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    CANCER:

    Myelodysplastic Syndrome

    1d
    Mutational dynamics in patients with del(5q) MDS treated with lenalidomide prior to transfusion dependency-Molecular results from the Sintrarev clinical trial. (PubMed, Hemasphere)
    Treatment with low-dose Len in transfusion-independent del(5q) MDS reduced the mutational burden of most genes and did not promote the expansion of preexisting clones or AML progression, especially TP53-mutated clones. Early administration of Len in del(5q) MDS patients without TD may be an effective therapeutic approach with a manageable safety profile regarding clonal evolution.
    Journal • Tumor mutational burden
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    TP53 (Tumor protein P53) • TMB (Tumor Mutational Burden) • DNMT3A (DNA methyltransferase 1) • SF3B1 (Splicing Factor 3b Subunit 1)
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    TP53 mutation • SF3B1 mutation • Chr del(5q)
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    lenalidomide
    1d
    Bone marrow immunosuppressive states associate with survival after guadecitabine and atezolizumab therapy in HMA-R/R MDS. (PubMed, Blood Neoplasia)
    These findings suggest that chronic inflammatory and senescent microenvironmental states constrain effective immune activation despite combined epigenetic and immune checkpoint therapy. Here, we identify distinct bone marrow microenvironments associated with patient survival after combined epigenetic and immune checkpoint therapy and suggest candidate biomarkers to guide patient stratification in HMA-R/R MDS.
    Journal • PD(L)-1 Biomarker • IO biomarker
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    CD34 (CD34 molecule)
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    Tecentriq (atezolizumab) • guadecitabine (SGI-110)
    2d
    Proteomics in myelodysplastic syndromes: towards novel prognostic and diagnostic biomarkers. (PubMed, Biochem Med (Zagreb))
    Collectively, these proteomic candidates illustrate the complex interplay of signaling, immune regulation, bone microenvironment, and metabolism in MDS. Their validation in clinical cohorts could enable early detection, refined risk stratification, and new therapeutic avenues, positioning proteomics as a central tool in the future management of MDS.
    Review • Journal
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    NTRK1 (Neurotrophic tyrosine kinase, receptor, type 1) • LDHA (Lactate dehydrogenase A) • SAA1 (Serum Amyloid A1) • THBS1 (Thrombospondin 1) • PTK7 (Protein Tyrosine Kinase 7) • CLU (Clusterin) • STAT1 (Signal Transducer And Activator Of Transcription 1) • CRTAM (Cytotoxic And Regulatory T Cell Molecule) • MAST4 (Microtubule Associated Serine/Threonine Kinase Family Member 4) • TLN1 (Talin 1) • VCL (Vinculin) • CEP55 (Centrosomal Protein 55) • IGF2BP3 (Insulin Like Growth Factor 2 MRNA Binding Protein 3) • PAK6 (P21 (RAC1) Activated Kinase 6) • PRDM16 (PR/SET Domain 16)
    2d
    Allogeneic Transplantation in the rare disease MDS/MPN with Neutrophilia: Age and Disease Burden Determine Outcome. (PubMed, Transplant Cell Ther)
    Although limited by its retrospective character, small sample size and incomplete molecular data, this study shows that long-term survival after allo-HCT is achievable in patients with MDS/MPN with Neutrophilia, particularly in younger individuals with low disease burden. However, relapse and NRM remain major challenges underscoring the need for optimized post-transplant strategies.
    Journal
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    TET2 (Tet Methylcytosine Dioxygenase 2)
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    TET2 mutation • EZH2 mutation
    2d
    Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis (clinicaltrials.gov)
    P2, N=56, Recruiting, National Heart, Lung, and Blood Institute (NHLBI) | Trial primary completion date: Jun 2026 --> Jun 2027
    Trial primary completion date
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    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
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    cyclophosphamide
    2d
    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
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
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    IDH1 mutation
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    azacitidine • Rezlidhia (olutasidenib)
    3d
    MAXILUS: A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants (clinicaltrials.gov)
    P3, N=106, Active, not recruiting, Bristol-Myers Squibb | Trial completion date: Dec 2027 --> Sep 2027
    Trial completion date
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    Reblozyl (luspatercept-aamt)
    3d
    AMICUS: Identification of Molecular Defects in Idiopathic Cytopenia of Undetermined Significance (clinicaltrials.gov)
    P=N/A, N=2, Terminated, Centre Hospitalier Universitaire, Amiens | N=10 --> 2 | Unknown status --> Terminated; Difficult recruitment
    Enrollment change • Trial termination
    3d
    A Phase 1b/2 Study of Ropeginterferon Alfa-2b Added to Azacitidine in Newly Diagnosed MDS/AML (≥ 10% Blasts) With TP53 Mutations (clinicaltrials.gov)
    P1/2, N=30, Not yet recruiting, M.D. Anderson Cancer Center
    New P1/2 trial
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    TP53 (Tumor protein P53)
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    TP53 mutation
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    azacitidine • Besremi (ropeginterferon alfa-2b-njft)
    3d
    AG120-C-001: Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation (clinicaltrials.gov)
    P1, N=291, Recruiting, Institut de Recherches Internationales Servier | Trial completion date: Mar 2026 --> Dec 2026 | Trial primary completion date: Mar 2026 --> Dec 2026
    Trial completion date • Trial primary completion date
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
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    IDH1 R132
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    Tibsovo (ivosidenib)
    3d
    Prognostic and therapeutic implications of BRAF mutations in acute myeloid leukemia. (PubMed, Leukemia)
    In this cohort, BRAF-mutant AML patients had poor overall survival with currently available treatments, including venetoclax-based regimens. Drug sensitivity data suggest possible avenues for targeted treatment of BRAF-mutated AML.
    Journal
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    KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • NRAS (Neuroblastoma RAS viral oncogene homolog)
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    KRAS mutation • BRAF mutation • BRAF V600 • RAS mutation
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    Venclexta (venetoclax)
    6d
    A Phase Ib Trial of Azacitidine, Venetoclax and Allogeneic NK Cells for Acute Myeloid Leukemia (ADVENT-AML) (clinicaltrials.gov)
    P1, N=32, Recruiting, M.D. Anderson Cancer Center | Trial primary completion date: Jun 2026 --> Jun 2028
    Trial primary completion date
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    FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • BCL2 (B-cell CLL/lymphoma 2) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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    Venclexta (venetoclax) • azacitidine