azacitidine / Generic mfg. - LARVOL DELTA

Dose-Reduced Venetoclax-Azacitidine in Elderly Acute Myeloid Leukemia: A Real-World Experience (SOHO 2025) - "Four patients required cytoreduction with hydroxyurea ± cytarabine for hyperleukocytosis. In this real-world Latin American cohort, shortened-duration VEN-AZA was feasible and highly effective, reproducing VIALE-A remission rates even in frail elderly patients. Curtailing VEN exposure appeared to lessen infectious complications without jeopardizing remission outcomes. Interpretation is limited by sample size and retrospective design."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2 • NPM1 • TP53

Individualized Acute Myeloid Leukemia Management in an Older Adult Patient With a Mechanical Heart Valve: A Case of Targeted Cytoreduction and Modified Maintenance (SOHO 2025) - "Cytoreduction was initiated with hydroxyurea and a single dose of gemtuzumab ozogamicin. Upon control of white blood cells, azacitidine and venetoclax were started... This specific case demonstrates that, with individualized therapy, it is feasible to achieve long-lasting, durable remission in AML in older patients with significant comorbidities. Gemtuzumab-assisted cytoreduction followed by modified decitabine/venetoclax enabled long-term disease control. A successful outcome for this high-risk patient required careful coordination of anticoagulation and supportive care."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KRAS • NPM1 • SRSF2

New Therapies and Targeted Treatments: MDS/ MPN Overlap Syndromes (SOHO 2025) - "This has led to the use of parenteral azanucleosides — decitabine and azacitidine — and, perhaps most successfully in the United States, the oral agent decitabine/ cedazuridine (Dec-C)...While decitabine led to improved response rates, it failed to show benefit in event-free survival (EFS) or overall survival (OS) over the hydroxyurea arm, 5 raising questions about the role of azanucleosides — at least in MP-CMML — and underscoring the urgent need for disease-specific therapies for patients with MPCMML and other MDS/MPNs...This yielded promising responses, 8 but the arm was closed, as itacitinib was discontinued by the manufacturer...9 Given these findings, in the phase 2 PREACH study in Australia comparing lenzilumab plus azacitidine in untreated, high-risk RAS- mutated CMML, investigators reported a 55% complete response (CR) rate, found to be durable at 18 months...STX-0712 is a cytotoxicity targeting antibody (CyTAC) that links an antibody designed to induce..."

IO biomarker • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • ABL1 • BCR • CCL2 • CCR2 • CSF2 • LILRB4 • SF3B1

PEVENAZA: A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy (clinicaltrials.gov) - P2 | N=164 | Active, not recruiting | Sponsor: Takeda | Trial completion date: Jun 2025 ➔ Sep 2025

Trial completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Real-World Comparison of Hypomethylating Agent-Venetoclax and Intensive Chemotherapy in Elderly Acute Myeloid Leukemia: Survival and Safety Insights From a Global Matched Cohort (SOHO 2025) - " We identified patients aged 65– 75 years with AML treated with either HMA + venetoclax (azacitidine or decitabine + venetoclax) or intensive chemotherapy (cytarabine + daunorubicin or idarubicin, without HMA) from TriNetX. In this large, real-world matched analysis of elderly AML patients, HMA + venetoclax was associated with significantly better overall survival and reduced rates of sepsis, pneumonia, cardiac arrest, and acute kidney injury compared with intensive chemotherapy. Despite increased neutropenia and ventilator use, overall complication and transfusion rates were comparable. These data support the preferential use of HMA + venetoclax as first-line therapy in older AML patients, particularly those at higher risk of treatment-related morbidity."

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

How to Select Intensive vs. Lower Intensity Therapy in Younger Patients (SOHO 2025) - "When combined with hypomethylating agent (HMA) therapies (decitabine and azacitidine) in single-arm clinical trials, rates of response among older patients were remarkable, with a composite remission rate (CCR) of 67% and a reported median overall survival (OS) of 17.5 months...These data would include response rates, MRD clearance, bridging to transplant, and survival outcomes. It is very possible that in the not-too-distant future, HMA-venetoclax will serve as the therapeutic backbone for the majority of treatment options offered to AML patients, regardless of age or functional status."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Oncology • FLT3 • IDH1 • IDH2 • NPM1 • TP53

