Midostaurin (systemic mastocytosis) ' Benefit assessment according to '35a Social Code Book V 1 Translation of Sections I 1 to I 6 of the dossier assessment Midostaurin (akute myeloische Leukämie) – Nutzenbewertung gemäß § 35a SGB V. Please note: This translation is provided as a service by IQWiG to English-language readers. However, solely the German original text is absolutely authoritative and legally binding. Midostaurin (systemic mastocytosis) Benefit assessment according to §35a SGB V1 EXTRACT Project: A23-111 Version: 1.0 Status: 9 Feb 2024 DOI: 10.60584/A23-111_en Extract of dossier assessment A23-111 Version 1.0 Midostaurin ( systemic mastocytosis) 9 Feb 2024 Institute for Quality and Efficiency in Health Care (IQWiG) - i - Publishing details Publisher Institute for Quality
Avapritinib (Ayvakyt) for the treatment of indolent systemic mastocytosis (ISM) ENGLISH | DEUTSCH ATOM RSS 1.0 RSS 2.0SIMPLE SEARCHADVANCED SEARCHHELPSERVICESLOGINBrowseTypeSubjectAuthor / EditorInstitutionYear AIHTA - Publications - Search - Avapritinib (Ayvakyt®) for the treatment of indolent systemic mastocytosis (ISM)Rothschedl, E. and Grössmann, N. (2023): Avapritinib (Ayvakyt® ) for the treatment of indolent systemic mastocytosis (ISM). Fact Sheet Nr. 157. PDF - Sie müssen einen PDF-Viewer auf Ihrem PC installiert haben wie z. B. GSview, Xpdf oder Adobe Acrobat Reader110kBItem Type: Horizon Scanning of MedicinesSubjects: QU Biochemistry > QU 300-375 CellsQV Pharmacology, toxicology, pharmacy > QV 60-370 PharmacologyQZ Pathology > QZ 200-380 Neoplasms.CystsWB Practice of medicine > WB 300
Avapritinib (Ayvakyt) - advanced systemic mastocytosis (AdvSM) View of Avapritinib (Ayvakyt) | Canadian Journal of Health Technologies Return to Article DetailsAvapritinib (Ayvakyt)
Avapritinib (Ayvakyt) for the treatment of advanced systemic mastocytosis (AdvSM) Avapritinib (Ayvakyt®) for the treatment of advanced systemic mastocytosis (AdvSM) - Repository of AIHTA GmbH English | Deutsch Atom RSS 1.0 RSS 2.0 * Simple search * Advanced search * Help * Services * Login * Browse * Type * Subject * Author / Editor * Institution * YearAIHTA - Publications - Search - Avapritinib (Ayvakyt®) for the treatment of advanced systemic mastocytosis (AdvSM) Wolf, S.(2022):Avapritinib (Ayvakyt®) for the treatment of advanced systemic mastocytosis (AdvSM). Oncology Fact Sheet Nr. 78.Preview PDF- Sie müssen einen PDF-Viewer auf Ihrem PC installiert haben wie z. B. GSview, Xpdf oder Adobe Acrobat Reader 119kB
Prognostic impact of expression of CD2, CD25, and/or CD30 in/on mast cells in systemic mastocytosis: a registry study of the European Competence Network on Mastocytosis. Expression of CD2, CD25 and/or CD30 in extracutaneous mast cells (MC) is a minor diagnostic criterion for systemic mastocytosis (SM) in the classification of the World Health Organization and International Consensus Classification. So far, it remains unknown whether expression of these antigens on MC is of prognostic significance in SM. We performed a retrospective multi-center study of patients with SM using the data set of the registry of the European Competence Network on Mastocytosis, including 5034 patients with various MC disorders. The percentage of CD2, CD25 and/or CD30 MC was considerably lower in patients
Prevalence of hypersensitivity reactions in various forms of mastocytosis: A pilot study of 2485 adult patients with mastocytosis collected in the ECNM registry. Hypersensitivity reactions (HR) are common in mastocytosis. However, little is known about triggers and risk factors. The registry of the European Competence Network on Mastocytosis (ECNM) enables reliable studies in a larger cohort of mastocytosis patients. We assessed prevalence, triggers and risk factors of HR in adults with mastocytosis in the ECNM registry. Data were collected in 27 ECNM centers. We analyzed potential triggers (Hymenoptera venoms, food, drug, inhalant and others) and risk factors at diagnosis and during follow-up. The study group consisted of 2485 adults with mastocytosis, 1379 women (55.5%) and 1106 men (44.5
