^
    4d
    Characterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Dynamic Features of Disease Progression. (PubMed, Clin Lymphoma Myeloma Leuk)
    A small proportion of CMML patients exhibit indolent features linked to better outcomes, contrasting with markers indicating earlier treatment initiation, which are associated with increased risk of progression; recognizing these markers aids in predicting disease aggressiveness.
    Journal
    |
    NRAS (Neuroblastoma RAS viral oncogene homolog) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1)
    |
    RUNX1 mutation • ASXL1 mutation
    5d
    Myelodysplastic syndrome/myeloproliferative neoplasm overlap syndromes: a practical guide to diagnosis and management. (PubMed, Leukemia)
    Recent advances have started to uncover the pathobiologic basis for these diseases, leading to novel clinical trials for MDS/MPN overlap syndromes, in particular CMML. This review is a practical guide for the diagnosis and management of MDS/MPN overlap syndromes and presents novel therapeutics being specifically designed for these diseases to improve their historically poor outcomes.
    Review • Journal
    |
    SF3B1 (Splicing Factor 3b Subunit 1)
    7d
    Intestinal Microbiota Transplant Prior to Allogeneic Stem Cell Transplant (MAST) Trial (clinicaltrials.gov)
    P2, N=50, Recruiting, Imperial College London | Not yet recruiting --> Recruiting
    Enrollment open
    10d
    Chemotherapy (Decitabine in Combination With FLAG-Ida) and Total-Body Irradiation Followed by Donor Stem Cell Transplant for the Treatment of Adults With Myeloid Malignancies at High Risk of Relapse (clinicaltrials.gov)
    P1/2, N=36, Not yet recruiting, Fred Hutchinson Cancer Center
    New P1/2 trial
    |
    cytarabine • decitabine • idarubicin hydrochloride • fludarabine IV • Neupogen (filgrastim) • Starasid (cytarabine ocfosfate)
    10d
    IgA vasculitis associated with chronic myelomonocytic leukemia. (PubMed, BMC Rheumatol)
    CMML can be associated with autoimmune diseases, such as polyarteritis nodosa and cutaneous leukocytoclastic vasculitis. However, this is the first report of IgA vasculitis occurring in the context of low risk CMML.
    Journal
    |
    NRAS (Neuroblastoma RAS viral oncogene homolog)
    |
    NRAS mutation
    10d
    Usefulness of Flow Cytometry Monocyte Partitioning in the Diagnosis of Chronic Myelomonocytic Leukemia in a Real-World Setting. (PubMed, Cancers (Basel))
    Our study demonstrates the utility of a practical and robust approach for monocyte subset analysis in the diagnosis of CMML.
    Journal • Real-world evidence
    |
    CD14 (CD14 Molecule) • NDUFA2 (NADH:Ubiquinone Oxidoreductase Subunit A2)
    17d
    Waldenström Macroglobulinemia and Chronic Myelomonocytic Leukemia: Case Report and Literature Review. (PubMed, Onco Targets Ther)
    In August 2020, he was diagnosed with Waldenström macroglobulinemia (WM) and underwent six cycles of chemotherapy with bendamustine, zanubrutinib, and rituximab. Then he accepted hematopoietic stem cell transplantation (HSCT). Unfortunately, after 6 months, the patient died as a consequence of severe pulmonary infection.
    Journal
    |
    CXCR4 (Chemokine (C-X-C motif) receptor 4) • TET2 (Tet Methylcytosine Dioxygenase 2)
    |
    TET2 mutation
    |
    Rituxan (rituximab) • Brukinsa (zanubrutinib) • bendamustine
    17d
    NCI-2020-05261: Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations (clinicaltrials.gov)
    P1/2, N=58, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ASXL1 (ASXL Transcriptional Regulator 1)
    |
    TP53 mutation • FLT3-ITD mutation • ASXL1 mutation • CBL mutation
    |
    azacitidine • Vanflyta (quizartinib)
    22d
    Prospective multi-center observational cohort study on the efficacy and prognostic analysis of functional precision medicine for guiding treatment in relapsed or refractory pediatric leukemia (ChiCTR2500098273)
    P=N/A, N=500, Not yet recruiting, The First Affiliated Hospital,Sun Yat-sen University; The First Affiliated Hospital,Sun Yat-sen University
    New trial
    23d
    FHD-286-C-002: FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies (clinicaltrials.gov)
    P1, N=144, Active, not recruiting, Foghorn Therapeutics Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    cytarabine • decitabine • FHD-286
    24d
    Solitary and multiple xanthogranulomas in adult patients: a single-center retrospective cohort study. (PubMed, Arch Dermatol Res)
    In conclusion, the presentation of multiple XGs in an adult is associated with malignancy (particularly hematologic malignancies) and non-malignant hematologic disorders, suggesting a possible link with immune dysregulation. Molecular testing in one patient with multiple XGs and CMML transformed to AML revealed the same KMT2a deletion in both the XGs and the bone marrow, raising the possibility of clonal relationships in some cases.
