Benign Hematology Updates

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Benign Hematology Updates

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News & Literature Highlights

March 2022

Hereditary/Acquired Bleeding Disorders Successful Kidney Transplant for Nephropathic Cystinosis in a Patient with Von Willebrand Disease Type III: The First Case Report
Case Reports in Nephrology and Dialysis

Hereditary/Acquired Bleeding Disorders Potentially Inappropriate Medications and Anticholinergic and Sedative Burden in Older Patients With Haemophilia or Von Willebrand Disease: The M'HEMORRH-AGE Study
Journal of Clinical Pharmacy and Therapeutics

Hereditary/Acquired Bleeding Disorders Patient-Reported Outcomes in Autosomal Inherited Bleeding Disorders: A Systematic Literature Review
Haemophilia

Hemoglobinopathies Sustained Improvements in Patient-Reported Quality of Life Up to 24 Months Post-Treatment With LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
ASH 2021 Annual Meeting and Exposition

Hemoglobinopathies Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia
New England Journal of Medicine

Bone Marrow Failure Disorders FDA Approves Treatment for Anemia in Adults With Rare Inherited Disorder
US Food and Drug Administration

Bone Marrow Failure Disorders Composite Endpoint to Evaluate Complement Inhibition Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
Journal of Haematology

Bone Marrow Failure Disorders Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia
New England Journal of Medicine

Autoimmune Cytopenias All-Trans Retinoic Acid Plus Low-Dose Rituximab Vs Low-Dose Rituximab in Corticosteroid-Resistant or Relapsed ITP
ASH 2021 Annual Meeting and Exposition

Autoimmune Cytopenias FDA Roundup
US Food and Drug Administration

Autoimmune Cytopenias Tacrolimus Plus High-Dose Dexamethasone Versus High-Dose Dexamethasone Alone as First-Line Treatment for Adult Immune Thrombocytopenia: The Phase 2, Open Label, Randomized Trial (TARGET 020)
ASH 2021 Annual Meeting and Exposition

February 2022

Hereditary/Acquired Bleeding Disorders Prevalence and Risk Factors for Hepatocellular Carcinoma in Individuals With Hemophilia in the Era of Direct-Acting Antiviral Agents: A National Inpatient Sample Study
ASH 2021 Annual Meeting and Exposition

Hereditary/Acquired Bleeding Disorders Anticoagulation Use and Outcomes Among Patients with Atrial Fibrillation and Von Willebrand Disease
ASH 2021 Annual Meeting and Exposition

Hemoglobinopathies Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial
ASH 2021 Annual Meeting and Exposition

Hemoglobinopathies ASH President Applauds Introduction of Legislation to Establish Sickle Cell Disease Medicaid Demonstration Program
American Society of Hematology

Bone Marrow Failure Disorders Strong Predictors of Chromosomal Aberrations and Myeloid Neoplasia Following Immunosuppression Therapy for Severe Aplastic Anemia: A Retrospective Cohort Study
ASH 2021 Annual Meeting and Exposition

Bone Marrow Failure Disorders Spectrum of Molecular Modes of Immune Escape in Idiopathic Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
ASH 2021 Annual Meeting and Exposition

Autoimmune Cytopenias Machine-Learning Model for Resistance/Relapse Prediction in Immune Thrombocytopenia Using Gut Microbiota and Function Signatures
ASH 2021 Annual Meeting and Exposition

Autoimmune Cytopenias Safety, Pharmacokinetics and Preliminary Efficacy of HMPL-523 in Adult Patients With Primary Immune Thrombocytopenia: A Randomized, Double-Blind and Placebo-Controlled Phase 1b Study
ASH 2021 Annual Meeting and Exposition

January 2022

Hereditary/Acquired Bleeding Disorders The von Willebrand Disease Aging and Bleeding Correlation (VWD ABC) Study
ASH 2021 Annual Meeting and Exposition

Hereditary/Acquired Bleeding Disorders Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results From a Phase 3 Study to Evaluate Efficacy and Safety in People With Hemophilia A or B Without Inhibitors (ATLAS-A/B)
ASH 2021 Annual Meeting and Exposition

