Effective treatment of apatinib in desmoplastic small round cell tumor: a case report and literature review

Abstract

BackgroundDesmoplastic small round cell tumor (DSRCT) is a rare malignant sarcoma with poor prognosis due to lack of effective treatments. Apatinib is a new potent oral small-molecule tyrosine kinase inhibitor, and targets the intracellular domain of vascular endothelial growth factor receptor 2 (VEGFR-2). In this study, we presented a case of intra-abdominal DSRCT which was effectively treated by apatinib.Case presentationA 32-year-old man was admitted due to increasing urination frequency and palpable mass in right lower abdomen for 2 months. The mass was resected and diagnosed DSRCT. The patient refused chemotherapy and radiotherapy,and used Chinese medicine only. Six months after the surgery, the patient re-hospitalized due to growing abdominal mass and ascites. Intraperitoneal cisplatin treatment showed little effect. Apatinib was then recommended. Apatinib revealed outstanding effect on reducing mass size and ascites during 2-month treatment. Apatinib therapy continued for additional 2 months, and the patient was in good condition. The only toxicity was hand-food syndrome, which was controllable and well tolerated.ConclusionIt is the first report that apatinib is effective on DSRCT. This report may provide an additional option for the treatment of metastatic DSRCT.

Phase II trial of VEGFR2 inhibitor apatinib for metastatic sarcoma: focus on efficacy and safety

“…Each patient had at least one measurable extracranial lesion, and responses were evaluated according to RECIST 1.1 37,38 . Based on benefits in previous retrospective studies of apatinib in sarcoma patients 25,27–30,39 , some sarcoma patients with nonmeasurable lesions were also enrolled and evaluated according to RECIST 1.1 37,38 . Nonmeasurable lesions included small lesions (longest diameter < 10 mm, or pathological lymph nodes with 10–15 mm short diameter) as well as truly nonmeasurable lesions.…”

“…Clinical investigations of apatinib in metastatic sarcomas have also shown encouraging results. Seven case reports of the use of apatinib to treat sarcoma have been published, and all seven patients were considered to have partial responses (PR) 22–27 , indicating that apatinib can be effective for treating malignant sarcomas, with manageable AEs. Three retrospective studies of apatinib for the treatment of sarcomas have also been conducted to date, including one by our group 28–30 .…”

Mediastinal desmoplastic small round cell tumor

“…The pleural DSRCT progresses rapidly and can be metastasized to the abdominal and pelvic cavities in the advanced stage, with poor prognosis. [ 17 , 18 ] The male patient in this case visited the hospital complaining of chest tightness, shortness of breath, and swelling pain in chest.…”

Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

“…VEGFR-2 and VEGFA expression was found to be markedly increased in DSRCT tumor sample and in the human DSRCT cell line, JN-DSRCT [78] The use of tyrosine kinase inhibitors (TKI) for VEGF, VEGFR, PDGFRα and other proteins involved in tumoral vascular proliferation has been explored in the clinical scenario. [79,84,85].…”

“…The best response to sorafenib was stable disease and the median progression free survival of of 3.5 and 4 months. On the other hand, as first line treatment, apatinib was used in only one patient [79]. Apatinib is another VEGFR-2 inhibitor with demonstrated activity in gastric and other tumors [90].…”