{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T22:54:18Z","timestamp":1792450458936,"version":"4.5.0"},"reference-count":85,"publisher":"American Society of Hematology","issue":"16","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,4,16]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Systemic mastocytosis (SM) has greatly benefited from the broad application of precision medicine techniques to hematolymphoid neoplasms. Sensitive detection of the recurrent KIT D816V mutation and use of next-generation sequencing (NGS) panels to profile the genetic landscape of SM variants have been critical adjuncts to the diagnosis and subclassification of SM, and development of clinical-molecular prognostic scoring systems. Multilineage KIT involvement and multimutated clones are characteristic of advanced SM (advSM), especially SM with an associated hematologic neoplasm (AHN). A major challenge is how to integrate conventional markers of mast cell disease burden (percentage of bone marrow mast cell infiltration and serum tryptase levels) with molecular data (serial monitoring of both KIT D816V variant allele frequency and NGS panels) to lend more diagnostic and prognostic clarity to the heterogeneous clinical presentations and natural histories of advSM. The approval of the multikinase\/KIT inhibitor midostaurin has validated the paradigm of KIT inhibition in advSM, and the efficacy and safety of second-generation agents, such as the switch-control inhibitor ripretinib (DCC-2618) and the D816V-selective inhibitor avapritinib (BLU-285) are being further defined in ongoing clinical trials. Looking forward, perhaps the most fruitful marriage of the advances in molecular genetics and treatment will be the design of adaptive basket trials that combine histopathology and genetic profiling to individualize treatment approaches for patients with diverse AHNs and relapsed\/refractory SM.<\/jats:p>","DOI":"10.1182\/blood.2019000932","type":"journal-article","created":{"date-parts":[[2020,2,27]],"date-time":"2020-02-27T23:20:08Z","timestamp":1582845608000},"page":"1365-1376","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":131,"title":["New developments in diagnosis, prognostication, and treatment of advanced systemic mastocytosis"],"prefix":"10.1182","volume":"135","author":[{"given":"Andreas","family":"Reiter","sequence":"first","affiliation":[{"name":"Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5478-7847","authenticated-orcid":false,"given":"Tracy I.","family":"George","sequence":"first","affiliation":[{"name":"Department of Pathology, University of Utah, Salt Lake City, UT;"},{"name":"ARUP Laboratories, Salt Lake City, UT; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"Gotlib","sequence":"first","affiliation":[{"name":"Division of Hematology, Stanford University School of Medicine\/Stanford Cancer Institute, Stanford, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"23","key":"2020041611581930300_B1","doi-asserted-by":"crossref","first-page":"10560","DOI":"10.1073\/pnas.92.23.10560","article-title":"Identification of a point mutation in the catalytic domain of the protooncogene c-kit in peripheral blood mononuclear cells of patients who have mastocytosis with an associated hematologic disorder","volume":"92","author":"Nagata","year":"1995","journal-title":"Proc Natl Acad Sci USA"},{"issue":"6","key":"2020041611581930300_B2","doi-asserted-by":"crossref","first-page":"1223","DOI":"10.1038\/leu.2015.24","article-title":"KIT mutation analysis in mast cell neoplasms: recommendations of the European Competence Network on Mastocytosis","volume":"29","author":"Arock","year":"2015","journal-title":"Leukemia"},{"issue":"5","key":"2020041611581930300_B3","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1002\/ajh.23672","article-title":"Sensitive KIT D816V mutation analysis of blood as a diagnostic test in mastocytosis","volume":"89","author":"Kristensen","year":"2014","journal-title":"Am J Hematol"},{"issue":"6","key":"2020041611581930300_B4","doi-asserted-by":"crossref","first-page":"810","DOI":"10.1111\/all.12409","article-title":"The KIT D816V allele burden predicts survival in patients with mastocytosis and correlates with the WHO type of the disease","volume":"69","author":"Hoermann","year":"2014","journal-title":"Allergy"},{"issue":"3","key":"2020041611581930300_B5","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1373\/clinchem.2017.277897","article-title":"Digital PCR: a sensitive and precise method for KIT D816V quantification in mastocytosis","volume":"64","author":"Greiner","year":"2018","journal-title":"Clin Chem"},{"issue":"2","key":"2020041611581930300_B6","doi-asserted-by":"crossref","first-page":"366","DOI":"10.3324\/haematol.2019.217950","article-title":"Molecular quantification of tissue disease burden is a new biomarker and independent predictor of survival in mastocytosis","volume":"105","author":"Greiner","year":"2020","journal-title":"Haematologica"},{"issue":"1","key":"2020041611581930300_B7","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1007\/s00277-013-1964-1","article-title":"The KIT D816V expressed allele burden for diagnosis and disease monitoring of systemic mastocytosis","volume":"93","author":"Erben","year":"2014","journal-title":"Ann Hematol"},{"issue":"2","key":"2020041611581930300_B8","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1182\/blood-2017-01-764423","article-title":"Response and progression on midostaurin in advanced systemic mastocytosis: KIT D816V and other molecular markers","volume":"130","author":"Jawhar","year":"2017","journal-title":"Blood"},{"key":"2020041611581930300_B9","first-page":"61","volume-title":"World Health Organization (WHO) Classification of Tumours: Pathology & Genetics\u2014Tumours of Haematopoietic and Lymphoid Tissues","author":"Horny","year":"2016"},{"issue":"6","key":"2020041611581930300_B10","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1158\/0008-5472.CAN-16-2234","article-title":"Advances in the classification and treatment of mastocytosis: current status and outlook toward the future","volume":"77","author":"Valent","year":"2017","journal-title":"Cancer Res"},{"issue":"5","key":"2020041611581930300_B11","doi-asserted-by":"crossref","first-page":"1124","DOI":"10.1038\/s41375-018-0346-z","article-title":"KIT D816 mutated\/CBF-negative acute myeloid leukemia: a poor-risk subtype associated with systemic mastocytosis","volume":"33","author":"Jawhar","year":"2019","journal-title":"Leukemia"},{"issue":"4","key":"2020041611581930300_B12","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1136\/mp.53.4.188","article-title":"Detection of c-kit mutation Asp 816 to Val in microdissected bone marrow infiltrates in a case of systemic mastocytosis associated with chronic myelomonocytic leukaemia","volume":"53","author":"Sotlar","year":"2000","journal-title":"Mol Pathol"},{"issue":"5","key":"2020041611581930300_B13","doi-asserted-by":"crossref","first-page":"586","DOI":"10.1002\/path.2677","article-title":"Variable presence of KITD816V in clonal haematological non-mast cell lineage diseases associated with systemic mastocytosis (SM-AHNMD)","volume":"220","author":"Sotlar","year":"2010","journal-title":"J Pathol"},{"issue":"7","key":"2020041611581930300_B14","doi-asserted-by":"crossref","first-page":"2366","DOI":"10.1182\/blood-2006-04-015545","article-title":"KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients","volume":"108","author":"Garcia-Montero","year":"2006","journal-title":"Blood"},{"issue":"3","key":"2020041611581930300_B15","doi-asserted-by":"crossref","first-page":"514","DOI":"10.1016\/j.jaci.2009.05.003","article-title":"Prognosis in adult indolent systemic