Real-World Data on Chronic Myelomonocytic Leukemia : Clinical and Molecular Characteristics, Treatment, Emerging Drugs, and Patient Outcomes
Castaño-Díez, Sandra (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
López-Guerra, Mónica (Centro de Investigación Biomédica en Red de Cáncer)
Bosch-Castañeda, Cristina (Universitat de Barcelona)
Bataller, Alex (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Charry, Paola (Hospital Clínic i Provincial de Barcelona)
Esteban, Daniel (Hospital Clínic i Provincial de Barcelona)
Guijarro, Francesca (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Jiménez-Vicente, Carlos (Hospital Clínic i Provincial de Barcelona)
Castillo-Girón, Carlos (Hospital Clínic i Provincial de Barcelona)
Cortes, Albert (Institut d'Investigació Biomèdica Sant Pau)
Martínez-Roca, Alexandra (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Triguero, Ana (Hospital Clínic i Provincial de Barcelona)
Álamo, José Ramón (Hospital Clínic i Provincial de Barcelona)
Bea, Sílvia (Centro de Investigación Biomédica en Red de Cáncer)
Costa, Dolors (Centro de Investigación Biomédica en Red de Cáncer)
Colomer, Dolors (Centro de Investigación Biomédica en Red de Cáncer)
Rozman, Maria (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Esteve Reyner, Jordi (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Díaz-Beyá, Marina (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Universitat Autònoma de Barcelona

Date:	2022
Abstract:	Despite emerging molecular information on chronic myelomonocytic leukemia (CMML), patient outcome remains unsatisfactory and little is known about the transformation to acute myeloid leukemia (AML). In a single-center cohort of 219 CMML patients, we explored the potential correlation between clinical features, gene mutations, and treatment regimens with overall survival (OS) and clonal evolution into AML. The most commonly detected mutations were TET2, SRSF2, ASXL1, and RUNX1. Median OS was 34 months and varied according to age, cytogenetic risk, FAB, CPSS and CPSS-Mol categories, and number of gene mutations. Hypomethylating agents were administered to 37 patients, 18 of whom responded. Allogeneic stem cell transplantation (alloSCT) was performed in 22 patients. Two-year OS after alloSCT was 60. 6%. Six patients received targeted therapy with IDH or FLT3 inhibitors, three of whom attained a long-lasting response. AML transformation occurred in 53 patients and the analysis of paired samples showed changes in gene mutation status. Our real-world data emphasize that the outcome of CMML patients is still unsatisfactory and alloSCT remains the only potentially curative treatment. However, targeted therapies show promise in patients with specific gene mutations. Complete molecular characterization can help to improve risk stratification, understand transformation, and personalize therapy.
Grants:	Instituto de Salud Carlos III FIS PI19/01476
Note:	This research was supported by grants from resident award Contractes Clínic de Recerca Emili Letang-Josep Font 2021, granted by Hospital Clínic de Barcelona; and it was funded by Instituto de Salud Carlos III (ISCIII) through the project "FIS PI19/01476" and co-funded by the European Union.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Published in:	Cancers, Vol. 14 Núm. 17 (september 2022), p. 4107, ISSN 2072-6694

DOI: 10.3390/cancers14174107
PMID: 36077644

21 p, 3.2 MB

The record appears in these collections:
Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP) > Josep Carreras Leukaemia Research Institute
Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut de Recerca Sant Pau
Articles > Research articles
Articles > Published articles