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Journal Articles

Therapeutic options in VEXAS syndrome: insights from a retrospective series

Estelle Bourbon, Maël Heiblig, Mathieu Gerfaud Valentin, Thomas Barba, Cécile-Audrey Durel, Jean Christophe Lega, Fiorenza Barraco, Pascal Sève, Yvan Jamilloux, Pierre Sujobert

Blood (2021) 137 (26): 3682–3684.

https://doi.org/10.1182/blood.2020010177

Published: 2021

Includes: Supplemental data

Journal Articles

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Malte U Ritter, Diana Amend, Yun Xu, Maksim Klimiankou, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 434.

https://doi.org/10.1182/blood-2019-129991

Published: 2019

Journal Articles

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Casey D.S. Katerndahl, PhD, Timothy J Ley, MD, Michelle A Cai, Timothy P Rooney, Jr., Sai Mukund Ramakrishnan, MS, Sridhar Nonavinkere Srivatsan, MS, Christopher A Miller, PhD

Blood (2019) 134 (Supplement_1): 534.

https://doi.org/10.1182/blood-2019-131883

Published: 2019

Journal Articles

Identification of Recurrent Alternative RNA Splicing in Adverse-Risk Acute Myeloid Leukemia

Govardhan Anande, Ashwin Unnikrishnan, PhD, Nandan Deshpande, PhD, Sylvain Mareschal, Aarif M. N. Batcha, Tobias Herold, MD, Sören Lehmann, MD PhD, Marc Wilkins, PhD, Jason Wong, PhD, John Pimanda, MBBS, PhD

Blood (2019) 134 (Supplement_1): 457.

https://doi.org/10.1182/blood-2019-129537

Published: 2019

Journal Articles

Myeloid-Lineage Enhancers Drive Oncogene Synergy and Represent a Novel Therapeutic Target in CSF3R-CEBPA Mutant AML

Theodore Braun, MD PhD, Cody Coblentz, Sarah A Carratt, PhD BS, Mariam Okhovat, PhD, Amy Foley, MEng., Zachary Schonrock, BS, Kimberly Nevonen, Rhonda E. Ries, MA, Shannon K. McWeeney, PhD, Claus Nerlov, PhD, Soheil Meshinchi, MD PhD, Lucia Carbone, PhD, Brian J. Druker, Julia E. Maxson, PhD

Blood (2019) 134 (Supplement_1): 543.

https://doi.org/10.1182/blood-2019-124773

Published: 2019

Journal Articles

Results of a Phase III Double-Blind Study of the Addition of Tocilizumab Vs. Placebo to Cyclosporin/Methotrexate Gvhd Prophylaxis after HLA-Matched Allogeneic Stem Cell Transplantation

Glen A Kennedy, MBBS FRACP FRCPA, Siok-Keen Tey, MBBS,PhDFRACP,FRCPA, Cameron Curley, MBBS, FRACP, FRCPA, Jason P Butler, MBBS, MMedSc, FRACP, FRCPA, Ashish Misra, MBBS, FRACP, Elango Subramoniapillai, MBBS, FRACP, James Morton, MBBS, FRACP, FRCPA, Simon Durrant, FRCP,FRCPath,MBBS, Andrea S Hendon, MBBS, PhD FRACP, FRCPA, John Moore, MDFRACP,FRCPA, David Ritchie, MD PhD, David Gottlieb, MBBS, MD FRACP, FRCPA, Julian Cooney, FRACP,FRCPA,MBBS,, Luke Buizen, BSc (Hons), MSc, Sanjoy K Paul, PhD, Geoff R. Hill, MD

Blood (2019) 134 (Supplement_1): 368.

https://doi.org/10.1182/blood-2019-126285

Published: 2019

Journal Articles

Stag2 Regulates Hematopoietic Differentiation and Self-Renewal through Alterations in Gene Expression and Topological Control

Blood (2019) 134 (Supplement_1): 279.

https://doi.org/10.1182/blood-2019-123464

Published: 2019

Journal Articles

Survival Outcomes in Patients with Intermediate Risk Acute Myeloid Leukemia: A Single-Center Retrospective Review

Lucia Garcia Mañó, Sandra Pérez, Bernardo López Andrade, MD, Maria Antonia Duran Pastor, Andrés Novo, MD, Antonio Gutiérrez, Antonia Sampol

Blood (2019) 134 (Supplement_1): 5112.

https://doi.org/10.1182/blood-2019-131987

Published: 2019

Journal Articles

Outcome of Patients with Newly-Diagnosed Therapy-Related AML (t-AML) and AML with Myelodysplasia-Related Changes (AML-MRC) Treated with Liposomal Daunorubicin and Cytarabine (CPX-351) at an Academic Community-Based Health System

John Khoury, MD, Nwabundo Anusim, MD, David Macari, MD, Angela Duenn, PharmD, Ishmael Jaiyesimi, DO, MS

Blood (2019) 134 (Supplement_1): 5117.

https://doi.org/10.1182/blood-2019-130776

Published: 2019

Journal Articles

Combination of Venetoclax and Hypomethylating Agents in Myeloid Malignancies in Patients Unfit for Intensive Chemotherapy - Real World Data from a Tertiary Care Centre in India. Is It Time to Bid Adieu to 3+7?

