PMID: 30240381Sep 22, 2018Paper

Atypical hemolytic uremic syndrome: a new drug program and first Polish adult patient treated with eculizumab

Polski merkuriusz lekarski : organ Polskiego Towarzystwa Lekarskiego
Jakub RuszkowskiAlicja Dębska-Ślizień

Abstract

Atypical hemolytic uremic syndrome (aHUS) is an extremely rare disease characterized by acute kidney injury, thrombocytopenia, and microangiopathic hemolytic anemia. Majority of patients have an underlying complement abnormality what makes aHUS possible to treat using eculizumab, antibody against the complement protein C5. Up to 2018, Polish adult patients were treated using glucocorticosteroids, plasma exchange or plasma infusion. Unfortunately, such protocols/ interventions were associated with poor prognosis; about 67% of adult patients with aHUS had been progressing to end-stage renal disease or had died within three years after diagnosis. Release of eculizumab significantly improve the prognosis of aHUS. We describe new drug program and case of first included adult patient: after ineffective nonspecific treatment of aHUS with plasma infusions (10 units), plasma exchanges (7 sessions) and glucocorticosteroids, we decided to introduce eculizumab. The clinical and laboratory tests showed effectiveness of the therapy after sixth infusion; no adverse effects were observed.

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