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CANCER:

Myelodysplastic Syndrome

1d
New P2 trial
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TP53 (Tumor protein P53) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • MECOM (MDS1 And EVI1 Complex Locus) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
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TP53 mutation
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cyclophosphamide • fludarabine IV
1d
MCC-20963: Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL) (clinicaltrials.gov)
P2, N=25, Completed, H. Lee Moffitt Cancer Center and Research Institute | Active, not recruiting --> Completed
Trial completion
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
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Inrebic (fedratinib)
1d
TMF-Allo: Faecal Microbiota Transplantation After Allogeneic Stem Cell Transplantation (clinicaltrials.gov)
P2, N=150, Recruiting, University Hospital, Clermont-Ferrand | Trial completion date: Dec 2027 --> May 2030 | Trial primary completion date: Oct 2026 --> May 2030
Trial completion date • Trial primary completion date
1d
MP0533-CP101: Study of MP0533 in Patients Acute Myeloid Leukemia or Myelodysplastic Syndrome (clinicaltrials.gov)
P1/2, N=249, Active, not recruiting, Molecular Partners AG | Recruiting --> Active, not recruiting | Trial completion date: Dec 2029 --> May 2027 | Trial primary completion date: Dec 2027 --> May 2026
Enrollment closed • Trial completion date • Trial primary completion date • First-in-human
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Venclexta (venetoclax) • azacitidine • Gazyva (obinutuzumab) • MP0533
1d
VA Induction and Bridging Transplantation for Hypoplastic/Secondary AML (clinicaltrials.gov)
P=N/A, N=50, Not yet recruiting, The First Affiliated Hospital of Soochow University
New trial
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Venclexta (venetoclax) • azacitidine
2d
A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS) (clinicaltrials.gov)
P2, N=10, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting | N=40 --> 10
Enrollment closed • Enrollment change
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Reblozyl (luspatercept-aamt)
2d
A Rare Case of Concurrent Chronic Lymphocytic Leukemia and Therapy-Unrelated Acute Myeloid Leukemia That Evolved From Myelodysplastic Neoplasm. (PubMed, Cureus)
Given the differing cytogenetics among each cell population, the CLL and AML may have developed as separate clonal processes. With a further understanding of the pathogenic mechanism, this may influence the treatment decision-making process.
Journal
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NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1)
2d
Prognostic value of measurable residual disease in high-risk MDS after intensive chemotherapy in HOVON-SAKK studies. (PubMed, Hemasphere)
Lastly, an exploratory cross-disease comparison showed that MRD-negative (MRDneg) hrMDS patients had similar survival outcomes to MRD-positive (MRDpos) AML patients. These findings demonstrate the prognostic value of MFC-MRD in intensively treated hrMDS and provide rationale for prospective trials of MRD-informed transplant and post-remission strategies.
Journal
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TP53 (Tumor protein P53)
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TP53 mutation
2d
Early Transplantation After Pretransplant Reduction of TP53-Altered Clones and Prolonged Dose-Adjusted Azacitidine Maintenance: A Three-Case Series. (PubMed, EJHaem)
Pretransplant treatment was intentionally limited to 2-3 cycles: three cycles of Aza in Case 1, intensive induction chemotherapy followed by one cycle of Aza in Case 2, and two cycles of venetoclax plus Aza in Case 3. In addition, for patients with FISH-detectable TP53 abnormalities, serial FISH may provide a practical adjunct for pretransplant decision-making in routine practice. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.
Journal
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TP53 (Tumor protein P53)
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Venclexta (venetoclax) • azacitidine
2d
Rapid Transformation of Myelodysplastic Neoplasm to Myelodysplasia-related Acute Myeloid Leukemia with Central Nervous System Leukemia in a Young Adult. (PubMed, Ann Afr Med)
Despite standard induction and intrathecal chemotherapy, bone marrow assessment demonstrated primary refractory disease. This case highlights aggressive secondary AML with adverse biology, CNS involvement, and induction failure.
Journal
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DNMT3A (DNA methyltransferase 1) • RUNX1 (RUNX Family Transcription Factor 1) • TET2 (Tet Methylcytosine Dioxygenase 2) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CD34 (CD34 molecule) • CD7 (CD7 Molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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TET2 mutation
2d
Fibrotic marrow limiting morphologic classification in MDS/MPN with SF3B1 mutation and thrombocytosis: diagnostic implications under the ICC 2022 framework. (PubMed, Virchows Arch)
After ruxolitinib therapy, he later developed leukocytosis and 2% circulating blasts. Repeat marrow demonstrated ≥ 15% ring sideroblasts. Retrospectively, the initial biopsy fulfills ICC 2022 criteria for MDS/MPN-SF3B1-T, highlighting the diagnostic value of genetics-integrated classification in fibrotic marrows.
Journal
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NRAS (Neuroblastoma RAS viral oncogene homolog) • JAK2 (Janus kinase 2) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1)
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SF3B1 mutation • NRAS G12
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Jakafi (ruxolitinib)
2d
Comparative efficacy of donor lymphocyte infusions in augmenting graft-versus-leukemia effect after allogeneic hematopoietic stem cell transplantation for patients with myeloid malignancies. (PubMed, Acta Haematol)
DLI is an effective treatment strategy for post-transplant relapse of all myeloid malignancies, with specific genetic subtypes showing poorer outcomes and survival.
Journal
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KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • NRAS (Neuroblastoma RAS viral oncogene homolog) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1)
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TP53 mutation • KRAS mutation • NRAS mutation