异基因造血干细胞移植治疗IV期儿童神经母细胞瘤

目的：儿童IV期神经母细胞瘤生存期短，且目前尚无有效治疗措施。 该文通过异基因造血干细胞移植治疗IV期儿童神经母细胞瘤来评价其安全性及疗效。方法：对1例7岁IV期神经母细胞瘤患儿进行异基因造血干细胞移植治疗，供者为患儿母亲，HLA配型半相合，采用氟达拉滨，马法兰为预处理方案，G-CSF动员的外周血和骨髓联合移植。结果植入成功，移植后＋10 d中性粒细胞>0.5×109/L，＋11 d血小板>20×109/L。移植后+8 d出现II度肠道急性移植物抗宿主反应，治疗后缓解。随访210 d健康生存。结论： 对IV期儿童神经母细胞瘤患者，以父母为供体的半相合移植是一种可选择的、安全的、有效的挽救性治疗措施之一。

Treatment of stage IV neuroblastoma with allogeneic hematopoietic stem cell transplantation in children

OBJECTIVE: At present there is no effective therapeutic approach for stage IV neuroblastoma. We report our experience with allogenic hematopoietic stem cell transplantation as a means of treating this disorder in one child. METHODS: A 7-year-old boy with stage IV neuroblastoma received allogenetc hematopoietic stem cell transplantation. The donor was his mother who was haploid HLA-matched to the patient. Conditioning regimen consisted of fludarabin and melphalan. Stem cells were collected from peripheral blood and bone marrow of the donor. RESULTS: The patient achieved hematopoietic reconstruction and was converted to full donor chimerism according to short tandem repeat sequence-polymerase chain reaction detection. The patient's neutrophil count recovered to more than 0.5 x 10(9)/L 10 days after transplantation. The patient's platelet count recovered to more than 20 x 10(9)/L 11 days after transplantation. Acute graft versus host disease occurred 8 days after transplantation and was improved after treatment. The patient survived in a 210-day-follow-up. CONCLUSIONS: Haploid HLA-matched allogeneic hematopoietic stem cell transplantation from parent donor was an alternative, safe and effective treatment for children with stage IV neuroblastoma.