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BackgroundIn younger, fit patients with acute myeloid leukemia (AML), intensive chemotherapy (IC) followed by consolidation or allogeneic hematopoietic stem cell transplantation (HSCT) is the standard approach. The authors performed a systematic review and meta-analysis to evaluate younger patients with AML treated with hypomethylating agents (HMA) plus venetoclax.MethodsThis systematic review and meta-analysis was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. MEDLINE and the Cochrane Library were systematically searched through February 2026. Studies included AML patients with a median age ResultsEight studies (two randomized controlled trials, two phase 2 trials, and four real-world studies), comprising 429 patients with a mean age of 54 years, were included. The pooled CR/CRi rate was 66% (95% confidence interval [CI], 48%-85%), with an MRD-negative rate of 69% (95% CI, 49%-90%). The pooled 1-year OS was 75% (95% CI, 63%-86%), exceeding Surveillance, Epidemiology, and End Results database cohorts (62%). The 1-year EFS was 59% (95% CI, 53%-65%), with low between-study heterogeneity. Overall, 66% of patients successfully proceeded to HSCT. Meta-regression analyses suggested a trend toward improved EFS and OS in studies using decitabine than azacitidine.ConclusionsIn younger patients with AML, HMA plus venetoclax yielded high response rates, MRD negativity, and a substantial proportion of patients proceeding to HSCT. These findings support HMA/venetoclax as an effective induction strategy in selected younger patients and provide a rationale for prospective randomized trials comparing this approach with IC-based regimens.

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Decitabine shows favorable efficacy in induction and maintenance therapy for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), but its role in pre-transplant conditioning still has not been established. We performed a systematic review and meta-analysis of comparative studies investigating the effects of adding decitabine to conditioning regimens in AML/MDS patients undergoing allogeneic hematopoietic stem cell transplantation. Meta-analysis was conducted with Review Manager 5.4. For time-to-event outcomes, pooled hazard ratios (HRs) were calculated using generic inverse-variance method. For dichotomous data, pooled risk ratios (RRs) were calculated using Mantel-Haenszel method. Fixed-effects model was adopted if there is no significant heterogeneity. Literature search and study selection identify 9 eligible studies, including 2 randomized controlled trials and 7 retrospective comparative studies. Meta-analysis show that addition of decitabine to conditioning regimen is associated with significantly better overall survival (HR 0.61; 95% CI: 0.49-0.76; P < 0.00001), reduced risk of relapse (HR 0.58; 95% CI: 0.46-0.73; P < 0.00001), and better disease-free survival (HR 0.67; 95% CI: 0.55-0.81; P < 0.0001) without increasing non-relapse mortality (HR 0.82; 95% CI: 0.56-1.18; P = 0.28) and grade II-IV acute graft-versus-host disease (RR 0.75; 95% CI: 0.54-1.04; P = 0.08).

Abstract licence: CC BY-NC-ND

BackgroundEpigenetic dysregulation is a key therapeutic target in pediatric hematologic malignancies resistant to conventional chemotherapy, where allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative option. In this setting, hypomethylating agents (HMAs), such as azacitidine (AZA) and decitabine (DAC), are increasingly used as pre-transplant bridging strategies to reduce disease burden and enhance treatment sensitivity. Their integration with HSCT may improve pre-transplant remission rates, prolong complete remission, and increase the likelihood of durable disease control and cure. However, evidence in pediatric patients remains limited. Here, we performed a systematic review and pooled analysis to evaluate the efficacy of pre-HSCT epigenetic therapy in pediatric hematologic malignancies.ResultsA pooled analysis of seven studies of 87 pediatric patients including 51 Myelodysplastic Syndromes (MDS) and 36 Juvenile Myelomonocytic Leukemia (JMML) treated with DAC or AZA prior to HSCT was performed. Statistical analyses used chi-squared or Fisher's exact tests for categorical variables and non-parametric tests for continuous variables, with correction for multiple comparisons. In MDS, Complete remission (CR) was achieved in 64.7% of patients and strongly associated with DAC, translating into improved post-HSCT survival compared to PD (84.2% vs. 42.8%). In JMML, partial remission (PR) and progressive disease (PD) predominated (47.2% and 41.7%, respectively), while CR was rare (11.1%); response was not associated with HMA type or genetic features, although older age correlated with PD. Comparative analysis confirmed superior responses in MDS (higher CR and SD) versus JMML, which showed higher PR and PD rates. PTPN11 mutations were significantly associated with multiple clinical variables, including treatment response.ConclusionsPre-transplant HMA therapy shows variable efficacy in pediatric myeloid malignancies. DAC induces rapid, deep responses in MDS, improving post-HSCT survival. JMML responds less, yet post-transplant survival remains favorable. Higher HMA responsiveness in MDS likely reflects predominant epigenetic dysregulation, whereas JMML is mainly driven by aberrant RAS/MAPK pathway activation. These results emphasize that pre-transplant strategies should be tailored to the specific biological and clinical context of each disease. However, their impact relative to upfront HSCT or conventional chemotherapy remains to be definitively determined.

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