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Updated: Sep 6, 2025
![Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography](/_next/image?url=https%3A%2F%2Fcloudfront.jove.com%2FCDNSource%2Fteasers%2F62334.jpg&w=3840&q=50)
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Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography
Published on: February 17, 2022
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Target Therapies for Systemic Mastocytosis: An Update
Mariarita Sciumè1, Claudio De Magistris2, Nicole Galli2
1Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Pharmaceuticals (Basel, Switzerland)
|June 24, 2022
Summary
Systemic mastocytosis (SM) involves abnormal mast cell growth and is linked to the KIT D816V mutation. Newer tyrosine kinase inhibitors (TKIs) like midostaurin and avapritinib offer improved treatment options for advanced SM.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Systemic mastocytosis (SM) is a clonal disorder characterized by abnormal mast cell proliferation in organs.
- SM subtypes range from indolent to aggressive forms, impacting patient prognosis.
- The KIT D816V mutation is frequently implicated in SM pathogenesis.
Purpose of the Study:
- To review current clinical data on therapies for systemic mastocytosis.
- To explore emerging therapeutic targets for SM management.
- To provide insights into the evolving treatment landscape for SM.
Main Methods:
- Review of clinical trial data and published literature on SM therapies.
- Analysis of treatment responses to tyrosine kinase inhibitors (TKIs).
- Evaluation of therapeutic strategies based on SM subtype and mutation status.
Main Results:
- Early TKIs were effective only in KIT D816V-negative SM.
- Newer TKIs, including midostaurin and avapritinib, show efficacy against the KIT D816V mutation.
- Treatment approaches vary from symptom management in indolent SM to cytoreductive therapy in advanced disease.
Conclusions:
- Advances in TKIs have significantly improved systemic mastocytosis treatment, particularly for advanced or KIT D816V-mutated cases.
- Personalized therapy based on SM subtype and genetic profile is crucial.
- Continued research into novel therapeutic targets is essential for further improving outcomes in SM.
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