☆ 4.7 Review

Translating recent advances in the pathogenesis of acute myeloid leukemia to the clinic

GENES & DEVELOPMENT (2022)

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Article Hematology

Phase 1 study of anti-CD47 monoclonal antibody CC-90002 in patients with relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes

Amer M. Zeidan et al.

Summary: Y CC-90002 is an anti-CD47 antibody that inhibits CD47-SIRP alpha interaction and enables macrophage-mediated killing of tumor cells in hematological cancer cell lines. The phase 1 clinical trial in patients with relapsed/refractory AML or high-risk MDS showed that CC-90002 led to mostly adverse events such as diarrhea and thrombocytopenia, with no objective responses observed. Further studies are needed to understand the lack of monotherapy activity and the development of anti-drug antibodies in this treatment.

ANNALS OF HEMATOLOGY (2022)

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Article Hematology

Effective therapy for AML with RUNX1 mutation by cotreatment with inhibitors of protein translation and BCL2

Christopher P. Mill et al.

Summary: Most RUNX1 mutations in acute myeloid leukemia (AML) are loss-of-function mutations. AML patients expressing mtRUNX1 have worse clinical outcomes than those without mutant RUNX1. Studies show that AML cells with mtRUNX1 have impaired ribosomal biogenesis and differentiation, as well as reduced levels of wild-type RUNX1, PU.1, and c-Myc. These cells are also more sensitive to protein translation inhibitors and a BCL2 inhibitor. Combination therapies involving omacetaxine are more effective for AML cells with mtRUNX1, with improved in vitro and in vivo efficacy.

BLOOD (2022)

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Review Hematology

Immune checkpoint inhibition in myeloid malignancies: Moving beyond the PD-1/PD-L1 and CTLA-4 pathways

Jan Philipp Bewersdorf et al.

Summary: Immune checkpoint inhibitors have shown mixed results in clinical trials in patients with acute myeloid leukemia and myelodysplastic syndromes, but there is optimism for the future of this field. Advances in understanding the immunologic landscape, potential biomarkers, and resistance mechanisms provide hope, but challenges such as the lack of validated biomarkers and the development of safe combination therapies still need to be addressed.

BLOOD REVIEWS (2021)

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Article Hematology

Synergistic activity of IDH1 inhibitor BAY1436032 with azacitidine in IDH1 mutant acute myeloid leukemia

Anuhar Chaturvedi et al.

Summary: The study demonstrates that the combination treatment of mIDH1 inhibitors and azacitidine has significant efficacy in AML patients, reducing leukemia stem cell numbers through different mechanisms and signaling pathways. The strong synergy between the two treatments is mediated through inhibition of MAPK/ERK and Rb/E2F signaling, suggesting an improved outcome for IDH1 mutated AML patients.

HAEMATOLOGICA (2021)

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Article Hematology

Venetoclax combines synergistically with FLT3 inhibition to effectively target leukemic cells in FLT3-ITD+ acute myeloid leukemia models

Raghuveer Singh Mali et al.

Summary: The combination of FLT3-ITD inhibition with venetoclax demonstrates impressive anti-tumor activity in FLT3-ITD+ AML preclinical models, highlighting the strong mechanistic rationale for clinical studies.

HAEMATOLOGICA (2021)

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Article Hematology

Flotetuzumab as salvage immunotherapy for refractory acute myeloid leukemia

Geoffrey L. Uy et al.

Summary: This study presents the results of a multicenter, open-label, phase 1/2 study of flotetuzumab in 88 adults with relapsed/refractory AML, showing clinical benefits in PIF/ER patients and encouraging evidence of activity. Flotetuzumab represents an innovative experimental approach associated with acceptable safety.

BLOOD (2021)

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Article Hematology

Ivosidenib or enasidenib combined with intensive chemotherapy in patients with newly diagnosed AML: a phase 1 study

Eytan M. Stein et al.

Summary: Ivosidenib and enasidenib, as targeted oral inhibitors, demonstrated good safety and efficacy when combined with intensive chemotherapy in newly diagnosed mIDH1/2 AML patients, achieving end-of-induction complete remission rates of 55% and 47% respectively.

BLOOD (2021)

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Meeting Abstract Hematology

Efficacy and Safety of Sabatolimab (MBG453) in Combination with Hypomethylating Agents (HMAs) in Patients (Pts) with Very High/High-Risk Myelodysplastic Syndrome (vHR/HRMDS) and Acute Myeloid Leukemia (AML): Final Analysis from a Phase Ib Study

Andrew M. Brunner et al.

BLOOD (2021)

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Meeting Abstract Hematology

AGILE: A Global, Randomized, Double-Blind, Phase 3 Study of Ivosidenib plus Azacitidine Versus Placebo plus Azacitidine in Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation

Pau Montesinos et al.

BLOOD (2021)

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Article Oncology

Mutant Isocitrate Dehydrogenase 1 Inhibitor Ivosidenib in Combination With Azacitidine for Newly Diagnosed Acute Myeloid Leukemia

Courtney D. DiNardo et al.

Summary: The combination therapy of ivosidenib and azacitidine showed promising results in treating IDH1-mutant acute myeloid leukemia, with a well-tolerated safety profile and deep, durable responses observed in patients, especially those achieving complete remission and mIDH1 mutation clearance in bone marrow cells.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Letter Oncology

Triplet therapy with venetoclax, FLT3 inhibitor and decitabine for FLT3-mutated acute myeloid leukemia

Abhishek Maiti et al.

