Impact of genotype on leukaemic transformation in polycythaemia vera and essential thrombocythaemia

The influence of driver mutations on leukaemic transformation was analysed in 1747 patients with polycythaemia vera or essential thrombocythaemia. With a median follow-up of 7·2 years, 349 patients died and 62 progressed to acute leukaemia or myelodysplastic syndrome. Taking death as a competing ris...

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Detalles Bibliográficos
Autores: Alvarez-Larrán, Alberto, Senín Magan, Maria Alicia, Fernández Rodríguez, M. Concepción, Pereira, Arturo, Arellano-Rodrigo, Arturo, Gómez, Montse, Ferrer-Marín, Francisca, Martínez-López, Joaquín, Camacho Díaz, Laura, Colomer Pujol, Dolors, Angona Figueras, Anna, Navarro, Blanca, Cervantes, Francisco, Besses Raebel, Carles, Bellosillo Paricio, Beatriz, Hernández-Boluda, Juan Carlos
Tipo de recurso: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2017
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:10230/34428
Acceso en línea:http://hdl.handle.net/10230/34428
http://dx.doi.org/10.1111/bjh.14762
Access Level:acceso abierto
Palabra clave:Leucèmia
Essential thrombocythaemia
Genotype
Myeloid leukaemia
Polycythaemia vera
Prognostic factors
Descripción
Sumario:The influence of driver mutations on leukaemic transformation was analysed in 1747 patients with polycythaemia vera or essential thrombocythaemia. With a median follow-up of 7·2 years, 349 patients died and 62 progressed to acute leukaemia or myelodysplastic syndrome. Taking death as a competing risk, CALR genotype was associated with a lower risk of transformation [subdistribution hazard ratio (SHR): 0·13, 95% confidence interval (CI): 0·2-0·9, P = 0·039], whereas JAK2 V617F showed borderline significance for higher risk (SHR: 2·05, 95% CI: 0·9-4·6, P = 0·09). Myelofibrotic transformation increased leukaemic risk, except in CALR-mutated patients. Next generation sequencing of 51 genes at the time of transformation showed additional mutations (median number: 3; range: 1-5) in 25 out of 29 (86%) assessable cases. Mutations (median: 1; range: 1-3) were detected in 67% of paired samples from the chronic phase. Leukaemia appeared in a JAK2 V617F negative clone in 17 (58%) cases, eleven of them being previously JAK2 V617F-positive. JAK2 V617F-mutated leukaemia was significantly associated with complex karyotype and acquisition of TP53 mutations, whereas EZH2 and RUNX1 mutations were more frequent in JAK2 V617F-negative leukaemia. Survival was longer in JAK2 V617F-unmutated leukaemia (343 days vs. 95 days, P = 0·003). In conclusion, CALR genotype is associated with a lower risk of leukaemic transformation. Leukaemia arising in a JAK2 V617F-negative clone is TP53 independent and shows better survival