异基因造血干细胞移植治疗伴TP53基因异常骨髓增生异常综合征/急性髓系白血病42例疗效分析

OBJECTIVE: TP53-abnormal MDS/acute myeloid leukemia（AML）patients' allogeneic hematopoietic stem cell transplantation（allo-HSCT）treatment's effectiveness and influencing factors should be studied. METHODS: 42 patients with TP53 gene status change MDS/AML who underwent allo-HSCT from 2014.8.1 to 2021.7.31 at the Hematology Hospital of the Chinese Academy of Medical Sciences were the subject of a retrospective analysis. The 42 patients were divided into three groups: the TP53 deletion group（group A）, TP53 mono-alle mutation group（group B）, and TP53 multi-hit group（group C）. The differences in clinical features and prognostic factors after transplantation were analyzed. RESULTS: There were 42 MDS/AML patients, including 21 patients with MDS, and 21 patients with AML. The median follow-up period was 34.0（7.5–75.0）months and the median patient age at the time of transplantation was 41.5（18–63）years old. The total OS was 66.3％（95％ CI 53.4％–82.4％）in 3 years after transplantation, and EFS was 61.0％（95％ CI 47.7％–78.0％）in 3 years. For 3 years after receiving hematopoietic stem cell transplantation, there were no statistically significant differences in 3-year OS and EFS in groups A, B, and C（P≥0.05）. The 3 years OS was 82.5％（95％ CI 63.1％–100.0％）in group A, 60.6％（95％ CI 43.5％–84.4％）in group B, and 57.1％（95％ CI 30.1％–100.0％）in group C. Univariate analysis revealed that the number of co-mutant genes, pre-HSCT treatment, and disease type did not affect prognosis, while age, karyotype, co-mutation, positive blast cell before transplantation, and positive blast cell after transplantation were common prognostic factors for OS and EFS（P<0.1）. MRD levels before transplantation were found to be independent risk factors for OS（P＝0.037, HR＝33.40, 95％ CI 1.24–901.17）in a multivariate analysis. CONCLUSION: Patients with MDS/AML who have TP53 mutations can benefit from allo-HSCT, but patients with complex karyotypes have a worse prognosis. Meanwhile, the final flow cytometry（FCM）monitoring blast cell test before HSCT has a certain guiding significance for prognostic assessment.