BLOOD CANCER JOURNAL (2021)

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Article Hematology

Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML

Maximilian Stahl et al.

Summary: Combination therapy of venetoclax is effective in many patients with relapsed or refractory AML, with azacitidine+venetoclax showing higher response rates compared to low-dose cytarabine+venetoclax. Clinical and molecular characteristics of patients may predict treatment outcomes in this population.

BLOOD ADVANCES (2021)

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Review Oncology

T-cell-based immunotherapy of acute myeloid leukemia: current concepts and future developments

Naval Daver et al.

Summary: AML is a heterogeneous disease with a broad spectrum of molecular abnormalities, requiring multiple therapeutic approaches for long-term disease control. Recent advancements in understanding and targeting these molecular aberrations have led to rapid evolution in AML treatment, with a focus on immunotherapies harnessing T cells. Multiple T-cell-based immunotherapies, including bispecific antibodies, CAR T-cell therapies, and immune checkpoint inhibitors, are in various stages of development for AML treatment.

LEUKEMIA (2021)

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Review Biochemistry & Molecular Biology

Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease

Ettaib El Marabti et al.

Summary: RNA splicing is a process where non-protein coding sequences are removed from RNA to produce mature protein-coding mRNA. Genetic alterations and changes in splicing factors can lead to various human genetic diseases and cancer. Therapies based on oligonucleotides and small molecules targeting splicing factors are currently being developed for different genetic disorders and cancer.

TRENDS IN MOLECULAR MEDICINE (2021)

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Article Oncology

Splicing Patterns in SF3B1-Mutated Uveal Melanoma Generate Shared Immunogenic Tumor-Specific Neoepitopes

Jeremy Bigot et al.

Summary: Mutations in the splicing factor SF3B1 in uveal melanoma generate immunogenic neoantigens that can be recognized and killed by specific CD8(+) T cells, providing a potential new therapeutic target for tumors. These findings suggest that mutations in splicing factors may be a valuable source for developing novel therapeutic strategies for various types of cancers.

CANCER DISCOVERY (2021)

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Review Hematology

Splicing factor mutations in hematologic malignancies

Sisi Chen et al.

Summary: Mutations in genes encoding RNA splicing factors are now known to play a key role in various blood disorders, leading to research on biological pathways disrupted by altered splicing and the development of new therapeutic approaches.

BLOOD (2021)

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Article Hematology

Phase II study of azacitidine with pembrolizumab in patients with intermediate-1 or higher-risk myelodysplastic syndrome

Kelly S. Chien et al.

Summary: The study showed that PD-1 and PD-L1 expression is upregulated in CD34(+) bone marrow cells of MDS patients and combining azacitidine with pembrolizumab had a significant overall response rate of 76% in previously untreated patients. However, the combination therapy had a lower response rate in patients who had failed HMA treatment. Adverse events, such as pneumonia and arthralgias, were common, but manageable, with corticosteroids required in 43% of cases.

BRITISH JOURNAL OF HAEMATOLOGY (2021)

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Article Oncology

A phase 1b/2 study of azacitidine with PD-L1 antibody avelumab in relapsed/refractory acute myeloid leukemia

Kapil Saxena et al.

Summary: Patients with relapsed/refractory acute myeloid leukemia (AML) have limited treatment options. The combination of azacitidine and the anti-PD-L1 immune checkpoint inhibitor avelumab showed limited clinical activity. High expression of PD-L2 on bone marrow blasts may contribute to resistance to anti-PD-L1 therapy in AML.

CANCER (2021)

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Article Oncology

Outcomes of TP53-mutant acute myeloid leukemia with decitabine and venetoclax

Kunhwa Kim et al.

Summary: Patients with TP53(mut) AML show lower response rates and shorter survival when treated with DEC10-VEN compared to those with wild-type TP53 AML.

CANCER (2021)

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Article Biochemistry & Molecular Biology

Pharmacologic modulation of RNA splicing enhances anti-tumor immunity

Sydney X. Lu et al.

Summary: The study demonstrates that modulation of splicing through specific drug classes generates genuine neoantigens and enhances anti-tumor immunity, augmenting checkpoint immunotherapy.

CELL (2021)

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Article Oncology

Vorasidenib, a Dual Inhibitor of Mutant IDH1/2, in Recurrent or Progressive Glioma; Results of a First-in-Human Phase I Trial

Ingo K. Mellinghoff et al.

Summary: Vorasidenib demonstrated favorable safety profile and preliminary antitumor activity in patients with non-enhancing mIDH LGG, with a median progression-free survival of 36.8 months.

CLINICAL CANCER RESEARCH (2021)

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Review Oncology

Maintenance therapies in acute myeloid leukemia: the renaissance of an old therapeutic concept

Jan Philipp Bewersdorf et al.

Summary: Recent research has shown that the oral hypomethylating agent CC-486 and FLT3 inhibitor sorafenib have demonstrated some maintenance efficacy in AML patients. There are still issues surrounding patient selection, timing, duration, and safety of maintenance therapies that need to be addressed.

CURRENT OPINION IN ONCOLOGY (2021)

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Article Oncology

Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia

Courtney D. DiNardo et al.

