Aim: Identification of
Dr.
Paroxysmal nocturnal hemoglobinuria is a clonal, hematopoietic stem cell disorder that manifests with hemolytic anemia and bone marrow failure. Eculizumab has been shown to improve anemia, decrease intravascular hemolysis and reduce the risk of thrombosis.
DESIGN AND METHODS:
This is a retrospective, single-center study of patients treated with Eculizumab and categorized according to response criteria. Complete response was defined as transfusion independence with normal hemoglobin for age/sex, absence of symptoms, and lactate dehydrogenase < 1.5 times the upper limit of normal. A good partial response was defined as decreased transfusions from pretreatment and lactate dehydrogenase <1.5 upper limit of normal without thrombosis. These patients did not achieve normal hemoglobins for age and sex. A suboptimal response was defined as unchanged transfusion needs and persistent of symptoms.
RESULTS:
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The recent availability of
No abstract available.
No abstract available.
