Treatment Options for Mastocytosis Diagnosed via Bone Biopsy
Disease Classification and Initial Management
- The National Comprehensive Cancer Network recommends that mastocytosis treatment depends on the World Health Organization (WHO) classification subtype, which includes indolent systemic mastocytosis (ISM), smoldering systemic mastocytosis (SSM), aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL) 3, 4
- Referral to specialized centers with expertise in mastocytosis is strongly recommended for optimal management, as stated by the National Comprehensive Cancer Network 1, 5
- All patients should be counseled about signs/symptoms and potential triggers of mast cell activation, and should carry injectable epinephrine to manage potential anaphylaxis, according to the National Comprehensive Cancer Network 1, 5
Treatment for Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM)
- Management focuses primarily on controlling mast cell activation symptoms with anti-mediator drug therapy, as recommended by the National Comprehensive Cancer Network 3, 6
- Regular monitoring includes health and physical exams and lab tests every 6-12 months and bone density scans every 1-3 years for patients with bone thinning, according to the National Comprehensive Cancer Network 3
- Clinical trials should be considered for patients with inadequate symptom control, as stated by the National Comprehensive Cancer Network 3
Treatment for Aggressive Systemic Mastocytosis (ASM)
- First-line therapy options include midostaurin, cladribine, or imatinib, as recommended by the National Comprehensive Cancer Network 1, 2, 5
- Allogeneic hematopoietic cell transplantation should be considered in eligible patients with adequate response to therapy, according to the National Comprehensive Cancer Network 1, 5
Treatment for Systemic Mastocytosis with Associated Hematologic Neoplasm (SM-AHN)
- Treatment is primarily governed by the associated non-mast cell neoplasm, as stated by the National Comprehensive Cancer Network 7
Treatment for Mast Cell Leukemia (MCL)
- Clinical trials are the preferred option, according to the National Comprehensive Cancer Network 7
- Midostaurin, cladribine, or multiagent chemotherapy may be used, as recommended by the National Comprehensive Cancer Network 5, 7
- Allogeneic hematopoietic cell transplantation should be considered for eligible patients, according to the National Comprehensive Cancer Network 5, 7
Monitoring and Response Assessment
- Re-staging should be performed with bone marrow tests, blood tests, and additional staging studies when there is return or progression of disease-related damage, as recommended by the National Comprehensive Cancer Network 1, 5, 7
- Response assessment should be based on improvement of disease-related symptoms and/or improvement of health findings, according to the National Comprehensive Cancer Network 3, 6