Vyxeos (cytarabine/daunorubicin liposomal formulation) / Jazz, Nippon Shinyaku - LARVOL DELTA

V-RULES: Real-world effectiveness and safety of CPX-351 in patients with secondary acute myeloid leukemia (AML). (ASCO 2025) - "The abstract will be released to the public on May 22, 2025 at 4:00 PM"

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

A PHASE I/II, OPEN-LABEL, MULTI-CENTER STUDY EVALUATING THE SAFETY AND EFFICACY OF RUXOLITINIB AND CPX-351 IN COMBINATION FOR THE TREATMENT OF ADVANCED PHASE MYELOPROLIFERATIVE NEOPLASMS (EHA 2025) - "Use of intensive induction chemotherapy in MPN-BP is associated with poor overall survival (OS) (median OS 3.9 months (mo); Mesa et al, Blood 2005), whereas decitabine with ruxolitinib (Rux) has shown early efficacy with a median OS of 9.4 mo in a phase 2 study (Mascarenhas et al, Blood Adv 2020)... The combination of CPX-351 + Rux was feasible and well tolerated with a safety profile similar to CPX-351 alone. Serious bleeding event was the main DLT at both rux dose levels which is a known risk associated with CPX-351. Median OS compares favorably to historical controls treated with "7+3" induction alone, but the study is limited by small numbers."

Clinical • Metastases • P1/2 data • Anemia • Cardiovascular • Cerebral Hemorrhage • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia

EVOLUTION OF REAL-WORLD TREATMENT PATHWAYS FOR SECONDARY ACUTE MYELOID LEUKEMIA IN ENGLAND (EHA 2025) - "At the onset of the COVID-19 pandemic (2020), venetoclax (VEN) was made available through an emergency measure as an alternative to intensive chemotherapy (IC) to protect high-risk patients and save resources. In February 2022, NICE recommended VEN+azacitidine (AZA) for treatment-naïve adults with AML ineligible for IC...Prior to CPX-351's NICE recommendation, 1L IC was primarily daunorubicin/cytarabine (DA) followed by fludarabine, high-dose cytarabine, and granulocyte-colony stimulating factor (FLAG)... In patients with sAML, CPX-351 was rapidly adopted post-NICE recommendation and remains the SOC IC. VEN use began during COVID-19, growing further after NICE's 2022 recommendation and replacing LDAC and AZA monotherapy. HCT remains key for optimal outcomes, with CPX-351 improving post-HCT survival vs DA or FLAG; VEN-treated patients had the poorest post-HCT survival."

Clinical • HEOR • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Novel Coronavirus Disease • Oncology

RISK STRATIFICATION OF NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX IN COMBINATION WITH INTENSIVE CHEMOTHERAPY (EHA 2025) - P1/2, P2 | "While IC+VEN may abrogate certain ELN22 adverse features, pts with TP53mut and MECOMr still do poorly and require novel therapies. These findings require independent validation."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2 • KMT2A • KRAS • NRAS • PTPN11 • TP53

REAL-LIFE OUTCOMES IN PATIENTS WITH ACUTE MYELOID LEUKEMIA SECONDARY TO MYELOPROLIFERATIVE NEOPLASIA: RESULTS OF MULTICENTRIC ENEA-STUDY. (EHA 2025) - "Patients received treatment based on clinical practice, with first-line regimen including hypomethylating agents (HMAs) alone, conventional chemotherapy (CHT), and HMAs combined with venetoclax (HMAs+VEN)... The CHT subgroup showed the longest mOS among the three strategies, especially in HSCT patients. The HMAs+VEN regimen had encouraging response rates, but survival benefits were less significant, likely due to higher median age and lower transplant rate. Notably, even patients not reaching cCR benefited significantly from HSCT, highlighting its potential to overcome adverse prognostic factors."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Essential Thrombocythemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • TP53

SECONDARY ACUTE ERYTHROID LEUKEMIA: A RARE ENTITY (EHA 2025) - "Most patients, due to performance status and comorbidities, were treated with low-intensity regimens such as monotherapy with HMA or combinations of HMA and Venetoclax... sAEL were extremely rare entities with a poor prognosis. Most patients presented with pancytopenia, complex karyotype, and the presence of TP53 mutation is common. Cytochemistry and immunophenotype did not reveal any specific differences to distinguish between primary and sAEL."

Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Breast Cancer • Esophageal Cancer • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • Thrombocytopenia • CD36 • GYPA • KIT • NPM1 • SCARB1 • TFRC • TP53

CLINICO-GENOMIC FEATURES OF THERAPY-RELATED MYELOID NEOPLASMS (T-MNS): A NATIONWIDE STUDY (EHA 2025) - "Finally, patients treated with intensive chemotherapy had better survival than those receiving HMA/Venetoclax (p=0.04). Our study provides an updated cartography of t-MNs, confirming a reduction in the prevalence of lymphoproliferative diseases as primary tumors (25% vs 41%), an increase of GU tumors (29% vs21%), and a reduction in t-MN following autoimmune diseases (2% vs 8%), as compared to the prior Italian Network report (PMID: 25653205). Previous treatment (namely PARPi) and molecular characteristics are major determinants of survival, which remains poor, despite changes in t-MN treatment approaches."

Acute Myelogenous Leukemia • Breast Cancer • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Solid Tumor • ASXL1 • DNMT3A • IDH2 • JAK2 • NPM1 • PTPN11 • TET2 • TP53

PROGNOSTIC IMPACT OF HIGH-RISK MUTATIONS IN SECONDARY ACUTE MYELOID LEUKEMIA PATIENTS TREATED WITH CPX-351: INSIGHTS INTO MOLECULAR PATTERNS AND TREATMENT RESPONSE (EHA 2025) - "Background: Acute Myeloid Leukemia (AML) is a heterogeneous disease, driven by the serial acquisition of somatic mutations.The ELN 2022 classification recognizes mutations of TP53, ASXL1, BCOR, EZH2, SF3B1, SRSF2, STAG2, U2AF1, ZRSR2 as indicators of poor prognosis when conventional chemotherapy is administered.However, the prognostic relevance of these mutations may differ with the introduction of novel therapies, including hypomethylating agents plus Venetoclax (HMA+VEN), where a different molecular pattern including RUNX1, FLT3-ITD, N/KRAS, CBL, and KIT mutations proved to predict treatment failure.CPX-351 is currently approved for secondary AML (s-AML), where high-risk mutations are particularly common, yet the impact of these mutations on the response to CPX-351 remains underexplored... CPX-351 is able to induce MRD negative CR in a large proportion of s-AML patients, with minimal influence from ELN2022 adverse mutations or HMA-VEN resistance profile. Notably,..."

Clinical • Tumor mutational burden • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • ASXL1 • BCOR • FLT3 • KIT • KRAS • RUNX1 • SF3B1 • SRSF2 • STAG2 • TMB • TP53 • U2AF1 • ZRSR2

Cord Blood Transplantation in Relapsed/Refractory Pediatric Acute Myeloid Leukemia (AML) (ASPHO 2025) - "Due to residual disease after induction I, she received a 2nd induction cycle with fludarabine, cytarabine, and gemtuzumab. Then, she received a 3rd cycle with venetoclax and azacitidine. Due to persistent disease, she received CPX-351 with gilteritinib due to newly detected FLT3-ITD (allelic frequency 0.047)...After 4 cycles of menin inhibitor and a brief course of hydroxyurea and dexamethasone, with reduced but persistent disease burden, she underwent an unrelated 6/8 (A, DRB1 mismatched) CBT following conditioning with clofarabine, fludarabine, and busulfan... Unrelated CBT can induce remission in patients with otherwise refractory disease, and potentially induce long-term disease control. This case represents the need for more research for a potential maintenance treatment regimen including single agent menin inhibitor for high risk patients with refractory disease post-CBT."

Clinical • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Pediatrics • Transplantation • HLA-DRB1 • NSD1 • NUP98

ALLOGENEIC STEM CELL TRANSPLANTATION OUTCOMES AFTER CPX-351 VS OTHER INDUCTION REGIMENS IN NEWLY DIAGNOSED SECONDARY ACUTE MYELOID LEUKEMIA (EHA 2025) - "No-CPX cohort included pts mainly treated before 019 and include 46% of fludarabine based regimen 7% hypometilating agents ± venetoclax 3% "3+7" 4% other regimens... in our real-life experience we observed a longer 1-year-OS (70% vs 5 %) and 1-year-PFS (54% vs 43%) after HSCT for sAML pts treated with CPX induction regimen in comparison with no-CPX cohort. There is a tendency in higher rate of complete remission achieved before HSCT with CPX-351 even if it was not statistically significative (p=0 077). Multicenter real-life studies with a larger number of cases are necessary to better understand the impact of CPX-351 on transplant outcomes."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Transplantation • FLT3 • NPM1

DRUG SCREENING IN A ZEBRAFISH MODEL OF MLL-AF9-DRIVEN ACUTE MYELOID LEUKEMIA (EHA 2025) - "Control and MA9/lyz-EFGP ZF 48 hpf embryos were treated with 10 µM of daunorubicin, doxorubicin, cytarabine, or Venetoclax, and 6 µM of flavopiridol... In summary, our results show that our platform is capable of identifying drugs and drug combinations that show potential in treating AML. We are already in the process of testing several drugs approved for various other indications for the potential utility in treating leukemia."

Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • KMT2A • MLLT3 • RUNX1

Testing the Use of Combination Therapy in Patients With Persistent Low Level Acute Myeloid Leukemia Following Initial Treatment, The ERASE Study (A MyeloMATCH Treatment Trial) (clinicaltrials.gov) - P2 | N=184 | Not yet recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Aug 2025 ➔ Aug 2026 | Trial primary completion date: Aug 2025 ➔ Aug 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Transplantation

Venetoclax + Azacitidine vs. Induction Chemotherapy in AML (clinicaltrials.gov) - P2 | N=172 | Recruiting | Sponsor: Massachusetts General Hospital | Trial completion date: Jan 2026 ➔ Jan 2028 | Trial primary completion date: Jan 2025 ➔ Jan 2027

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • BCL2

MYCOTIC ANEURYSM AS A FATAL COMPLICATION IN A PATIENT WITH ACUTE LEUKEMIA: A CASE REPORT (EHA 2025) - "However, blood cultures showed growth of the filamentous fungus Scedosporium (Lomentospora) prolificans, prompting the initiation of antifungal therapy with Isavuconazole.Subsequently, the patient developed neurological symptoms, including headache, dysarthria, deviation of the labial commissure, and a non-reactive right pupil... Patients with Acute Myeloid Leukemia (AML) may develop life-threatening complications, particularly in the context of neutropenia following intensive chemotherapy.Mycotic aneurysm is a critical complication that should be considered in immunocompromised patients presenting with infectious and neurological symptoms.A high index of clinical suspicion and detailed radiological review are essential for early diagnosis and appropriate management."

Case report • Clinical • Acute Myelogenous Leukemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Ocular Infections • Ocular Inflammation • Oncology • Ophthalmology • Pain • Subarachnoid Hemorrhage

MICROBIOTA-INSPIRED COMPOUNDS AS NOVEL DRUG CANDIDATES TARGETING NPM1-MUTATED ACUTE MYELOID LEUKEMIA (EHA 2025) - "The co-administration of UCM-19286 with daunorubicin and cytarabine resulted in synergistic cytotoxicity in OCI-AML3 cells in vitro... UCM-19286 has been identified as a promising first-in-class NPM1 inhibitor for the treatment of NPM1mut AML. Its high preclinical efficacy, favorable pharmacokinetic profile, and synergistic potential with conventional chemotherapeutics underscore its translational relevance. On going investigations aim to validate its therapeutic benefits in vivo and further optimize its formulation for clinical application."

Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • FBXW7 • NPM1

CPX-351 FOR TRANSPLANT ELIGIBLE, HIGHER RISK PATIENTS WITH MYELODYSPLASTIC SYNDROMES: MOLECULAR RESPONSES AND LONG-TERM FOLLOW-UP (EHA 2025) - P1 | "CPX-351 may be a promising treatment approach for transplant-eligible, high-risk MDS patients, warranting further study in this population. CPX-351 therapy was associated with mutational clearance in 24% pts. The correlation of mutational clearance and treatment outcomes in CPX-351 needs further study."

Clinical • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Transplantation • ASXL1 • BCOR • RUNX1 • SETBP1 • STAG2 • TP53

THE IMPACT OF MEASURABLE RESIDUAL DISEASE ON OUTCOMES IN PATIENTS WITH FLT3-ITD MUTATED ACUTE MYELOID LEUKEMIA (EHA 2025) - "Various types of induction therapies were used such as 7+3 (cytarabine/anthracycline) or CPX-351 +/- midostaurin +/- gemtuzamab... Our findings highlight the importance of highly sensitive FLT3-ITD MRD testing and its associated prognostic impact for pts with FLT3-ITD mutated AML after achieving response. Pts with undetectable MRD have significantly longer survival. For detectable MRD, HCT remains an important treatment modality to extend survival."

Clinical • Residual disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • DNMT3A • FLT3 • IDH2 • NPM1 • RUNX1 • TET2 • WT1

EFFICACY OF CPX-351 IN SECONDARY ACUTE MYELOID LEUKEMIA DEFINED ACCORDING TO WHO 2022 CLASSIFICATION. (EHA 2025) - "Our data show that CPX-351 has high activity in s-AML, defined according to WHO 2022 as patients with MDS-related gene mutations had a superimposable outcome to the other patients, therefore, CPX-351 is a reasonable option also for patients with genetically defined s-AML."

