Molecular profiling and targeted inhibitor therapy in atypical chronic myeloid leukaemia in blast crisis

Atypical chronic myeloid leukaemia (aCML) is a rare myelodysplastic/myeloproliferative (MDS/MPN) neoplasm morphologically characterised by leukocytosis, immature granulocytes in the peripheral blood (PB) and granulocytic and megakaryocytic dysplasia in the bone marrow (BM).1 Blast crisis is frequent and associated with an exceedingly poor prognosis.2 Allogeneic stem cell transplantation is the only potentially curative option but in those patients ineligible, treatment has largely relied on agents used for MDS and MPN with varying degrees of success.3 Mutations in genes associated with other myeloid malignancies are detected in all patients with 60% of cases harbouring more than one mutation.4 This underlying molecular complexity allied to heterogeneous clinical features means that no current standard of care exists for patients with aCML. Personalised treatment of patients with aCML requires characterisation of the individuals' molecular landscape with selection and addition of an appropriate inhibitor(s) to existing cytoreductive...

from # All Medicine by Alexandros G. Sfakianakis via alkiviadis.1961 on Inoreader http://ift.tt/2mLWVIm