Avapritinib achieves long-term disease control with favorable safety in patients with indolent systemic mastocytosis over 3 years
Background - Indolent systemic mastocytosis (ISM), a clonal mast cell disease primarily driven by the KIT D816V mutation, can cause long-term debilitating symptoms and poor quality of life. Most patients rely on symptom-directed best supportive care (BSC) medications, which do not treat the underlyi...
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| Hauptverfasser: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
August 2026
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| In: |
The journal of allergy and clinical immunology
Year: 2026, Jahrgang: 158, Heft: 2, Pages: 421-431 |
| ISSN: | 1097-6825 |
| DOI: | 10.1016/j.jaci.2026.04.001 |
| Online-Zugang: | Verlag, kostenfrei, Volltext: https://doi.org/10.1016/j.jaci.2026.04.001 Verlag, kostenfrei, Volltext: https://www.sciencedirect.com/science/article/pii/S0091674926002538 
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| Verfasserangaben: | Cem Akin, MD, PhD, Vito Sabato, MD, PhD, Jason Gotlib, MD, MS, Jens Panse, MD, Iván Álvarez-Twose, MD, PhD, Deepti H. Radia, MRCPI, FRCPath, Cristina Bulai Livideanu, MD, MSc, Joseph Jurcic, MD, Hanneke Oude Elberink, MD, PhD, Paul Van Daele, MD, PhD, Sonia Cerquozzi, MD, Ingunn Dybedal, MD, PhD, Andreas Reiter, MD, Thanai Pongdee, MD, Stéphane Barete, MD, PhD, Celalettin Ustun, MD, Philippe Schafhausen, MD, Peter Vadas, MD, PhD, Prithviraj Bose, MD, Daniel J. DeAngelo, MD, PhD, Lindsay Rein, MD, Pankit Vachhani, MD, Massimo Triggiani, MD, PhD, Patrizia Bonadonna, MD, Karin Hartmann, MD, Sigurd Broesby-Olsen, MD, Mattias Mattsson, MD, PhD, Tracy I. George, MD, William Shomali, MD, Matthew Giannetti, MD, Frank Siebenhaar, PD Dr Med, Hui-Min Lin, PhD, Ilda Bidollari, MD, MBA, Benjamin Lampson, MD, PhD, Janet Hong, PharmD, Ashley Doyle, PharmD, Tsewang Tashi, MD, and Mariana Castells, MD, PhD |
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| 245 | 1 | 0 | |a Avapritinib achieves long-term disease control with favorable safety in patients with indolent systemic mastocytosis over 3 years |c Cem Akin, MD, PhD, Vito Sabato, MD, PhD, Jason Gotlib, MD, MS, Jens Panse, MD, Iván Álvarez-Twose, MD, PhD, Deepti H. Radia, MRCPI, FRCPath, Cristina Bulai Livideanu, MD, MSc, Joseph Jurcic, MD, Hanneke Oude Elberink, MD, PhD, Paul Van Daele, MD, PhD, Sonia Cerquozzi, MD, Ingunn Dybedal, MD, PhD, Andreas Reiter, MD, Thanai Pongdee, MD, Stéphane Barete, MD, PhD, Celalettin Ustun, MD, Philippe Schafhausen, MD, Peter Vadas, MD, PhD, Prithviraj Bose, MD, Daniel J. DeAngelo, MD, PhD, Lindsay Rein, MD, Pankit Vachhani, MD, Massimo Triggiani, MD, PhD, Patrizia Bonadonna, MD, Karin Hartmann, MD, Sigurd Broesby-Olsen, MD, Mattias Mattsson, MD, PhD, Tracy I. George, MD, William Shomali, MD, Matthew Giannetti, MD, Frank Siebenhaar, PD Dr Med, Hui-Min Lin, PhD, Ilda Bidollari, MD, MBA, Benjamin Lampson, MD, PhD, Janet Hong, PharmD, Ashley Doyle, PharmD, Tsewang Tashi, MD, and Mariana Castells, MD, PhD |
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| 520 | |a Background - Indolent systemic mastocytosis (ISM), a clonal mast cell disease primarily driven by the KIT D816V mutation, can cause long-term debilitating symptoms and poor quality of life. Most patients rely on symptom-directed best supportive care (BSC) medications, which do not treat the underlying driver of ISM. Avapritinib, an oral, potent, selective KIT D816V inhibitor, is approved in adults with ISM. - Objective - We sought to understand the long-term efficacy and safety of avapritinib in ISM. - Methods - The PIONEER trial (NCT03731260) enrolled adults with moderate to severe ISM symptoms. Patients initiated avapritinib 25 mg once daily (QD; recommended dose) plus BSC in part 1, 2, or 3; open-label part 3 is ongoing with up to 5 years of follow-up. As per investigator discretion and disease burden, a dose increase up to avapritinib 50 mg QD was permitted in part 3. - Results - As of February 21, 2025, 226 patients initiated avapritinib at 25 mg QD. The median (range) treatment duration was 40.0 (0.7-67.2) months. Patients receiving avapritinib experienced durable and clinically meaningful symptom improvement (mean change, −19.39 [n = 127] in the Indolent Systemic Mastocytosis Symptom Assessment Form total symptom score) through approximately 3 years. Avapritinib continued to be well tolerated for a longer term with a safety profile comparable with the previously reported placebo-controlled portion. Most treatment-related adverse events (TRAEs) were grades 1 to 2, with limited grade 3 or higher reported. Edema events were the most frequent TRAEs (mostly grade 1). Serious TRAEs occurred in 3 patients (1%), and 7 patients (3%) discontinued treatment because of TRAEs. - Conclusions - Long-term follow-up (median, ∼3 years) demonstrates that avapritinib is effective and well tolerated. Avapritinib shows a favorable benefit-risk profile as a chronic ISM treatment. | ||
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| 650 | 4 | |a PIONEER study | |
| 650 | 4 | |a quality of life | |
| 650 | 4 | |a safety | |
| 650 | 4 | |a systemic mastocytosis | |
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