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

CPX-351 IN PATIENTS WITH NEWLY DIAGNOSED POST MYELOPROLIFERATIVE NEOPLASMS ACUTE MYELOID LEUKEMIA . FINAL RESULTS OF THE CPX-TA SMP PHASE II TRIAL. (EHA 2025) - "CPX-351 showed 44% response rates, which is comparable to 7+3 rates in this high-risk population with poor molecular risk. Toxicity profile is manageable. TP53-mut and/or TET2-mut groups have particularly poor outcomes"

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Essential Thrombocythemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Mucositis • Myelofibrosis • Myeloproliferative Neoplasm • Neutropenia • Oncology • Polycythemia Vera • Thrombocytopenia • Thrombocytosis • ASXL1 • CALR • JAK2 • TET2 • TP53

LONG-TERM REAL-WORLD EXPERIENCE WITH CPX-351 TREATMENT FOR ACUTE MYELOID LEUKEMIA IN ENGLAND (EHA 2025) - "Twenty-four (4%) patients received azacitidine for prior malignancy; no patients received midostaurin in combination with CPX-351... This is the largest RW evidence study of CPX-351 to date, complementing the 5-year follow-up data from the pivotal phase 3 trial that supported its regulatory approval. OS in patients aged ≥60 years was comparable to the phase 3 trial, including patients aged ≥70 years. Additionally, the findings help address the gap in CPX-351 outcomes data for patients <60 years; in this patient group OS was favorable."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

V-RULES: REAL-WORLD EFFECTIVENESS AND SAFETY OF CPX-351 IN PATIENTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA (EHA 2025) - "These results highlight the effectiveness and safety of CPX-351 for the treatment of t-AML and AML-MRC in the US RW setting, consistent with the pivotal trial and published RW data. Notably, this study showed favorable outcomes for younger patients (<60 years) who were not included in the pivotal trial. Patients who received HCT also had improved outcomes, as did those with MRm."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • TP53

Double hit Cytogenetics: Secondary AML Featuring 11Q21 Translocation and Monosomy 7 With 7Q Deletion (ASPHO 2025) - "Design/ Case Report An 18-year-old male with a history of osteosarcoma of the left femur underwent resection with total knee prosthetic reconstruction and completed chemotherapy protocol AOST0331, which included doxorubicin, methotrexate, and cisplatin, in 2022...The patient was started on the Vyxeos protocol (daunorubicin and cytarabine) and is currently undergoing treatment with the FLAG-Ida regimen (fludarabine, cytarabine, idarubicin, and intrathecal cytarabine)... This case describes a patient with a complex karyotype who developed s-AML two years after completing chemotherapy for osteosarcoma. While the patient's monosomy 7 and 7q deletion, along with the standard dosing of doxorubicin, suggest an alkylator-associated s-AML, the atypical timing of the diagnosis raises the possibility that the 11q21 translocation contributed to the development of s-AML. Thus, this case underscores the importance of genetic analysis in understanding the mechanisms behind..."

Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Pain • Pediatrics • Sarcoma • Solid Tumor

Subgaleal Edema as a Unique Reaction Related to Liposomal Cytarabine-Daunorubicin (ASPHO 2025) - ""Phase I/II study of CPX-351 followed by fludarabine, cytarabine, and granulocyte-colony stimulating factor for children with relapsed acute myeloid leukemia: a report from the Children's Oncology Group." Journal of Clinical Oncology... While dermatologic toxicity is a known side effect of liposomal cytarabine-daunorubicin, this is the first report of a severe dermatologic reaction with subgaleal edema in a pediatric patient. We report this as a rare side effect of liposomal cytarabine-daunorubicin in a pediatric patient and describe a reasonable therapeutic option to aid in symptom resolution."

Acute Myelogenous Leukemia • Cardiovascular • Dermatology • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics

The Clinical Safety and Efficacy of Cytarabine and Daunorubicin Liposome (CPX-351) in Acute Myeloid Leukemia Patients: A Systematic Review. (PubMed, Cancer Rep (Hoboken)) - "This review presents strong evidence supporting CPX-351 as a therapeutic alternative with superior efficacy and comparable safety to standard chemotherapy across diverse AML populations. This represents a breakthrough in therapy, with demonstrated efficacy in AML cases."

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

The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety. (PubMed, Drugs) - "This review examines the mechanisms underlying CPX-351's therapeutic effects and highlights its expanding role in AML treatment by summarizing efficacy and safety data from preclinical models, the pivotal clinical trial, and real-world studies. Particular focus is given to recent findings on CPX-351's intestinal and cardioprotective properties, which together strengthen its safety and efficacy profile compared with conventional chemotherapy."

Clinical • Journal • Review • Acute Myelogenous Leukemia • Cardiovascular • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology