Department of Gastrointestinal colorectal and anal surgery, China-Japan Union Hospital of Jilin University, Changchun, China.
Department of Pathology, China-Japan Union Hospital of Jilin University, Changchun, China.
BMC Cancer. 2018 Mar 27;18(1):338. doi: 10.1186/s12885-018-4135-x.
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.
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.
It is the first report that apatinib is effective on DSRCT. This report may provide an additional option for the treatment of metastatic DSRCT.
促结缔组织增生性小圆细胞肿瘤（DSRCT）是一种罕见的恶性肉瘤，由于缺乏有效治疗方法，预后较差。阿帕替尼是一种新型强效口服小分子酪氨酸激酶抑制剂，作用于血管内皮生长因子受体 2（VEGFR-2）的细胞内结构域。本研究报告了一例阿帕替尼治疗有效的腹腔内 DSRCT 病例。
一名 32 岁男性因尿频和右下腹可触及肿块 2 个月而入院。肿块切除后诊断为 DSRCT。患者拒绝化疗和放疗，仅使用中药。术后 6 个月，患者因腹部肿块和腹水增大再次住院。腹腔内顺铂治疗效果不佳。随后建议使用阿帕替尼。阿帕替尼治疗 2 个月后，肿块大小和腹水明显减少，疗效显著。阿帕替尼治疗继续进行了 2 个月，患者情况良好。唯一的毒性是手足综合征，可控制且耐受性良好。
这是首例报告阿帕替尼对 DSRCT 有效的病例。本报告可能为转移性 DSRCT 的治疗提供了另一种选择。
