Discussion
DISEASE, TUMOR GROWTH, BOWEL PERFORATION
This randomized crossover trial found that fluoropyrimidine-based chemotherapy was not effective for patients with low-grade mucinous AA, as there was not a single objective response and not a significant difference in tumor growth while during chemotherapy vs observation. Due both to the rarity and heterogeneity of AA, it has been difficult to objectively determine whether systemic chemotherapy is effective in the treatment of this disease. This study represents the first prospective, randomized trial for low-grade mucinous AA to answer this pivotal question, to our knowledge. Moreover, chemotherapy significantly decreased QOL during the chemotherapy period compared with the observation period. Similarly, delaying the start of chemotherapy with a 6-month observation period did not reduce OS nor increase rate of bowel perforation or obstruction.The results from this prospective, randomized trial are consistent with multiple prior retrospective analyses suggesting chemotherapy is ineffective in low-grade AA.To our knowledge there are no reports of objective response from cytotoxic systemic chemotherapy specifically in low-grade AA. It is important to note that this is in contrast to high-grade AA, which is known to be responsive to cytotoxic chemotherapy on the basis of many prior reports.