Systemic mastocytosis in adults: 2023 update on diagnosis, risk stratification and management.
Animesh D PardananiPublished in: American journal of hematology (2023)
Treatment goals for ISM patients are primarily directed toward anaphylaxis prevention/symptom control/osteoporosis treatment. Patients with advanced SM frequently need MC cytoreductive therapy to reverse disease-related organ dysfunction. Tyrosine kinase inhibitors (TKI) (midostaurin, avapritinib) have changed the treatment landscape in SM. While deep biochemical, histological and molecular responses have been documented with avapritinib treatment, its efficacy as monotherapy against a multimutated AMN disease component in SM-AMN patients remains unclear. Cladribine continues to have a role for MC debulking, whereas interferon-α has a diminishing role in the TKI era. Treatment of SM-AMN primarily targets the AMN component, particularly if an aggressive disease such as acute leukemia is present. Allogeneic stem cell transplant has a role in such patients. Imatinib has a therapeutic role only in the rare patient with an imatinib-sensitive KIT mutation.
Keyphrases
- end stage renal disease
- ejection fraction
- stem cells
- newly diagnosed
- chronic kidney disease
- clinical trial
- chronic myeloid leukemia
- tyrosine kinase
- randomized controlled trial
- combination therapy
- prognostic factors
- peritoneal dialysis
- single cell
- open label
- replacement therapy
- stem cell transplantation
- high dose
- dendritic cells
- immune response
- single molecule
- global health
- postmenopausal women