^
    5d
    Pacritinib w/ Talazoparib in Pts w/ Myeloproliferative Neoplasms Unresponsive to JAK2 Inhibition (clinicaltrials.gov)
    P1, N=24, Recruiting, Fox Chase Cancer Center | Not yet recruiting --> Recruiting
    Enrollment open • Combination therapy
    |
    Talzenna (talazoparib) • Vonjo (pacritinib)
    5d
    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=70, Recruiting, Novartis Pharmaceuticals | Trial completion date: Jun 2028 --> Feb 2028
    Trial completion date
    |
    Venclexta (venetoclax) • azacitidine • decitabine • spartalizumab (PDR001) • Inqovi (decitabine/cedazuridine) • sabatolimab (MBG453)
    6d
    BRAF mutation in myeloid neoplasm: incidences and clinical outcomes. (PubMed, Leuk Lymphoma)
    Although the acquisition of BRAF mutation during disease progression did not correlate with unfavorable outcomes, its clearance through chemotherapy or stem cell transplant exhibited favorable outcomes (median overall survival of 34.8 months versus 10.4 months, p = 0.047). Furthermore, G469A was the most frequently observed BRAF mutation, differing from solid tumors and hairy cell leukemia, where V600E mutations were predominant.
    Clinical data • Journal
    |
    BRAF (B-raf proto-oncogene)
    |
    BRAF V600E • BRAF mutation • BRAF V600 • RAS mutation • BRAF G469A
    7d
    LAUREL: A Phase I Open-Label Dose Escalation Study of Intravenous INKmune In Patients With MDS or AML (clinicaltrials.gov)
    P1, N=2, Terminated, Inmune Bio, Inc. | Trial completion date: Jan 2025 --> Apr 2024 | Recruiting --> Terminated | Trial primary completion date: Jul 2024 --> Mar 2024; Slow/low Enrollment
    Trial completion date • Trial termination • Trial primary completion date
    |
    INKmune (primed NK cell therapy)
    9d
    The elevation of red blood cell distribution width is an independent prognostic factor for juvenile myelomonocytic leukemia. (PubMed, Blood Sci)
    RDW is an independent prognostic variable in JMML subjects. RDW may be regarded as an inexpensive biomarker to predict the clinical outcome in patients with JMML.
    Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    PTPN11 mutation
    9d
    High-Dose Post-Transplant Cyclophosphamide, Bortezomib, and Sitagliptin for the Prevention of GVHD (clinicaltrials.gov)
    P1/2, N=0, Withdrawn, Indiana University | N=72 --> 0 | Not yet recruiting --> Withdrawn
    Enrollment change • Trial withdrawal • Post-transplantation
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    bortezomib • cyclophosphamide
    14d
    Dose-Escalation Study of Oral Administration of LP-108 as Monotherapy and in Combination With Azacitidine in Patients With Relapsed or Refractory MDS, CMML, or AML (clinicaltrials.gov)
    P1, N=36, Active, not recruiting, Newave Pharmaceutical Inc | Recruiting --> Active, not recruiting
    Enrollment closed • Combination therapy
    |
    BCL2 (B-cell CLL/lymphoma 2)
    |
    azacitidine • LP-108
    15d
    Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL. (clinicaltrials.gov)
    P1, N=100, Recruiting, Newave Pharmaceutical Inc | Trial completion date: Aug 2024 --> Oct 2025 | Trial primary completion date: Aug 2024 --> Oct 2025
    Trial completion date • Trial primary completion date
    |
    BCL2 (B-cell CLL/lymphoma 2)
    |
    LP-118
    18d
    Analysis of CSF3R mutations in atypical chronic myeloid leukemia and other myeloid malignancies. (PubMed, Ann Diagn Pathol)
    In conclusion, CSF3R mutations were found at a higher frequency in aCML patients than in previous studies, which might reflect ethnic differences. Additional studies are needed to confirm these findings and the relationship between CSF3R and CEBPA mutations.