Evaluation of allergic diseases in children diagnosed with cutaneous mastocytosis. Cutaneous mastocytosis (CM) is the most common type of mastocytosis in children. The atopy frequency in these patients is typically similar to that in the general population, but a higher incidence of anaphylaxis is reported. This study aimed to evaluate the presence of allergic diseases in children diagnosed
How I diagnose and treat systemic mastocytosis with an associated hematologic neoplasm. Over the last decade significant advances have been made by honing in on the diagnostic evaluation and the significance of molecular profiles in patients with systemic mastocytosis (SM), non-advanced and advanced.This is reflected in the 2022 iterations of the World Health Organization Edition 5 of patients with advanced systemic mastocytosis with an associated hematologic neoplasm and is a personal perspective using some illustrative patient cases treated at our centre, Guys and St Thomas's Hospitals, London: UK centre of excellence in Mastocytosis.
Confirmed Systemic Mastocytosis in a Pediatric Patient With Widespread Cutaneous Symptoms. Mastocytosis is characterized by the clonal expansion of mast cells, with deposition into various organs and variable clinical presentation depending on subtype. It generally results from a mutation in the KIT gene, which encodes for production of receptor tyrosine kinases, the constitutive activity of which results in abnormal cell growth and proliferation. In pediatric patients, the cutaneous mastocytosis (CM) form predominates, and systemic mastocytosis (SM) is rarely reported. Accordingly, clinical course and management are not well described. We describe a case of SM in a 10-year-old child who was initially suspected of having widespread CM. The child had initially minimal systemic symptoms
Midostaurin (Rydapt) - for the treatment of adult patients with aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, or mast cell leukaemia Published 9 July 2018 Statement of Advice: midostaurin 25mg soft capsules (Rydapt®) SMC2100 Novartis Pharmaceuticals UK Ltd 8 June 2018 ADVICE: in the absence of a submission from the holder of the marketing authorisation midostaurin (Rydapt®) is not recommended for use within NHSScotland. Indication under review: as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, or mast cell leukaemia. The holder of the marketing authorisation has not made a submission to SMC regarding this product
Standardized indolent systemic mastocytosis evaluations across a healthcare system: implications for screening accuracy. Timely diagnosis of systemic mastocytosis (SM) remains challenging due to care heterogeneity. We implemented a standardized approach for SM screening and diagnosis utilizing a novel healthcare system-wide international screening registry. A retrospective analysis assessed rates of SM, cutaneous mastocytosis (CM), and molecular diagnoses before and two years after care standardization. Accuracy of individual and combined SM screening tests - basal serum tryptase (BST) ≥11.5 and ≥20.0 ng/mL, REMA ≥2, monomorphic maculopapular CM, and elevated BST based upon tryptase genotype - was analyzed. Tryptase genotyping and high-sensitivity KIT p.D816V testing increased substantially
Epidemiology of mastocytosis: a population-based study (Sweden). Mastocytosis is a disease characterized by accumulation of aberrant mast cells and mediator-related symptoms and is divided into systemic mastocytosis (SM) and cutaneous mastocytosis (CM). The epidemiology of mastocytosis remains incompletely understood. To estimate the incidence, prevalence, overall survival (OS) and burden of comorbidities in adult mastocytosis patients identified in Swedish population-based registries. Individuals (≥ 20 years of age) with a mastocytosis diagnosis in the National Patient Register (NPR) and/or the Swedish Cancer Register (SCR) between 2001 and 2018, were identified. In a matched cohort design, for each case five randomly selected mastocytosis-free comparators matched on age, sex, and county
Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries. We identified 71 patients with AdvSM (aggressive SM [ASM], SM with an associated hematologic neoplasm [SM-AHN, e.g., acute myeloid leukemia, SM-AML], mast cell leukemia [MCL]) in two national registries (DRST/GREM) who received an allogeneic hematopoietic cell
Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee. Systemic Mastocytosis (SM) is a multifaceted clinically heterogeneous disease. Advanced SM (AdvSM) comprises three entities: aggressive SM (ASM), mast cell leukaemia (MCL) and SM with an associated hematologic of these targeted agents is 'curative' in all patients and the duration of responses varies. The median overall survival, depending on the WHO subtype and scoring result, is approximately 1 to 4 years. Although the European Competence Network on Mastocytosis (ECNM) and American Initiative in Mast Cell Diseases (AIM) consensus groups recommend allogeneic haematopoietic cell transplantation (allo-HCT) in drug
NCCN Guidelines® Insights: Systemic Mastocytosis, Version 3.2024. Mastocytosis is a heterogeneous group of disorders comprising cutaneous mastocytosis, systemic mastocytosis, and mast cell sarcoma. It is associated with a variety of symptoms related to the release of mast cell mediators and mast cell tissue infiltration. Referral to specialized centers with expertise in the management of mastocytosis and multidisciplinary collaboration with subspecialists (eg, allergists for the management of anaphylaxis and drug hypersensitivities, anesthesiologists for invasive procedures or surgery, high-risk obstetrician for pregnancy) is recommended. The NCCN Guidelines for Systemic Mastocytosis provide evidence- and consensus-based recommendations for the diagnosis and comprehensive care of patients
T-cell immune profile in blood of systemic mastocytosis: Association with disease features. Systemic mastocytosis (SM) is a heterogeneous disease characterized by an expansion of KIT-mutated mast cells (MC). KIT-mutated MC display activated features and release MC mediators that might act on the tumour microenvironment and other immune cells. Here, we investigated the distribution of lymphocyte subsets in blood of patients with distinct subtypes of SM and determined its association with other disease features. We studied the distribution of TCD4 and TCD4 cytotoxic cells and their subsets, as well as total NK- and B cells, in blood of 115 SM patients-38 bone marrow mastocytosis (BMM), 67 indolent SM (ISM), 10 aggressive SM (ASM)- and 83 age-matched healthy donors (HD), using spectral flow
Interactions Between Eosinophils and IL5Rα+ Mast Cells in Non-Advanced Systemic Mastocytosis. Bidirectional interactions between eosinophils and mast cells (MCs) have been reported in various allergic diseases. Bone marrow (BM) eosinophilia, and to a lesser extent blood eosinophilia, is common in systemic mastocytosis (SM), but its significance remains unknown. To describe blood and BM
Altered B-cell, plasma cell and antibody immune profiles in blood of systemic mastocytosis. Systemic mastocytosis (SM) is a heterogeneous disease characterised by an expansion of KIT-mutated constitutively activated mast cells (MC) which release MC mediators that might act on the tumour microenvironment including other immune cells. Here we investigated the blood distribution of B-cell, plasma cell (PC) and antibody-isotype compartments in SM. We used spectral flow cytometry and the EuroFlow Immunomonitoring panel and Lymphocyte Screening Tube to quantify B-cells, PC and their subsets in blood of 108 SM patients - 35 bone marrow mastocytosis (BMM), 64 indolent SM (ISM), 9 aggressive SM (ASM)- vs 117 age-matched healthy donors (HD) and paired bone marrow (BM) samples of 31 SM vs 17 controls