Sumeet Prakash Mirgh, MBBS, MDDM, Jyotsna Kapoor, MSc, Shinto Francis Thekuddan, MD DM, Vishvdeep Khushoo, MBBS, MDDM, Faran Naim, MD DNB, Ambar Garg, MD DNB, Narendra Agrawal, MD DM, Rayaz Ahmed, MDDM, Dinesh Bhurani, MD DM, FRCPA

Blood (2019) 134 (Supplement_1): 5149.

https://doi.org/10.1182/blood-2019-131746

Published: 2019

Journal Articles

A Phase 1a/b Dose Escalation Study of Apto-253 in Patients with Relapsed or Refractory AML or High-Risk MDS

Stephen B Howell, MD, Hongying Zhang, MD PhD, William G Rice, PhD

Blood (2019) 134 (Supplement_1): 5148.

https://doi.org/10.1182/blood-2019-129685

Published: 2019

Journal Articles

Assessing Efficacy and Safety of Daunorubicin and Cytarabine Liposomal in Patients with Acute Myeloid Leukemia

Elizabeth Rogers, PharmD, MPH, Maho Hibino, PharmD, Rebecca Garcia Hunt, PharmD, Leslie Renee Ellis, MD, Rupali Bhave, MD, Dianna S. Howard, MD, Susan Lyerly, P.A.-C., Sarah Dralle, NP, Megan Manuel, NP, Timothy S. Pardee, MD PhD, Bayard L Powell, MD

Blood (2019) 134 (Supplement_1): 5134.

https://doi.org/10.1182/blood-2019-131328

Published: 2019

Journal Articles

Outcomes of Patients Receiving Direct Oral Anticoagulants for Myeloproliferative Neoplasm Associated Venous Thromboembolism

Andrew Doyle, MBBS, BSc, FRPATH, Karen Breen, MD, Donal P. McLornan, MB, BCh (Hons), MRCP, PhD FRCPath, Deepti Radia, Beverley J. Hunt, Gavin Ling, MBBS, BSc, FRCPath, Claire N Harrison, Professor, Natalia Curto Garcia, MD MBBS

Blood (2019) 134 (Supplement_1): 4183.

https://doi.org/10.1182/blood-2019-128170

Published: 2019

Journal Articles

A Randomized, Open-Label, Multicenter, Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of KRT-232 Compared with Ruxolitinib in Patients with Phlebotomy-Dependent Polycythemia Vera

Jason Gotlib, MD MS, Nashat Gabrail, MD, Casey L. O'Connell, MD, Regina Garcia-Delgado, MD, Timothy Sbardellati, Wayne M. Rothbaum, Jesse McGreivy, Claire N Harrison, Professor, Jean-Jacques Kiladjian, MD PhD

Blood (2019) 134 (Supplement_1): 4168.

https://doi.org/10.1182/blood-2019-123546

Published: 2019

Journal Articles

Cord Blood Regulatory T Cells Prevent Mutiple Myeloma Progression By Suppressing Inflammation

Mitsutaka Nishimoto, MD PhD, Ke Zeng, MD PhD, Meixian Huang, Mi-Ae Lyu, PhD, Nina D. Shah, MD, Swaminathan P. Iyer, MD, Robert Z. Orlowski, MD, PhD, Simrit Parmar, MBBS

Blood (2019) 134 (Supplement_1): 3095.

https://doi.org/10.1182/blood-2019-128418

Published: 2019

Journal Articles

Single-Cell RNA Sequencing Reveals a Distinct Transcriptome Signature of Hematopoiesis in GATA2 Deficiency

Zhijie Wu, MD PhD, Shouguo Gao, PhD, Sachiko Kajigaya, PhD, Jinguo Chen, PhD, Rongye Shi, BS, Carrie Diamond, MD, Cindy Palmer, BSN, Amy P. Hsu, Katherine R. Calvo, MD PhD, Dennis D. Hickstein, MD, Steven M. Holland, MD, Neal S. Young, MD

Blood (2019) 134 (Supplement_1): 3735.

https://doi.org/10.1182/blood-2019-124774

Published: 2019

Journal Articles

CPX-351 As Induction Chemotherapy Yields Similar Responses and Survival Outcomes in Younger Patients (<60 Years Old) Compared to Older Patients (≥60 Years Old) with Acute Myeloid Leukemia

Dasom Lee, MD, Hannah H Asghari, MD, Yehuda E. Deutsch, MD, Onyee Chan, MD, Eric Padron, MD, Andrew T Kuykendall, MD, Alan F. List, MD, Rami S. Komrokji, MD, Hugo F. Fernandez, MD, Jeffrey E. Lancet, MD, David A Sallman, MD, Kendra L. Sweet, MD, Chetasi Talati, MD

Blood (2019) 134 (Supplement_1): 3894.

