Back to Search
Start Over
Ravulizumab in Atypical Hemolytic Uremic Syndrome: An Analysis of 2-Year Efficacy and Safety Outcomes in 2 Phase 3 Trials.
- Source :
-
Kidney medicine [Kidney Med] 2024 Jun 14; Vol. 6 (8), pp. 100855. Date of Electronic Publication: 2024 Jun 14 (Print Publication: 2024). - Publication Year :
- 2024
-
Abstract
- Rationale & Objective: Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) caused by complement dysregulation. Ravulizumab is a C5i approved for the treatment of aHUS. This analysis assessed long-term outcomes of ravulizumab in adults and pediatric patients with aHUS.<br />Study Design: This analysis reports 2-year data from 2 phase 3, single-arm studies.<br />Setting & Participants: One study included C5i-naïve adults (NCT02949128), and the other included 2 cohorts of pediatric patients (C5i-naïve and those who switched to ravulizumab from eculizumab [pediatric switch patients]; NCT03131219).<br />Exposure: Patients received intravenous ravulizumab every 4-8 weeks, with the dose depending on body weight.<br />Outcomes: The primary endpoint in the studies of C5i-naïve patients was complete TMA response, which consisted of platelet count normalization, lactate dehydrogenase normalization, and ≥25% improvement in serum creatinine concentrations from baseline, at 2 consecutive assessments ≥4 weeks apart.<br />Analytical Approach: All analyses used descriptive statistics. No formal statistical comparisons were performed.<br />Results: In total, 86 and 92 patients were included in efficacy and safety analyses, respectively. Complete TMA response rates over 2 years were 61% and 90% in C5i-naïve adults and pediatric patients, respectively. The median increase in estimated glomerular filtration rate from baseline was maintained over 2 years in C5i-naïve adults (35 mL/min/1.73 m <superscript>2</superscript> ) and pediatric patients (82.5 mL/min/1.73 m <superscript>2</superscript> ). Most adverse events and serious adverse events occurred during the first 26 weeks. No meningococcal infections were reported. Improvement in the Functional Assessment of Chronic Illness Therapy - Fatigue score achieved by 26 weeks was maintained over 2 years.<br />Limitations: Limitations were the small sample of pediatric switch patients and limited availability of genetic data.<br />Conclusions: Long-term treatment with ravulizumab is well tolerated and associated with improved hematologic and renal parameters and quality of life in adults and pediatric patients with aHUS.<br /> (© 2024 The Authors.)
Details
- Language :
- English
- ISSN :
- 2590-0595
- Volume :
- 6
- Issue :
- 8
- Database :
- MEDLINE
- Journal :
- Kidney medicine
- Publication Type :
- Academic Journal
- Accession number :
- 39105067
- Full Text :
- https://doi.org/10.1016/j.xkme.2024.100855