Publication: Búsqueda de nuevos factores pronósticos y de terapias combinadas en el tratamiento de neoplasias mieloproliferativas
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2018-09-07
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Universidad Complutense de Madrid
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Abstract
La mielofibrosis (MF) es una neoplasia mieloproliferativa crónica (NMPc) caracterizadapor una proliferación clonal de la célula madre mieloide patológica y por diferentesgrados de fibrosis de la médula ósea. La mutación más frecuente en MF es JAK2V617Fque se encuentra en aproximadamente el 50-60% de los pacientes con MF. Lasmutaciones de CALR se encuentran en un 35-50% de los pacientes. La diversidad clínicay de respuesta al tratamiento de estos pacientes no puede ser explicada con lasmutaciones en JAK2, CALR y MPL. Por lo que resulta necesario el estudio de nuevasmutaciones.Hasta la fecha, el único tratamiento específico aprobado para el tratamiento de la MFes el ruxolitinib, un inhibidor específico de JAK1/2. Ha demostrado ser un tratamientoeficaz, pero la búsqueda de tratamientos combinados con ruxolitinib responde a lanecesidad de conseguir un aumento de la eficacia del tratamiento y disminución de sutoxicidad...
Myelofibrosis (MF) is a Philadelphia chromosome-negative myeloproliferativeneoplasm (NMPc). It is a clonal disorders characterized by increased production ofmature blood cells and bone marrow fibrosis. There are three types of MF: primary MF(PMF), post-polycythemia vera MF (PV-MF) and post-essential thrombocythemia MF(ET-MF). The most frequent mutation is JAK2V617F. It can be found around 50-60% ofMF. The CALR mutations are in approximately 35-50% of patients. The variety ofclinical manifestations and response to treatment cannot be solely explained by thesemutations. Thus, it is necessary looking for new mutations in MF patients.The only therapy approved to treat MF is ruxolitinib, a JAK1/2 inhibitor. It has showedto be an effective therapy. However, the screening of new approaches, likecombination therapy with ruxolitinib, is an interesting way to increase its efficacy anddecrease the toxicity of ruxolitinib...
Myelofibrosis (MF) is a Philadelphia chromosome-negative myeloproliferativeneoplasm (NMPc). It is a clonal disorders characterized by increased production ofmature blood cells and bone marrow fibrosis. There are three types of MF: primary MF(PMF), post-polycythemia vera MF (PV-MF) and post-essential thrombocythemia MF(ET-MF). The most frequent mutation is JAK2V617F. It can be found around 50-60% ofMF. The CALR mutations are in approximately 35-50% of patients. The variety ofclinical manifestations and response to treatment cannot be solely explained by thesemutations. Thus, it is necessary looking for new mutations in MF patients.The only therapy approved to treat MF is ruxolitinib, a JAK1/2 inhibitor. It has showedto be an effective therapy. However, the screening of new approaches, likecombination therapy with ruxolitinib, is an interesting way to increase its efficacy anddecrease the toxicity of ruxolitinib...
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Tesis de la Universidad Complutense de Madrid, Facultad de Ciencias Químicas, Departamento de Bioquímica y Biología Molecular I, leída el 05-07-2017