decitabine / Generic mfg. - LARVOL DELTA

Efficacy of Decitabine in Clearance of MRD (clinicaltrials.gov) - P1/2 | N=0 | Withdrawn | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | N=300 ➔ 0 | Suspended ➔ Withdrawn

Enrollment change • Trial withdrawal • Acute Myelogenous Leukemia

Navitoclax, Venetoclax, and Decitabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Previously Treated With Venetoclax (clinicaltrials.gov) - P1 | N=17 | Active, not recruiting | Sponsor: City of Hope Medical Center | Trial completion date: May 2025 ➔ Apr 2026 | Trial primary completion date: May 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Decitabine in Combination With Standard of Care Therapy for the Treatment of Surgically Resectable HPV-Negative Head and Neck Cancer (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Mayo Clinic

New P1 trial • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Retrospective review of relapse after allogeneic stem cell transplant for myeloid malignancy. (ASCO 2025) - "3 received gilteritinib, 1 decitabine and GCSF, and 1 oral azacitidine. Patients with myeloid malignancies who experienced relapse after alloHSCT had poor prognosis. Development of GVHD prior to relapse trended toward improved survival after relapse. In this cohort, maintenance chemotherapy prior increased time to relapse and DLI improved survival after relapse."

Retrospective data • Review • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

Venetoclax duration outcomes analysis in MDS/AML within a community hospital. (ASCO 2025) - "Most received decitabine (81%), had adverse ELN risk (83%), and were de novo AML (66%). In this community hospital cohort, Ven 7 patients had significantly worse 1yrOS vs Ven 28; there was no difference in OS. Our findings, consistent with other retrospective studies, imply shorter Ven durations may be as effective but with minimal difference in hematologic toxicities. A selection bias in prescribing patterns likely exists in this and other retrospective studies."

Clinical • Acute Myelogenous Leukemia • Hematological Disorders • Myelodysplastic Syndrome • Neutropenia • Thrombocytopenia

Real world outcomes of venetoclax combined with hypomethylating agents in acute myeloid leukemia and myelodysplastic syndrome: Insights from a tertiary care centre in Pakistan. (ASCO 2025) - "Indications for first line treatment were advanced age or frailty in 27 (50%) , active infections in 3 (3.9%) and cardiac dysfunction in 2 (3.8%) .Total 44 (81.5%) patients received azacytidine and 10 (18.5%) decitabine. Venetoclax with HMAs represent an effective therapeutic strategy for AML and MDS in the real-world setting. This regimen is associated with promising outcomes and may offer an alternative to traditional treatment regimens for patients in Pakistan and similar settings"

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology

Impact of antecedent myelodysplastic syndrome treatment on outcomes and treatment response in patients with subsequent secondary acute myeloid leukemia. (ASCO 2025) - "Data extracted includes previous treatment for MDS (azacitidine, decitabine, cytarabine, venetoclax, daunorubicin, or hematopoietic stem cell transplant (HCT)) Patients whose antecedent MDS was treated were compared to previously untreated patients. Patients with sAML who had precedent treatment for MDS have inferior responses therapy and significantly reduced survival"

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

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

Hydroxyurea vs. hypomethylating vs. other agents in chronic myelomonocytic leukemia: A retrospective inspection of treatment strategies among 457 Mayo Clinic patients. (ASCO 2025) - "209 patients received CMML-directed therapy: HU (N=102), HMA (N=78; azacitidine 32 and decitabine 46). In the current retrospective study that included a large number of patients with CMML, chemotherapy with HU or HMA did not appear to affect overall survival but might have increased the risk of blast transformation. The study also suggests superiority of HU for the treatment of leukocytosis and HMA for anemia. Predictors of blast transformation-free survival (BTFS) in 457 patients diagnosed with chronic myelomonocytic leukemia (CMML)."

Retrospective data • Anemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • ASXL1

Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation. (PubMed, Ann Pharmacother) - P1/2 | "Revumenib is an innovative targeted treatment with promising activity in r/r acute leukemia with KMT2Ar."

