Objective  To evaluate the efficacy and safety of VCD/IE combined with apatinib for patients with advanced Ewing sarcoma, unresponsive to chemotherapy.

  Methods  The clinical data of patients with advanced Ewing sarcoma, who received VCD/IE regimen combined with apatinib in Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, from January 2017 to December 2021, were retrospectively collected. Their clinical data were compared with those of patients who received apatinib as monotherapy during the same period. The primary endpoint was progression-free survival (PFS). The secondary endpoints evaluated were objective response rate (ORR), disease control rate (DCR), overall survival (OS), and adverse events (AE).

  Results  Eleven patients who received the combined regimen met the inclusion criteria of this study. Ten patients managed with apatinib monotherapy were enrolled. The median follow-up time was 19.5 months and 14.5 months, respectively. Most participants were male (72.7% and 70%, respectively), aged (18.1 ±6.7) and (20.8 ±15.7) years old, respectively. The ORR, DCR, mPFS, and mOS of those in the combined regimen were 27.3% (3/11), 81.8% (9/11), 6.4 months, and 10.9 months, respectively. Meanwhile, the ORR, DCR, mPFS, and mOS of those in apatinib monotherapy were 0, 62.5% (5/8), 1.8 months, and 4.1 months, respectively. Patients that underwent the combined regimen experienced grade 3-4 AE, such as chemotherapy-related neutropenia, anemia, thrombocytopenia, and febrile neutropenia. In comparison, patients who underwent apatinib monotherapy experienced grade 1-2 adverse reactions. All adverse reactions were generally tolerable.

  Conclusions  VCD/IE regimen combined with apatinib is superior than apatinib monotherapy in managing patients with advanced Ewing sarcoma unresponsive to chemotherapy; all adverse reactions were tolerable.