The survival outcomes of nonadvanced SM are excellent. Patients with SM can have a significant symptom burden that substantially affects their quality of life.4,5 These symptoms are related to release of mast cell mediators and organ infiltration. Patients with SM can present with a variety of symptoms as well as the presence of B findings (high disease burden without organ dysfunction) and C findings (organ dysfunction from mast cell infiltration). There are 5 subtypes of SM: indolent SM (ISM) and smoldering SM (SSM), considered together as nonadvanced SM aggressive SM (ASM) SM with an associated hematologic neoplasm (SM-AHN) or myeloid neoplasm and mast cell leukemia (MCL), the last 3 considered together as advanced SM (~10% of the overall SM population). These are frequently the bone marrow and gastrointestinal tract. SM, on the other hand, refers to the neoplastic mast cell infiltration of 1 or more extracutaneous tissues. Both the 5th edition of the World Health Organization classification of myeloid neoplasms and the International Consensus Classification of Myeloid Neoplasms organize mastocytosis into 2 broad types: cutaneous mastocytosis and SM.2,3 The former is mostly seen in children and the mastocytosis is confined to the cutaneous tissues only. KIT inhibitors have helped alter the treatment paradigm for patients with SM. The hematologic neoplasm systemic mastocytosis (SM) is marked by the clonal proliferation of mast cells in the skin, bone marrow, gastrointestinal tract, spleen, and/or liver, caused by an activating KIT mutation in the KIT gene.1 Notably, in approximately 95% of patients, this mutation is identified as KIT D816V in exon 17, and in most instances, patients with harbor other mutations. The hematologic neoplasm systemic mastocytosis is marked by the clonal proliferation of mast cells in the skin, bone marrow, gastrointestinal tract, spleen, and/or liver, caused by an activating KIT mutation in the KIT gene. The company is developing elenestinib (BLU-263), an investigational, orally available, potent and highly selective KIT inhibitor, for the treatment of indolent SM and other mast cell disorders.KIT Inhibitors Lead to Shift in Treatment of Systemic Mastocytosis Its pipelines also include Elenestinib (BLU-263) (KIT), AYVAKIT (avapritinib) (KIT), Wild-type KIT research program, GAVRETO (pralsetinib) (RET), BLU-945 (EGFR), BLU-525 (EGFR), BLU-451 (EGFR exon 20 insertions), BLU-222 (CDK2), AYVAKIT (PDGFRA), GAVRETO (RET), BLU-222 (CDK2) and BLU-852 (MAP4K1). The firm delivers its approved medicines, including AYVAKIT/AYVAKYT (avapritinib) and GAVRETO (pralsetinib), to patients in the United States and Europe, and it is globally advancing multiple programs for systemic mastocytosis (SM), lung cancer, breast cancer and other genomically defined cancers, and cancer immunotherapy. The company is headquartered in Cambridge, Massachusetts and currently employs 660 full-time employees. is a precision therapy company, which engages in the invention of medicines for people with cancer and blood disorders.
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