Comprehensive Analysis of Acquired Genetic Variants and Their Prognostic Impact in Systemic Mastocytosis
- Biology, MedicineCancers
Assessment of multilineage involvement of haematopoiesis by KIT D816V and additional mutations in genes known to be associated with the prognosis of SM have become of great help to identify good vs. poor-prognosis SM patients who could benefit from a closer follow-up and, eventually, also early cytoreductive treatment.
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Nintedanib Targets KIT D816V Neoplastic Cells Derived from Induced Pluripotent Stem cells of Systemic Mastocytosis.
- Biology, MedicineBlood
This study identified nintedanib, an FDA approved angiokinase inhibitor that targets VEGFR, PDGFR and FGFR, as a novel KIT D816V inhibitor and suggested nintinganib as a new drug candidate for K IT D 816V targeted therapy of advanced SM.
New Developments in Diagnosis, Prognostication, and Treatment of Advanced Systemic Mastocytosis.
- Medicine, BiologyBlood
Looking forward, perhaps the most fruitful marriage of the advances in molecular genetics and treatment will be the design of adaptive basket trials that combine histopathology and genetic profiling to individualize treatment approaches for patients with diverse AHNs and relapsed/refractory SM.
Advances in the Classification and Treatment of Mastocytosis: Current Status and Outlook toward the Future.
- MedicineCancer research
An overview of recent advances in the field of mastocytosis is provided, with emphasis on classification, prognostication, and emerging new treatment options in advanced systemic mastocyTosis.
Stage-Specific Human Induced Pluripotent Stem Cells Map the Progression of Myeloid Transformation to Transplantable Leukemia.
- BiologyCell stem cell
Molecular profiling of myeloid progenitor cells in multi-mutated advanced systemic mastocytosis identifies KIT D816V as a distinct and late event
Data indicate that SM-AHNMD is a multi-mutated neoplasm, KIT D816V or other mutations are rare in CFU-GM colonies of ISM/SSM patients, which might explain at least in part their better prognosis.
Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts.
An overview of recent developments in the field of mastocytosis is provided, with emphasis on the updated WHO classification, refined criteria, additional prognostic parameters, and novel therapeutic approaches.