Recombinant ADAMTS13 for Hereditary Thrombotic Thrombocytopenic Purpura.

Asmis, Lars M; Serra, Andreas; Krafft, Alexander; Licht, Abraham; Leisinger, Elke; Henschkowski-Serra, Jana; Ganter, Michael T; Hauptmann, Steffen; Tinguely, Marianne; Kremer Hovinga, Johanna A (2022). Recombinant ADAMTS13 for Hereditary Thrombotic Thrombocytopenic Purpura. The New England journal of medicine, 387(25), pp. 2356-2361. Massachusetts Medical Society 10.1056/NEJMoa2211113

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A 27-year-old patient with a history of severe obstetrical complications and arterial thrombosis received a diagnosis of hereditary thrombotic thrombocytopenic purpura (TTP) due to severe ADAMTS13 deficiency when she presented with an acute episode in the 30th week of her second pregnancy. When the acute episode of hereditary TTP became plasma-refractory and fetal death was imminent, weekly injections of recombinant ADAMTS13 at a dose of 40 U per kilogram of body weight were initiated. The patient's platelet count normalized, and the growth of the fetus stabilized. At 37 weeks 1 day of gestation, a small-for-gestational-age boy was delivered by cesarean section. At the time of this report, the patient and her son were well, and she continued to receive injections of recombinant ADAMTS13 every 2 weeks. (Funded by the Swiss National Science Foundation.).

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