Oncology and Therapy最新文献

{"title":"Optimizing Real-World Outcomes in High-Risk Relapsed/Refractory (r/r) FL with CAR-T Cell Therapy: A Vodcast and Case Example.","authors":"Kai Hübel","doi":"10.1007/s40487-024-00269-4","DOIUrl":"10.1007/s40487-024-00269-4","url":null,"abstract":"<p><p>Follicular lymphoma (FL) is often considered a chronic disease with frequent relapses, shortening both response duration and survival after every relapse. Selecting the most appropriate therapy at the right time within the treatment timeline is key to optimize outcomes. The aim of this vodcast, featuring Dr. Kai Hübel, is to outline the severity of FL by referring to a patient case as well as highlight chimeric antigen receptor (CAR)-T cells as an effective therapy in relapsed/refractory (r/r) FL. The patient was in their early 50s, diagnosed with FL in the early 2010s and presented with a third relapse. The patient complained of night sweats and fatigue but was still capable of self-care (Eastern Cooperative Oncology Group Performance Status Scale 2). The patient received eight cycles of rituximab-cyclophosphamide-doxorubicin-vincristine-prednisolone (R-CHOP), followed by irradiation and rituximab maintenance (first-line) and then received rituximab 4 × weekly, followed by rituximab maintenance (second-line). The patient relapsed during rituximab maintenance; the patient was rituximab refractory. The patient received six cycles of bendamustine/obinutuzumab followed by obinutuzumab maintenance. The patient relapsed during obinutuzumab maintenance, achieved a partial remission after irradiation and was switched to R/lenalidomide. Due to several high-risk features, CAR-T cell therapy was initiated. Dr. Hubel underlines how earlier treatment with CAR-T cell therapy would have been beneficial for this patient. Results of the ELARA trial as well as comparative studies have shown tisagenlecleucel to be more effective than standard of care in extensively pretreated r/r FL, including high-risk patients. In conclusion, CAR-T cell therapy is a promising therapy option for patients with multiply r/r FL. A vodcast feature is available for this article.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"217-221"},"PeriodicalIF":3.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11187031/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140185872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating Public Economic Gains from Early Breast Cancer and Curative Treatment: A Case Study in Human Epidermal Growth Factor Receptor (HER-2) Positive Targeted Therapies.","authors":"Svenn Alexander Kommandantvold, Nikos Kotsopoulos, Isabel Monteiro, Ana Ladeiras, Andrew Hogan, Felipe Barboza Magalhães de Araujo, Mark P Connolly","doi":"10.1007/s40487-024-00264-9","DOIUrl":"10.1007/s40487-024-00264-9","url":null,"abstract":"<p><strong>Introduction: </strong>Cancer diagnosis influences the choices that patients make regarding current and future labor market activity. These choices have implications for governments based on resulting changes in taxes paid and benefits received. In this analysis we explore how human growth receptor 2 (HER2)-positive residual invasive breast cancer and different treatments influence government accounts excluding health costs.</p><p><strong>Methods: </strong>HER2-positive early breast cancer (eBC) health states from a published disease model were used to establish likelihood of working and wage impact at different stages of disease. The indirect productivity losses for an average woman aged 49 years were translated into fiscal consequences to government by applying an established government perspective-modeling framework. The fiscal projections (discounted) include gross tax revenue by disease stage, government transfer costs related to time off work and early retirement ,and net fiscal balance (e.g., gross taxes-transfers) in three countries Canada, Portugal, and Brazil.</p><p><strong>Results: </strong>The net fiscal balance in Canada for a healthy woman was C$109,551 compared with a HER2-positive eBC woman treated with trastuzumab emtansine (C$69,767) or trastuzumab (C$62,971). A similar pattern was observed in the three countries but reflecting the overall tax burden in each country, labor force activity, and available public benefits. Age at diagnosis was an important determinant of the likely net fiscal balance, as this influences the remaining working years.</p><p><strong>Discussion: </strong>Women diagnosed with HER2-positive eBC were estimated to pay less lifetime gross taxes and receive more in sickness benefits compared with healthy women. Treatments that improve outcomes are likely to offer fiscal gains for government from improved work force participation.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"277-292"},"PeriodicalIF":3.