Avapritinib versus placebo in indolent systemic mastocytosis
Open accessAbstract
BACKGROUND: Indolent systemic mastocytosis (ISM) is a clonal mast-cell disease driven by the KIT D816V mutation. We assessed the efficacy and safety of avapritinib versus placebo, both with best supportive care, in patients with ISM. METHODS: We randomized patients with moderate to severe ISM (total symptom score [TSS] of ≥28; scores range from 0 to 110, with higher numbers indicating more severe symptoms) two to one to avapritinib 25 mg once daily (n=141) or placebo (n=71). The primary end point was mean change in TSS based on the 14-day average of patient-reported severity of 11 symptoms. Secondary end points included reductions in serum tryptase and blood KIT D816V variant allele fraction (≥50%), reductions in TSS (≥50% and ≥30%), reduction in bone marrow mast cells (≥50%), and quality of life measures. RESULTS: From baseline to week 24, avapritinib-treated patients had a decrease of 15.6 points (95% CI, −18.6 to −12.6) in TSS compared to a decrease of 9.2 points (−13.1 to −5.2) in the placebo group; P<0.003. From baseline to Week 24, 76/141 patients (54%; 45% to 62%) in the avapritinib group compared to 0/71 patients in the placebo group achieved a ≥50% reduction in serum tryptase level; P<0.001. Edema and increases in alkaline phosphatase were more common with avapritinib than placebo; there were few treatment discontinuations because of adverse events. CONCLUSIONS: In this trial, avapritinib was superior to placebo in reducing uncontrolled symptoms and mast-cell burden in patients with ISM. The long-term safety and efficacy of this approach for patients with ISM remain the focus of the ongoing trial. (Funded by Blueprint Medicines Corporation; ClinicalTrials.gov number, NCT03731260.)
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Tyrosine kinase inhibitors targeting mutated KIT D816V such as midostaurin and avapritinib reduce bone marrow mast cells, skin mast cells, serum tryptase, and MCA symptoms.
“Tyrosine kinase inhibitors addressing mutated KIT on codon D816V such as midostaurin and avapritinib have been shown to reduce bone marrow MCs, skin MCs, serum tryptase, and symptoms of MCA.28”
From Mast cell activation syndrome: Current understanding and research needs (2024) · cites this paper as [28]
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WARE, J. E., KOSINSKI, M., & KELLER, S. D. (1996) · Medical Care · doi:10.1097/00005650-199603000-00003 · Cited by 17,392 · see citing works
EuroQol: a new facility for the measurement of health-related quality of life
(1990) · Health Policy · doi:10.1016/0168-8510(90)90421-9 · Cited by 15,736 · see citing works
Arber, D. A., Orazi, A., Hasserjian, R. P., Borowitz, M. J., Calvo, K. R., Kvasnicka, H.-M., Wang, S. A., Bagg, A., Barbui, T., Branford, S., et al. (2022) · Blood · doi:10.1182/blood.2022015850 · Cited by 2,945 · see citing works
Self-citationMastocytosis: 2016 updated WHO classification and novel emerging treatment concepts
Valent, P., Akin, C., & Metcalfe, D. D. (2016) · Blood · doi:10.1182/blood-2016-09-731893 · Cited by 680 · see citing works
Self-citationHartmann, K., Escribano, L., Grattan, C. E. H., Brockow, K., Carter, M. C., Álvarez‐Twöse, I., Matito, A., Broesby‐Olsen, S., Siebenhaar, F., Lange, M., et al. (2015) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2015.08.034 · Cited by 395 · see citing works
Self-citationEscribano, L., Álvarez‐Twöse, I., Sánchez‐Muñoz, L., García‐Montero, A. C., Núñez, R., Almeida, J., Jara‐Acevedo, M., Teodósio, C., García‐Cosío, M., Bellas, C., et al. (2009) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2009.05.003 · Cited by 290 · see citing works
Epidemiology of systemic mastocytosis in Denmark
Cohen, S. S., Skovbo, S., Vestergaard, H., Kristensen, T. K., Møller, M. B., Bindslev‐Jensen, C., Fryzek, J. P., & Broesby‐Olsen, S. (2014) · British Journal of Haematology · doi:10.1111/bjh.12916 · Cited by 235 · see citing works
Self-citationKristensen, T., Vestergaard, H., & Møller, M. B. (2011) · The Journal of Molecular Diagnostics · doi:10.1016/j.jmoldx.2010.10.004 · Cited by 208 · see citing works
Mesa, R. A., Gotlib, J. R., Gupta, V. K., Catalano, J. V., Deininger, M. W., Shields, A. L., Miller, C. B., Silver, R. T., Talpaz, M., Winton, E. F., et al. (2013) · Journal of Clinical Oncology · doi:10.1200/jco.2012.44.4489 · Cited by 190 · see citing works
Self-citationSystemic mastocytosis in adults: 2021 Update on diagnosis, risk stratification and management
Pardanani, A. D. (2021) · American Journal of Hematology · doi:10.1002/ajh.26118 · Cited by 181 · see citing works
Hafliðadóttir, S. H., Juhl, C. B., Nielsen, S. M., Henriksen, M., Harris, I. A., Bliddal, H., & Christensen, R. (2021) · Trials · doi:10.1186/s13063-021-05454-8 · Cited by 181 · see citing works
The placebo effect: advances from different methodological approaches
Meissner, K., Bingel, U., Colloca, L., Wager, T. D., Watson, A., & Flaten, M. A. (2011) · The Journal of Neuroscience · doi:10.1523/jneurosci.4099-11.2011 · Cited by 178 · see citing works
Case-Control Cohort Study of Patients' Perceptions of Disability in Mastocytosis
Hermine, O., Lortholary, O., Leventhal, P. S., Catteau, A., Soppelsa, F., Baude, C., Cohen-Akenine, A., Palmérini, F., Hanssens, K., Yang, Y., et al. (2008) · PLoS ONE · doi:10.1371/journal.pone.0002266 · Cited by 170 · see citing works
Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial
DeAngelo, D. J., Radia, D. H., George, T. I., Robinson, W. A., Quiery, A. T., Drummond, M. W., Bose, P., Hexner, E. O., Winton, E. F., Horny, H., et al. (2021) · Nature Medicine · doi:10.1038/s41591-021-01538-9 · Cited by 160 · see citing works
Self-citationGotlib, J. R., Reiter, A., Radia, D. H., Deininger, M. W., George, T. I., Panse, J., Vannucchi, A. M., Platzbecker, U., Álvarez‐Twöse, I., Mital, A., et al. (2021) · Nature Medicine · doi:10.1038/s41591-021-01539-8 · Cited by 154 · see citing works
Self-citationNew developments in diagnosis, prognostication, and treatment of advanced systemic mastocytosis
Reiter, A., George, T. I., & Gotlib, J. R. (2020) · Blood · doi:10.1182/blood.2019000932 · Cited by 140 · see citing works
Self-citationJones, B. D. M., Razza, L. B., Weissman, C. R., Karbi, J., Vine, T., Mulsant, L. S., Brunoni, A. R., Husain, M. I., Mulsant, B. H., Blumberger, D. M., et al. (2021) · JAMA Network Open · doi:10.1001/jamanetworkopen.2021.25531 · Cited by 121 · see citing works
Pardanani, A. (2013) · Blood · doi:10.1182/blood-2013-01-453183 · Cited by 101 · see citing works
Development and validation of the mastocytosis quality of life questionnaire: MC-QoL