mastocytosis: a long-term study of the Spanish Network on Mastocytosis in a series of 145 patients","volume":"124","author":"Escribano","year":"2009","journal-title":"J Allergy Clin Immunol"},{"issue":"1","key":"2020041611581930300_B16","doi-asserted-by":"crossref","first-page":"58","DOI":"10.2353\/jmoldx.2008.070061","article-title":"Systemic mastocytosis associated with chronic idiopathic myelofibrosis: a distinct subtype of systemic mastocytosis associated with a [corrected] clonal hematological non-mast [corrected] cell lineage disorder carrying the activating point mutations KITD816V and JAK2V617F [published correction appears in J Mol Diagn. 2009;10(3):276]","volume":"10","author":"Sotlar","year":"2008","journal-title":"J Mol Diagn"},{"issue":"14","key":"2020041611581930300_B17","doi-asserted-by":"crossref","first-page":"2460","DOI":"10.1182\/blood-2013-04-496448","article-title":"Comprehensive mutational profiling in advanced systemic mastocytosis","volume":"122","author":"Schwaab","year":"2013","journal-title":"Blood"},{"issue":"5","key":"2020041611581930300_B18","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1182\/blood.2018886507","article-title":"Frequency and prognostic impact of KIT and other genetic variants in indolent systemic mastocytosis","volume":"134","author":"Mu\u00f1oz-Gonz\u00e1lez","year":"2019","journal-title":"Blood"},{"issue":"5","key":"2020041611581930300_B19","doi-asserted-by":"crossref","first-page":"1115","DOI":"10.1038\/leu.2015.4","article-title":"Molecular profiling of myeloid progenitor cells in multi-mutated advanced systemic mastocytosis identifies KIT D816V as a distinct and late event","volume":"29","author":"Jawhar","year":"2015","journal-title":"Leukemia"},{"issue":"8","key":"2020041611581930300_B20","doi-asserted-by":"crossref","first-page":"e43090","DOI":"10.1371\/journal.pone.0043090","article-title":"Single nucleotide polymorphism array lesions, TET2, DNMT3A, ASXL1 and CBL mutations are present in systemic mastocytosis","volume":"7","author":"Traina","year":"2012","journal-title":"PLoS One"},{"issue":"24","key":"2020041611581930300_B21","doi-asserted-by":"crossref","first-page":"4846","DOI":"10.1182\/blood-2011-12-397588","article-title":"In aggressive forms of mastocytosis, TET2 loss cooperates with c-KITD816V to transform mast cells","volume":"120","author":"Soucie","year":"2012","journal-title":"Blood"},{"issue":"1","key":"2020041611581930300_B22","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1038\/leu.2015.284","article-title":"Additional mutations in SRSF2, ASXL1 and\/or RUNX1 identify a high-risk group of patients with KIT D816V(+) advanced systemic mastocytosis","volume":"30","author":"Jawhar","year":"2016","journal-title":"Leukemia"},{"issue":"3","key":"2020041611581930300_B23","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1111\/bjh.13865","article-title":"ASXL1 and CBL mutations are independently predictive of inferior survival in advanced systemic mastocytosis","volume":"175","author":"Pardanani","year":"2016","journal-title":"Br J Haematol"},{"issue":"21","key":"2020041611581930300_B24","doi-asserted-by":"crossref","first-page":"2814","DOI":"10.1182\/bloodadvances.2018020628","article-title":"Impact of somatic and germline mutations on the outcome of systemic mastocytosis","volume":"2","author":"Mu\u00f1oz-Gonz\u00e1lez","year":"2018","journal-title":"Blood Adv"},{"issue":"5","key":"2020041611581930300_B25","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1002\/gcc.22526","article-title":"Incidence and prognostic impact of cytogenetic aberrations in patients with systemic mastocytosis","volume":"57","author":"Naumann","year":"2018","journal-title":"Genes Chromosomes Cancer"},{"issue":"12","key":"2020041611581930300_B26","doi-asserted-by":"crossref","first-page":"1461","DOI":"10.1002\/ajh.25265","article-title":"Cytogenetic