{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2024,7,25]],"date-time":"2024-07-25T11:58:20Z","timestamp":1721908700257},"reference-count":49,"publisher":"Springer Science and Business Media LLC","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2021,9,29]]},"DOI":"10.7759\/cureus.18385","type":"journal-article","created":{"date-parts":[[2021,9,30]],"date-time":"2021-09-30T03:47:24Z","timestamp":1632973644000},"source":"Crossref","is-referenced-by-count":0,"title":["The Role of Avapritinib for the Treatment of Systemic Mastocytosis"],"prefix":"10.7759","author":[{"given":"Vikram","family":"Sumbly","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ian","family":"Landry","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Saba","family":"Iqbal","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zamaraq","family":"Bhatti","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mohsen S","family":"Alshamam","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Salman","family":"Ashfaq","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vincent","family":"Rizzo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","reference":[{"key":"ref1","doi-asserted-by":"publisher","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 P","year":"2007","unstructured":"Valent P, Akin C, Escribano L, et al.. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest. 2007, 37:435-53. 10.1111\/j.1365-2362.2007.01807.x","journal-title":"Eur J Clin Invest"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-03-643544","article-title":"The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia","volume":"127","author":"Arber DA","year":"2016","unstructured":"Arber DA, Orazi A, Hasserjian R, et al.. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016, 127:2391-405. 10.1182\/blood-2016-03-643544","journal-title":"Blood"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-99-120192","article-title":"Systemic mastocytosis in United States: a population based study","volume":"132","author":"Bista A","year":"2018","unstructured":"Bista A, Uprety D, Vallatharasu Y, et al.. Systemic mastocytosis in United States: a population based study. Blood. 2018, 132:1830. 10.1182\/blood-2018-99-120192","journal-title":"Blood"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25371","article-title":"Systemic mastocytosis in adults: 2019 update on diagnosis, risk stratification and management","volume":"94","author":"Pardanani A","year":"2019","unstructured":"Pardanani A. Systemic mastocytosis in adults: 2019 update on diagnosis, risk stratification and management. Am J Hematol. 2019, 94:363-77. 10.1002\/ajh.25371","journal-title":"Am J Hematol"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1016\/B978-0-12-809843-1.00014-0","article-title":"Mastocytosis","author":"Naeim F","year":"2018","unstructured":"Naeim F, Nagesh Rao P, Song SX, Phan RT. Mastocytosis. Atlas of Hematopathology (Second Edition). Naeim F, Nagesh Rao P, Song SX, Phan RT (ed): Academic Press, Cambridge; 2018. 245-59. 10.1016\/B978-0-12-809843-1.00014-0"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2016.146563","article-title":"Advanced systemic mastocytosis: from molecular and genetic progress to clinical practice","volume":"101","author":"Ustun C","year":"2016","unstructured":"Ustun C, Arock M, Kluin-Nelemans HC, et al.. Advanced systemic mastocytosis: from molecular and genetic progress to clinical practice. Haematologica. 2016, 101:1133-43. 10.3324\/haematol.2016.146563","journal-title":"Haematologica"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.1016\/s0145-2126(01)00038-8","article-title":"Diagnostic criteria and classification of mastocytosis: a consensus proposal","volume":"25","author":"Valent P","year":"2001","unstructured":"Valent P, Horny HP, Escribano L, et al.. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res. 2001, 25:603-25. 