Recombinant ADAMTS13 for Hereditary Thrombotic Thrombocytopenic Purpura

This brief report describes the successful use of recombinant ADAMTS13 in a 27 year old patient with hereditary TTP during a high risk pregnancy. The patient, who had a history of fetal loss and stroke due to severe ADAMTS13 deficiency (<10% activity), presented with a plasma-refractory acute TTP episode at 30 weeks’ gestation. Weekly infusions of recombinant ADAMTS13 (40 U/kg) normalized her platelet count, stabilized fetal growth, and enabled delivery of a small-for-gestational age infant via cesarean section at 37 weeks. The case highlights recombinant ADAMTS13’s potential to overcome limitations of plasma based therapies (e.g., suboptimal ADAMTS13 levels, logistical burdens) and underscores the importance of early diagnosis in patients with obstetrical or thrombotic complications suggestive of hereditary TTP.

This brief report describes the successful use of recombinant ADAMTS13 in a 27 year old patient with hereditary TTP during a high risk pregnancy. The patient, who had a history of fetal loss and stroke due to severe ADAMTS13 deficiency (<10% activity), presented with a plasma-refractory acute TTP episode at 30 weeks’ gestation. Weekly infusions of recombinant ADAMTS13 (40 U/kg) normalized her platelet count, stabilized fetal growth, and enabled delivery of a small-for-gestational age infant via cesarean section at 37 weeks. The case highlights recombinant ADAMTS13’s potential to overcome limitations of plasma based therapies (e.g., suboptimal ADAMTS13 levels, logistical burdens) and underscores the importance of early diagnosis in patients with obstetrical or thrombotic complications suggestive of hereditary TTP.