Le Quintrec M, et al. - In this trial, researchers attempted to examine the efficacy of eculizumab in pediatric and adult patients with C3 glomerulopathy. Data illustrated that eculizumab served as a potential treatment for patients with crescentic rapidly progressive C3 glomerulopathy. Its benefit appeared to be limited in patients with non–rapidly progressing forms.

Methods

• The design of this research was a case series study of C3 glomerulopathy.
• Experts enrolled pediatric and adult patients with C3 glomerulopathy treated with eculizumab between 2010 and 2016, identified via the C3 glomerulopathy French registry database.
• During this study, a questionnaire was sent to participating French pediatric and adult nephrology centers, as well as one pediatric referral center in Quebec, Canada.
• Global or partial clinical renal response were included as the outcomes.
• Measurements consisted of evolution of serum creatinine and proteinuria values.

Results

• Researchers analyzed 26 patients (13 children/adolescents).
• Among the study participants, 22 (85%) patients had received steroids, plasma exchange, or immunosuppressive therapy before eculizumab, and 3 of them presented with rapid progression of their kidney disease despite treatment.
• A total of 11 (42%) patients had chronic kidney disease, 7 (27%) had rapidly progressive disease, and 3 (12%) required dialysis, at the initiation of eculizumab therapy.
• Findings revealed that 6 (23%) patients had a global clinical response; 6 (23%), a partial clinical response; and 14 (54%), no response, after eculizumab treatment (median duration, 14 months).
• A lower estimated glomerular filtration rates, a more rapidly progressive course and more extracapillary proliferation was discovered on kidney biopsy in patients who had a global clinical response vs patients who had a partial clinical or no response.
• Between the responders and nonresponders, age, extent of renal fibrosis, frequency of nephrotic syndrome, low serum C3 and C3 nephritic factor and elevated soluble C5b-9 concentrations or complement gene variants did not exhibit any difference.