Journal • Review • Acute Myelogenous Leukemia • Cardiovascular • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pediatrics • Thrombosis • Transplantation • KMT2A

GSK-3484862, a DNMT1 degrader, promotes DNMT3B expression in lung cancer cells. (PubMed, NAR Cancer) - "The FDA-approved nucleoside analogs azacytidine and decitabine are effective demethylating agents for hematologic malignancies but their general use has been limited by their toxicity and ineffectiveness against solid tumors. GSK-3484862, a dicyanopyridine-containing, DNMT1-selective inhibitor and degrader, offers a promising lead for developing novel demethylating therapeutics. These demethylation events correlate with upregulation of DNMT3B expression. These findings suggest that combining inhibitors targeting DNMT1, the maintenance methyltransferase, with those targeting DNMT3A/3B, the de novo methyltransferases, or using pan-DNMT inhibitors, could enhance anticancer efficacy and reduce resistance."

Journal • Hematological Disorders • Hematological Malignancies • Lung Cancer • Oncology • Solid Tumor • DNMT1 • DNMT3A • DNMT3B • TERT

LYT-200-2022-02: A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS) (clinicaltrials.gov) - P1 | N=90 | Recruiting | Sponsor: PureTech | Trial completion date: May 2025 ➔ Mar 2026 | Trial primary completion date: Feb 2025 ➔ Dec 2025

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

INDIVIDUALIZED AND INCLUSIVE STRATEGIES FOR TREATING JEHOVAH’S WITNESSES WITH ACUTE LEUKEMIA AND HIGH-GRADE MYELOID NEOPLASMS (EHA 2025) - "Treatments included: 7+3, attenuated decitabine+venetoclax, and azacitidine and sorafenib...One, treated with dasatinib+HyperCVAD, relapsed after 9 mos, then received Ponatinib+Blinatumomab, and underwent stem cell transplant...The third pt was treated with cyclophosphamide/topotecan for a co-occurring neuroblastoma without response, switching to blinatumomab then hospice due to neuroblastoma progression.For MDS - 1 pt received luspatercept for 8 mos before cessation due to cytopenia (Hgb of 2.9 g/dL)...TPO agonists included romiplostim at 2 to 4 mcg/kg weekly or daily eltrombopag, while G-CSF (Filgrastim, 5 mcg/kg) was administered based on ANC levels. Our study of Jehovah's Witness pts with acute leukemia and high-grade MDS underscores the possibility of individualized leukemia-directed care despite transfusion limitations. Despite significant baseline cytopenias, individualized treatment approaches and the use of hematopoietic growth factors can facilitate the..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Neuroblastoma • Oncology • Septic Shock • Solid Tumor • NPM1

SEQUENTIAL ALLOGENEIC STEM CELL TRANSPLANTATION FOR VENETOCLAX-EXPOSED RELAPSED OR REFRACTORY ACUTE MYELOID LEUKEMIA OR MYELODYSPLASTIC SYNDROME WITH INCREASED BLASTS-2 (EHA 2025) - "Three different 10-day preconditioning backbone regimens were used: ACTIVE (actinomycin D 12.5 mcg/kg (days 1-3), cytarabine 20 mg/m2 (days 1-10), venetoclax 600 mg (days 1-10), De-ACTIVE (decitabine 20 mg/m2 (days 1-10) added to ACTIVE) or De-CAVE (decitabine 20 mg/m2 (days 1-10), cytarabine 20 mg/m2 (days 1-10), venetoclax 600 mg (days 1-10)...Gilteritinib, revumenib, and BCR-ABL1 inhibitor exposure was reported in 16 % (4/25), 12 % (3/25), and 8 % (2/25), respectively...Cladribine (40 %, 10/25), gilteritinib (12 %, 3/25), trametinib (16 %, 4/25), navitoclax (28 %, 7/25), and ponatinib (8 %, 2/25) were added to the preconditioning... Upfront sequential alloSCT demonstrates high antileukemic efficacy in poor-risk, R/R AML, or MDS-IB2 patients with previous venetoclax exposure."

Acute Myelogenous Leukemia • Infectious Disease • Mucositis • Myelodysplastic Syndrome • Septic Shock • Transplantation • BCR • TP53

Enhanced FOXO1 expression as a predictor of decitabine response and prolonged survival in high-risk myelodysplastic syndrome. (PubMed, Hematology) - "Univariate and multivariate Cox analyses revealed that elevated FOXO1 expression was linked to prolonged overall survival and leukemia-free survival. Elevated FOXO1 expression in high-risk MDS patients undergoing decitabine treatment enhances patient prognosis and survival."