Siebenhaar, F., von Tschirnhaus, E., Hartmann, K., Rabenhorst, A., Staubach, P., Peveling‐Oberhag, A., Wagner, N., Martus, P., Carter, M. C., Metcalfe, D. D., et al. (2016) · Allergy · doi:10.1111/all.12842 · Cited by 76 · see citing works
Self-citationValent, P., Hartmann, K., Schwaab, J., Alvarez-Twose, I., Brockow, K., Bonadonna, P., Hermine, O., Niedoszytko, M., Carter, M. C., Hoermann, G., et al. (2022) · The Journal of Allergy and Clinical Immunology: In Practice · doi:10.1016/j.jaip.2022.03.007 · Cited by 72 · see citing works
Self-citationPieri, L., Bonadonna, P., Elena, C., Papayannidis, C., Grifoni, F. I., Rondoni, M., Girlanda, S., Mauro, M., Magliacane, D., Elli, E. M., et al. (2016) · American Journal of Hematology · doi:10.1002/ajh.24382 · Cited by 67 · see citing works
Self-citationPatient-reported disease-specific quality-of-life and symptom severity in systemic mastocytosis
van Anrooij, B., Kluin‐Nelemans, H. C., Safy‐Khan, M., Blok, B. M. J. F.-D., & Elberink, J. N. G. O. (2016) · Allergy · doi:10.1111/all.12920 · Cited by 59 · see citing works
Midostaurin in patients with indolent systemic mastocytosis: An open-label phase 2 trial
van Anrooij, B., Elberink, J. N. G. O., Span, L. F. R., de Monchy, J. G. R., Rosati, S., Mulder, A. B., & Kluin-Nelemans, J. C. (2018) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2018.06.003 · Cited by 58 · see citing works
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Jennings, S. V., Slee, V. M., Zack, R. M., Verstovsek, S., George, T. I., Shi, H., Lee, P., & Castells, M. C. (2018) · Immunology and Allergy Clinics of North America · doi:10.1016/j.iac.2018.04.006 · Cited by 53 · see citing works
Self-citationMastocytosis: update on pharmacotherapy and future directions
Cardet, J. C., Akin, C., & Lee, M. J. (2013) · Expert Opinion on Pharmacotherapy · doi:10.1517/14656566.2013.824424 · Cited by 49 · see citing works
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Akin, C., Arock, M., & Valent, P. (2022) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2022.04.020 · Cited by 44 · see citing works
Self-citationBroesby‐Olsen, S., Kristensen, T., Vestergaard, H., Brixen, K., Møller, M. B., & Bindslev‐Jensen, C. (2013) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2013.02.019 · Cited by 42 · see citing works
Self-citationMesa, R. A., Sullivan, E. M., Dubinski, D. G., Carroll, B., Slee, V. M., Jennings, S. V., Finnerty, C. C., Bohannon, L. S., Mathias, S. D., Lahue, B. J., et al. (2022) · Cancer · doi:10.1002/cncr.34420 · Cited by 29 · see citing works
Self-citationPyatilova, P., Akin, C., Alvarez-Twose, I., Arock, M., Bonadonna, P., Brockow, K., Butterfield, J. H., Broesby-Olsen, S., Carter, M. C., Castells, M., et al. (2022) · The Journal of Allergy and Clinical Immunology In Practice · doi:10.1016/j.jaip.2022.05.037 · Cited by 27 · see citing works
Self-citationHarrison, C. N., Schaap, N. P., Vannucchi, A. M., Kiladjian, J., Jourdan, É., Silver, R. T., Schouten, H. C., Passamonti, F., Zweegman, S., Talpaz, M., et al. (2021) · HemaSphere · doi:10.1097/hs9.0000000000000562 · Cited by 24 · see citing works
Estevinho, M. M., Afonso, J., Rosa, I., Lago, P. M., Trindade, E., Correia, L., Dias, C. C., Magro, F., & GEDII [Portuguese IBD Group] (2018) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjy100 · Cited by 23 · see citing works
