Clinical efficacy and safety of switching from eculizumab to ravulizumab in adult patients with aHUS- real-world data

• Published in: BMC nephrology 2024-06, Vol.25 (1), p.202-8, Article 202
• Main Authors: Schönfelder, Kristina, Kühne, Lucas, Schulte-Kemna, Lena, Kaufeld, Jessica, Rohn, Hana, Kribben, Andreas, Schröppel, Bernd, Brinkkötter, Paul T, Gäckler, Anja
• Format: Article
• Language: English
• Subjects: Adult; Adults; Aged; Antibodies, Monoclonal, Humanized - therapeutic use; Atypical hemolytic uremic syndrome; Atypical Hemolytic Uremic Syndrome - drug therapy; Cardiovascular disease; Care and treatment; Complement component C5; Complement Inactivating Agents - therapeutic use; Creatinine; Drug Substitution; Drug therapy; Eculizumab; Female; Genetic analysis; Genetic testing; Hematology; Hemoglobin; Hemolytic uremic syndrome; Humans; Information management; Kidney diseases; Kidney transplant recipients; Kidney transplantation; Kidney transplants; Kidneys; Male; Middle Aged; Mutation; Organ transplant recipients; Patient outcomes; Patients; Quality of life; Ravulizumab; Renal function; Renal replacement therapy; Retrospective Studies; Thrombotic microangiopathy; Transplantation; Treatment Outcome; Young Adult
• ISSN: 1471-2369; 1471-2369
• DOI: 10.1186/s12882-024-03638-3

The complement factor 5 (C5)-inhibitor eculizumab has been established as standard-of-care for the treatment of atypical hemolytic uremic syndrome (aHUS). In 2021, the long-acting C5-inhibitor ravulizumab was approved, extending intervals of intravenous treatment from two to eight weeks resulting in improvement of quality of life for patients and lowering direct and indirect therapy associated costs. This multicenter, retrospective data analysis of 32 adult patients with aHUS (including 10 kidney transplant recipients) treated with eculizumab for at least three months and switched to ravulizumab aims to evaluate the safety and efficacy of switching medication in the real-world setting. Hematologic parameters, kidney function, concurrent therapy and aHUS associated events were evaluated three months before and until up to 12 months after switching to ravulizumab. Mean age (range) at ravulizumab initiation was 41 years (19-78 years) and 59% of the patients were female. Genetic analysis was available for all patients with 72% showing a pathogenic variant. Median time (range) on eculizumab before switching was 20 months (3-120 months). No new events of TMA or worsening of renal function were reported during up to 12 months of follow-up during ravulizumab treatment. This is the largest, non-industry derived, multi-center retrospective analysis of adult patients with aHUS switching C5-inhibitor treatment from eculizumab to ravulizumab in the real-world setting. Switching to ravulizumab was safe and efficient resulting in sustained hematological stability and preservation of renal function.