目的：复发和难治性神经母细胞瘤（neuroblastoma, NB）疗效差，生存时间短，死亡率高。羟基喜树碱（10-hydroxycamptothecin, HCPT）是中药提取物，对消化道肿瘤、肺癌、卵巢癌等有较好疗效，但它在NB中的治疗反应国内外尚未见报道。该研究探讨HCPT治疗复发和难治性NB的疗效。方法：10例复发和2例难治性NB患儿接受HCPT化疗，其中5例复发患儿接受HCPT单药治疗，余7例患儿接受HCPT联合治疗。HCPT单药治疗用法：7.5 mg/m2，静脉滴注，d 1～14；HCPT联合化疗采用改良新A1与改良B方案交替应用。改良新A1方案：CTX 1 200 mg/m2，d 1；VP16 100 mg/m2，d 1～5；HCPT 5 mg/m2，d 1～3；CDDP 90 mg/m2，d 4。改良B方案：IFO 1.5 g/m2，d 1～5；HCPT 5 mg/m2，d 1～3；CBP 450 mg/m2，d 2，均采用静脉滴注。结果：部分缓解4例（33.3％），疾病稳定8例（66.7％）。有效患者中位缓解时间3.5(2～5)个月。HCPT单药治疗不良反应轻微，HCPT联合化疗的主要不良反应为骨髓抑制及消化道反应，无化疗相关的死亡。结论：HCPT治疗复发和难治性NB有一定的近期疗效，毒性可耐受，远期效果需进一步研究。［中国当代儿科杂志，2009，11（5）：361-363］

OBJECTIVE: The patients with recurrent or refractory neuroblastoma have a very poor prognosis and high mortality. 10-hydroxycamptothecin (HCPT) is a new agent extracted from comptotheca acuminate, a native plant. It has been shown to be very effective in some solid tumors such as gastric and colon cancers, lung cancers and ovary cancers. However, its efficacy in neuroblastoma has not been determined. This study aimed to investigate the therapeutic effects of HCPT in the treatment of recurrent or refractory neuroblastoma in children. METHODS: Ten children with recurrent neuroblastoma and two children with refractory neuroblastoma were treated with HCPT. Of them, 5 children with recurrent neuroblastoma were treated with HCPT alone, and the other 7 patients received combination chemotherapy of HCPT plus other agents. The HCPT alone treatment group was injected with HCPT (7.5 mg/m2 daily) for 14 consecutive days. The combination chemotherapy group was alternately treated with the modified new protocol A1 (cyclophosomide 1 200 mg/m2 on day 1, etoposide 100 mg/m2 on days 1-5, HCPT 5 mg/m2 on days 1-3, cisplatin 90 mg/m2 on day 4) and the modified protocol B (ifosfomide 1.5 g/m2 on days 1-5, HCPT 5 mg/m2 on days 1-3, carboplatin 450 mg/m2 on day 2). RESULTS: Four patients (33.3%) achieved partial remission and 8 patients (66.7%) had stable disease. The median remission duration was 3.5 months (2-5 months). HCPT treatment as a single agent resulted in mild side effects. Myelosuppression and digestive disorders were found as the main adverse events in the combined chemotherapy group. No chemotherapy related deaths were found. CONCLUSIONS: HCPT is safe and effective in the treatment of recurrent or refractory neuroblastoma. The toxicities of HCPT are tolerable. The long-term efficacy of HCPT warrants further research.［Chin J Contemp Pediatr, 2009, 11 (5):361-363］