Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Multicenter Study
. 2024 Jun 19;25(1):202.
doi: 10.1186/s12882-024-03638-3.

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

Kristina Schönfelder  1 , Lucas Kühne  2 , Lena Schulte-Kemna  3 , Jessica Kaufeld  4 , Hana Rohn  5 , Andreas Kribben  1 , Bernd Schröppel  3 , Paul T Brinkkötter  2 , Anja Gäckler  6   7
Affiliations
Multicenter Study

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

Kristina Schönfelder et al. BMC Nephrol. .

Abstract

Background: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

Keywords: Atypical hemolytic uremic syndrome; Eculizumab; Kidney transplant recipients; Ravulizumab; Thrombotic microangiopathy.

PubMed Disclaimer

Conflict of interest statement

K.S. and H.R. declare no competing interests. L.K. has received consulting fees from Alexion and research funding from Sanofi-Genzyme. L.S.K. reports consultant honoraria and travel grants from Alexion. J.K. reports speaker honoraria and participation in advisory boards from Alexion, Sanofi, and Chiesi. A.K. has received grants for clinical studies, speaker fees, honoraria and travel expenses from Actelion, Amgen, Amicus, Alexion, Astellas, Bayer, Baxter, Binding Site, Bristol-Myers Squibb, Chiesi, CytoSorbents, Fresenius, GlaxoSmithKline, Hexal, Janssen, Kyowa Kirin, MSD, Novartis, Otsuka, Peripal, Pfizer, Roche, Sanofi, Shire, Teva and Vifor Fresenius Medical. B.S. has received consultant/speaker honoraria from Alexion, Amgen, Novartis, Astellas, Bristol Myers Squibb, Boehringer Ingelheim, Bayer, Vifor Pharma, Astra Zeneca, Janssen-Cilag, Pfizer, GlaxoSmithKlein, Lilly. P.T.B. has received speaker honoraria and consultant fees from Sanofi-Genzyme, AstraZeneca, Alexion, Bayer, Travere, Pfizer, Novartis, Roche, Boehringer Ingelheim, and participated in advisory boards for Alexion, Sanofi-Genzyme, Novartis, Travere, and Bayer. A.G. has received speaker/consultant honoraria and study fees from Alexion/Astra Zeneca, Boehringer Ingelheim, Chiesi, Novartis, Sanofi, Roche and Vifor Pharma.

Figures

Fig. 1
Fig. 1
Platelets and hemoglobin. Platelets (A-C) and hemoglobin (D-F) approx. 3 months before and up to approx. 12 months after switch from eculizumab to ravulizumab. A + D all patients; B + E non-transplanted patients and renal transplant recipients; C + F patients with diagnosis of aHUS > 6 months and < 6 months before switch of medication. * p < 0.05 vs. approx. 3 months before switching to ravulizumab
Fig. 2
Fig. 2
LDH and haptoglobin. LDH (A-C) and haptoglobin (D-F) approx. 3 months before and up to approx. 12 months after switch from eculizumab to ravulizumab. A + D all patients; B + E non-transplanted patients and renal transplant recipients; C + F patients with diagnosis of aHUS > 6 months and < 6 months before switch of medication
Fig. 3
Fig. 3
Serum creatinine. Serum creatinine approx. 3 months before and up to approx. 12 months after switch from eculizumab to ravulizumab. A all patients; B non-transplanted patients and renal transplant recipients; C patients with diagnosis of aHUS > 6 months and < 6 months before switch of medication
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Laurence J, Haller HM, Mannucci PM, Nangaku M, Praga M. Rodriguez de Córdoba S: Atypical hemolytic uremic syndrome (aHUS): essential aspects of an acute diagnosis. Clin Adv Hematol Oncol. 2016;14 Suppl 11(11):2–15. - PubMed
    1. Fremeaux-Bacchi V, Fakhouri F, Garnier A, Bienaimé F, Dragon-Durey M, Ngo S, Moulin B, Servais A, Provot F, Rostaing L, et al. Genetics and outcome of atypical hemolytic uremic syndrome: a nationwide French series comparing children and adults. Clin J Am Soc Nephrol. 2013;8(4):554–562. doi: 10.2215/CJN.04760512. - DOI - PMC - PubMed
    1. Le Quintrec M, Zuber J, Moulin B, Kamar N, Jablonski M, Lionet A, Chatelet V, Mousson C, Mourad G, Bridoux F, et al. Complement genes strongly predicht recurrence and graft outcome in adult renal transplant recipients with atypical hemolytic and uremic syndrome. Am J Transplant. 2013;13(3):663–675. doi: 10.1111/ajt.12077. - DOI - PubMed
    1. Fakhouri F, Hourmant M, Campistol J, Cataland S, Espinosa M, Gaber A, Menne J, Minetti E, Provot F, Rondeau E, et al. Terminal complement inhibitor eculizumab in adult patients with atypical hemolytic uremic syndrome: a single-arm, open-label trial. Am J Kidney Dis. 2016;68(1):84–93. doi: 10.1053/j.ajkd.2015.12.034. - DOI - PubMed
    1. Zuber J, Frimat M, Caillard S, Kamar N, Gatault P, Petiprez F, Couzi L, Jourde-Chiche N, Chatelet V, Gaisne R, et al. Use of higly individualized complement blockade has revolutionized clinical outcomes after kidney transplantation and renal epidemiology of atypical hemolytic uremic syndrome. J Am Soc Nephrol. 2019;30(12):2449–2463. doi: 10.1681/ASN.2019040331. - DOI - PMC - PubMed

Publication types

LinkOut - more resources