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    CANCER:

    Myelodysplastic Syndrome

    2d
    Do myelodysplasia-related gene mutations alter transplant decisions in NPM1-mutated acute myeloid leukemia? Comment on: "Prognostic implications of myelodysplasiarelated gene mutations in NPM1-mutated acute myeloid leukemia: a systematic review and meta-analysis". (PubMed, Haematologica)
    Not available.
    Retrospective data • Journal
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    NPM1 (Nucleophosmin 1)
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    NPM1 mutation
    2d
    Predisposition to ALL and Solid Tumors Rather Than Bone Marrow Failure in FANCM-Associated Fanconi Anemia. (PubMed, JCO Precis Oncol)
    Biallelic FANCM variants define a distinct FA subtype lacking early BMF, leading to missed diagnoses and severe toxicity upon malignancy. Recognizing this presentation is crucial for timely FA detection and for implementing adapted therapeutic and follow-up strategies.
    Journal
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    FANCM (FA Complementation Group M) • BCL11B (BAF Chromatin Remodeling Complex Subunit BCL11B) • HOXA13 (Homeobox A13) • ZNF384 (Zinc Finger Protein 384)
    2d
    MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases (clinicaltrials.gov)
    P2, N=132, Not yet recruiting, Masonic Cancer Center, University of Minnesota
    New P2 trial
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    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)
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    cyclophosphamide • fludarabine IV • Grafapex (treosulfan)
    3d
    IIT-2018-Gamma-DeltaTcell: Expanded/Activated Gamma Delta T-cell Infusion Following Hematopoietic Stem Cell Transplantation and Post-transplant Cyclophosphamide (clinicaltrials.gov)
    P1, N=38, Recruiting, University of Kansas Medical Center | Trial completion date: Jan 2025 --> Jan 2028 | Trial primary completion date: Jan 2024 --> Jan 2027
    Trial completion date • Trial primary completion date
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    cyclophosphamide • INB-100
    3d
    Leveraging paired germline and somatic analysis to improve the classification of DDX41 variants. (PubMed, Br J Haematol)
    Current ACMG and CanVIG-UK guidelines do not permit co-occurrence with recurrent somatic driver variants as evidence favouring pathogenicity, despite this being a convincing finding. This study proposes modifying certain rules as a basis for developing DDX41-specific guidance, as it will significantly impact decisions surrounding bone marrow transplantation.
    Journal
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    DDX41 (DEAD-Box Helicase 41)
    3d
    Overexpression of the TGF-β target CCN2 in megakaryocytes: a common feature of MDS with mutated SF3B1 : Uncovering novelinsights into the bone marrow microenvironment in MDS. (PubMed, Virchows Arch)
    In aggregate, these findings may contribute to a better understanding of disease pathophysiology and help elucidate the role of potentially clinically relevant TGF-β signaling. This is particularly significant given the clinical use of agents targeting TGF-β-signaling such as luspatercept, as well as the emergence of several CCN2-targeting therapies currently undergoing clinical or preclinical evaluation with promising results.
    Journal
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    RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TGFB1 (Transforming Growth Factor Beta 1) • CTGF (Connective tissue growth factor)
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    RUNX1 mutation • ASXL1 mutation • SF3B1 mutation
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    Reblozyl (luspatercept-aamt)
    3d
    DSP-5336-101: A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1) (clinicaltrials.gov)
    P1/2, N=606, Recruiting, Sumitomo Pharma America, Inc. | N=362 --> 606
    Enrollment change
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    FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
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    FLT3-ITD mutation • FLT3 mutation • NPM1 mutation
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    Venclexta (venetoclax) • Xospata (gilteritinib) • azacitidine • enzomenib (DSP-5336)
    4d
    US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov)
    P1/2, N=13, Completed, ExCellThera inc. | Recruiting --> Completed | Trial completion date: Jun 2027 --> Dec 2025 | Trial primary completion date: Jun 2026 --> Dec 2025
    Trial completion • Trial completion date • Trial primary completion date
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    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
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    cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan • Zemcelpro (dorocubicel)
    4d
    NCI-2019-03057: Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation (clinicaltrials.gov)
    P2, N=14, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Feb 2026 --> Mar 2027
    Trial completion date
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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    IDH2 mutation • IDH1 R132 • IDH2 R172
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    Lynparza (olaparib)
    4d
    A Proof of Concept Pilot Study of the Addition of Venetoclax to Standard Remission Induction Chemotherapy Fludarabine or Cladrabine, Cytarabine, and Granulocyte Colony Stimulating Factor (G-CSF) (FLAG or CLAG) for Frontline Therapy of Secondary Acute Myeloid Leukemia (clinicaltrials.gov)
    P2, N=20, Suspended, University of Michigan Rogel Cancer Center | Recruiting --> Suspended
    Trial suspension
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    Venclexta (venetoclax) • cytarabine • fludarabine IV
    4d
    Phase IB/II of CPX-351 for Relapse Prevention in AML (clinicaltrials.gov)
    P1/2, N=24, Recruiting, Georgetown University | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
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    Vyxeos (cytarabine/daunorubicin liposomal formulation)