Summary: The combination of fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin with venetoclax showed to be an effective treatment regimen in both newly diagnosed and relapsed or refractory AML patients, resulting in deep remissions and high rates of successful transplantation.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Review Oncology

Therapeutic implications of menin inhibition in acute leukemias

Ghayas C. Issa et al.

Summary: Menin inhibitors are novel targeted agents currently being developed for the treatment of genetically defined subsets of acute leukemia, showing promising early results in specific leukemia types, such as NPM1 mutations. These inhibitors target various gene regulatory roles of menin in leukemogenesis and are being investigated in clinical studies for relapsed acute leukemias.

LEUKEMIA (2021)

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Article Oncology

Phase I First-in-Human Dose Escalation Study of the oral SF3B1 modulator H3B-8800 in myeloid neoplasms

David P. Steensma et al.

Summary: H3B-8800 treatment was associated with mostly low-grade treatment-related adverse events and induced red blood cell transfusion independence in a subset of MDS patients.

LEUKEMIA (2021)

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Article Oncology

Venetoclax-based combinations in AML and high-risk MDS prior to and following allogeneic hematopoietic cell transplant

Jan Philipp Bewersdorf et al.

Summary: The study found that using venetoclax as consolidation therapy before allo-HCT in AML or MDS patients can improve the 12-month survival rate, and venetoclax-based salvage therapy after allo-HCT can provide a potential salvage treatment option with a 12-month survival rate around 43.4%.

LEUKEMIA & LYMPHOMA (2021)

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Meeting Abstract Hematology

Safety and Efficacy of Menin Inhibition in Patients (Pts) with MLL-Rearranged and NPM1 Mutant Acute Leukemia: A Phase (Ph) 1, First-in-Human Study of SNDX-5613 (AUGMENT 101)

Eytan M. Stein et al.

BLOOD (2021)

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Article Hematology

Safety and Efficacy of Pembrolizumab Prior to Allogeneic Stem Cell Transplantation for Acute Myelogenous Leukemia

Nicholas P. Tschernia et al.

Summary: This study compared the clinical outcomes of AML patients receiving high-dose cytarabine followed by pembrolizumab prior to alloSCT with a historical control group. One-year survival did not significantly differ between the groups, but there was no 100-day mortality in the ICI group, and no increase in the severity of GVHD.

TRANSPLANTATION AND CELLULAR THERAPY (2021)

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Article Oncology

A Therapeutic Strategy for Preferential Targeting of TET2-Mutant and TET Dioxygenase-Deficient Cells in Myeloid Neoplasms

Yihong Guan et al.

Summary: This study highlights the synthetic lethality between IDH1/2 mutations and TET dioxygenase deficiency. The TET-selective small-molecule inhibitor shows promising potential in restricting clonal evolution of TET2-mutant cells, indicating a new class of targeted agents for TET2-mutant neoplasms.

BLOOD CANCER DISCOVERY (2021)

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Article Oncology

Targeting public neoantigens for cancer immunotherapy

Alexander H. Pearlman et al.

Summary: Immunotherapy can target public neoantigens for cancer treatment, which focuses on proteins crucial for tumor growth and can be applied to a broad range of patients. However, identifying suitable public neoantigen targets and developing corresponding therapeutic agents still pose challenges.

NATURE CANCER (2021)

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Article Oncology

Epigenetic targeted therapy of stabilized BAP1 in ASXL1 gain-of-function mutated leukemia

Lu Wang et al.

Summary: The study uncovered gain-of-function mutations in ASXL1 that stabilize BAP1 protein and induce widespread epigenetic changes in myeloid neoplasms. By developing a chemical inhibitor targeting ASXL1-altered leukemia, the research provides a potential therapeutic strategy for this type of cancer. This breakthrough sheds light on the molecular mechanisms of ASXL1 mutations in leukemia pathogenesis and highlights small-molecular catalytic inhibitors of BAP1 as a promising targeted therapy.

NATURE CANCER (2021)

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Article Hematology

Molecular patterns of response and treatment failure after frontline venetoclax combinations in older patients with AML

C. D. DiNardo et al.

BLOOD (2020)

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Article Hematology

Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia

Gail J. Roboz et al.

BLOOD (2020)

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Article Hematology

Bispecific and split CAR T cells targeting CD13 and TIM3 eradicate acute myeloid leukemia

Xin He et al.

BLOOD (2020)

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Article Medicine, Research & Experimental

Menin inhibitor MI-3454 induces remission in MLL1-rearranged and NPM1-mutated models of leukemia

Szymon Klossowski et al.

JOURNAL OF CLINICAL INVESTIGATION (2020)

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Article Chemistry, Medicinal

Structure-Based Design and Identification of FT-2102 (Olutasidenib), a Potent Mutant-Selective IDH1 Inhibitor

Justin A. Caravella et al.

JOURNAL OF MEDICINAL CHEMISTRY (2020)

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Article Multidisciplinary Sciences

Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemia

Hannah J. Uckelmann et al.

SCIENCE (2020)

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Review Oncology

Therapeutic Targeting of the Macrophage Immune Checkpoint CD47 in Myeloid Malignancies

Mark P. Chao et al.

FRONTIERS IN ONCOLOGY (2020)

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Article Hematology

Venetoclax plus LDAC for newly diagnosed AML ineligible for intensive chemotherapy: a phase 3 randomized placebo-controlled trial

Andrew H. Wei et al.

