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
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
Hematology
Edmond Chiche, Ramy Rahme, Sarah Bertoli, Pierre-Yves Dumas, Jean-Baptiste Micol, Yosr Hicheri, Florence Pasquier, Pierre Peterlin, Patrice Chevallier, Xavier Thomas, Michael Loschi, Alexis Genthon, Ollivier Legrand, Mohamad Mohty, Emmanuel Raffoux, Patrick Auberger, Alexis Caulier, Magalie Joris, Caroline Bonmati, Gabrielle Roth-Guepin, Caroline Lejeune, Arnaud Pigneux, Norbert Vey, Christian Recher, Lionel Ades, Thomas Cluzeau
Summary: This study retrospectively analyzed the efficacy and safety of CPX-351 in 103 patients from 12 French centers, finding a favorable safety profile and a high overall response rate. Presence of high-risk molecular prognosis subgroups did not significantly impact response to CPX-351, while spliceosome mutations were associated with better overall survival.
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
Jad Othman, Charlotte Wilhelm-Benartzi, Richard Dillon, Steve Knapper, Sylvie D. Freeman, Leona M. Batten, Joanna Canham, Emily L. Hinson, Julie Wych, Sophie Betteridge, William Villiers, Michelle Kleeman, Amanda Gilkes, Nicola Potter, Ulrik Malthe Overgaard, Priyanka Mehta, Panagiotis Kottaridis, Jamie Cavenagh, Claire Hemmaway, Claire Arnold, Mike Dennis, Nigel H. Russell
Summary: This study compared the efficacy of CPX-351 and FLAG-Ida in younger patients, and found no significant difference in overall survival between the two treatments. However, the use of CPX-351 significantly prolonged relapse-free survival, particularly in patients with MDS-related gene mutations.
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
Jorge E. Cortes, Tara L. Lin, Geoffrey L. Uy, Robert J. Ryan, Stefan Faderl, Jeffrey E. Lancet
Summary: CPX-351, a liposomal encapsulation of daunorubicin and cytarabine, demonstrated significant improvement in median overall survival for patients with newly diagnosed high-risk/secondary acute myeloid leukemia compared to conventional 7 + 3 chemotherapy. Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis showed that CPX-351 provided higher quality-adjusted survival benefits, supporting its clinical benefit in this patient population.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Article
Hematology
Pierre Peterlin, Yannick Le Bris, Pascal Turlure, Patrice Chevallier, Audrey Menard, Marie-Pierre Gourin, Pierre-Yves Dumas, Sylvain Thepot, Ana Berceanu, Sophie Park, Marie -Anne Hospital, Thomas Cluzeau, Simon Bouzy, Jose-Miguel Torregrosa-Diaz, Louis Drevon, Rosa Sapena, Fatiha Chermat, Lionel Ades, Sophie Dimicoli-Salazar, Sylvie Chevret, Marie-Christine Bene, Pierre Fenaux
Summary: CPX-351 has been shown to be safe and effective in treating patients with higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia, allowing for bridging to allogeneic hematopoietic stem cell transplantation in most patients.
LANCET HAEMATOLOGY
(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.
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
Alex Legg, Alexandrina Lambova, Anne Broe, Julia Levy, Greg Medalla
Summary: This retrospective study aimed to describe and compare the characteristics and survival outcomes of younger and older patients with AML treated with CPX-351. The results suggest that CPX-351 is an effective treatment for both younger and older adults with AML.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)
Article
Oncology
Jorge E. Cortes, Tara L. Lin, Kobby Asubonteng, Stefan Faderl, Jeffrey E. Lancet, Thomas Prebet
Summary: CPX-351, a dual-drug liposomal encapsulation, has shown improved outcomes in older adults with high-risk/secondary AML compared to traditional treatment. A post hoc analysis of a phase 3 study revealed higher remission rates, longer overall survival, and post-transplant survival with CPX-351, particularly in adverse-risk AML patients.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2022)