异基因造血干细胞移植治疗IV期儿童神经母细胞瘤

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

摘要:

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。

Abstract:

ObjectiveAt 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.MethodsA 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. ResultsThe 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 0.5×109/L 10 days after transplantation. The patient′s platelet count recovered to more than 20×109/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.ConclusionsHaploid HLA-matched allogeneic hematopoietic stem cell transplantation from parent donor was an alternative, safe and effective treatment for children with stage IV neuroblastoma.