Article
Oncology
Marie-Emilie Dourthe, Florence Rabian, Karima Yakouben, Florian Chevillon, Aurelie Cabannes-Hamy, Francoise Mechinaud, Audrey Grain, Delphine Chaillou, Ilhem Rahal, Sophie Caillat-Zucman, Emmanuelle Lesprit, Jerome Naudin, Julie Roupret-Serzec, Nathalie Parquet, Anne Brignier, Valerie Guerin-El Khourouj, Elodie Lainey, Aurelie Caye-Eude, Helene Cave, Emmanuelle Clappier, Stephanie Mathis, Elie Azoulay, Jean Hugues Dallel, Nathalie Dhedin, Isabelle Madelaine, Jerome Larghero, Nicolas Boissel, Andre Baruchel
Summary: Tisagenlecleucel therapy has shown promising efficacy in relapsed/refractory BCP-ALL patients, with a high complete remission rate at D28. However, factors such as prior therapy and disease burden can impact the risk of relapse and overall survival. Detectable MRD at D28 and loss of BCA may define patients at high risk of relapse, requiring additional interventions.
Article
Oncology
Andrew H. Wei, Josep-Maria Ribera, Richard A. Larson, David Ritchie, Armin Ghobadi, Yuqi Chen, Abraham Anderson, Cedric E. Dos Santos, Janet Franklin, Hagop Kantarjian
Summary: This study identified platelets, tumor burden, and percentage of T cells as prognostic and predictive biomarkers for outcomes in adults with Philadelphia chromosome-negative relapsed/refractory B-cell precursor acute lymphoblastic leukemia receiving blinatumomab or chemotherapy. Adjusting these biomarkers may help in identifying subgroups most likely to benefit from blinatumomab treatment.
Article
Hematology
Sandheeah Ramdeny, Asima Chaudhary, Austen Worth, Sara Ghorashian, Mary Slatter, Su Han Lum, Ajay Vora
Summary: This study reported two patients with B-precursor acute lymphoblastic leukemia (BCP-ALL) and congenital T-cell immunodeficiency who achieved excellent responses to blinatumomab, demonstrating the efficacy of this antibody in such patients.
Article
Oncology
Manon Queudeville, Anthony S. Stein, Franco Locatelli, Martin Ebinger, Rupert Handgretinger, Nicola Goekbuget, Lia Gore, Yi Zeng, Priya Gokani, Gerhard Zugmaier, Hagop M. Kantarjian
Summary: The study found that a lower baseline bone marrow blast percentage (bBMB%) is associated with better treatment outcomes in patients with B-cell acute lymphoblastic leukemia (B-ALL) receiving blinatumomab. This suggests that bBMB% <50% is associated with improved efficacy following blinatumomab treatment for B-ALL.
Article
Hematology
Sara Ghorashian, Elad Jacoby, Barbara De Moerloose, Susana Rives, Denise Bonney, Geoff Shenton, Peter Bader, Nicole Bodmer, Agueda Molinos Quintana, Blanca Herrero, Mattia Algeri, Franco Locatelli, Kim Vettenranta, Berta Gonzalez, Andishe Attarbaschi, Stephen Harris, Jean Pierre Bourquin, Andre Baruchel
Summary: This study analyzed the feasibility, efficacy, and safety of tisagenlecleucel treatment in young children and infants with acute lymphoblastic leukemia. The results suggest that tisagenlecleucel has antitumor activity and an acceptable safety profile for this age group.
LANCET HAEMATOLOGY
(2022)
Article
Oncology
Ibrahim Aldoss, Salman Otoukesh, Jianying Zhang, Sally Mokhtari, Dat Ngo, Mona Mojtahedzadeh, Monzr M. Al Malki, Amandeep Salhotra, Haris Ali, Ahmed Aribi, Karamjeet S. Sandhu, Shukaib Arslan, Paul Koller, Brian Ball, Forrest Stewart, Peter Curtin, Andrew Artz, Ryotaro Nakamura, Guido Marcucci, Stephen J. Forman, Anthony S. Stein, Vinod Pullarkat
Summary: Blintumomab shows promising activity in relapsed/refractory acute lymphoblastic leukemia (ALL) patients, but there are risk factors for treatment failure, particularly extramedullary relapse and retention of CD19 expression.
Article
Oncology
Min'er Gu, Yahong Xia, Jingying Zhang, Yongmin Tang, Weiqun Xu, Hua Song, Xiaojun Xu
Summary: This study demonstrates for the first time that blinatumomab can further eradicate MRD in B-ALL patients after achieving MFC-MRD undetectable, providing effective treatment for extremely low levels of MRD (up to <10(-6)).
Review
Oncology
Hoda Pourhassan, Vaibhav Agrawal, Vinod Pullarkat, Ibrahim Aldoss
Summary: Blinatumomab is an effective targeted immunotherapy for relapsed/refractory Philadelphia chromosome-positive and Philadelphia chromosome-negative acute lymphoblastic leukemia (ALL), with favorable safety and activity. Multiple completed and ongoing studies have shown promising results, including improved response rates and survival outcomes, decreased treatment toxicity, and reduced need for multi-agent chemotherapy regimens. The early use of blinatumomab has demonstrated success in both Ph-negative and Ph-positive B-ALL, even in older adults with historically inferior outcomes. This review summarizes the current data and future directions for the early use of blinatumomab in newly diagnosed adults with B-cell ALL.
FRONTIERS IN ONCOLOGY
(2023)
Article
Immunology
Han-Yu Cao, Hui Chen, Song-Bai Liu, Wen-Jie Gong, Chong-Sheng Qian, Tong-Tong Zhang, Chao-Ling Wan, Si-Man Huang, Nan Xu, Hai-Ping Dai, Sheng-Li Xue
Summary: The treatment of B-cell acute lymphoblastic leukemia (B-ALL) with central nervous system (CNS) involvement is challenging due to weak permeability of chemotherapeutic agents to the blood-brain barrier (BBB) and potential complications of current anti-CNS leukemia treatments. Immunotherapy, particularly blinatumomab, shows potential in treating CNS leukemia with both cerebral spinal fluid and cerebral parenchymal involvement, but further studies are needed.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Oncology
Nicola Stefano Fracchiolla, Mariarita Sciume, Cristina Papayannidis, Antonella Vitale, Sabina Chiaretti, Mario Annunziata, Fabio Giglio, Prassede Salutari, Fabio Forghieri, Davide Lazzarotto, Monia Lunghi, Annalisa Imovilli, Barbara Scappini, Massimiliano Bonifacio, Michelina Dargenio, Carmela Gurrieri, Elisabetta Todisco, Marzia Defina, Maria Ilaria Del Principe, Patrizia Zappasodi, Marco Cerrano, Lidia Santoro, Elena Tagliaferri, Enrico Barozzi, Pasquale De Roberto, Marta Canzi, Elisa Buzzatti, Chiara Sartor, Francesco Passamonti, Robin Foa, Antonio Curti, Thomas Wirth
Summary: This retrospective study evaluated the efficacy and safety of blinatumomab and inotuzumab ozogamicin in the treatment of relapsed B-lymphoblastic leukemia. The study demonstrated the feasibility and effectiveness of sequential immunotherapy using these two drugs in terms of minimal residual disease, overall survival, and disease-free survival.
Review
Cell & Tissue Engineering
Aurelio Matos Andrade, Vitoria Rodrigutes Teixera, Robert Pogue, Ana Claudia Morais Godoy Figueiredo, Juliana Lott Carvalho
Summary: This study evaluated the cost-effectiveness of tisagenlecleucel compared with conventional salvage therapies for pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). The results showed that tisagenlecleucel is more expensive than traditional therapies, but its ICER value is still good, and it is more effective than conventional drugs in terms of life years and QALY gained.
Article
Health Care Sciences & Services
Niamh Carey, Joy Leahy, Lea Trela-Larsen, Laura McCullagh, Michael Barry
Summary: This study evaluates the cost-effectiveness of tisagenlecleucel versus blinatumomab for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia in Ireland. The results indicate that tisagenlecleucel is not cost effective and further research may not be of value.
INTERNATIONAL JOURNAL OF TECHNOLOGY ASSESSMENT IN HEALTH CARE
(2022)
Article
Medicine, Research & Experimental
Maziar Moradi-Lakeh, Mohsen Yaghoubi, Patrick Seitz, Mehdi Javanbakht, Elisabeth Brock
Summary: This analysis explores the cost-effectiveness of tisagenlecleucel in pALL and DLBCL in Switzerland compared to historical standard-of-care treatments. The study used cost-utility models, clinical trial data, and expert input to determine that tisagenlecleucel is a cost-effective option in both indications.
ADVANCES IN THERAPY
(2021)
Article
Oncology
Max S. Topp, Nicola Goekbuget, Gerhard Zugmaier, Anthony S. Stein, Herve Dombret, Yuqi Chen, Josep-Maria Ribera, Ralf C. Bargou, Heinz-August Horst, Hagop M. Kantarjian
Summary: This study analyzed long-term follow-up data from two clinical trials involving blinatumomab, finding that one-third of patients with relapsed and/or refractory B-cell precursor acute lymphoblastic leukemia achieving a deep response with blinatumomab lived for 3 years or longer. These results suggest that long-term survival is possible after treatment with blinatumomab, with some patients even undergoing stem cell transplantation showing improved survival outcomes.
Article
Oncology
Katarzyna Pawinska-Wasikowska, Aleksandra Wieczorek, Walentyna Balwierz, Karolina Bukowska-Strakova, Marta Surman, Szymon Skoczen
Summary: This study retrospectively assessed the efficacy and toxicity of blinatumomab in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL). The results showed that blinatumomab is not only a feasible therapeutic option, but also an effective treatment with acceptable toxicity profile in pretreated children with relapsed/refractory BCP-ALL, paving the way for hematopoietic stem cell transplantation (HSCT).
Review
Economics
Amy Gye, Stephen Goodall, Richard De Abreu Lourenco
Summary: This review aimed to identify the sources of variability in cost-effectiveness analyses of CAR-T therapies for different patient populations. The study found that while there was some consistency in the clinical evidence and economic models used, the outcomes varied, especially in young patients. The long-term treatment benefit in young patients was associated with greater uncertainty due to potential lifelong benefits with cell and gene therapies.
Article
Clinical Neurology
Kate A. Carey, Michelle A. Farrar, Nadine A. Kasparian, Deborah J. Street, Richard De Abreu Lourenco
Summary: This study aimed to understand the factors that influence decision-making in the treatment of infantile spinal muscular atrophy (SMA). The results showed that treatments with a higher chance of improving functioning and mobility were preferred, while treatments with higher costs, invasive delivery, and risk of adverse events were less preferred. The study also revealed that cost influenced treatment choices for healthcare professionals and the general population, while change in mobility and mode of administration were most influential for parents.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2022)
Article
Medicine, Research & Experimental
Sibel Saya, Lucy Boyd, Patty Chondros, Mairead McNamara, Michelle King, Shakira Milton, Richard De Abreu Lourenco, Malcolm Clark, George Fishman, Julie Marker, Cheri Ostroff, Richard Allman, Fiona M. Walter, Daniel Buchanan, Ingrid Winship, Jennifer McIntosh, Finlay Macrae, Mark Jenkins, Jon Emery
Summary: This study aims to investigate whether using personalized CRC risk assessment based on PRS in primary care can increase risk-appropriate CRC screening. By providing risk reports and personalized screening recommendations in general practice, it can accurately predict CRC risk and improve screening rates.
Review
Oncology
Amy Brown, Jackie Yim, Scott Jones, Alex Tan, Emily Callander, Kerrianne Watt, Richard De Abreu Lourenco, Tilley Pain
Summary: This study assessed literature on men's preferences and perceptions of prostate cancer radiation therapy. A total of 27 studies were included, covering various stages and themes related to information needs, preferences and decisions, experiences, side effects, and support. However, there was a lack of research on patient preferences and perceptions, despite the numerous clinical and technical studies published in this area.
CLINICAL AND TRANSLATIONAL RADIATION ONCOLOGY
(2023)
Review
Health Care Sciences & Services
Maria Farris, Stephen Goodall, Richard De Abreu Lourenco
Summary: This study aims to summarize the key methodological challenges faced by health technology assessment (HTA) agencies in assessing gene therapy, as well as the consideration of broad elements of value. The study selected economic evaluations of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English. The key challenges identified include the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect.
INTERNATIONAL JOURNAL OF TECHNOLOGY ASSESSMENT IN HEALTH CARE
(2023)
Review
Public, Environmental & Occupational Health
Rachael H. Dodd, Ashleigh R. Sharman, Deborah McGregor, Emily Stone, Candice Donnelly, Richard De Abreu Lourenco, Henry Marshall, Nicole M. Rankin
Summary: International lung cancer screening trials using low-dose computed tomography have shown clinical effectiveness in reducing lung cancer mortality. This systematic review aims to synthesize the key messages and strategies for increasing awareness and knowledge of screening. The review found that providing information on screening recommendations, benefits and harms, cost and insurance coverage, and eligibility criteria are important for increasing knowledge and intention to screen. Educational videos and digital awareness campaigns may be effective in increasing engagement, while group based learning is suitable for increasing health professionals' knowledge.
PREVENTIVE MEDICINE
(2023)
Article
Medicine, General & Internal
Daniel A. Goldstein, Gary M. Ginsberg, Dorit Hofnung-Gabbay, Richard De Abreu Lourenco, Herbert H. Loong, Boon Cher Goh, Kelvin K. W. Chan, Massimo Di Maio, Francesco Perrone, Peter S. Hall, Alona Zer, Eli Rosenbaum
Summary: The US FDA has recently approved new dosing options for immune checkpoint inhibitors, including extended-interval fixed dosing. The economic impact of this new dosing strategy compared to the standard dosing strategy is unknown.