Hemoglobinopathies Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence After Treatment With Betibeglogene Autotemcel Gene Therapy: Results From Up to 7 Years of Follow-Up
ASH 2021 Annual Meeting and Exposition

Hemoglobinopathies Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults With Sickle Cell Disease: A Phase 1 Dose Escalation Study
ASH 2021 Annual Meeting and Exposition

Bone Marrow Failure Disorders When Does a PNH Clone Have Clinical Significance?
Hematology ASH Education Program

Bone Marrow Failure Disorders Efficacy and Safety of Pegcetacoplan Treatment in Complement-Inhibitor Naive Patients With Paroxysmal Nocturnal Hemoglobinuria: Results From the Phase 3 Prince Study
ASH 2021 Annual Meeting and Exposition

Autoimmune Cytopenias Tacrolimus Plus High-Dose Dexamethasone Versus High-Dose Dexamethasone Alone as First-Line Treatment for Adult Immune Thrombocytopenia: The Phase 2, Open Label, Randomized Trial (TARGET 020)
ASH 2021 Annual Meeting and Exposition

Autoimmune Cytopenias Updated Phase I/II Safety and Efficacy Results for Oral Bruton Tyrosine Kinase Inhibitor Rilzabrutinib in Patients with Relapsed/Refractory Immune Thrombocytopenia
ASH 2021 Annual Meeting and Exposition

December 2021

Hereditary/Acquired Bleeding Disorders Economic Burden Associated With Major Surgery in Patients With von Willebrand Disease: A United States Retrospective Administrative Database Analysis
Journal of Blood Medicine

Hereditary/Acquired Bleeding Disorders Subdural Hemorrhage Due to Acquired von Willebrand Syndrome in a Patient With Polycythemia Vera
Cureus

Hemoglobinopathies Significance of Genetic Modifiers of Hemoglobinopathies Leading Towards Precision Medicine
Scientific Reports (Nature)

Hemoglobinopathies Extended Phenotyping Does Not Preclude the Occurrence of Delayed Haemolytic Transfusion Reactions in Sickle Cell Disease
British Journal of Haematology

Bone Marrow Failure Disorders Randomized Multicenter Noninferiority Phase III Clinical Trial of the First Biosimilar of Eculizumab
Annals of Hematology

Bone Marrow Failure Disorders How We('ll) Treat Paroxysmal Nocturnal Hemoglobinuria: Diving Into the Future
British Journal of Haematology

Autoimmune Cytopenias Targeted Therapies for Immune Thrombocytic Purpura: A Meta-analysis of Randomized Controlled Trials
Annals of Hematology

Autoimmune Cytopenias Thromboembolic Complications in Autoimmune Hemolytic Anemia: Retrospective Study
European Journal of Haematology

November 2021

Hereditary/Acquired Bleeding Disorders Editor's Pick: von Willebrand Factor and ADAMTS13 in COVID-19 and Beyond: A Question of Balance
European Medical Journal

Hereditary/Acquired Bleeding Disorders PERSEPT 3: A Phase 3 Clinical Trial to Evaluate the Haemostatic Efficacy of Eptacog Beta (Recombinant Human FVIIa) in Perioperative Care in Subjects With Haemophilia A or B With Inhibitors
Haemophilia

Hemoglobinopathies Pathogenic BCL11A Variants Provide Insights Into the Mechanisms of Human Fetal Hemoglobin Silencing
PLoS Genetics

Hemoglobinopathies The Role of RBC Oxidative Stress in Sickle Cell Disease: From the Molecular Basis to Pathologic Implications
Antioxidants (Basel)

Bone Marrow Failure Disorders Advancing Therapeutic Complement Inhibition in Hematologic Diseases: PNH and Beyond
Blood

Bone Marrow Failure Disorders A Novel Patient-Reported Outcome Instrument Assessing the Symptoms of Paroxysmal Nocturnal Hemoglobinuria, the PNH-SQ
Journal of Patient-Reported Outcomes

Autoimmune Cytopenias Treatment-Refractory, Primary Immune Thrombocytopenic Purpura in a Patient With Celiac Disease
American Journal of Case Reports

Autoimmune Cytopenias Novel Therapies for Immune Thrombocytopenia
British Journal of Haematology

