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Journal Articles

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

Free
Tsewang Tashi, Hanneke Oude Elberink, Cem Akin, Mariana Castells, Jens Panse, Deepti Radia, Frank Siebenhaar, Cristina Bulai Livideanu, Karin Hartmann, Sigurd Broesby-Olsen, Tracy George, Vito Sabato, Paul van Daele, Sonia Cerquozzi, Ingunn Dybedal, Joseph Jurcic, Stéphane Barete, Andreas Reiter, Thanai Pongdee, Ruchi Desai, Philippe Schafhausen, Celalettin Ustun, Prithviraj Bose, Peter Vadas, Massimo Triggiani, Patrizia Bonadonna, Daniel Deangelo, Lindsay Rein, Pankit Vachhani, Ilda Bidollari, Hui-Min Lin, Janet Hong, Benjamin Lampson, Ashley Doyle, Ivan Alvarez-Twose, Jason Gotlib
Journal: Blood
Blood (2025) 146 (Supplement 1): 2024.
https://doi.org/10.1182/blood-2025-2024
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

Free
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
Journal: Blood
Blood (2025) 146 (Supplement 1): 80.
https://doi.org/10.1182/blood-2025-80
Published: 2025
Journal Articles

Interpreting tryptase levels and avoiding common pitfalls in screening for clonal mast cell disease

Free
Mariana Castells, Matthew Giannetti, Cem Akin, Joseph Jurcic, Aaron Zakharyan, Ray Coghlan, Karin Hartmann, Vito Sabato
Journal: Blood
Blood (2025) 146 (Supplement 1): 4774.
https://doi.org/10.1182/blood-2025-4774
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

Free
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
Journal: Blood
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

Free
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
Journal: Blood
Blood (2025) 146 (Supplement 1): 5573.
https://doi.org/10.1182/blood-2025-5573
Published: 2025
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

Free
Tsewang Tashi, Olivier Hermine, Mariana Castells, Mar Guilarte, Vito Sabato, Marcus Maurer, Jens Panse, Ivan Alvarez-Twose, Renata Cabral, Robert Bird, Stéphane Barete, Laurence Bouillet, Maud Hermans, Paul Van Daele, David González-De-Olano, Elizabeth A. Griffiths, Joseph Jurcic, Ingunn Dybedal, Gandhi Laurent Damaj, Philippe Schafhausen, Chiara Elena, Tse-Chieh Teh, Pankit Vachhani, Caroline Labe, Saranya Venugopal, Kevin He, Javier Muñoz-González, Ben Lampson, Robyn Scherber, Prithviraj Bose, Clive Grattan, Thanai Pongdee, Tracy I. George, Cristina Bulai Livideanu
Journal: Blood
Blood (2023) 142 (Supplement 1): 76.
https://doi.org/10.1182/blood-2023-188904
Published: 2023
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

Free
Cem Akin, MDPhD, Hanneke Oude Elberink, MD PhD, Jason Gotlib, MD MS, Vito Sabato, MD PhD, Karin Hartmann, MD, Sigurd Broesby-Olsen, MD, Mariana Castells, MD PhD, Tsewang Tashi, MD, Mark L Heaney, MD, Tracy I. George, MD, Frank Siebenhaar, MD, Deepti H. Radia, MRCPI, FRCPath, Massimo Triggiani, MD PhD, Paul van Daele, MD, Daniel J. DeAngelo, MD PhD, Iván Alvarez-Twose, MD PhD, Andreas Reiter, MD, Peter Vadas, MD PhD, Patrizia Bonadonna, MD, Paggy Hew, MS, Hui-Min Lin, PhD, Maria Roche, MS, Marcus Maurer, MD
Journal: Blood
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

Free
Cem Akin, MD PhD, Vito Sabato, Jason Gotlib, MD MS, Mariana Castells, Michael W. Deininger, Hanneke Oude Elberink, Mark L Heaney, Paul van Daele, Deepti Radia, Massimo Triggiani, Daniel J. DeAngelo, MD, Iván Alvarez-Twose, Sigurd Broesby-Olsen, Tracy I. George, MD, Karin Hartmann, Siebenhaar Frank, Andreas Reiter, Peter Vadas, Patrizia Bonadonna, Jens P. Panse, Petra Staubach-Renz, Knut Brockow, Diamant Thaci, Hui-Min Lin, Andrew Morrison, Brenton Mar, Marcus Maurer
Journal: Blood
Blood (2019) 134 (Supplement_1): 2950.
https://doi.org/10.1182/blood-2019-126035
Published: 2019
Journal Articles

