-target management algorithms can be applied to UP, aiming for clinical remission in the short term, and endoscopic remission and maintenance of remission in the long term. During their disease, approximately one-third of UP patients require advanced therapies. Escalation to biologic therapy is required for refractory or steroid dependent UP. For optimal patient care and management of UP, it is necessary to include these patients in future randomized clinical trials.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141317207","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

Checkpoint inhibition for early-stage hormone receptor-positive breast cancer. 检查点抑制治疗早期激素受体阳性乳腺癌。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-24 DOI: 10.1080/14712598.2024.2370395

Ilana Schlam, Chiara Corti, Sarah Sammons, Elizabeth A Mittendorf, Sara M Tolaney

{"title":"Checkpoint inhibition for early-stage hormone receptor-positive breast cancer.","authors":"Ilana Schlam, Chiara Corti, Sarah Sammons, Elizabeth A Mittendorf, Sara M Tolaney","doi":"10.1080/14712598.2024.2370395","DOIUrl":"10.1080/14712598.2024.2370395","url":null,"abstract":"Introduction: Most patients with breast cancer have early-stage hormone receptor (HR)-positive, human epidermal growth factor receptor-2 (HER2)-negative disease. Even though the prognosis for most of these patients is good, there is a need to identify patients at risk for poor outcomes and to develop strategies to mitigate this risk.Areas covered: The addition of immunotherapy to standard neoadjuvant chemotherapy represents a promising option for select patients with HR-positive early breast cancer. Three randomized clinical trials have shown favorable results to date. In this review, we discuss the findings of I-SPY2, CheckMate 7FL (NCT04109066), and KEYNOTE-756 (NCT03725059).Expert opinion: Despite the promising results of these trials, there are unanswered questions that need to be considered before incorporating neo/adjuvant immunotherapy in the treatment paradigm of early-stage HR-positive breast cancer. One example of an unanswered question is patient selection. Because the regimens used in these protocols are associated with long-term toxicities, identifying the patients who are more likely to derive a benefit from these agents, such as through the use of biomarkers, is critical. A second example is the optimal integration of adjuvant therapies that improve invasive disease-free survival, such as abemaciclib and ribociclib, which are not safely administered concurrently with immunotherapy.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445942","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

Progress in second-line antibody therapies for advanced esophageal squamous cell carcinoma. 晚期食管鳞状细胞癌二线抗体疗法的进展。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-11 DOI: 10.1080/14712598.2024.2366493

Nozomu Ogura, Shun Yamamoto, Ken Kato

{"title":"Progress in second-line antibody therapies for advanced esophageal squamous cell carcinoma.","authors":"Nozomu Ogura, Shun Yamamoto, Ken Kato","doi":"10.1080/14712598.2024.2366493","DOIUrl":"10.1080/14712598.2024.2366493","url":null,"abstract":"Introduction: The prognosis of advanced esophageal squamous cell carcinoma (ESCC) is poor. Although cytotoxic drugs have been widely used in advanced ESCC, several antibody agents have recently been reported to be effective.Areas covered: Nivolumab and pembrolizumab are anti-PD-1 antibodies that improve immunosuppression by binding to programmed death-1 (PD-1), leading to an antitumor effect. Randomized phase III trials have found these immune checkpoint inhibitors (ICIs) to be effective as second-line treatment. ATTRACTION-3, which compared nivolumab monotherapy with taxane monotherapy in patients with previously treated advanced ESCC, reported prolonged overall survival in the nivolumab group. KEYNOTE-181 found that overall survival was longer in patients with PD-L1-positive ESCC who received second-line treatment with pembrolizumab than in those who received chemotherapy. Sym004 and amivantamab are antibodies that target the epidermal growth factor receptor and have demonstrated efficacy in the treatment of other tumors in recent phase I studies. Furthermore, clinical trials on antibody-drug conjugates such as enfortumab vedotin and DS-7300 for solid tumors are currently ongoing.Expert opinion: The standard first-line treatments for patients with advanced ESCC contain ICIs. Therefore, drugs with different mechanisms of action that can overcome resistance to ICIs are needed as second-line or later-line treatments to improve clinical outcomes in these patients.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300393","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

How close are we to a success stratification tool for improving biological therapy in ulcerative colitis? 我们离改进溃疡性结肠炎生物疗法的成功分层工具还有多远？

