Health-Related Quality of Life from a Phase 4 Global Clinical Study to Evaluate Discontinuation and Rechallenge of Pexidartinib in Patients with Tenosynovial Giant Cell Tumor (TGCT) Previously Treated with Pexidartinib
Author(s)
Desai J1, Wagner AJ2, Carrasco Garcia I3, Cesari M4, Gordon M5, Lin CC6, Papai Z7, Ryan CW8, Tap WD9, Trent JC10, Gelderblom H11, Grimison P12, López Pousa A13, Van Tine BA14, Dai D15, Rubinacci M15, Tecson K15, Wooddell M15, Stacchiotti S16
1Peter MacCallum Cancer Centre, Melbourne, VIC, Australia, 2Dana-Farber Cancer Institute, Center for Sarcoma and Bone Oncology, Boston, MA, USA, 3Virgen del Rocio University Hospital, Seville, Spain, 4IRCCS Istituto Ortopedico Rizzoli, Bologna, Bologna, Italy, 5HonorHealth Research Institute, Scottsdale, AZ, USA, 6National Taiwan University Hospital, Taipei, Taiwan, 7Semmelweis University, Budapest, Hungary, 8Oregon Health & Science University, Portland, OR, USA, 9Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, NY, USA, 10Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami, FL, USA, 11Leiden University Medical Center, Leiden, Netherlands, 12Chris O’Brien Lifehouse, Sydney, Australia, 13Hospital de la Santa Cruz i Sant Pau, Barcelona, Spain, 14Siteman Cancer Center and Washington University School of Medicine, St. Louis, MO, USA, 15Daiichi Sankyo, Inc., Basking Ridge, NJ, USA, 16Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy
Presentation Documents
OBJECTIVES: Tenosynovial giant cell tumor (TGCT) is a rare, nonmalignant neoplasm of the synovium, bursae, or tendon sheath and is associated with a median onset age of 40 years and deteriorating health-related quality of life (HRQOL). Pexidartinib, the only systemic therapy indicated for treatment of adults with TGCT with severe morbidity/functional limitations not amenable to surgical improvement, has demonstrated robust TGCT tumor responses and improved functional outcomes (FDA-approved in 2019). This phase 4, global, open-label, non-randomized study (NCT04526704) of TGCT patients previously treated with pexidartinib evaluated HRQOL during treatment and discontinuation/rechallenge with pexidartinib.
METHODS: Patients with TGCT from prior trials (NCT02371369; NCT01004861; NCT02734433; NCT03291288) enrolled after the previous trials’ end-of-treatment (EOT) visits. Patients chose to continue pexidartinib (Treatment Continuation Cohort [TCC]; same dose) or discontinue with option to re-initiate (Treatment-Free/Re-Treatment Cohort [TF/RTC]) at the investigator’s/patient’s discretion. Change from Baseline in patient-reported outcomes (PROMIS physical function [PF], EQ-5D-5L) was a secondary endpoint.
RESULTS: From October 2020-April 2021, 32 patients enrolled (21 in TCC, 11 in TF/RTC). Patients had a median age of 47.5 (range: 21-81) years, median of 7.8 (4.6-32.6) years from diagnosis to informed consent, and median of 55.7 (26.7-91.0) months of prior pexidartinib treatment. In the TCC, 24-month average paired change (APC) from Baseline was -2.78 (95% confidence interval: -6.20, 0.65) and -2.4 (-9.6, 4.8) for PROMIS PF and EQ-5D-5L VAS, respectively. During the TF period, 24-month APC was -1.93 (-6.37, 2.51) and 3.4 (-3.6, 10.4) for PROMIS PF and EQ-5D-5L VAS, respectively. Two of three patients who re-initiated treatment had clinically significant (≥10 point) increases in PROMIS PF and EQ-5D-5L VAS.
CONCLUSIONS: Despite previous literature reporting HRQOL deterioration over time for TGCT patients, HRQOL was generally sustained during this study’s 2-year follow-up. Further HRQOL investigations for patients on long-term continuous and intermittent pexidartinib treatment are warranted.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 6, S1 (June 2024)
Code
CO22
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
Oncology, Rare & Orphan Diseases