Distinct Diagnostic and Clinical Features of Bone Marrow Mastocytosis: A Study of the Registry of the European Competence Network on Mastocytosis (ECNM)

Free
Massimiliano Bonifacio, MD, Patrizia Bonadonna, MD, Hanneke Oude Elberink, MD, Björn van Anrooij, MD, Aleksandra Górska, MD, Magdalena Lange, MD, Emir Hadzijusufovic, DVM, Luigi Scaffidi, Ilaria Tanasi, MD, Cecelia Perkins, Virginia Valeria Ferretti, Anja Illerhaus, PhD, Thilo Jakob, MD, Roberta Parente, MD, Mohamad Jawhar, MD, Francesca Caroppo, MD, Alexander Zink, MD, Alex Kilbertus, MD, Akif Selim Yavuz, MD, Michael Doubek, MD PhD, Hans Hagglund, MD PhD, Jens P. Panse, MD, Vito Sabato, MD PhD, Elisabeth Aberer, MD, Nadja Jaekel, MD, Peter Vandenberghe, MD PhD, David Fuchs, MD, Knut Brockow, MD, Anna Belloni Fortina, MD, Andreas Reiter, MD, Massimo Triggiani, MD, Khalid Shoumariyeh, MD, Karin Hartmann, MD, Chiara Elena, Olivier Hermine, MD PhD, Jason Gotlib, MD, Marek Niedoszytko, MD, Hanneke Kluin-Nelemans, MD PhD, Wolfgang R. Sperr, MD, Peter Valent, MD, Roberta Zanotti, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4200.
https://doi.org/10.1182/blood.V130.Suppl_1.4200.4200
Published: 2017
Journal Articles

Selection and Efficacy of Cytoreductive Agents in Patients with Mastocytosis Included in the Registry of the European Competence Network on Mastocytosis (ECNM)

Free
Chiara Elena, Serena Merante, MD, Virginia Valeria Ferretti, Lambertus CM Span, MD PhD, Aleksandra Górska, MD, Massimiliano Bonifacio, MD, Cecelia Perkins, Olivier Hermine, MD PhD, Anja Illerhaus, PhD, Sabine Mueller, Roberta Parente, MD, Mohamad Jawhar, MD, Elena Fontana, Alexander Zink, Alex Kilbertus, MD, Akif Selim Yavuz, Michael Doubek, MD PhD, Hans Hagglund, MD PhD, Jens P. Panse, MD, Vito Sabato, Haifa Kathrin Al Ali, Elisabeth Aberer, MD, Marie Anne Morren, Magdalena Lange, MD, David Fuchs, MD, Knut Brockow, MD, Anna Belloni Fortina, Andreas Reiter, MD, Massimo Triggiani, MD, Nikolas von Bubnoff, MD, Karin Hartmann, MD, Jason Gotlib, MD, Luigi Scaffidi, Marek Niedoszytko, MD, Hanneke Kluin-Nelemans, MD PhD, Luca Malcovati, MD, Wolfgang R. Sperr, MD, Peter Valent, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1650.
https://doi.org/10.1182/blood.V130.Suppl_1.1650.1650
Published: 2017
Journal Articles

Prognostic Factors and Survival Prediction in 1,088 Patients with Mastocytosis Collected in the Registry of the European Competence Network on Mastocytosis (ECNM Registry)

Free
Wolfgang R. Sperr, MD, Michael Kundi, MD, Hanneke Oude Elberink, MD, Björn van Anrooij, MD, Karoline V Gleixner, MD, Emir Hadzijusufovic, DVM, Aleksandra Górska, MD, Magdalena Lange, MD, Anja Rabenhorst, PhD, Serena Merante, MD, Chiara Elena, MD, Anna Belloni Fortina, MD, Elena Fontana, MD, Juliana Schwaab, MD, Mohamad Jawhar, MD, Roberta Zanotti, MD, Patrizia Bonadonna, MD, Massimo Triggiani, MD, Cecelia Perkins, MPH, Jason R. Gotlib, MD, Michael Doubek, Khalid Shoumaryeh, MD, David Fuchs, MD, Vito Sabato, MD, Knut Brockow, MD, Agnes Bretterklieber, MD, Nadja Jaekel, MD, Andreas Reiter, MD, Karin Hartmann, MD, Marek Niedoszytko, MD, Hanneke C. Kluin-Nelemans, MD PhD, Peter Valent, MD
Journal: Blood
Blood (2016) 128 (22): 396.
https://doi.org/10.1182/blood.V128.22.396.396
Published: 2016