abnormalities in systemic mastocytosis: WHO subcategory-specific incidence and prognostic impact among 348 informative cases","volume":"93","author":"Shah","year":"2018","journal-title":"Am J Hematol"},{"issue":"18","key":"2020041611581930300_B27","doi-asserted-by":"crossref","first-page":"3769","DOI":"10.1182\/blood-2009-05-220145","article-title":"Prognostically relevant breakdown of 123 patients with systemic mastocytosis associated with other myeloid malignancies","volume":"114","author":"Pardanani","year":"2009","journal-title":"Blood"},{"issue":"3","key":"2020041611581930300_B28","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1002\/ajh.25371","article-title":"Systemic mastocytosis in adults: 2019 update on diagnosis, risk stratification and management","volume":"94","author":"Pardanani","year":"2019","journal-title":"Am J Hematol"},{"issue":"1","key":"2020041611581930300_B29","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1182\/asheducation-2018.1.127","article-title":"The new tool \u201cKIT\u201d in advanced systemic mastocytosis","volume":"2018","author":"Shomali","year":"2018","journal-title":"Hematology Am Soc Hematol Educ Program"},{"issue":"5","key":"2020041611581930300_B30","doi-asserted-by":"crossref","first-page":"683","DOI":"10.1586\/17474086.2014.955008","article-title":"The serum tryptase test: an emerging robust biomarker in clinical hematology","volume":"7","author":"Valent","year":"2014","journal-title":"Expert Rev Hematol"},{"issue":"12","key":"2020041611581930300_B31","doi-asserted-by":"crossref","first-page":"1564","DOI":"10.1038\/ng.3696","article-title":"Elevated basal serum tryptase identifies a multisystem disorder associated with increased TPSAB1 copy number","volume":"48","author":"Lyons","year":"2016","journal-title":"Nat Genet"},{"issue":"1","key":"2020041611581930300_B32","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1159\/000501079","article-title":"Why the 20% + 2 tryptase formula is a diagnostic gold standard for severe systemic mast cell activation and mast cell activation syndrome","volume":"180","author":"Valent","year":"2019","journal-title":"Int Arch Allergy Immunol"},{"issue":"5","key":"2020041611581930300_B33","doi-asserted-by":"crossref","first-page":"392","DOI":"10.1111\/eci.12607","article-title":"Impact of centralized evaluation of bone marrow histology in systemic mastocytosis","volume":"46","author":"Jawhar","year":"2016","journal-title":"Eur J Clin Invest"},{"issue":"4","key":"2020041611581930300_B34","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1309\/AJCP1Q0YSXEAHNKK","article-title":"Hidden mastocytosis in acute myeloid leukemia with t(8;21)(q22;q22)","volume":"140","author":"Johnson","year":"2013","journal-title":"Am J Clin Pathol"},{"issue":"11","key":"2020041611581930300_B35","doi-asserted-by":"crossref","first-page":"2673","DOI":"10.1038\/s41375-019-0460-6","article-title":"Identification of a leukemia-initiating stem cell in human mast cell leukemia","volume":"33","author":"Eisenwort","year":"2019","journal-title":"Leukemia"},{"issue":"8","key":"2020041611581930300_B36","doi-asserted-by":"crossref","first-page":"1285","DOI":"10.1182\/blood-2012-07-442400","article-title":"Mast cell leukemia","volume":"121","author":"Georgin-Lavialle","year":"2013","journal-title":"Blood"},{"issue":"1","key":"2020041611581930300_B37","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.leukres.2014.09.010","article-title":"Chronic mast cell leukemia: a novel leukemia-variant with distinct morphological and clinical features","volume":"39","author":"Valent","year":"2015","journal-title":"Leuk Res"},{"issue":"40","key":"2020041611581930300_B38","doi-asserted-by":"crossref","first-page":"68950","DOI":"10.18632\/oncotarget.10711","article-title":"Imatinib in systemic mastocytosis: a phase IV clinical trial in patients