摘要
目的对以CEM(卡铂+依托泊苷+马法兰)为预处理方案的自体外周血造血干细胞移植术治疗20例Ⅳ期神经母细胞瘤患儿的疗效及不良反应进行评价。方法 2007年5月-2009年11月我科收治的Ⅳ期神经母细胞瘤患儿20例,中位年龄4.3岁(1.8～8.7岁),中位体重15.5 kg(9～22.5 kg);原发灶4例为纵隔,16例为肾上腺。14例患儿存在骨髓转移。5例经强化疗、手术减积治疗后仍存在残留病灶,为带瘤移植。预处理方案为CEM方案。结果移植后中位随访时间为17.35个月(2～36个月),1例失访,带瘤移植组(n=5)中3例死亡,1例颅内复发放疗后仍存活,1例无疾病进展存活。移植前完全缓解组(n=15)随访14例,4例死亡,8例无疾病进展存活,2例复发仍存活。回输的CD34+细胞中位数为5.346(1.54～10.3)×106/kg,全部患儿移植后均获得造血重建,在骨髓空巢期均出现发热,4例出现败血症,致病菌分别为少酸链球菌、近平滑念珠菌、大肠埃希菌、表皮葡萄球菌感染,经抗感染治疗后痊愈。髓外毒性包括:18例Ⅱ度肝功能损害,16例Ⅰ度口腔黏膜炎,13例Ⅰ度腹泻,5例Ⅱ度腹泻,2例Ⅰ度心脏不良反应。无移植相关性死亡。结论自体外周血造血干细胞移植术对移植前达到完全缓解的Ⅳ期神经母细胞瘤患儿有较好疗效,对部分缓解期患儿可提高缓解率,不良反应可逆,耐受性可。
Objective To investigate the clinical effects and toxicity of patients treated with CEM conditioning regimen combined with autologous peripheral blood stem cell transplantation in high-risk stage IV neuroblastoma of childhood. Methods Twenty patients with high-risk neuroblastoma were treated in our hospital from May of 2007 to November of 2009. The median age was 4.3 years old. Primary sites of the tumors included mediastinum （ n = 4 ）, and adrenal gland （ n = 16 ）. Fourteen patients had bone-marrow metastases. Fifteen patients got CR （ complete remission） while 5 patients had tumor residual disease after intensive induction therapy with 7 cycles and resection of the primary tumor before ASCT. All the 20 patients received autologous peripheral blood stem cell transplantation. The conditioning regimen consisted of melphalan, etoposide and carboplatin. Results With a median follow-up of 17.35 months, one of the patients were lost to follow-up. In the subgroup of patients （ n = 5 ） who had tumor residual disease before ASCT, three patients died and 1 patient suffered tumor recurrence ; in the subgroup of patients got CR （ n = 14）, 4 patients died, 8 patients live without recurrence and 2 patients suffered recurrence but still alive. All the patients recovered from bone marrow suppression, the non-hematologic toxicity included liver function damage,oral mucositis, diarrhea and cardiac toxicity. All the 20 patients had febrile neutropenia and 4 had septicemia. Conclusion For patients who had CR before transplantation, high-dose chemotherapy combined with autologous peripheral stem cell transplantation achieved good outcome in patients with high risk stage IV neuroblastoma. The outcome of patients with tumor residual disease could be improved by ASCT. Transplantation-related toxicities were tolerable.
出处
《中国小儿血液与肿瘤杂志》
CAS
2011年第4期164-168,共5页
Journal of China Pediatric Blood and Cancer
关键词
神经母细胞瘤
自体外周血干细胞移植
诱导化疗
儿童
Neuroblastoma
Autologous peripheral blood stem cell transplantation
Induction chemotherapy
Childhood
