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Journal Articles

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

Phase I Trial of Ibrutinib, Lenalidomide and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma (RRMM)

Sikander Ailawadhi, MD, Aneel Paulus, MD, Victoria R. Alegria, Betsy Laplant, MS, Muhamad Alhaj Moustafa, MD, Dustin Chapin, Keisha Jackson, Prachi Jani, MD, Salman Ahmed, MD, Brett Edwards, Alak Manna, PhD, Vivek Roy, MD, Taimur Sher, MD, Asher A. Chanan-Khan, MD

Blood (2019) 134 (Supplement_1): 1912.

https://doi.org/10.1182/blood-2019-128233

Published: 2019

Journal Articles

ATG5 Dependent Autophagy in Graft-Versus-Host Disease Target Organs Mitigates Disease Severity Post Allogeneic Bone Marrow Transplant

Austin Taylor, BA, Kay Oravecz-Wilson, Hideaki Fujiwara, Stephanie Kim, Dan Peltier, Molly Radosevich, Yaping Sun, Pavan Reddy

Blood (2019) 134 (Supplement_1): 1914.

https://doi.org/10.1182/blood-2019-131879

Published: 2019

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

High Molecular Weight Kininogen but Not Factor XII Deficiency Attenuates Acetaminophen-Induced Liver Injury in Mice

Michael Wayne Henderson, MSc, Erica Sparkenbaugh, PhD BS, Denis Noubouossie, MD PhD, Reiner Mailer, Thomas Renne, MD PhD, Keith R McCrae, MD, Nigel S. Key, MBChB, FRCP, Rafal Pawlinski, PhD

Blood (2019) 134 (Supplement_1): 3621.

https://doi.org/10.1182/blood-2019-127206

Published: 2019

Journal Articles

Long-Term Efficacy and Safety of Copanlisib in Multiply Relapsed or Refractory Patients with Marginal Zone Lymphoma

Martin Dreyling, MD, Panayiotis Panayiotidis, MD, George A Follows, MD PhD, Luigina Mollica, MD FRCPC, PhD, Arnon Nagler, MD MSc, Muhit Özcan, MD, Armando Santoro, Don Stevens, MD, David Trevarthen, MD, Florian Hiemeyer, MSc, Li Liu, PhD, Jose Garcia-Vargas, MD, Barrett H Childs, MD, Pier Luigi Zinzani, MD PhD

Blood (2019) 134 (Supplement_1): 1531.

https://doi.org/10.1182/blood-2019-121932

Published: 2019

Journal Articles

Updated Phase 1 Results of a First-in-Human Open-Label Study of Lcar-B38M, a Structurally Differentiated Chimeric Antigen Receptor T (CAR-T) Cell Therapy Targeting B-Cell Maturation Antigen (Bcma)

Blood (2019) 134 (Supplement_1): 1858.

https://doi.org/10.1182/blood-2019-130008

Published: 2019

Journal Articles

Results from a Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Intermediate, or High Risk, Relapsed/Refractory Myelofibrosis

Blood (2019) 134 (Supplement_1): 558.

https://doi.org/10.1182/blood-2019-131217

Published: 2019

Journal Articles

AG-636 for the Treatment of Adults with Advanced Lymphoma: Initiation of a Phase 1 Clinical Study

Scott F. Huntington, MD MPH, Avyakta Kallam, MD, Frank G. Basile, MD, Danielle Ulanet, Huansheng Xu, PhD, Feng Yin, PhD, Michelle Mobilia, BS, Michael Cooper, MD, Bijal Shah, MD, John P. Leonard, MD, Gottfried R. von Keudell, MD PhD, Ajay K. Gopal, MD

Blood (2019) 134 (Supplement_1): 1286.

https://doi.org/10.1182/blood-2019-123420

Published: 2019

Journal Articles

Biomarkers of Cardiopulmonary, Renal, and Liver Dysfunction in an Adult Sickle Cell Disease Cohort

Xu Zhang, PhD, Binal N. Shah, Roberto Machado, MD, Santosh L. Saraf, MD, Victor R Gordeuk, MD

Blood (2019) 134 (Supplement_1): 3574.

https://doi.org/10.1182/blood-2019-131813

Published: 2019

Journal Articles

Early Hyperbilirubinemia Is an Independent Predictor of Outcome after Allogeneic Stem Cell Transplantation and Correlates with Markers of Endothelial Cell Dysfunction