lacking exon 17 KIT mutations and review of the literature","volume":"8","author":"\u00c1lvarez-Twose","year":"2016","journal-title":"Oncotarget"},{"issue":"6","key":"2020041611581930300_B39","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.3324\/haematol.2017.163964","article-title":"The clinical and molecular diversity of mast cell leukemia with or without associated hematologic neoplasm","volume":"102","author":"Jawhar","year":"2017","journal-title":"Haematologica"},{"issue":"7","key":"2020041611581930300_B40","doi-asserted-by":"crossref","first-page":"603","DOI":"10.1016\/S0145-2126(01)00038-8","article-title":"Diagnostic criteria and classification of mastocytosis: a consensus proposal","volume":"25","author":"Valent","year":"2001","journal-title":"Leuk Res"},{"key":"2020041611581930300_B41","first-page":"291","volume-title":"World Health Organization (WHO) Classification of Tumours: Pathology & Genetics\u2014Tumours of Haematopoietic and Lymphoid Tissues","author":"Valent","year":"2001"},{"key":"2020041611581930300_B42","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1016\/j.ebiom.2019.03.089","article-title":"Single-cell analysis reveals the KIT D816V mutation in haematopoietic stem and progenitor cells in systemic mastocytosis","volume":"43","author":"Grootens","year":"2019","journal-title":"EBioMedicine"},{"issue":"12","key":"2020041611581930300_B43","doi-asserted-by":"crossref","first-page":"2342","DOI":"10.1038\/leu.2016.190","article-title":"Splenomegaly, elevated alkaline phosphatase and mutations in the SRSF2\/ASXL1\/RUNX1 gene panel are strong adverse prognostic markers in patients with systemic mastocytosis","volume":"30","author":"Jawhar","year":"2016","journal-title":"Leukemia"},{"issue":"21","key":"2020041611581930300_B44","doi-asserted-by":"crossref","first-page":"2964","DOI":"10.1182\/bloodadvances.2018026245","article-title":"Mayo alliance prognostic system for mastocytosis: clinical and hybrid clinical-molecular models","volume":"2","author":"Pardanani","year":"2018","journal-title":"Blood Adv"},{"issue":"3","key":"2020041611581930300_B45","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1038\/s41408-019-0189-5","article-title":"World Health Organization class-independent risk categorization in mastocytosis","volume":"9","author":"Pardanani","year":"2019","journal-title":"Blood Cancer J"},{"issue":"31","key":"2020041611581930300_B46","doi-asserted-by":"crossref","first-page":"2846","DOI":"10.1200\/JCO.19.00640","article-title":"MARS: mutation-adjusted risk score for advanced systemic mastocytosis","volume":"37","author":"Jawhar","year":"2019","journal-title":"J Clin Oncol"},{"issue":"12","key":"2020041611581930300_B47","doi-asserted-by":"crossref","first-page":"e638","DOI":"10.1016\/S2352-3026(19)30166-8","article-title":"International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study","volume":"6","author":"Sperr","year":"2019","journal-title":"Lancet Haematol"},{"issue":"8","key":"2020041611581930300_B48","doi-asserted-by":"crossref","first-page":"5466","DOI":"10.4049\/jimmunol.180.8.5466","article-title":"Unique effects of KIT D816V in BaF3 cells: induction of cluster formation, histamine synthesis, and early mast cell differentiation antigens","volume":"180","author":"Mayerhofer","year":"2008","journal-title":"J Immunol"},{"issue":"12","key":"2020041611581930300_B49","doi-asserted-by":"crossref","first-page":"1635","DOI":"10.1084\/jem.20050807","article-title":"Mastocytosis in mice expressing human Kit receptor with the activating Asp816Val mutation","volume":"202","author":"Zappulla","year":"2005","journal-title":"J Exp Med"},{"issue":"2","key":"2020041611581930300_B50","doi-asserted-by":"crossref","first-page":"721","DOI":"10.1182\/blood-2004-12-4617","article-title":"Activation mutations of human c-KIT resistant to imatinib mesylate are sensitive