Loading
Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record.
What is in development for Systemic mastocytosis, drawn from the whole corpus: 6 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Avapritinib carries intracranial bleeding risk at low platelet counts and cognitive effects; bezuclastinib and elenestinib are designed to avoid them.
The associated neoplasm in SM-AHN, not the mast cells, usually determines survival; combination strategies with hypomethylating agents and transplant are being studied.
Diagnostic delay of years in indolent disease; blood KIT D816V testing and tryptase genotyping are shortening it.
Long-term safety of chronic KIT inhibition in indolent patients with a normal life expectancy.
Dated changes read from the records linked to this cancer: approvals, regulatory steps, reported trials, guideline versions and milestones. Newest first; no date is inferred.
On EdgeAll 13 changes by month →When this page itself was last checked or edited.
H1 and H2 antihistamines, cromolyn, leukotriene antagonists, omalizumab for anaphylaxis, epinephrine autoinjector, bone protection; avapritinib 25 mg daily for moderate to severe symptoms uncontrolled by these (PIONEER). (NCCN Category 2A)
PIONEER; FDA approval May 2023, the first therapy for the indolent form.
Advanced systemic mastocytosis
Avapritinib 200 mg daily (platelets above 50 x 10^9/L) as preferred agent; midostaurin as alternative or where platelets are low. (NCCN Category 2A (preferred: avapritinib))
Overall response rate 75 percent and complete remission rate 36 percent in 53 evaluable patients with advanced systemic mastocytosis; maximum tolerated dose not reached; recommended dose 200 mg daily; approved June 2021.