Improved Fatigue and Quality-of-Life in Patients with Paroxysmal Nocturnal Hemoglobinuria During Treatment with Eculizumab: Data from the Global PNH Registry

Original Publication Date: 
Friday, August 30, 2013

This study was presented at the European Hematology Association annual meeting in June 2013.

Abstract EHA18ABSSUB-4921

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

Paroxysmal nocturnal hemoglobinuria (PNH) causes hemolysis, or destruction of some or all of a patient’s red blood cells. This hemolysis and the other symptoms of PNH have a major impact on patients’ quality of life. Eculizumab (Soliris®) can prevent hemolysis in people with PNH.

The PNH Registry is a worldwide collection of data on patients with PNH. An international research team evaluated changes in quality of life and fatigue in 62 patients (median age 41) in the PNH Registry who had been treated with eculizumab for 12 months. The patients completed questionnaires to assess their fatigue and quality of life before starting treatment and every 6 months after that.

Key Findings:

  • After 6 months of eculizumab treatment, patients had less fatigue, pain, shortness of breath, and nausea and vomiting. They also had better social and physical functioning.
  • Most of these aspects of quality of life continued to improve over the next 6 months.
  • At 12 months, patients also had less insomnia and better emotional functioning than before they started treatment.
  • At least 40% of patients had improvements in all measures of functioning and in the symptoms of fatigue, pain, and shortness of breath at 6 and 12 months and in insomnia at 12 months.


  • Patients with PNH had significant improvements in fatigue and other PNH-related symptoms after starting eculizumab treatment.
  • These improvements were associated with significant increases in quality of life.
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