Taylor, F. C., Akin, C., Lamoureux, R. E., Padilla, B., Green, T., Boral, A. L., Mazar, I., Mar, B. G., Shields, A. L., & Siebenhaar, F. (2021) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-021-02035-5 · Cited by 20 · see citing works
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Akin, C., Elberink, H. O., Gotlib, J. R., Sabato, V., Hartmann, K., Broesby‐Olsen, S., Castells, M., Deininger, M. W., Heaney, M. L., George, T. I., et al. (2020) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2019.12.062 · Cited by 17 · see citing works
Self-citationPadilla, B., Shields, A. L., Taylor, F. C., Li, X., Mcdonald, J., Green, T., Boral, A. L., Lin, H., Akin, C., Siebenhaar, F., et al. (2021) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-021-02037-3 · Cited by 15 · see citing works
Self-citationShields, A. L., Taylor, F. C., Lamoureux, R. E., Padilla, B., Severson, K., Green, T., & Mar, B. G. (2019) · Value in Health · doi:10.1016/j.jval.2019.09.2471 · Cited by 2 · see citing works
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Self-citationAkin, C., Sabato, V., Gotlib, J. R., Panse, J., Álvarez‐Twöse, I., Radia, D. H., Livideanu, C. B., Jurcic, J. G., Elberink, H. O., van Daele, P., et al. (2026) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2026.04.001 · Cited by 2 · see citing works
Self-citationDiagnosis and management of systemic mastocytosis in a community hematology setting
Dranitsaris, G., Neuhalfen, H., Peevyhouse, A., Powell, D., Miller, K., Green, T., & Graff, T. (2023) · Journal of Oncology Pharmacy Practice · doi:10.1177/10781552231221149 · Cited by 2 · see citing works
Hamilton, M. J., Greene, L. W., Madigan, L. M., Wang, S. A., Yi, C. A., Kuykendall, A. T., George, T. I., & Castells, M. (2024) · Frontiers in Allergy · doi:10.3389/falgy.2024.1401187 · Cited by 2 · see citing works
Self-citationMakeeva, A. (2025) · American Journal of Cancer Research · doi:10.62347/tytu4465 · Cited by 2 · see citing works
Hymenoptera anaphylaxis and mast cell activation disorders
González‐de‐Olano, D., & Álvarez‐Twöse, I. (2025) · Current Opinion in Allergy and Clinical Immunology · doi:10.1097/aci.0000000000001088 · Cited by 2 · see citing works
Successful venom immunotherapy with avapritinib in a patient with systemic mastocytosis
Depina, L., Karmarkar, S., Moeller, S., Lazarovich, M., de Magalhães, A. R., Castells, M., & Giannetti, M. P. (2025) · Annals of Allergy Asthma & Immunology · doi:10.1016/j.anai.2025.06.015 · Cited by 2 · see citing works
Self-citationMastocytosis in the age of precision medicine
Siebenhaar, F., Brehler, R., Christen, D., Hartmann, K., Altrichter, S., Joest, M., Kampe, K. A., Lang, C. C. V., Lippert, U., Mülleneisen, N. K., et al. (2025) · Allergo Journal International · doi:10.1007/s40629-025-00327-x · Cited by 1 · see citing works
Self-citationTreatment of indolent systemic mastocytosis with sarilumab is not supported in a randomized trial
Komarow, H. D., Wang, J., Eisch, R. A., Scott, L., Brittain, E., & Metcalfe, D. D. (2025) · Journal of Allergy and Clinical Immunology Global · doi:10.1016/j.jacig.2025.100498 · Cited by 1 · see citing works
Liu, C., Chu, J., Wan, Y., Wang, H., Yu, H., Zhang, K., Xie, Z., Tu, S., Wang, N., & Yang, L. J. (2026) · Frontiers in Oncology · doi:10.3389/fonc.2025.1717065 · Cited by 1 · see citing works