to the tyrosine kinase inhibitor PKC412","volume":"106","author":"Growney","year":"2005","journal-title":"Blood"},{"issue":"9","key":"2020041611581930300_B51","doi-asserted-by":"crossref","first-page":"e7258","DOI":"10.1371\/journal.pone.0007258","article-title":"Masitinib (AB1010), a potent and selective tyrosine kinase inhibitor targeting KIT","volume":"4","author":"Dubreuil","year":"2009","journal-title":"PLoS One"},{"issue":"11","key":"2020041611581930300_B52","doi-asserted-by":"crossref","first-page":"2047","DOI":"10.1007\/s00432-015-1988-0","article-title":"Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study","volume":"141","author":"Hochhaus","year":"2015","journal-title":"J Cancer Res Clin Oncol"},{"issue":"12","key":"2020041611581930300_B53","doi-asserted-by":"crossref","first-page":"3906","DOI":"10.1158\/1078-0432.CCR-08-0366","article-title":"Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis","volume":"14","author":"Verstovsek","year":"2008","journal-title":"Clin Cancer Res"},{"issue":"8","key":"2020041611581930300_B54","doi-asserted-by":"crossref","first-page":"3222","DOI":"10.1182\/blood-2003-11-3816","article-title":"A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib","volume":"103","author":"Akin","year":"2004","journal-title":"Blood"},{"issue":"26","key":"2020041611581930300_B55","doi-asserted-by":"crossref","first-page":"2530","DOI":"10.1056\/NEJMoa1513098","article-title":"Efficacy and safety of midostaurin in advanced systemic mastocytosis","volume":"374","author":"Gotlib","year":"2016","journal-title":"N Engl J Med"},{"issue":"2","key":"2020041611581930300_B56","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1038\/leu.2017.234","article-title":"Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial","volume":"32","author":"DeAngelo","year":"2018","journal-title":"Leukemia"},{"key":"2020041611581930300_B57","author":"Radia"},{"issue":"5","key":"2020041611581930300_B58","doi-asserted-by":"crossref","first-page":"799","DOI":"10.3324\/haematol.2017.179895","article-title":"The KIT and PDGFRA switch-control inhibitor DCC-2618 blocks growth and survival of multiple neoplastic cell types in advanced mastocytosis","volume":"103","author":"Schneeweiss","year":"2018","journal-title":"Haematologica"},{"issue":"6","key":"2020041611581930300_B59","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1111\/j.1365-2362.2007.01807.x","article-title":"Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria","volume":"37","author":"Valent","year":"2007","journal-title":"Eur J Clin Invest"},{"issue":"13","key":"2020041611581930300_B60","doi-asserted-by":"crossref","first-page":"2393","DOI":"10.1182\/blood-2012-09-458521","article-title":"International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis","volume":"121","author":"Gotlib","year":"2013","journal-title":"Blood"},{"issue":"12","key":"2020041611581930300_B61","doi-asserted-by":"crossref","first-page":"1500","DOI":"10.6004\/jnccn.2018.0088","article-title":"Systemic mastocytosis, version 2.2019, NCCN clinical practice guidelines in oncology","volume":"16","author":"Gotlib","year":"2018","journal-title":"J Natl Compr Canc Netw"},{"issue":"23","key":"2020041611581930300_B62","doi-asserted-by":"crossref","first-page":"5727","DOI":"10.1182\/blood-2009-02-205237","article-title":"Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors","volume":"113","author":"Lim","year":"2009","journal-title":"Blood"},{"issue":"10","key":"2020041611581930300_B63","doi-asserted-by":"crossref","first-page":"1766","DOI":"10.1200\/JCO.2009.25.1066","article-title":"Effect of complete remission and responses less than complete remission on survival in acute myeloid leukemia: a combined Eastern Cooperative Oncology Group, Southwest Oncology Group, and M. D. Anderson Cancer Center Study","volume":"28","author":"Walter","year":"2010","journal-title":"J