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11187022/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139742216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Podcast on Emerging Treatment Options for Pediatric Patients with ALK-Positive Anaplastic Large Cell Lymphoma and Inflammatory Myofibroblastic Tumors.","authors":"Eric Lowe, Yael P Mossé","doi":"10.1007/s40487-024-00275-6","DOIUrl":"10.1007/s40487-024-00275-6","url":null,"abstract":"<p><p>Anaplastic large cell lymphoma (ALCL) and inflammatory myofibroblastic tumor (IMT) are rare cancers observed predominantly in children and young adults. ALCL accounts for 10-15% of all pediatric non-Hodgkin lymphomas and is commonly diagnosed at an advanced stage of disease. In children, 84-91% of cases of ALCL harbor an anaplastic lymphoma kinase (ALK) gene translocation. IMT is a rare mesenchymal neoplasm that also tends to occur in children and adolescents. Approximately 50-70% of IMT cases involve rearrangements in the ALK gene. A combination of chemotherapeutic drugs is typically used for children with ALK-positive ALCL, and the only known curative therapy for ALK-positive IMT is complete surgical resection. Crizotinib, a first-generation ALK inhibitor, was approved in the USA in 2021 for pediatric patients and young adults with relapsed or refractory ALK-positive ALCL; however, its safety and efficacy have not been established in older adults. In 2022, crizotinib was approved for adult and pediatric patients with unresectable, recurrent, or refractory ALK-positive IMT. This podcast provides an overview of ALK-positive ALCL and IMT. We discuss the current treatment landscape, the role of ALK tyrosine kinase inhibitors, and areas of future research.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"247-255"},"PeriodicalIF":3.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11187053/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140872712","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions.","authors":"Ruofei Yuan, Bo Wang, Ying Wang, Pinan Liu","doi":"10.1007/s40487-024-00279-2","DOIUrl":"10.1007/s40487-024-00279-2","url":null,"abstract":"<p><p>Neurofibromatosis type 2 (NF2)-related schwannomatosis is a rare autosomal dominant monogenic disorder caused by mutations in the NF2 gene. The hallmarks of NF2-related schwannomatosis are bilateral vestibular schwannomas (VS). The current treatment options for NF2-related schwannomatosis, such as observation with serial imaging, surgery, radiotherapy, and pharmacotherapies, have shown limited effectiveness and serious complications. Therefore, there is a critical demand for novel effective treatments. Gene therapy, which has made significant advancements in treating genetic diseases, holds promise for the treatment of this disease. This review covers the genetic pathogenesis of NF2-related schwannomatosis, the latest progress in gene therapy strategies, current challenges, and future directions of gene therapy for NF2-related schwannomatosis.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"257-276"},"PeriodicalIF":3.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11187037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Podcast on Pre- or Perioperative Chemoimmunotherapy for Stage III Non-Small Cell Lung Cancer: Shared Agreement from the Thoracic Surgeon and Oncologist Perspectives.","authors":"Luca Bertolaccini, Andrea De Giglio, Eleonora Gariazzo, Giulio Metro","doi":"10.1007/s40487-024-00267-6","DOIUrl":"10.1007/s40487-024-00267-6","url":null,"abstract":"<p><p>Management of stage II-III non-small cell lung cancer (NSCLC) has been dramatically revolutionized by studies testing the addition of immunotherapy (IO) to chemotherapy in the pre- or perioperative setting. That is because the integration of chemoimmunotherapy (chemo-IO) with surgery has consistently shown a significant improvement in pathological complete response (path CR) rate, event-free survival, and, more recently, overall survival, versus preoperative chemotherapy alone. Particularly, resectable stage III NSCLCs represent a disease entity with a high risk of distant recurrence after radical surgery, for whom pre- or perioperative chemo-IO should be considered as the preferential treatment option. However, owing to the heterogeneity of stage III NSCLC, a standard definition of resectability is not established yet, being often subjective according to the expertise and clinical background of the thoracic surgeon. In addition, careful patient selection on the basis of tumor biomarkers, meticulous staging of the disease, and accurate monitoring of treatment-related adverse events are critical factors that could prevent the ineligibility for surgery of patients treated with pre- or perioperative chemo-IO. Finally, the impact of downstaging for initially borderline resectable tumors, as well as the exact number of preoperative chemo-IO cycles needed and the indications for adjuvant IO, still need to be fully elucidated. In this podcast, we will touch upon the above-mentioned topics from the perspectives of the thoracic surgeon and the oncologist, and suggest a shared agreement between two of the main actors involved in the treatment of resectable stage III NSCLCs.Podcast audio available for this publication.