{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,7]],"date-time":"2026-05-07T23:47:11Z","timestamp":1778197631403,"version":"3.51.4"},"reference-count":81,"publisher":"MDPI AG","issue":"20","license":[{"start":{"date-parts":[[2023,10,12]],"date-time":"2023-10-12T00:00:00Z","timestamp":1697068800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Advanced systemic mastocytosis (SM) is a heterogeneous group of myeloid neoplasms characterized by an uncontrolled expansion of mast cells (MC) in one or more internal organs, SM-induced tissue damage, and poor prognosis. Advanced SM can be categorized into aggressive SM (ASM), MC leukemia (MCL), and SM with an associated hematologic neoplasm (SM\u2013AHN). In a vast majority of all patients, neoplastic cells display a KIT mutation, mostly D816V and rarely other KIT variants. Additional mutations in other target genes, such as SRSF2, ASXL1, or RUNX1, may also be identified, especially when an AHN is present. During the past 10 years, improved treatment approaches have led to a better quality of life and survival in patients with advanced SM. However, despite the availability of novel potent inhibitors of KIT D816V, not all patients enter remission and others relapse, often with a multi-mutated and sometimes KIT D816V-negative disease exhibiting multi-drug resistance. For these patients, (poly)chemotherapy, antibody-based therapies, and allogeneic hematopoietic stem cell transplantation may be viable treatment alternatives. In this article, we discuss treatment options for patients with drug-resistant advanced SM, including novel KIT-targeting drugs, antibody-based drugs, and stem cell-eradicating therapies.<\/jats:p>","DOI":"10.3390\/ijms242015125","type":"journal-article","created":{"date-parts":[[2023,10,12]],"date-time":"2023-10-12T12:46:13Z","timestamp":1697114773000},"page":"15125","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Antibody-Based and Cell Therapies for Advanced Mastocytosis: Established and Novel Concepts"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0456-5095","authenticated-orcid":false,"given":"Peter","family":"Valent","sequence":"first","affiliation":[{"name":"Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, 1090 Vienna, Austria"},{"name":"Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cem","family":"Akin","sequence":"additional","affiliation":[{"name":"Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, MI 48106, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3990-4408","authenticated-orcid":false,"given":"Michel","family":"Arock","sequence":"additional","affiliation":[{"name":"Department of Hematological Biology, Piti\u00e9-Salp\u00eatri\u00e8re Hospital, Sorbonne University, 75013 Paris, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karoline V.","family":"Gleixner","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, 1090 Vienna, Austria"},{"name":"Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hildegard","family":"Greinix","sequence":"additional","affiliation":[{"name":"Division of Hematology, Medical University of Graz, 8010 Graz, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olivier","family":"Hermine","sequence":"additional","affiliation":[{"name":"Service d\u2019H\u00e9matologie, Imagine Institute Universit\u00e9 de Paris, INSERM U1163, Centre National de R\u00e9f\u00e9rence des Mastocytoses, H\u00f4pital Necker, Assistance Publique H\u00f4pitaux de Paris, 75015 Paris, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hans-Peter","family":"Horny","sequence":"additional","affiliation":[{"name":"Institute of Pathology, Ludwig-Maximilians University, 80539 Munich, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1304-4224","authenticated-orcid":false,"given":"Daniel","family":"Ivanov","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, 1090 Vienna, Austria"},{"name":"Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0007-7230","authenticated-orcid":false,"given":"Alberto","family":"Orfao","sequence":"additional","affiliation":[{"name":"Servicio Central de Citometria, Centro de Investigacion del Cancer (IBMCC; CSIC\/USAL) Instituto Biosanitario de Salamanca (IBSAL), CIBERONC and Department of Medicine, University of Salamanca, 37007 Salamanca, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Werner","family":"Rabitsch","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Stem Cell Transplantation Unit, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Reiter","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology, University Hospital Mannheim, 68135 Mannheim, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Axel","family":"Schulenburg","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Stem Cell Transplantation Unit, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karl","family":"Sotlar","sequence":"additional","affiliation":[{"name":"Institute of Pathology, University Hospital Salzburg, Paracelsus Medical University, 5020 Salzburg, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wolfgang R.","family":"Sperr","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, 1090 Vienna, Austria"},{"name":"Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, 1090 Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Celalettin","family":"Ustun","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Hematology, Oncology, and Cell Therapy, Coleman Foundation Blood and Marrow Transplant Center at Rush University Medical Center, Chicago, IL 60612, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,10,12]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1420","DOI":"10.1182\/blood-2016-09-731893","article-title":"Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts","volume":"129","author":"Valent","year":"2017","journal-title":"Blood"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1365","DOI":"10.1182\/blood.2019000932","article-title":"New developments in diagnosis, prognostication, and treatment of advanced systemic mastocytosis","volume":"135","author":"Reiter","year":"2020","journal-title":"Blood"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1146\/annurev-pathmechdis-031521-042618","article-title":"New Insights into the Pathogenesis of Mastocytosis: Emerging Concepts in Diagnosis and Therapy","volume":"18","author":"Valent","year":"2023","journal-title":"Annu. 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