Journal • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • FOXO1

Budget Impact Analysis of Venetoclax Combination Therapies for the Treatment of Newly Diagnosed Acute Myeloid Leukemia Patients Who Are Aged 75 Years or Older, or Who Have Comorbidities That Preclude Use of Intensive Induction Chemotherapy (ISPOR 2025) - "The most up-to-date market share, reflecting the current treatment landscape projected venetoclax + azacitidine or decitabine to capture 53% and 15% market share, respectively, from existing treatments (azacitidine, low-dose cytarabine [LDAC], decitabine, ivosidenib, gemtuzumab ozogamicin, glasdegib + LDAC, and ivosidenib + azacitidine). Use of venetoclax combinations for the treatment of FDA-approved indication of ND AML reduced the budget impact and provides potential financial benefit for US payers."

Clinical • Combination therapy • HEOR • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Multi-antigen Specific CD8+ T Cells With Decitabine and Lymphodepleting Chemotherapy for the Treatment of Patients With Relapsed or Refractory AML or MDS Following an Allogeneic Hematopoietic Cell Transplantation From a Matched Donor (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: City of Hope Medical Center | N=31 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

Molecular mechanistic approach to reveal decitabine's effect on DNMT gene modulation and its inhibitory role in heavy metal-induced proliferation in urinary bladder cancer cell line. (PubMed, Toxicol In Vitro) - "Furthermore, decitabine treatment effectively inhibited Cd- and Cr-induced proliferation and downregulated DNMT gene expression. In conclusion, heavy metals such as Cd and Cr may contribute to urinary bladder carcinogenesis through DNMT upregulation, while decitabine showed prominent protective effects by suppressing DNMT expression and inhibiting cell proliferation."

Journal • Preclinical • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • DNMT1 • DNMT3B

A Rare Case of Acute Myeloid Leukemia With Refractory Hypercalcemia in the ICU (ATS 2025) - "Case report: A 68-year-old male with a history of acute myeloid leukemia (AML) complicated by 17% blasts and a TP53 mutation, previously treated with decitabine and venetoclax, presented with lethargy, shortness of breath, dizziness, nausea, and poor oral intake...Treatment was initiated with intravenous fluids, calcitonin, hydrocortisone, and furosemide. Despite these interventions, his calcium levels remained persistently high, prompting the initiation of pamidronate...Given the severity and refractoriness of hypercalcemia in AML, bisphosphonate resistant cases warrant further investigation. This condition represents a clinical emergency and underscores the need for more research into its pathophysiology and optimal management strategies."

Clinical • Acute Myelogenous Leukemia • Endocrine Disorders • Febrile Neutropenia • Hematological Malignancies • Leukemia • Metabolic Disorders • Nephrology • Oncology • TP53

Low-Risk Myelodysplastic Syndrome in Lung Transplant Candidates: Pause at Candidate Selection? (ATS 2025) - "Posttransplant, he was placed on tacrolimus, mycophenolate, and prednisone, but developed leukopenia and anemia, necessitating discontinuation of mycophenolate...He began chemotherapy with decitabine, venetoclax, and vincristine...His condition worsened despite transfusions and luspatercept-aamt, progressing to AML with 24.1% blasts and mutations in U2AF1, NRAS, and SETBP1 and MECOM rearrangement. Chemotherapy with azacitidine and venetoclax was initiated, but he succumbed to diffuse alveolar hemorrhage. Despite low risk MDS features, both patients in our report progressed to AML after lung transplant; their disease was resistant to conventional therapies. Further research on predictors of MDS progression after solid organ transplant and the impact of immunosuppressive medications on MDS disease progression is needed to help risk stratify older lung transplant candidates with low-risk MDS."

Acute Myelogenous Leukemia • Anemia • Antibody-mediated Rejection • Chronic Obstructive Pulmonary Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Nephrology • Oncology • Pulmonary Disease • Renal Disease • Respiratory Diseases • Septic Shock • Solid Organ Transplantation • Transplantation • CD34 • MECOM • NCAM1 • NRAS • SETBP1 • TP53 • U2AF1

Mucorales and Actinomyces Co-infection Presenting as Erosive Pulmonary Mass in a Neutropenic Patientmucorales and Actinomyces Co-infection Presenting as Erosive Pulmonary Mass in a Neutropenic Patient (ATS 2025) - "He underwent recent 1 cycle chemotherapy with venetoclax and decitabine...Of note patient was on outpatient posaconazole for fungal prophylaxis, however therapeutic levels of the drug were not achieved...However given underlying malignancy and tenuous clinical condition alongside profound thrombocytopenia, he elected a palliative approach to his overall diagnoses, and was discharged with isavuconazole and amoxicillin...The treatment of invasive Mucor infection requires both surgical debridement and anti-fungal therapy. In certain hosts such as this patient, surgical intervention is often noted to be of prohibitively high risk, which ultimately mitigates the clinician's ability to cure the infection."

