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Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Lactate Dehydrogenase to Carboxyhemoglobin Ratio As a Biomarker of Heme Release to Heme Processing Is Associated with Higher Tricuspid Regurgitant Jet Velocity and Early Death in Sickle Cell Disease

Annelies J. Van Vuren, MD, Laurel Mendelsohn, BS, Richard van Wijk, PhD, Caterina P. Minniti, MD, John Baird, MD, Gregory J Kato, MD, Eduard J. van Beers, MD PhD

Blood (2019) 134 (Supplement_1): 2274.

https://doi.org/10.1182/blood-2019-124048

Published: 2019

Journal Articles

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Minke A.E. Rab, MD, Richard van Wijk, PhD, Celeste K. Kanne, BA, Brigitte A. van Oirschot, Jennifer Bos, Camille Boisson, Maite E. Houwing, MD, Jorn J. Gerritsma, MD, Karin Fijnvandraat, MD PhD, Celine Renoux, PhD, Marije Bartels, MD PhD, Erfan Nur, MD PhD, Marjon H. Cnossen, MD PhD, Philippe Joly, PhD, Romain Fort, MD, Philippe Connes, PhD, Eduard J. van Beers, MD PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 2275.

https://doi.org/10.1182/blood-2019-128870

Published: 2019

Journal Articles

Opioid Analgesics Are Associated with Albuminuria in Adult Patients with Sickle Cell Anemia

Ashley Thrower, BS, Zhou Laura, Vimal K. Derebail, MD, David Wichlan, Ashley Smith, RN, Jianwen Cai, PhD, Kenneth I. Ataga, MD

Blood (2019) 134 (Supplement_1): 2308.

https://doi.org/10.1182/blood-2019-131368

Published: 2019

Journal Articles

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Thidarat Suksangpleng, PhD, Kalaya Tachavanich, M.Sc., Suchada Riolueang, MSc, Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2220.

https://doi.org/10.1182/blood-2019-131720

Published: 2019

Journal Articles

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Wasan Theerajangkhaphichai, MD, Adisak Tantiworawit, MD, Jirapas Sripetchwandee, PhD, Nattayaporn Apaijai, PhD, Sirawit Sriwichaiin, MD, Napatsorn Saiyasit, Kewarin Jinawong, Busarin Arunsak, Pokpong Piriyakhuntorn, MD, Thanawat Rattanathammethee, MD, Sasinee Hantrakool, MD, Chatree Chai-Adisaksopha, MD PhD, Ekarat Rattarittamrong, MD, Lalita Norasetthada, MD, Pimlak Charoenkwan, MD, Nipon Chattipakorn, MD PhD, Siriporn Chattipakorn, DDS, PhD

Blood (2019) 134 (Supplement_1): 2251.

https://doi.org/10.1182/blood-2019-125182

Published: 2019

Journal Articles

Mn Porphyrin-Based Redox Active Drugs Improve Anemia and Reduce Organ Damage in a Murine Model of Sickle Cell Disease

Madhan Thamilarasan, PhD, Rodolfo Estupinan, Rahima Zennadi, PhD

Blood (2019) 134 (Supplement_1): 2255.

https://doi.org/10.1182/blood-2019-131140

Published: 2019

Journal Articles

Rate of Sickle Hemoglobin Recovery in Sickle Cell Disease Patients Undergoing Red Blood Cell (RBC) Exchange Transfusion Is Associated with Age of Patients and Number of RBC Units Transfused

John Chinawaeze Aneke, MBBS, Aliraza Rajabali, Nafanta Fadiga, Stéphanie Forté, MD MSc, FRCPC, George A. Tomlinson, PhD BSc, MSc, Christine M. Cserti-Gazdewich, BSc, MD FRCPC, Christopher J. Patriquin, MDFRCPC,MSc, Richard Ward, MSc, MRCP, FRCPath, Jacob Pendergrast, MDFRCPC, David Barth, MD FRCPC, Kevin H. M. Kuo, MDMSc,FRCPC

Blood (2019) 134 (Supplement_1): 2170.

https://doi.org/10.1182/blood-2019-122072

Published: 2019

Journal Articles

Comparison of α LEPRA and α LELY Co-Inheritance with a SPTA1 Whole Gene Deletion, a Family Study

Molly S. Hein, Lea Coon, MS, LCGC, Tavanna R. Porter, MS, LCGC, Rong He, MD, Michelle E. Savedra, MT(ASCP), Laura J. Train, BS, Linnea M. Baudhuin, PhD, Andre M. Oliveira, MD PhD, Phuong L. Nguyen, MD, James D. Hoyer, MD, Aruna Rangan, MD, Jennifer L. Oliveira, MD

Blood (2019) 134 (Supplement_1): 2228.

https://doi.org/10.1182/blood-2019-125691

Published: 2019

Journal Articles

Benefit and Safety of Isovolemic Hemodilution Prior to Red Cell Exchange in Chronically Transfused Sickle Cell Patients

Katayoun Fomani, MD, Cindy Xu, Swathi Ratkal, MD, Vijay Nandi, PhD, Abena Appiah-Kubi, MD MPH, Banu Aygun, MD, James Louie, MD, Patricia Shi, MD

Blood (2019) 134 (Supplement_1): 2291.

https://doi.org/10.1182/blood-2019-122259

Published: 2019

Journal Articles

Ndepth: A Randomized Controlled Trial of a Novel Dose-Prediction Equation to Determine Maximum Tolerated Dose for Hydroxyurea Therapy in Pediatric Patients with Sickle Cell Anemia

Alex George, MD PhD, Bogdan Dinu, MD, Norma Estrada, Charles Minard, PhD, Richard L Hurwitz, MD, Donald Mahoney, MD, Amber M Yates, MD, Mary Vaughan, FNP - C, Alaundra Carmouche, FNP - C, Gladstone Airewele, MDMPH, Kirk E Susan, P.A., Titilope Adenike Fasipe, MD PhD, Precious Uwaezuoke, FNP, Russell E. Ware, MD PhD

Blood (2019) 134 (Supplement_1): 2267.

https://doi.org/10.1182/blood-2019-127414

Published: 2019

Journal Articles

Manifestations of Reduced Kidney Function Occur at a Higher Estimated Glomerular Filtration Rate in Sickle Cell Anemia

Santosh L. Saraf, MD, Vimal K. Derebail, MD, Xu Zhang, PhD, Mark T Gladwin, MD, Victor R Gordeuk, MD, Jane A. Little, MD

Blood (2019) 134 (Supplement_1): 2268.

https://doi.org/10.1182/blood-2019-124737

Published: 2019

Journal Articles

Predictors of the Rowland Universal Dementia Assessment Scale (RUDAS) Performance in Adults with Sickle Cell Disease

Stéphanie Forté, MD MSc, FRCPC, Florence Blais, Mathias Castonguay, Nafanta Fadiga, Richard Ward, MSc, MRCP, FRCPath, Béland Sébastien, PhD, Melanie Cohn, PhD CPsych, Denis Soulières, MD, Kevin H. M. Kuo, MDMSc,FRCPC

Blood (2019) 134 (Supplement_1): 2294.

https://doi.org/10.1182/blood-2019-129342

Published: 2019

Journal Articles

Association between GLUT1 and HbF Levels in Red Blood Cells from Patients with Sickle Cell Disease

Alireza Paikari, MD, Ankush Goyal, MA, Christelle Cousin, PhD, Vincent Petit, DVM, PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 2265.

https://doi.org/10.1182/blood-2019-131058

Published: 2019

Journal Articles

Untargeted Metabolomics on Dried Blood Spots for the Diagnosis and Clinical Follow up of Rare Hereditary Anemias

Birgit van Dooijeweert, MD, Melissa Broeks, Msc, Judith Jans, PhD, Eduard J. van Beers, MD PhD, Nanda Verhoeven, PhD, Richard van Wijk, PhD, Marije Bartels, MD PhD

Blood (2019) 134 (Supplement_1): 3376.

https://doi.org/10.1182/blood-2019-131693

Published: 2019

Journal Articles

A Case of Mistaken Identity: TTP-like State Due to Heroin Overdose

Phani Borra, MD, Cory Edward Mellon, MD

Blood (2019) 134 (Supplement_1): 4905.

https://doi.org/10.1182/blood-2019-128537

Published: 2019

Journal Articles

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Sule Unal, MD, Tekin Aksu, MD, Cagrı Coskun, MD, Fatma Gumruk, MD

Blood (2019) 134 (Supplement_1): 4794.

https://doi.org/10.1182/blood-2019-124272

Published: 2019

Journal Articles

Symptomatic Bilirubin Gallstones in Patients with PNH Treated with Eculizumab

David J. Araten, MD, H. Leon Pachter, Robert J Dring, Elliot Newman, Steven M Cohen

Blood (2019) 134 (Supplement_1): 4795.

https://doi.org/10.1182/blood-2019-124571

Published: 2019

Journal Articles

Paroxysmal Cold Hemoglobinuria Successfully Treated with Complement Inhibition

Sarah A Lau-Braunhut, MD, Hannah Stone, Griffin Collins, MD, Sigbjørn Berentsen, MD PhD, Matt Zinter, MD, Benjamin Braun, MD PhD

Blood (2019) 134 (Supplement_1): 4798.

https://doi.org/10.1182/blood-2019-126365

Published: 2019

Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

Published: 2019

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Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Lactate Dehydrogenase to Carboxyhemoglobin Ratio As a Biomarker of Heme Release to Heme Processing Is Associated with Higher Tricuspid Regurgitant Jet Velocity and Early Death in Sickle Cell Disease

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Opioid Analgesics Are Associated with Albuminuria in Adult Patients with Sickle Cell Anemia

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Mn Porphyrin-Based Redox Active Drugs Improve Anemia and Reduce Organ Damage in a Murine Model of Sickle Cell Disease

Rate of Sickle Hemoglobin Recovery in Sickle Cell Disease Patients Undergoing Red Blood Cell (RBC) Exchange Transfusion Is Associated with Age of Patients and Number of RBC Units Transfused

Comparison of α LEPRA and α LELY Co-Inheritance with a SPTA1 Whole Gene Deletion, a Family Study

Benefit and Safety of Isovolemic Hemodilution Prior to Red Cell Exchange in Chronically Transfused Sickle Cell Patients

Ndepth: A Randomized Controlled Trial of a Novel Dose-Prediction Equation to Determine Maximum Tolerated Dose for Hydroxyurea Therapy in Pediatric Patients with Sickle Cell Anemia

Manifestations of Reduced Kidney Function Occur at a Higher Estimated Glomerular Filtration Rate in Sickle Cell Anemia

Predictors of the Rowland Universal Dementia Assessment Scale (RUDAS) Performance in Adults with Sickle Cell Disease

Association between GLUT1 and HbF Levels in Red Blood Cells from Patients with Sickle Cell Disease

Untargeted Metabolomics on Dried Blood Spots for the Diagnosis and Clinical Follow up of Rare Hereditary Anemias

A Case of Mistaken Identity: TTP-like State Due to Heroin Overdose

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Symptomatic Bilirubin Gallstones in Patients with PNH Treated with Eculizumab

Paroxysmal Cold Hemoglobinuria Successfully Treated with Complement Inhibition

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

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