Eculizumab as first-line treatment for patients with severe presentation of complement factor H antibody–mediated hemolytic uremic syndrome.
Background: Complement factor H (FH) antibody–mediated hemolytic uremic syndrome (HUS) has varying prevalence globally. Plasmapheresis and immunosuppressive drugs are the standard treatment. Recently, eculizumab has been reported as an effective alternative. This study aims to report four children w...
| Published in: | Pediatric Nephrology Vol. 40; no. 4; pp. 1041 - 1048 |
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| Main Authors: | , , , , , , , , , |
| Format: | case study research tables/charts Journal Article |
| Published: |
Springer Nature
Apr2025
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| Online Access: | View this record in EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=183485106&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 183485106 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 0931041X EF1 jtl: Pediatric Nephrology issn: 0931041X maglogo: N pubinfo: dt: Apr2025 vid: 40 iid: 4 pid: 237 pub: Springer Nature place: New York, New York artinfo: ui: 183485106 180147342 183485106 183485106 10.1007/s00467-024-06530-2 183485106 ppf: 1041 ppct: 7 formats: fmt: – @attributes: type: T – @attributes: type: P tig: atl: Eculizumab as first-line treatment for patients with severe presentation of complement factor H antibody–mediated hemolytic uremic syndrome. aug: au: Coccia, Paula A. Alconcher, Laura F. Ferraris, Veronica Lucarelli, Lucas I. Grillo, Maria A. Arias, Maria Andrea Saurit, Mariana Ratto, Viviana M. dos Santos, Celia Sánchez-Luceros, Analía affil: https://ror.org/00bq4rw46 Division of Pediatric Nephrology, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina sug: subj: Complement Immunology Atypical Hemolytic Uremic Syndrome Immunology Atypical Hemolytic Uremic Syndrome Drug Therapy Severity of Illness Antibodies, Monoclonal Therapeutic Use Immunosuppression Drug Efficacy Patient Safety Human Male Female Immunosuppressive Agents Therapeutic Use Infant, Newborn Infant Child, Preschool Child Retrospective Design Record Review Autoantibodies Prednisone Therapeutic Use Mycophenolate Mofetil Therapeutic Use Treatment Duration Drug Tapering Infant, Newborn: birth-1 month Infant: 1-23 months Child, Preschool: 2-5 years Child: 6-12 years Male Female ab: Background: Complement factor H (FH) antibody–mediated hemolytic uremic syndrome (HUS) has varying prevalence globally. Plasmapheresis and immunosuppressive drugs are the standard treatment. Recently, eculizumab has been reported as an effective alternative. This study aims to report four children with FH antibody–mediated HUS managed with eculizumab plus immunosuppression as first-line therapy. Methods: A retrospective chart review was conducted for children aged ≤ 18 years old with complement-mediated HUS in two referral centers. Patients with FH antibody–mediated HUS treated with eculizumab as first-line therapy were included. Results: Four children (aged 6–11 years old) were included. Dialysis was necessary in three patients. Eculizumab was administered 5–23 days after onset. None of them received plasmapheresis. Prednisone and mycophenolate mofetil were added after receiving positive FH antibody results. Hematological signs and kidney function improved after the second eculizumab dose. Eculizumab was discontinued in three patients after 6 months. One patient required rituximab due to persistent high FH antibody titers; discontinuation of eculizumab occurred after 15 months without recurrence. No treatment-related complications were observed. During a mean 12-month follow-up (range 6–24 months), no relapses were recorded and all patients ended with normal GFR. Conclusion: Our data suggest that a short course of 6 months of C5 inhibitor might be sufficient to reverse thrombotic microangiopathy symptoms and improve kidney function in patients with severe FH antibody–mediated HUS. Simultaneously, adding immunosuppressive agents might reduce the risk of relapse and allow cessation of C5 inhibition in a shorter period of time. pubtype: Academic Journal doctype: case study research tables/charts Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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