^
    1m
    Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion (clinicaltrials.gov)
    P2, N=29, Active, not recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Jun 2026 --> May 2027
    Trial completion date
    |
    Jakafi (ruxolitinib)
    1m
    Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes (clinicaltrials.gov)
    P2, N=49, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Apr 2026 --> Apr 2027
    Trial completion date
    |
    RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    Chr del(11q)
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation) • pomalidomide
    1m
    A Phase Ib/II study of ceralasertib, a selective inhibitor of ATR, in patients with relapsed or refractory MDS and CMML. (PubMed, Blood Adv)
    In conclusion, ceralasertib 160mg BID d1-7 and 15-21 was established as monotherapy dosing with a response rate of 30% in patients with R/R MDS and CMML. NCT03770429.
    P1/2 data • Journal
    |
    RUNX1 (RUNX Family Transcription Factor 1) • TNFRSF8 (TNF Receptor Superfamily Member 8)
    |
    RUNX1 mutation
    |
    ceralasertib (AZD6738)
    1m
    Vibecotamab for measurable residual disease in acute myeloid leukemia and for myelodysplastic syndromes and chronic myelomonocytic leukemia after hypomethylating agent failure: a phase II study. (PubMed, J Hematol Oncol)
    Vibecotamab was active in low-blast myeloid diseases, although the durability of responses was modest. Additional studies of CD123-targeting bispecific antibodies, alone or in combination, are warranted for these diseases.
    P2 data • Journal
    |
    CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    vibecotamab (XmAb14045)
    1m
    MT2015-29: Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders (clinicaltrials.gov)
    P2, N=300, Recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2026 --> Jun 2027
    Trial completion date • Trial primary completion date
    |
    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • IKZF1 (IKAROS Family Zinc Finger 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    NPM1 mutation • MLL rearrangement
    |
    cyclophosphamide
    2ms
    Chronic Myelomonocytic Leukemia Revisited: A Comprehensive Review with Emphasis on the Oligomonocytic Subtype. (PubMed, Hum Pathol)
    Cases harboring biallelic TET2 inactivation or TET2+SRSF2 co-mutation show the highest bone marrow monocyte burden, frequent classical monocyte (MO1; CD14+/CD16-) elevation, and highest progression risk representing biologically true OM-CMML, whereas SF3B1-mutated and biallelic TP53 mutated cases show MDS-directed biology and warrant reclassification. This review synthesizes current diagnostic frameworks, molecular heterogeneity, risk stratification approaches, and evolving classification proposals, thereby providing a practical guide for pathologists navigating OM-CMML in the modern genomic era.
    Journal
    |
    TP53 (Tumor protein P53) • SF3B1 (Splicing Factor 3b Subunit 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2) • CD14 (CD14 Molecule)
    |
    TP53 mutation • TET2 mutation • SF3B1 mutation • SRSF2 mutation
    2ms
    EP0042-101: Study to Evaluate the Safety and Tolerability of EP0042 (clinicaltrials.gov)
    P1/2, N=70, Recruiting, Ellipses Pharma | Trial completion date: Dec 2026 --> Dec 2027 | Trial primary completion date: Oct 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    Venclexta (venetoclax) • azacitidine • EP0042
    2ms
    CD69 blockade restores the bone marrow niche and delays leukemogenesis in a mouse model of Nras G12D-driven chronic myelomonocytic leukemia. (PubMed, Cancer Biol Ther)
    Anti-CD69 monoclonal antibody treatment was associated with reduced generation of granulocyte-macrophage progenitor cells, prolonged survival, and decreased Treg accumulation in the BME. Our findings suggest that CD69 may serve as a biomarker of BME immunological dysfunction in CMML.
    Preclinical • Journal
    |
    NRAS (Neuroblastoma RAS viral oncogene homolog) • CD69 (CD69 Molecule)
    |
    NRAS mutation • NRAS G12
    2ms
    Cumulative incidence and prognostic factors for leukemic transformation in chronic myelomonocytic leukemia: a competing risk analysis. (PubMed, BMC Cancer)
    This study shows how competing-risk analysis complements standard Kaplan-Meier estimates of overall and progression-free survival when the cumulative incidence of a specific event, leukaemic transformation, is the quantity of interest. Although the modest, single-centre sample and non-randomised treatment allocation preclude definitive treatment recommendations, cytogenetic risk was the strongest disease-related prognostic factor, and molecular profiling provided additional risk discrimination within the NGS-tested subgroup. Cumulative incidence functions should therefore be reported routinely alongside Kaplan-Meier estimates in CMML and other myeloid neoplasms with non-negligible competing mortality.
    Journal
    |
    TP53 (Tumor protein P53) • SETBP1 (SET Binding Protein 1)
    2ms
    Relevance of Peripheral Cells in the Pathophysiology of Chronic Myelomonocytic Leukemia (CMML) (clinicaltrials.gov)
    P=N/A, N=50, Recruiting, Centre Hospitalier Universitaire de Nice | Unknown status --> Recruiting | Trial completion date: Nov 2018 --> Nov 2028 | Trial primary completion date: Nov 2018 --> Nov 2027
    Enrollment open • Trial completion date • Trial primary completion date
    2ms
    A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm (clinicaltrials.gov)
    P2, N=0, Withdrawn, M.D. Anderson Cancer Center | Trial completion date: Aug 2029 --> Jun 2026 | Trial primary completion date: Aug 2027 --> Jun 2026
    Trial completion date • Trial primary completion date
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
    |
    IDH1 mutation
    |
    azacitidine • Rezlidhia (olutasidenib)
    2ms
    Comparative efficacy of donor lymphocyte infusions in augmenting graft-versus-leukemia effect after allogeneic hematopoietic stem cell transplantation for patients with myeloid malignancies. (PubMed, Acta Haematol)
    DLI is an effective treatment strategy for post-transplant relapse of all myeloid malignancies, with specific genetic subtypes showing poorer outcomes and survival.
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • NRAS (Neuroblastoma RAS viral oncogene homolog) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1)
    |
    TP53 mutation • KRAS mutation • NRAS mutation