BLOOD (2020)

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Article Hematology

SF3B1-mutant MDS as a distinct disease subtype: a proposal from the International Working Group for the Prognosis of MDS

Luca Malcovati et al.

BLOOD (2020)

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Review Immunology

Neoantigens in Hematologic Malignancies

Melinda A. Biernacki et al.

FRONTIERS IN IMMUNOLOGY (2020)

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Article Oncology

Updated results from phase I dose-escalation study of AMG 330, a bispecific T-cell engager molecule, in patients with relapsed/refractory acute myeloid leukemia (R/R AML).

Farhad Ravandi et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Article Oncology

Tolerability and efficacy of the first-in-class anti-CD47 antibody magrolimab combined with azacitidine in MDS and AML patients: Phase Ib results.

David Andrew Sallman et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Article Oncology

Phase Ib/II study of the IDH1-mutant inhibitor ivosidenib with the BCL2 inhibitor venetoclax +/- azacitidine in IDH1-mutated hematologic malignancies.

Curtis Andrew Lachowiez et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Article Oncology

Effect of enasidenib (ENA) plus azacitidine (AZA) on complete remission and overall response versus AZA monotherapy in mutant-IDH2 (mIDH2) newly diagnosed acute myeloid leukemia (ND-AML).

Courtney Denton Dinardo et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Article Hematology

Synergistic targeting of FLT3 mutations in AML via combined menin-MLL and FLT3 inhibition

Margarita M. Dzama et al.

BLOOD (2020)

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Article Hematology

TP53 mutations in myelodysplastic syndromes and secondary AML confer an immunosuppressive phenotype

David A. Sallman et al.

BLOOD (2020)

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Article Oncology

Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation (SORMAIN)

Andreas Burchert et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Article Hematology

Molecular mechanisms mediating relapse following ivosidenib monotherapy in IDH1-mutant relapsed or refractory AML

Sung Choe et al.

BLOOD ADVANCES (2020)

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Article Hematology

Patterns of care and clinical outcomes with cytarabine-anthracycline induction chemotherapy for AML patients in the United States

Amer M. Zeidan et al.

BLOOD ADVANCES (2020)

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Article Oncology

Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial

Li Xuan et al.

LANCET ONCOLOGY (2020)

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Article Oncology

Safety and efficacy of BAY1436032 in IDH1-mutant AML: phase I study results

Michael Heuser et al.

LEUKEMIA (2020)

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Article Medicine, General & Internal

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

C. D. DiNardo et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Article Cell Biology

Immune landscapes predict chemotherapy resistance and immunotherapy response in acute myeloid leukemia

Jayakumar Vadakekolathu et al.

SCIENCE TRANSLATIONAL MEDICINE (2020)

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Article Medicine, Research & Experimental

CBFB-MYH11 fusion neoantigen enables T cell recognition and killing of acute myeloid leukemia

Melinda A. Biernacki et al.

JOURNAL OF CLINICAL INVESTIGATION (2020)

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Article Multidisciplinary Sciences

Mutational landscape and clinical outcome of patients with de novo acute myeloid leukemia and rearrangements involving 11q23/KMT2A

Marius Bill et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)

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Article Hematology

10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial

Courtney D. DiNardo et al.

LANCET HAEMATOLOGY (2020)

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Article Hematology

TP53 abnormalities correlate with immune infiltration and associate with response to flotetuzumab immunotherapy in AML

Jayakumar Vadakekolathu et al.

BLOOD ADVANCES (2020)

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Review Hematology

CD123 bi-specific antibodies in development in AML: What do we know so far?

Michael J. Slade et al.

BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY (2020)

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Review Hematology

CD33 directed bispecific antibodies in acute myeloid leukemia

Mary C. Clark et al.

BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY (2020)

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Meeting Abstract Hematology

A Phase 1 Study of LY3410738, a First-in-Class Covalent Inhibitor of Mutant IDH in Advanced Myeloid Malignancies (Trial in Progress)

Eytan M. Stein et al.

BLOOD (2020)

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Meeting Abstract Hematology

Complete Responses in Relapsed/Refractory Acute Myeloid Leukemia (AML) Patients on a Weekly Dosing Schedule of Vibecotamab (XmAb14045), a CD123 x CD3 T Cell-Engaging Bispecific Antibody; Initial Results of a Phase 1 Study

Farhad Ravandi et al.

BLOOD (2020)

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Meeting Abstract Hematology

Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia in the Expansion Cohort of a Phase 1b Study

Naval Daver et al.

BLOOD (2020)

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Meeting Abstract Hematology

A Phase II Study of Double Immune Checkpoint Inhibitor Blockade with Nivolumab and Ipilimumab with or without Azacitidine in Patients with Myelodysplastic Syndrome (MDS)

Kiyomi Morita et al.

BLOOD (2020)

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Meeting Abstract Hematology

The STIMULUS Program: Clinical Trials Evaluating Sabatolimab (MBG453) Combination Therapy in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) or Acute Myeloid Leukemia (AML)

Amer M. Zeidan et al.

BLOOD (2020)

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Meeting Abstract Hematology

Efficacy and Safety of Sabatolimab (MBG453) in Combination with Hypomethylating Agents (HMAs) in Patients with Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (HR-MDS): Updated Results from a Phase 1b Study

Andrew M. Brunner et al.

