Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population

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The pathophysiology of paroxysmal nocturnal hemoglobinuria and treatment with eculizumab

Paroxysmal nocturnal hemoglobinuria is a rare disorder of hemopoietic stem cells. Affected individuals have a triad of clinical associations - intravascular hemolysis, an increased risk of thromboembolism, and bone marrow failure. Most of the symptoms experienced in this disease occur due to the absence of complement regulatory proteins on the surface of the red blood cells. Complement activati...

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Paroxysmal Nocturnal Hemoglobinuria (PNH) — Symptoms and Diagnosis

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired defect in the myeloid stem cell lineage and can be seen as a rare, chronic, morbid disorder. Formally known as Marchiafava-Micheli syndrome, it received its current name as a descriptive term for the disease. Individuals afflicted with the disease wake up to dark, “cola” colored urine due to RBC breakdown and release of hemoglobin in the ...

متن کامل

Paroxysmal Nocturnal Hemoglobinuria (PNH) — Symptoms and Diagnosis

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired defect in the myeloid stem cell lineage and can be seen as a rare, chronic, morbid disorder. Formally known as Marchiafava-Micheli syndrome, it received its current name as a descriptive term for the disease. Individuals afflicted with the disease wake up to dark, “cola” colored urine due to RBC breakdown and release of hemoglobin in the ...

متن کامل

Paroxysmal Nocturnal Hemoglobinuria (PNH) — Symptoms and Diagnosis

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired defect in the myeloid stem cell lineage and can be seen as a rare, chronic, morbid disorder. Formally known as Marchiafava-Micheli syndrome, it received its current name as a descriptive term for the disease. Individuals afflicted with the disease wake up to dark, “cola” colored urine due to RBC breakdown and release of hemoglobin in the ...

متن کامل

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ژورنال

عنوان ژورنال: Blood

سال: 2020

ISSN: 0006-4971,1528-0020

DOI: 10.1182/blood-2020-141648