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Journal Articles

Probably Pathogenic KMT2D Variants Presenting with Cytopenias and Primary Immune Deficiency

Sun Young Kim, MD PhD, Alexander Ing, MS, CGC, Kristin Clemenz, MS, CGC, Kai Lee Yap, PhD, Sherif M. Badawy, MD, Robert I Liem, MD MS, Alexis A. Thompson, MD MPH, A. Kyle Mack, MD

Blood (2019) 134 (Supplement_1): 2334.

https://doi.org/10.1182/blood-2019-131468

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

Blood (2019) 134 (Supplement_1): 2305.

https://doi.org/10.1182/blood-2019-127613

Published: 2019

Journal Articles

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

Isabella McCary, BS, Christine Guelcher, PPCNP-BC, Jan Kuhn, BA, BSN, MSN, Regina Butler, RN-BC, Michael F. Guerrera, MD, Gita Massey, Leslie J. Raffini, MD

Blood (2019) 134 (Supplement_1): 2396.

https://doi.org/10.1182/blood-2019-126944

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

Risk Factors for Pediatric Cerebral Sinovenous Thrombosis: A Case-Control Study with Pediatric Neuroradiologist Case Validation

Austin R Sellers, MS, Anh Thy Nguyen, MSPH, Marisol Betensky, MDMPH, Katie Mills, DNP, APRN, Neil A Goldenberg, MD PhD, Nir Shimony, MD

Blood (2019) 134 (Supplement_1): 2427.

https://doi.org/10.1182/blood-2019-130906

Published: 2019

Journal Articles

Comparison of Health Outcomes in Adolescent and Young Adults (AYA) Oncology Patients Treated at a Pediatric Versus Adult Institute

Amy Moskop, MD, Ke Yan, PhD, Sarah K. Dobrozsi, MD, Julie Panepinto, MDMSPH

Blood (2019) 134 (Supplement_1): 4743.

https://doi.org/10.1182/blood-2019-121616

Published: 2019

Journal Articles

Characterization of Community-Based Socioeconomic Factors, Utilization, and Adherence in Children with Sickle Cell Disease

Ronay Thomas, BS, Patrick T. McGann, MD, Andrew Beck, MD MPH, Amanda Pfeiffer, Kyesha M James, BS

Blood (2019) 134 (Supplement_1): 4686.

https://doi.org/10.1182/blood-2019-130637

Published: 2019

Journal Articles

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent β-Thalassemia Treated at the Bambino Gesù Children's Hospital, Rome, Italy

Pietro Merli, MD, Annalisa Ruggeri, MD, Mattia Algeri, MD, Giuseppina Li Pira, PhD, Giulia Ceglie, MD, Katiana Gruppioni, MPH, Sanjeev Kommera, MS, Jenfue Maa, PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 969.

https://doi.org/10.1182/blood-2019-123440

Published: 2019

Journal Articles

KLF4 Is Needed for Optimal Hydroxyurea Induction of Gamma-Globin in K562 Cells

Alireza Paikari, MD, Ankush Goyal, MA, Celeste K. Kanne, BA, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 971.

https://doi.org/10.1182/blood-2019-131214

Published: 2019

Journal Articles

Safety of Intravenous Arginine Therapy in Children with Sickle Cell Disease Hospitalized for Vaso-Occlusive Pain: A Randomized Placebo-Controlled Trial in Progress

Claudia R. Morris, MD, Janet Figueroa, MPH, Amanda Watt, Kirshma Khemani, MD, Polly Kumari, MBBS, Morgan Barnett, MPH, Nitya Bakshi, MBBS, MS, Peter A. Lane, MD, Carlton Dampier, MD

Blood (2019) 134 (Supplement_1): 995.

https://doi.org/10.1182/blood-2019-122983

Published: 2019

Journal Articles

Cytogenetic Characteristics and Outcomes of Patients Receiving CTL019 CAR T Cell Therapy

Allison Barz Leahy, MD, Kaitlin J. Stanley, MD, Regina M. Myers, MD, Amanda M. DiNofia, MD, Lisa Wray, MD, Susan R. Rheingold, MD, Colleen Callahan, CRNP, Diane Baniewicz, CRNP, Amy E. Barry, PPCNP-BC, Maria M. Patino, BS, Stephen P. Hunger, MD, Stephan Grupp, MD PhD, David M. Barrett, Shannon L. Maude, MD PhD

Blood (2019) 134 (Supplement_1): 1464.

https://doi.org/10.1182/blood-2019-130060

Published: 2019

Journal Articles

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Sherif M. Badawy, MD, Leonardo Barrera, MPH, Graham Zolkowski, BS, Zeinab Alward, MBChB, MPH

Blood (2019) 134 (Supplement_1): 420.

https://doi.org/10.1182/blood-2019-131334

Published: 2019

Journal Articles

Oral Arginine Therapy As a Novel Adjuvant in the Management of Acute Pain in Children with Sickle Cell Anemia in Nigeria: A Randomized Placebo-Controlled Trial

Richard Onalo, FCPaed, Peter Cooper, FCPaed, Antoinette Cilliers, FCPaed, Uche Nnebe-Agumadu, FCPaed, Oluseyi Oniyangi, FCPaed, Damilare Oladimeji, MSc Statistics, Claudia R. Morris, MD

Blood (2019) 134 (Supplement_1): 613.