BLOOD (2020)

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Meeting Abstract Hematology

Final Results from a Phase II Study Combining Azacitidine and Pembrolizumab in Patients with Higher-Risk Myelodysplastic Syndrome after Failure of Hypomethylating Agent Therapy

Kelly S. Chien et al.

BLOOD (2020)

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Article Oncology

Coexpression profile of leukemic stem cell markers for combinatorial targeted therapy in AML

S. Haubner et al.

LEUKEMIA (2019)

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Article Oncology

Targeting an RNA-Binding Protein Network in Acute Myeloid Leukemia

Eric Wang et al.

CANCER CELL (2019)

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Article Biochemistry & Molecular Biology

Immune signature drives leukemia escape and relapse after hematopoietic cell transplantation

Cristina Toffalori et al.

NATURE MEDICINE (2019)

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Article Hematology

RUNX1-targeted therapy for AML expressing somatic or germline mutation in RUNX1

Christopher P. Mill et al.

BLOOD (2019)

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Article Hematology

Mutational landscape of the transcriptome offers putative targets for immunotherapy of myeloproliferative neoplasms

Fiorella Schischlik et al.

BLOOD (2019)

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Review Hematology

Epidemiology of acute myeloid leukemia: Recent progress and enduring challenges

Rory M. Shallis et al.

BLOOD REVIEWS (2019)

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Article Oncology

Inhibition of Bcl-2 Synergistically Enhances the Antileukemic Activity of Midostaurin and Gilteritinib in Preclinical Models of FLT3-Mutated Acute Myeloid Leukemia

Jun Ma et al.

CLINICAL CANCER RESEARCH (2019)

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Article Oncology

Acute Myeloid Leukemia, Version 3.2019

Martin S. Tallman et al.

JOURNAL OF THE NATIONAL COMPREHENSIVE CANCER NETWORK (2019)

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Article Oncology

Temporal patterns and predictors of receiving no active treatment among older patients with acute myeloid leukemia in the United States: A population-level analysis

Amer M. Zeidan et al.

CANCER (2019)

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Article Oncology

Therapeutic Targeting of RNA Splicing Catalysis through Inhibition of Protein Arginine Methylation

Jia Yi Fong et al.

CANCER CELL (2019)

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Article Hematology

Idarubicin, cytarabine, and nivolumab in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a single-arm, phase 2 study

Farhad Ravandi et al.

LANCET HAEMATOLOGY (2019)

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Meeting Abstract Hematology

Use of CD70 Targeted Chimeric Antigen Receptor (CAR) T Cells for the Treatment of Acute Myeloid Leukemia (AML)

Mark Leick et al.

BLOOD (2019)

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Meeting Abstract Hematology

Azacytidine Sensitizes AML Cells for Effective Elimination By CD123 CAR T-Cells

Nadia El Khawanky et al.

BLOOD (2019)

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Meeting Abstract Hematology

A Phase I Study of CC-90002, a Monoclonal Antibody Targeting CD47, in Patients with Relapsed and/or Refractory (R/R) Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS): Final Results

Amer M. Zeidan et al.

BLOOD (2019)

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Meeting Abstract Hematology

Phase 1-2 Study of Pembrolizumab Combined with the Anti-LAG-3 Antibody MK-4280 for the Treatment of Hematologic Malignancies

Philippe Armand et al.

BLOOD (2019)

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Review Hematology

Immunotherapy in acute myeloid leukemia and myelodysplastic syndromes: The dawn of a new era?

Yuxin Liu et al.

BLOOD REVIEWS (2019)

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Article Oncology

The distribution of T-cell subsets and the expression of immune checkpoint receptors and ligands in patients with newly diagnosed and relapsed acute myeloid leukemia

Patrick Williams et al.

CANCER (2019)

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Meeting Abstract Hematology

Preliminary Results from a Phase 1 First-in-Human Study of AMG 673, a Novel Half-Life Extended (HLE) Anti-CD33/CD3 BiTE® (Bispecific T-Cell Engager) in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Marion y Subklewe et al.

BLOOD (2019)

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Meeting Abstract Hematology

Phase 1 First-in-Human Trial of AMV564, a Bivalent Bispecific (2:2) CD33/CD3 T-Cell Engager, in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML)

Peter Westervelt et al.

BLOOD (2019)

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Meeting Abstract Hematology

Olutasidenib (FT-2102) Induces Rapid Remissions in Patients with IDH1-Mutant Myelodysplastic Syndrome: Results of Phase 1/2 Single Agent Treatment and Combination with Azacitidine

Jorge E. Cortes et al.

BLOOD (2019)

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Meeting Abstract Hematology

Efficacy and Safety of Azacitidine (AZA) in Combination with the Anti-PD-L1 Durvalumab (durva) for the Front-Line Treatment of Older Patients (pts) with Acute Myeloid Leukemia (AML) Who Are Unfit for Intensive Chemotherapy (IC) and Pts with Higher-Risk Myelodysplastic Syndromes (HR-MDS): Results from a Large, International, Randomized Phase 2 Study

Amer M. Zeidan et al.

