Pexidartinib improves physical functioning and stiffness in patients with tenosynovial giant cell tumor
| Autor: | Andrew J. Wagner, Dale Shuster, William D. Tap, Michiel A. J. van de Sande, Jayesh Desai, Javier Martin-Broto, Emanuela Palmerini, Rebecca M. Speck, John H. Healey, Heather L. Gelhorn, Xin Ye, Thierry Alcindor, Silvia Stacchiotti, Qiang Wang, Hans Gelderblom, Kristen N. Ganjoo |
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| Přispěvatelé: | Daiichi-Sankyo |
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Adult
Male medicine.medical_specialty Pexidartinib MEDLINE Aminopyridines Giant Cell Tumor of Tendon Sheath Tenosynovial giant cell tumor Placebo law.invention 03 medical and health sciences 0302 clinical medicine Text mining Randomized controlled trial Double-Blind Method law Internal medicine Medicine Humans Orthopedics and Sports Medicine In patient Pyrroles 030212 general & internal medicine Patient Reported Outcome Measures Orthopedic surgery 030222 orthopedics business.industry General Medicine Middle Aged Lower Extremity Surgery Female business RD701-811 Research Article |
| Zdroj: | Digital.CSIC. Repositorio Institucional del CSIC instname Acta Orthopaedica article-version (VoR) Version of Record Acta Orthopaedica, 92(4), 493-499. TAYLOR & FRANCIS LTD Acta Orthopaedica, Vol 92, Iss 4, Pp 493-499 (2021) |
| Popis: | [Background and purpose] The ENLIVEN trial showed that, after 25 weeks, pexidartinib statistically significantly reduced tumor size more than placebo in patients with symptomatic, advanced tenosynovial giant cell tumor (TGCT) for whom surgery was not recommended. Here, we detail the effect of pexidartinib on patient-reported physical function and stiffness in ENLIVEN. [Patients and methods] This was a planned analysis of patient-reported outcome data from ENLIVEN, a double-blinded, randomized phase 3 trial of adults with symptomatic, advanced TGCT treated with pexidartinib or placebo. Physical function was assessed using the Patient-Reported Outcomes Measurement Information System (PROMIS)-physical function (PF), and worst stiffness was assessed using a numerical rating scale (NRS). A mixed model for repeated measures was used to compare changes in PROMIS-PF and worst stiffness NRS scores from baseline to week 25 between treatment groups. Response rates for the PROMIS-PF and worst stiffness NRS at week 25 were calculated based on threshold estimates from reliable change index and anchor-based methods. [Results] Between baseline and week 25, greater improvements in physical function and stiffness were experienced by patients receiving pexidartinib than patients receiving placebo (change in PROMIS-PF = 4.1 [95% confidence interval (CI) 1.8–6.3] vs. –0.9 [CI −3.0 to 1.2]; change in worst stiffness NRS = –2.5 [CI −3.0 to −1.9] vs. –0.3 [CI −0.9 to 0.3]). Patients receiving pexidartinib had higher response rates than patients receiving placebo for meaningful improvements in physical function and stiffness. Improvements were sustained after 50 weeks of pexidartinib treatment. [Interpretation] Pexidartinib treatment provided sustained, meaningful improvements in physical function and stiffness for patients with symptomatic, advanced TGCT. This study was supported by Daiichi Sankyo. De-identified individual participant data and supporting clinical study documents are available on request, depending on circumstances, at https://vivli.org. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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