Plain English summary
tumor, DT/AF, outcome(s, Fibromatosis, Desmoid tumors
FIBROMATOSIS, TUMOR, DESMOID, DESMOID TUMORS
Desmoid tumors are associated with significant symptoms that affect patients’ quality of life. The effect of treatment on patient-reported outcomes is as important as its effect on the tumor. The efficacy and safety of nirogacestat have been evaluated in an appropriately designed clinical study. Although patient-reported outcome(s) may be included in a clinical study, these outcomes are infrequently included in drug labeling for oncology drugs primarily due to challenges related to the reliability and validity of the measurements. The GODDESS tool was developed to assess Desmoid Tumor/Aggressive Fibromatosis (DT/AF) symptom severity and their impact on patients’ lives. This paper presents the results of the analysis of patient-reported outcomes in the study of nirogacestat. GODDESS was found to be dependable, valid, responsive, and interpretable as a means of examining the effect of treatment on patient-reported outcomes.