October 2021

Hemoglobinopathies Developing an Emergency Department Order Set to Treat Acute Pain in Sickle Cell Disease
Journal of the American College of Emergency Physicians Open (JACEP Open)

Hereditary/Acquired Bleeding Disorders Bernard Soulier Syndrome: A Rare, Frequently Misdiagnosed and Poorly Managed Bleeding Disorder
BMJ Case Reports

Hereditary/Acquired Bleeding Disorders Plasma-Derived VWF/FVIII Concentrate in Pediatric Patients With Severe Hemophilia A
Journal of Blood Medicine

Hemoglobinopathies Manifestations of HbSE Sickle Cell Disease: A Systematic Review
Journal of Translational Medicine

Bone Marrow Failure Disorders Concept Confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in Rare Paroxysmal Nocturnal Hemoglobinuria
Journal of Patient Reported Outcomes

Bone Marrow Failure Disorders COVID-19 Vaccines Induce Severe Hemolysis in Paroxysmal Nocturnal Hemoglobinuria
Blood

Bone Marrow Failure Disorders COVID‐19 in Patients With Paroxysmal Nocturnal Haemoglobinuria: An Italian Multicentre Survey
British Journal of Haematology

Autoimmune Cytopenias Platelet Desialylation and TFH cells—The Novel Pathway of Immune Thrombocytopenia
Experimental Hematology & Oncology

Autoimmune Cytopenias IL-6 Expression Helps Distinguish Castleman’s Disease From IgG4-Related Disease in the Lung
BMC Pulmonary Medicine

Autoimmune Cytopenias Low-Dose Decitabine Modulates T-Cell Homeostasis and Restores Immune Tolerance in Immune Thrombocytopenia
Blood

September 2021

Hereditary/Acquired Bleeding Disorders Novel Simulation Method Predicts Blood Flow Conditions Behind von Willebrand Disease
EurekAlert! Science News

Hereditary/Acquired Bleeding Disorders New Data Reinforce Safety Profile of Emicizumab in Patients With Hemophilia A
Pharmacy Times

Hemoglobinopathies Techniques for the Detection of Sickle Cell Disease: A Review
Micromachines (Basel)

Hemoglobinopathies Impact of Hemoglobin Biophysical Studies on Molecular Pathogenesis and Drug Therapy for Sickle Cell Disease
Molecular Aspects of Medicine

Bone Marrow Failure Disorders Pregnancy with Paroxysmal Nocturnal Hemoglobinuria: A Case Series with Review of the Literature
Saudi Journal of Medicine & Medical Sciences

Bone Marrow Failure Disorders Addition of Iptacopan, an Oral Factor B Inhibitor, to Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria and Active Haemolysis: An Open-Label, Single-Arm, Phase 2, Proof-of-Concept Trial
The Lancet Hematology

Autoimmune Cytopenias Multicentric Castleman’s Disease in HIV patients: A Single-Center Cohort Diagnosed From 2008 to 2018
Infection

Autoimmune Cytopenias Immune Thrombocytopenia: Recent Advances in Pathogenesis and Treatments
HemaSphere

August 2021

Hereditary/Acquired Bleeding Disorders Novel Tool Developed to Measure the Impact of Hemophilia on Families
Hematology Advisor

Hereditary/Acquired Bleeding Disorders Response to Treatment for Gastro-intestinal Bleeding in Patients Affected by von Willebrand Disease
International Society on Thrombosis and Haemostasis 2021

Hemoglobinopathies Fixing the Sickle Cell Disease Gene
NIH Research Matters

Hemoglobinopathies Neutrophil Binding on E-selectin: A Potential Biomarker of Clinical Course and Response to Therapy in Sickle Cell Disease
International Society on Thrombosis and Haemostasis 2021

Bone Marrow Failure Disorders Clinically Meaningful and Long-Term Improvements in Fatigue With the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: Post-hoc Analyses From the PEGASUS Study Week 48
International Society on Thrombosis and Haemostasis 2021

Bone Marrow Failure Disorders Recurrent Thromboembolic Risk in Paroxysmal Nocturnal Hemoglobinuria Patients Not on Anticoagulation Treated With Terminal Complement Inhibition
International Society on Thrombosis and Haemostasis 2021

