Pregnancy outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab: a Japanese experience and updated review

Pregnancy with paroxysmal nocturnal hemoglobinuria (PNH) is associated with significant risk of complications, such as life-threatening thrombosis. Recently, eculizumab has come into clinical use and revolutionized the treatment of PNH. However, clinical information regarding eculizumab use for PNH...

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Bibliographic Details
Published in:International journal of hematology 2016-06, Vol.103 (6), p.703-712
Main Authors: Miyasaka, Naoyuki, Miura, Osamu, Kawaguchi, Tatsuya, Arima, Nobuyoshi, Morishita, Eriko, Usuki, Kensuke, Morita, Yasuyoshi, Nishiwaki, Kaichi, Ninomiya, Haruhiko, Gotoh, Akihiko, Imashuku, Shinsaku, Urabe, Akio, Shichishima, Tsutomu, Nishimura, Jun-ichi, Kanakura, Yuzuru
Format: Article
Language:English
Subjects:
Adult
Antibodies, Monoclonal, Humanized - therapeutic use
Female
Hematology
Hemoglobinuria, Paroxysmal - drug therapy
Humans
Medicine
Medicine & Public Health
Oncology
Original Article
Pregnancy
Pregnancy Complications, Hematologic - drug therapy
Pregnancy Outcome
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Summary:Pregnancy with paroxysmal nocturnal hemoglobinuria (PNH) is associated with significant risk of complications, such as life-threatening thrombosis. Recently, eculizumab has come into clinical use and revolutionized the treatment of PNH. However, clinical information regarding eculizumab use for PNH during pregnancy is limited. The present report describes pregnancies with PNH treated with eculizumab that were registered with the Japan PNH study group and reviews the literature. In case 1, the patient received eculizumab throughout pregnancy and delivered a healthy neonate at term, although breakthrough hemolysis occurred at 20 weeks of gestation. In case 2, the patient discontinued eculizumab before pregnancy and developed preeclampsia at 27 weeks of gestation. She received eculizumab and delivered a preterm, but healthy, neonate by cesarean section. In case 3, the patient received eculizumab from 18 weeks of gestation and delivered a healthy neonate at term without any complications. Reports of 11 pregnant women treated with eculizumab were identified in the literature. Of 14 pregnancies, including our own cases, breakthrough hemolysis and preeclampsia occurred in five and two cases, respectively. There were no thrombotic complications, maternal or neonatal deaths, or fetal structural abnormalities. Thus, eculizumab appears to be safe and effective for managing PNH during pregnancy.
ISSN:0925-5710
1865-3774
DOI:10.1007/s12185-016-1946-x