To the editors,
Ravulizumab, a long-acting C5-inhibitor, has been shown to be efficacious and safe in clinical studies in pediatric patients with preceding eculizumab treatment [1] and in therapy-naïve pediatric and adult patients [2, 3] with atypical hemolytic uremic syndrome (aHUS).
We present here the first real-world data of six pediatric patients with genetically proven aHUS switched to ravulizumab after a median time of 69 (range 6–123) months of eculizumab treatment. Four patients were diagnosed with a complement factor H (CFH) mutation, one patient with a complement factor 3 (C3) mutation, and one with DEAP-HUS (compare Supplementary Table 1 for detailed patient characteristics). Hematological and renal parameters remained stable as shown exemplarily for kidney function and lactate dehydrogenase (LDH) (Supplementary Figure 1). Comprehensive complement surveillance showed stable AP50 suppression and suppressed sC5b-9 levels 3 months after therapy switch as compared to before (Supplementary Figure 1). None of the patients reported any side effects of ravulizumab treatment in the current investigation interval of a median of 220 (range 90–274) days. Importantly, all patients reported a subjective benefit in quality of life due to the extended dosing interval.
Our data support the conclusion of Tanaka et al. [1] and add six definitely diagnosed patients with aHUS to the existing evidence that ravulizumab is effective and safe in pediatric patients. Thus, our data are a significant contribution to the growing body of evidence. In addition, we were able to show that the switch to ravulizumab is feasible in a real-life setting.
Supplementary Information
(PDF 668 kb)
(PDF 561 kb)
Author contribution
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Funding
Open Access funding enabled and organized by Projekt DEAL.
Data Availability
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Declarations
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Conflict of interest
Rasmus Ehren has no conflict of interest. Sandra Habbig has received honoraria for lecturing from Sanofi-Aventis Deutschland GmbH and honoraria for participation on Advisory Boards of Alexion Pharma GmbH and Greenovation Biotech GmbH.
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References
- 1.Tanaka K, Adams B, Aris AM, Fujita N, Ogawa M, Ortiz S, Vallee M, Greenbaum LA. The long-acting C5 inhibitor, ravulizumab, is efficacious and safe in pediatric patients with atypical hemolytic uremic syndrome previously treated with eculizumab. Pediatr Nephrol. 2021;36:889–898. doi: 10.1007/s00467-020-04774-2. [DOI] [PMC free article] [PubMed] [Google Scholar]
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- 3.Rondeau E, Scully M, Ariceta G, Barbour T, Cataland S, Heyne N, Miyakawa Y, Ortiz S, Swenson E, Vallee M, Yoon S-S, Kavanagh D, Haller H, 311 Study Group The long-acting C5 inhibitor, Ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment. Kidney Int. 2020;97:1287–1296. doi: 10.1016/j.kint.2020.01.035. [DOI] [PubMed] [Google Scholar]
Associated Data
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Supplementary Materials
(PDF 668 kb)
(PDF 561 kb)
Data Availability Statement
Not applicable.
Code availability
Not applicable.