10.1016\/s0145-2126(01)00038-8","journal-title":"Leuk Res"},{"key":"ref8","doi-asserted-by":"publisher","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 A","year":"2009","unstructured":"Pardanani A, Lim KH, Lasho TL, Finke C, McClure RF, Li CY, Tefferi A. Prognostically relevant breakdown of 123 patients with systemic mastocytosis associated with other myeloid malignancies. Blood. 2009, 114:3769-72. 10.1182\/blood-2009-05-220145","journal-title":"Blood"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-02-205237","article-title":"Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors","volume":"113","author":"Lim KH","year":"2009","unstructured":"Lim KH, Tefferi A, Lasho TL, et al.. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood. 2009, 113:5727-36. 10.1182\/blood-2009-02-205237","journal-title":"Blood"},{"key":"ref10","doi-asserted-by":"crossref","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 J","year":"2019","unstructured":"L\u00fcbke J, Naumann N, Kluger S, et al.. Inhibitory effects of midostaurin and avapritinib on myeloid progenitors derived from patients with KIT D816V positive advanced systemic mastocytosis. Leukemia. 2019, 33:1195-1205.","journal-title":"Leukemia"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(20)30269-2","article-title":"Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial","volume":"21","author":"Heinrich MC","year":"2020","unstructured":"Heinrich MC, Jones R, von Mehren M, et al.. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020, 21:935-946. 10.1016\/S1470-2045(20)30269-2","journal-title":"Lancet Oncol"},{"key":"ref12","doi-asserted-by":"publisher","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 M","year":"2015","unstructured":"Arock M, Sotlar K, Akin C, et al.. KIT mutation analysis in mast cell neoplasms: recommendations of the European Competence Network on Mastocytosis. Leukemia. 2015, 29:1223-32. 10.1038\/leu.2015.24","journal-title":"Leukemia"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.7150\/thno.51872","article-title":"Cytogenetic and molecular aberrations and worse outcome for male patients in systemic mastocytosis","volume":"11","author":"Kluin-Nelemans HC","year":"2021","unstructured":"Kluin-Nelemans HC, Jawhar M, Reiter A, et al.. Cytogenetic and molecular aberrations and worse outcome for male patients in systemic mastocytosis. Theranostics. 2021, 11:292-303. 10.7150\/thno.51872","journal-title":"Theranostics"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22094900","article-title":"New insights into the pathogenesis of systemic mastocytosis","volume":"22","author":"Li Z","year":"2021","unstructured":"Li Z. New insights into the pathogenesis of systemic mastocytosis. Int J Mol Sci. 2021, 22:4900. 10.3390\/ijms22094900","journal-title":"Int J Mol Sci"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1046\/j.1523-1747.1998.00414.x","article-title":"Identification of activating c-kit mutations in adult-, but not in childhood-onset indolent mastocytosis: a possible explanation for divergent clinical behavior","volume":"111","author":"B\u00fcttner C","year":"1998","unstructured":"B\u00fcttner C, Henz BM, Welker P, Sepp NT, Grabbe J. Identification of activating c-kit mutations in adult-, but not in childhood-onset indolent mastocytosis: a possible explanation for divergent clinical behavior. J Invest Dermatol. 1998, 111:1227-31. 10.1046\/j.1523-1747.1998.00414.x","journal-title":"J Invest Dermatol"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9440(10)63870-9","article-title":"One-step detection of c-kit point mutations using peptide nucleic acid-mediated polymerase chain reaction clamping and hybridization probes","volume":"162","author":"Sotlar K","year":"2003","unstructured":"Sotlar K, Escribano L, Landt O, et al.. One-step detection of c-kit point mutations using peptide nucleic acid-mediated polymerase chain reaction clamping and hybridization probes. Am J Pathol. 2003, 162:737-46. 10.1016\/S0002-9440(10)63870-9","journal-title":"Am J Pathol"},{"key":"ref17","doi-asserted-by":"publisher","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 AC","year":"2006","unstructured":"Garcia-Montero AC, Jara-Acevedo M, Teodosio C, et al.. 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. Blood. 2006, 108:2366-72. 10.1182\/blood-2006-04-015545","journal-title":"Blood"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.4.1609","article-title":"Activating and dominant inactivating c-KIT catalytic domain mutations in distinct clinical forms of human mastocytosis","volume":"96","author":"Longley BJ Jr","year":"1999","unstructured":"Longley BJ Jr, Metcalfe DD, Tharp M, et al.. Activating and dominant inactivating c-KIT catalytic domain mutations in distinct clinical forms of human mastocytosis. Proc Natl Acad Sci U S A. 1999, 96:1609-14. 10.1073\/pnas.96.4.1609","journal-title":"Proc Natl Acad Sci U S A"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2141.1997.d01-2042.x","article-title":"A new c-kit mutation in a case of aggressive mast cell disease","volume":"96","author":"Pignon JM","year":"1997","unstructured":"Pignon JM, Giraudier S, Duquesnoy P, et al.. A new c-kit mutation in a case of aggressive mast cell disease. Br J Haematol. 1997, 96:374-6. 10.1046\/j.1365-2141.1997.d01-2042.x","journal-title":"Br J Haematol"},{"key":"ref20","doi-asserted-by":"publisher","DOI":"10.1016\/s0145-2126(01)00028-5","article-title":"Classes of c-KIT activating mutations: proposed mechanisms of action and implications for disease classification and therapy","volume":"25","author":"Longley BJ","year":"2001","unstructured":"Longley BJ, Reguera MJ, Ma Y. Classes of c-KIT activating mutations: proposed mechanisms of action and implications for disease classification and therapy. Leuk Res. 2001, 25:571-6. 10.1016\/s0145-2126(01)00028-5","journal-title":"Leuk Res"},{"key":"ref21","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.01153-06","article-title":"Neoplasia driven by mutant c-KIT is mediated by intracellular, not plasma membrane, receptor signaling","volume":"27","author":"Xiang Z","year":"2007","unstructured":"Xiang Z, Kreisel F, Cain J, Colson A, Tomasson MH. Neoplasia driven by mutant c-KIT is mediated by intracellular, not plasma membrane, receptor signaling. Mol Cell Biol. 2007, 27:267-82. 10.1128\/MCB.01153-06","journal-title":"Mol Cell Biol"},{"key":"ref22","doi-asserted-by":"publisher","DOI":"10.1084\/jem.20050807","article-title":"Mastocytosis in mice expressing human Kit receptor with the activating Asp816Val mutation","volume":"202","author":"Zappulla JP","year":"2005","unstructured":"Zappulla JP, Dubreuil P, Desbois S, et al.. Mastocytosis in mice expressing human Kit receptor with the activating Asp816Val mutation. J Exp Med. 2005, 202:1635-41. 10.1084\/jem.20050807","journal-title":"J Exp Med"},{"key":"ref23","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-04-496448","article-title":"Comprehensive mutational profiling in advanced systemic mastocytosis","volume":"122","author":"Schwaab J","year":"2013","unstructured":"Schwaab J, Schnittger S, Sotlar K, et al.. Comprehensive mutational profiling in advanced systemic mastocytosis. Blood. 2013, 122:2460-6. 10.1182\/blood-2013-04-496448","journal-title":"Blood"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0096209","article-title":"Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis","volume":"9","author":"De Vita S","year":"2014","unstructured":"De Vita S, Schneider RK, Garcia M, et al.. Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. PLoS One. 2014, 9:e96209. 10.1371\/journal.pone.0096209","journal-title":"PLoS One"},{"key":"ref25","doi-asserted-by":"publisher","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 M","year":"2015","unstructured":"Jawhar M, Schwaab J, Schnittger S, et al.. Molecular profiling of myeloid progenitor cells in multi-mutated advanced systemic mastocytosis identifies KIT D816V as a distinct and late event. Leukemia. 2015, 29:1115-22. 