NDLI: Atypical Chronic Myeloid Leukemia: New Developments from Molecular Diagnosis to Treatment

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Atypical Chronic Myeloid Leukemia: New Developments from Molecular Diagnosis to Treatment

Content Provider	MDPI
Author	Castellino, Alessia Santambrogio, Elisa Rapezzi, Davide Massaia, Massimo
Copyright Year	2021
Description	Atypical Chronic Myeloid Leukemia, BCR-ABL1 negative (aCML) is a rare hematological entity, included in the group of myelodysplastic (MDS)/myeloproliferative (MPN) overlap syndromes. It is characterized by an aggressive course, a high rate of acute myeloid leukemia (AML) transformation, and a dismal outcome. The clinical presentation includes splenomegaly and leukocytosis with neutrophilia and left-shifted granulocytosis accompanied by granulocytic dysplasia and sometimes multilineage dysplasia. In past years, the disease incidence was likely underestimated, as diagnosis was only based on morphological features. Recently, the improving knowledge in the molecular biology of MDS/MPN neoplasms has made it possible to distinguish aCML from other overlapping syndromes, basing on next generation sequencing. Among the most commonly mutated genes, several involve the Jak-STAT, MAPK, and ROCK signaling pathways, which could be actionable with targeted therapies that are already used in clinical practice, opening the way to tailored treatment in aCML. However, currently, there are few data available for small samples, and allogeneic transplant remains the only curative option for eligible patients.
Starting Page	1104
e-ISSN	16489144
DOI	10.3390/medicina57101104
Journal	Medicina
Issue Number	10
Volume Number	57
Language	English
Publisher	MDPI
Publisher Date	2021-10-14
Access Restriction	Open
Subject Keyword	Medicina Oncology Atypical Chronic Myeloid Leukemia Myelodysplastic (mds)/myeloproliferative (mpn) Overlap Syndromes Next-generation Sequencing Target Therapy Allogenic Transplant
Content Type	Text
Resource Type	Article

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