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Journal Articles

DARTs point the way forward in AML

Clinical Trials & Observations
Saar Gill
Journal: Blood
Blood (2021) 137 (6): 720–721.
https://doi.org/10.1182/blood.2020009020
Published: 2021
Journal Articles

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Ekta Seth Chhabra, Tongyao Liu, John Kulman, Susannah Patarroyo-White, Buyue Yang, Qi Lu, Douglas Drager, Nancy Moore, Jiayun Liu, Amy M. Holthaus, Jurg M. Sommer, Ayman Ismail, Deana Rabinovich, Zhan Liu, Arjan van der Flier, Allison Goodman, Chris Furcht, Mark Tie, Tyler Carlage, Randy Mauldin, Terrence M. Dobrowsky, Zhiqian Liu, Oblaise Mercury, Lily Zhu, Baisong Mei, Volker Schellenberger, Haiyan Jiang, Glenn F. Pierce, Joe Salas, Robert Peters
Journal: Blood
Blood (2020) 135 (17): 1484–1496.
https://doi.org/10.1182/blood.2019001292
Published: 2020
Includes: Supplemental data
Journal Articles

An Adaptive Design Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients with Non-Primary Hemophagocytic Lymphohistiocytosis

Veronica Asnaghi, MD, Maria Ballabio, MD, Philippe Jacqmin, Kathy de Graaf, Cristina De Min, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2333.
https://doi.org/10.1182/blood-2019-123995
Published: 2019
Journal Articles

Clinical Evidence of Granulocyte-Monocyte Progenitor (GMP) Stem Cell Involvement in Plinabulin's Mechanism of Action (MoA) for the Prevention of Docetaxel (Doc) Chemotherapy (Chemo)-Induced Neutropenia (CIN)

Douglas W. Blayney, MD, Lan Huang, PhD, Ramon Mohanlal, PhD MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4861.
https://doi.org/10.1182/blood-2019-127227
Published: 2019
Journal Articles

Pharmacokinetics of Ticagrelor in Infants and Toddlers Aged <24 Months with Sickle Cell Disease

Baba PD Inusa, MD, Adlette Inati, MD, Philip Maes, MD, Jessie Githanga, MBChB, MMed, Miguel R. Abboud, MD, Maurizio Miano, Elena Cela, Videlis Nduba, Mohammad Niazi, Magnus Åstrand, Kevin Persson, Anders Rikard Berggren, MD PhD, Glenn Carlson, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1005.
https://doi.org/10.1182/blood-2019-129855
Published: 2019
Journal Articles

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Charles T. Quinn, MD, Omar Niss, MD, Amanda Pfeiffer, Jennifer Korpik, Holly Bonar, Mary Reynaud, Russell E. Ware, MD PhD, Patrick T. McGann, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 892.
https://doi.org/10.1182/blood-2019-130414
Published: 2019
Journal Articles

A Phase 2a Open-Label Study to Investigate Safety and Tolerability (including the MTD), Efficacy, Pharmacokinetics, Pharmacodynamics and Immunogenicity of MT-3724 in Combination with Lenalidomide in Subjects with Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma

Christine Burnett, Gary Gordon, MD PhD, Thomas Strack, MD, Tara Lehner, MS, PMP, Jack P Higgins, PhD, Jason Tache, DO
Journal: Blood
Blood (2019) 134 (Supplement_1): 1597.
https://doi.org/10.1182/blood-2019-128503
Published: 2019
Journal Articles

Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

Theodosia A. Kalfa, MD PhD, Frans A Kuypers, PhD, Marilyn J. Telen, MD, Punam Malik, MD, Diamantis G. Konstantinidis, PhD BSc, Jeremy H. Estepp, MD, Hyon J. Kim, MD PhD, Santosh L. Saraf, MD, Lindsey Wilson, Maria D Ribadeneira, PhD, Sanjeev Forsyth, Patricia Schroeder, Adam Drake, Olga Polyanskaya, Patrick Kelly, MD, Lukasz Biernat, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 616.
https://doi.org/10.1182/blood-2019-121889
Published: 2019
Journal Articles

Autism-Associated Chromatin Remodeler CHD8 Governs the Survival and Differentiation of Hematopoietic Stem/Progenitor Cells

Zhaowei Tu, PhD, Chen Wang, MSc, Chuntao Zhao, PhD, Lei Huang, PhD, Nathan Salomonis, PhD, Richard Q Lu, PhD, Yi Zheng, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1191.
https://doi.org/10.1182/blood-2019-127313
Published: 2019
Journal Articles

Hyper-Sialylated IgG M254, an Innovative Therapeutic Candidate, Evaluated in Healthy Volunteers and in Patients with Immune Thrombocytopenia Purpura: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics

Santiago Arroyo, MD PhD, Renger G Tiessen, MD, William S Denney, PhD, Jim Jin, MS, Mattheus Paulus (Thijs) van Iersel, MD, Heidi Zeitz, MD CCRA, Anthony M Manning, PhD, Martin R. Schipperus, MD PhD, James B. Bussel, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1090.
https://doi.org/10.1182/blood-2019-125993
Published: 2019
Journal Articles

Pharmacokinetics of Extended Half-Life Factor VIII Products By myPKFiT R Is Useful for Personalized Treatment in Children with Severe Hemophilia a

Risa Matsumura, Keita Tomioka, MD, Shiho Nishimura, MD, Yoko Mizoguchi, MD PhD, Hiroshi Kawaguchi, MD PhD, Masao Kobayashi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1127.
https://doi.org/10.1182/blood-2019-128149
Published: 2019
Journal Articles

Can Monosomy 7 be Targeted By Next Generation Cereblon-Modulating Agents?

