Eculizumab: A guide to its use in atypical haemolytic uraemic syndrome

被引：0
作者：
Keating G.M. [1 ]
机构：
[1] Adis, North Shore, Auckland 0754, 41 Centorian Drive, Mairangi Bay
来源：
Drugs & Therapy Perspectives | 2014年 / 30卷 / 5期
关键词：
Chronic Kidney Disease; Chronic Kidney Disease Stage; Paroxysmal Nocturnal Haemoglobinuria; Eculizumab; Thrombotic Microangiopathy;
D O I：
10.1007/s40267-014-0120-x
中图分类号：
学科分类号：
摘要：
The recombinant humanized monoclonal antibody eculizumab (Soliris®) is a complement inhibitor that is indicated for use in the treatment of atypical haemolytic uraemic syndrome (aHUS). In two open-label, single-arm, multinational, 26-week, phase II trials, intravenous eculizumab inhibited complement-mediated thrombotic microangiopathy in patients aged ≥12 years with aHUS. At 26 weeks, the platelet count was significantly increased in patients with progressing thrombotic microangiopathy, and thrombotic microangiopathic event-free status was achieved in 80 % of patients with a long disease duration and chronic kidney disease who had received previous long-term plasma exchange/infusion. The beneficial effects of eculizumab on renal function were maintained long term and the proportion of patients meeting criteria for improvement in renal function increased over 2 years of therapy. Intravenous eculizumab was generally well tolerated. In conclusion, eculizumab is a valuable new agent for use in the treatment of aHUS. © 2014 Springer International Publishing.
引用
收藏
页码：166 / 172
页数：6
相关论文
共 35 条
  • [1] Westra D., Wetzels J.F., Volokhina E.B., Et al., A new era in the diagnosis and treatment of atypical haemolytic uraemic syndrome, Neth J Med, 70, 3, pp. 121-129, (2012)
  • [2] Loirat C., Fremeaux-Bacchi V., Atypical hemolytic uremic syndrome, Orphanet J Rare Dis, 6, (2011)
  • [3] Legendre C.M., Licht C., Muus P., Et al., Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome, N Engl J Med, 368, 23, pp. 2169-2181, (2013)
  • [4] Campistol J.M., Arias M., Ariceta G., Et al., An update for atypical haemolytic uraemic syndrome: Diagnosis and treatment: A consensus document, Nefrologia, 33, 1, pp. 27-45, (2013)
  • [5] Waters A.M., Licht C., aHUS caused by complement dysregulation: New therapies on the horizon, Pediatr Nephrol, 26, 1, pp. 41-57, (2011)
  • [6] Hodgkins K.S., Bobrowski A.E., Lane J.C., Et al., Clinical grand rounds: Atypical hemolytic uremic syndrome, Am J Nephrol, 35, 5, pp. 394-400, (2012)
  • [7] Noris M., Remuzzi G., Atypical hemolytic-uremic syndrome, N Engl J Med, 361, 17, pp. 1676-1687, (2009)
  • [8] Langman C., Systemic multi-organ complications in atypical hemolytic uremic syndrome (aHUS): Retrospective study in a medical practice setting, Haematologica, 97, SUPPL. 1, pp. 195-196, (2012)
  • [9] Zuber J., Fakhouri F., Roumenina L.T., Et al., Use of eculizumab for atypical haemolytic uraemic syndrome and C3 glomerulopathies, Nat Rev Nephrol, 8, 11, pp. 643-657, (2012)
  • [10] Schmidtko J., Peine S., El-Housseini Y., Et al., Treatment of atypical hemolytic uremic syndrome and thrombotic microangiopathies: A focus on eculizumab, Am J Kidney Dis, 61, 2, pp. 289-299, (2013)
1234