Okada Keiko, Nakano Yoshiko, Yamasaki Kai, Nitani Chika, Fujisaki Hiroyuki, Hara Junichi
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Cancer Med. 2016 Aug;5(8):1947-9. doi: 10.1002/cam4.784. Epub 2016 Jun 5.
Metastatic neuroblastoma is an aggressive malignancy with a poor prognosis. Recent findings have shown that sorafenib decreases cell viability and increases apoptosis in human neuroblastoma cell lines. We report an experience of compassionate use of sorafenib in children with treatment-refractory neuroblastoma. Sorafenib showed transient anti-tumor activity in all four patients without adverse effects. However, progression was observed after a short stabilization phase. While sorafenib showed minimal anti-tumor activity in our patients, it might still be effective in patients with neuroblastoma in an earlier stage.
转移性神经母细胞瘤是一种侵袭性恶性肿瘤,预后较差。最近的研究结果表明,索拉非尼可降低人神经母细胞瘤细胞系的细胞活力并增加细胞凋亡。我们报告了索拉非尼在治疗难治性神经母细胞瘤儿童中的同情用药经验。索拉非尼在所有四名患者中均显示出短暂的抗肿瘤活性,且无不良反应。然而,在短暂的稳定期后观察到疾病进展。虽然索拉非尼在我们的患者中显示出最小的抗肿瘤活性,但它可能对早期神经母细胞瘤患者仍然有效。