End Points and Assessments
death, mucinous and cystic lesions, Tumor, cancer, bowel obstruction
TUMOR, BOWEL PERFORATION, CANCER, BOWEL OBSTRUCTION, COMPLICATION, SECONDARY, CAVITY, APPENDIX CANCER, TUMOR GROWTH, COMPLICATIONS
The primary end point was the difference in tumor growth (percentage change), using the mpRECIST method, between the chemotherapy and observation periods. The mpRECIST was developed for this trial specifically to address the limitations of standard RECIST in appendix cancer. mpRECIST measures 5 lesions (mucinous and cystic lesions are allowed) in the peritoneal cavity in contrast to the maximum of 2 lesions, per standard RECIST. A computed tomography scan of the abdomen and pelvis was performed at baseline and every 3 months as standard of care. Tumor markers (carcinoembryonic antigen [CEA], cancer antigen 125 [CA125], and CA19-9) were measured in peripheral blood collected at baseline and 3, 6, 9, and 12 months. All patients with available 3-, 6-, 9-, or 12-month data were combined to compare percentage change in each marker level between the observation and treatment periods. Additional secondary efficacy end points were the objective response rate, the rate of bowel complications (defined as bowel obstruction requiring hospitalization or bowel perforation), and differences in OS between early and delayed chemotherapy approaches. Safety monitoring was conducted for the composite safety end point of death or bowel complication.