Shaw P J, Pinkerton C R, Yaniv I
Department of Oncology, Royal Alexandra Hospital for Children, Westmead, Sydney, Australia.
Bone Marrow Transplant. 1996 Dec;18(6):1043-7.
Carboplatin and melphalan are two drugs whose toxicity profile makes them suitable for use in high doses followed by stem cell rescue. We report the use of high-dose carboplatin, with the dose based on glomerular filtration rate (GFR), combined with melphalan followed by autologous stem cell rescue in children with advanced stage or chemoresistant solid tumours. Thirty children were treated. After multiagent induction chemotherapy before BMT, 13 were in CR, five in VGPR, 11 in PR and one had progressive disease. They received melphalan, 180 mg/m2 and carboplatin, followed by autologous stem cell rescue. The dose of carboplatin was varied by GFR rather than fixed by surface area. The dose given ranged from 0.7 to 2.6 g/m2. Haematological and gastrointestinal toxicities were severe. Life-threatening or fatal toxicity was attributable to opportunistic infection in two cases and regimen-related in two cases. Of the 30 patients, 15 are alive and 13 disease-free at 4-36 months post-BMT. This simple two-drug combination has been used as consolidation of initial remission for patients with high-risk tumours. The toxicity is severe but tolerable. Use of a carboplatin dose based on GFR should optimise effectiveness in patients with good renal function and avoid excessive toxicity where renal function is impaired.
卡铂和美法仑是两种药物，其毒性特征使其适用于大剂量使用并随后进行干细胞救援。我们报告了在患有晚期或化疗耐药实体瘤的儿童中使用基于肾小球滤过率（GFR）的大剂量卡铂，联合美法仑，随后进行自体干细胞救援。30名儿童接受了治疗。在骨髓移植前进行多药诱导化疗后，13名处于完全缓解（CR），5名处于非常好的部分缓解（VGPR），11名处于部分缓解（PR），1名病情进展。他们接受了180mg/m²的美法仑和卡铂，随后进行自体干细胞救援。卡铂的剂量根据GFR而变化，而非按体表面积固定。给予的剂量范围为0.7至2.6g/m²。血液学和胃肠道毒性严重。危及生命或致命的毒性在两例中归因于机会性感染，两例中归因于方案相关。30名患者中，15名在骨髓移植后4至36个月存活，13名无疾病。这种简单的两药联合已被用作高危肿瘤患者初始缓解后的巩固治疗。毒性严重但可耐受。基于GFR使用卡铂剂量应能优化肾功能良好患者的疗效，并避免肾功能受损时的过度毒性。
