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Correspondence: Richard L. Haspel, MD, Beth Israel Deaconess Medical Center, Department of Pathology, 330 Brookline Avenue, Yamins 309, Boston, MA 02215; Phone 617-667-4344; Fax 617-667-7120; e-mail haspelr{at}yahoo.com
Abstract
A 45-year-old woman presents for evaluation as a result of persistent anemia. Ultimately, she is diagnosed with hemolytic anemia ascribed to paroxysmal nocturnal hemoglobinuria. She is otherwise well and has an otherwise normal complete blood cell count. She requires transfusion about once every 6 weeks for symptomatic anemia. She wonders whether she would benefit from eculizumab.
A double-blind randomized trial included 87 patients with a transfusion requirement of at least 4 red cell units in the previous 12 months and a platelet count of at least 100,000.3 Forty-four patients received placebo and 43 received eculizumab for 26 weeks. Eculizumab-treated patients had reduced need for transfusion (0 versus 10 units in patients not treated with eculizumab). Quality of life (QOL) scores improved with eculizumab treatment but not with placebo, although baseline QOL scores were not reported. Adverse events were similar in both groups.
A Phase III non-randomized trial included 97 patients with less pronounced transfusion requirements (at least 1 red cell transfusion in the last 2 years) and lower average platelet counts (at least 30,000).5 Patients were followed for 52 weeks and compared before and after treatment—both transfusion requirements and QOL improved with the greatest effects on dyspnea and fatigue. Of 44 serious adverse events, only 7 were considered possibly related to eculizumab and there were no severe bacterial infections.
These findings confirm that eculizumab decreases hemolysis and the resultant symptoms and transfusion requirements. Extended patient follow-up will further define adverse effects associated with long-term treatment and whether eculizumab reduces the incidence of other complications of PNH (e.g., thromboembolism).4 Based on this analysis, eculizumab should be considered in patients with significant symptoms from hemolysis that are not adequately managed with transfusion (Grade 1A recommendation). The cost and therefore a potential lack of access to this medication in some healthcare communities may influence decisions about its use.
Footnotes
1 Beth Israel Deaconess Hospital, Department of Pathology, Boston, MA 
2 St James’s University Hospital, Leeds, UK
Disclosures
Conflict-of-interest disclosure: R.L.H. declares no competing financial interests. P.H. has received honoraria/research funding from Alexion Pharmaceuticals Inc.
Off-label drug use: None disclosed.
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