Autoimmune Cytopenias Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia: ADVANCE SC, a Global Phase 3 Clinical Trial in Progress
International Society on Thrombosis and Haemostasis 2021

Autoimmune Cytopenias Time Course of Response to Fostamatinib, an Oral Spleen Tyrosine Kinase (SYK) Inhibitor for the Treatment of Immune Thrombocytopenia (ITP)
International Society on Thrombosis and Haemostasis 2021

July 2021

Hereditary/Acquired Bleeding Disorders Type 3 von Willebrand Disease in Pregnancy: A Systemic Literature Review
American Journal of Perinatology

Hereditary/Acquired Bleeding Disorders First-in-Human Results Show Promise With BT200, a New Anti-von Willebrand Factor Agent
Hematology Advisor

Hemoglobinopathies Luspatercept Safe, Effective in Nontransfusion-Dependent Beta-Thalassemia
HemOnc Today [Healio]

Hemoglobinopathies Cost-Effectiveness of a Hypothetical Cell or Gene Therapy Cure for Sickle Cell Disease
Scientific Reports [Nature Research]

Bone Marrow Failure Disorders Agios Launches myAgios® Patient Support Services for Pyruvate Kinase Deficiency
Globe Newswire

Bone Marrow Failure Disorders FDA Approves New Treatment for Adults With Serious Rare Blood Disease
U.S. Food & Drug Administration

Autoimmune Cytopenias How I Treat Warm Autoimmune Hemolytic Anemia
Blood

Autoimmune Cytopenias Clinical Spectrum, Outcome and Management of Immune Thrombocytopenia Associated With Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia
Haematologica

June 2021

Hereditary/Acquired Bleeding Disorders Criteria for Low von Willebrand Factor Diagnosis and Risk Score to Predict Future Bleeding
Journal of Thrombosis and Haemostasis

Hereditary/Acquired Bleeding Disorders Elevated von Willebrand Factor Levels During Heavy Menstrual Bleeding Episodes Limit the Diagnostic Utility for von Willebrand Disease
Research and Practice in Thrombosis and Haemostasis

Hemoglobinopathies Impact of Renal Function on Hydroxyurea Exposure in Sickle-Cell Disease Patients
British Journal of Clinical Pharmacology

Hemoglobinopathies Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial
JAMA

Bone Marrow Failure Disorders Reduced Red Blood Cell Surface Level of Factor H as a Mechanism Underlying Paroxysmal Nocturnal Hemoglobinuria
Leukemia

Bone Marrow Failure Disorders The Variable Manifestations of Disease in Pyruvate Kinase Deficiency and Their Management
Haematologica

Autoimmune Cytopenias The Role of Novel Agents in Treating CLL-Associated Autoimmune Hemolytic Anemia
Journal of Clinical Medicine

Autoimmune Cytopenias Practical Recommendations for the Management of Patients With ITP During the COVID-19 Pandemic
Mediterranean Journal of Hematology and Infectious Diseases

May 2021

Hereditary/Acquired Bleeding Disorders Future Directions in Acquired Hemophilia A
Blood

Hemoglobinopathies Cancer Diagnosis in Patient Given LentiGlobin Revised to Severe Anemia
Sickle Cell Disease News

Hemoglobinopathies A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease
New England Journal of Medicine

Bone Marrow Failure Disorders Coronavirus Disease COVID-19: EBMT Recommendations Version 15
European Group for Blood and Marrow Transplantation

Bone Marrow Failure Disorders C3 Inhibition With Pegcetacoplan in Subjects With Paroxysmal Nocturnal Hemoglobinuria Treated With Eculizumab
American Journal of Hematology

Autoimmune Cytopenias Immune Thrombocytopenia in a 22‐Year‐Old Post Covid‐19 Vaccine
American Journal of Hematology

Autoimmune Cytopenias Cytogenetic and Molecular Aberrations and Worse Outcome for Male Patients in Systemic Mastocytosis
Theranostics

Hereditary/Acquired Bleeding Disorders Activation of von Willebrand Factor Via Mechanical Unfolding of Its Discontinuous Autoinhibitory Molecule
Nature Communications

