Chemotherapy combined with apatinib for the treatment of desmoplastic small round cell tumors: A case report

J Cancer Res Ther. 2020 Sep;16(5):1177-1181. doi: 10.4103/jcrt.JCRT_589_20.


Desmoplastic small round cell tumor (DSRCT) is a type of soft-tissue sarcoma with poor prognosis. Current treatments include multidisciplinary treatment options such as surgery, chemotherapy, and radiotherapy. Apatinib is an oral, small-molecule, anti-tumor, angiogenesis-targeted drug, which acts mainly on the intracellular binding site of vascular endothelial growth factor receptor-2. In this study, we administered apatinib in combination with chemotherapy to achieve good disease control. This is a 31-year-old male who presented with upper abdominal pain, nausea, and anorexia for over a month. Imaging revealed multiple solid masses and ascites in the liver and abdominal cavity. He was diagnosed as having cholangiocarcinoma with metastasis to the liver, both lungs, bone, and multiple lymph nodes in the neck, abdominal and pelvic cavity, retroperitoneum, and palpitate angle, based on a percutaneous biopsy of the liver and an abdominal mass, and other examinations. Computed tomography revealed disease progression after two cycles of gemcitabine combined with nedaplatin chemotherapy. Next-generation sequencing detection based on the Illumina high-throughput sequencing platform suggested EWSR1 exon7- Wilms tumor 1 exon8 fusion. The pathology was verified and diagnosed as DSRCT. The chemotherapy regimen was changed to cyclophosphamide, epirubicin, vincristine, and oral apatinib for two cycles. The lesions were mostly reduced, and partial response was evaluated. This case is the first report of the efficacy of apatinib combined with systemic chemotherapy in the treatment of DSRCT, which can become an alternative treatment for this disease.

Keywords: Apatinib; chemotherapy; desmoplastic small round cell tumor.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Cyclophosphamide / administration & dosage
  • Deoxycytidine / administration & dosage
  • Deoxycytidine / analogs & derivatives
  • Desmoplastic Small Round Cell Tumor / drug therapy*
  • Desmoplastic Small Round Cell Tumor / genetics
  • Desmoplastic Small Round Cell Tumor / pathology
  • Epirubicin / administration & dosage
  • Gemcitabine
  • Humans
  • Male
  • Neoplasm Metastasis
  • Oncogene Proteins, Fusion / genetics
  • Pyridines / administration & dosage
  • RNA-Binding Protein EWS / genetics*
  • Treatment Outcome
  • Vincristine / administration & dosage
  • WT1 Proteins / genetics*


  • EWSR1 protein, human
  • Oncogene Proteins, Fusion
  • Pyridines
  • RNA-Binding Protein EWS
  • WT1 Proteins
  • WT1 protein, human
  • Deoxycytidine
  • Epirubicin
  • Vincristine
  • apatinib
  • Cyclophosphamide
  • Gemcitabine