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NEW STUDY: Eculizumab in Pregnant Patients with PNH

The New England Journal of Medicine (NEJM) recently published a study focusing on the use of eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria (PNH).

A team of doctors acknowledged that eculizumab prevents complications of PNH and can lead to an improved quality of life, as well as survival, but noted that there are limited data on the use of eculizumab in pregnant women with PNH. In order to solicit data, the team of doctors created a questionnaire and sent it to pregnant women who belong to the International PNH Interest Group, as well as physicians associated with the International PNH Registry. The questionnaire focused on the birth and developmental records of the child dren born, as well as adverse events in mothers with PNH. The data collected was used to determine the safety and efficacy of eculizumab during pregnancy.

94 questionnaires were distributed and 75 were returned, representing a response rate of 80 per cent. Data on 75 pregnancies in 61 women with PNH were evaluated, and it was concluded that eculizumab provided benefit for pregnant women with PNH, as indicated by a high rate of fetal survival and a low rate of maternal complications.

To read the full study, please click HERE.