Bone Marrow Failure Disorders
News & Literature Highlights
March 2022
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Bone Marrow Failure Disorders
FDA Approves Treatment for Anemia in Adults With Rare Inherited Disorder
US Food and Drug Administration
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Bone Marrow Failure Disorders
Composite Endpoint to Evaluate Complement Inhibition Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
Journal of Haematology
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Bone Marrow Failure Disorders
Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia
New England Journal of Medicine
February 2022
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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
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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
January 2022
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Bone Marrow Failure Disorders
When Does a PNH Clone Have Clinical Significance?
Hematology ASH Education Program
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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
December 2021
November 2021
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Bone Marrow Failure Disorders
Advancing Therapeutic Complement Inhibition in Hematologic Diseases: PNH and Beyond
Blood
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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
October 2021
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Bone Marrow Failure Disorders
Concept Confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in Rare Paroxysmal Nocturnal Hemoglobinuria
Journal of Patient Reported Outcomes
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Bone Marrow Failure Disorders
COVID-19 Vaccines Induce Severe Hemolysis in Paroxysmal Nocturnal Hemoglobinuria
Blood
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Bone Marrow Failure Disorders
COVID‐19 in Patients With Paroxysmal Nocturnal Haemoglobinuria: An Italian Multicentre Survey
British Journal of Haematology
September 2021
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Bone Marrow Failure Disorders
Pregnancy with Paroxysmal Nocturnal Hemoglobinuria: A Case Series with Review of the Literature
Saudi Journal of Medicine & Medical Sciences
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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
August 2021
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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
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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
July 2021
June 2021
May 2021
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Bone Marrow Failure Disorders
Coronavirus Disease COVID-19: EBMT Recommendations Version 15
European Group for Blood and Marrow Transplantation
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Bone Marrow Failure Disorders
C3 Inhibition With Pegcetacoplan in Subjects With Paroxysmal Nocturnal Hemoglobinuria Treated With Eculizumab
American Journal of Hematology
April 2021
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Bone Marrow Failure Disorders
A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective Erythropoiesis in Pyruvate Kinase Deficiency
Frontiers in Physiology
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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
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Bone Marrow Failure Disorders
Narrative Review of Aplastic Anemia—The Importance of Supportive Treatment
Annals of Palliative Medicine
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Bone Marrow Failure Disorders
Detectable Mutations Precede Late Myeloid Neoplasia in Aplastic Anemia
Haematologica
