Assessment of volumetric and modified Choi MRI response in Desmoid Fibromatosis treatment with Sorafenib versus Nirogacestat
Author Block: R. F. Valenzuela, E. Duran-Sierra, M. Antony, J. Espinoza, C. M. Costelloe, J. E. Madewell, W. Murphy, B. Amini, S. Lo; Houston, TX/US
Purpose: Desmoid fibromatosis patients are typically treated with active surveillance and drugs on progression, particularly sorafenib, and most recently, with nirogacestat. This study aimed to determine the signal and volume changes after therapy and over time, comparing both drugs using MRI T2-WI-derived features, including modified Choi (mChoi) and volumetric measurements.
Methods or Background: Retrospective study including 17 patients with single-lesion extremity desmoid fibromatosis and standard-of-care MRI, including T2-WI, from March 2021-February 2024. Volumetric tumor segmentations were created on T2-STIR images. Diameter, mChoi, and volume measurements were computed at four time points across the patient’s treatment: Pre-treatment (Pre-Rx), post-treatment 1 (Rx1), post-treatment 2 (Rx2), and post-treatment 3 (Rx3). The percentage change in diameter (RECIST), mChoi, and volume across time were computed with respect to Rx1 and compared in nirogacestat vs. sorafenib patients.
Results or Findings: Eight patients were treated with nirogacestat and nine patients with sorafenib. Nirogacestat-group: 1) mChoi detected two cases of progression not detected by RECIST or volume, 2) from Pre-Rx to Rx3, the average mChoi decreased by 66% over 20 months (-3.3%/month), and 3) from Pre-Rx to Rx3, the average volume reduced by 49% over 20 months (-2.45%/month). Sorafenib group: 1) RECIST, mChoi, and volume detected two progressions. mChoi detected one response not detected by RECIST or volume, 2) from Pre-Rx to Rx3, the average mChoi decreased by 49% over 34 months (-1.44%/month), and 3) from Pre-Rx to Rx3, the average volume decreased by 4% over 34 months (-0.09%/month).
Conclusion: Patients treated with nirogacestat exhibited larger volumetric and mChoi changes, with the fastest response and similar sustained positive treatment effects compared to those treated with sorafenib, indicating the potential of nirogacestat as an effective treatment option for desmoid tumors.
Limitations: Small sample size.
Funding for this study: The John S. Dunn, Sr. Distinguished Chair in Diagnostic imaging and M.R Evelyn Hudson Foundation Endowed Professorship.
Has your study been approved by an ethics committee? Not applicable
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