肿瘤细胞治疗研究
英文原题：WT1 and PRAME RNA-loaded dendritic cell vaccine as maintenance therapy in de novo AML after intensive induction chemotherapy.
强化诱导化疗可使>60%的急性髓系白血病（AML）患者达到完全缓解（CR），但对于不适合异基因造血干细胞移植（allo-HSCT）的复发患者，总生存期（OS）较差。
强化诱导化疗可使超过60%的急性髓系白血病（AML）患者达到完全缓解（CR），但对于不适合异基因造血干细胞移植（allo-HSCT）的复发患者，总生存期（OS）较差。口服阿扎胞苷可用于首次缓解的AML维持治疗，但可能伴随显著副作用，应探索毒性更低的策略。20例首次完全缓解（CR1）且不适合allo-HSCT的AML患者接受了FDC101治疗，这是一种自体RNA负载成熟树突状细胞（mDC）疫苗，表达两种白血病相关抗原（LAA）。每剂包含每种抗原2.5-5 × 10 6个mDC，每周给药至第4周，第6周给药，之后每月给药，持续2年研究期。对患者进行安全性和长期生存随访。治疗耐受良好，仅出现轻微且短暂的注射部位反应。20例患者中11例（55%）维持CR，6例复发患者中4例在挽救治疗后达到CR2并接受了allo-HSCT。五年OS为75%（95% CI：50-89），其中≥60岁患者中70%为长期生存者。该DC疫苗维持治疗在CR1的AML患者中耐受良好，并伴随令人鼓舞的5年长期生存。
Intensive induction chemotherapy achieves complete remissions (CR) in >60% of patients with acute myeloid leukemia (AML) but overall survival (OS) is poor for relapsing patients not eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Oral azacytidine may be used as maintenance treatment in AML in first remission, but can be associated with substantial side effects, and less toxic strategies should be explored. Twenty AML patients in first CR (CR1) ineligible for allo-HSCT were treated with FDC101, an autologous RNA-loaded mature dendritic cell (mDC) vaccine expressing two leukemia-associated antigens (LAAs). Each dose consisted of 2.5-5 × 10 6 mDCs per antigen, given weekly until week 4, at week 6, and then monthly, during the 2-year study period. Patients were followed for safety and long-term survival. Treatment was well tolerated, with mild and transient injection site reactions. Eleven of 20 patients (55%) remained in CR, while 4 of 6 relapsing patients achieved CR2 after salvage therapy and underwent allo-HSCT. OS at five years was 75% (95% CI: 50-89), with 70% of patients ≥60 years of age being long-term survivors. Maintenance therapy with this DC vaccine was well tolerated in AML patients in CR1 and was accompanied by encouraging 5-year long-term survival.
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