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-26 DOI: 10.1080/14712598.2024.2371049

Panu Wetwittayakhlang, Gynter Kotrri, Talat Bessissow, Peter L Lakatos

{"title":"How close are we to a success stratification tool for improving biological therapy in ulcerative colitis?","authors":"Panu Wetwittayakhlang, Gynter Kotrri, Talat Bessissow, Peter L Lakatos","doi":"10.1080/14712598.2024.2371049","DOIUrl":"10.1080/14712598.2024.2371049","url":null,"abstract":"Introduction: Biological therapies have become the standard treatment for ulcerative colitis (UC). However, clinical remission rates post-induction therapy remain modest at 40-50%, with many initial responders losing response over time. Current treatment strategies frequently rely on a 'trial and error' approach, leading to prolonged periods of ineffective and costly therapies for patients, accompanied by associated treatment complications.Area covered: This review discusses current evidence on risk stratification tools for predicting therapeutic efficacy and minimizing adverse events in UC management. Recent studies have identified predictive factors for biologic therapy response. In the context of personalized medicine, the goal is to identify patients at high risk of progression and complications, as well as those likely to respond to specific therapies. Essential risk stratification tools include clinical decision-making aids, biomarkers, genomics, multi-omics factors, endoscopic, imaging, and histological assessments.Expert opinion: Employing risk stratification tools to predict therapeutic response and prevent treatment-related complications is essential for precision medicine in the biological management of UC. These tools are necessary to select the most suitable treatment for each individual patient, thereby enhancing efficacy and safety.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141431719","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

Treatment options for advanced hepatocellular carcinoma: the potential of biologics. 晚期肝细胞癌的治疗方案：生物制剂的潜力。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-24 DOI: 10.1080/14712598.2024.2363234

Federico Rossari, Silvia Foti, Silvia Camera, Mara Persano, Andrea Casadei-Gardini, Margherita Rimini

{"title":"Treatment options for advanced hepatocellular carcinoma: the potential of biologics.","authors":"Federico Rossari, Silvia Foti, Silvia Camera, Mara Persano, Andrea Casadei-Gardini, Margherita Rimini","doi":"10.1080/14712598.2024.2363234","DOIUrl":"10.1080/14712598.2024.2363234","url":null,"abstract":"Introduction: Advanced hepatocellular carcinoma (HCC) represents a significant global health burden, whose treatment has been recently revolutionized by the advent of biologic treatments. Despite that, innovative therapeutic regimens and approaches, especially immune-based, remain to be explored aiming at extending the therapeutic benefits to a wider population of patients.Areas covered: This review comprehensively discusses the evolving landscape of biological treatment modalities for advanced HCC, including immune checkpoint inhibitors, antiangiogenic monoclonal antibodies, tumor-targeting monoclonal antibodies either naked or drug-conjugated, therapeutic vaccines, oncolytic viruses, adoptive cell therapies, and cytokine-based therapies. Key clinical trials and preclinical studies are examined, highlighting the actual or potential impact of these interventions in reshaping treatment paradigms for HCC.Expert opinion: Tailored and rational combination strategies, leveraging the synergistic effects of different modalities, represent a promising approach to maximize treatment efficacy in advanced HCC, which should aim at conversion endpoints to increase the fraction of patients eligible for curative approaches. The identification of predictive biomarkers holds the key to optimizing patient selection and improving therapeutic outcomes.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442391","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 nadofaragene firadenovec-vncg for the treatment of high-risk BCG-unresponsive non-muscle-invasive bladder cancer. 评估纳多法拉基因 Firadenovec-vncg 用于治疗对卡介苗无反应的高风险非肌肉浸润性膀胱癌。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-13 DOI: 10.1080/14712598.2024.2365802

