Review
Oncology
Claire Andrews, Vinod Pullarkat, Christian Recher
Summary: CPX-351, a dual-drug liposomal encapsulation of daunorubicin and cytarabine, has been approved for treating newly diagnosed therapy-related AML or AML with myelodysplasia-related changes. In clinical trials, CPX-351 significantly improved overall survival in high-risk or secondary AML patients aged 60-75 years. FLT3 gene mutations are found in approximately 30% of newly diagnosed AML patients and may have a negative impact. CPX-351 combined with FLT3 inhibitors shows promise as a treatment option for FLT3 mutation-positive AML patients.
FRONTIERS IN ONCOLOGY
(2023)
Review
Oncology
Roberto M. Lemoli, Pau Montesinos, Akriti Jain
Summary: CPX-351, a liposomal encapsulation of daunorubicin/cytarabine, has been approved for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) and AML with myelodysplasia-related changes in adults. Real-world studies have evaluated its efficacy and safety, including its use in younger adults, measurable residual disease negativity, and outcomes by mutation. This review aims to provide information for prescribers to make informed treatment decisions.
CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY
(2023)
Article
Chemistry, Multidisciplinary
Melanie Donnette, Mourad Hamimed, Joseph Ciccolini, Yael Berda-Haddad, Elise Kaspi, Geoffroy Venton, Bruno Lacarelle, Regis Costello, L''Houcine Ouafik, Laure Farnault, Raphaelle Fanciullino
Summary: The pharmacokinetics of liposomal CPX-351 in treating AML patients were studied, revealing a major impact of CDA status on cytarabine PK and safety.
JOURNAL OF CONTROLLED RELEASE
(2021)
Article
Hematology
Andrew H. Matthews, Alexander E. Perl, Selina M. Luger, Alison W. Loren, Saar I. Gill, David L. Porter, Daria V. Babushok, Ivan P. Maillard, Martin P. Carroll, Noelle V. Frey, Elizabeth O. Hexner, Mary Ellen Martin, Shannon R. McCurdy, Edward A. Stadtmauer, Vikram R. Paralkar, Ximena Jordan Bruno, Wei -Ting Hwang, David Margolis, Keith W. Pratz
Summary: Using retrospective observational data, this study compared the outcomes of newly diagnosed AML patients receiving CPX-351 or ven/aza. The results showed no significant difference in overall survival between the two treatment methods. However, CPX-351 was associated with higher rates of infections, febrile neutropenia, and longer hospital stays.
Article
Oncology
Luana Fianchi, Fabio Guolo, Francesco Marchesi, Chiara Cattaneo, Michele Gottardi, Francesco Restuccia, Anna Candoni, Elettra Ortu La Barbera, Rita Fazzi, Crescenza Pasciolla, Olimpia Finizio, Nicola Fracchiolla, Mario Delia, Federica Lessi, Michelina Dargenio, Valentina Bonuomo, Maria Ilaria Del Principe, Patrizia Zappasodi, Marco Picardi, Claudia Basilico, Monica Piedimonte, Paola Minetto, Antonio Giordano, Patrizia Chiusolo, Lucia Prezioso, Caterina Buquicchio, Lorella Maria Antonia Melillo, Daniele Zama, Francesca Farina, Valentina Mancini, Irene Terrenato, Michela Rondoni, Irene Urbino, Mario Tumbarello, Alessandro Busca, Livio Pagano
Summary: This study evaluated the absolute infectious risk in 200 AML patients treated with CPX-351 and found it to have a good safety profile, with an incidence of infectious complications comparable to that of pivotal studies. The only factor significantly associated with mortality was a lack of response to CPX-351 treatment.