Clin Oncol"},{"issue":"414","key":"2020041611581930300_B64","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aao1690","article-title":"A precision therapy against cancers driven by KIT\/PDGFRA mutations","volume":"9","author":"Evans","year":"2017","journal-title":"Sci Transl Med"},{"issue":"16","key":"2020041611581930300_B65","doi-asserted-by":"crossref","first-page":"4283","DOI":"10.1158\/0008-5472.CAN-18-3139","article-title":"ATP-competitive inhibitors midostaurin and avapritinib have resistance profiles in exon 17-mutant KIT","volume":"79","author":"Apsel Winger","year":"2019","journal-title":"Cancer Res"},{"issue":"6","key":"2020041611581930300_B66","doi-asserted-by":"crossref","first-page":"790","DOI":"10.1182\/blood-2015-03-633404","article-title":"Correlation of mutation profile and response in patients with myelofibrosis treated with ruxolitinib","volume":"126","author":"Patel","year":"2015","journal-title":"Blood"},{"issue":"5","key":"2020041611581930300_B67","doi-asserted-by":"crossref","first-page":"1195","DOI":"10.1038\/s41375-019-0450-8","article-title":"Inhibitory effects of midostaurin and avapritinib on myeloid progenitors derived from patients with KIT D816V positive advanced systemic mastocytosis","volume":"33","author":"L\u00fcbke","year":"2019","journal-title":"Leukemia"},{"issue":"8","key":"2020041611581930300_B68","doi-asserted-by":"crossref","first-page":"1050","DOI":"10.1158\/2159-8290.CD-18-1453","article-title":"Clonal selection with RAS pathway activation mediates secondary clinical resistance to selective FLT inhibition in acute myeloid leukemia","volume":"9","author":"McMahon","year":"2019","journal-title":"Cancer Discov"},{"key":"2020041611581930300_B69","doi-asserted-by":"crossref","unstructured":"Miles LA , BowmanRL, MerlinskyTR, et al. Single cell DNA sequencing identifies combinatorial mutation patterns and clonal architecture in myeloid malignancies [abstract]. Blood.2019;134(suppl 1). Abstract 913.","DOI":"10.1182\/blood-2019-128763"},{"issue":"8","key":"2020041611581930300_B70","doi-asserted-by":"crossref","first-page":"1009","DOI":"10.1182\/blood-2014-12-614743","article-title":"Long-term efficacy and safety of cladribine (2-CdA) in adult patients with mastocytosis","volume":"126","author":"Barete","year":"2015","journal-title":"Blood"},{"issue":"19","key":"2020041611581930300_B71","first-page":"1885","article-title":"Mast cells, mastocytosis, and related disorders","volume":"373","author":"Theoharides","year":"2015","journal-title":"N Engl J Med"},{"issue":"4","key":"2020041611581930300_B72","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1007\/s11899-015-0280-3","article-title":"Tyrosine kinase inhibitors and therapeutic antibodies in advanced eosinophilic disorders and systemic mastocytosis","volume":"10","author":"Gotlib","year":"2015","journal-title":"Curr Hematol Malig Rep"},{"issue":"15","key":"2020041611581930300_B73","doi-asserted-by":"crossref","first-page":"2264","DOI":"10.1182\/bloodadvances.2019000152","article-title":"A phase 2 study of brentuximab vedotin in patients with CD30-positive advanced systemic mastocytosis","volume":"3","author":"Gotlib","year":"2019","journal-title":"Blood Adv"},{"issue":"3","key":"2020041611581930300_B74","doi-asserted-by":"crossref","first-page":"E552","DOI":"10.3390\/ijms20030552","article-title":"Bone marrow mast cell antibody-targetable cell surface protein expression profiles in systemic mastocytosis","volume":"20","author":"Dasilva-Freire","year":"2019","journal-title":"Int J Mol Sci"},{"issue":"7","key":"2020041611581930300_B75","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.1038\/leu.2015.16","article-title":"Aberrant expression of CD123 (interleukin-3 receptor-\u03b1) on neoplastic mast