Eculizumab Shows No Benefit in Kids With Rare Renal Disorder
A meta-analysis found that paediatric patients with Shiga toxin–producing Escherichia coli –associated haemolytic uremic syndrome (STEC-HUS) and neurologic complications who received eculizumab did not show a significant improvement in neurologic outcomes compared with those who received standard supportive care, despite eculizumab being more commonly used for these patients.
METHODOLOGY:
HUS, characterised by microangiopathic haemolytic anaemia, thrombocytopenia, and acute kidney injury, is primarily caused by STEC in 90% of paediatric cases. Even though eculizumab has shown efficacy in atypical HUS, its use in STEC-HUS is still under investigation.
Researchers conducted a systematic review and meta-analysis of seven European studies involving paediatric patients with STEC-HUS and neurologic complications (n = 529; age range, 0-18 years) treated with eculizumab to examine its effect on neurologic prognosis.
Most patients were younger than 5 years; 135 (25.5%) patients developed neurologic complications ranging from mild irritability to seizures, stupor, and coma, and only 59 (11.1%) received eculizumab.
All the included studies used a retrospective cohort design, with only two being multicentric.
TAKEAWAY:
Patients with neurologic involvement had significantly higher odds of receiving eculizumab therapy than those without (odds ratio, 13.03; 95% CI, 4.40-38.75); 74.5% of patients in the eculizumab group vs 19.3% of those in the non-eculizumab group showed neurologic symptoms.
Among patients with neurologic involvement, 64% of those in the eculizumab group vs 89% of those in the non-eculizumab group showed improvement, with no statistically significant difference between groups ( P = .10).
IN PRACTICE:
"While eculizumab may offer benefits for specific subgroups, current evidence does not support its routine use to improve neurological outcomes," the authors wrote.
SOURCE:
This study was led by Rachele Spagnol, University of Padua, Padua, Italy, and was published online on May 08, 2025, in European Journal of Pediatrics .
LIMITATIONS:
The included studies were retrospective in nature, which may have introduced potential biases. They also had small sample sizes and short follow-up durations, which limited the ability to assess long-term neurologic outcomes. Additionally, the heterogeneity in diagnostic criteria and neurologic outcome assessment across studies may have affected result comparability.
DISCLOSURES:
This study received support through the Research4Residents project within the Residency Program in Pediatrics at the University of Padua, Italy. Open access funding was provided by Università degli Studi di Padova within the CRUI-CARE Agreement. The authors declared no competing interests.
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