Thomas Luft, MD PhD, Hao Dai, Aleksandar Radujkovic, MD, David Schult, Joshua Majer-Lauterbach, Olga Blau, MD PhD, Lars Bullinger, Sihe Jiang, Carsten Müller-Tidow, MD, Peter Dreger, Olaf Penack, MD

Blood (2019) 134 (Supplement_1): 4487.

https://doi.org/10.1182/blood-2019-124284

Published: 2019

Journal Articles

First-in-Human Study of Abbv-621, a TRAIL Receptor Agonist Fusion Protein, in Patients (Pts) with Relapsed/Refractory (RR) Acute Myeloid Leukemia (AML) and Diffuse Large B-Cell Lymphoma (DLBCL)

Maja J.A. de Jonge, MD, Benedito A. Carneiro, MD, Lot Devriese, MD PhD, Toshihiko Doi, MD PhD, Sudhir Penugonda, MD MPH, Adam M. Petrich, MD, Silpa Nuthalapati, PhD, Monica Motwani, PhD, Dimple A. Modi, PhD, Yu-Wei Chang, PhD, Emiliano Calvo, MD PhD, Victor Moreno, MD PhD

Blood (2019) 134 (Supplement_1): 3924.

https://doi.org/10.1182/blood-2019-129783

Published: 2019

Journal Articles

Cytomegalovirus Induced Thrombocytopenia with No Platelets in an Immunocompetent Young Male

Sujatha Baddam, MD, Jose Cavo, Jorge Diaz Castro, MD

Blood (2019) 134 (Supplement_1): 4904.

https://doi.org/10.1182/blood-2019-132257

Published: 2019

Journal Articles

A Phase 3 Trial Comparing the Efficacy and Safety of Acalabrutinib in Combination with Venetoclax with or without Obinutuzumab, Compared with Investigator's Choice of Chemoimmunotherapy in Patients with Previously Untreated Chronic Lymphocytic Leukemia (CLL) without Del(17p) or TP53 Mutation

Jennifer R Brown, MD PhD, Barbara F. Eichhorst, MD, Paolo Ghia, MD PhD, Arnon P. Kater, MD PhD, Jianyong Li, MD, Sudha Khurana, Mostafa Elhamy, Min Hui Wang, PhD, John F. Seymour, MBBS, PhD FRACP

Blood (2019) 134 (Supplement_1): 4318.

https://doi.org/10.1182/blood-2019-123057

Published: 2019

Journal Articles

Ivosidenib (AG-120) in Patients with IDH1 -Mutant Relapsed/Refractory Myelodysplastic Syndrome: Updated Enrollment of a Phase 1 Dose Escalation and Expansion Study

Blood (2019) 134 (Supplement_1): 4254.

https://doi.org/10.1182/blood-2019-123946

Published: 2019

Journal Articles

Updated Preliminary Results from a Phase II Study Combining Azacitidine and Pembrolizumab in Patients with Higher-Risk Myelodysplastic Syndrome

Kelly S. Chien, MD, Gautam M. Borthakur, MD, Kiran Naqvi, MDMPH, Naval G. Daver, MD, Jorge E. Cortes, MD, Courtney D. DiNardo, MDMSc, Elias Jabbour, MD, Michael Andreeff, MD PhD, Yesid Alvarado, MD, Prithviraj Bose, MD, Nitin Jain, MD, Kimberly Sheppard, BSN, RN, Cheri Klingner-Winton, Sherry A. Pierce, BSN, BA, Xiao Qin Dong, Kelly Soltysiak, PhD, Hagop M. Kantarjian, MD, Guillermo Garcia-Manero, MD

Blood (2019) 134 (Supplement_1): 4240.

https://doi.org/10.1182/blood-2019-129598

Published: 2019

Journal Articles

SEL120 - a First-in-Class CDK8/19 Inhibitor As a Novel Option for the Treatment of Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome - Data from Preclinical Studies and Introduction to a Phase Ib Clinical Trial

Blood (2019) 134 (Supplement_1): 2651.

https://doi.org/10.1182/blood-2019-127835

Published: 2019

Journal Articles

Daratumumab (DARA) Subcutaneous (SC) Delivery in Relapsed or Refractory Multiple Myeloma (RRMM): Population Pharmacokinetics (PPK) and Exposure-Response (E-R) Analysis

Melody Luo, PhD, Dolly A. Parasrampuria, PhD, Saad Z. Usmani, MD, Maria-Victoria Mateos, Hareth Nahi, MD PhD, Christoph Heuck, Ming Qi, MD PhD, Steven Sun, Pamela L. Clemens, PhD, Honghui Zhou, Yu-Nien (Tom) Sun, PhD, Steven Xu, PhD

Blood (2019) 134 (Supplement_1): 3151.

https://doi.org/10.1182/blood-2019-123405

Published: 2019

Journal Articles

Parsaclisib, a potent and highly selective PI3Kδ inhibitor, in patients with relapsed or refractory B-cell malignancies

Andres Forero-Torres, Radhakrishnan Ramchandren, Abdulraheem Yacoub, Michael S. Wertheim, William J. Edenfield, Paolo Caimi, Martin Gutierrez, Luke Akard, Carolina Escobar, Justin Call, Daniel Persky, Swaminathan Iyer, Douglas J. DeMarini, Li Zhou, Xuejun Chen, Fitzroy Dawkins, Tycel J. Phillips

Blood (2019) 133 (16): 1742–1752.

https://doi.org/10.1182/blood-2018-08-867499

Published: 2019

Includes: Supplemental data

Journal Articles

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank Leebeek, K Meijer, MD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, Erhard Seifried, MD, J Schwaeble, MD, Halvard Bonig, MD, Eileen K Sawyer, PhD, W Miesbach

Blood (2018) 132 (Supplement 1): 3476.

https://doi.org/10.1182/blood-2018-99-109995

Published: 2018

NARROW

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Phase I Trial of Ibrutinib, Lenalidomide and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma (RRMM)

ATG5 Dependent Autophagy in Graft-Versus-Host Disease Target Organs Mitigates Disease Severity Post Allogeneic Bone Marrow Transplant

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

High Molecular Weight Kininogen but Not Factor XII Deficiency Attenuates Acetaminophen-Induced Liver Injury in Mice

Long-Term Efficacy and Safety of Copanlisib in Multiply Relapsed or Refractory Patients with Marginal Zone Lymphoma

Updated Phase 1 Results of a First-in-Human Open-Label Study of Lcar-B38M, a Structurally Differentiated Chimeric Antigen Receptor T (CAR-T) Cell Therapy Targeting B-Cell Maturation Antigen (Bcma)

Results from a Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Intermediate, or High Risk, Relapsed/Refractory Myelofibrosis

AG-636 for the Treatment of Adults with Advanced Lymphoma: Initiation of a Phase 1 Clinical Study

Biomarkers of Cardiopulmonary, Renal, and Liver Dysfunction in an Adult Sickle Cell Disease Cohort

Early Hyperbilirubinemia Is an Independent Predictor of Outcome after Allogeneic Stem Cell Transplantation and Correlates with Markers of Endothelial Cell Dysfunction

First-in-Human Study of Abbv-621, a TRAIL Receptor Agonist Fusion Protein, in Patients (Pts) with Relapsed/Refractory (RR) Acute Myeloid Leukemia (AML) and Diffuse Large B-Cell Lymphoma (DLBCL)

Cytomegalovirus Induced Thrombocytopenia with No Platelets in an Immunocompetent Young Male

A Phase 3 Trial Comparing the Efficacy and Safety of Acalabrutinib in Combination with Venetoclax with or without Obinutuzumab, Compared with Investigator's Choice of Chemoimmunotherapy in Patients with Previously Untreated Chronic Lymphocytic Leukemia (CLL) without Del(17p) or TP53 Mutation

Ivosidenib (AG-120) in Patients with IDH1 -Mutant Relapsed/Refractory Myelodysplastic Syndrome: Updated Enrollment of a Phase 1 Dose Escalation and Expansion Study

Updated Preliminary Results from a Phase II Study Combining Azacitidine and Pembrolizumab in Patients with Higher-Risk Myelodysplastic Syndrome

SEL120 - a First-in-Class CDK8/19 Inhibitor As a Novel Option for the Treatment of Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome - Data from Preclinical Studies and Introduction to a Phase Ib Clinical Trial

Daratumumab (DARA) Subcutaneous (SC) Delivery in Relapsed or Refractory Multiple Myeloma (RRMM): Population Pharmacokinetics (PPK) and Exposure-Response (E-R) Analysis

Parsaclisib, a potent and highly selective PI3Kδ inhibitor, in patients with relapsed or refractory B-cell malignancies

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

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American Society of Hematology

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American Society of Hematology

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