Tumor volume score (TVS), modified recist, and tissue damage score (TDS) as novel methods for assessing response in tenosynovial giant cell tumors (TGCT) treated with pexidartinib: Relationship with patient-reported outcomes (PROs).

Authors

null

Charles Peterfy

Spire Sciences, Inc., Boca Raton, FL

Charles Peterfy , Xin Ye , Heather Gelhorn , Rebecca M Speck , Peter J. Countryman , Vicki Leigh Keedy , Zev A. Wainberg , Arun S. Singh , Bartosz Chmielowski , Daniel D. Von Hoff , Hani M. Babiker , Vivek Khemka , Gregory Michael Cote , Geoffrey Shapiro , Andrew J. Wagner , John H. Healey , Henry Hsu , Paul S. Lin , Sandra Tong , William D. Tap

Organizations

Spire Sciences, Inc., Boca Raton, FL, Daiichi Sankyo Co., Ltd., Edison, NJ, Evidera, Bethesda, MD, Vanderbilt University Medical Center, Nashville, TN, David Geffen School of Medicine at University of California Los Angeles, Los Angeles, CA, University of California Los Angeles Medical Center, Los Angeles, CA, Translational Genomics Research Institute (TGen) and HonorHealth, Phoenix, AZ, University of Arizona Cancer Center, Tucson, AZ, HonorHealth Research Institute, Scottsdale, AZ, Massachusetts General Hospital, Boston, MA, Dana-Farber Cancer Institute, Boston, MA, Memorial Sloan-Kettering Cancer Center, New York, NY, Plexxikon, Inc., Berkeley, CA, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

Research Funding

Pharmaceutical/Biotech Company

Background: TGCT is a locally aggressive neoplasm of joint and tendon sheath synovia that may cause pain, limit joint function and destroy bone and local tissues. Measuring TGCT with RECIST is a challenge due to irregular shape and asymmetrical growth, and local tissue damage is not assessed. We reported earlier results of a longitudinal trial of pexidartinib, a selective CSF1R kinase inhibitor, using RECIST as well as novel TVS, modified RECIST and TDS. Here we examine concordance of these MRI measures with PROs. Methods: Patients (pts) with progressive TGCT in a single-arm, multi-center trial of pexidartinib (1000 mg po daily) were assessed by MRI every 2 months by 2 central radiologists (blind to visit order). For RECIST, longest measurable dimensions of up to 2 tumors per joint or tendon sheath were summed (SLD). Modified RECIST summed short axis dimensions (SSD). TVS was based on 10% increments of the estimated maximally distended normal synovial cavity or tendon sheath. TDS scored bone erosion (ERO), cartilage loss (CAR) and bone marrow edema (BME) in multiple regions of each joint. The relationship with PROs (Worst Pain numerical rating scale [NRS] and Worst Stiffness NRS) was assessed. Results: 15 pts (7 knees, 3 hips, 2 ankles, 1 elbow, 1 wrist, 1 thigh) with PRO data and evaluable MRI scans at baseline and Month 7 were assessed. All SLD, SSD and TVS scores improved with respective median changes of -25%, -39% and -50%. Baseline ERO, CAR, and BME ranged 0-19, 0-34, and 0-15, respectively. Median change for each was 0%: ERO worsened in 1 pt, CAR did not change, and BME improved in 4 and worsened in 2. Worst Pain NRS and Worst Stiffness NRS improved in 11 and 9 pts, respectively. Conclusions: TVS demonstrated the greatest pexidartinib effect size, followed by SSD and then conventional RECIST. All had good concordance with PROs. Clinical trial information: NCT01004861

Concordance of MRI measures with PROs.

Pain improved (Yes)Pain improved (No)Stiffness improved (Yes)Stiffness improved (No)
SLD improved (Yes)11193
SLD improved (No)0000
SSD improved (Yes)11294
SSD improved (No)0000
TVS improved (Yes)10284
TVS improved (No)1010

Disclaimer

This material on this page is ©2024 American Society of Clinical Oncology, all rights reserved. Licensing available upon request. For more information, please contact licensing@asco.org

Abstract Details

Meeting

2017 ASCO Annual Meeting

Session Type

Poster Session

Session Title

Sarcoma

Track

Sarcoma

Sub Track

Soft Tissue Tumors

Clinical Trial Registration Number

NCT01004861

Citation

J Clin Oncol 35, 2017 (suppl; abstr 11048)

DOI

10.1200/JCO.2017.35.15_suppl.11048

Abstract #

11048

Poster Bd #

371

Abstract Disclosures