Document Detail

Predictors of hemoglobin response to eculizumab therapy in paroxysmal nocturnal hemoglobinuria.
MedLine Citation:
PMID:  23046169     Owner:  NLM     Status:  MEDLINE    
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) 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 (CR) 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 (GPR) 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: Thirty patients with PNH clones were treated with eculizumab and classified as complete responders (four patients), good partial responders (16), and suboptimal responders (10) over 863 patient-months of treatment. Complete responders had a decrease in red cell clone size, while good partial responders had an increase. Thirteen patients treated did not meet inclusion criteria for the clinical trials of eculizumab due to lack of transfusions or thrombocytopenia; eight had at least a GPR.
CONCLUSIONS: Eculizumab is efficacious in patients with PNH, but responses can vary and may depend on underlying marrow failure, underlying inflammatory conditions and red cell clone size following treatment. Normalization of hemoglobin with decrease in red cell clone size may predict CR.
Amy E DeZern; Donna Dorr; Robert A Brodsky
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural     Date:  2012-11-22
Journal Detail:
Title:  European journal of haematology     Volume:  90     ISSN:  1600-0609     ISO Abbreviation:  Eur. J. Haematol.     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2012-12-17     Completed Date:  2013-02-06     Revised Date:  2014-01-09    
Medline Journal Info:
Nlm Unique ID:  8703985     Medline TA:  Eur J Haematol     Country:  England    
Other Details:
Languages:  eng     Pagination:  16-24     Citation Subset:  IM    
Copyright Information:
© 2012 John Wiley & Sons A/S.
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MeSH Terms
Antibodies, Monoclonal, Humanized / administration & dosage,  adverse effects,  therapeutic use*
Hemoglobins / metabolism*
Hemoglobinuria, Paroxysmal / blood*,  drug therapy*,  mortality
Middle Aged
Retrospective Studies
Treatment Outcome
Young Adult
Grant Support
5K12HL087169-05/HL/NHLBI NIH HHS; P01 CA70970/CA/NCI NIH HHS; P30 CA006973/CA/NCI NIH HHS
Reg. No./Substance:
0/Antibodies, Monoclonal, Humanized; 0/Hemoglobins; A3ULP0F556/eculizumab

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