    Journal
    |
    ASXL1 (ASXL Transcriptional Regulator 1) • SRSF2 (Serine and arginine rich splicing factor 2) • CSF3R (Colony Stimulating Factor 3 Receptor) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    ASXL1 mutation • SRSF2 mutation • CEBPA mutation • CSF3R T618I • CSF3R mutation
    21d
    EVI-3: Combining Active and Passive DNA Hypomethylation (clinicaltrials.gov)
    P2, N=196, Recruiting, Kirsten Grønbæk | Trial completion date: Sep 2027 --> Dec 2027 | Trial primary completion date: Sep 2024 --> Dec 2025
    Trial completion date • Trial primary completion date • Combination therapy
    |
    azacitidine
    23d
    Identifying Potential SOS1 Inhibitors via Virtual Screening of Multiple Small Molecule Libraries against KRAS-SOS1 Interface. (PubMed, Chembiochem)
    Comparative analysis of the average binding free energies of these predicted potent compounds with known SOS1 small molecule inhibitors revealed that the identified compounds display similar or even superior predicted binding affinities compared to the known inhibitors. These findings offer valuable insights into the potential of these compounds as candidates for further development as effective anti-cancer agents.
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase)
    |
    KRAS mutation
    23d
    SH2B3 alterations in a novel genetic condition, juvenile myelomonocytic leukemia, and myeloproliferative neoplasia. (PubMed, Haematologica)
    Not available.
    Journal
    |
    SH2B3 (SH2B Adaptor Protein 3)
    |
    SH2B3 alteration
    27d
    Alpha/Beta T Cell and CD19+ B Cell Depletion in Allogeneic Stem Cell Transplantation in Patients With Malignant Diseases (clinicaltrials.gov)
    P2, N=20, Recruiting, University of Florida | Not yet recruiting --> Recruiting
    Enrollment open
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    FLT3 mutation • CBL mutation • Chr t(9;11)
    1m
    Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer (clinicaltrials.gov)
    P2, N=31, Active, not recruiting, Roswell Park Cancer Institute | Trial completion date: Aug 2023 --> Aug 2024 | Trial primary completion date: Aug 2022 --> Aug 2023
    Trial completion date • Trial primary completion date
    |
    cyclophosphamide • fludarabine IV
    1m
    SWOG-S1117: Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=282, Active, not recruiting, National Cancer Institute (NCI)
    Trial completion date • Combination therapy
    |
    lenalidomide • azacitidine • Zolinza (vorinostat)
    1m
    Ascorbic Acid and Chemotherapy for the Treatment of Relapsed or Refractory Lymphoma, CCUS, and Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=80, Recruiting, Mayo Clinic | N=55 --> 80
    Enrollment change
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • BCL2 (B-cell CLL/lymphoma 2) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • DNMT3A (DNA methyltransferase 1) • SF3B1 (Splicing Factor 3b Subunit 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • CD4 (CD4 Molecule) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    IDH2 mutation • DNMT3A mutation • TET2 mutation • SF3B1 mutation • EZH2 mutation • SRSF2 mutation • U2AF1 mutation
    |
    cisplatin • carboplatin • gemcitabine • Rituxan (rituximab) • cytarabine • cyclophosphamide • ifosfamide • oxaliplatin • etoposide IV • decitabine • Hemady (dexamethasone tablets) • Mabtas (rituximab biosimilar) • Starasid (cytarabine ocfosfate) • dexamethasone injection
    1m
    Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=25, Active, not recruiting, National Cancer Institute (NCI) | N=60 --> 25 | Trial completion date: Jun 2023 --> Dec 2024
    Enrollment change • Trial completion date • Metastases
    |
    carboplatin • veliparib (ABT-888) • topotecan
    1m
    Unraveling trajectories from aplastic anemia to hematologic malignancies: genetic and molecular insights. (PubMed, Front Oncol)
    Monosomy 7's prevalence and the occurrence of PTPN11 mutations suggest predictive and prognostic significance. Clonal evolution underscores the complexity of disease progression.
    Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • KMT2C (Lysine Methyltransferase 2C)
    |
    PTPN11 mutation • KMT2C mutation • MLL3 mutation
    2ms
    Therapy-related chronic myelomonocytic leukemia does not have the high-risk features of a therapy-related neoplasm. (PubMed, Blood Adv)
    Compared to a cohort of therapy-related myelodysplastic syndrome, tCMML had lower TP53 mutation frequency (12% vs 44.4%, P <.001) and less unfavorable outcomes. In summary, tCMML does not exhibit the high-risk features and poor outcomes of t-MNs.
    Journal
    |
    TP53 (Tumor protein P53) • NRAS (Neuroblastoma RAS viral oncogene homolog) • NF1 (Neurofibromin 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SETBP1 (SET Binding Protein 1)
    |
    TP53 mutation • NF1 mutation • TET2 mutation • CBL mutation • SETBP1 mutation
    2ms
    HA-1 T TCR T Cell Immunotherapy for the Treatment of Patients With Relapsed or Refractory Acute Leukemia After Donor Stem Cell Transplant (clinicaltrials.gov)
    P1, N=24, Suspended, Fred Hutchinson Cancer Center | Trial completion date: Jul 2025 --> Jul 2028 | Trial primary completion date: Oct 2024 --> Oct 2027
    Trial completion date • Trial primary completion date
    |
    HLA-A (Major Histocompatibility Complex, Class I, A) • CD4 (CD4 Molecule)
    |
    HLA-A*02:01
    |
    fludarabine IV
    2ms
    Prognostic impact of the bone marrow tumor microenvironment, HLA-I and HLA-Ib expression in MDS and CMML progression to sAML. (PubMed, Oncoimmunology)
    This study underlines the independent prognostic value of the tumor microenvironment in MDS/CMML progression to sAML, which shows the most pronounced immune escape. Moreover, new prognostic markers, like HLA-G expression and spatial T cell distribution, were described for the first time, which might also serve as therapeutic targets.
    Journal • PD(L)-1 Biomarker • IO biomarker
    |
    PD-L1 (Programmed death ligand 1) • CD8 (cluster of differentiation 8) • PD-1 (Programmed cell death 1) • PD-L2 (Programmed Cell Death 1 Ligand 2) • CD28 (CD28 Molecule) • HLA-G (Major Histocompatibility Complex, Class I, G)
    |
    CD28 expression
    2ms
    AZD6738 for Patients With Progressive MDS or CMML (clinicaltrials.gov)
    P1, N=52, Recruiting, Massachusetts General Hospital | Trial primary completion date: May 2022 --> May 2024
    Trial primary completion date
    |
    SF3B1 (Splicing Factor 3b Subunit 1) • SRSF2 (Serine and arginine rich splicing factor 2) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    SF3B1 mutation • U2AF1 mutation
    |
    ceralasertib (AZD6738)
    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 2023 --> Dec 2024
    Trial completion date
    |
    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    sirolimus • clofarabine • melphalan
    2ms
    NCI-2019-04959: Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm (clinicaltrials.gov)
    P1/2, N=55, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting | N=42 --> 55
    Enrollment closed • Enrollment change
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    FLT3-ITD mutation • FLT3 mutation • FLT3 D835
    |
    Venclexta (venetoclax) • Xospata (gilteritinib)
    2ms
    A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P3, N=42, Recruiting, Otsuka Australia Pharmaceutical Pty Ltd | Not yet recruiting --> Recruiting | Phase classification: P3b --> P3
    Enrollment open • Phase classification
    |
    Inqovi (decitabine/cedazuridine) • Onureg (azacitidine oral)
    2ms
    Long-term remission of infantile Takayasu arteritis associated with germline CBL syndrome after allogeneic hematopoietic stem cell transplantation: A case report and literature review. (PubMed, Transpl Immunol)
    The pathogenesis of vasculitis in CBL syndrome appears to involve dysregulated T cell function and possibly increased angiogenesis. This case advances the understanding of vascular involvement in CBL syndrome and of the genetic, immune, and vascular interplay in TA, offering insights for treating CBL syndrome and broader TA.
