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Journal Articles

Improved diagnostic screening and classification of clonal mast cell diseases by ultrasensitive KIT p.D816V detection

Paula Navarro-Navarro, María González-Tablas, Alba Pérez-Pons, Laura Sánchez-Muñoz, Ana Henriques, Iván Álvarez-Twose, Paula Nafria, David Araco-Sánchez, Cristina Morales-Cabeza, Horacio Caligaris, Lei Chen, David González-de-Olano, Óscar González-López, Luis Escribano, Andrés C. García-Montero, María Jara-Acevedo, Alberto Orfao

Blood (2025) 146 (22): 2696–2709.

https://doi.org/10.1182/blood.2025029507

Published: 2025

Includes: Supplemental data

Journal Articles

Improved overall survival in patients with advanced systemic mastocytosis treated with avapritinib versus real-world therapy based on mutation-adjusted risk score (MARS) stratification

Blood (2025) 146 (Supplement 1): 5595.

https://doi.org/10.1182/blood-2025-5595

Published: 2025

Journal Articles

Efficacy and safety results from the primary analysis of the pivotal summit trial: Bezuclastinib in adults with non-advanced systemic mastocytosis

Lindsay Rein, Nathan Boggs, Prithviraj Bose, Brian Modena, Vito Sabato, Karin Hartmann, Cem Akin, Tracy George, Cecilia Arana Yi, Hanneke Oude Elberink, Deepti Radia, Andreas Reiter, Miguel Piris-Villaespesa, Ingunn Dybedal, Jens Panse, Stephen Oh, Pankit Vachhani, Anthony Hunter, Mariana Castells, Cristina Bulai Livideanu, Paul van Daele, Arnold Kirshenbaum, Ivan Alvarez-Twose, Jennifer Vaughn, Minakshi Taparia, Sonia Cerquozzi, Andrzej Mital, Marek Hus, ALESSANDRA ROMANO, John Fahrenholz, Frederick Lansigan, Cristina Papayannidis, Helena Pomares, Michela Rondoni, Celalettin Ustun, Richard Herrscher, Michael Manning, Stéphane Barete, Mar Guilarte, Candido Rivera, Jonathan Bernstein, Peter Vadas, Chiara Elena, Derek McCulloch, Nina Orfali, Clodagh Keohane, Francesco Mannelli, Philippe Schafhausen, Amanda Pilla, Jenna Zhang, Lei Sun, Nisha Shah, Hina Jolin, Rachael Easton, Jessica Sachs, Frank Siebenhaar, Daniel Deangelo

Blood (2025) 146 (Supplement 1): 80.

https://doi.org/10.1182/blood-2025-80

Published: 2025

Journal Articles

Effect of avapritinib on skin lesions in patients with advanced systemic mastocytosis using a novel, artificial intelligence-based technology

Blood (2025) 146 (Supplement 1): 2030.

https://doi.org/10.1182/blood-2025-2030

Published: 2025

Journal Articles

Avapritinib achieves deep and durable symptom control with a well-tolerated safety profile in ism: Long-term outcomes from pioneer

Blood (2025) 146 (Supplement 1): 2024.

https://doi.org/10.1182/blood-2025-2024

Published: 2025

Journal Articles

Changes in long-term bone health in patients receiving avapritinib for the treatment of indolent systemic mastocytosis in the pioneer study

Mariana Castells, Vishnu Sundaresh, Jason Gotlib, Tsewang Tashi, Vito Sabato, Cristina Bulai Livideanu, Cem Akin, Deepti Radia, Frank Siebenhaar, Daniel Deangelo, Tracy George, Hui-Min Lin, Janet Hong, Ashley Doyle, Guang Yang, Rachel Erlich, Kate Newberry, Benjamin Lampson, Ivan Alvarez-Twose

Blood (2025) 146 (Supplement 1): 5582.

https://doi.org/10.1182/blood-2025-5582

Published: 2025

Journal Articles

An analysis of clonal dynamics in patients with indolent systemic mastocytosis treated with avapritinib in the pioneer study

Jens Panse, Tsewang Tashi, Jason Gotlib, Mariana Castells, Ivan Alvarez-Twose, Cem Akin, Cristina Bulai Livideanu, Vito Sabato, Juliana Schwaab, Andreas Reiter, Sigurd Broesby-Olsen, Frank Siebenhaar, Hui-Min Lin, Janet Hong, Benjamin Lampson, Guang Yang, Tracy George, Deepti Radia

Blood (2025) 146 (Supplement 1): 5573.

https://doi.org/10.1182/blood-2025-5573

Published: 2025

Journal Articles

Ultra-Sensitive KIT testing Uncovers Previously Undetected KIT mutations in Patients with Indolent Systemic Mastocytosis: Results from the Pioneer Trial

Deepti H. Radia, Tsewang Tashi, Ivan Alvarez-Twose, Patrizia Bonadonna, Hanneke Oude Elberink, Sonia Cerquozzi, Tracy I. George, Guang Yang, Mark Rosenzweig, Hui-Min Lin, Ben Lampson, Rachel L. Erlich, Jason Gotlib

Blood (2024) 144 (Supplement 1): 3164.

https://doi.org/10.1182/blood-2024-207798

Published: 2024

Journal Articles

Overall Survival and Duration of Treatment in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib Versus Midostaurin or Best Available Therapy in a Real-World Setting

Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Luebke, Priyanka J. Bobbili, Aolin Wang, Saša Dimitrijević, Erin Sullivan, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Manasi Mohan, Teshawna Badu, Mei Sheng Duh, Peter Valent, Daniel J. DeAngelo

Blood (2024) 144 (Supplement 1): 1801.

https://doi.org/10.1182/blood-2024-204015

Published: 2024

Journal Articles

Elenestinib, an Investigational, Next Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves Symptoms, and Has a Favorable Safety Profile in Patients with Indolent Systemic Mastocytosis: Analysis of the Harbor Trial

Blood (2023) 142 (Supplement 1): 76.

https://doi.org/10.1182/blood-2023-188904

Published: 2023

Journal Articles

Frequency and prognostic impact of blood-circulating tumor mast cells in mastocytosis

Ana Henriques, Javier I. Muñoz-González, Laura Sánchez-Muñoz, Almudena Matito, Lidia Torres-Rivera, María Jara-Acevedo, Carolina Caldas, Andrea Mayado, Alba Pérez-Pons, Andrés C. García-Montero, Iván Álvarez-Twose, Alberto Orfao

Blood (2022) 139 (4): 572–583.

https://doi.org/10.1182/blood.2021012694

Published: 2022

Includes: Supplemental data

Journal Articles

Pioneer Part 2: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib in Indolent Systemic Mastocytosis

Blood (2020) 136 (Supplement 1): 41–42.

https://doi.org/10.1182/blood-2020-136632

Published: 2020

Journal Articles

Pioneer: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy

Blood (2019) 134 (Supplement_1): 2950.

https://doi.org/10.1182/blood-2019-126035

Published: 2019

Journal Articles

Frequency and prognostic impact of KIT and other genetic variants in indolent systemic mastocytosis

Javier I. Muñoz-González, Iván Álvarez-Twose, María Jara-Acevedo, Ana Henriques, Esther Viñas, Carlos Prieto, Laura Sánchez-Muñoz, Carolina Caldas, Andrea Mayado, Almudena Matito, Noelia Dasilva-Freire, Alberto Orfao, Andrés C. García-Montero

Blood (2019) 134 (5): 456–468.

https://doi.org/10.1182/blood.2018886507

Published: 2019

Includes: Supplemental data

Journal Articles

KIT D816V–mutated bone marrow mesenchymal stem cells in indolent systemic mastocytosis are associated with disease progression

Andres C. Garcia-Montero, Maria Jara-Acevedo, Ivan Alvarez-Twose, Cristina Teodosio, Laura Sanchez-Muñoz, Carmen Muñiz, Javier I. Muñoz-Gonzalez, Andrea Mayado, Almudena Matito, Carolina Caldas, Jose M. Morgado, Luis Escribano, Alberto Orfao

Blood (2016) 127 (6): 761–768.

https://doi.org/10.1182/blood-2015-07-655100

Published: 2016

Journal Articles

KIT D816V Mutation Positive Bone Marrow Mesenchymal Stem Cells in Indolent Systemic Mastocytosis Are Associated with Disease Progression

Andres C Garcia-Montero, PhD, Maria Jara-Acevedo, MSc, Ivan Alvarez-Twose, MD, Cristina Teodosio, Laura Sanchez-Muñoz, Javier Ignacio Muñoz-Gonzalez, Andrea Mayado, Almudena Matito, MD PhD, Carolina Caldas, Jose M Morgado, MSc, Luis Escribano, MD PhD, Alberto Orfao, MD PhD

Blood (2015) 126 (23): 4058.

https://doi.org/10.1182/blood.V126.23.4058.4058

Published: 2015

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Improved diagnostic screening and classification of clonal mast cell diseases by ultrasensitive KIT p.D816V detection

Improved overall survival in patients with advanced systemic mastocytosis treated with avapritinib versus real-world therapy based on mutation-adjusted risk score (MARS) stratification

Efficacy and safety results from the primary analysis of the pivotal summit trial: Bezuclastinib in adults with non-advanced systemic mastocytosis

Effect of avapritinib on skin lesions in patients with advanced systemic mastocytosis using a novel, artificial intelligence-based technology

Avapritinib achieves deep and durable symptom control with a well-tolerated safety profile in ism: Long-term outcomes from pioneer

Changes in long-term bone health in patients receiving avapritinib for the treatment of indolent systemic mastocytosis in the pioneer study

An analysis of clonal dynamics in patients with indolent systemic mastocytosis treated with avapritinib in the pioneer study

Ultra-Sensitive KIT testing Uncovers Previously Undetected KIT mutations in Patients with Indolent Systemic Mastocytosis: Results from the Pioneer Trial

Overall Survival and Duration of Treatment in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib Versus Midostaurin or Best Available Therapy in a Real-World Setting

Elenestinib, an Investigational, Next Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves Symptoms, and Has a Favorable Safety Profile in Patients with Indolent Systemic Mastocytosis: Analysis of the Harbor Trial

Frequency and prognostic impact of blood-circulating tumor mast cells in mastocytosis

Pioneer Part 2: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib in Indolent Systemic Mastocytosis

Pioneer: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy

Frequency and prognostic impact of KIT and other genetic variants in indolent systemic mastocytosis

KIT D816V–mutated bone marrow mesenchymal stem cells in indolent systemic mastocytosis are associated with disease progression

KIT D816V Mutation Positive Bone Marrow Mesenchymal Stem Cells in Indolent Systemic Mastocytosis Are Associated with Disease Progression

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American Society of Hematology

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