Background Eculizumab is an orphan drug for Atypical Haemolytic Uremic Syndrome (aHUS). The disease is characterised by non-immune haemolytic anaemia, thrombocytopenia and renal impairment.

Purpose To assess the efficacy of eculizumab in adult patients, resistant to plasma treatment (PT), diagnosed with aHUS.

Materials and methods A systematic literature review was conducted focused on the efficacy. MEDLINE, EMBASE, CRD, and the Cochrane Library were searched to 2012 September to identify relevant studies.

Health technology agency reports, meta-analyses, systematic reviews, the European Medicines Agency drug assessment report, randomised controlled trials, controlled observational studies, and uncontrolled intervention studies evaluating the efficacy of eculizumab in adult PT-resistant patients with aHUS were included.

Study Selection, quality assessment, data extraction, and qualitative synthesis of the evaluated literature were undertaken independently by two researchers. Disagreements were resolved by consensus.

Results Only one prospective uncontrolled intervention study was included. The median follow-up of the study was 64 weeks (range: 2–90 weeks). No deaths were reported during the follow-up period. 87% of patients achieved a minimally important difference of 0.06 in the EuroQol 5D measurements. The thrombotic microangiopathy (TMA) event-free status (no PT sessions, new dialysis, and decrease in platelet count of >25% from baseline for at least 12 weeks) was achieved in 87% of the patients. The TMA intervention rate (PT session and dialysis/patient/day) was reduced from a median of 0.88 to 0 events/person/day. 53% of patients achieved a sustained change in estimated glomerular filtration rate (eGFR) ≥15 mL/min/1.73 m²), 76% of patients achieved a sustained 25% reduction from baseline in serum creatinine, 65% of patients improved at least one chronic kidney disease (CKD) stage.

Conclusions Eculizumab in the PT-resistant population improves quality of life and renal function, reduces the percentage of patients in dialysis and the necessity for PT. This systematic review could be used as a basis for developing recommendations for the use of eculizumab in this population.

No conflict of interest.