    Clinical • Observational data • Retrospective data • Review • Journal
    |
    CBL (Cbl proto-oncogene)
    2ms
    NCI-2022-02837: Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation (clinicaltrials.gov)
    P1/2, N=48, Recruiting, Stanford University | Trial completion date: Jan 2024 --> Aug 2024 | Trial primary completion date: Jan 2024 --> Aug 2024
    Trial completion date • Trial primary completion date
    |
    IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
    |
    IDH2 mutation
    |
    Idhifa (enasidenib)
    2ms
    Veliparib and Topotecan With or Without Carboplatin in Treating Patients With Relapsed or Refractory Acute Leukemia, High-Risk Myelodysplasia, or Aggressive Myeloproliferative Disorders (clinicaltrials.gov)
    P1, N=12, Active, not recruiting, National Cancer Institute (NCI)
    Trial completion date • Combination therapy
    |
    carboplatin • veliparib (ABT-888) • topotecan
    2ms
    Chronic myelomonocytic leukemia: 2024 update on diagnosis, risk stratification and management. (PubMed, Am J Hematol)
    Drug therapy is currently not disease-modifying and includes hydroxyurea and hypomethylating agents; a recent phase-3 study (DACOTA) comparing hydroxyurea and decitabine, in high-risk MP-CMML, showed similar overall survival at 23.1 versus 18.4 months, respectively, despite response rates being higher for decitabine (56% vs. 31%). These include systemic inflammatory autoimmune diseases, leukemia cutis and lysozyme-induced nephropathy; the latter requires close monitoring of renal function during leukocytosis and is a potential indication for cytoreductive therapy.
    Journal
    |
    DNMT3A (DNA methyltransferase 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2)
    |
    DNMT3A mutation • ASXL1 mutation • TET2 mutation • SRSF2 mutation
    |
    decitabine • hydroxyurea
    2ms
    A comprehensive genomic profiling of myeloid malignancies demonstrates mutational spectrum of DNA variants, FLT3-ITDs, and gene fusions (AACR 2024)
    The Oncomine Myeloid Assay is a fast, robust, and reproducible solution for comprehensive genomic profiling of myeloid malignancies. We describe the mutational spectrum of DNA variants and RNA fusions in a range of clinical research samples. (For research use only.
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • FGFR1 (Fibroblast growth factor receptor 1) • DNMT3A (DNA methyltransferase 1) • MYD88 (MYD88 Innate Immune Signal Transduction Adaptor) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2) • CREBBP (CREB binding protein) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • ZMYM2 (Zinc Finger MYM-Type Containing 2) • CALR (Calreticulin) • KAT6A (Lysine Acetyltransferase 6A) • ANKRD26 (Ankyrin Repeat Domain Containing 26)
    |
    FLT3-ITD mutation • ASXL1 mutation • TET2 mutation • SRSF2 mutation • FGFR1 fusion
    |
    Oncomine Myeloid Assay GX • Oncomine Myeloid Research Assay
    2ms
    Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial (clinicaltrials.gov)
    P1, N=2, Terminated, OHSU Knight Cancer Institute | Active, not recruiting --> Terminated; Low accrual
    Trial termination
    |
    Keytruda (pembrolizumab) • Opdivo (nivolumab) • Avastin (bevacizumab) • Venclexta (venetoclax) • Lynparza (olaparib) • Mekinist (trametinib) • erlotinib • Gilotrif (afatinib) • Yervoy (ipilimumab) • Ibrance (palbociclib) • dasatinib • Zelboraf (vemurafenib) • Tafinlar (dabrafenib) • carboplatin • Imfinzi (durvalumab) • sorafenib • Rozlytrek (entrectinib) • imatinib • sunitinib • everolimus • Nerlynx (neratinib) • Iclusig (ponatinib) • Kadcyla (ado-trastuzumab emtansine) • Cotellic (cobimetinib) • Lorbrena (lorlatinib) • Lenvima (lenvatinib) • bortezomib • doxorubicin hydrochloride • capecitabine • Verzenio (abemaciclib) • Xtandi (enzalutamide capsule) • Cabometyx (cabozantinib tablet) • albumin-bound paclitaxel • Stivarga (regorafenib) • abiraterone acetate • oxaliplatin • Aliqopa (copanlisib) • Vizimpro (dacomitinib) • Zydelig (idelalisib) • daunorubicin • Zolinza (vorinostat) • Idhifa (enasidenib) • Farydak (panobinostat) • Erivedge (vismodegib) • Nubeqa (darolutamide) • bicalutamide • leucovorin calcium • cabazitaxel • Vesanoid (tretinoin) • fluorouracil topical