    Retrospective data • Journal
    |
    KMT2A (Lysine Methyltransferase 2A)
    24d
    NCI-2019-01558: CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1, N=38, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation)
    27d
    Selinexor Combined With Azacitidine Therapy in High-Risk Myeloid Neoplasms Patients (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China
    New P2 trial
    |
    azacitidine • Xpovio (selinexor)
    28d
    Clinicopathologic Characteristics and Prognostic Profile of Chronic Myeloid Neoplasms With Somatic NF1 Mutations in Adult Patients. (PubMed, Eur J Haematol)
    Adult CMNs with mutated NF1 show higher AMC, high-risk molecular cytogenetic features, and inferior survival. Therefore, testing for NF1 mutations could be considered part of risk assessment for patients with CMNs.
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase) • NF1 (Neurofibromin 1) • SF3B1 (Splicing Factor 3b Subunit 1) • SRSF2 (Serine and arginine rich splicing factor 2)
    |
    KRAS mutation • SRSF2 mutation
    28d
    Features of RUNX1-mutated patients with chronic myelomonocytic leukemia in a national (ABCMML) and international cohort (cBioPortal). (PubMed, Wien Med Wochenschr)
    Regarding phenotype, in both cohorts, RUNX1-mutated patients had a significantly higher proportion of patients with thrombocytopenia and lower metric values for platelets as compared to wildtype patients. Validation of data, as we have done in this study in CMML patients using two independent cohorts, ensures the quality standards that are required for their use in clinical decision making.
    Journal
    |
    RUNX1 (RUNX Family Transcription Factor 1)
    |
    RUNX1 mutation
    29d
    Two distinct fetal-type signatures characterize juvenile myelomonocytic leukemia. (PubMed, Hemasphere)
    However, in the second group, despite evidence of developmental dysregulation as indicated by the aberrant signature of the master oncofetal regulator LIN28B, JMML clustered separately from healthy prenatal and postnatal fractions. These findings highlight the intricate relationship between JMML and development, which will help inform future therapeutic approaches for this rare but severe form of leukemia.
    Journal
    |
    LIN28B (Lin-28 Homolog B)
    1m
    Monocytic meningitis complicating histiocytosis and response to MEK-inhibitor: a case series. (PubMed, Ann Hematol)
    Treatment with binimetinib rapidly improved the patient's clinical condition...Cobimetinib also proved effective in managing CNS symptoms...In cases of neurological involvement in histiocytosis, lumbar puncture and liquid biopsy can sometime overcome the need for a meningeal biopsy. The molecular characterization of histiocytosis is essential for considering the use of targeted therapy, but the lack of an identified mutation should not preclude the use of anti-MEK therapy.
    Journal
    |
    MAP2K1 (Mitogen-activated protein kinase kinase 1)
    |
    Cotellic (cobimetinib) • Mektovi (binimetinib)
    1m
    STIMULUS-MDS2: Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) (clinicaltrials.gov)
    P3, N=530, Terminated, Novartis Pharmaceuticals | Completed --> Terminated; Despite positive trends identified post unblinding after Overall Survival primary analysis, the study did not show statistical significance in its primary endpoint, overall survival. No new safety findings were detected.
    Trial termination
    |
    azacitidine • sabatolimab (MBG453)
    1m
    Clonal Evolution of Myeloid Malignancies Treated With Microtransplantation: A Single-Centre Experience. (PubMed, J Cell Mol Med)
    Microtransplantation is a promising therapy for patients ineligible for regular allogeneic stem transplantation. Further larger and randomised studies are required to establish its place as a maintenance therapy in myeloid malignancies.
    Retrospective data • Journal
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    1m
    NCI-2022-02837: Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation (clinicaltrials.gov)
    P1/2, N=17, Completed, Stanford University | Active, not recruiting --> Completed
    Trial completion
    |
    IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
    |
    IDH2 mutation
    |
    Idhifa (enasidenib)
    1m
    Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies (clinicaltrials.gov)
    P2, N=35, Completed, Sidney Kimmel Cancer Center at Thomas Jefferson University | Active, not recruiting --> Completed
    Trial completion
    |
    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cyclophosphamide • fludarabine IV
    1m
    Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov)
    P1/2, N=55, Completed, Fujifilm Pharmaceuticals U.S.A., Inc. | N=41 --> 55
    Enrollment change
    |
    FF-10501-01
    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=50, Active, not recruiting, National Cancer Institute (NCI) | N=78 --> 50 | Trial primary completion date: Dec 2025 --> Sep 2024
    Enrollment change • Trial primary 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
    IMM01+Azacitidine VS Placebo +Azacitidine in Patients With Newly Diagnosed Chronic Myelomonocytic Leukemia (CMML1-2) (clinicaltrials.gov)
    P3, N=170, Recruiting, ImmuneOnco Biopharmaceuticals (Shanghai) Inc. | Trial primary completion date: Oct 2026 --> Apr 2026
    Trial primary completion date
    |
    azacitidine • timdarpacept (IMM01)
    1m
    A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation (clinicaltrials.gov)
    P1, N=50, Active, not recruiting, Dana-Farber Cancer Institute | Enrolling by invitation --> Active, not recruiting
    Enrollment closed
    |
    IL2 (Interleukin 2)
    |
    cyclophosphamide • fludarabine IV • CIML NK
    2ms
    Oncogenic and microenvironmental signals drive cell type specific apoptosis resistance in juvenile myelomonocytic leukemia. (PubMed, Cell Death Dis)
    Interestingly, stem and progenitor cells expressing the oncogenic PTPN11 mutant showed no increased apoptosis resistance. BCL-XL inhibition was the most effective in killing myeloid cells in vitro but was insufficient to completely resolve myeloproliferation in vivo.
    Journal • IO biomarker
    |
    BCL2 (B-cell CLL/lymphoma 2) • BCL2L1 (BCL2-like 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    2ms
    ASTX727-06: A Study for Participants Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose) (clinicaltrials.gov)
    P2, N=332, Enrolling by invitation, Taiho Oncology, Inc. | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    Inqovi (decitabine/cedazuridine)
    2ms
    Features of TP53-mutated patients with chronic myelomonocytic leukemia in a national (ABCMML) and international cohort (cBIOPORTAL). (PubMed, Wien Med Wochenschr)
    Decreased hemoglobin values and the presence of blast cells in peripheral blood were significantly associated with TP53 mutations in the cBIOPORTAL group but not in the ABCMML database. Our study indicates the necessity of sufficient patient numbers for the comparison of CMML patients regarding outcome and phenotype according to their molecular subtype, particularly in the case of rare mutations.
    Journal
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    2ms
    LILRB4 STAR-T Cells in the Treatment of R/R AML/CMML (clinicaltrials.gov)
    P1, N=11, Completed, Peking University People's Hospital | Trial primary completion date: Dec 2023 --> Aug 2024 | Not yet recruiting --> Completed
    Trial completion • Trial primary completion date
    |
    LILRB4 (Leukocyte Immunoglobulin Like Receptor B4)
    |
    cyclophosphamide • fludarabine IV • YTS104
    2ms
    CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT (clinicaltrials.gov)
    P=N/A, N=50, Recruiting, Children's Hospital of Philadelphia | Trial completion date: Jan 2026 --> Jun 2027 | Trial primary completion date: Feb 2025 --> Jun 2026
    Trial completion date • Trial primary completion date
    |
    cyclophosphamide
    2ms
    ACCELERATE: A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients with Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=358, Not yet recruiting, Center for International Blood and Marrow Transplant Research
    New P2 trial
    |
    Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV
    2ms
    Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=72, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2024 --> Oct 2025
    Trial completion date
    |
    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    sirolimus • clofarabine • melphalan
    2ms
    Myeloid neoplasms with PHF6 mutations: context-dependent genomic and prognostic characterization in 176 informative cases. (PubMed, Blood Cancer J)
    Multivariable analysis also identified high-risk karyotype (HR 5.1, 95% CI 1.2-20.9, p = 0.02), and hemoglobin <10 g/dL (HR 2.7, 95% CI 1.0-7.2, p = 0.04), as independent predictors of inferior OS in patients with MDS. The current study provides disease-specific information on genotype and prognosis of PHF6-mutated myeloid neoplasms.
