Allogeneic hematopoietic stem cell transplantation could improve the survival of acute myeloid leukemia patients with ASXL1 mutations.

Abstract	Objective: To discover the function of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in ASXL1-mutated acute myeloid leukemia (AML) patients.Methods: We analyzed the prognostic value of ASXL1 mutations and explored the role of allo-HSCT in 581 AML patients.Results: According to the definition of intermediate- and adverse-risk AML groups in the European Leukemia Net (ELN), ASXL1-mutated patients had shorter OS and DFS than ASXL1-wild-type patients in the intermediate- and adverse-risk AML groups (3-year OS: 47.5% vs. 60.8%, P<0.001; 3-year DFS: 28.5% vs. 48.9%, P<0.001). Among the cytogenetically normal acute myeloid leukemia (CN-AML), differences were found in both OS (47.4% vs.65.2%, P<0.001) and DFS (21.0% vs. 52.1%, P<0.001) between ASXL1-mutated patients and ASXL1 wild-type patients.In the ASXL1-mutated AML cohort, the patients received allo-HSCT had longer 3-year OS (P=0.0005) and 3-year DFS (P<0.0001) than those who did not receive allo-HSCT. Multivariate analysis revealed that ASXL1 mutation was an independent prognostic factor for OS (HR 2.248, 95% CI 1.155-4.375, P=0.017), and allo-HSCT had a positive impact on OS (HR 7.568, 95% CI 3.597-15.92, P<0.001) and DFS (HR 2.611, 95% CI 1.688-4.039, P<0.001) in ASXL1-mutated patients.Conclusion: The results indicate that the presence of ASXL1 mutations is a factor predictive of poor prognosis in AML patients and allo-HSCT could improve the survival of AML patients with ASXL1 mutations.
Authors	Lili Zhou, Jingnan An, Chang Hou, Zixuan Ding, Huiying Qiu, Xiaowen Tang, Aining Sun, Suning Chen, Yang Xu, Tianhui Liu, Depei Wu
Journal	Hematology (Amsterdam, Netherlands) (Hematology) Vol. 26 Issue 1 Pg. 340-347 (Dec 2021) ISSN: 1607-8454 [Electronic] England
PMID	33840380 (Publication Type: Journal Article)
Chemical References	ASXL1 protein, human Repressor Proteins
Topics	Adolescent Adult Aged Child Female Hematopoietic Stem Cell Transplantation Humans Leukemia, Myeloid, Acute (epidemiology, genetics, therapy) Male Middle Aged Mutation Repressor Proteins (genetics) Survival Analysis Transplantation, Homologous Treatment Outcome Young Adult

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