10.1038\/leu.2015.4","journal-title":"Leukemia"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2013.095133","article-title":"SRSF2-p95 hotspot mutation is highly associated with advanced forms of mastocytosis and mutations in epigenetic regulator genes","volume":"99","author":"Hanssens K","year":"2014","unstructured":"Hanssens K, Brenet F, Agopian J, et al.. SRSF2-p95 hotspot mutation is highly associated with advanced forms of mastocytosis and mutations in epigenetic regulator genes. Haematologica. 2014, 99:830-5. 10.3324\/haematol.2013.095133","journal-title":"Haematologica"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.1038\/leu.2009.37","article-title":"Frequent TET2 mutations in systemic mastocytosis: clinical, KITD816V and FIP1L1-PDGFRA correlates","volume":"23","author":"Tefferi A","year":"2009","unstructured":"Tefferi A, Levine RL, Lim KH, et al.. Frequent TET2 mutations in systemic mastocytosis: clinical, KITD816V and FIP1L1-PDGFRA correlates. Leukemia. 2009, 23:900-4. 10.1038\/leu.2009.37","journal-title":"Leukemia"},{"key":"ref28","doi-asserted-by":"publisher","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 E","year":"2012","unstructured":"Soucie E, Hanssens K, Mercher T, et al.. In aggressive forms of mastocytosis, TET2 loss cooperates with c-KITD816V to transform mast cells. Blood. 2012, 120:4846-9. 10.1182\/blood-2011-12-397588","journal-title":"Blood"},{"key":"ref29","doi-asserted-by":"publisher","DOI":"10.1016\/j.ccr.2011.06.025","article-title":"The role of TET2 in hematologic neoplasms","volume":"20","author":"Holmfeldt L","year":"2011","unstructured":"Holmfeldt L, Mullighan CG. The role of TET2 in hematologic neoplasms. Cancer Cell. 2011, 20:1-2. 10.1016\/j.ccr.2011.06.025","journal-title":"Cancer Cell"},{"key":"ref30","doi-asserted-by":"publisher","DOI":"10.1016\/j.ccell.2015.04.006","article-title":"SRSF2 mutations contribute to myelodysplasia by mutant-specific effects on exon recognition","volume":"27","author":"Kim E","year":"2015","unstructured":"Kim E, Ilagan JO, Liang Y, et al.. SRSF2 mutations contribute to myelodysplasia by mutant-specific effects on exon recognition. Cancer Cell. 2015, 27:617-30. 10.1016\/j.ccell.2015.04.006","journal-title":"Cancer Cell"},{"key":"ref31","doi-asserted-by":"publisher","DOI":"10.1038\/nature09586","article-title":"Impaired hydroxylation of 5-methylcytosine in myeloid cancers with mutant TET2","volume":"468","author":"Ko M","year":"2010","unstructured":"Ko M, Huang Y, Jankowska AM, et al.. Impaired hydroxylation of 5-methylcytosine in myeloid cancers with mutant TET2. Nature. 2010, 468:839-43. 10.1038\/nature09586","journal-title":"Nature"},{"key":"ref32","doi-asserted-by":"publisher","DOI":"10.1007\/BF02982057","article-title":"RUNX1\/AML1: a central player in hematopoiesis","volume":"74","author":"Okuda T","year":"2001","unstructured":"Okuda T, Nishimura M, Nakao M, Fujita Y. RUNX1\/AML1: a central player in hematopoiesis. Int J Hematol. 2001, 74:252-7. 10.1007\/BF02982057","journal-title":"Int J Hematol"},{"key":"ref33","doi-asserted-by":"publisher","DOI":"10.14348\/molcells.2019.0252","article-title":"The clinical, molecular, and mechanistic basis of RUNX1 mutations identified in hematological malignancies","volume":"43","author":"Yokota A","year":"2020","unstructured":"Yokota A, Huo L, Lan F, Wu J, Huang G. The clinical, molecular, and mechanistic basis of RUNX1 mutations identified in hematological malignancies. Mol Cells. 2020, 43:145-52. 10.14348\/molcells.2019.0252","journal-title":"Mol Cells"},{"key":"ref34","doi-asserted-by":"publisher","DOI":"10.1084\/jem.20171151","article-title":"Expression of mutant Asxl1 perturbs hematopoiesis and promotes susceptibility to leukemic transformation","volume":"215","author":"Nagase R","year":"2018","unstructured":"Nagase R, Inoue D, Pastore A, et al.. Expression of mutant Asxl1 perturbs hematopoiesis and promotes susceptibility to leukemic transformation. J Exp Med. 2018, 215:1729-47. 