https://doi.org/10.1182/blood-2019-127701

Published: 2019

Journal Articles

Hypomethylating Agent and Venetoclax Combination Yields Comparable Outcomes to CPX-351 in Newly Diagnosed Acute Myeloid Leukemia

Hannah Asghari, MD, Dasom Lee, MD, Yehuda E. Deutsch, MD, Onyee Chan, MD, Najla Al Ali, M.Sc, Stephanie Boisclair, MD, Constance Terrell, PharmD, BCOP, Eric Padron, MD, Andrew T Kuykendall, MD, Alan F. List, MD, Hugo F. Fernandez, MD, Jeffrey E. Lancet, MD, David A Sallman, MD, Rami S. Komrokji, MD, Kendra L. Sweet, MD, Chetasi Talati, MD

Blood (2019) 134 (Supplement_1): 3895.

https://doi.org/10.1182/blood-2019-130379

Published: 2019

Journal Articles

Targeting DHODH with AG-636 Induces Apoptosis and Differentiation and Inhibits Mitochondrial Function in AML, Translating into Anti-Tumor Efficacy in Vitro and i n Vivo

Zhihong Zeng, Natalia Baran, PhDMD, Sergej Konoplev, Antonio Cavazos, Qi Zhang, Vinitha Mary Kuruvilla, MSc, Philip Lorenzi, Hagop M. Kantarjian, MD, Michael Andreeff, MD PhD, Courtney D. DiNardo, MDMSc, Joshua Murtie, Danielle Ulanet, Marina Y Konopleva, MD PhD

Blood (2019) 134 (Supplement_1): 3911.

https://doi.org/10.1182/blood-2019-125469

Published: 2019

Journal Articles

Germline Mutations in ETV6 are Associated with In Vivo cytoplasmic Localization of ETV6 and Cause Transcriptional Dysregulation of Interferon Response Genes

Marlie H Fisher, Greg Kirkpatrick, PhD, Courtney L Jones, PhD, Brett M. Stevens, PhD, Michael Callaghan, MD, Eric Pietras, PhD, Kenneth L. Jones, PhD, Christopher C. Porter, MD, Jorge Di Paola, MD

Blood (2019) 134 (Supplement_1): 2316.

https://doi.org/10.1182/blood-2019-132032

Published: 2019

NARROW

Therapeutic options in VEXAS syndrome: insights from a retrospective series

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Identification of Recurrent Alternative RNA Splicing in Adverse-Risk Acute Myeloid Leukemia

Myeloid-Lineage Enhancers Drive Oncogene Synergy and Represent a Novel Therapeutic Target in CSF3R-CEBPA Mutant AML

Results of a Phase III Double-Blind Study of the Addition of Tocilizumab Vs. Placebo to Cyclosporin/Methotrexate Gvhd Prophylaxis after HLA-Matched Allogeneic Stem Cell Transplantation

Stag2 Regulates Hematopoietic Differentiation and Self-Renewal through Alterations in Gene Expression and Topological Control

Survival Outcomes in Patients with Intermediate Risk Acute Myeloid Leukemia: A Single-Center Retrospective Review

Outcome of Patients with Newly-Diagnosed Therapy-Related AML (t-AML) and AML with Myelodysplasia-Related Changes (AML-MRC) Treated with Liposomal Daunorubicin and Cytarabine (CPX-351) at an Academic Community-Based Health System

Combination of Venetoclax and Hypomethylating Agents in Myeloid Malignancies in Patients Unfit for Intensive Chemotherapy - Real World Data from a Tertiary Care Centre in India. Is It Time to Bid Adieu to 3+7?

A Phase 1a/b Dose Escalation Study of Apto-253 in Patients with Relapsed or Refractory AML or High-Risk MDS

Assessing Efficacy and Safety of Daunorubicin and Cytarabine Liposomal in Patients with Acute Myeloid Leukemia

Outcomes of Patients Receiving Direct Oral Anticoagulants for Myeloproliferative Neoplasm Associated Venous Thromboembolism

A Randomized, Open-Label, Multicenter, Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of KRT-232 Compared with Ruxolitinib in Patients with Phlebotomy-Dependent Polycythemia Vera

Cord Blood Regulatory T Cells Prevent Mutiple Myeloma Progression By Suppressing Inflammation

Single-Cell RNA Sequencing Reveals a Distinct Transcriptome Signature of Hematopoiesis in GATA2 Deficiency

CPX-351 As Induction Chemotherapy Yields Similar Responses and Survival Outcomes in Younger Patients (<60 Years Old) Compared to Older Patients (≥60 Years Old) with Acute Myeloid Leukemia

Hypomethylating Agent and Venetoclax Combination Yields Comparable Outcomes to CPX-351 in Newly Diagnosed Acute Myeloid Leukemia

Targeting DHODH with AG-636 Induces Apoptosis and Differentiation and Inhibits Mitochondrial Function in AML, Translating into Anti-Tumor Efficacy in Vitro and i n Vivo

Germline Mutations in ETV6 are Associated with In Vivo cytoplasmic Localization of ETV6 and Cause Transcriptional Dysregulation of Interferon Response Genes

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American Society of Hematology

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