肿瘤细胞治疗研究
英文原题：Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas.
对于部分高危儿童肉瘤患者，常规治疗后的单倍体HSCT巩固治疗似乎具有一定意义，但对大多数患者而言并非如此。有必要评估其作为后续体液或细胞免疫治疗基础的应用前景。
原发性播散性或转移性复发性肉瘤患儿的预后仍然很差，尽管包括大剂量化疗在内的常规治疗已得到强化。由于单倍体相合造血干细胞移植（haplo-HSCT）通过介导移植物抗白血病效应在血液系统恶性肿瘤治疗中有效，我们也在儿童肉瘤中评估了这种方法。
接受单倍体HSCT作为临床试验一部分的骨尤文肉瘤或软组织肉瘤患者，分别使用CD3+或TCRα/β+和CD19+去除，评估了治疗的可行性和生存情况。
我们识别出15例原发性播散性疾病患者和14例转移性复发患者，他们接受了单倍体相合供者移植以改善预后。3年无事件生存（EFS）率为18.1%，主要由疾病复发决定。生存取决于移植前治疗的反应（完全缓解或非常好的部分缓解患者的3年EFS率为36.4%）。然而，没有一例转移性复发患者能够被挽救。
BACKGROUND: Prognosis of children with primary disseminated or metastatic relapsed sarcomas remains dismal despite intensification of conventional therapies including high-dose chemotherapy. Since haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is effective in the treatment of hematological malignancies by mediating a graft versus leukemia effect, we evaluated this approach in pediatric sarcomas as well. METHODS: Patients with bone Ewing sarcoma or soft tissue sarcoma who received haplo-HSCT as part of clinical trials using CD3+ or TCRα/β+ and CD19+ depletion respectively were evaluated regarding feasibility of treatment and survival. RESULTS: We identified 15 patients with primary disseminated disease and 14 with metastatic relapse who were transplanted from a haploidentical donor to improve prognosis. Three-year event-free survival (EFS) was 18,1% and predominantly determined by disease relapse. Survival depended on response to pre-transplant therapy (3y-EFS of patients in complete or very good partial response: 36,4%). However, no patient with metastatic relapse could be rescued. CONCLUSION: Haplo-HSCT for consolidation after conventional therapy seems to be of interest for some, but not for the majority of patients with high-risk pediatric sarcomas. Evaluation of its future use as basis for subsequent humoral or cellular immunotherapies is necessary.
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