    Journal
    |
    PHF6 (PHD Finger Protein 6)
    2ms
    Analysis of SRSF2 Gene Mutation in Patients with Chronic Myelomonocytic Leukemia (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi)
    SRSF2 mutations have a high incidence in CMML, occurring frequently in older patients, and often coexisting with ASXL1 and TET2 mutations. Patients with CMML carrying both SRSF2mut ASXL1mut double mutations have a higher risk of acute leukemia transformation.
    Retrospective data • Journal
    |
    NRAS (Neuroblastoma RAS viral oncogene homolog) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2) • SETBP1 (SET Binding Protein 1)
    |
    ASXL1 mutation • TET2 mutation • SRSF2 mutation
    2ms
    Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab. (clinicaltrials.gov)
    P2, N=33, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Nov 2025 --> Feb 2028 | Trial primary completion date: Nov 2025 --> Feb 2028
    Trial completion date • Trial primary completion date
    |
    Venclexta (venetoclax) • azacitidine • decitabine • spartalizumab (PDR001) • Inqovi (decitabine/cedazuridine) • sabatolimab (MBG453)
    2ms
    A Study to Evaluate S227928 as a Single Agent and in Combination With Venetoclax in Patients With R/R AML, MDS/AML, or CMML (clinicaltrials.gov)
    P1/2, N=132, Recruiting, Servier Bio-Innovation LLC | Initiation date: Nov 2024 --> Feb 2025 | Not yet recruiting --> Recruiting
    Enrollment open • Trial initiation date
    |
    Venclexta (venetoclax)
    2ms
    Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (clinicaltrials.gov)
    P1, N=100, Recruiting, Jacqueline Garcia, MD | Trial primary completion date: Feb 2025 --> Dec 2025
    Trial primary completion date
    |
    KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • NRAS (Neuroblastoma RAS viral oncogene homolog) • BCR (BCR Activator Of RhoGEF And GTPase) • BCL2 (B-cell CLL/lymphoma 2) • NPM1 (Nucleophosmin 1) • NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • MECOM (MDS1 And EVI1 Complex Locus) • NUP214 (Nucleoporin 214) • GATA2 (GATA Binding Protein 2) • DEK (DEK Proto-Oncogene) • RIT1 (Ras Like Without CAAX 1) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • KRAS mutation • NRAS mutation • RUNX1 mutation • RAS mutation • ASXL1 mutation • CBL mutation • Chr del(5q)
    |
    Venclexta (venetoclax) • azacitidine • Inqovi (decitabine/cedazuridine) • fludarabine IV • busulfan
    2ms
    Axatilimab with or Without Azacitidine for the Treatment of Patients with Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1/2, N=52, Suspended, Uma Borate | Recruiting --> Suspended
    Trial suspension
    |
    azacitidine • Niktimvo (axatilimab-csfr)
    2ms
    Noonan Syndrome, Cancer Risk, and Growth Hormone Treatment. (PubMed, J Clin Res Pediatr Endocrinol)
    In the light of current data, especially in cases for whom growth hormone therapy is considered, the diagnosis should be genetically confirmed, and the results of genetic analysis should be compared with the cases reported in the literature. Families should be informed about possible cancer risk and in cases predisposing to juvenile myelomonocytic leukemia, early initiation of growth hormone therapy should be avoided.
    Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    2ms
    CLAG-M or FLAG-Ida Chemotherapy and Reduced-Intensity Conditioning Donor Stem Cell Transplant for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1, N=120, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Dec 2027 --> Mar 2027 | Trial primary completion date: Dec 2025 --> Feb 2027
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cytarabine • cyclophosphamide • idarubicin hydrochloride • mitoxantrone • cladribine • fludarabine IV • cyclosporin A microemulsion • Neupogen (filgrastim) • Starasid (cytarabine ocfosfate)