azacitidine / Generic mfg. - LARVOL DELTA

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia. (PubMed, N Engl J Med) - P3 | "The incidence of febrile neutropenia was higher in the venetoclax-azacitidine group than in the control group. (Funded by AbbVie and Genentech; VIALE-A ClinicalTrials.gov number, NCT02993523.)."

Journal • P3 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia

Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed NPM1-Mutated or KMT2A-Rearranged AML. (PubMed, J Clin Oncol) - P1/2 | "In older adults newly diagnosed with NPM1m or KMT2Ar AML, the combination of azacitidine, venetoclax, and revumenib was able to be safely administered with high rates of CR and clinical activity."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • KMT2A • NPM1

Long-term results from the AGILE study of azacitidine plus ivosidenib vs placebo in newly diagnosed IDH1-mutated AML. (PubMed, Blood Adv) - P3 | "These long-term efficacy and safety results confirm the benefit of ivosidenib-azacitidine in this challenging-to-treat population and support its use as a standard of care with the longest reported survival benefit for intensive chemotherapy-ineligible patients with IDH1-mutated AML. ClinicalTrials.gov registration ID: NCT03173248."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • IDH1

OPTI-AML: PROSPECTIVE RANDOMIZED PHASE 2 TRIAL OF 28 VERSUS 14 DAY SCHEDULE OF VENETOCLAX AND AZACITIDINE FOR 2 CYCLES IN NEWLY DIAGNOSED GENOMICALLY AGNOSTIC ACUTE MYELOID LEUKEMIA PATIENTS ≥60 YEARS (EHA 2026) - P1/2 | "Our study demonstrates the challenges in delivering continuous AV28 per label, however AV14 appears to not be equivalent to AV28 for all genomic subgroups given the heterogeneous nature of AML. Development of aza/ven-based triplet regimens warrants further study to optimize ven duration in molecularly defined subsets."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • IDH1 • IDH2 • KRAS • NPM1 • TP53

Long-Term Outcomes of Azacitidine, Venetoclax and Gilteritinib in Newly Diagnosed FLT3-Mutated AML. (PubMed, Blood Adv) - P1/2 | "Randomized studies comparing this regimen to standard of care approaches are warranted. This trial is registered at www.clinicaltrials.gov #NCT04140487."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Transplantation • FLT3

Efficacy and safety of risk-stratified chemotherapy with azacitidine for children with myeloid leukaemia with Down syndrome: protocol for a multicentre phase II trial (JCCG AML-D24). (PubMed, BMJ Open) - "The results of this study will be disseminated through publication in peer-reviewed journals and presentation at national and international scientific conferences. jRCTs041250031."

Clinical protocol • Journal • P2 data • Acute Myelogenous Leukemia • Developmental Disorders • Genetic Disorders • Hematological Malignancies • Leukemia • Oncology • Pediatrics • GATA1

Cutaneous involvement by T-Lymphoblastic lymphoma with loss of immaturity markers in a patient treated for acute myeloid leukemia (EADV 2026) - "First-line azacitidine plus venetoclax achieved complete marrow response after six cycles...Following the first cycle of vincristina and prednisone, disease progression was confirmed, with the T-lymphoblastic lymphoma now demonstrating peripheral expression and hepatic infiltration...Cutaneous T-LBL may lose immaturity markers, mimicking mature T-cell lymphoma. In hematologic patients with new skin eruptions, biopsy with attention to CD4/CD8 co-expression and CD99 is essential to identify precursor T-cell neoplasms when CD1a and TdT are absent."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Lymphoblastic Lymphoma • Lymphoma • Peripheral T-cell Lymphoma • Skin Cancer • T Cell Non-Hodgkin Lymphoma • CD1a • CD4 • CD52 • CD8 • CD99 • IL2RA • ISG20 • MME

Drug-Induced Sweet Syndrome: A Systematic Review of Triggers, Clinical Features, and Treatment Outcomes (EADV 2026) - "The most frequently implicated agents were azathioprine (7.2%), G-CSF (6.8%), ATRA (5.9%), azacitidine (5.1%), trimethoprim–sulfamethoxazole (4.2%), and COVID-19 vaccines (1.3%).The median time to onset was 10.6 days after drug initiation. Three distinct clinical phenotypes of DISS are identified: (1) gastro-intestinal and inflammatory, (2) pulmonary and (3) localized mucocutaneous. Early drug withdrawal and corticosteroid therapy remain key to favorable outcomes."

Clinical • Review • Dermatology • Infectious Disease • Inflammatory Bowel Disease • Musculoskeletal Pain • Novel Coronavirus Disease • Pruritus • Vasculitis

Unreported Chemotherapy-Induced Rash: Expanding the Spectrum of Cutaneous Toxicities (EADV 2026) - "Case Presentation Case 1 – A 63 year old female with Acute Myeloid Leukemia was started on Injection Azacytidine after which patient presented with erythematous plaques all over the body and underlying sepsis...She was a known case of Carcinoma of Breast and was started on adjuvant Abemaciclib...Case 3- A 75 year old male with periampullary adenocarcinoma was given 2 cycles of chemotherapy with gemcitabine and capecitabine...Despite the benefits, they often provoke side effects on the skin which impair quality of life in patients and require discontinuation of therapy. Timely diagnosis of such rashes are very important for best outcome."

Acute Myelogenous Leukemia • Ampulla of Vater Adenocarcinoma • Breast Cancer • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Septic Shock • Solid Tumor

Facial erythema nodosum: a rare adverse event of Azacitidine in a patient with acute myeloid leukemia (EADV 2026) - "This case highlights the importance of recognizing Azacitidine as a potential trigger for erythema nodosum. Dermatologists should remain vigilant for atypical presentations of EN, such as facial involvement, when associated with medications."

Adverse events • Clinical • Acute Myelogenous Leukemia • Dermatology • Hematological Malignancies • Infectious Disease • Leukemia • Vasculitis

Azacitidine in MDS/AML: A Population Pharmacokinetic Model to Explain Variability (ESMO 2026) - No abstract available

Clinical • PK/PD data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome • Oncology

Preclinical Evaluation of 5-Azacitidine in Combination with Cisplatin Across Distinct Germ Cell Tumor Histologies (ESMO 2026) - No abstract available

Combination therapy • Preclinical • Germ Cell Tumors • Oncology

TET1 and TET3 are predominantly involved in 5-Azacytidine-driven oxidative DNA demethylation in HAP1 cells (ESMO 2026) - No abstract available

Epigenetic controller • Oncology

A Phase II Clinical Study of Liposomal Mitoxantrone Combined with Azacitidine in Relapsed/Refractory Angioimmunoblastic T-Cell Lymphoma (ESMO 2026) - No abstract available

Clinical • P2 data • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

DNA methylation status at diagnosis in MDS predicts response to azacitidine. (PubMed, J Clin Invest) - "Epigenetic signatures at distal genomic elements provide superior predictive power for AZA response compared to promoter-centric or transcriptional analyses. These findings establish a robust framework for personalized treatment strategies in MDS."

Journal • Tumor mutational burden • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TMB

Unveiling the Complete Biosynthetic Pathway of Azacitidine: A Rare 1,3,5-Triazine Nucleoside Derived From GTP. (PubMed, Angew Chem Int Ed Engl) - "Subsequent decarboxylation by AzaB/AzaC, phosphoribosyl transfer by AzaD, and dephosphorylation by AzaG together with other endogenous hydrolases yield azacitidine. This work uncovers a distinct biosynthetic logic for triazine-containing nucleosides and provides a molecular basis for the synthetic biology-driven production of azacitidine."

Journal • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Phase I trial of a novel first-in-class drug ONC201 as a post-transplant maintenance for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) (ASH 2025) - P1 | "GVHD prophylaxis included tacrolimus and methotrexate with or withoutantithymocyte globulin. ONC201 was well tolerated with no observed DLT and a manageable safety profile. The AEprofile was not different from AEs expected in a post-transplant population. Rates of cytopeniaswere low."

Clinical • P1 data • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Liver Failure • Myelodysplastic Syndrome • Neutropenia • Thrombocytopenia • Transplantation • ASXL1 • BCOR • DRD2 • STAG2 • TP53 • U2AF1

Effects of Arsenic Trioxide in MRL/lpr Mice: Disease-Context Support from Human Transcriptomic and DNA Methylation Datasets. (PubMed, Biol Trace Elem Res) - "The second further evaluated these findings using C57BL/6 controls and MRL/lpr mice treated with ATO, 5-Azacytidine (5-Aza), or their combination. ATO ameliorates lupus-like immune and hepatorenal injury in MRL/lpr mice, accompanied by DNMT1/SOCS1-related transcriptional changes and reduced JAK2/STAT3/IL-17A-related inflammatory transcription. Public human transcriptomic and DNA methylation data support the disease relevance of this regulatory context."

Journal • Preclinical • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Renal Disease • Systemic Lupus Erythematosus • DNMT1 • IL17A • JAK2 • SOCS1 • TET2 • TLR4

Efficacy and Safety of Venetoclax Combined with Azacitidine versus Azacitidine Monotherapy in the Treatment of Higher-Risk Myelodysplastic Syndromes (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi) - "The VEN combined with AZA regimen not only significantly improves treatment response rates in patients with HR-MDS but also effectively extends patient survival, with a favorable safety profile and overall manageable adverse reactions."