Clinical • Hematological Disorders • Infectious Disease • Musculoskeletal Diseases • Oncology • Orthopedics • Pulmonary Disease • Respiratory Diseases • Septic Shock • Thrombocytopenia

Tragic Transformations and Differentiation Syndrome (ATS 2025) - "This abstract is funded by: None Introduction: Differentiation syndrome (DS) is a life-threatening adverse drug reaction caused by differentiating agents, including all-trans retinoic acid, arsenic trioxide, inhibitors of mutant isocitrate dehydrogenase (e.g., enasidenib), and the FLT3 inhibitor gilteritinib...Case: An 80-year-old female with a history of polycythemia vera and newly diagnosed AML, on decitabine, venetoclax, and enasidenib...Enasidenib was discontinued due to suspected DS, and she was treated with dexamethasone for five days...Despite initial oxygenation improvement with prone positioning, she remained hypotensive requiring norepinephrine, vasopressin, angiotensin II, and hydrocortisone...Leukocytosis worsened, and decitabine and hydroxyurea were initiated due to concern for uncontrolled acute leukemia...Prompt initiation of systemic glucocorticoids is critical in the management of DS, and this condition should be considered in patients receiving active..."

Cardiovascular • Febrile Neutropenia • Hematological Malignancies • Hepatology • Leukemia • Liver Failure • Myocardial Ischemia • Oncology • Polycythemia Vera • Renal Disease • Respiratory Diseases • FLT3

Impact of Decitabine on Native Lung Immune Profile in a Murine Single-lung Transplant Model (ATS 2025) - "DAC treatment reduced pro-inflammatory infiltration and promoted anti-inflammatory immune responses in the native lung, suggesting systemic immunoprotection in lung transplantation."

Preclinical • Inflammation • Transplant Rejection • Transplantation • CCR2 • CD36 • CD8 • MRC1 • PTPRC • SCARB1

Safety and Tolerability of Concurrent Lung Stereotactic Body Radiation Therapy (SBRT), Decitabine, and Venetoclax Prior to Hematopoietic Stem Cell Transplant (ATS 2025) - "Although high-grade toxicities of combining Stereotactic Body Radiation Therapy (SBRT) with certain pharmacologic agents such as bevacizumab exist, the toxigenic effects of combining radiation and hypomethylating agents are not well known, making the treatment decisions in this case difficult and potentially informative.Patient Presentation: A 67-year-old former smoker initially presented with myalgia, fever, chills and weight loss. This case illustrates the absence of unexpected adverse effects associated with concurrent AML chemotherapy and SBRT radiation, underscoring the safety of this approach despite concerns for potential toxicity. While the simultaneous diagnoses of AML and Lung Adenocarcinoma in this patient are rare, this case shows that combining these treatments can be safe and efficacious for patients. This case challenges the hesitancy that envelops administering concurrent radiation and AML chemotherapy and provides valuable insight to future cases of..."

Clinical • Bone Marrow Transplantation • Fatigue • Lung Adenocarcinoma • Lung Cancer • Musculoskeletal Pain • Non Small Cell Lung Cancer • Oncology • Pain • Solid Tumor • Transplantation

Little to show for much effort and investment: An industry perspective on MDS drug development (MDS 2025) - "Randomized studies incorporated lenalidomide, vorinostat, entinostat (MS-275), durvalumab, valproic acid, idarubicin, eltrombopag, pevonedistat (MLN4924), eprenetapopt (APR-246), sabatolimab (MBG453), magrolimab (Hu5F9-G4), or tamibarotene (SY-1425)...While the approval of luspatercept and imetelstat for lower-risk patients provides a glimmer of hope, this track record of failure in higher-risk MDS is enough to make a prudent investor or senior pharmaceutical executive think twice before committing further resources to development in this difficult group of diseases...Nor is it because the existing therapies are so good that they're hard to beat, although the practice of giving a few cycles of azacitidine or decitabine in local clinics before referring a patient to a trial center has been an unfortunate barrier to accrual...In this session I will discuss some potential fixes for a few of these problems. But solutions to other barriers are less clear, and will require..."

Acute Myelogenous Leukemia • Hematological Malignancies • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • FLT3 • TP53