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Background: Advanced systemic mastocytosis advSM is characterized by presence of the KIT DV mutation and pathologic accumulation of neoplastic mast cells MCs in various tissues, leading to severe symptoms and organ damage eg, cytopenias, liver dysfunction, portal hypertension, malabsorption, and weight loss.

Objective: We aimed to determine whether midostaurin improves health-related quality of life QOL and MC mediator-related symptoms in patients with advSM. Methods: In patients with systemic mastocytosis 89 patients with advSM fulfilling the strict inclusion criteria of the D study [ClinicalTrials.

MC mediator-related symptoms were evaluated by using a specific physician-reported questionnaire. Results: Over the first 6 cycles of treatment with midostaurin ie, 6 months , patients experienced significant improvements in total SF and Memorial Symptom Assessment Scale scores, as well as in subscores of each instrument. These improvements were durable during 36 months of follow-up. Keywords: Advanced systemic mastocytosis; KIT mutation; mastocytosis; mediator symptoms; midostaurin; quality of life; skin lesions of mastocytosis; tryptase.

Published by Elsevier Inc. All rights reserved. Abstract Background: Advanced systemic mastocytosis advSM is characterized by presence of the KIT DV mutation and pathologic accumulation of neoplastic mast cells MCs in various tissues, leading to severe symptoms and organ damage eg, cytopenias, liver dysfunction, portal hypertension, malabsorption, and weight loss. Substances Protein Kinase Inhibitors Staurosporine midostaurin.