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"207-215"},"PeriodicalIF":3.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11187012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140132776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adjuvant CDK4/6 Inhibitors for Early Breast Cancer: How to Choose Wisely?","authors":"Elisa Agostinetto, Luca Arecco, Evandro de Azambuja","doi":"10.1007/s40487-023-00250-7","DOIUrl":"10.1007/s40487-023-00250-7","url":null,"abstract":"","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"19-29"},"PeriodicalIF":2.7,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10881905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138292029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Retrospective Study of Cemiplimab Effectiveness in Elderly Patients with Squamous Cell Carcinoma of the Skin: Insights from a Real-Life Scenario.","authors":"Giuseppe Di Lorenzo, Aieta Michele, Leo Silvana, Domenico Bilancia, Rossella Di Trolio, Michela Rosaria Iuliucci, Concetta Ingenito, Roberta Rubino, Arianna Piscosquito, Michele Caraglia, Marianna Donnarumma, Ferdinando Costabile, Raffaele Conca, Marco Pisino, Angelo Vaia, Luca Scafuri, Antonio Verde, Carlo Buonerba","doi":"10.1007/s40487-023-00256-1","DOIUrl":"10.1007/s40487-023-00256-1","url":null,"abstract":"<p><strong>Introduction: </strong>This retrospective study investigates the efficacy of cemiplimab, a monoclonal antibody targeting the PD-1 receptor, in treating squamous cell carcinoma (SCC) of the skin.</p><p><strong>Methods: </strong>The study analyzes data from 50 patients with SCC, focusing on various clinical parameters, including patient demographics, tumor characteristics, treatment history, disease status at the beginning of therapy, and survival outcomes.</p><p><strong>Results: </strong>Of the patients who received at least one cycle of cemiplimab, 42% showed a clinical response. Adverse reactions were generally low, with the safety profile deemed excellent. During a median follow-up of 9.6 months, 17 patients experienced progression or death. Among these, 15 patients had died at the time of the analysis. The median progression-free survival (PFS) for the entire cohort was approximately 20.8 months, while median overall survival (OS) was not reached. Univariate Cox regression analysis for PFS showed that tumors in the arms and legs were associated with higher progression risk, while age above 65 years was not statistically significant. Distant metastasis exhibited a trend towards improved PFS. In terms of OS, distant metastasis was a significant predictor of reduced survival, while age above 65 years was not statistically significant. In a multivariate model, only the absence of distant metastasis remained significant, with an adjusted odds ratio (OR) of 12.3 (95% confidence interval 1.3-112.1).</p><p><strong>Conclusion: </strong>These findings provide valuable insights into the real-world effectiveness of cemiplimab in SCC management.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"147-155"},"PeriodicalIF":2.7,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10881452/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138811194","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Repotrectinib's Clinical Benefit and Its Brain Penetration in a Patient with Meningeal Carcinomatosis from G2032R-Mutated ROS-1 Positive Non-Small Cell Lung Cancer.","authors":"Giulio Metro, Eleonora Gariazzo, Silvia Costabile, Sara Baglivo, Fausto Roila, Francesca Colamartini, Barbara Palumbo, Pietro Chiarini, Stefania Gori, Antonio Conti, Luca Marcomigni, Guido Bellezza, Gianluigi Lunardi","doi":"10.1007/s40487-023-00251-6","DOIUrl":"10.1007/s40487-023-00251-6","url":null,"abstract":"<p><p>In this work, we report on a clinically significant response of meningeal carcinomatosis to repotrectinib in a woman with a heavily pretreated ROS1-rearranged non-small cell lung cancer (NSCLC) that harbored the concomitant solvent front G2032R mutation. Meningeal carcinomatosis has a higher incidence in oncogene addicted NSCLC due to increased life expectancy, yet no report has ever documented the activity of repotrectinib in this context. In line with its activity, we documented the presence of the drug at potentially active concentrations in the cerebrospinal fluid. Nevertheless, the short-lived response reported by our patient highlights the importance for novel ROS1-tyrosine kinase inhibitors (TKIs) to be specifically developed to be able to penetrate the blood-brain barrier.