{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Wang L"],"funding":["National Key Research and Development Program of China"],"pagination":["586"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11852425"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["17(4)"],"pubmed_abstract":["<h4>Purpose</h4>This study aimed to investigate the efficacy and the clinical and molecular predictors of response and survival following venetoclax plus hypomethylating agents (VEN + HMAs) in adult relapsed/refractory acute myeloid leukemia (R/R AML) patients.<h4>Methods</h4>We retrospectively analyzed 197 adult R/R AML patients who received the VEN + HMAs regimen. Molecular profiling was performed using targeted next-generation sequencing (NGS) of 139 genes to explore potential response and survival genetic predictors.<h4>Results</h4>The median treatment cycle was 1 (1-4) cycle. The composite complete remission (CRc) rate, encompassing complete remission (CR) and CR with incomplete hematologic recovery (CRi), was 44.7%, while the overall response rate (ORR) reached 59.9%. With a median follow-up period of 14.0 months (range: 0.7-54.0 months), the 1-year and 2-year overall survival (OS) rates were 55.4% and 40.2%, respectively. Multivariate analyses revealed that mutations in <i>NPM1</i> and <i>SRSF2</i> were significantly associated with improved response rates. Conversely, prior exposure to HMA therapy, early relapse, and the presence of <i>GATA2</i> mutations were linked to lower response rates. Regarding survival outcomes, the <i>CBFB-MYH11</i> fusion gene, as well as mutations in <i>NPM1</i> and <i>IDH1/2</i>, were found to be favorable prognostic factors for OS, whereas mutations in <i>FLT3-ITD</i>, <i>TP53</i>, <i>DNMT3A</i>, and <i>GATA2</i> were associated with worse OS.<h4>Conclusions</h4>The VEN + HMAs regimen demonstrated considerable efficacy in the treatment of R/R AML patients, with both response rates and overall survival being influenced by distinct genetic features. These findings provide valuable insights into optimizing personalized treatment strategies for this challenging patient population."],"journal":["Cancers"],"pubmed_title":["Clinical and Molecular Predictors of Response and Survival Following Venetoclax Plus Hypomethylating Agents in Relapsed/Refractory Acute Myeloid Leukemia: A Single-Center Study in Chinese Patients."],"pmcid":["PMC11852425"],"funding_grant_id":["2021YFC2500302"],"pubmed_authors":["Xu L","Fu Q","Jiang H","Zhang X","Huang X","Jiang Q","Gao H","Wang Y","Tang F","Wang L"],"additional_accession":[]},"is_claimable":false,"name":"Clinical and Molecular Predictors of Response and Survival Following Venetoclax Plus Hypomethylating Agents in Relapsed/Refractory Acute Myeloid Leukemia: A Single-Center Study in Chinese Patients.","description":"<h4>Purpose</h4>This study aimed to investigate the efficacy and the clinical and molecular predictors of response and survival following venetoclax plus hypomethylating agents (VEN + HMAs) in adult relapsed/refractory acute myeloid leukemia (R/R AML) patients.<h4>Methods</h4>We retrospectively analyzed 197 adult R/R AML patients who received the VEN + HMAs regimen. Molecular profiling was performed using targeted next-generation sequencing (NGS) of 139 genes to explore potential response and survival genetic predictors.<h4>Results</h4>The median treatment cycle was 1 (1-4) cycle. The composite complete remission (CRc) rate, encompassing complete remission (CR) and CR with incomplete hematologic recovery (CRi), was 44.7%, while the overall response rate (ORR) reached 59.9%. With a median follow-up period of 14.0 months (range: 0.7-54.0 months), the 1-year and 2-year overall survival (OS) rates were 55.4% and 40.2%, respectively. Multivariate analyses revealed that mutations in <i>NPM1</i> and <i>SRSF2</i> were significantly associated with improved response rates. Conversely, prior exposure to HMA therapy, early relapse, and the presence of <i>GATA2</i> mutations were linked to lower response rates. Regarding survival outcomes, the <i>CBFB-MYH11</i> fusion gene, as well as mutations in <i>NPM1</i> and <i>IDH1/2</i>, were found to be favorable prognostic factors for OS, whereas mutations in <i>FLT3-ITD</i>, <i>TP53</i>, <i>DNMT3A</i>, and <i>GATA2</i> were associated with worse OS.<h4>Conclusions</h4>The VEN + HMAs regimen demonstrated considerable efficacy in the treatment of R/R AML patients, with both response rates and overall survival being influenced by distinct genetic features. These findings provide valuable insights into optimizing personalized treatment strategies for this challenging patient population.","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Feb","modification":"2025-04-03T23:44:24.625Z","creation":"2025-04-03T23:44:24.625Z"},"accession":"S-EPMC11852425","cross_references":{"pubmed":["40002181"],"doi":["10.3390/cancers17040586"]}}