"Mast"ering drug discovery with iPSCs.

  title={"Mast"ering drug discovery with iPSCs.},
  author={Adrienne Dorrance},
  volume={137 15},
2 Citations

Comprehensive Analysis of Acquired Genetic Variants and Their Prognostic Impact in Systemic Mastocytosis

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.

Precision Medicine in Systemic Mastocytosis

The clinical, diagnostic, and therapeutic issues of SM are reviewed, with special emphasis on the translational implications of SM genetics for a precision medicine approach in clinical practice.



Nintedanib Targets KIT D816V Neoplastic Cells Derived from Induced Pluripotent Stem cells of Systemic Mastocytosis

A panel of patient-specific KIT D816V induced pluripotent stem cells from patients with aggressive SM and mast cell leukemia is generated to develop a patient- specific SM disease model for mechanistic and drug discovery studies and suggests nintedanib as a new drug candidate for targeted therapy of advanced SM.

New Developments in Diagnosis, Prognostication, and Treatment of Advanced Systemic Mastocytosis.

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.

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.

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.