Journal article
Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria
RJ Kelly, B Höchsmann, J Szer, A Kulasekararaj, S De Guibert, A Röth, IC Weitz, E Armstrong, AM Risitano, CJ Patriquin, L Terriou, P Muus, A Hill, MP Turner, H Schrezenmeier, RP De Latour
New England Journal of Medicine | MASSACHUSETTS MEDICAL SOC | Published : 2015
Abstract
BACKGROUND Eculizumab, a humanized monoclonal antibody against complement protein C5 that inhibits terminal complement activation, has been shown to prevent complications of paroxysmal nocturnal hemoglobinuria (PNH) and improve quality of life and overall survival, but data on the use of eculizumab in women during pregnancy are scarce. METHODS We designed a questionnaire to solicit data on pregnancies in women with PNH and sent it to the members of the International PNH Interest Group and to the physicians participating in the International PNH Registry. We assessed the safety and efficacy of eculizumab in pregnant patients with PNH by examining the birth and developmental records of the chi..
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