For any patient with aHUS, the primary treatment goal is to reach remission.1 Remission can be broadly defined as maintaining the following for at least 3 months, during a minimum of 6 months treatment:1,3
It is recommended to treat for at least 6 months, within which there should be at least 3 months of remission*1,6
- While haematological parameters improve rapidly with C5 inhibition, improvement in renal outcomes is time-dependent – in some patients renal function continues to improve for up to 2 years after treatment initiation7-9
Balance individual risk factors and clinical needs – once a patient achieves remission, their continued need for treatment should be assessed carefully on a case-by-case basis, with informed and mutually agreed decisions made around treatment continuation and monitoring1-3
Following discontinuation of a C5 inhibitor patients should be monitored on an ongoing basis, including regular urine and blood tests for at least 24 months1,3
*Remission can be broadly defined as maintaining each of the following for at least 3 months: normalisation of platelet count, LDH and haptoglobin; improvement or stabilisation of kidney function (including stabilisation of residual CKD); optimal control of blood pressure and other extrarenal manifestations.1
†Analysis of data from the Global aHUS Registry. Included were patients who had received ≥1 month of C5 inhibitor therapy; had evidence of clinical response (LDH <1.5× ULN or platelet count ≥150,000/μL or eGFR >30 mL/min/1.73 m2 or a decrease in serum creatinine by 25% vs baseline), and had discontinued C5 inhibitor therapy with ≥6 months of follow-up after discontinuation, unless TMA recurred sooner. Pathogenic variants were more common among patients with versus those without TMA recurrence (30.3% [10/33] vs 15.0% [17/113]). The pathogenic variants tested were: CFH, C3, CFI, CFB, MCP, THBD and DGKE.2
C5, complement component 5; aHUS, atypical haemolytic uraemic syndrome; CKD, chronic kidney disease; ESRD, end-stage renal disease; LDH, lactate dehydrogenase; TMA, thrombotic microangiopathy.