Moving Beyond Romidepsin: New Strategies for Relapsed/Refractory PTCL (SOHO 2025) - "Current FDA-approved agents include pralatrexate, 2 belinostat, 3 and brentuximab vedotin...In this study, romidepsin plus CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) was compared to CHOP in untreated T-cell lymphomas (TCLs) but failed to show an improvement in progression-free survival (PFS) — the primary endpoint of this study 6 Brentuximab vedotin has the highest response rate of 87% in R/R anaplastic large cell lymphoma (ALCL), 4 but the response rates are lower in other subtypes...7 Duvelisib — a dual PI3K delta/ gamma inhibitor — has shown an overall response rate (ORR) of 48%, a complete response (CR) rate of 33%, and a median duration of response of 7.89 months...Other PI3K inhibitors include tenalisib 10 and linperlisisb, 11 which demonstrate similar results...Inhibitors of the JAK/ STAT pathway like ruxolitinib are showing promising results in PTCL — both as single agents and in combination...The ORACLE study compared 5-azacitidine against..."

Oncology • Peripheral T-cell Lymphoma • CD5 • CD7 • CD70 • EZH2 • GATA3 • IL2RA • KLRD1 • SIRPA • TNFRSF8

Comparative efficacy of venetoclax and hypomethylating agents in acute myeloid leukemia treatment: a meta-analysis of clinical trials and Real-World outcomes. (PubMed, Ann Hematol) - "This meta-analysis, comprising 24 studies, evaluated the efficacy of venetoclax (VEN) in combination with hypomethylating agents (HMAs), including azacitidine (AZA) and decitabine (DEC), in untreated patients with acute myeloid leukemia (AML), comparing outcomes from clinical trials and real-world practice. In real-world studies, the VEN + HMA combination was associated with a significantly higher CRc rate (67%, 95% CI: 48-85%) compared to HMA monotherapy (17%, 95% CI: 13-21%; p < 0.005), although no significant difference in OS was observed between these groups (9.35 vs. 9.38 months; p = 0.964). These findings highlight the need to optimize the implementation of VEN + HMA regimens in clinical practice, as real-world outcomes remain inferior to those reported in clinical trials."

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

From Low Risk to Blast Crisis: A Case for Mastering Hematological Malignancies (SOHO 2025) - "Leukemia was suspected, and he was treated with hydroxyurea for cytoreduction...His intended treatment was azacitidine, planned to begin 1 week after presentation...A thorough review of CBC results and differential at each presentation is key in the early identification of a potential blast cell crisis. Our case demonstrates the importance of a comprehensive understanding of hematologic malignancies and how recognizing the signs and symptoms of disease progression and crises is a skill relevant to all physicians."

Clinical • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Effective Treatment of Peritoneal Myeloid Sarcoma With Decitabine and Venetoclax: A Case Report (SOHO 2025) - "To our knowledge, this is the first reported case of primary peritoneal MS treated with decitabine and venetoclax. Prior studies showed improved overall survival with azacitidine and venetoclax in older, treatment-naïve AML patients and demonstrated symptom resolution in a patient with bladder MS using this regimen. Our patient achieved a durable clinical and radiographic response, highlighting the efficacy of decitabine and venetoclax combination therapy in older patients ineligible for induction chemotherapy with peritoneal MS."

Case report • Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Sarcoma • Solid Tumor • ABL1 • ASXL1 • CD33 • CD34 • FLT3 • TET2

Does the Timing of Response Impact the Outcome of Relapsed/Refractory Acute Myeloid Leukemia Treated with Venetoclax in Combination with Hypomethylating Agents? A Proof of Concept from a Monocentric Observational Study. (PubMed, J Clin Med) - " This prospective single-center study included 33 adult patients with R/R AML treated with VEN plus either azacitidine (AZA) or decitabine (DEC) from 2018 to 2021. The absence of a prognostic disadvantage for late responders supports flexible treatment schedules and suggests that the continuation of therapy may be beneficial even without early blast clearance. Tailored approaches based on performance status and comorbidities are warranted, and future studies should incorporate minimal residual disease (MRD)-based monitoring to refine response assessment."

Journal • Observational data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Transplantation

A Rare Presentation of Acute Myeloid Leukemia Associated With Philadelphia Chromosome (SOHO 2025) - "Hematology-oncology recommended leukapheresis and hydroxyurea for cytoreduction...The patient started azacitidine/venetoclax induction but failed to achieve response with increasing peripheral blasts...He started cladribine, low-dose cytarabine (LDAC), and dasatinib...He continued dasatinib with excellent disease control for 2 years before switching to asciminib due to edema...Our case highlights prolonged remission with TKI monotherapy after LDAC induction, suggesting that patients may achieve sustained disease control without transplantation. Further research is needed to evaluate TKI monotherapy outcomes and identify factors predicting durable response in Ph+ AML."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR

Hypomethylating-Agent Prophylaxis Following Allo-HSCT in Adult Acute Lymphoblastic Leukemia Worsens Survival: Evidence From the TriNetX Network (SOHO 2025) - "In this large propensity-matched cohort, decitabine/azacitidine maintenance failed to lower relapse risk and instead produced a 28% absolute rise in 1-year mortality alongside greater infectious and hematologic toxicity. Routine post-transplant HMA prophylaxis in adult ALL should be reconsidered until validated by prospective trials."

Clinical • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • T Acute Lymphoblastic Leukemia

VEXAS Syndrome: Genetics, Gender Differences, Clinical Insights, Diagnostic Pitfalls, and Emerging Therapies. (PubMed, Int J Mol Sci) - "Azacitidine and decitabine have shown promise in reducing disease burden, but hematopoietic stem cell transplantation (HSCT) remains the only curative treatment, albeit with significant risks. This review provides a comprehensive analysis of VEXAS syndrome, examining its clinical features, differential diagnoses, diagnostic challenges, and treatment approaches, including both pharmacological and non-pharmacological strategies. By enhancing clinical awareness and optimizing therapeutic interventions, this article aims to bridge emerging genetic insights with practical patient management, ultimately improving outcomes for those affected by this complex and often life-threatening disease."

Journal • Review • Bone Marrow Transplantation • Dermatology • Hematological Disorders • Hematological Malignancies • Immunology • Inflammation • Myelodysplastic Syndrome • Oncology • Targeted Protein Degradation • Thrombocytopenia • Transplantation

Personalized Therapy and Real-World Outcomes in Adult Acute Myeloid Leukemia: Lessons From a Nationwide Prospective Cohort in Turkey (SOHO 2025) - P | "Low-intensity therapy—mainly azacitidine or decitabine—was used in 25.9%, with targeted agents (eg, venetoclax) integrated more frequently in the low-intensity group compared with the high-intensity group (19.1% vs 3.4%, respectively; P < 0.001). This large-scale real-world analysis highlights the prognostic impact of ELN risk classification, performance status, and treatment intensity in AML. While intensive chemotherapy remains standard, the addition of targeted therapies to low-intensity regimens offers clinical benefit in selected patients. Real-world registries are essential for refining personalized treatment strategies and improving access to novel therapies and allogeneic transplantation."

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

Macrophage reprogrammer Bexmarilimab Plus Azacitidine in Myelodysplastic Syndrome: PK/PD and biomarker results from the Phase 1/2 BEXMAB Study (ESMO 2025) - No abstract available

Biomarker • P1/2 data • PK/PD data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Outcomes in Acute Myeloid Leukemia With MECOM Rearrangement (MECOMr-AML) Treated With Hypomethylating Agent Plus Venetoclax (SOHO 2025) - "HMA+ven contained azacitidine (74%) and decitabine (26%) for a median of 2 cycles (range, 1-12). MECOMr-AML treated with HMA +ven is associated with poor prognosis particularly with co-occurring −7, though responders have a trend toward improved mOS."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • GATA2 • MECOM • NF1 • NRAS • PTPN11 • RUNX1 • SF3B1 • TP53

Phase II Study of Combination Azacitidine and Entinostat and Nivolumab in Patients With Metastatic Non-Small Cell Lung Cancer (IASLC-WCLC 2025) - P2 | "Further correlative evaluation, including in-depth genomic and transcriptomic analyses, are ongoing to accurately identify patients most likely to benefit from this therapeutic approach. Final integration of these translational studies with clinical outcomes will work to identify the biologic effect of epigenetic therapy in re-shaping the tumor microenvironment to personalize this epigenetic combinatorial approach."

Clinical • Metastases • P2 data • Gene Therapies • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Pulmonary Disease • Solid Tumor

Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: Johns Hopkins All Children's Hospital | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

Efficacy and Safety of Venetoclax Plus Azacitidine for Patients With High-Risk Myelodysplastic Syndromes—A Meta-Analysis (SOHO 2025) - "Context: The current standard of care for high-risk myelodys-plastic syndromes (MDSs) is treatment with hypomethylating agents (HMAs) such as azacitidine or decitabine; however, it is associated with lower rates of response. Venetoclax combined with HMA demonstrates promising efficacy in high-risk MDS, with notable response rates and potential to bridge to stem cell transplantation. However, its use is limited by notable hematologic and infectious toxicities, warranting careful monitoring and tailored supportive care."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • BCL2

PEMAZA: MRD-guided Treatment in NPM1mut AML Patients (clinicaltrials.gov) - P2 | N=26 | Terminated | Sponsor: Technische Universität Dresden | Trial completion date: Dec 2026 ➔ Feb 2025 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Feb 2025; Feasibility

Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • NPM1

Bone marrow vacuolization to curative strategies: Evolving paradigms in VEXAS syndrome management. (PubMed, Curr Res Transl Med) - "This includes infection control, transfusion administration, hypomethylating agents such as azacitidine, which provide the dual benefit of reducing mutant clones alongside inflammation, as well as immunosuppressive drugs, steroids, and Janus Kinase (JAK) inhibitors...Some investigational therapies targeting specific pathways show promise, particularly nucleotide-binding domain, Leucine-rich Repeat-containing family, pyrin domain containing 3 (NLRP3) inflammasome blockers (IL-1β/IL-6 inhibitors) and proteasome inhibitors like bortezomib (Bortezomib), which exploit the proteostasis defects in UBA1-mutated cells. Core obstacles still lie in the absence of a standardized treatment paradigm due to gaps in genotype-phenotype expression and variability, alongside insufficient biomarkers able to guide therapy selection and directed personalized therapeutic interventions. This review highlights the curative strategies, therapeutic challenges, and advancements in VEXAS syndrome,..."

Journal • Review • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Myelodysplastic Syndrome • Oncology • Transplantation • IL1B • NLRP3

Triplet Therapy With Decitabine, Venetoclax, and Quizartinib for FLT3-ITD Mutated AML: A Phase 1/ 2 Study (SOHO 2025) - "Context: Newly diagnosed patients with FLT3-ITD mutated (FLT3m) acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy experience poor outcomes with median overall survival (OS) <1 year with azacitidine+venetoclax...Forty-seven R/R AML patients were heavily pretreated (median 3 [1-5] prior therapies), 85% (39/46) had received ≥1 prior FLT3 inhibitors—with 76% having prior exposure to gilteritinib and 37% having undergone prior ASCT... Decitabine+venetoclax+quizartinib combination showed remarkable responses in the frontline setting; 94% achieved CRc, with median ANC and platelet recovery of 37 and 36 days, respectively. Median OS was not reached."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

Treatment of Higher-Risk Myelodysplastic Syndrome With Induction Chemotherapy Followed by Azacitidine and Lenalidomide: Results of a Phase 2 Study (SOHO 2025) - "Standard therapies include the hypomethylating agents azacitidine and decitabine...Induction chemotherapy included cytarabine 3 g/wk for 8 consecutive weeks, followed by azacitidine 75 mg/m2/day for 5 consecutive days of a 28-day cycle, given concomitantly with lenalidomide 10 mg/day continuously... The sequence of induction chemotherapy followed by hypomethylating agents plus immunomodulators seems to be a good choice in higher-risk MDS patients."

P2 data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Checkpoint immunotherapy is associated with preferential activation of tumor antigen-specific CD4+ T cells in MDS. (PubMed, Blood Neoplasia) - P1 | "Based upon this hypothesis, early phase trials combining immune checkpoint inhibitors (ICIs) with azacitidine in patients with myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) were undertaken, but clinical and immunologic efficacy have proven disappointing...We hypothesized that combining vaccination against the New York esophageal squamous cell carcinoma 1 (NY-ESO-1) tumor antigen with decitabine and an ICI would allow us to understand antigen-specific immune responses in patients with MDS. To test this hypothesis, we developed an investigator-initiated phase 1 trial in transplant-ineligible patients with MDS/low blast count AML incorporating the anti-programmed cell death protein-1 (PD-1) ICI nivolumab...Approaches to augment the number and function of cDC1 populations in myeloid disease might overcome this defect and enhance the efficacy of immunotherapy for patients with MDS. This trial was registered at www.ClinicalTrials.gov as #NCT03358719."

Clinical • IO biomarker • Journal • Acute Myelogenous Leukemia • Esophageal Cancer • Esophageal Squamous Cell Carcinoma • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Squamous Cell Carcinoma • Transplantation • CD4 • CTAG1B