BLOOD (2019)

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Meeting Abstract Hematology

Olutasidenib (FT-2102), an IDH1m Inhibitor As a Single Agent or in Combination with Azacitidine, Induces Deep Clinical Responses with Mutation Clearance in Patients with Acute Myeloid Leukemia Treated in a Phase 1 Dose Escalation and Expansion Study

Justin M. Watts et al.

BLOOD (2019)

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Article Oncology

A Menin-MLL Inhibitor Induces Specific Chromatin Changes and Eradicates Disease in Models of MLL-Rearranged Leukemia

Andrei V. Krivtsov et al.

CANCER CELL (2019)

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Article Medicine, Research & Experimental

Mutated nucleophosmin 1 as immunotherapy target in acute myeloid leukemia

Dyantha van der Lee et al.

JOURNAL OF CLINICAL INVESTIGATION (2019)

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Article Oncology

Randomized comparison of low dose cytarabine with or without glasdegib in patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome

Jorge E. Cortes et al.

LEUKEMIA (2019)

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Article Medicine, General & Internal

Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma

Stephen J. Schuster et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

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Article Oncology

Efficacy, Safety, and Biomarkers of Response to Azacitidine and Nivolumab in Relapsed/Refractory Acute Myeloid Leukemia: A Nonrandomized, Open-Label, Phase II Study

Naval Daver et al.

CANCER DISCOVERY (2019)

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Article Oncology

Enhancing venetoclax activity in acute myeloid leukemia by co-targeting MCL1

T-C Teh et al.

LEUKEMIA (2018)

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Article Oncology

The MLL recombinome of acute leukemias in 2017

C. Meyer et al.

LEUKEMIA (2018)

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Review Oncology

The emerging role of immune checkpoint based approaches in AML and MDS

Prajwal Boddu et al.

LEUKEMIA & LYMPHOMA (2018)

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Article Hematology

Blinatumomab for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukemia

Nicola Goekbuget et al.

BLOOD (2018)

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Review Hematology

Blocking don't eat me signal of CD47-SIRPα in hematological malignancies, an in-depth review

Atlantis Russ et al.

BLOOD REVIEWS (2018)

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Article Oncology

Final results of a phase 2, open-label study of indisulam, idarubicin, and cytarabine in patients with relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome

Rita Assi et al.

CANCER (2018)

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Article Biochemistry & Molecular Biology

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia

Miriam Y. Kim et al.

CELL (2018)

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Article Oncology

A Multi-center Phase I Trial of Ipilimumab in Patients with Myelodysplastic Syndromes following Hypomethylating Agent Failure

Amer M. Zeidan et al.

CLINICAL CANCER RESEARCH (2018)

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Article Biochemistry & Molecular Biology

Somatic mutations precede myeloid leukemia years before diagnosis

Pinkal Desai et al.

NATURE MEDICINE (2018)

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Article Biochemistry & Molecular Biology

H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers

Michael Seiler et al.

NATURE MEDICINE (2018)

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Article Medicine, General & Internal

Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML

C. D. DiNardo et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Article Medicine, General & Internal

Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia

Jae H. Park et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Article Multidisciplinary Sciences

Mutant ASXL1 cooperates with BAP1 to promote myeloid leukaemogenesis

Shuhei Asada et al.

NATURE COMMUNICATIONS (2018)

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Article Immunology

Targeting LAG-3 and PD-1 to Enhance T Cell Activation by Antigen-Presenting Cells

Felix S. Lichtenegger et al.

FRONTIERS IN IMMUNOLOGY (2018)

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Article Oncology

Synthetic Lethal and Convergent Biological Effects of Cancer-Associated Spliceosomal Gene Mutations

Stanley Chun-Wei Lee et al.

CANCER CELL (2018)

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Article Multidisciplinary Sciences

Acquired resistance to IDH inhibition through trans or cis dimer-interface mutations

Andrew M. Intlekofer et al.

NATURE (2018)

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Meeting Abstract Hematology

Remissions in Relapse/Refractory Acute Myeloid Leukemia Patients Following Treatment with NKG2D CAR-T Therapy without a Prior Preconditioning Chemotherapy

David A. Sallman et al.

BLOOD (2018)

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Meeting Abstract Hematology

First-in-Human CLL1-CD33 Compound CAR T Cell Therapy Induces Complete Remission in Patients with Refractory Acute Myeloid Leukemia: Update on Phase 1 Clinical Trial

Fang Liu et al.

BLOOD (2018)

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Review Hematology

Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel

Hartmut Doehner et al.

BLOOD (2017)

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Article Oncology

TTI-621 (SIRPαFc): A CD47-Blocking Innate Immune Checkpoint Inhibitor with Broad Antitumor Activity and Minimal Erythrocyte Binding

Penka S. Petrova et al.

CLINICAL CANCER RESEARCH (2017)

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Article Oncology

Complementary activities of DOT1L and Menin inhibitors in MLL-rearranged leukemia

C. Dafflon et al.

LEUKEMIA (2017)

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Article Oncology

Pan-mutant-IDH1 inhibitor BAY1436032 is highly effective against human IDH1 mutant acute myeloid leukemia in vivo

A. Chaturvedi et al.

LEUKEMIA (2017)

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Article Medicine, General & Internal

Clonal Hematopoiesis and Risk of Atherosclerotic Cardiovascular Disease

S. Jaiswal et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Medicine, General & Internal

Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation

R. M. Stone et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Medicine, General & Internal

Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia

Hagop Kantarjian et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Multidisciplinary Sciences

Anticancer sulfonamides target splicing by inducing RBM39 degradation via recruitment to DCAF15

Ting Han et al.

