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目的观察造血干细胞移植治疗儿童血液肿瘤及其他难治性血液病的临床疗效。方法自2007年4月至2009年12月,我们共对53例血液病患儿实施了造血干细胞移植治疗,其中自体移植15例,异基因移植38例,观察其临床疗效和并发症。结果 15例患者行自体外周血造血干细胞移植后造血功能均能顺利重建,无严重并发症发生。异基因移植患儿除1例发生颅内出血早期死亡外,其余均顺利达到造血重建,但其中急性移植物抗宿主病发生率为18.4%,慢性移植物抗宿主病发生率2.6%;15例患儿于移植后0～24个月分别死于颅内出血、感染和疾病复发或进展;移植后6个月至2年存活率65%。结论造血干细胞移植是治疗儿童血液肿瘤及其他难治性血液病的有效方法,可明显改善预后;临床可根据患者原发疾病诊断、供者情况及家庭经济条件选择移植方式。
Objective To observe the clinical efficacy of hematopoietic stem cell transplantation in the treatment of hematological malignancies and other refractory hematological diseases in children. Methods From April 2007 to December 2009, a total of 53 children with hematological diseases underwent hematopoietic stem cell transplantation. Among them, 15 were autotransplanted and allogeneic were transplanted. The clinical efficacy and complications were observed. Results 15 cases of patients with autologous peripheral blood stem cell transplantation hematopoietic function can be successfully reconstructed, no serious complications. In allogeneic transplantation children except 1 case of early death of intracranial hemorrhage, the rest were successfully achieved hematopoietic reconstitution, but the incidence of acute graft-versus-host disease was 18.4%, the incidence of chronic graft-versus-host disease was 2.6%; 15 patients Children died of intracranial hemorrhage 0-24 months after transplantation, infection and disease recurrence or progression; 6 months after transplantation 2-year survival rate of 65%. Conclusion Hematopoietic stem cell transplantation is an effective method to treat hematological malignancies and other refractory hematological diseases in children, which can significantly improve the prognosis. The transplantation can be selected according to the diagnosis of primary diseases, the status of donors and the family economic conditions.