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"163-171"},"PeriodicalIF":2.7,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10881448/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136399696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Is HER2-Low a New Clinical Entity or Merely a Biomarker for an Antibody Drug Conjugate?","authors":"Heidi Ko, Rebecca A Previs, Kyle C Strickland, Jonathan Klein, Brian Caveney, Chiara Chiruzzi, Marcia Eisenberg, Eric A Severson, Shakti Ramkissoon, Kamal S Saini","doi":"10.1007/s40487-023-00249-0","DOIUrl":"10.1007/s40487-023-00249-0","url":null,"abstract":"","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"13-17"},"PeriodicalIF":2.7,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10881909/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"92156925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Non-Small Cell Lung Cancer Testing on Reference Specimens: An Italian Multicenter Experience.","authors":"Francesco Pepe, Gianluca Russo, Alessandro Venuta, Claudia Scimone, Mariantonia Nacchio, Pasquale Pisapia, Gaia Goteri, Francesca Barbisan, Caterina Chiappetta, Angelina Pernazza, Domenico Campagna, Marco Giordano, Giuseppe Perrone, Giovanna Sabarese, Annalisa Altimari, Dario de Biase, Giovanni Tallini, Daniele Calistri, Elisa Chiadini, Laura Capelli, Alfredo Santinelli, Anna Elisa Gulini, Elisa Pierpaoli, Manuela Badiali, Stefania Murru, Riccardo Murgia, Elena Guerini Rocco, Konstantinos Venetis, Nicola Fusco, Denise Morotti, Andrea Gianatti, Daniela Furlan, Giulio Rossi, Laura Melocchi, Maria Russo, Caterina De Luca, Lucia Palumbo, Saverio Simonelli, Antonella Maffè, Paola Francia di Celle, Tiziana Venesio, Maria Scatolini, Enrico Grosso, Sara Orecchia, Matteo Fassan, Mariangela Balistreri, Elisabetta Zulato, Daniela Reghellin, Elena Lazzari, Maria Santacatterina, Maria Liliana Piredda, Manuela Riccardi, Licia Laurino, Elena Roz, Domenico Longo, Daniela Petronilla Romeo, Carmine Fazzari, Andrea Moreno-Manuel, Giuseppe Diego Puglia, Andrey D Prjibelski, Daria Shafranskaya, Luisella Righi, Angela Listì, Domenico Vitale, Antonino Iaccarino, Umberto Malapelle, Giancarlo Troncone","doi":"10.1007/s40487-023-00252-5","DOIUrl":"10.1007/s40487-023-00252-5","url":null,"abstract":"<p><strong>Introduction: </strong>Biomarker testing is mandatory for the clinical management of patients with advanced non-small cell lung cancer (NSCLC). Myriads of technical platforms are now available for biomarker analysis with differences in terms of multiplexing capability, analytical sensitivity, and turnaround time (TAT). We evaluated the technical performance of the diagnostic workflows of 24 representative Italian institutions performing molecular tests on a series of artificial reference specimens built to mimic routine diagnostic samples.</p><p><strong>Methods: </strong>Sample sets of eight slides from cell blocks of artificial reference specimens harboring exon 19 EGFR (epidermal growth factor receptor) p.E746_AT50del, exon 2 KRAS (Kirsten rat sarcoma viral oncogene homologue) p.G12C, ROS1 (c-ros oncogene 1)-unknown gene fusion, and MET (MET proto-oncogene, receptor tyrosine kinase) Δ exon 14 skipping were distributed to each participating institution. Two independent cell block specimens were validated by the University of Naples Federico II before shipment. Methodological and molecular data from reference specimens were annotated.</p><p><strong>Results: </strong>Overall, a median DNA concentration of 3.3 ng/µL (range 0.1-10.0 ng/µL) and 13.4 ng/µL (range 2.0-45.8 ng/µL) were obtained with automated and manual technical procedures, respectively. RNA concentrations of 5.7 ng/µL (range 0.2-11.9 ng/µL) and 9.3 ng/µL (range 0.5-18.0 ng/µL) were also detected. KRAS exon 2 p.G12C, EGFR exon 19 p.E736_A750del hotspot mutations, and ROS1 aberrant transcripts were identified in all tested cases, whereas 15 out of 16 (93.7%) centers detected MET exon 14 skipping mutation.</p><p><strong>Conclusions: </strong>Optimized technical workflows are crucial in the decision-making strategy of patients with NSCLC. Artificial reference specimens enable optimization of diagnostic workflows for predictive molecular analysis in routine clinical practice.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":" ","pages":"73-95"},"PeriodicalIF":2.7,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10881930/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139418318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}