SCIENCE (2017)

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Article Genetics & Heredity

Analysis of 100,000 human cancer genomes reveals the landscape of tumor mutational burden

Zachary R. Chalmers et al.

GENOME MEDICINE (2017)

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Review Oncology

The genetics of myelodysplastic syndrome: from clonal haematopoiesis to secondary leukaemia

Adam S. Sperling et al.

NATURE REVIEWS CANCER (2017)

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Article Biochemistry & Molecular Biology

Restoration of TET2 Function Blocks Aberrant Self-Renewal and Leukemia Progression

Luisa Cimmino et al.

CELL (2017)

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Article Hematology

Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response

Michael D. Amatangelo et al.

BLOOD (2017)

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Article Hematology

Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia

Eytan M. Stein et al.

BLOOD (2017)

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Letter Biophysics

Severe acute GvHD following administration of ipilimumab for early relapse of AML after haploidentical stem cell transplantation

F-X Gros et al.

BONE MARROW TRANSPLANTATION (2017)

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Article Hematology

Clinical significance of somatic mutation in unexplained blood cytopenia

Luca Malcovati et al.

BLOOD (2017)

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Article Oncology

Blockade of the PD-1/PD-L1 axis augments lysis of AML cells by the CD33/CD3 BiTE antibody construct AMG 330: reversing a T-cell-induced immune escape mechanism

C. Krupka et al.

LEUKEMIA (2016)

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Article Oncology

TP53 mutations in newly diagnosed acute myeloid leukemia: Clinicomolecular characteristics, response to therapy, and outcomes

Tapan M. Kadia et al.

CANCER (2016)

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Article Oncology

T-Cell Immunoglobulin and ITIM Domain (TIGIT) Associates with CD8+ T-Cell Exhaustion and Poor Clinical Outcome in AML Patients

Yaxian Kong et al.

CLINICAL CANCER RESEARCH (2016)

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Review Cell Biology

Spliceosomal gene mutations in myelodysplasia: molecular links to clonal abnormalities of hematopoiesis

Daichi Inoue et al.

GENES & DEVELOPMENT (2016)

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Article Biochemistry & Molecular Biology

Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins

Stanley Chun-Wei Lee et al.

NATURE MEDICINE (2016)

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Article Medicine, General & Internal

TP53 and Decitabine in Acute Myeloid Leukemia and Myelodysplastic Syndromes

J. S. Welch et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

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Article Medicine, General & Internal

Ipilimumab for Patients with Relapse after Allogeneic Transplantation

Matthew S. Davids et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

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Article Medicine, General & Internal

Genomic Classification and Prognosis in Acute Myeloid Leukemia

Elli Papaemmanuil et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

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Article Cell Biology

Individualized vaccination of AML patients in remission is associated with induction of antileukemia immunity and prolonged remissions

Jacalyn Rosenblatt et al.

Science Translational Medicine (2016)

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Article Hematology

Clonal hematopoiesis of indeterminate potential and its distinction from myelodysplastic syndromes

David P. Steensma et al.

BLOOD (2015)

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Article Oncology

Pharmacologic Inhibition of the Menin-MLL Interaction Blocks Progression of MLL Leukemia In Vivo

Dmitry Borkin et al.

CANCER CELL (2015)

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Letter Hematology

MLL partial tandem duplication leukemia cells are sensitive to small molecule DOT1L inhibition

Michael W. M. Kuehn et al.

HAEMATOLOGICA (2015)

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Article Oncology

T cells are functionally not impaired in AML: increased PD-1 expression is only seen at time of relapse and correlates with a shift towards the memory T cell compartment

Frauke M. Schnorfeil et al.

JOURNAL OF HEMATOLOGY & ONCOLOGY (2015)

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Article Genetics & Heredity

The landscape of somatic mutations in infant MLL-rearranged acute lymphoblastic leukemias

Anna K. Andersson et al.

NATURE GENETICS (2015)

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Article Biochemistry & Molecular Biology

Isocitrate dehydrogenase 1 and 2 mutations induce BCL-2 dependence in acute myeloid leukemia

Steven M. Chan et al.

NATURE MEDICINE (2015)

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Article Multidisciplinary Sciences

Cancer-associated ASXL1 mutations may act as gain-of-function mutations of the ASXL1-BAP1 complex

Anand Balasubramani et al.

NATURE COMMUNICATIONS (2015)

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Article Oncology

PD-1hiTIM-3+ T cells associate with and predict leukemia relapse in AML patients post allogeneic stem cell transplantation

Y. Kong et al.

BLOOD CANCER JOURNAL (2015)

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Article Oncology

Hypomethylation and up-regulation of PD-1 in T cells by azacytidine in MDS/AML patients: A rationale for combined targeting of PD-1 and DNA methylation

Andreas D. Orskov et al.

ONCOTARGET (2015)

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Letter Hematology

MLL partial tandem duplication leukemia cells are sensitive to small molecule DOT1L inhibition

Michael W. M. Kuehn et al.

HAEMATOLOGICA (2015)

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Review Cell Biology

Bispecific T-cell engagers for cancer immunotherapy

Amelia M. Huehls et al.

