Efficacy of Systemic Chemotherapy in Patients With Low-grade Mucinous Appendiceal Adenocarcinoma

• DOI: 10.1001/jamanetworkopen.2023.16161
• Publication Date: 2023-06-01T15:32:56Z
• Authors: John Paul Shen; Abdelrahman M. Yo...; Fadl A. Zeineddine; Mohammad A. Zeine...; Rebecca S. Tidwell; Karen A. Beaty; Lisa C. Scofield; Safia Rafeeq; Nicholas Hornstein; Elizabeth Lano; Cathy Eng; Aurelio Matamoros; Wai Chin Foo; Abhineet Uppal; Christopher Scally; Paul Mansfield; Melissa Taggart; Kanwal P. Raghav; Michael J. Overman; Keith Fournier
• Keywords: Mucinous Tumor; Clinical endpoint

Importance Appendiceal adenocarcinoma is a rare tumor, and given the inherent difficulties in performing prospective trials such disease, there are currently minimal high-quality data to guide treatment decisions, highlighting need for more preclinical clinical investigation this disease. Objective To prospectively evaluate effectiveness of fluoropyrimidine-based systemic chemotherapy patients with inoperable low-grade mucinous appendiceal adenocarcinoma. Design, Setting, Participants This open-label randomized crossover trial recruited at single tertiary care comprehensive cancer center from September 2013 January 2021. The collection cutoff was May 2022. Enrollment up 30 planned. Eligible had histological evidence metastatic adenocarcinoma, radiographic imaging demonstrating presence peritoneal carcinomatosis were not considered candidates complete cytoreductive surgery. Key exclusion criteria concurrent or recent investigational therapy, bowel obstruction, use total parenteral nutrition. Data analyzed November 2021 Interventions Patients either 6 months observation followed by chemotherapy, initial observation. Main Outcomes Measures primary end point percentage difference tumor growth groups. secondary points included patient-reported outcomes periods, objective response rate, rate complications, differences overall survival (OS). Results A 24 enrolled, median (range) age 63 (38 82) years, equal proportion men women (eg, 12 [50%]); all ECOG performance status 0 1. 11 receive first, 13 first. Most (15 [63%]) treated fluorouracil capecitabine as agent; 3 (13%) received doublet (leucovorin calcium [folinic acid], fluorouracil, oxaliplatin folinic acid, irinotecan hydrochloride), bevacizumab added cytotoxic 5 (21%). Fifteen available during periods. Tumor while receiving increased 8.4% (95% CI, 1.5% 15.3%) baseline but significantly different than (4.0%; 95% −0.1% 8.0%; P = .26). Of 18 who any none an (14 [77.8%] stable disease; 4 [22.2%] progressive disease). Median OS 53.2 (8.1 95.5) months, no significant between observation-first group (76.0 [8.6 95.5] months) treatment-first (53.2 [8.1 64.1] months; hazard ratio, 0.64; 0.16-2.55; .48). Patient-reported quality-of-life metrics identified that treatment, experienced worse fatigue (mean [SD] score, 18.5 [18.6] vs 28.9 [21.3]; .02), peripheral neuropathy 6.67 [12.28] 38.89 [34.88]; .01), financial difficulty 8.9 [15.2] [33.0]; .001) compared Conclusions Relevance In did derive benefit fluorouracil-based responses, when delayed chemotherapy. Trial Registration ClinicalTrials.gov Identifier: NCT01946854