April 2021

Hemoglobinopathies Usefulness of NGS for Diagnosis of Dominant Beta-Thalassemia and Unstable Hemoglobinopathies in Five Clinical Cases
Frontiers in Physiology

Bone Marrow Failure Disorders A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective Erythropoiesis in Pyruvate Kinase Deficiency
Frontiers in Physiology

Autoimmune Cytopenias Weathering the COVID-19 Storm: Lessons From Hematologic Cytokine Syndromes
Blood Reviews

Hereditary/Acquired Bleeding Disorders ASH ISTH NHF WFH 2021 Guidelines on the Management of von Willebrand Disease
Blood Advances

Hemoglobinopathies EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
BioSpace

Bone Marrow Failure Disorders Characterization of Breakthrough Hemolysis Events Observed in the Phase 3 Randomized Studies of Ravulizumab Versus Eculizumab in Adults With Paroxysmal Nocturnal Hemoglobinuria
Haematologica

Autoimmune Cytopenias Immune Thrombocytopenic Purpura Complicated by Hepatitis C Virus–Related Membranoproliferative Glomerulonephritis After Rituximab Therapy
Internal Medicine

Hereditary/Acquired Bleeding Disorders ASH ISTH NHF WFH 2021 Guidelines on the Diagnosis of von Willebrand Disease
Blood Advances

Hemoglobinopathies What Are the Key Considerations When Prescribing Pharmacotherapy for Sickle Cell Anemia?
Expert Opinion on Pharmacotherapy

Bone Marrow Failure Disorders Narrative Review of Aplastic Anemia—The Importance of Supportive Treatment
Annals of Palliative Medicine

Autoimmune Cytopenias Severe Warm Autoimmune Hemolytic Anemia in COVID-19 Managed With Least Incompatible RBC Product and Glucocorticoids
Annals of Hematology

Hereditary/Acquired Bleeding Disorders Efanesoctocog Alfa Granted FDA Fast Track Designation for Treatment of Hemophilia A
Sanofi

Hemoglobinopathies CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
New England Journal of Medicine

Bone Marrow Failure Disorders Detectable Mutations Precede Late Myeloid Neoplasia in Aplastic Anemia
Haematologica

Autoimmune Cytopenias Efficacy of Recombinant Erythropoietin in Autoimmune Hemolytic Anemia: A Multicenter International Study
Haematologica

Hereditary/Acquired Bleeding Disorders 2021 Clinical Trials Update: Innovations in Hemophilia Therapy
American Journal of Hematology

Class Categories

Hereditary/Acquired Bleeding Disorders

Autoimmune Cytopenias

Bone Marrow Failure Disorders

Hemoglobinopathies

BENIGN HEMATOLOGY BASICS

Benign Hematology Overview

Hereditary and Acquired Bleeding Disorders: Hemophilia

Hereditary and Acquired Bleeding Disorders: von Willebrand Disorders

Autoimmune Cytopenias

Bone Marrow Disorders

Hemoglobinopathies

Bone Marrow Failure Disorders

Meet the Faculty

Resources

Hereditary/Acquired Bleeding Disorders von Willebrand Disease: Piecing Together the VWD Puzzle

Hereditary/Acquired Bleeding Disorders Mild Hemophilia Is Not Always Mild…All About Mild Hemophilia

Hemoglobinopathies Neurocognitive Risk in Sickle Cell Disease: Utilizing Neuropsychology Services to Manage Cognitive Symptoms and Functional Limitations

Hemoglobinopathies GRNDaD: Big Data and Sickle Cell Disease

Bone Marrow Failure Disorders Clinical Manifestations [of paroxysmal nocturnal hemoglobinuria]

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Videos

Hemoglobinopathies Understanding Sickle Cell Disease and the Transition Into Adult Care

Bone Marrow Failure Disorders Diagnosis and Management of PNH: A Case Study

Hereditary/Acquired Bleeding Disorders Insights Into Hemophilia Treatment Trends: An Interview With Michelle L. Witkop, DNP, FNP-BC, From the National Hemophilia Foundation

Autoimmune Cytopenias Treating Patients With Chronic ITP

Hemoglobinopathies Beta-Thalassemia in Adults

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