IMMUNOLOGY AND CELL BIOLOGY (2015)

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Article Pathology

Next-generation sequencing of acute myeloid leukemia identifies the significance of TP53, U2AF1, ASXL1, and TET2 mutations

Robert S. Ohgami et al.

MODERN PATHOLOGY (2015)

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Article Oncology

Mapping the HLA ligandome landscape of acute myeloid leukemia: a targeted approach toward peptide-based immunotherapy

C. Berlin et al.

LEUKEMIA (2015)

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Article Hematology

TET2 mutations predict response to hypomethylating agents in myelodysplastic syndrome patients

Rafael Bejar et al.

BLOOD (2014)

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Article Oncology

Expression of PD-L1, PD-L2, PD-1 and CTLA4 in myelodysplastic syndromes is enhanced by treatment with hypomethylating agents

H. Yang et al.

LEUKEMIA (2014)

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Article Biochemistry & Molecular Biology

Age-related mutations associated with clonal hematopoietic expansion and malignancies

Mingchao Xie et al.

NATURE MEDICINE (2014)

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Article Medicine, General & Internal

Clonal Hematopoiesis and Blood-Cancer Risk Inferred from Blood DNA Sequence

Giulio Genovese et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

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Article Medicine, General & Internal

Age-Related Clonal Hematopoiesis Associated with Adverse Outcomes

Siddhartha Jaiswal et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

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Article Oncology

Distribution and levels of cell surface expression of CD33 and CD123 in acute myeloid leukemia

A. Ehninger et al.

BLOOD CANCER JOURNAL (2014)

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Article Medicine, General & Internal

Genomic and Epigenomic Landscapes of Adult De Novo Acute Myeloid Leukemia

Timothy J. Ley et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

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Article Hematology

TP53 alterations in acute myeloid leukemia with complex karyotype correlate with specific copy number alterations, monosomal karyotype, and dismal outcome

Frank G. Rueckner et al.

BLOOD (2012)

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Article Hematology

Frequency and prognostic impact of mutations in SRSF2, U2AF1, and ZRSR2 in patients with myelodysplastic syndromes

Felicitas Thol et al.

BLOOD (2012)

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Article Hematology

Structural insights into inhibition of the bivalent menin-MLL interaction by small molecules in leukemia

Aibin Shi et al.

BLOOD (2012)

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Article Medicine, General & Internal

Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): a randomised, open-label, phase 3 study

Sylvie Castaigne et al.

LANCET (2012)

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Article Genetics & Heredity

Recurrent somatic TET2 mutations in normal elderly individuals with clonal hematopoiesis

Lambert Busque et al.

NATURE GENETICS (2012)

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Article Oncology

Tet2 Loss Leads to Increased Hematopoietic Stem Cell Self-Renewal and Myeloid Transformation

Kelly Moran-Crusio et al.

CANCER CELL (2011)

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Article Medicine, General & Internal

Somatic SF3B1 Mutation in Myelodysplasia with Ring Sideroblasts

E. Papaemmanuil et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

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Article Cell & Tissue Engineering

TIM-3 Is a Promising Target to Selectively Kill Acute Myeloid Leukemia Stem Cells

Yoshikane Kikushige et al.

CELL STEM CELL (2010)

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Article Biochemistry & Molecular Biology

CD47 Is an Adverse Prognostic Factor and Therapeutic Antibody Target on Human Acute Myeloid Leukemia Stem Cells

Ravindra Majeti et al.

CELL (2009)

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Article Medicine, General & Internal

Mutation in TET2 in Myeloid Cancers

Francois Delhommeau et al.

NEW ENGLAND JOURNAL OF MEDICINE (2009)

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Article Oncology

Phase I safety and pharmacokinetic study of CT-011, a humanized antibody interacting with PD-1, in patients with advanced hematologic malignancies

Raanan Berger et al.

CLINICAL CANCER RESEARCH (2008)

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Article Oncology

HOX expression patterns identify a common signature for favorable AML

M. Andreeff et al.

LEUKEMIA (2008)

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Article Oncology

Strictly target cell-dependent activation of T cells by bispecific single-chain antibody constructs of the BiTE class

Klaus Brischwein et al.

JOURNAL OF IMMUNOTHERAPY (2007)

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Review Oncology

MLL translocations, histone modifications and leukaemia stem-cell development

Andrei V. Krivtsov et al.

NATURE REVIEWS CANCER (2007)

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Article Oncology

Pediatric acute myeloid leukemia with NPM1 mutations is characterized by a gene expression profile with dysregulated HOX gene expression distinct from MLL-rearranged leukemias

C. G. Mullighan et al.

LEUKEMIA (2007)

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Article Multidisciplinary Sciences

The tumor suppressor menin regulates hematopoiesis and myeloid transformation by influencing Hox gene expression

YX Chen et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)

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Article Biochemistry & Molecular Biology

The menin tumor suppressor protein is an essential oncogenic cofactor for MLL-associated leukemogenesis

A Yokoyama et al.

CELL (2005)

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Article Oncology

Extremely potent, rapid and costimulation-independent cytotoxic T-cell response against lymphoma cells catalyzed by a single-chain bispecific antibody

T Dreier et al.

INTERNATIONAL JOURNAL OF CANCER (2002)

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