1. Safety and efficacy of asciminib treatment in chronic myeloid leukemia patients in real-life clinical practice
- Author
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Garcia-Gutierrez, Valentin, Luna, Alejandro, Alonso-Dominguez, Juan M., Estrada Barreras, Natalia, Boque, Concepcion, Xicoy, Blanca, Giraldo, Pilar, Angona, Anna, Álvarez Larrán, Alberto, Sanchez-Guijo, Fermin, Ramírez, María José, Mora, Elvira, Vélez, Patricia, Rosell, Ana, Colorado Araujo, Mercedes, Cuevas, Beatriz, Sagüés, Miguel, Cortés, Montserrat, Pérez Encinas, Manuel, Casado Montero, Luis Felipe, Moreno Vega, Melania, Serrano, Luis, Gomez, Valle, Garcia-Hernandez, Carmen, Lakhwani, Sunil, Paz Coll, Antonio, De Paz, Raquel, Suarez-Varela, Sara, Fernandez-Ruiz, Andrés, Perez Lopez, Raul, Ortiz-Fernández, Almudena, Jiménez-Velasco, Antonio, Steegmann-Olmedillas, Juan Luis, Hernandez-Boluda, Juan Carlos, Universitat Autònoma de Barcelona, [Garcia-Gutiérrez V, Luna A] Hematology, Hospital Universitario Ramón y Cajal. IRYCIS, Madrid, Spain. [Alonso-Dominguez JM] Hospital Universitario Fundación Jiménez Díaz, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), UAM, Madrid, Spain. [Estrada N, Xicoy B] Institut Català d’Oncologia, Hospital Germans Trias i Pujol, Josep Carreras Leukemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain. [Boque C] Institut Català d’Oncologia – L’Hospitalet de Llobregat, L’Hospitalet de Llobregat, Spain. [Cortes M] Hospital General de Granollers, Granollers, Spain, and Departament de Salut
- Subjects
Adult ,Male ,Niacinamide ,medicine.medical_specialty ,Heterocyclic Compounds::Acids, Heterocyclic::Nicotinic Acids::Niacinamide [CHEMICALS AND DRUGS] ,MEDLINE ,Fusion Proteins, bcr-abl ,Drug development ,Hemic and Lymphatic Diseases::Hematologic Diseases::Bone Marrow Diseases::Myeloproliferative Disorders::Leukemia, Myelogenous, Chronic, BCR-ABL Positive [DISEASES] ,lcsh:RC254-282 ,Chemical Actions and Uses::Pharmacologic Actions::Molecular Mechanisms of Pharmacological Action::Enzyme Inhibitors::Protein Kinase Inhibitors [CHEMICALS AND DRUGS] ,Young Adult ,Text mining ,Leukemia, Myelogenous, Chronic, BCR-ABL Positive ,hemic and lymphatic diseases ,Correspondence ,compuestos heterocíclicos::ácidos heterocíclicos::ácidos nicotínicos::niacinamida [COMPUESTOS QUÍMICOS Y DROGAS] ,Medicine ,In real life ,Humans ,Intensive care medicine ,Protein Kinase Inhibitors ,Aged ,Aged, 80 and over ,Leucèmia mieloide crònica - Tractament ,business.industry ,acciones y usos químicos::acciones farmacológicas::mecanismos moleculares de acción farmacológica::inhibidores enzimáticos::inhibidores de proteínas cinasas [COMPUESTOS QUÍMICOS Y DROGAS] ,Myeloid leukemia ,Hematology ,Middle Aged ,lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,Tiroxina - Inhibidors ,Clinical Practice ,Treatment Outcome ,Oncology ,Molecularly targeted therapy ,Proteïna quinasa CK2 ,Pyrazoles ,Female ,enfermedades hematológicas y linfáticas::enfermedades hematológicas::enfermedades de la médula ósea::trastornos mieloproliferativos::leucemia mielogenosa crónica BCR-ABL positiva [ENFERMEDADES] ,business - Abstract
Chronic myeloid leukemia; Asciminib; Treatment Leucemia mieloide crónica; Asciminib; Tratamiento Leucèmia mieloide crònica; Asciminib; Tractament Despite the excellent overall survival (OS) of chronic myeloid leukemia (CML) patients, a significant proportion will fail currently available tyrosine-kinase inhibitors (TKIs) due to resistance or intolerance. Intolerant patients are usually managed successfully with alternative second-generation tyrosine-kinase inhibitors (2GTKIs). However, more than half of the patients will eventually discontinue second-line treatment due to loss of response or toxicity. Ponatinib is an effective drug in the setting of resistance to 2GTKIs, however with life-threatening side effects and varying responses. Asciminib is a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor that potently and specifically inhibits BCR-ABL1 via binding to a pocket distinct from the ATP binding site of the kinase. Asciminib has the potential to overcome resistance to prior TKIs, and also offers the possibility of dual inhibition of BCR-ABL1 in combination with ATP-binding TKIs. Asciminib has been evaluated in a phase I study in patients with Ph-positive leukemia failing prior TKIs, with promising results. Our aim is to share the first data on the use of asciminib in CML patients in clinical practice, allowed by Novartis under a managed-access program (MAP).
- Published
- 2021