decitabine / Generic mfg. - LARVOL DELTA

Dosing decitabine and venetoclax for terminal differentiation to improve outcomes in TP53 mutant MDS and AML. (ASCO 2025) - P2 | "Venetoclax (Ven) added to the hypomethylating agents (HMA) of Decitabine or Azacitidine is the current standard of care for elderly patients with AML and is frequently used in high-risk MDS (HR-MDS). In this cohort, of elderly patients with poor risk TP53 mutated MDS and AML the use of a non-cytotoxic dosing schedule of Decitabine and Ven resulted in over half the patients achieving a CR and transfusion independence. The median OS of 11.3 months compares favorably to currently approved cytotoxic dosing of HMA/Ven."

Acute Myelogenous Leukemia • Myelodysplastic Syndrome • TP53

Epigenetic agents plus anti-PD-1 reshapes tumor microenvironment and restores antitumor efficacy in Hodgkin lymphoma. (PubMed, Blood) - P2 | "In this study, we evaluated the efficacy and safety of a triplet regimen consisting of the histone deacetylase inhibitor chidamide, decitabine and anti-PD-1 camrelizumab (CDP) in 52 patients with relapsed/refractory cHL who had previously received DP therapy (NCT04233294)...The classical CD30+ HRS-like cells interacted with the abundant immunosuppressive IL21+CD4+ T helper cells, forming a positive feedback loop that supported their survival...CDP treatment promoted the activation of diverse tumor-reactive CD8+ T cells and suppressed the proliferation of IL21+CD4+ T cells by inhibiting STAT1/3 signaling, thereby alleviating their immunosuppressive effects. These findings provide insights into the cHL microenvironment that contributes to anti-PD-1 resistance and highlight the therapeutic effectiveness of dual epi-immunotherapy in overcoming immunotherapy resistance."

Biomarker • Journal • Tumor microenvironment • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Oncology • CD4 • CD8 • IL21 • STAT1 • TNFRSF8

Transcriptomic immune subtyping and connectivity mapping nominate DNA methyltransferase inhibitors for immunedepleted melanoma. (PubMed, Melanoma Res) - "After aggregation and filtering, DNA methyltransferase (DNMT) inhibitors showed the most consistent positive pattern; fdcyd, azacitidine, and RG-108, but not decitabine, were robust to sample-type sensitivity analysis. Across both folds, all seven canonical genes replicated and all four DNMT inhibitors retained above-median ranks, but the cold-elevated signature module was markedly less stable than the hot-elevated module (25.7 vs. 88.7% overlap). This suggestive pattern converges with a phase II trial of azacitidine/carboplatin priming before anti-programmed death-ligand 1 rechallenge in ICB-resistant melanoma, supporting prospective evaluation."

Journal • Cutaneous Melanoma • Melanoma • Oncology • Solid Tumor • CD3D • CD8 • CXCL10 • CXCL9 • GZMB • PRF1

NSD2 Expression Remodels DNA Methylation, Creating a Targetable Epigenetic Dependency in t(4;14) Multiple Myeloma (IMS 2026) - "Cell sensitivity to decitabine (DAC) and the DNMT1 inhibitor GSK-3685032 were evaluated via flow cytometry (Annexin V and live/dead staining)... NSD2-mediated increases in H3K36me2 promote DNAm via recruitment of the DNMT3A/B PWWP-domains, creating a distinct epigenetic state in t(4;14) MM. This high-methylation state creates a dependency that can be exploited by DNA hypomethylating agents; inhibiting DNAm in t(4;14) leads to ERV de-repression, triggering viral defense pathways (IFN signaling) and apoptosis. Overall, NSD2 creates an epigenetic dependency in t(4;14) MM that can be therapeutically exploited with DNA hypomethylating agents."

Epigenetic controller • Hematological Malignancies • Multiple Myeloma • ANXA5 • DNMT3A • NSD2

Daratumumab combining with venetoclax, three-day multi-frequency decitabine, vincristine, dexamethasone (VDVD) as the salvage treatment of relapsed/refractory T-cell acute lymphoblastic leukemia. (PubMed, Haematologica) - No abstract available

Journal • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma

Venetoclax plus decitabine as a bridge to allogeneic haematopoietic stem-cell transplantation in older patients with acute myeloid leukaemia (VEN-DEC GITMO): final report of a multicentre, single-arm, phase 2 trial. (PubMed, Lancet Haematol) - P2 | "Venetoclax plus decitabine induction can significantly enhance the feasibility of allogeneic HSCT in older patients with acute myeloid leukaemia who are deemed fit for transplantation."

