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    BIOMARKER:

    Chr del(5q)

    5d
    Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations (clinicaltrials.gov)
    P2, N=25, Suspended, Abhay Singh, MD MPH | Recruiting --> Suspended
    Trial suspension • Tumor mutational burden
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    TET2 (Tet Methylcytosine Dioxygenase 2)
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    TET2 mutation • Chr del(5q)
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    Promacta (eltrombopag)
    7d
    Busulfan, Etoposide, and Total-Body Irradiation in Treating Patients Undergoing Donor Stem Cell or Bone Marrow Transplant for Advanced Hematologic Cancer (clinicaltrials.gov)
    P2, N=30, Completed, City of Hope Medical Center | Active, not recruiting --> Completed
    Trial completion
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    Chr del(5q)
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    etoposide IV • busulfan • cyclosporine
    21d
    Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (clinicaltrials.gov)
    P1, N=102, Active, not recruiting, Jacqueline Garcia, MD | Trial completion date: Mar 2027 --> Mar 2030
    Trial 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)
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    TP53 mutation • KRAS mutation • NRAS mutation • RUNX1 mutation • RAS mutation • ASXL1 mutation • CBL mutation • Chr del(5q)
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    Venclexta (venetoclax) • azacitidine • Inqovi (decitabine/cedazuridine) • fludarabine IV • busulfan
    23d
    5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies (clinicaltrials.gov)
    P1, N=20, Recruiting, Benjamin Tomlinson | Trial completion date: Dec 2026 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Nov 2026
    Trial completion date • Trial primary completion date
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    ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
    |
    Chr del(5q)
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    azacitidine • decitabine
    24d
    TP53-mutant AML with ribosomal gene loss exhibits impaired protein translation and sensitivity to HSP90 inhibition. (PubMed, Sci Adv)
    Chemical screening identified HSP90 inhibition as a selective vulnerability in AML with low RPG expression. These findings highlight a previously unappreciated TP53-altered AML subset characterized by converging genomic and translational defects and suggest that ribosomal stress may serve as a therapeutic entry point for targeted intervention of this patient subgroup.
    Journal
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    TP53 (Tumor protein P53) • HSP90AA1 (Heat Shock Protein 90 Alpha Family Class A Member 1Heat Shock Protein 90 Alpha Family Class A Member 1)
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    TP53 mutation • Chr del(17p) • Chr del(5q)
    1m
    How should myelodysplastic neoplasms with isolated deletion 5q and TP53 multihit alterations be classified? (PubMed, Leukemia)
    Moreover, the superior outcomes of MDS-del(5q) TP53 multihit patients persisted significant even when compared with MDS-LB TP53 multihit cases without complex karyotype (survival of 70.2 vs 39.9 months; time to AML progression of 31.9 vs 11.4 months). These findings indicate that, in MDS-del(5q) the adverse impact of TP53 multihit alterations may be less important than in other MDS subtypes.
    Journal
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    TP53 (Tumor protein P53) • SF3B1 (Splicing Factor 3b Subunit 1)
    |
    SF3B1 mutation • Chr del(5q)
    2ms
    Acute Myeloid Leukemia, Myelodysplasia-Related (AML-MR), With del(5q) and Double Minutes Containing Chromosomal Segment 11q24 Leading to Amplification and Expression of FLI1. (PubMed, Case Rep Hematol)
    After eight years and treatment with lenalidomide with excellent clinical response, she developed progressive cytopenias and transformation to acute myeloid leukemia, myelodysplasia-related (AML-MR)...The patient was treated with combination azacitidine and venetoclax and an investigational immunotherapy within a clinical trial...Our findings expand the spectrum of dmin-associated oncogenic amplifications in myeloid neoplasms and highlight FLI1 and ETS1 as recurrent targets of 11q24-derived ecDNA amplification. Recognition of such rare events underscores the importance of integrative cytogenomic profiling for uncovering novel mechanisms of leukemic transformation and potential therapeutic targets.
    Journal • IO biomarker
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    TP53 (Tumor protein P53) • SF3B1 (Splicing Factor 3b Subunit 1) • KMT2A (Lysine Methyltransferase 2A) • FLI1 (Fli-1 Proto-Oncogene ETS Transcription Factor) • ETS1 (ETS Proto-Oncogene 1) • EGR1 (Early Growth Response 1)
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    TP53 mutation • SF3B1 mutation • Chr del(5q)
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    Venclexta (venetoclax) • lenalidomide • azacitidine
    2ms
    A Single-arm, Prospective Study of a Cladribine-Bridged LABU Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Relapsed/Refractory MDS/AML in Elderly Patients (clinicaltrials.gov)
    P=N/A, N=30, Recruiting, The First Affiliated Hospital of Soochow University
    New trial
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    TP53 (Tumor protein P53) • SF3B1 (Splicing Factor 3b Subunit 1)
    |
    TP53 mutation • SF3B1 mutation • Chr del(5q)
    |
    cytarabine • azacitidine • cladribine • melphalan • busulfan
    3ms
    Revumenib in Combination With 7+3 + Midostaurin in AML (clinicaltrials.gov)
    P1, N=22, Recruiting, Richard Stone, MD | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • SRSF2 (Serine and arginine rich splicing factor 2) • CREBBP (CREB binding protein) • 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) • KAT6A (Lysine Acetyltransferase 6A) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • FLT3-ITD mutation • NPM1 mutation • Chr del(5q)
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    midostaurin • daunorubicin • Revuforj (revumenib)
    3ms
    ReSPECT: Study of Rezafungin Compared to Standard Antimicrobial Regimen for Prevention of Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation (clinicaltrials.gov)
    P3, N=602, Completed, Mundipharma Research Limited | Active, not recruiting --> Completed
    Trial completion
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    Chr del(5q)
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    Noxafil (posaconazole)
    3ms
    ReSPECT: Study of Rezafungin Compared to Standard Antimicrobial Regimen for Prevention of Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation (clinicaltrials.gov)
    P3, N=602, Active, not recruiting, Mundipharma Research Limited | Completed --> Active, not recruiting
    Enrollment closed
    |
    Chr del(5q)
    |
    Noxafil (posaconazole)
    3ms
    MBG453 in Lower Risk MDS (clinicaltrials.gov)
    P2, N=10, Terminated, Massachusetts General Hospital | N=20 --> 10 | Trial completion date: Nov 2026 --> Nov 2025 | Active, not recruiting --> Terminated; The trial closed early due to changes in support for the study drug.
    Enrollment change • Trial completion date • Trial termination
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    Chr del(5q)
    |
    sabatolimab (MBG453)