Systemic Mastocytosis: Clinical Update and Future Directions

Publication date: Available online 5 August 2015 Source:Clinical Lymphoma Myeloma and Leukemia Author(s): Douglas Tremblay, Nicole Carreau, Marina Kremyanskaya, John Mascarenhas Systemic mastocytosis (SM) is defined as the accumulation of abnormal mast cells (MC) in one or more extracutaneous tissues. Symptoms are due to either MC activation or organ infiltration and vary depending on disease subtype. More benign forms of SM, such as indolent SM (ISM) have a life expectancy similar to the general population while more aggressive subtypes, such as mast cell leukemia (MCL), have a median survival measured on the order of months. Treatment of ISM is directed at controlling the symptoms associated with MC activation. In advanced forms, such as aggressive SM (ASM) and MCL, agents targeting MC proliferation such as KIT tyrosine kinase inhibitors (TKIs), cladribine, and thalidomide may be employed. Newer agents based on pre-clinical rationale are also being actively investigated. However, the only potentially curative therapy for ASM/MCL remains hematopoietic stem cell transplantation. Given that SM is a relatively rare disease, clinicians are often underprepared to evaluate, diagnose, and effectively treat this clinically heterogeneous condition. This manuscript will better familiarize the clinician with this orphan disease and review current and future treatment approaches.
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research