Mifamurtide: a review of its use in the treatment of osteosarcoma.
Mifamurtide: a review of its use in the treatment of osteosarcoma.
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DOI：
10.2165/11204910-000000000-00000
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发表时间：
2010-06-01
期刊：
影响因子：
--
通讯作者：
Frampton, James E
中科院分区：
文献类型：
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作者：
Frampton, James E
米法莫肽（脂质体胞壁酰三肽磷脂酰乙醇胺; Mepact）是一种具有抗肿瘤作用的免疫调节剂，似乎通过激活单核细胞和巨噬细胞介导。在欧盟，mifamurtide适用于儿童、青少年和年轻成人，用于治疗肉眼完全手术切除后的高级别、可切除、非转移性骨肉瘤;通过静脉输注联合术后多药化疗给药。在美国，mifamurtide目前是一种研究药物，具有治疗骨肉瘤的孤儿药地位。在一项大型、随机、开放标签、多中心、III期试验中，在三种或四种药物联合化疗（多柔比星、顺铂和高剂量甲氨蝶呤联合或不联合异环磷酰胺）基础上添加辅助（术后）mifamurtide可显著改善新诊断、高级别、非转移性、可切除骨肉瘤患者的总生存期。在入组本试验的一小群转移性疾病患者中，结局模式通常相似。米法莫肽通常耐受性良好;药物给药引起的不良事件包括寒战、发热、头痛、恶心和肌痛。根据现有数据，可以考虑将mifamurtide纳入局部骨肉瘤的治疗方案。
Mifamurtide (liposomal muramyl tripeptide phosphatidyl ethanolamine; Mepact) is an immunomodulator with antitumor effects that appear to be mediated via activation of monocytes and macrophages. In the EU, mifamurtide is indicated in children, adolescents, and young adults for the treatment of high-grade, resectable, non-metastatic osteosarcoma after macroscopically complete surgical resection; it is administered by intravenous infusion in conjunction with postoperative multiagent chemotherapy. In the US, mifamurtide is currently an investigational agent that holds orphan drug status for the treatment of osteosarcoma. In a large, randomized, open-label, multicenter, phase III trial, the addition of adjuvant (postoperative) mifamurtide to three- or four-drug combination chemotherapy (doxorubicin, cisplatin, and high-dose methotrexate with, or without, ifosfamide) was associated with a statistically significant improvement in overall survival in patients with newly diagnosed, high-grade, non-metastatic, resectable osteosarcoma. The pattern of outcome was generally similar in a small cohort of patients with metastatic disease who were enrolled in this trial. Mifamurtide is generally well tolerated; adverse events attributed to administration of the drug include chills, fever, headache, nausea, and myalgias. Based on the available data, mifamurtide can be considered for inclusion in treatment protocols for localized osteosarcoma.