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Proof of Concept for the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non–Transfusion-Dependent Thalassemia: Interim Results from an Ongoing, Phase 2, Open-Label, Multicenter Study

Kevin H.M. Kuo - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Alpha-thalassemia

N/A

An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

Kenneth W. Wood - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Central Nervous System (CNS)

Sickle cell anemia

Hydroxyurea

Study Design and Initial Baseline Characteristics in Solace-Kids: Crizanlizumab in Pediatric Patients with Sickle Cell Disease

Matthew M. Heeney - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Musculoskeletal

Acute chest syndrome

Erythropoietin

Steadfast: A Randomized, Multicenter, Open-Label, Phase II Study Comparing the Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Chronic Kidney Disease Due to Sickle Cell Nephropathy

Kenneth I. Ataga - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell nephropathy

SelG1

Factors Influencing Time from Initial Presentation to Start of Plasma Exchange (PEX) in Patients with Acute Thrombotic Thrombocytopenic Purpura (TTP)

Rory McCulloch - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Thrombotic thrombocytopenic purpura

N/A

Extrapolation and Justification of Nplate Dosing to Improve Overall Survival in Acute Radiation Syndrome

Sameer Doshi - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

N/A

Romiplostim

Higher Symptom Burden in Patients with Immune Thrombocytopenia Experiencing Fatigue: Results from the ITP World Impact Survey (I-WISh)

James B. Bussel - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Autoimmune (AI)

Immune thrombocytopenia

N/A

Long-Term Prophylaxis with Simoctocog Alfa for the Management of Haemophilia Α: Immunogenicity, Efficacy and Safety Results from the Nuprotect PUP Extension Study

Ri J. Liesner - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

rFVIII

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Ngrier - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

Evithrom

Effectiveness and Safety Outcomes in Patients with Hemophilia a Receiving Antihemophilic Factor (Recombinant) for at Least 5 Years in a Real-World Setting: 6-Year Interim Analysis of the Ahead International and German Studies

Dimitrios A. Tsakiris - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

Antihemophilic factor

Diagnostic Accuracy of the Tina-Quant D-Dimer Gen.2 Assay with Age-Adjusted Cut-Off Ranges for Evaluation of Patients with Suspected Deep Vein Thrombosis or Pulmonary Embolism

Thomas Bertsch - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Deep vein thrombosis

Fibrinogen (IV)

Single-Cell Transcriptional Landscapes of Human Bone Marrow Reveal Distinct Erythroid Phenotypes Underpinned By Genotype in Diamond-Blackfan Anemia

Deena Iskander - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Genetic Disorder

Diamond-Blackfan anemia

N/A

Upfront Alternative Donor Transplant Versus Immunosuppressive Therapy in Patients with Severe Aplastic Anemia Who Lack Fully HLA Matched Related Donor: Systematic Review and Meta-Analysis of Retrospective Studies. on Behalf of the Severe Aplastic Anemia Working Party of European Group for Blood and Marrow Transplantation (SAAWP of EBMT)

Hind Alotaibi - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Aplastic anemia

N/A

Tfeb Links MYC Signaling to Epigenetic Control of Acute Myeloid Leukemia Cell Death and Differentiation

Seongseok Yun - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute myelocytic leukemia

N/A

Safety and Efficacy of CD19 CAR T-Cells for Pediatric Relapsed Acute Lymphoblastic Leukemia with Active CNS Involvement

Elad Jacoby - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute lymphoblastic leukemia

N/A

Treatment Patterns and Outcomes of Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) Treated with Hypomethylating Agents (HMA) in the United States (US)

Timothy S. Pardee - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute promyelocytic leukemia

N/A

Prophylactic Ruxolitinib for Cytokine Release Syndrome (CRS) in Relapse/Refractory (R/R) AML Patients Treated with Flotetuzumab

Geoffrey L Uy - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Autoimmune (AI)

Cytokine release syndrome

Flotetuzumab

Immune Senescence and Exhaustion Correlate with Response to Flotetuzumab, an Investigational CD123×CD3 Bispecific Dart® Molecule, in Acute Myeloid Leukemia

Jayakumar Vadakekolathu - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute myelocytic leukemia

Flotetuzumab

Identification of Prognostic Immunophenotypes at First Diagnosis in Patients with Acute Myeloid Leukemia (AML) By a Standardized Multicolor Flow Cytometry (MFC) Panel Originally Designed to Detect Measurable Residual Disease (MRD) at Follow-up

Maximilian Alexander A Rohnert - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute myelocytic leukemia

N/A

A Three-Gene Immune Signature Including IDO1, BIN1 and PLXNC1 Predicts Survival in Acute Myeloid Leukemia

Simone Ragaini - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute myelocytic leukemia

N/A

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