azacitidine / Generic mfg. - LARVOL DELTA

Phase 1/2, multi-center, open-label clinical study to evaluate the safety, pharmacokinetics, and efficacy of Zefamenib combined with chemotherapy or targeted Agents in patients with Acute Myeloid Leukemia (ASH 2025) - P1/2 | "Menin inhibitor revumenib combined with venetoclax and azacitidine have shown complete remission (CR) rate of 88.4% in newly diagnosed AML patients harboring these aberrations...Cohort A and D will enroll untreated AML patients, who will receive zefamenib combined with standard 7+3 chemotherapy for induction and zefamenib combined with high dose cytarabine for consolidation...This study will be the first to clarify the feasibility of zefamenib with intensive chemotherapy or targeted agents in the frontline treatment of AML patients or the efficacy and safety of zefamenib with targeted agents in R/R AML patients harboring NPM1 mutation or KMT2A/NUP98 rearrangements. Results of this study will provide ideas and evidence to improve the prognosis of Chinese AML patients with these genetic aberrations."

Clinical • P1/2 data • PK/PD data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • KMT2A • MEIS1 • NPM1 • NUP98

PyramIDH: A phase 3 study of ivosidenib monotherapy or azacitidine monotherapy in patients with mutant isocitrate dehydrogenase 1 myelodysplastic syndromes who have not received prior hypomethylating agent therapy (ASH 2025) - P1, P3 | "As of July 15, 2025, 34 sites are open for enrollment in 9 countries. In total, 19 patients have been prescreened, 4 have been screened, and 3 patients have been enrolled."

Clinical • Monotherapy • P3 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • IDH1

Comparing the efficacy and safety of the ABC-14 regimen (Azacitidine, Venetoclax, and Chidamide) with traditional "3+7" intensive induction regimen or AB-14 regimen (Venetoclax Combined with Azacitidine) in newly diagnosed AML: Study protocol for a prospective, multicenter, randomized, open-label clinical trial (ASH 2025) - P2 | "To provide a alternative treatment for induction therapy of newly diagnosed AML indiscriminately. Trial registration ClinicalTrials.gov NCT06451861, Registered on 2024.06.11, https://clinicaltrials.gov/study/NCT06451861 Keywords Acute myeloid leukemia, unfit-AML, fit-AML, induction therapy, Protocol"

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3 • MCL1 • RUNX1 • TP53

Ivosidenib combined with venetoclax and azacitidine for the treatment of newly diagnosed IDH1-mutant Acute Myeloid Leukemia: A prospective, two cohorts, multicenter study (ASH 2025) - "Previous data showed that the multi-drug combination regimen of IVO demonstrates good tolerability and efficacy. This study will be the first to clarify the feasibility of multi-drug regimens in the treatment of IDH1m AML patients in China, providing ideas and evidence to improve the prognosis of Chinese IDH1m AML patients."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • IDH1

Real-world outcomes associated with azacitidine and venetoclax in Myelodysplastic Syndromes (ASH 2025) - "Conclusion : Treatment with azacitidine and venetoclax in patients with higher-risk MDS may be helpful to achieve disease control quickly providing benefit in those intending to proceed to SCT. In our population, azacitidine and venetoclax did not provide long term disease control, especially in TP53-mutated disease."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Aplastic Anemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Septic Shock • Thrombocytopenia • TP53

Venetoclax-based salvage therapy achieves high remission rates after 7+3 or low-dose cytarabine (ASH 2025) - "Introduction: In the Brazilian public health system (SUS), salvage chemotherapy for fit patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) after standard 7+3 induction (7 days of cytarabine plus 3 days of an anthracycline) is usually FLAG-IDA (fludarabine, cytarabine, granulocyte colony-stimulating factor [G-CSF], and idarubicin) or MEC (mitoxantrone, etoposide, and cytarabine)...Although the venetoclax–cytarabine (VEN-ARAC) or venetoclax–azacitidine (VEN-AZA) combination is approved as first-line therapy for unfit patients, it is generally not available in SUS... In this real-world study, VEN-based salvage therapy induced rapid and high CR rates after 7+3 or LDAC, with a favorable safety profile, and may serve as a less-toxic bridge to allo-HSCT."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Azole-adjusted venetoclax-based regimens in unfit AML patients: A real-world brazilian experience from a resource-limited setting (ASH 2025) - "After 2020, unfit patients were treated with Venetoclax-based regimens (n=54), either combined with Azacitidine 75 mg/m²/day SC for 7 days (AZA-VEN) or with low-dose Cytarabine 20 mg/m²/day SC for 10 days (LoDAC-VEN). All patients received, after ramp-up, azole-adjusted dosing of Venetoclax: 100 mg when combined with Voriconazole or 200 mg with Fluconazole... In this real-world study, Venetoclax-based regimens with azole-adjusted dosing showed better outcomes in unfit AML patients compared to LoDAC or BSC, reinforcing the applicability of such regimens in resource-limited settings. While overall survival in our cohort was lower than reported in pivotal trials, likely due to infectious complications and limited access to supportive care, response rates were comparable. These results support broader incorporation of this approach across diverse healthcare systems."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Real-world characteristics, treatment modifications, and outcomes for patients with Acute Myeloid Leukemia (AML) receiving hypomethylating agents (HMA) + venetoclax (VEN) as first-line treatment (1L) (ASH 2025) - "Background: Older patients with acute myeloid leukemia (AML), those with multiple comorbidities, and/or poor performance status are generally considered ineligible for intense chemotherapy and are often treated with combination therapy of hypomethylating agents (HMAs) (azacitidine or decitabine) and venetoclax (VEN) as the standard of care. This real-world study showed that 1L combination of HMA+VEN was effective in managing AML patients that are ineligible for intense chemotherapy. However, treatment modifications due to cytopenia were common. The longer observed rwOS is likely reflective of a younger, high-functioning patient population with favorable ELN risk profile, subsequent therapies used (including stem cell transplant), and/or improvements in dose optimization in clinical practice."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia

Utilization patterns of venetoclax-based regimens for AML in a community based setting & representative populational analyses (ASH 2025) - "In first line setting, 22 patients (52.3%) received Venetoclax with azacitidine, 17 patients 40.1% received decitabine, 2 patients (4.8%) with cladribine and cytarabine, and 2 patients (4.8%) received Venetoclax alone...6 patients (40%) received azacitidine with venetcloax, 6 patients (40%) received decitabine, 1 patient (6.7%) received cytarabine, 1 patient (6.7%) received enasidenib, and one patient (6.7%) received nelarabine, cyclophosphamide, and cytarabine... The median age at diagnosis was 64 years (range 21-86). 47% patients were female gender. 24.5% of patients were Black or African-American, 71.4% were White, 1% were Asian, and 3.1% were more than one race."

Clinical • Acute Myelogenous Leukemia • Febrile Neutropenia • Hepatology • Liver Failure • Neutropenia • Renal Disease • CEBPA • DDX41 • NPM1 • TP53

Validation of the ELN2024 less-intensive prognostic stratification in newly diagnosed AML patients receiving venetoclax-azacitidine: A real-world study from China (ASH 2025) - "In this real-world Chinese cohort, ELN 2024 Less-Intensive stratification clearly separated survival outcomes and outperformed ELN 2022 in VA-treated AML. MRD negativity remained a robust predictor of favorable RFS, and PTPN11 mutations were associated with early relapse. Larger multicenter studies are warranted."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • PTPN11

Administration of chemoterapy at home for "unfit" hematological patients: A safe and cost-effective way. (ASH 2025) - "Materials and During 2022 we provide identification of patients suffering from Myelodysplastic Syndrome, Multiple Myeloma, and Chronic Lymphatic Leukemia requiring chemotherapy and among these the number of patients defined as UNFIT.We activated two projects of home sub cutaneous or oral chemotherapy as follow: PROJECT N° 1:DOMICILIATION OF CHEMOTHERAPY PER ORAL WITH OBLIGATION OF AIFA CARD for patients affected by Multiple Myeloma and Chronic lymphocytic leukemia PROJECT N° 2: DOMICILIATION OF Sub Cutaneos CHEMOTHERAPY for patients affected by Multiple Myeloma in therapy with VELCADE; - Myelodysplastic syndrome in therapy with VIDAZA. The path for the future is set to effectively respond to the ever-increasing needs of patients due to social and demographic changes. The Hematology Department of the Ragusa Local Health Authority intends to continue in this direction, as being treated at home is very different, psychologically and emotionally, from being..."

Clinical • Cost effectiveness • HEOR • Chronic Lymphocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome

Less is more: Improving outcomes in haemato-oncology with optimal antibiotic use for febrile neutropenia (ASH 2025) - "Management of FN: Rapid administration of piperacillin-tazobactam and amikacin (Piptaz/Ami) for fever (≥38°C) with neutropenia (≤0.5x10 9 /L or expected neutropenia) after taking BCs...Two patients had relapsed AML, 2 newly diagnosed AML (on Venetoclax/Azacytidine and one on palliative Hydroxycarbamide), and 1 post-haploidentical HSCT for lymphoma... Patient population: 49% had AML/MDS; 66% had active disease (presentation or relapse). Treatment modalities: intensive chemotherapy (37%) autologous (37%) or allogeneic HSCT (13%). Median age was 57 years (23-85)."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cerebral Hemorrhage • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Neutropenia • Pneumonia • Renal Disease

