Highlights
– Longitudinal UK registry data from 104 confirmed congenital TTP (cTTP) patients (median follow-up 63 months) demonstrate that regular plasma prophylaxis markedly reduces acute TTP episodes and end-organ events (notably stroke/TIA and renal impairment).
– Despite biochemical and clinical benefits of plasma prophylaxis, many patients continue to experience substantial symptom burden (headache, fatigue, abdominal pain, mood disturbance) and practical problems (infusion reactions and transfusion dependence), prompting transition to recombinant ADAMTS13 in many cases.
