Midostaurin therapy for advanced systemic mastocytosis: Mayo Clinic experience in 33 consecutive cases
Sign Up to like & getrecommendations! Published in 2022 at "American Journal of Hematology"
DOI: 10.1002/ajh.26498
Abstract: We retrospectively examined our experience with midostaurin therapy in 33 consecutive patients (median age 68 years; 58% females) with advanced systemic mastocytosis (adv‐SM): aggressive SM (ASM; n = 17), SM associated with another hematologic neoplasm (SM‐AHN;… read more here.
Keywords: therapy; systemic mastocytosis; midostaurin therapy; advanced systemic ... See more keywords
Therapeutic management and outcome of patients with advanced systemic mastocytosis treated with midostaurin: A comprehensive real‐life study in the French national healthcare database
Sign Up to like & getrecommendations! Published in 2022 at "Hematological Oncology"
DOI: 10.1002/hon.3062
Abstract: Advanced Systemic Mastocytosis (Adv‐SM) is rare and has a poor prognosis. Midostaurin (Rydapt®) is one of the few treatments for Adv‐SM in Europe. The study aims were to describe the characteristics of patients treated with… read more here.
Keywords: systemic mastocytosis; advanced systemic; treatment; treated midostaurin ... See more keywords
SETD2 and histone H3 lysine 36 methylation deficiency in advanced systemic mastocytosis
Sign Up to like & getrecommendations! Published in 2018 at "Leukemia"
DOI: 10.1038/leu.2017.183
Abstract: The molecular basis of advanced systemic mastocytosis (SM) is not fully understood and despite novel therapies the prognosis remains dismal. Exome sequencing of an index-patient with mast cell leukemia (MCL) uncovered biallelic loss-of-function mutations in… read more here.
Keywords: setd2 histone; setd2; systemic mastocytosis; advanced systemic ... See more keywords
Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial
Sign Up to like & getrecommendations! Published in 2018 at "Leukemia"
DOI: 10.1038/leu.2017.234
Abstract: Patients with advanced systemic mastocytosis (SM) (e.g. aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL)) have limited treatment options and exhibit reduced survival. Midostaurin is an oral multikinase… read more here.
Keywords: median follow; advanced systemic; systemic mastocytosis; patients advanced ... See more keywords
Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial
Sign Up to like & getrecommendations! Published in 2021 at "Nature Medicine"
DOI: 10.1038/s41591-021-01539-8
Abstract: Advanced systemic mastocytosis (AdvSM) is a rare, KIT D816V-driven hematologic neoplasm characterized by mast cell infiltration and shortened survival. We report the results of a prespecified interim analysis of an ongoing pivotal single-arm phase 2 trial… read more here.
Keywords: response; systemic mastocytosis; interim analysis; advanced systemic ... See more keywords
Midostaurin in Advanced Systemic Mastocytosis: A Systematic Review and Meta-analysis.
Sign Up to like & getrecommendations! Published in 2022 at "American journal of therapeutics"
DOI: 10.1097/mjt.0000000000001508
Abstract: Please view the PDF To see the formatted meeting abstract. read more here.
Keywords: review meta; mastocytosis systematic; systemic mastocytosis; systematic review ... See more keywords
Midostaurin in daily clinical practice of patients with advanced systemic mastocytosis
Sign Up to like & getrecommendations! Published in 2025 at "British Journal of Haematology"
DOI: 10.1111/bjh.70065
Abstract: Midostaurin, a multikinase/KIT inhibitor, is approved for the treatment of advanced systemic mastocytosis (AdvSM). We evaluated dosing regimens, safety, efficacy and overall survival (OS) of 79 patients from the ‘German Registry on Disorders of Eosinophils… read more here.
Keywords: systemic mastocytosis; response; advanced systemic; modified valent ... See more keywords
A New Prognostic Score for Advanced Systemic Mastocytosis Based on Clinical and Genetic Characteristics of 210 Consecutive Patients
Sign Up to like & getrecommendations! Published in 2018 at "Blood"
DOI: 10.1182/blood-2018-99-115804
Abstract: According to the WHO classification, advanced systemic mastocytosis (advSM) comprises aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL). SM-AHN is the most common subtype of advSM, diagnosed in… read more here.
Keywords: risk; advanced systemic; clinical genetic; advsm ... See more keywords
Response and resistance to cladribine in patients with advanced systemic mastocytosis: a registry-based analysis.
Sign Up to like & getrecommendations! Published in 2022 at "Annals of hematology"
DOI: 10.1182/blood-2022-163650
Abstract: We sought to evaluate the efficacy of the purine analogue cladribine in 79 patients with advanced systemic mastocytosis (AdvSM) using data from the 'German Registry on Disorders of Eosinophils and Mast Cells (GREM)'. The overall… read more here.
Keywords: cladribine patients; cladribine; patients advanced; systemic mastocytosis ... See more keywords
Avapritinib for Advanced Systemic Mastocytosis.
Sign Up to like & getrecommendations! Published in 2022 at "Blood"
DOI: 10.1182/blood.2021014612
Abstract: Avapritinib, a selective inhibitor of KIT D816V, was approved by the Food and Drug Administration in 2021 for treatment of advanced systemic mastocytosis (AdvSM), and by the European Medicines Agency in 2022 for AdvSM patients… read more here.
Keywords: advanced systemic; avapritinib advanced; systemic mastocytosis;
From Pathways to Patients in Atopic Dermatitis: Advanced Systemic Therapies
Sign Up to like & getrecommendations! Published in 2025 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms262311487
Abstract: Atopic dermatitis is the most prevalent chronic inflammatory skin disease, posing a significant individual and healthcare burden. Traditionally managed with topical agents and broad immunosuppressants, the treatment landscape has shifted significantly in recent years. This… read more here.
Keywords: pathways patients; dermatitis; systemic therapies; patients atopic ... See more keywords