Conflict of interest: Nothing to report
Article first published online: 5 MAY 2010
Copyright © 2010 Wiley-Liss, Inc.
American Journal of Hematology
Volume 85, Issue 8, pages 553–559, August 2010
How to Cite
Hillmen, P., Elebute, M., Kelly, R., Urbano-Ispizua, A., Hill, A., Rother, R. P., Khursigara, G., Fu, C.-L., Omine, M., Browne, P. and Rosse, W. (2010), Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am. J. Hematol., 85: 553–559. doi: 10.1002/ajh.21757
Hillmen has received honoraria and research funding from and served as a consultant to and member of the Advisory Board of Alexion Pharmaceuticals. Kelly has received grant support from and served on Advisory Boards for Alexion. Omine received research funding and is a consultant for Alexion. Urbano-Ispizua has served on the Alexion Advisory Board. Rother and Khursigara are employees of Alexion Pharmaceuticals and own stock in the company.
- Issue published online: 15 JUL 2010
- Article first published online: 5 MAY 2010
- Accepted manuscript online: 5 MAY 2010 12:00AM EST
- Manuscript Accepted: 2 MAY 2010
- Manuscript Revised: 30 APR 2010
- Manuscript Received: 11 NOV 2009
Erratum: Erratum: “Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinura” by Hillmen et al., Am J Hematol 553–559, 2010, DOI number 21757
Vol. 85, Issue 11, 911, Article first published online: 14 SEP 2010
Paroxysmal nocturnal hemoglobinuria (PNH) is a debilitating and life-threatening disease in which lysis of PNH red blood cells frequently manifests with chronic hemolysis, anemia, and thrombosis. Renal damage in PNH is associated with chronic hemosiderosis and/or microvascular thrombosis. We determined the incidence of renal dysfunction or damage, defined by stages of chronic kidney disease (CKD), in a large cohort of PNH patients and evaluated the safety and efficacy of the complement inhibitor eculizumab in altering its progression. Renal dysfunction or damage was observed in 65% of the study population at baseline with 21% of patients with later stage CKD or kidney failure (glomerular filtration rate [GFR] ≤60 ml/min/1.73 m2; Stage 3, 4, or 5). Eculizumab treatment was safe and well-tolerated in patients with renal dysfunction or damage and resulted in the likelihood of improvement as defined as categorical reduction in CKD stage (P < 0.001) compared with baseline and to placebo (P = 0.04). Improvement in renal function was more commonly seen in patients with baseline CKD Stages 1–2 (67.1% improvement, P < 0.001) although improvement was also observed in patients with CKD Stages 3–4 (P = 0.05). Improvements occurred quickly and were sustained for at least 18 months of treatment. Patients categorized at CKD Stages 3–5 did not worsen during treatment with eculizumab. Overall, 40 (21%) of 195 patients who demonstrated renal dysfunction or damage at baseline were no longer classified as such after 18 months of treatment. Administration of eculizumab to patients with renal dysfunction or damage was well tolerated and was usually associated with clinical improvement. Am. J. Hematol. 85:553–559, 2010. © 2010 Wiley-Liss, Inc.