cells","volume":"29","author":"Pardanani","year":"2015","journal-title":"Leukemia"},{"issue":"8","key":"2020041611581930300_B76","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.1038\/leu.2016.30","article-title":"Complete response to gemtuzumab ozogamicin in a patient with refractory mast cell leukemia","volume":"30","author":"\u00c1lvarez-Twose","year":"2016","journal-title":"Leukemia"},{"issue":"1","key":"2020041611581930300_B77","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1189\/jlb.1112609","article-title":"The pan-Bcl-2 blocker obatoclax promotes the expression of Puma, Noxa, and Bim mRNA and induces apoptosis in neoplastic mast cells","volume":"95","author":"Peter","year":"2014","journal-title":"J Leukoc Biol"},{"issue":"2","key":"2020041611581930300_B78","doi-asserted-by":"crossref","first-page":"324","DOI":"10.1111\/bjh.13951","article-title":"Beneficial effects of JAK inhibitor therapy in systemic mastocytosis","volume":"176","author":"Dowse","year":"2017","journal-title":"Br J Haematol"},{"issue":"7728","key":"2020041611581930300_B79","doi-asserted-by":"crossref","first-page":"526","DOI":"10.1038\/s41586-018-0623-z","article-title":"Functional genomic landscape of acute myeloid leukaemia","volume":"562","author":"Tyner","year":"2018","journal-title":"Nature"},{"issue":"29","key":"2020041611581930300_B80","doi-asserted-by":"crossref","first-page":"3264","DOI":"10.1200\/JCO.2014.55.2018","article-title":"Hematopoietic stem-cell transplantation for advanced systemic mastocytosis","volume":"32","author":"Ustun","year":"2014","journal-title":"J Clin Oncol"},{"issue":"8","key":"2020041611581930300_B81","doi-asserted-by":"crossref","first-page":"1348","DOI":"10.1016\/j.bbmt.2016.04.018","article-title":"Consensus opinion on allogeneic hematopoietic cell transplantation in advanced systemic mastocytosis","volume":"22","author":"Ustun","year":"2016","journal-title":"Biol Blood Marrow Transplant"},{"issue":"1","key":"2020041611581930300_B82","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1038\/s41467-018-08201-x","article-title":"Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation","volume":"10","author":"Czechowicz","year":"2019","journal-title":"Nat Commun"},{"issue":"19","key":"2020041611581930300_B83","doi-asserted-by":"crossref","first-page":"2069","DOI":"10.1182\/blood-2018-06-858159","article-title":"Anti-CD117 antibody depletes normal and myelodysplastic syndrome human hematopoietic stem cells in xenografted mice","volume":"133","author":"Pang","year":"2019","journal-title":"Blood"},{"issue":"1","key":"2020041611581930300_B84","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1016\/j.jaip.2018.09.024","article-title":"The data registry of the European Competence Network on Mastocytosis (ECNM): set up, projects, and perspectives","volume":"7","author":"Valent","year":"2019","journal-title":"J Allergy Clin Immunol Pract"},{"key":"2020041611581930300_B85","unstructured":"Mu\u00f1oz-Gonz\u00e1lez JI , OrfaoA. Validation of the MARS, IPSM, Mayo and REMA prognostic scores for systemic mastocytosis. Presented at the 17th Annual Meeting of the European Competence Network on Mastocytosis. 5 October 2019. Salzburg, Austria."}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/135\/16\/1365\/1724162\/bloodbld2019000932c.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/135\/16\/1365\/1724162\/bloodbld2019000932c.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,27]],"date-time":"2023-09-27T19:18:07Z","timestamp":1695842287000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/135\/16\/1365\/452574\/New-developments-in-diagnosis-prognostication-and"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,4,16]]},"references-count":85,"journal-issue":{"issue":"16","published-print":{"date-parts":[[2020,4,16]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2019000932","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,4,16]]}}}