10.1084\/jem.20171151","journal-title":"J Exp Med"},{"key":"ref35","doi-asserted-by":"publisher","DOI":"10.1586\/14789450.2015.1033409","article-title":"Functional proteomics of the epigenetic regulators ASXL1, ASXL2 and ASXL3: a convergence of proteomics and epigenetics for translational medicine","volume":"12","author":"Katoh M","year":"2015","unstructured":"Katoh M. Functional proteomics of the epigenetic regulators ASXL1, ASXL2 and ASXL3: a convergence of proteomics and epigenetics for translational medicine. Expert Rev Proteomics. 2015, 12:317-28. 10.1586\/14789450.2015.1033409","journal-title":"Expert Rev Proteomics"},{"key":"ref36","doi-asserted-by":"publisher","DOI":"10.1002\/cyto.b.10072","article-title":"Immunophenotypic analysis of mast cells in mastocytosis: when and how to do it. Proposals of the Spanish Network on Mastocytosis (REMA)","volume":"58","author":"Escribano L","year":"2004","unstructured":"Escribano L, Diaz-Agustin B, L\u00f3pez A, et al.. Immunophenotypic analysis of mast cells in mastocytosis: when and how to do it. Proposals of the Spanish Network on Mastocytosis (REMA). Cytometry B Clin Cytom. 2004, 58:1-8. 10.1002\/cyto.b.10072","journal-title":"Cytometry B Clin Cytom"},{"key":"ref37","doi-asserted-by":"publisher","DOI":"10.1097\/01.pas.0000138181.89743.7b","article-title":"CD25 indicates the neoplastic phenotype of mast cells: a novel immunohistochemical marker for the diagnosis of systemic mastocytosis (SM) in routinely processed bone marrow biopsy specimens","volume":"28","author":"Sotlar K","year":"2004","unstructured":"Sotlar K, Horny HP, Simonitsch I, et al.. CD25 indicates the neoplastic phenotype of mast cells: a novel immunohistochemical marker for the diagnosis of systemic mastocytosis (SM) in routinely processed bone marrow biopsy specimens. Am J Surg Pathol. 2004, 28:1319-25. 10.1097\/01.pas.0000138181.89743.7b","journal-title":"Am J Surg Pathol"},{"key":"ref38","doi-asserted-by":"publisher","DOI":"10.1111\/his.12221","article-title":"CD30 expression by bone marrow mast cells from different diagnostic variants of systemic mastocytosis","volume":"63","author":"Morgado JM","year":"2013","unstructured":"Morgado JM, Perbellini O, Johnson RC, et al.. CD30 expression by bone marrow mast cells from different diagnostic variants of systemic mastocytosis. Histopathology. 2013, 63:780-7. 10.1111\/his.12221","journal-title":"Histopathology"},{"key":"ref39","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2015.1.98","article-title":"Diagnosis and management of mastocytosis: an emerging challenge in applied hematology","volume":"2015","author":"Valent P","year":"2015","unstructured":"Valent P. Diagnosis and management of mastocytosis: an emerging challenge in applied hematology. Hematology Am Soc Hematol Educ Program. 2015, 2015:98-105. 10.1182\/asheducation-2015.1.98","journal-title":"Hematology Am Soc Hematol Educ Program"},{"key":"ref40","doi-asserted-by":"publisher","DOI":"10.1111\/all.12436","article-title":"Proposed diagnostic algorithm for patients with suspected mastocytosis: a proposal of the European Competence Network on Mastocytosis","volume":"69","author":"Valent P","year":"2014","unstructured":"Valent P, Escribano L, Broesby-Olsen S, et al.. Proposed diagnostic algorithm for patients with suspected mastocytosis: a proposal of the European Competence Network on Mastocytosis. Allergy. 2014, 69:1267-74. 10.1111\/all.12436","journal-title":"Allergy"},{"key":"ref41","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-020-01275-2","article-title":"Avapritinib: first approval","volume":"80","author":"Dhillon S","year":"2020","unstructured":"Dhillon S. Avapritinib: first approval. Drugs. 2020, 80:433-9. 10.1007\/s40265-020-01275-2","journal-title":"Drugs"},{"key":"ref42","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1513098","article-title":"Efficacy and safety of midostaurin in advanced systemic mastocytosis","volume":"374","author":"Gotlib J","year":"2016","unstructured":"Gotlib J, Kluin-Nelemans HC, George TI, et al.. Efficacy and safety of midostaurin in advanced systemic mastocytosis. N Engl J Med. 2016, 374:2530-41. 