From the Guidelines
Treatment for mastocytosis should focus on symptom management, with H1 antihistamines like cetirizine (10mg daily) or fexofenadine (180mg daily) and H2 blockers such as ranitidine (150mg twice daily) or famotidine (20mg twice daily) as first-line treatments, and midostaurin (100mg twice daily) for advanced systemic mastocytosis with specific genetic mutations, as recommended by the most recent guidelines 1.
Key Treatment Options
- H1 antihistamines: cetirizine (10mg daily) or fexofenadine (180mg daily) to control itching, flushing, and hives
- H2 blockers: ranitidine (150mg twice daily) or famotidine (20mg twice daily) to manage gastrointestinal symptoms
- Oral cromolyn sodium (200mg four times daily) to stabilize mast cells for severe symptoms
- Midostaurin (100mg twice daily) for advanced systemic mastocytosis with specific genetic mutations, such as KIT D816V mutation
- Corticosteroids for acute flares
- Epinephrine auto-injector for anaphylactic reactions
Important Considerations
- Regular monitoring of disease progression through blood tests and bone marrow examinations is essential
- Patients should avoid known triggers such as alcohol, extreme temperatures, certain medications, and physical stress
- Treatment is lifelong for most patients as mastocytosis results from abnormal accumulation of mast cells that release histamine and other inflammatory mediators when triggered
- Referral to specialized centers with expertise in mastocytosis and multidisciplinary collaboration with subspecialists is recommended, as stated in the recent guidelines 1
Recent Guidelines
The most recent guidelines from 2024 1 emphasize the importance of symptom management and targeted therapies for advanced systemic mastocytosis, and provide evidence- and consensus-based recommendations for the diagnosis and comprehensive care of patients with systemic mastocytosis.
From the FDA Drug Label
RYDAPT is indicated for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL). The recommended dose of RYDAPT for patients with ASM, SM-AHN, and MCL is 100 mg orally twice daily with food. Continue treatment until disease progression or unacceptable toxicity occurs.
The treatment option for mastocytosis (Mast Cell Disorder) is midostaurin (RYDAPT), which is indicated for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL). The recommended dose is 100 mg orally twice daily with food until disease progression or unacceptable toxicity occurs 2.
- Key points:
- Midostaurin (RYDAPT) is the treatment option for mastocytosis.
- The recommended dose is 100 mg orally twice daily with food.
- Treatment should continue until disease progression or unacceptable toxicity occurs.
- The drug is indicated for adult patients with ASM, SM-AHN, or MCL.
From the Research
Treatment Options for Mastocytosis
The treatment options for mastocytosis are varied and depend on the individual patient's clinical presentation and prognosis. Some of the main treatment options include:
- H1 and H2 antihistamines, which are the mainstay of treatment for most categories of mastocytosis 3
- Corticosteroids, which are added for more severe symptoms 3
- Cladribine and interferon alpha, which have been shown to be therapeutically effective in some patients 4
- Sodium cromoglicate, which is licensed for the treatment of mastocytosis and can be effective in controlling gastrointestinal symptoms, as well as symptoms such as bone pain, fatigue, and headache when used in conjunction with inhaled sodium cromoglicate 5
- Avoidance of known symptom triggers, which is an important aspect of treatment 6
- Cytoreductive therapies, such as interferon-alpha and cladribine, which may be considered for patients with treatment-refractory symptoms or smoldering mastocytosis 4, 6
- Leukotriene receptor antagonists and PUVA therapy, which may also be used to treat symptoms 7
Specific Treatment Considerations
Some specific considerations for treatment include:
- The use of epinephrine solution for emergency use in patients who suffer from systemic reactions such as hypotension or syncope 7
- The need for a multidisciplinary preoperative assessment, adequate premedications, and close intra- and postoperative monitoring in patients undergoing surgery 6
- The potential need for increased doses of sodium cromoglicate during the menstrual period or when challenged with a food to which the patient is sensitive 5