Clinical • Journal • Monotherapy • Retrospective data • Allergy • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Nephrology • Neutropenia • Renal Disease

Novel Insights into Hypoxia-Driven Transcriptomic and Epigenetic Landscapes in Grade 3 Meningioma (EANO 2026) - "Findings were cross-validated using public meningioma datasets, an Indian meningioma patient cohort and cell line via RT-qPCR, and azacytidine-based demethylation assay. Transcriptomic analysis under hypoxia revealed induction of a canonical HIF1A-driven transcriptional program promoting glycolytic and angiogenic pathways while suppressing DNA repair and replication in meningioma... This study presents the first integrated transcriptomic-epigenomic landscape of hypoxia in grade 3 meningioma, uncovering regulatory networks and candidate biomarkers with prognostic and therapeutic potential. The author acknowledges the Ministry of Education, Government of India, for the Institutional Research Fellowship."

Brain Cancer • Meningioma • Oncology • Solid Tumor • BRCA1 • CA9 • CDH2 • E2F1 • HIF1A • IGFBP3 • KLF4 • LRP1B • NDRG1 • RTN4IP1 • TP53 • VIM

PPARGC1A as an Epigenetic Biomarker of Differentiation in Differentiated Thyroid Cancer (ETA 2026) - "Similarly, thyroid cancer redifferentiation models, including treatments with LY294002 (PI3K inhibitor), PD325901 (ERK/MAPK inhibitor), Trichostatin A (histone deacetylase inhibitor) and 5-azacytidine (DNA methyltransferase inhibitor), also resulted in an increased PPARGC1A expression alongside the TDS, reinforcing the link between PPARGC1A and differentiation state. In conclusion, we have identified PPARGC1A as an epigenetic factor with a great potential as a biomarker of differentiation in DTC. Understanding its role may provide new insights into tumour dedifferentiation and open new avenues for therapeutic strategies in advanced thyroid carcinomas."

Biomarker • Oncology • Solid Tumor • Thyroid Gland Carcinoma • PPARGC1A

Therapy-related acute myeloid leukemia with 24-month latency after CD19 CAR-T cell therapy in relapsed/refractory diffuse large B-cell lymphoma: a case report. (PubMed, Front Med (Lausanne)) - "After first-line R-CHOP chemotherapy and subsequent relapse, he received two courses of CD19 CAR-T cell therapy (June 2020 and February 2021) with sintilimab maintenance...The patient received azacitidine plus venetoclax, achieving partial remission, followed by child-to-parent haploidentical allogeneic hematopoietic stem cell transplantation...While causality cannot be established from a single case, these findings are consistent with the hypothesis that prolonged genotoxic stress and inflammatory cytokine exposure may promote clonal selection of TP53-mutated hematopoietic cells. This report underscores the necessity of extended hematologic surveillance beyond the first 12 months post-CAR-T infusion, baseline CHIP screening prior to therapy, and systematic longitudinal genomic profiling in future cases to validate these observations."

IO biomarker • Journal • Acute Myelogenous Leukemia • B Cell Lymphoma • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Novel Coronavirus Disease • Oncology • Pneumonia • Respiratory Diseases • Transplantation • CD34 • KIT • TP53

Triptolide sensitizes cancer cells to nucleoside DNA methyltransferase inhibitors through inhibition of DCTPP1-mediated cell-intrinsic resistance. (PubMed, Nat Commun) - "However, while DNA methyltransferase inhibitors (DNMTi) such as decitabine and azacitidine are effective in treating myelodysplatic syndrome/leukemia, they have had limited utility for the majority of other cancers. We show that high DCTPP1 expression mediates cell-intrinsic resistance to nucleoside DNMTi, and that triptolide and its analogs could overcome this resistance. These findings nominate combining DNMTi with triptolide or its analogs as a rational cancer therapeutic strategy."

Journal • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Ophthalmology • DCTPP1

Gilteritinib plus azacitidine in patients with newly diagnosed FLT3 mutation-positive acute myeloid leukemia who were ineligible for intensive chemotherapy: final results of the phase III LACEWING study. (PubMed, Haematologica) - "Corresponding two-year OS rates were 18.8%, 12.9%, and 4.5%. Overall response rates were 70.2% versus 36.8% (GIL+AZA vs AZA, nominal p."

Journal • P3 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

Venetoclax-Based Regimens in Newly Diagnosed FLT3-Mutated Older or Unfit Acute Myeloid Leukemia: A Systematic Review and Meta-Analysis of Comparative and Single-Arm Outcomes. (PubMed, Hematol Oncol) - "The randomized non-venetoclax LACEWING trial showed higher CRc with gilteritinib + AZA than AZA (58.1% vs. 26.5%; HR for OS 0.916; p = 0.753), illustrating that improved remission does not guarantee survival benefit...Triplet therapy produces the strongest remission-depth and MRD signals but has not shown superiority over VEN-HMA in a randomized frontline comparison. On current evidence, triplets are a promising biologically rational strategy requiring direct randomized validation against the contemporary VEN-HMA standard."

Clinical • Journal • Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • FLT3