Vera Adema, PhD, Cassandra M Kerr, MS, Wencke Walter, PhD, Stephan Hutter, PhD, Yasunobu Nagata, MD PhD, Hassan Awada, MD, Sunisa Kongkiatkamon, Christina A. Snider, BA, Milo Co, Rainey Magdalena, Arda Durmaz, Manja Meggendorfer, PhD, Aziz Nazha, MD, Jacob Scott, MD, Hetty E. Carraway, MD MBA, Mikkael A. Sekeres, MD MS, Francesc Sole, PhD, Torsten Haferlach, MD, Valeria Visconte, PhD, Jaroslaw P. Maciejewski, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1270.
https://doi.org/10.1182/blood-2019-128967
Published: 2019
Journal Articles

Identification of Novel Combination Therapies Active in BCL2 Inhibitor-Resistant Patient-Derived AML Models

Courtney L Andersen, PhD, Amanda L Christie, MS, Alan Rosen, MS, Kim Maratea, DVM, PhD, Maureen Hattersley, Jon Travers, PhD, Justin Cidado, PhD, Sai Pulukuri, PhD, Jamal Saeh, PhD, Edwin Clark, PhD, Corinne Reimer, PhD, Jerome Mettetal, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1273.
https://doi.org/10.1182/blood-2019-129475
Published: 2019
Journal Articles

Nascent Transcript and Single Cell Rnaseq Analysis Defines the Mechanism of Action of the LSD1 Inhibitor INCB059872 in Myeloid Leukemia

Gretchen Johnston, Haley E. Ramsey, PhDMSc, Kristy Stengel, PhD, Shilpa Sampathi, PhD, Pankaj Acharya, PhD, Maria Arrate, MSc, Matthew C. Stubbs, PhD, Timothy Burn, PhD, Scott Hiebert, PhD, Michael R. Savona, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2509.
https://doi.org/10.1182/blood-2019-130458
Published: 2019
Journal Articles

Identification of a New Targeted Therapy for T-Cell Acute Lymphoblastic Leukemia and Studies on Its Mechanism of Action

Kinjal Shah, MS, Julhash U. Kazi, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2752.
https://doi.org/10.1182/blood-2019-129823
Published: 2019
Journal Articles

Improved Anti-Leukemic Pre-Clinical Efficacy of a Protac Based MDM2 Degrader in a Large AML Cohort

Malathi Kandarpa, PhD, Luke F. Peterson, PhD, Harish Potu, PhD, Megha Ramappan, Yihong Liu, Avery Polk, Shaomeng Wang, PhD, Moshe Talpaz, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2670.
https://doi.org/10.1182/blood-2019-131949
Published: 2019
Journal Articles

Modulation of the IL-6/STAT3 Signaling Axis in CD4 + T Cells As a Potential Immune Mechanism of Action of Azacytidine in High-Risk Myelodysplastic Syndromes

Eleftheria Lamprianidou, Chrysoula Kordella, Anastasiya Kazachenka, Emmanouela Zoulia, Elsa Bernard, PhD, Anastasia Filia, Stamatia Laidou, Theodoros P. Vassilakopoulos, MD PhD, Sotirios Papageorgiou, MD PhD, Vassiliki Pappa, MD, Athanasios Galanopoulos, MD PhD, Nora-Athina Viniou, MD PhD, Evangelia Nakou, Anastasia Hadzidimitriou, PhD, George Kassiotis, Elli Papaemmanuil, PhD, Ioannis Mitroulis, Ioannis Kotsianidis
Journal: Blood
Blood (2019) 134 (Supplement_1): 2988.
https://doi.org/10.1182/blood-2019-126504
Published: 2019
Journal Articles

First China Approved Rituximab Biosimilar HLX01: Pharmacokinetics, Safety and Efficacy Comparison to Reference Rituximab in the Phase 3 Diffuse Large B-Cell Lymphoma Study

Yuankai Shi, MD PhD, Yongping Song, MD PhD, Yan Qin, MD PhD, Qingyuan Zhang, MD, Xiaohong Han, MD, Xiaonan Hong, MD, Dong Wang, MD, Xiaofeng Zeng, MD PhD, Pei Hu, MD PhD, Shuiyu Zhao, Kaka Y. Hong, MSSB, Chaoming Ma, Jiancheng Cheng, Peng Ting, PhD, Kevin Li, MD, Weidong Jiang, PhD, Scott Liu, PhD, Eugene Liu, MD PhD, Xin Zhang, MD, Katherine Chai, PhD MsLaw, Dan Yao, PhD, Alvin Y. Luk, PhDMBA
Journal: Blood
Blood (2019) 134 (Supplement_1): 2878.
https://doi.org/10.1182/blood-2019-130603
Published: 2019
Journal Articles

Phase 1 Dose-Finding Study Investigating CA-4948, an IRAK4 Kinase Inhibitor, in Patients with R/R NHL: Report of Initial Efficacy and Updated Safety Information

Anas Younes, MD, Grzegorz Nowakowski, MD, Allison C. Rosenthal, DO, Lori A. Leslie, MD, Han W. Tun, MD, Matthew A. Lunning, DO, FACP, Iris Isufi, MD, Robert Martell, MD PhD, Krish Patel, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5327.
https://doi.org/10.1182/blood-2019-131851
Published: 2019
Journal Articles

Phase 1 Study of Bisthianostat, an Orally Efficacious Pan-HDAC Inhibitor: Part Results of Safety, Pharmacokinetics and Efficacy in Patients with Relapsed or Refractory Multiple Myeloma

Hong-Hui Huang, PhD MD, Jian Hou, PhD MD, Yang-Ming Zhang, PhD, Yu-Bo Zhou, PhD, Li Jia, PhD, Fa-Jun Nan, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5591.
https://doi.org/10.1182/blood-2019-129683
Published: 2019