Di Stasi Antonio, Jimenez Antonio M, Minagawa Kentaro, Al-Obaidi Mustafa, Rezvani Katayoun
Stem Cell Transplantation and Cell Therapy Unit, The University of Alabama at Birmingham , Birmingham, AL , USA.
Stem Cell Transplantation and Cell Therapy Unit, Rush University Medical Center , Chicago, IL , USA.
Front Immunol. 2015 Feb 4;6:36. doi: 10.3389/fimmu.2015.00036. eCollection 2015.
We performed a systematic review of data from nine clinical trials of WT1 peptide vaccination in patients with myelodysplastic syndromes and/or acute myeloid leukemia (MDS/AML), published between 2004 and 2012. A total of 51 patients were eligible for analysis. Vaccination with WT1 peptides proved safe and feasible in patients with MDS/AML, in studies from different institutions. Additionally, clinical responses and clinical benefit were observed, with some patients achieving and maintaining remission long-term (more than 8 years). A significant correlation between induction of WT1-specific T cells and normalization/reduction of WT1 mRNA levels and progression-free survival was noted in a number of studies. However, larger studies are warranted to confirm these results. Interestingly, the majority of trials reported the presence of WT1-specific T cells with limited or absent functionality prior to vaccination, which increased in frequency and function after vaccination. In conclusion, WT1 peptide vaccination strategies were safe in this heterogeneous group of patient with MDS/AML. Larger and more homogeneous studies or randomized clinical trials are needed to quantify the contribution of WT1 peptide vaccines to clinical responses and long-term survival.
我们对2004年至2012年间发表的9项关于WT1肽疫苗接种治疗骨髓增生异常综合征和/或急性髓系白血病（MDS/AML）患者的临床试验数据进行了系统评价。共有51例患者符合分析条件。在不同机构开展的研究中，WT1肽疫苗接种在MDS/AML患者中被证明是安全可行的。此外，还观察到了临床反应和临床获益，部分患者实现并长期维持缓解（超过8年）。多项研究指出，WT1特异性T细胞的诱导与WT1 mRNA水平的正常化/降低以及无进展生存期之间存在显著相关性。然而，需要开展更大规模的研究来证实这些结果。有趣的是，大多数试验报告称，接种疫苗前存在功能有限或无功能的WT1特异性T细胞，接种后其频率和功能均有所增加。总之，WT1肽疫苗接种策略在这类异质性MDS/AML患者群体中是安全的。需要开展更大规模、更具同质性的研究或随机临床试验，以量化WT1肽疫苗对临床反应和长期生存的贡献。
