Eculizumab Protects Against TE and Prolongs Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Study

Original Publication Date: 
Friday, August 30, 2013

Note: Two studies from the Global PNH Registry presented at the European Hematology Association annual meeting in June focused on the effectiveness of eculizumab (Soliris®).

Abstract EHA18ABSSUB-5522

Gerard Socie, H. Schrezenmeier, P. Muus, J. Szer, A. Urbano-Ispizua, J. Maciejewski, R. Brodsky, M. Bessler, Y. Kanakura, W. Rosse, G. Khursigara, C. Bedrosian, P. Hillmen 

Thromboembolisms, or clots that block vessels, are one of the main causes of death in patients with paroxysmal nocturnal hemoglobinuria (PNH). Clinical trials have shown that eculizumab (Soliris®) reduces rates of hemolysis, or the destruction of platelets, and thromboembolism.

The purpose of this study was to evaluate eculizumab’s effectiveness in reducing rates of thromboembolism and death in 1,047 patients with PNH (mean age 45 years) who were enrolled in the PNH Registry, a worldwide collection of data. About half the patients were male. Half were treated with eculizumab, 28% used anticoagulant drugs (such as heparin or warfarin), and 18% used both eculizumab and anticoagulant drugs. On average, 23 months of follow-up data were available on these patients.

Key Findings:

  • At 2 years, 1% of patients treated with eculizumab had thromboembolism, compared to 3% in other patients.
  • The patients who were most likely to have thromboembolism were those who had recent red blood cell transfusions, a history of liver problems, and headaches.
  • After 2 years, 4% of patients treated with eculizumab had died, compared with 7% of other patients.
  • Risk of death was highest in patients who were 60 years old or older and had a score lower than 80 on the Karnofsky Performance Status Scale, which measures performance status on a scale of 0 (death) to 100 (normal health and no evidence of disease).


  • This analysis of an international group of “real-world” patients with PNH showed that eculizumab is associated with a lower risk of thromboembolism and death, which is consistent with the results of previous studies.
  • Recent red blood cell transfusions, which can indicate hemolysis, were associated with a greater risk of thromboembolism.
  • Not surprisingly, older age, lower performance status, and bone marrow transplantation were associated with a higher risk of death.
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