Zine-Eddine Khene, Yair Lotan

{"title":"An evaluation of nadofaragene firadenovec-vncg for the treatment of high-risk BCG-unresponsive non-muscle-invasive bladder cancer.","authors":"Zine-Eddine Khene, Yair Lotan","doi":"10.1080/14712598.2024.2365802","DOIUrl":"10.1080/14712598.2024.2365802","url":null,"abstract":"Introduction: BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) represent a significant therapeutic challenge in the treatment of bladder cancer. Nadofaragene firadenovec, represents a breakthrough in this area, offering a novel approach for the treatment of BCG-unresponsive NMIBC.Areas covered: This overview explores the historical development of nadofaragene firadenovec, assessing its efficacy and safety, and discusses future NMIBC therapy directions.Expert opinion: Patients with high grade NMIBC who are BCG unresponsive will have a growing number of treatment alternatives to bladder removal. Nadofaragene firadenovec offers good short-term efficacy but lacks significant durability for most patients. Its strengths include ease of administration and low risk of adverse events. This will need to balance with risk of progression and cost. Furthermore, the likely approval of other agents will require consideration of which therapy to use and for which patient. The need for biomarkers to tailor treatment choices to individual patient needs is becoming more critical. The treatment field is rapidly advancing, with several Phase 3 single-arm trials underway, indicating a potential broader range of treatment options for NMIBC. Further research will be necessary to determine the optimal choice for patients.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300392","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

Do we need alternative PD-1 inhibitors for the treatment of renal cell carcinoma? 治疗肾细胞癌是否需要其他 PD-1 抑制剂？

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-19 DOI: 10.1080/14712598.2024.2369190

Matteo Rosellini, Andrea Marchetti, Elisa Tassinari, Veronica Mollica, Francesco Massari, Matteo Santoni

引用次数: 0

Comparison of the efficacy and safety of rapid-acting insulin analogs, lispro versus aspart, in the treatment of diabetes: a systematic review of randomized controlled trials. 比较速效胰岛素类似物力司宝和阿斯巴特治疗糖尿病的疗效和安全性：随机对照试验的系统回顾。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-06-01 Epub Date: 2024-06-28 DOI: 10.1080/14712598.2024.2371046

Rahul Kapur, Shivani Mittra, Geetanjali Tonpe, Adithi P, Praveen Raj, Uwe Gudat, Sandeep N Athalye

{"title":"Comparison of the efficacy and safety of rapid-acting insulin analogs, lispro versus aspart, in the treatment of diabetes: a systematic review of randomized controlled trials.","authors":"Rahul Kapur, Shivani Mittra, Geetanjali Tonpe, Adithi P, Praveen Raj, Uwe Gudat, Sandeep N Athalye","doi":"10.1080/14712598.2024.2371046","DOIUrl":"10.1080/14712598.2024.2371046","url":null,"abstract":"Introduction: We evaluated a potential move from one rapid-acting insulin analog to another, or their biosimilars, to aid better and faster decisions for diabetes management.Methods: A systematic literature review was performed according to PRISMA reporting guidelines. The MEDLINE/EMBASE/COCHRANE databases were searched for randomized control trials (RCTs) comparing aspart/lispro in type-1 (T1D) and type-2 (T2D) diabetes. The methodological quality of the included studies was assessed using the Cochrane Collaboration's risk of bias assessment criteria.Results: Of the 753 records retrieved, the six selected efficacy/safety RCTs and the additional three hand-searched pharmacokinetics/pharmacodynamics RCTs showed some heterogeneity in the presentation of the continuous variables; however, collectively, the outcomes demonstrated that lispro and aspart had comparable efficacy and safety in adult patients with T1D and T2D. Both treatments yielded a similar decrease in glycated hemoglobin (HbA1c) and had similar dosing and weight changes, with similar treatment-emergent adverse events (TEAE) and serious adverse event (SAE) reporting, similar hypoglycemic episodes in both T1D and T2D populations, and no clinically significant differences for hyperglycemia, occlusions or other infusion site/set complications.Conclusions: Aspart and lispro demonstrate comparative safety and efficacy in patients with T1D/T2D. Since both are deemed equally suitable for controlling prandial glycemic excursions and both have similar safety attributes, they may be used interchangeably in clinical practice.Prospero registration number: CRD42023376793.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141456111","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

Bispecific antibodies targeting CD20xCD3 in immunotherapy for adult B-cell lymphoma: insights from the 65th American Society of Hematology 2023 annual meeting. 针对成人 B 细胞淋巴瘤免疫疗法中 CD20xCD3 的双特异性抗体：第 65 届美国血液学会 2023 年年会的启示。

IF 4.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-05-01 Epub Date: 2024-05-08 DOI: 10.1080/14712598.2024.2351995

Hao Sun, Haizhou Xing, Lijie Han, Yongping Song, Zhongxing Jiang, Yanyan Liu, Jifeng Yu

引用次数: 0