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    CANCER:

    Myelodysplastic Syndrome

    22h
    Predictive Clinical and Biological Parameters in Acute Leukemia, Myelodysplastic Syndromes and Myeloproliferative Disorders-HEMATO-BIO-IPC-2013-015 (clinicaltrials.gov)
    P=N/A, N=650, Completed, Institut Paoli-Calmettes | Recruiting --> Completed
    Trial completion
    1d
    Exportin 1 Inhibitor Combined With Venetoclax Induces Apoptosis in Myelodysplastic Syndrome by Mitochondria-Induced Apoptosis Pathway. (PubMed, J Clin Lab Anal)
    Our study found that inhibition of XPO1 is a promising modality in the treatment of MDS, especially when combined with Venetoclax, which could be a potential target for MDS therapy.
    Journal • IO biomarker
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    XPO1 (Exportin 1)
    |
    Venclexta (venetoclax) • Xpovio (selinexor)
    1d
    HIF-1α impairs NK cell differentiation-maturation and cytotoxicity in myelodysplastic syndrome via JAK1/STAT5/SOCS2 pathway. (PubMed, J Immunol)
    Collectively, these findings suggest that the hypoxic microenvironment in MDS enhances HIF-1α expression, which subsequently impairs NK cell maturation and inhibits their cytotoxicity. Targeting HIF-1α may delay MDS progression by enhancing NK cell function via the JAK1/STAT5/SOCS2 signaling pathway.
    Journal
    |
    HIF1A (Hypoxia inducible factor 1, alpha subunit) • JAK1 (Janus Kinase 1) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • GZMB (Granzyme B) • SOCS2 (Suppressor Of Cytokine Signaling 2) • NKG2D (killer cell lectin like receptor K1)
    2d
    A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy (clinicaltrials.gov)
    P1, N=36, Recruiting, Children's Oncology Group | Trial completion date: Jun 2026 --> Mar 2028 | Trial primary completion date: Jun 2026 --> Mar 2028
    Trial completion date • Trial primary completion date
    |
    NF1 (Neurofibromin 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    RAS mutation • CBL mutation
    |
    cytarabine • methotrexate • leucovorin calcium • fludarabine IV • Rytelo (imetelstat) • Starasid (cytarabine ocfosfate)
    3d
    MyeChild 01: International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia (clinicaltrials.gov)
    P3, N=700, Active, not recruiting, University of Birmingham | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    cytarabine • cyclophosphamide • Mylotarg (gemtuzumab ozogamicin) • mitoxantrone • fludarabine IV • busulfan
    3d
    D7770C00001: A study of AZD2962, an IRAK4 inhibitor, in Participants with Haematologic Neoplasms (2025-520786-44-00)
    P1/2, N=7, Suspended, AstraZeneca AB | Recruiting --> Suspended
    Trial suspension
    3d
    TAK-226-3001: A phase 3 trial comparing elritercept versus epoetin alfa for treatment of anemia due to lower-risk MDS in ESA-nave adults who require RBC transfusions (2025-523544-12-00)
    P2/3, N=16, Not yet recruiting, Takeda Development Center Americas Inc.
    New P2/3 trial
    |
    elritercept (KER-050)
    4d
    Concurrent SF3B1 Mutation and BCR::ABL1 Demonstrating a Myelodysplastic Syndrome Phenotype: A Case Report. (PubMed, EJHaem)
    Single-cell DNA sequencing suggested that the SF3B1 mutation likely preceded BCR::ABL1 and blunted the expected granulopoiesis, thereby explaining the myelodysplastic syndrome phenotype without leukocytosis. This case illustrates how single-cell analysis can reveal meaningful clonal interactions that would not be evident with traditional bulk sequencing.
    Journal
    |
    ABL1 (ABL proto-oncogene 1) • SF3B1 (Splicing Factor 3b Subunit 1)
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    SF3B1 mutation
    4d
    Behçet's syndrome-like features revealing myelodysplastic syndrome with TP53 mutation: a case report. (PubMed, Front Immunol)
    After confirmation of diagnosis and prognostic assessment, the patient ultimately underwent successful allogeneic stem cell transplantation. This case highlights the importance of comprehensive genomic profiling in diagnosing, risk-stratifying, and managing patients with rare hematological entities presenting with autoinflammatory phenomena.
    Journal
    |
    TP53 (Tumor protein P53) • SF3B1 (Splicing Factor 3b Subunit 1)
    |
    TP53 mutation • SF3B1 mutation
    5d
    TACrolimus Targeted Immunosuppression Cessation in ALlogeneic HCT (clinicaltrials.gov)
    P1, N=50, Not yet recruiting, Stanford University | Initiation date: Feb 2026 --> Jun 2026
    Trial initiation date
    |
    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)
    5d
    BHARAT-1: A First in Human Study of AUR 103 Calcium to Evaluate Safety, Pharmacokinetics and Pharmacodynamics (clinicaltrials.gov)
    P1, N=27, Terminated, Aurigene Discovery Technologies Limited | N=80 --> 27 | Recruiting --> Terminated; Patient recruitment problems.
    Enrollment change • Trial termination • First-in-human
    |
    AUR-103
    6d
    UHKT-PTC-TMI-1: Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML/MDS (clinicaltrials.gov)
    P1/2, N=16, Recruiting, Institute of Hematology and Blood Transfusion, Czech Republic
    New P1/2 trial
    |
    cyclophosphamide • melphalan • fludarabine IV