Abstract:
Objective To investigate the efficacy and safety of apatinib in recurrent and progressive desmoid fibromatosis (DF).
Methods From October 2017 to September 2020, all patients who presented with recurrent or progressive DF at West China Hospital, Sichuan University were included in this study and initialy received a daily dose of 250 mg of apatinib. We adjusted the dose based on drug-related toxicity.
Results Seventeen patients showed partial response (45.9%), 18 patients had stable disease (48.6%), and 2 patients had progressive disease (5.4%). The objective response rate was 45.9% and disease control rate was 94.6%. After apatinib treatment, the range of motion (ROM), visual analog scale (VAS) score, Disabilities of the Arm, Shoulder, and Hand (DASH) score, Harris score, and Hospital for Special Surgery (HSS) score of the patients significantly improved compared to those prior to treatment. The most common adverse events were hand-foot syndrome 14/37 (37.8%), gastrointestinal reaction 11/37 (29.7%), and paramenia 8/37 (21.6%).
Conclusions Apatinib was efficacious in the treatment of progressive or recurrent DF with mild adverse events. Thus, it can likely be considered as a novel option for the treatment of DF.