Key result
Apatinib at 500 mg/d effectively reduced tumor mass size by 40% and diminished ascites in a 32-year-old man with metastatic desmoplastic small round cell tumor.
Why the study?
Does apatinib reduce tumor size and ascites in a patient with metastatic desmoplastic small round cell tumor?
Case Report (n=1)
No
Does apatinib reduce tumor size and ascites in a patient with metastatic desmoplastic small round cell tumor?
Apatinib demonstrated efficacy in reducing tumor burden and ascites in a single patient with metastatic desmoplastic small round cell tumor, providing a potential targeted therapy option.
Single-case apatinib response suggests potential activity in metastatic desmoplastic small round cell tumor; leaves open confirmation in prospective trials.
BACKGROUND: Desmoplastic 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 PRESENTATION: A 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. CONCLUSION: It is the first report that apatinib is effective on DSRCT. This report may provide an additional option for the treatment of metastatic DSRCT.
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Shi et al. (2018) conducted a case report in Desmoplastic small round cell tumor (DSRCT) (n=1). Apatinib was evaluated on Tumor size and ascites reduction. Apatinib at 500 mg/d effectively reduced tumor mass size by 40% and diminished ascites in a 32-year-old man with metastatic desmoplastic small round cell tumor.
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