https://doi.org/10.1182/blood-2019-122510

Published: 2019

Journal Articles

Real-World Use of Emicizumab in Patients with Hemophilia with and without Inhibitors

Isabella McCary, BS, Christine Guelcher, PPCNP-BC, Jan Kuhn, BA, BSN, MSN, Regina Butler, RN-BC, Gita Massey, Michael F. Guerrera, MD, Lance Ballester, MS, Leslie J. Raffini, MD

Blood (2019) 134 (Supplement_1): 1137.

https://doi.org/10.1182/blood-2019-122569

Published: 2019

Journal Articles

Two Different Platelet Thresholds for Bleeding Prophylaxis in Children with Malignancies or Non-Malignant Disorders: The Bambino Gesù Children's Hospital Experience

Pierpaolo Berti, MD, Guglielmo Trua, MD, Francesco Lamura, MD, Laila Costantino, MD, Costantino Damico, MD, Alessandra Paoloni, MD, Maria Teresa Romano, MD, Katia Girardi, MD, Luisa Strocchio, MD, Elisabetta Cicchetti, Alessandra Bernardi, Valentina De Nava, Antonio Cappelli, Andrea Onetti Muda, MD PhD, Mauro Montanari, MD

Blood (2019) 134 (Supplement_1): 1173.

https://doi.org/10.1182/blood-2019-129132

Published: 2019

Journal Articles

The Cost of Healthcare for Pediatric Patients with Sickle Cell Disease

Maite E. Houwing, MD, Frederick Thielen, Anne P.J. de Pagter, MD PhD, Jan A Hazelzet, MD PhD, Hedwig Blommestein, Carin A. Uyl-de Groot, Marjon H. Cnossen, MD PhD

Blood (2019) 134 (Supplement_1): 1030.

https://doi.org/10.1182/blood-2019-130372

Published: 2019

Journal Articles

Severe Bleeding Events in Hemophilia Α Patients Receiving Emicizumab Prophylaxis

Karen L. Zimowski, MD, Glaivy M. Batsuli, MD, Paulette Bryant, MD, Jenny McDaniel, MD, Kelly Tickle, MSN, APN, PPCNP-BC, Shannon L. Meeks, MD, Robert F. Sidonio, Jr., MD MSc

Blood (2019) 134 (Supplement_1): 1126.

https://doi.org/10.1182/blood-2019-126659

Published: 2019

Journal Articles

Pilot Study on the Safety of Intravenous Arsenic Trioxide and Oral Realgar-Indigo Naturalis Formula Administrated in Children with Acute Promyelocytic Leukemia: A Single-Center Study

Linya Wang, MD, Yuanyuan Zhang, MD, Ruidong Zhang, MD PhD, Jiaole Yu, PhD MD, Ying Wu, MD, Wei Lin, Peijing Qi, Jia Fan, Jing Li, PhD, Jiayan Lin, Xueling Zheng, Hairu Hu, Xiaoxia Peng, Hui Jiang, PhD, Huyong Zheng, MD PhD

Blood (2019) 134 (Supplement_1): 2611.

https://doi.org/10.1182/blood-2019-128196

Published: 2019

Journal Articles

Modeling Transient Myeloproliferative Disorder Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology

Sonali Barwe, PhD MSc, E. Anders Kolb, MD, Anilkumar Gopalakrishnapillai, PhD

Blood (2019) 134 (Supplement_1): 2738.

https://doi.org/10.1182/blood-2019-130934

Published: 2019

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Probably Pathogenic KMT2D Variants Presenting with Cytopenias and Primary Immune Deficiency

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

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

Risk Factors for Pediatric Cerebral Sinovenous Thrombosis: A Case-Control Study with Pediatric Neuroradiologist Case Validation

Comparison of Health Outcomes in Adolescent and Young Adults (AYA) Oncology Patients Treated at a Pediatric Versus Adult Institute

Characterization of Community-Based Socioeconomic Factors, Utilization, and Adherence in Children with Sickle Cell Disease

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent β-Thalassemia Treated at the Bambino Gesù Children's Hospital, Rome, Italy

KLF4 Is Needed for Optimal Hydroxyurea Induction of Gamma-Globin in K562 Cells

Safety of Intravenous Arginine Therapy in Children with Sickle Cell Disease Hospitalized for Vaso-Occlusive Pain: A Randomized Placebo-Controlled Trial in Progress

Cytogenetic Characteristics and Outcomes of Patients Receiving CTL019 CAR T Cell Therapy

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Oral Arginine Therapy As a Novel Adjuvant in the Management of Acute Pain in Children with Sickle Cell Anemia in Nigeria: A Randomized Placebo-Controlled Trial

Real-World Use of Emicizumab in Patients with Hemophilia with and without Inhibitors

Two Different Platelet Thresholds for Bleeding Prophylaxis in Children with Malignancies or Non-Malignant Disorders: The Bambino Gesù Children's Hospital Experience

The Cost of Healthcare for Pediatric Patients with Sickle Cell Disease

Severe Bleeding Events in Hemophilia Α Patients Receiving Emicizumab Prophylaxis

Pilot Study on the Safety of Intravenous Arsenic Trioxide and Oral Realgar-Indigo Naturalis Formula Administrated in Children with Acute Promyelocytic Leukemia: A Single-Center Study

Modeling Transient Myeloproliferative Disorder Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology

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American Society of Hematology

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