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El gen del receptor denominado FLT3 es uno de los genes más comúnmente mutados en la leucemia mieloide aguda (LMA), en una frecuencia de alrededor del 30% de los pacientes (pts). La mutación más frecuente que confiere mal pronóstico es la duplicación en tándem dentro del dominio yuxtamembrana de dicho receptor (FLT3-ITD). Con menor frecuencia se detectan mutaciones puntuales del dominio de tirosina kinasa (FLT3-TKD). El sorafenib y la midostaurina son inhibidores de multikinasas (ITK) no específicos para las mutaciones del FLT3, han sido evaluados como monoterapia con respuestas transitorias en pts recaídos o refractarios (r/r) debido posiblemente a la falta de potencia y especificidad. Resultados recientes de ensayos clínicos muestran los posibles beneficios de los inhibidores de FLT3 (IFLT3) en pts con LMA de reciente diagnóstico en combinación con quimioterapia estándar de inducción, consolidación, mantenimiento y post trasplante.
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