Chernak, B. J., & Rampal, R. K. (2026) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2025.2604560 · Cited by 1 · see citing works
Valent, P., Hartmann, K., Stefanzl, G., Filik, Y., Bauer, K., Degenfeld‐Schonburg, L., Gleixner, K. V., Siebenhaar, F., Niedoszytko, M., Sperr, W. R., et al. (2026) · Current Allergy and Asthma Reports · doi:10.1007/s11882-026-01257-6 · Cited by 1 · see citing works
Self-citationInvestigational Approaches in Treatment of Nonadvanced Mastocytosis
Pongdee, T. (2026) · Current Allergy and Asthma Reports · doi:10.1007/s11882-026-01263-8 · Cited by 1 · see citing works
Self-citationGiannetti, M. P. (2026) · Current Allergy and Asthma Reports · doi:10.1007/s11882-026-01268-3 · Cited by 1 · see citing works
Self-citationYue, Z., Wang, Y., Zeng, L., Jin, T., & Sheng, Y. (2024) · Clinical Pharmacology in Drug Development · doi:10.1002/cpdd.1398 · Cited by 1 · see citing works
Mastozytose im Zeitalter der Präzisionsmedizin
Siebenhaar, F., Brehler, R., Christen, D., Hartmann, K., Altrichter, S., Joest, M., Kampe, K. A., Lang, C. C. V., Lippert, U., Mülleneisen, N. K., et al. (2025) · Allergo Journal · doi:10.1007/s15007-025-6465-8 · Cited by 1 · see citing works
Self-citationKomarow, H. D., Nilsson, G. P., Okayama, Y., Mekori, Y. A., Olivera, A., Valent, P., Hartmann, K., Brockow, K., Cruse, G., Lyons, J. J., et al. (2025) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2025.06.029 · Cited by 1 · see citing works
Self-citationSelecting optimal therapy in systemic mastocytosis: current state and future directions
Ike, I., Samperio, V. M., & Dasanu, C. A. (2025) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2025.2503847 · Cited by 1 · see citing works
Targeted Therapy for the Predominant Form of Mastocytosis
Metcalfe, D. D. (2023) · NEJM Evidence · doi:10.1056/evide2300089
EXABS-115-MPN Current and Emerging Therapies for Systemic Mastocytosis
Bose, P. (2023) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/s2152-2650(23)00289-6
Self-citationDermatologische Klinik des Universitätsspitals Basel
Hartmann, K., Brenner, M., Brandt, O., Contassot, E., Cvijetic, I., Erni, B., Geiges, M. L., Izakovic, J., Kunz, M., Merkel, T., et al. (2023) · Aktuelle Dermatologie · doi:10.1055/a-2122-7078
Self-citationDifferentialdiagnose der Eosinophilie aus hämatologischer Sicht
Sockel, K., Reiter, A., Bornhäuser, M., & Schwaab, J. (2024) · Aktuelle Rheumatologie · doi:10.1055/a-2307-3824
Self-citationManaging Systemic Mastocytosis
Gotlib, J. R. (2024) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/s2152-2650(24)00330-6
Self-citationDegboé, Y. (2025) · Revue du Rhumatisme · doi:10.1016/j.rhum.2025.01.023
Indolent Systemic Mastocytosis: An Incidental Diagnosis
Khalid, A., Faiz, A., & Kaell, A. T. (2025) · Annals of Internal Medicine Clinical Cases · doi:10.7326/aimcc.2024.0118
Beyond the classical Philadelphia chromosome–negative myeloproliferative neoplasms
Patel, A. A., & Stein, B. L. (2025) · doi:10.1182/ashsap9.chapter38
Molhem, A. M., Aldamen, R., Masoud, A. T., Abuelsamen, Q. R., Alsaleeli, F. M., Almomani, O. M., Shawaqfeh, A. E., Marie, H. F. S., AlHomoud, R. A., Beitho, M. N. I., et al. (2025) · Archives of Dermatological Research · doi:10.1007/s00403-025-04430-5
Chronic Anaphylaxis With Indolent Systemic Mastocytosis: A Case Report
Worth, S., George, T. I., Shaheen, D., Roche, M. R., & Vachhani, P. J. (2025) · Case Reports in Hematology · doi:10.1155/crh/9562195
Self-citationDegboé, Y., Masson, M. H., & Livideanu, C. B. (2025) · Revue du Rhumatisme · doi:10.1016/j.rhum.2025.10.266
Refractory cutaneous symptoms in systemic mastocytosis
Gleason, L. K., Bhatti, S. A., Wallace, M., Lee, J. B., Alpdoğan, Ö., & Nikbakht, N. (2025) · JAAD Case Reports · doi:10.1016/j.jdcr.2025.09.012
Ieven, T., Govaerts, I., Frans, G., Vandebotermet, M., Tuyls, S., Wilmer, A. P., Schrijvers, R., Vandenberghe, P., & Breynaert, C. M. (2026) · Clinical Reviews in Allergy & Immunology · doi:10.1007/s12016-026-09137-7
Brockow, K., Franz, T. M., Saak, M., Lorenz, H., Plötz, J., Stark, L., Schäbitz, A., Aydin, G., Rupp, S., Untch, J., et al. (2026) · Allergo Journal International · doi:10.1007/s40629-026-00373-z
Depression in mastocytosis: A neglected dimension of a clonal mast cell disease
Heneberg, P. (2026) · Autoimmunity Reviews · doi:10.1016/j.autrev.2026.104100
Mastocytosis in clinical practice – current standards of care
Masiarz, A., Gawryś, U., Tochman, W., & Podhorecka, M. (2026) · Journal of Pre-Clinical and Clinical Research · doi:10.26444/jpccr/221743
Jankovski, L., Herman, R., Rakuša, M., Kopač, P., Kačar, M., Škerget, M., Janež, A., & Jensterle, M. (2026) · Biomolecules · doi:10.3390/biom16060821
Systemic Mastocytosis in Adults: 2026 Update on Diagnosis, Risk Stratification and Management
Pardanani, A. D. (2026) · American Journal of Hematology · doi:10.1002/ajh.70395
Small molecules and their importance as therapeutic options in daily practice
Tachdjian, R. (2026) · Annals of Allergy Asthma & Immunology · doi:10.1016/j.anai.2026.06.014
Mast cell activation disorders: mechanisms, comorbidities and look-alike
Theoharides, T. C., Kempuraj, D., & Maitland, A. L. (2026) · Expert Review of Clinical Immunology · doi:10.1080/1744666x.2026.2699311
Barète, S., & Dalle, S. (2026) · Elsevier eBooks · doi:10.1016/b978-2-294-77608-3.00062-4
Hu, M., He, J., Luo, Y., Alvarado, D., Miller, D. C., Scheffel, J., Frischbutter, S., Siebenhaar, F., & Pyatilova, P. (2026) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2026.08.019
Self-citationRamírez, G., Khoudari, A., Gonzalez, Y., & Davis-Yadley, A. H. (2026) · ACG Case Reports Journal · doi:10.14309/crj.0000000000002310
Hereditary alpha-tryptasemia: ten years on
(2026) · Frontiers research topics · doi:10.3389/978-2-8325-8910-6
Cătană, A., Porr, C., Mondoc, L.-M., Mocanu, L., Olteanu, A. L., Draghila, L., & Iancu, G. M. (2026) · Diagnostics · doi:10.3390/diagnostics16213499
- Authors
- Gotlib, J., Castells, M., Elberink, H. O., Siebenhaar, F., Hartmann, K., Broesby-Olsen, S., George, T. I., Panse, J., Alvarez-Twose, I., Radia, D. H., Tashi, T., Bulai Livideanu, C., Sabato, V., Heaney, M., Van Daele, P., Cerquozzi, S., Dybedal, I., Reiter, A., Pongdee, T., Barete, S., Ustun, C., Schwartz, L., Ward, B. R., Schafhausen, P., Vadas, P., Bose, P., DeAngelo, D. J., Rein, L., Vachhani, P., Triggiani, M., Bonadonna, P., Rafferty, M., Butt, N. M., Oh, S. T., Wortmann, F., Ungerstedt, J., Guilarte, M., Taparia, M., Kuykendall, A. T., Arana Yi, C., Ogbogu, P., Gaudy-Marqueste, C., Mattsson, M., Shomali, W., Giannetti, M. P., Bidollari, I., Lin, H.-M., Sulllivan, E., Mar, B., Scherber, R., Roche, M., Akin, C., & Maurer, M.
- Affiliations
- Stanford Medicine (US)
- Brigham and Women's Hospital (US)
- University Medical Center Groningen (NL)
- Humboldt-Universität zu Berlin (DE)
- University of Basel (CH)
- Department of Dermatology and Allergy Centre, Odense University Hospital, Odense, Denmark
- University of Utah (US)
- Universitätsklinikum Aachen (DE)
- Institute of Mastocytosis Studies of Castilla-La Mancha, Virgen del Valle Hospital, Toledo, Spain
- National Health Service (GB)
- Huntsman Cancer Institute (US)
- Department of Dermatology, Centre of Reference for Mastocytosis, Toulouse University Hospital, Toulouse, France
- University of Antwerp (BE)
- Columbia University Irving Medical Center (US)
- Department of Internal Medicine and Immunology, Erasmus Medical Center, Rotterdam, the Netherlands
- University of Calgary (CA)
- Oslo University Hospital (NO)
- Heidelberg University (DE)
- Mayo Clinic (US)
- Unit of Dermatology, Centre of Reference for Mastocytosis, Pitié-Salpêtrière Hospital, Assistance Publique–Hôpitaux de Paris, Sorbonne Université, Paris
- Department of Internal Medicine, Division of Hematology, Oncology and Cell Therapy, Section of Bone Marrow Transplantation and Cellular Therapy, Rush Medical College, Chicago
- Virginia Commonwealth University (US)
- University Medical Center Hamburg-Eppendorf (DE)
- St. Michael's Hospital (CA)
- The University of Texas MD Anderson Cancer Center (US)
- Dana-Farber Cancer Institute (US)
- Duke University (US)
- University of Alabama at Birmingham (US)
- University of Salerno (IT)
- Azienda Ospedaliera Universitaria Integrata Verona (IT)
- Beatson West of Scotland Cancer Centre (GB)
- Clatterbridge Cancer Centre NHS Foundation Trust (GB)
- Barnes-Jewish Hospital (US)
- University Hospital Schleswig-Holstein (DE)
- Karolinska University Hospital (SE)
- Vall d'Hebron Institut de Recerca (ES)
- University of Alberta (CA)
- Moffitt Cancer Center (US)
- Division of Hematology and Medical Oncology, Mayo Clinic, Phoenix, AZ
- Rainbow Babies & Children's Hospital (US)
- Service de Dermatologie et de cancérologie cutanée, Assistance Publique–Hopitaux de Marseille, Aix-Marseille Université, Marseille, France
- Uppsala University (SE)
- Blueprint Medicines (United States) (US)
- Blueprint Medicines Corporation, Cambridge, MA
- University of Michigan (US)
- ORCID
- Gotlib, Jason
- Castells, Mariana
- Elberink, Hanneke Oude
- Siebenhaar, Frank
- Hartmann, Karin
- George, Tracy I.
- Panse, Jens
- Radia, Deepti H.
- Tashi, Tsewang
- Sabato, Vito
- Heaney, Mark
- Cerquozzi, Sonia
- Dybedal, Ingunn
- Reiter, Andreas
- Pongdee, Thanai
- Schwartz, Lawrence
- Ward, Brant R.
- Vadas, Peter
- Bose, Prithviraj
- DeAngelo, Daniel J.
- Rein, Lindsay
- Vachhani, Pankit
- Triggiani, Massimo
- Bonadonna, Patrizia
- Oh, Stephen T.
- Wortmann, Friederike
- Ungerstedt, Johanna
- Guilarte, Mar
- Taparia, Minakshi
- Kuykendall, Andrew T.
- Mattsson, Mattias
- Shomali, William
- Giannetti, Matthew P.
- Mar, Brenton
- Scherber, Robyn
- Roche, Maria
- Akin, Cem
- Maurer, Marcus
- Venue
- NEJM Evidence
- Publisher
- Massachusetts Medical Society
- Published
- 2023-05-23
- Volume
- 2
- Issue
- 6
- Pages
- EVIDoa2200339-EVIDoa2200339
- ISSN
- 2766-5526 (ISSN-L)
- OpenAlex
- W4377694353
- Cited by
- 81
- References
- 36
- Citations by year
- 2026: 20 · 2025: 35 · 2024: 16 · 2023: 10
- FWCI
- 8.84
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- NEJM Evidence (publishedVersion)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (acceptedVersion, other-oa)
- University of Southern Denmark Research Portal (University of Southern Denmark) (acceptedVersion, other-oa)
- Topics
- Life Sciences › Immunology and Microbiology › Immunology › Mast cells and histamine
- Health Sciences › Medicine › Gastroenterology › Gastrointestinal Tumor Research and Treatment
- Health Sciences › Medicine › Rheumatology › Eosinophilic Disorders and Syndromes
- Keywords
- indolent systemic mastocytosis, avapritinib, KIT D816V mutation, tyrosine kinase inhibitors, total symptom score, serum tryptase, variant allele frequency, bone marrow mast cells, quality of life, edema, alkaline phosphatase, best supportive care
- MeSH terms
- Avapritinib, Humans, Pyrazoles, Pyrroles, Triazines, Mastocytosis, Systemic
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