    2ms
    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 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024
    Trial completion date • Trial primary completion date
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation)
    2ms
    KRAS mutations, autoimmunity and female sex in chronic myelomonocytic leukemia. (PubMed, Leuk Res)
    No abstract available
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase)
    |
    KRAS mutation
    2ms
    Onvansertib for the Treatment of Recurrent or Refractory Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1, N=25, Recruiting, Mayo Clinic | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial primary completion date
    |
    onvansertib (PCM-075)
    2ms
    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, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Jan 2027 --> Sep 2024 | Trial primary completion date: Jan 2027 --> Sep 2024
    Trial completion date • Trial primary completion date • Combination therapy
    |
    azacitidine • sabatolimab (MBG453)
    2ms
    A Study of BLEX 404 Oral Liquid in Patients With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-Risk Myelodysplastic Syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML) (clinicaltrials.gov)
    P2, N=52, Not yet recruiting, BioLite, Inc. | Trial completion date: Jun 2025 --> Jun 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
    Trial completion date • Trial primary completion date
    |
    azacitidine
    2ms
    Veliparib and Temozolomide in Treating Patients With Acute Leukemia (clinicaltrials.gov)
    P1, N=66, Active, not recruiting, National Cancer Institute (NCI)
    Trial completion date • Combination therapy
    |
    Chr t(15;17)
    |
    temozolomide • veliparib (ABT-888)
    2ms
    NCI-2018-01813: Quizartinib With Azacitidine or Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (clinicaltrials.gov)
    P1/2, N=73, Completed, M.D. Anderson Cancer Center | N=200 --> 73
    Enrollment change
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    FLT3 mutation
    |
    cytarabine • azacitidine • Vanflyta (quizartinib) • Starasid (cytarabine ocfosfate)
    2ms
    Chimeric Antigen Receptor T Cell Therapy Redirected to CD4 (CD4CAR)as a Second Line Treatment for Chronic Myelomonocytic Leukemia, CMML. (clinicaltrials.gov)
    P1, N=30, Recruiting, Huda Salman | Not yet recruiting --> Recruiting
    Enrollment open
    |
    CD4 (CD4 Molecule)
    |
    ICG122
    2ms
    Infrequent Presentations of Chronic NPM1-Mutated Myeloid Neoplasms: Clinicopathological Features of Eight Cases from a Single Institution and Review of the Literature. (PubMed, Cancers (Basel))
    Tailoring patient management on an individualized basis, favoring intensive treatment aligned with AML protocols, is crucial, regardless of blast percentage. Research on the impact of NPM1 mutations in non-acute myeloid neoplasms is ongoing, requiring challenging prospective studies with substantial patient cohorts and extended follow-up periods for validation.
    Review • Journal
    |
    NPM1 (Nucleophosmin 1)
    |
    NPM1 mutation
    3ms
    A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire. (PubMed, Haematologica)
    By integrating genetic information with clinical and hematological features, we have devised a predictive classifier. This classifier effectively identifies a subgroup of patients who are at a heightened risk of unfavorable post-transplant outcomes who would benefit novel therapeutic agents and post-transplant strategies.
    Journal
    |
    ASXL1 (ASXL Transcriptional Regulator 1) • JAK3 (Janus Kinase 3) • SETBP1 (SET Binding Protein 1)
    |
    RAS mutation • ASXL1 mutation • JAK3 mutation
    3ms
    Study of NMS-03592088 in Patients With Relapsed or Refractory AML or CMML (clinicaltrials.gov)
    P1/2, N=200, Recruiting, Nerviano Medical Sciences | N=140 --> 200 | Trial completion date: Sep 2023 --> Feb 2026 | Trial primary completion date: Sep 2023 --> Dec 2025
    Enrollment change • Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    FLT3-ITD mutation
    |
    NMS-088