Review
Oncology
Shaykhah Alotaibi, Dietger Niederwieser, Syed Osman Ahmed, Jaime Sanz, Mohamad Mohty, Mahmoud Aljurf
Summary: AML treatment has evolved with the introduction of CPX-351 as a novel therapeutic approach, showing promise in improving the treatment outcomes for AML patients.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2022)
Article
Oncology
Teresa Bernal, Ainhoa Fernandez Moreno, Almudena de LaIglesia, Celina Benavente, Ana Garcia-Noblejas, Daniel Garcia Belmonte, Rosalia Riaza, Olga Salamero, Maria Angeles Foncillas, Alicia Roldan, Victor Noriega Concepcion, Laura Llorente Gonzalez, Juan Miguel Bergua Burgues, Soraya Lorente de Una, Gabriela Rodriguez-Macias, Adolfo de la Fuente Burguera, Maria Jose Garcia Perez, Jose Luis Lopez-Lorenzo, Pilar Martinez, Concepcion Alaez, Marta Callejas, Carmen Martinez-Chamorro, Jose Rifon Roca, Lourdes Amador Barciela, Armando V. Mena Duran, Karoll Gomez Correcha, Esperanza Lavilla Rubira, Maria Luz Amigo, Ferran Vall-llovera, Ana Garrido, Maria Garcia-Fortes, Dunia de Miguel Llorente, Anastasia Aules Leonardo, Carlos Cervero, Rosa Coll Jorda, Manuel M. Perez-Encinas, Marta Polo Zarzuela, Angela Figuera, Guillermo Rad, David Martinez-Cuadron, Pau Montesinos
Summary: This study aimed to compare the treatment outcomes of CPX-351 and standard chemotherapy in real-life patients. Through retrospective analysis and propensity score matching, it was found that CPX-351 showed similar efficacy in terms of complete remission rate and overall survival compared to standard chemotherapy. The study suggests that CPX-351 may have clinical benefits in real-life settings.
Article
Hematology
Yixin Hu, Kenneth J. Caldwell, Mihaela Onciu, Sara M. Federico, Marta Salek, Sara Lewis, Shaohua Lei, Jinghui Zhang, Kim E. Nichols, Clifford M. Takemoto, Brandon M. Triplett, Jason E. Farrar, Jeffrey E. Rubnitz, Raul C. Ribeiro, Marcin W. Wlodarski
Summary: CP X-351, a fixed ratio of liposomal cytarabine to daunorubicin, is effective and well-tolerated in pediatric sMDS/AML patients, showing complete morphologic remission without significant toxicity. Prospective studies are warranted to further evaluate its potential in this population.
Article
Immunology
Xiqin Tong, Fuling Zhou
Summary: This study analyzed the mutation status of 31 mitochondrial metabolism-related genes in AML patients and constructed a prognosis model based on five genes, which accurately distinguished high-risk and low-risk patients. It was also found that high-risk patients had more immune-cell infiltration and poor immunotherapy response.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Oncology
Christina Rautenberg, Friedrich Stoelzel, Christoph Roellig, Matthias Stelljes, Verena Gaidzik, Michael Lauseker, Oliver Kriege, Mareike Verbeek, Julia Marie Unglaub, Felicitas Thol, Stefan W. Krause, Mathias Haenel, Charlotte Neuerburg, Vladan Vucinic, Christian-Friedrich Jehn, Julia Severmann, Maxi Wass, Lars Fransecky, Jens Chemnitz, Udo Holtick, Kerstin Schaefer-Eckart, Josephine Schroeder, Sabrina Kraus, William Krueger, Ulrich Kaiser, Sebastian Scholl, Kathrin Koch, Lea Henning, Guido Kobbe, Rainer Haas, Nael Alakel, Maximilian-Alexander Roehnert, Katja Sockel, Maher Hanoun, Uwe Platzbecker, Tobias A. W. Holderried, Anke Morgner, Michael Heuser, Tim Sauer, Katharina S. Goetze, Eva Wagner-Drouet, Konstanze Doehner, Hartmut Doehner, Christoph Schliemann, Johannes Schetelig, Martin Bornhaeuser, Ulrich Germing, Thomas Schroeder, Jan Moritz Middeke
Summary: The study found that CPX-351 is an effective treatment for therapy-related acute myeloid leukemia or AML with myelodysplasia-related changes, especially for patients undergoing allogeneic hematopoietic cell transplantation. However, patients may experience significant non-hematologic toxicities during treatment.
BLOOD CANCER JOURNAL
(2021)
Article
Oncology
Akriti G. Jain, Somedeb Ball, Luis E. Aguirre, Katherine A. Tobon, Onyee Chan, Eric Padron, Andrew Kuykendall, Rami Komrokji, David Sallman, Jeffrey E. Lancet, Kendra Sweet
Summary: This study is the first to report on the use of next-generation sequencing (NGS) at best response and before allogeneic stem cell transplant (alloSCT) in patients treated with CPX-351 for acute myeloid leukemia. The study found that overall survival (OS) and relapse-free survival (RFS) were numerically longer among patients who cleared mutations, but this did not reach statistical significance. Additionally, alloSCT improved RFS regardless of mutational clearance.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)
Review
Oncology
Matteo Molica, Salvatore Perrone, Carla Mazzone, Laura Cesini, Martina Canichella, Paolo de Fabritiis
Summary: Therapy-related acute myeloid leukemia (t-AML) and acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) are aggressive forms of AML that have traditionally had poor outcomes with standard chemotherapy. However, the liposomal formulation CPX-351, which combines cytarabine and daunorubicin at a fixed molar ratio, has shown improved responses and survival rates in older patients with these high-risk AML subcategories. Future research aims to explore dose intensification, combination with targeted therapies, and the mechanism behind the improved responses. CPX-351 has the potential to change the treatment paradigm for high-risk and elderly AML patients.
Review
Oncology
Xiaomei Zhuang, Han Zhong Pei, Tianwen Li, Junbin Huang, Yao Guo, Yuming Zhao, Ming Yang, Dengyang Zhang, Zhiguang Chang, Qi Zhang, Liuting Yu, Chunxiao He, Liqing Zhang, Yihang Pan, Chun Chen, Yun Chen
Summary: Gain-of-function mutations of IDH1/2 are crucial in the development of AML, and ivosidenib and enasidenib have been approved as therapeutic targets. However, drug resistance remains a major challenge, highlighting the need for novel treatment strategies.
FRONTIERS IN ONCOLOGY
(2022)
Review
Biochemistry & Molecular Biology
Daniela Damiani, Mario Tiribelli
Summary: The prognosis of acute myeloid leukemia (AML) is poor due to tumor cell immune escape, which weakens T-cells. Inhibiting immune checkpoints (ICs) through immune checkpoint inhibitors (ICIs) has emerged as a promising therapeutic strategy for AML. However, the results of clinical trials testing ICIs, alone or in combination with other treatments, in AML are conflicting.
Article
Immunology
Ding Li, Xuan Wu, Cheng Cheng, Jiaming Liang, Yinfeng Liang, Han Li, Xiaohan Guo, Ruchun Li, Wenzhou Zhang, Wenping Song
Summary: This study developed a prognostic risk signature for acute myeloid leukemia (AML) by evaluating the expression profiles of genes related to lipid metabolism and immune modifications. The risk signature effectively stratified AML patients into low- and high-risk groups with significant differences in survival time.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Nanoscience & Nanotechnology
Daniela Rubanova, Svitlana Skoroplyas, Alena Libanska, Eva Randarova, Josef Bryja, Michaela Chorvatova, Tomas Etrych, Lukas Kubala
Summary: This study explored the use of nanocarriers to deliver dexamethasone for treating rheumatoid arthritis. The results showed that this method increased the accumulation of dexamethasone in the arthritic area, had a dose-dependent healing effect, and reduced the accumulation of the RA mediator RANKL. This study provides a feasible strategy for improving rheumatoid arthritis therapy.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
Article
Nanoscience & Nanotechnology
Thanpisit Lomphithak, Apiwit Sae-Fung, Simone Sprio, Anna Tampieri, Siriporn Jitkaew, Bengt Fadeel
Summary: In this study, iron-doped hydroxyapatite (FeHA) was evaluated as a potential nanomedicine approach for treating PDAC. The results showed that FeHA, in combination with a sublethal dose of the GPX4 inhibitor RSL3, could trigger ferroptosis in KRAS mutant PANC-1 cells while sparing normal human cells. The induction of ferroptosis by FeHA plus RSL3 was found to be dependent on the metalloreductase STEAP3 in PDAC cells.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
Review
Nanoscience & Nanotechnology
Marco Felipe Salas-Orozco, Ana Cecilia Lorenzo-Leal, Idania de Alba Montero, Nuria Patino Marin, Miguel Angel Casillas Santana, Horacio Bach
Summary: The emergence of antibiotic-resistant bacteria in severe infections is a growing concern, particularly in hospital environments. Metal-based nanomaterials have shown potential as an alternative to combat these bacteria, but there is a risk of bacterial resistance and environmental accumulation.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
