Summary of findings 2. Ravulizumab versus placebo or alternative treatment for adults with atypical haemolytic uraemic syndrome (aHUS).

Ravulizumab versus placebo or alternative treatment for adults with aHUS
Patient or population: adults with aHUS Settings: inpatient Intervention: ravulizumab Comparison: placebo or alternative treatment
Outcomes	Illustrative comparative risks* (95% CI)		Relative effect (95% CI)	No. of Participants (studies)	Quality of the evidence (GRADE)	Comments
	Assumed risk	Corresponding risk
	Risk with placebo or alternative treatment	Risk with ravulizumab treatment
Death	N/A	4/58	N/A	58 (1 single‐arm study)	⊕⊝⊝⊝ very low	Four deaths occurred, including one in a patient ultimately excluded based on eligibility criteria but who had received one dose of ravulizumab. No deaths were considered treatment‐related by the study investigators
Requirement for KRT	N/A	N/A	N/A	56 (1 single‐arm study)	⊕⊝⊝⊝ very low	29/56 were undergoing dialysis at initiation of ravulizumab therapy. Of these patients, 12 (41%) continued to require regular dialysis after 26 weeks of treatment representing a 59% reduction in dialysis requirement
Disease remission	N/A	30/56	N/A	56 (1 single‐arm study)	⊕⊝⊝⊝ very low	30/56 patients treated with ravulizumab achieved complete TMA response after 26 weeks of treatment. Median time to complete TMA response was 86 days
Change in eGFR	N/A	N/A	N/A	56 (1 single‐arm study)	⊕⊝⊝⊝ very low	In patients treated with ravulizumab, mean change in eGFR over 26 weeks was 35 ± 35 mL/min/1.73 m²
HRQoL	N/A	N/A	N/A	44 (1 single‐arm study)	⊕⊝⊝⊝ very low	A clinically meaningful improvement in FACIT‐F score (≥ 3‐point increase) was observed in 84% of patients treated with ravulizumab
Adverse events	N/A	58/58	N/A	58 (1 single‐arm study)	⊕⊝⊝⊝ very low	Adverse events occurred in 100% of patients treated with ravulizumab. Serious adverse events occurred in 52% of patients. The most commonly reported events included headache (36%), diarrhoea (31%), vomiting (26), hypertension (22%), nausea (22%) and urinary tract infection (17%)
Meningococcal infection	N/A	0/58	N/A	58 (1 single‐arm study)	⊕⊝⊝⊝ very low	No patients treated with ravulizumab developed meningococcal infection
*The basis for the assumed risk (e.g. the median control group risk across studies) is provided in footnotes. The corresponding risk (and its 95% CI) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). CI: Confidence interval; RR: Risk Ratio; N/A: not applicable; KRT: Kidney replacement therapy; TMA: Thrombotic microangiopathy; eGFR: Estimated glomerular filtration rate; HRQoL: Health‐related quality of life; FACIT‐F: Functional assessment of chronic illness therapy ‐ fatigue.
GRADE Working Group grades of evidence High quality: Further research is very unlikely to change our confidence in the estimate of effect. Moderate quality: Further research is likely to have an important impact on our confidence in the estimate of effect and may change the estimate. Low quality: Further research is very likely to have an important impact on our confidence in the estimate of effect and is likely to change the estimate. Very low quality: We are very uncertain about the estimate.