Article
Urology & Nephrology
Matthew J. Roberts, Ciara Conduit, Ian D. Davis, Rachel M. Effeney, Scott Williams, Jarad M. Martin, Michael S. Hofman, George Hruby, Renu Eapen, Chris Gianacas, Nathan Papa, Richard De Abreu Lourenco, Haryana M. Dhillon, Ray Allen, Antoinette Fontela, Baldeep Kaur, Louise Emmett
Summary: The efficacy of early salvage radiation therapy (SRT) compared to surveillance in patients with PSA recurrence after radical prostatectomy, but no identifiable recurrent disease on PSMA-PET/CT, is unknown.
Editorial Material
Medicine, General & Internal
Gemma McErlean, Ashish Bajel, Abir Bhattacharyya, Ngaire Brown, Richard De Abreu Lourenco, Matthew Greenwood, Ian Kerridge, Nancy Kim, David Kliman, Della Maneze, Tracey O'Brien, Jeff Szer, Ida Twist
INTERNAL MEDICINE JOURNAL
(2023)
Article
Medicine, General & Internal
Eng-Siew Koh, Hui K. Gan, Clare Senko, Roslyn J. Francis, Martin Ebert, Sze Ting Lee, Eddie Lau, Mustafa Khasraw, Anna K. Nowak, Dale L. Bailey, Bradford A. Moffat, Greg Fitt, Rodney J. Hicks, Robert Coffey, Roel Verhaak, Kyle M. Walsh, Elizabeth H. Barnes, Richard De Abreu Lourenco, Mark Rosenthal, Lucas Adda, Farshad Foroudi, Arian Lasocki, Alisha Moore, Paul A. Thomas, Paul Roach, Michael Back, Robyn Leonard, Andrew M. Scott
Summary: FET-PET has the potential to impact adjuvant radiotherapy planning, differentiate between treatment-induced pseudoprogression and true tumor progression, and predict prognosis in glioblastoma management. The FIG study is a multicenter phase II study aiming to investigate the impact of FET-PET versus standard MRI on radiotherapy volume delineation and the accuracy and management impact of FET-PET in distinguishing pseudoprogression from true tumor progression.
Article
Primary Health Care
Jon D. Emery, Mark A. Jenkins, Sibel Saya, Patty Chondros, Jasmeen Oberoi, Shakira Milton, Kitty Novy, Emily Habgood, Napin Karnchanachari, Marie Pirotta, Lyndal Trevena, Adrian Bickerstaffe, Richard De Abreu Lourenco, Anna Crothers, Driss Ait Ouakrim, Louisa Flander, James G. Dowty, Fiona M. Walter, Malcolm Clark, Sally Doncovio, Dariush Etemadmoghadam, George Fishman, Finlay Macrae, Ingrid Winship, Jennifer G. McIntosh
Summary: Using a risk assessment and decision support tool can increase the rate of risk-appropriate colorectal cancer screening. It is important to start screening for colorectal cancer at the appropriate age and choose the most cost-effective testing method.
BRITISH JOURNAL OF GENERAL PRACTICE
(2023)
Meeting Abstract
Medicine, General & Internal
E. S. Koh, A. Moore, R. J. Francis, M. A. Ebert, H. K. Gan, S. T. Lee, E. Lau, A. Rossi, A. Grose, S. P. Ng, E. H. Barnes, B. A. Moffat, F. E. Scott, L. Adda, F. Foroudi, A. K. Nowak, D. L. Bailey, M. Back, R. De Abreu Lourenco, A. M. Scott
INTERNAL MEDICINE JOURNAL
(2022)
Article
Oncology
Ciara Conduit, Blossom Mak, Wenjia Qu, Juliana Di Lulio, Ronan Burder, Matthias Bressel, Thomas Cusick, Haryana M. Dhillon, Richard De Abreu Lourenco, Craig Underhill, Javier Torres, Megan Crumbaker, Florian Honeyball, Anthony Linton, Ray Allen, Ian D. Davis, Susan J. Clark, Lisa G. Horvath, Kate L. Mahon
Summary: The study aims to assess the efficacy and safety of intermittent docetaxel chemotherapy guided by circulating mGSTP1 in men with metastatic castration-resistant prostate cancer. It plans to recruit 120 patients for randomised observation and evaluate the utility of mGSTP1 as a biomarker in guiding treatment.
THERAPEUTIC ADVANCES IN MEDICAL ONCOLOGY
(2022)