paroxysmal nocturnal hemoglobinuria (PNH) | Page 33 | Aplastic Anemia & MDS International Foundation

paroxysmal nocturnal hemoglobinuria (PNH)

Predictors of Hemoglobin Response to Eculizumab Therapy in Paroxysmal Nocturnal Hemoglobinuria

Dezern AE, Dorr D, Brodsky RA
Primary Author: 
Dezern AE
Journal Title: 
Eur J Haematol
Original Publication Date: 
Oct 2012

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.

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.


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