Real-World Data on Chronic Myelomonocytic Leukemia: Clinical and Molecular Characteristics, Treatment, Emerging Drugs, and Patient Outcomes

• Published in: Cancers 2022, Vol.14 (17)
• Main Authors: Castaño-Díez, Sandra, López-Guerra, Mónica, Bosch-Castañeda, Cristina, Bataller, Alex, Charry, Paola, Esteban, Daniel, Guijarro, Francesca, Jiménez-Vicente, Carlos, Castillo-Girón, Carlos, Cortes, Albert, Martínez-Roca, Alexandra, Triguero, Ana, Álamo, José Ramón, Beà, Silvia, Costa, Dolors, Colomer, Dolors, Rozman, María, Esteve, Jordi, Díaz-Beyá, Marina
• Format: Report
• Language: English
• Subjects: Care and treatment; Chronic myelomonocytic leukemia; Diagnosis; Patient outcomes
• ISSN: 2072-6694; 2072-6694
• DOI: 10.3390/cancers14174107

Chronic myelomonocytic leukemia (CMML) is an infrequent disease with poor prognosis and risk of progression into acute myeloid leukemia (AML). Stem cell transplantation (alloSCT) is the only potentially curative option. New targeted drugs (NTDs) directed at specific gene mutations are useful in AML, but little is known about how CMML progresses to AML and if these drugs are effective in CMML. In our study, 38% of patients received hypomethylating agents but less than half of them responded. Six patients received NTDs and responded well. AlloSCT was possible in only 10% of patients. Progression to AML occurred in 25% of patients, and there were changes in their gene mutations between the time of diagnosis and the time of progression. Although prognosis is poor in CMML, analyzing gene mutations can help to better stratify the risk of each patient and to identify potentially effective NTDs for each patient.