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Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis

J Allergy Clin Immunol. 2020. doi: 10.1016/j.jaci.2020.03.044


Advanced systemic mastocytosis (advSM) is characterized by presence of the KIT D816V 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). Treatment with midostaurin, an orally active multikinase/KIT inhibitor now approved for advSM in the United States and the European Union, resulted in a high rate of response accompanied by reduced MC infiltration of the bone marrow and lowered serum tryptase level.


We aimed to determine whether midostaurin improves health-related quality of life (QOL) and MC mediator–related symptoms in patients with advSM.


In 116 patients with systemic mastocytosis (89 patients with advSM fulfilling the strict inclusion criteria of the D2201 study [ identifier NCT00782067]), QOL and symptom burden were assessed during treatment with midostaurin by using the 12-Item Short-Form Health Survey (SF-12) and the Memorial Symptom Assessment Scale patient-reported questionnaires, respectively. MC mediator–related symptoms were evaluated by using a specific physician-reported questionnaire.


Over the first 6 cycles of treatment with midostaurin (ie, 6 months), patients experienced significant improvements in total SF-12 and Memorial Symptom Assessment Scale scores, as well as in subscores of each instrument. These improvements were durable during 36 months of follow-up. Similarly, we found substantial improvements (67%-100%) in all MC mediator–related symptoms.


QOL and MC mediator–related symptoms significantly improve with midostaurin treatment in patients with advSM ( identifier, NCT00782067).


Key words: Advanced systemic mastocytosis, mutation, mastocytosis, mediator symptoms, midostaurin, quality of life, tryptase, skin lesions of mastocytosis.

Abbreviations used: AdvSM-Advanced systemic mastocytosis, ASM-Aggressive systemic mastocytosis, BM-Bone marrow, ITT-Intention-to-treat, MC-Mast cell, MC-QoL-Mastocytosis Quality of Life Questionnaire, MQLQ-Mastocytosis Quality-of-Life Questionnaire, MSAS-Memorial Symptom Assessment Scale, PEP-Primary efficacy population, QOL-Quality of life, SF-12-12-Item Short-Form Health Survey, SM-Systemic mastocytosis, SM-AHN-Systemic mastocytosis with an associated hematologic neoplasm, SSC-Study steering committee.