Journal • P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Novel Coronavirus Disease • Oncology • Pneumonia • Respiratory Diseases • Septic Shock • Thrombocytopenia • Transplantation

Treatment of Higher-Risk Myelodysplastic Syndrome With High-Dose Chemotherapy Followed by Lenalidomide and Azacitidine: A 4-Year Survival Update (SOHO 2026) - "Standard therapies include the hypomethylating agents azacitidine and decitabine...We offered our patients induction chemotherapy including cytarabine 3 g every week for 8 consecutive weeks followed by azacitadine 75 mg/m2 per day for 5 consecutive days per 28-day cycle, concomitant with lenalidomide 10 mg/day continuously... The combination of induction chemotherapy followed by hypomethylating agents plus immunomodulators seems to be a good choice in low income countries in treatment of higher-risk MDS patients."

Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Single-cell proteogenomics analysis of AML samples treated with decitabine and venetoclax reveals divergent treatment escape mechanisms (AACR 2024) - "Most importantly, we report that divergent phenotypic transitions, from stem-like toward more differentiated phenotypes (monocytic or erythroid), were frequently observed in AML relapse following Dec+Ven therapy. Close monitoring of phenotypic alterations during HMA+Ven treatment may facilitate the precise identification of AML patients predisposed to relapse."

Genomic analysis • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BCL2 • BCL2L1 • KRAS • MCL1 • NRAS • TP53

Olutasidenib in post-venetoclax patients with mIDH1 AML. (ASCO 2023) - P1/2 | "VEN was used with azacytidine (AZA) in 8 patients, after AZA (1), and with decitabine (5), cytarabine +/- idarubicin (5), and dinaciclib (2). Olutasidenib induced durable remissions in patients with mIDH1 R/R AML, including those failing prior treatment with a venetoclax-based regimen. Clinical trial information: NCT02719574."

Clinical • Acute Myelogenous Leukemia • Hematological Disorders • IDH1

Real-world outcomes of venetoclax plus hypomethylating agents in unfit acute myeloid leukaemia: Results of the GIMEMA AML2320 trial. (PubMed, Br J Haematol) - P, P3 | "The Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) AML2320 is a prospective, multicentre, observational study (NCT04589728) including 193 newly diagnosed unfit AML patients receiving VEN plus azacitidine or decitabine between November 2020 and December 2021. The GIMEMA AML2320 trial confirmed the efficacy of VEN/HMAs in a prospective, real-world unfit population. Early remission was associated with improved outcomes."

Clinical • Journal • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Venetoclax and Azacitidine Therapy in Acute Myeloid Leukemia Patients With Severe Renal Impairment: A Retrospective Cohort Propensity Score–Matched Analysis (SOHO 2026) - " Using the TriNetX Global Network, we identified adults with AML who received VEN with azacitidine or decitabine (initiated within 14 days). In this large PSM analysis, AML patients with severe CKD/end-stage renal disease treated with VEN/HMA demonstrated comparable OS, TLS, and sepsis rates to those without severe CKD. These real-world findings align with pharmacokinetic data indicating that no dose adjustment is required, supporting the feasibility of VEN/HMA in this underrepresented population. AKI surveillance remains important given the observed trend toward higher incidence."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Venetoclax and Decitabine vs Intensive Chemotherapy as Induction for Young Patients with Newly Diagnosed AML. (PubMed, Blood) - P3 | "Patients aged 18-59 years eligible for intensive chemotherapy were randomized 1:1 to receive VEN-DEC or IA-12 (idarubicin and cytarabine). In conclusion, VEN-DEC demonstrated non-inferior response rates with superior safety over IA-12 in young AML patients. The trial was registered at ClinicalTrials.gov as #NCT05177731."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Thrombocytopenia • RUNX1 • RUNX1T1

Outcomes of Consolidative Allogeneic Transplantation After Venetoclax Plus Hypomethylating Agent Induction in Acute Myeloid Leukemia: A Single-Center Retrospective Study (SOHO 2026) - "Induction consisted of VEN with 5-day decitabine (DEC) (VEN7/14-DEC5; n = 16, 66.7%) or 7-day azacitidine (VEN7/14-AZA7; n = 8, 33.3%), with a median of 2 cycles (range, 1–4) before allo-HSCT...One patient received ponatinib maintenance... In our cohort, allo-HSCT after VEN+HMA bridging demonstrated favorable outcomes despite a substantial proportion of patients being MRD-positive prior to transplantation. Transplantrelated mortality and graft-versus-host disease remain the key challenges. CR: complete response, CRi: CR with incomplete hematologic recovery, ECOG: Eastern Cooperative Oncology Group, ELN: European LeukemiaNet, GVHD: graft-versus-host disease, MAC: myeloablative conditioning, MFC: multiparameter flow cytometry, MLFS: morphologic leukemia-free state, MRD: minimal residual disease, MSD: matched sibling donors, RIC: reduced-intensity conditioning."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

[VIRTUAL] Efficacy and Safety of Sabatolimab (MBG453) in Combination with Hypomethylating Agents (HMAs) in Patients with Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (HR-MDS): Updated Results from a Phase 1b Study (ASH 2020) - P1b | "Study Design and This is a phase Ib, open-label, multicenter, dose-escalation study of sabatolimab + HMA (decitabine [Dec] or azacitidine [Aza]) in patients (pts) with AML or HR-MDS (NCT03066648). Sabatolimab + HMA is well tolerated in pts with AML and HR-MDS and continues to show promising antileukemic activity and emerging durability. These results support TIM-3 as a potential therapeutic target and provide a basis for further development of sabatolimab + HMA in pts with AML or higher-risk MDS. Co-senior authors Uma Borate and Andrew H. Wei contributed equally to the work."

Clinical • Combination therapy • P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Endocrine Disorders • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Rheumatology • Septic Shock • Thrombocytopenia • Transplantation • HAVCR2

Venetoclax With Hypomethylating Agents in Newly Diagnosed AML Unfit for Intensive Chemotherapy (clinicaltrials.gov) - P4 | N=24 | Completed | Sponsor: Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh

New P4 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Choice of hypomethylating agent for newly diagnosed TP53-mutant acute myeloid Leukemia: a COMMAND registry study. (PubMed, Leukemia) - "Although azacitidine (AZA) and decitabine (DEC) demonstrate comparable efficacy in AML, prior data suggest that DEC may induce deeper TP53 mutation clearance and higher response rates; however, direct comparisons in TP53-mutant (TP53-MT) AML are lacking. We conducted a large multicenter retrospective analysis to compare outcomes between DEC- and AZA-based induction, including combinations with venetoclax (VEN)...No significant pairwise differences were observed between regimens. These findings from a large multicenter cohort suggest that AZA- and DEC-based induction yield comparable survival outcomes in TP53-MT AML."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • TP53

Synergistic epigenetic modulation and ferroptosis induction via codelivery of Decitabine and Sorafenib for potent anti-tumor therapy. (PubMed, Drug Deliv) - "Mechanistic studies confirmed that Dac potentiates Sor-induced ferroptosis through epigenetic modulation. Thus, Exo&PM/D + S represents a promising nanotherapeutic strategy that synergizes epigenetic modulation with ferroptosis induction for enhanced anti-tumor efficacy."

Journal • Neuroblastoma • Oncology • Solid Tumor

Prospective Comparison of Outcomes with Azacitidine and Decitabine in AML Patients Ineligible for Intensive Chemotherapy. (PubMed, Blood) - No abstract available

Journal • Acute Myelogenous Leukemia

A phase II study of venetoclax (VEN) in combination with 10-day decitabine (DEC) in older/unfit pts with newly diagnosed (ND) or pts with relapsed/refractory (R/R) acute myeloid leukemia (AML), or high-risk myelodysplastic syndrome (HR-MDS). (ASCO 2024) - P2 | "DEC10-VEN demonstrated expected safety and efficacy in a particularly high-risk cohort of ND and R/R pts, with no evidence that 10-days of DEC provides improved responses over standard 5-days DEC."

Clinical • Combination therapy • P2 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • ASXL1

TARGETED AGENTS COMBINED WITH CHOP COMPARED WITH CHOP AS THE FIRST-LINE THERAPY FOR PERIPHERAL T-CELL LYMPHOMA: PRELIMINARY RESULTS FROM A PHASE 2 GUIDANCE-03 TRIAL (ICML 2023) - P2 | "Standard CHOP (Cyclophosphamide, doxorubicin, vincristine, and prednisolone) chemotherapy is still the most widely used regimen for front-line management...X (i.e., targeted agent) was added from the second to sixth cycles as following, intravenous decitabine 10 mg/m2 on day -5 to -1 if with TP53 mutation. Subcutaneous azacytidine 100 mg on day −7 to −1 if with TET2/KMT2D mutation. Oral chidamide 20 mg on day1, 4, 8, 11 if with CREBBP/EP300 mutation. Oral lenalidomide 25 mg on day 1–10 if without above mutations... Preliminary analysis showed targeted agents combined with CHOP was effective and safe compared with standard CHOP in PTCL. Therapeutic strategy specifically towards molecular features may change current PTCL management in front-line setting."

Clinical • P2 data • Hematological Malignancies • Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • CREBBP • EP300 • KMT2D • TET2 • TP53

Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN) (ASH 2024) - P2/3 | "Background : Azacitidine (AZA) and decitabine (DEC) are parenteral DNA methyltransferase inhibitors (DNMTis) approved for the treatment of patients with MDS and acute myeloid leukemia...Median age was 72 years (range, 26–87), 35% (n=31) of patients were female, prior treatments included DNMTis (9% [n=8]), luspatercept (3% [n=3]), lenalidomide (3% [n=3]), and erythropoiesis‑stimulating agents (3% [n=2])...Based on the results of the phase 1 trial, 140/20 mg AZA/CED was selected as the RP2D. Enrollment for the phase 2 randomized, crossover (oral vs SC) trial is ongoing and combination dosing is in preparation."

Clinical • P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia

Clinical Outcomes of Adults With Core Binding Factor Acute Myeloid Leukemia (CBF‑AML): A Single Center Experience (SOHO 2023) - "Other regimens included: cladribine, idarubicin and cytarabine (CLIA, n=1), fludarabine, cytarabine and gemtuzumab ozogamicin (FLAG-GO, n=1), idarubicin and cytarabine (3+7 regimen, n=1) and decitabine, venetoclax and GO (n=1). Although CBF-AML is considered a favorable risk AML, outcomes for relapsed disease remains poor. Incorporation of GO and CD117 tyrosine kinase inhibitors (i.e. dasatinib) into induction and consolidation regimens may mitigate the relapse risk and improves long-term outcomes."

Clinical • Clinical data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KIT

Notable Efficacy of Co-Treatment with FHD-286, a Dual BRG1/BRM ATP-Ase Inhibitor, and Menin or BET Inhibitor, Decitabine or Venetoclax Against AML with MLL-r or Mutant NPM1 (ASH 2023) - "BRG1 (SMARCA4) and BRM (SMARCA2) are the core ATPase within the multi-protein, ATP-dependent, chromatin remodeling BAF complexes that regulate gene transcription. Finally, co-treatment with FHD-286 and OTX015 or SNDX-5613 (oral gavage) was significantly more effective than each drug alone in reducing the AML burden and overall survival of mice engrafted with a separate PDX model of AML cells with mtNPM1 and FLT3-ITD, without significant toxicity. These findings demonstrate the pre-clinical efficacy of FHD-286-based rational combinations and underscore their promise against AML with MLL1r or mtNPM1."

Clinical • IO biomarker • Acute Myelogenous Leukemia • BCL2 • BRD4 • CASP3 • CD123 • CD33 • CD99 • CDK4 • CDKN1A • CEBPA • CLEC12A • FLT3 • HEXIM1 • IL3RA • ITGAM • MCL1 • MEF2C • MYC • NPM1 • PBX3 • PLK1 • SMARCA2 • SMARCA4

Phase I/II Study of Quizartinib, Venetoclax, and Decitabine Triple Combination in FLT3-ITD Mutated AML (ASH 2023) - "These patients have a median overall survival (OS) of 9.9 months when treated with the standard of care regimen (azacitidine and venetoclax). The combination of DAC + VEN + Quiz demonstrated activity in heavily pretreated and prior FLT3i-exposed (including 78% with prior gilteritinib exposure) R/R FLT3-ITDm pts, with a CRc rate of 68% and a median OS of 7.1 months. In the frontline setting, all pts achieved CRc with no early mortality, median count recovery of 40 days, and median OS not reached. The study continues to accrue, and updated results will be reported at the meeting."

P1/2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Gastrointestinal Disorder • Infectious Disease • Neutropenia • Pneumonia • Respiratory Diseases • Septic Shock • FLT3

Efficacy and Safety of Venetoclax Combined With Hypomethylating Agents in Acute Myeloid Leukemia and Myelodysplastic Syndromes: A Systematic Review and Meta-Analysis (SOHO 2026) - "Hypomethylating agents (HMAs), such as azacitidine and decitabine, are standard therapies; however, response rates remain suboptimal, and resistance is common. Venetoclax combined with HMAs demonstrated improved response rates in AML and MDS, compared with historical HMA monotherapy, particularly in newly diagnosed disease. However, increased hematologic toxicity warrants careful monitoring. The limited number of randomized trials highlights the need for further high-quality studies to confirm these findings and guide clinical practice."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology