Paroxysmal nocturnal hemoglobinuria

Eculizumab

Regimen:

IV 600 mg weekly for 4 doses, followed by 900 mg 1 week later; then 900 mg every 2 weeks as maintenance treatment

Supportive medications:

Meningococcal vaccine at least 2 weeks prior to treatment initiation; may revaccinate per current guidelines

Reference:

1) Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006;355:1233-1243

2) Schubert, J., Hillmen, P., Röth, A., Young, N. S., Elebute, M. O., Szer, J., Gianfaldoni, G., Socié, G., Browne, P., Geller, R., Rother, R. P. and Muus, P. (2008), Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria. British Journal of Haematology, 142: 263–272

3) Kelly RJ, Hill A, Arnold LM, et al.(2011) Long term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood 117(25):6786–6792

4) Hill A, Kelly RJ, Kulasekararaj AG, et al. Eculizumab in paroxysmal nocturnal hemoglobinuria (PNH): a report of all 153 patients treated in the UK. Blood. Vol. 120.(21) 2012. p. 3472