A phase 4, multicenter, global clinical study to evaluate discontinuation and rechallenge of pexidartinib in patients with tenosynovial giant cell tumor previously treated with pexidartinib
Pexidartinib is a medication for people with tenosynovial giant cell tumor (TGCT) for whom surgery is not recommended. This study examined what happens when patients stop taking pexidartinib (after it has helped them) to see whether it is safe and effective to restart if needed. Thirty-two patients were followed for 2 years: 21 chose to continue pexidartinib and did not experience disease progression, while 11 chose to stop pexidartinib; 54.5% of them had disease progression within 2 years. Three patients who stopped pexidartinib restarted treatment after their tumor size increased or their symptoms worsened. Restarting pexidartinib was safe and effective in these patients.
Authors
- Andrew J. Wagner (ORCID: https://orcid.org/0000-0002-4384-9448)
- Brian Andrew Van Tine (ORCID: https://orcid.org/0000-0003-4572-6668)
- Jonathan C. Trent (ORCID: https://orcid.org/0000-0001-6169-5238)
- Peter Grimison (ORCID: https://orcid.org/0000-0002-2653-1657)
- Kristen M. Tecson (ORCID: https://orcid.org/0000-0002-6732-8698)
- Hans A. J. Gelderblom (ORCID: https://orcid.org/0000-0001-9270-8636)
- Marilena Cesari (ORCID: https://orcid.org/0000-0003-1505-758X)
- William D. Tap (ORCID: https://orcid.org/0000-0001-7779-2796)
- Christopher W. Ryan (ORCID: https://orcid.org/0000-0002-4259-1123)
- Silvia Stacchiotti (ORCID: https://orcid.org/0000-0002-1742-8666)
- Zsuzsanna Pápai (ORCID: https://orcid.org/0009-0004-7742-3682)
- Jayesh Desai (ORCID: https://orcid.org/0000-0003-4246-9344)
- Margaret Wooddell (ORCID: https://orcid.org/0009-0008-3657-6614)
- Antonio López–Pousa (ORCID: https://orcid.org/0000-0003-1066-3951)
- M. Rubinacci
- Michael A. Gordon (ORCID: https://orcid.org/0000-0002-6212-6421)
- Abdul Waheed Rajper (ORCID: https://orcid.org/0009-0000-6468-7380)
- Irene Carrasco García
- Dong Dai
- Chia‐Chi Lin (ORCID: https://orcid.org/0009-0006-5084-0819)
Institutions
- Semmelweis University (HU)
- The Royal Melbourne Hospital (AU)
- Memorial Sloan Kettering Cancer Center (US)
- Oregon Health & Science University (US)
- Washington University in St. Louis (US)
- Cornell University (US)
- Leiden University Medical Center (NL)
- Peter MacCallum Cancer Centre (AU)
- Hospital de Sant Pau (ES)
- Chris O’Brien Lifehouse (AU)
- University of Miami Health System (US)
- Dana-Farber Cancer Institute (US)
- Istituto Ortopedico Rizzoli (IT)
- HonorHealth (US)
- National Taiwan University Hospital (TW)
- Hospital Universitario Virgen del Rocío (ES)
- Fondazione IRCCS Istituto Nazionale dei Tumori (IT)
- Sylvester Comprehensive Cancer Center (US)
- Daiichi Sankyo (United States) (US)
Publication Details
- Journal
- Cancer
- Published
- 2024-11-12
- DOI
- https://doi.org/10.1002/cncr.35634
- Citations
- 8
- Primary Topic
- Musculoskeletal synovial abnormalities and treatments
- Type
- article
- Field-Weighted Citation Impact
- 3.76
Funders
- Amgen
- Bristol-Myers Squibb
- Eli Lilly and Company
- AstraZeneca
- GlaxoSmithKline
- BeiGene
- Daiichi-Sankyo
- Daiichi Sankyo Europe