10.1056\/NEJMoa1513098","journal-title":"N Engl J Med"},{"key":"ref43","doi-asserted-by":"publisher","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 S","year":"2008","unstructured":"Verstovsek S, Tefferi A, Cortes J, et al.. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res. 2008, 14:3906-15. 10.1158\/1078-0432.CCR-08-0366","journal-title":"Clin Cancer Res"},{"key":"ref44","doi-asserted-by":"publisher","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 A","year":"2015","unstructured":"Hochhaus A, Baccarani M, Giles FJ, et al.. Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study. J Cancer Res Clin Oncol. 2015, 141:2047-60. 10.1007\/s00432-015-1988-0","journal-title":"J Cancer Res Clin Oncol"},{"key":"ref45","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2014.55.2018","article-title":"Hematopoietic stem-cell transplantation for advanced systemic mastocytosis","volume":"32","author":"Ustun C","year":"2014","unstructured":"Ustun C, Reiter A, Scott BL, et al.. Hematopoietic stem-cell transplantation for advanced systemic mastocytosis. J Clin Oncol. 2014, 32:3264-74. 10.1200\/JCO.2014.55.2018","journal-title":"J Clin Oncol"},{"key":"ref46","doi-asserted-by":"publisher","DOI":"10.1158\/0008-5472.CAN-18-3139","article-title":"ATP-competitive inhibitors midostaurin and avapritinib have distinct resistance profiles in exon 17-mutant KIT","volume":"79","author":"Apsel Winger B","year":"2019","unstructured":"Apsel Winger B, Cortopassi WA, Garrido Ruiz D, et al.. ATP-competitive inhibitors midostaurin and avapritinib have distinct resistance profiles in exon 17-mutant KIT. Cancer Res. 2019, 79:4283-92. 10.1158\/0008-5472.CAN-18-3139","journal-title":"Cancer Res"},{"key":"ref47","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-18-1858","article-title":"Robust activity of avapritinib, potent and highly selective inhibitor of mutated KIT, in patient-derived xenograft models of gastrointestinal stromal tumors","volume":"25","author":"Gebreyohannes YK","year":"2019","unstructured":"Gebreyohannes YK, Wozniak A, Zhai ME, et al.. Robust activity of avapritinib, potent and highly selective inhibitor of mutated KIT, in patient-derived xenograft models of gastrointestinal stromal tumors. Clin Cancer Res. 2019, 25:609-18. 10.1158\/1078-0432.CCR-18-1858","journal-title":"Clin Cancer Res"},{"key":"ref48","doi-asserted-by":"publisher","DOI":"10.18632\/oncotarget.4213","article-title":"Mastocytosis: a mutated KIT receptor induced myeloproliferative disorder","volume":"6","author":"Chatterjee A","year":"2015","unstructured":"Chatterjee A, Ghosh J, Kapur R. Mastocytosis: a mutated KIT receptor induced myeloproliferative disorder. Oncotarget. 2015, 6:18250-64. 10.18632\/oncotarget.4213","journal-title":"Oncotarget"},{"key":"ref49","doi-asserted-by":"publisher","DOI":"10.1021\/acs.molpharmaceut.9b00274","article-title":"Avapritinib: a selective inhibitor of KIT and PDGFR\u03b1 that reverses ABCB1 and ABCG2-mediated multidrug resistance in cancer cell lines","volume":"16","author":"Wu CP","year":"2019","unstructured":"Wu CP, Lusvarghi S, Wang JC, Hsiao SH, Huang YH, Hung TH, Ambudkar SV. Avapritinib: a selective inhibitor of KIT and PDGFR\u03b1 that reverses ABCB1 and ABCG2-mediated multidrug resistance in cancer cell lines. Mol Pharm. 2019, 16:3040-52. 10.1021\/acs.molpharmaceut.9b00274","journal-title":"Mol Pharm"}],"container-title":["Cureus"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cureus.com\/articles\/71828-the-role-of-avapritinib-for-the-treatment-of-systemic-mastocytosis","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,2,9]],"date-time":"2024-02-09T19:34:27Z","timestamp":1707507267000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cureus.com\/articles\/71828-the-role-of-avapritinib-for-the-treatment-of-systemic-mastocytosis"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,9,29]]},"references-count":49,"URL":"https:\/\/doi.org\/10.7759\/cureus.18385","relation":{},"ISSN":["2168-8184"],"issn-type":[{"value":"2168-8184","type":"print"}],"subject":[],"published":{"date-parts":[[2021,9,29]]}}}