Enhanced antileukemic activity of momelotinib in combination with venetoclax and azacitidine compared to gilteritinib-based combination (ASH 2025) - "Despite extensive genomic insights, conventional chemotherapies, specifically anthracyclines and cytarabine , have remained the cornerstone of treatment for the past four decades...In contrast, selective inhibitors of JAK-STAT (ruxolitinib) or FLT3 (gilteritinib or quizartinib) alone failed to demonstrate comparable synergy, highlighting the distinct polypharmacological profile of momelotinib in mediating the cytotoxic response...Notably, while the efficacy of gilteritinib-based regimens was restricted to FLT3-mutant AML, the momelotinib combination demonstrated activity across both FLT3 mutant and FLT3 wild-type contexts. Altogether these preclinical data support clinical evaluation of momelotinib in combination with venetoclax and azacitidine as a potential effective treatment in AML."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • ACVR1 • FLT3 • IRAK1

Post-allogenic hematopoietic stem cell transplant ( Allo-HSCT), hypomethylating-agent maintenance improves survival without increasing GVHD (Graft versus host disease) in AML/MDS( Acute myeloid leukemia/Myelodysplastic syndrome): Updated systematic review and meta-analysis (ASH 2025) - "Hypomethylating agents (HMAs) such as azacitidine and decitabine are increasingly used as post-transplant maintenance, but the magnitude of benefit after the latest clinical reports is unclear. Importantly, HMA maintenance did not increase the risk of grade III–IV acute GVHD (RR = 0.86, 95% CI: 0.38–1.94) or chronic GVHD (RR = 0.94, 95% CI: 0.65–1.34) Conclusions This updated meta-analysis demonstrates that HMA maintenance after allo-HSCT confers clinically meaningful gains in overall and relapse-free survival, driven by both reduced relapse and lower non-relapse mortality, without increasing severe acute or chronic GVHD. These data support integrating HMAs into post-transplant protocols for high-risk AML/MDS."

Retrospective data • Review • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation

Single-center retrospective analysis of venetoclax-based triple-drug combination therapy for relapsed Acute Myeloid Leukemia post allogeneic hematopoietic stem cell transplantation. (ASH 2025) - "Objective: To evaluate the efficacy and safety of venetoclax (VEN), cytarabine, and azacitidine (AZA) combination therapy in relapsed acute myeloid leukemia (AML) patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The VEN-cytarabine-AZA triple-drug regimen is an effective option for relapsed AML after allo-HSCT. However, prolonged neutropenia and thrombocytopenia necessitate vigilant monitoring for severe infections."

Combination therapy • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Pneumonia • Respiratory Diseases • Thrombocytopenia • Transplantation • CBFB • KMT2A • NUP98

Frequency of and finding risk factors for thromboembolism and major bleeding in patients with myelodysplastic syndrome undergoing allogeneic transplantation (ASH 2025) - "Four were on decitabine, 1 was on lenalidomide, and 1 was on azacitidine. Rates of TEE and bleed in MDS patients undergoing allo-SCT were similar and common at 26% and 22%, respectively. Bleeding, but not TEE, seemed to impact survival in line with one other report (Gergi et al. Blood 2024)."

Clinical • Acute Graft versus Host Disease • Cardiovascular • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Thrombosis • Transplantation

Azacitidine (AZA) and donor lymphocyte infusion (DLI) is a viable salvage approach for chimerism-only relapse following allogeneic blood or marrow transplantation (BMT) (ASH 2025) - "All except one received reduced-intensity conditioning, and all received post-transplant cyclophosphamide as GVHD prophylaxis backbone. Our data support AZA-DLI as a feasible strategy for managing post-BMT relapse even in a predominant haplo-BMT population, or when used many years after BMT. Pre-emptive intervention with DLI in a setting of mixed chimerism is associated with superior outcomes, while monitoring for GVHD is warranted."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Transplantation

Myelodysplastic syndrome in Tanzania; The current status of management (ASH 2025) - "Erythropoiesis-stimulating agents, lenalidomide, and cyclosporine were administered to 4.9%, 6.6%, and 8.2% of patients, respectively. Azacitidine, a hypomethylating agent, was offered to only 1.6% of patients...Supportive care remains the mainstay of management, with limited use of disease-specific therapies. This emphasizes the pressing need to strengthen diagnostic infrastructure and make treatment options available to align with global standards and improve the outcomes of MDS patients in low-resource settings."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome

Oral decitabine-cedazuridine versus parenteral hypomethylating agents in treatment-Naïve TP53-mutated Myelodysplastic Syndromes (ASH 2025) - "Pts were grouped by initial therapy: Dec-C or pHMA (azacitidine or decitabine)...Venetoclax was used with HMA in 22 pts (Dec-C, n=4; pHMA, n=18), and those pts were excluded from further analyses...The lower incidence of febrile neutropenia with Dec-C may reflect variability in initial dosing schedules. Although limited by small sample size and retrospective design, these real-world findings can inform clinical use of Dec-C as a potentially effective frontline option for patients with m TP53 -MDS."

Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • TP53

Addition of venetoclax to hypomethylating agent offers limited incremental benefit for patients with treatment-naïve TP53-mutated myelodysplastic syndromes (ASH 2025) - "The HMA used included azacitidine (HMA: n=18; HMA-Ven: n=11), decitabine (HMA: n=15; HMA-Ven: n=7), and decitabine-cedazuridine (HMA: n=21; HMA-Ven: n=4). However, pts with increased blasts and m TP53 appeared to respond better to HMA-Ven. Allo-SCT should be considered for all eligible pts with m TP53 -MDS, as it remains the single independent predictor of improved OS in this subgroup."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • TP53

Lupus-like manifestations in MDS/CMML: Evidence for clonal origin and therapeutic implications (ASH 2025) - "For lupus manifestations, 15 patients received hydroxychloroquine (HCQ) alone and 4 achieved compplete remission (CR); 12 patients received HCQ in combination with oral corticosteroids and 6 achieved CR. Seven patients with active LE received specific treatment for MDS/CMML: 1 with MDS-IB2 received allo-SCT, achieving hematology and LE CR.; 1 with AML received azacytidine (AZA) and venetoclax, achieving hematological and LE CR...Improvement of systemic manifestations following hematologic treatment suggests that clinical features may be clone-dependent. In patients with atypical or treatment-resistant lupus, especially in older adults, men, or those with chilblain CLE, cytopenia, a myeloid neoplasm should be systematically considered, and clone-directed hematologic therapy may be considered."

Chronic Myelomonocytic Leukemia • Cutaneous Lupus Erythematosus • Glomerulonephritis • Hematological Disorders • Hematological Malignancies • Immunology • Inflammatory Arthritis • Leukemia • Lupus • Lupus Nephritis • Myelodysplastic Syndrome • Nephrology • Systemic Lupus Erythematosus • ASXL1 • KRAS • NRAS • SRSF2 • TET2

Molecular correlates of response and survival with venetoclax plus hypomethylating agent in treatment-Naïve high-risk myelodysplastic syndrome (ASH 2025) - "HMA backbones included azacitidine (75 mg/m² IV/SC, days 1–7), IV decitabine (20 mg/m², days 1–5), or oral decitabine-cedazuridine (35 mg/100 mg, days 1–5). However, we observed trends suggesting prognostic relevance for specific mutations ( TP53 , ASXL1 , RAS vs. RUNX1 , TET2 , DNMT3A ). These findings highlight the utility of Ven-HMA as a bridge to transplant, frequent need for dose modification, and underscore the complex interplay of molecular genetics in determining outcomes, which warrants validation in larger studies."

Tumor mutational burden • Hematological Malignancies • Myelodysplastic Syndrome • ASXL1 • DNMT3A • KRAS • NRAS • RUNX1 • SF3B1 • SRSF2 • TET2 • TMB • U2AF1 • ZRSR2

Refractory anemia with ring sideroblasts: Epidemiological survival trends and implications in a SEER population-based study (ASH 2025) - "Available chemotherapies include luspatercept, hypomethylating agents like decitabine and azacitidine in more aggressive or refractory disease, and lenalidomide in del(5q) mutation co-ocurrence. Older patients had notably higher all-cause mortality, likely due to comorbidities as well as increased risk for malignant transformation. More notably, chemotherapy, on average, was associated with worse outcomes, likely reflecting patients with more aggressive disease compared to most untreated counterparts. There is no immediately clear explanation for the modest but significant survival advantage female gender provides."

Clinical • Anemia • Hematological Malignancies • Myelodysplastic Syndrome • SF3B1

Outcomes with azacitidine versus decitabine in AML/MDS (ASH 2025) - "In AML/MDS, both azacitidine and decitabine demonstrate activity. Decitabine achieved higher overall and complete response rates but was associated with serious infections and hematologic adverse events. Azacitidine showed modest effectiveness but better tolerability."

Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Respiratory Diseases • Septic Shock • Thrombocytopenia

Prescribing patterns in MDS/MPN syndromes (ASH 2025) - "Additionally, the prescribing patterns of Hydroxyurea, Hypomethylating Agents (HMA)- Azacitidine/Decitabine and JAK inhibitors Ruxolitinib/Cedazuridine/Pacritinib/Momelotinib/Fedratinib were analyzed in these patients. In this real-world analysis, our data demonstrate that over 90% of patients diagnosed with MDS/MPN overlap syndromes do not receive any treatment in routine clinical practice. The most prescribed medication in this cohort of patients with MDS/MPN was hydroxyurea, followed by hypomethylating medications (decitabine and azacitidine). Being untreated was associated with a significant decrement in survival, with untreated patients having up to twice the risk of death than those treated."

Atypical Chronic Myeloid Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm