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Journal Articles

Avapritinib treatment of patients with advanced systemic mastocytosis: 4-year safety, effect on bone and first-line efficacy results of the pathfinder clinical study

Free
Andreas Reiter, Tracy George, Deepti Radia, Michael Deininger, Johannes Luebke, Hui-Min Lin, Ilda Bidollari, Joana Caetano-Lopes, Guang Yang, Amber King, Saša Dimitrijević, Daniel Deangelo, Jason Gotlib
Journal: Blood
Blood (2025) 146 (Supplement 1): 1022.
https://doi.org/10.1182/blood-2025-1022
Published: 2025
Journal Articles

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

Free
Deepti Radia, Tracy George, Ivan Alvarez-Twose, Francesco Mannelli, Michael Deininger, Kristen Pettit, Celalettin Ustun, Cristina Bulai Livideanu, Ingunn Dybedal, Elizabeth O. Hexner, Hui-Min Lin, Ilda Bidollari, Amber King, Saša Dimitrijević, Javier I. Muñoz-González, Brian D'Alessandro, Mira Patel, Anthony Melione, Jason Gotlib, Andreas Reiter, Daniel Deangelo, Frank Siebenhaar, Sigurd Broesby-Olsen, Karin Hartmann
Journal: Blood
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

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

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

Diagnostic evolution in systemic mastocytosis: Clinical impact of who 2022 criteria on smoldering systemic mastocytosis identification in pioneer

Free
Tracy George, Mariana Castells, Cem Akin, Hanneke Oude Elberink, Karin Hartmann, Sigurd Broesby-Olsen, Frank Siebenhaar, Hui-Min Lin, Janet Hong, Benjamin Lampson, Jason Gotlib
Journal: Blood
Blood (2025) 146 (Supplement 1): 5578.
https://doi.org/10.1182/blood-2025-5578
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

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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 3164.
https://doi.org/10.1182/blood-2024-207798
Published: 2024
Journal Articles

Avapritinib As First-Line Therapy in Patients with Advanced Systemic Mastocytosis: Efficacy and Safety from the Pathfinder Clinical Study

Free
Deepti H. Radia, Jason Gotlib, Mark W. Drummond, Tracy I. George, Hui-Min Lin, Saša Dimitrijević, Javier I. Munoz-Gonzalez, Ilda Bidollari, Michael W. Deininger, Daniel J. DeAngelo, Andreas Reiter
Journal: Blood
Blood (2022) 140 (Supplement 1): 1509–1511.
https://doi.org/10.1182/blood-2022-166986
Published: 2022
Journal Articles

An Updated Analysis on Safety and Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis from the Explorer Clinical Study: Long-Term Efficacy and Safety

Free
Daniel J. DeAngelo, Deepti H. Radia, Tracy I. George, William A. Robinson, Albert T. Quiery, Mark W. Drummond, Prithviraj Bose, Elizabeth O. Hexner, Elliott Winton, Hans-Peter Horny, Meera Tugnait, Hui-Min Lin, Saša Dimitrijević, Javier I. Munoz-Gonzalez, Ilda Bidollari, Michael W. Deininger, Jason Gotlib
Journal: Blood
Blood (2022) 140 (Supplement 1): 3932–3934.
https://doi.org/10.1182/blood-2022-167593
Published: 2022
Journal Articles

Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis: Hematologic and Bone Marrow Responses from the Phase 2 Open-Label, Single-Arm, Pathfinder Study

Free
Tracy I. George, Kristin H. Karner, Karen A. Moser, Anton Rets, Michelle Fredericks, Andreas Reiter, Deepti H. Radia, Michael W. Deininger, Hui-Min Lin, Jayita Sen, Jason Gotlib, Saša Dimitrijević, Daniel J. DeAngelo
Journal: Blood
Blood (2021) 138 (Supplement 1): 2565.
https://doi.org/10.1182/blood-2021-146873
Published: 2021
Journal Articles

Effective Control of Advance Systemic Mastocytosis with Avapritinib: Mutational Analysis from the Explorer Clinical Study

Free
Michael W. Deininger, Daniel J. DeAngelo, Deepti H. Radia, Tracy I. George, Guang Yang, Jayita Sen, Hui-Min Lin, Brenton Mar, Jason Gotlib
Journal: Blood
Blood (2021) 138 (Supplement 1): 318.
https://doi.org/10.1182/blood-2021-150872
Published: 2021
Journal Articles

Pure Pathologic Response Is Associated with Improved Overall Survival in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib in the Phase I EXPLORER Study

Free
Jason Gotlib, MD MS, Deepti H. Radia, MRCPI, FRCPath, Tracy I. George, MD, William A Robinson, MD PhD, Albert T. Quiery, MD, Mark W. Drummond, PhD FRCPath, Prithviraj Bose, MD, Elizabeth O. Hexner, MD, Elliott F. Winton, MD, Hans-Peter Horny, Prof, Dr, Meera Tugnait, PhD, Oleg Schmidt-Kittler, PhD, Erica K. Evans, PhD, Hui-Min Lin, PhD, Brenton G. Mar, MD PhD, Michael W. Deininger, MD PhD, Daniel J. DeAngelo, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 37–38.
https://doi.org/10.1182/blood-2020-137413
Published: 2020
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

Increased Detection of KIT D816V Mutation in Peripheral Blood Samples from Patients with Indolent Systemic Mastocytosis (ISM) in the Phase 2 Pioneer Study Using a High Sensitivity Droplet Digital (dd) PCR Assay Compared with Next Generation Sequencing (NGS)

Free
Tracy I. George, MD, Gerard Hoehn, PhD, Hui-Min Lin, PhD, Stephen Miller, PhD, Cem Akin, MDPhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 7–8.
https://doi.org/10.1182/blood-2020-136930
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

Superior Clinical Benefit of Brentuximab Vedotin in Mycosis Fungoides Versus Physician's Choice Irrespective of CD30 Level or Large Cell Transformation Status in the Phase 3 ALCANZA Study

Free
Youn H. Kim, MD, H. Miles Prince, MD FRACP, FRCPA, Sean Whittaker, MB, ChB, FRCP, MD, Steven M. Horwitz, MD, Madeleine Duvic, MD, Oliver Bechter, Jose A. Sanches, Rudolf Stadler, MD, Julia Scarisbrick, MD FRCP, MBBS, Pietro Quaglino, MD, Pier Luigi Zinzani, MD, Pascal Wolter, MD, Herbert Eradat, MD, Lauren C. Pinter-Brown, MD, Pablo L. Ortiz-Romero, Oleg E. Akilov, MD, Judith Trotman, FRACP, Kerry Taylor, Michael Weichenthal, MD, Jan Walewski, MD, David C. Fisher, MD PhD, Marise McNeeley, Alejandro A. Gru, MD, Yinghui Wang, MS, Maria Corinna Palanca-Wessels, MD PhD, Julie Lisano, Martha Li, Hui-Min Lin, PhD, Meredith Little, MPH, William L. Trepicchio, PhD, Reinhard Dummer, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1646.
https://doi.org/10.1182/blood-2018-99-112847
Published: 2018
Journal Articles

Updated Analyses of the International, Open-Label, Randomized, Phase 3 Alcanza Study: Longer-Term Evidence for Superiority of Brentuximab Vedotin Versus Methotrexate or Bexarotene for CD30-Positive Cutaneous T-Cell Lymphoma (CTCL)

Free
Steven M. Horwitz, MD, Julia J. Scarisbrick, MD FRCP, MBBS †, Reinhard Dummer, MD, Madeleine Duvic, MD, Youn H. Kim, MD, Jan Walewski, MD, Sean Whittaker, MB, ChB, FRCP, MD, Pietro Quaglino, MD, Pier Luigi Zinzani, MD, Pascal Wolter, MD, Herbert Eradat, MD, Jose A. Sanches, PhD, Pablo L. Ortiz-Romero, MD PhD, Oleg E. Akilov, MD, Judith Trotman, FRACP, Kerry Taylor, FRACP,FRCPA,MBBS, Stephane Dalle, MD PhD, Michael Weichenthal, MD, David C. Fisher, MD, Rudolf Stadler, MD, Tatyana A Feldman, MD, Timothy M. Kuzel, MD, Alejandro A. Gru, MD, Yinghui Wang, MS, Maria Corinna Palanca-Wessels, MD PhD, Hui-Min Lin, PhD, Yi Liu, PhD, Meredith Little, MPH, H. Miles Prince, MD FRACP, FRCPA
Journal: Blood
Blood (2017) 130 (Supplement 1): 1509.
https://doi.org/10.1182/blood.V130.Suppl_1.1509.1509
Published: 2017