When someone is diagnosed with systemic mastocytosis (SM), they and their families will likely have numerous questions about treatment options.
Thanks to recent scientific discoveries, several newly approved and experimental treatments have shown promise in the management of SM. A paper recently published in Expert Opinion on Pharmacotherapy outlines current treatment options and those on the horizon.
The authors noted avapritinib “has emerged as the preferred therapy for ISM as well as advanced SM.” Avapritinib is a newer, targeted drug that blocks the abnormal KIT D816V mutation responsible for 90% of SM cases. This treatment significantly reduces mast cells, helps relieve symptoms and improves organ function in both indolent and advanced SM.
Learn more about SM treatment and care
Midostaurin is another targeted medicine approved for advanced SM. It works similarly to avapritinib, but may not be as effective in patients without the KIT D816V mutation.
Cladribine (2-chlorodeoxyadenosine) is a chemotherapy drug that has been shown to reduce mast cell numbers. However, it can cause immunosuppression and can lead to severely low blood cell counts, so it’s typically only used when other treatments are not an option for a patient.
Allogeneic hematopoietic cell transplantation (allo-HCT) may be considered for individuals with advanced SM who do not respond to other therapies. Eligibility is determined on a case by case basis, due to the risks involved. Complications include graft-versus-host disease (GVHD), a condition in which transplanted cells attack the recipient’s tissues.
With no cure, therapies for SM focus on symptom management and are tailored to individual needs. A comprehensive treatment plan typically involves avoiding factors that can trigger mast cells, such as insect stings and certain foods; drugs like omalizumab, which helps prevent allergic reactions; and medications to treat symptoms like antihistamines and corticosteroids.
New treatments are being studied, including drugs like bezuclastinib and elenestinib, which also target the KIT mutation. Early results show promise.
“Clinical trials involving these and other agents and/or combinations in the first line and relapsed/refractory settings are warranted in SM, and should be pursued when and where available,” the study’s authors conclude.
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