Anti-CFH-associated hemolytic uremic syndrome: do we still need plasma exchange?

Autor: Ferri M; Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France., Zotta F; Division of Nephrology, Laboratory of Nephrology, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy., Donadelli R; Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Ranica, Bergamo, Italy., Dossier C; Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France., Duneton C; Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France., El-Sissy C; Assistance Publique-Hôpitaux de Paris, Department of Biological Immunology, Hôpital Européen Georges Pompidou, and INSERM UMR S1138, Complément et Maladies, Centre de Recherche des Cordeliers, Paris, France., Fremeau-Bacchi V; Assistance Publique-Hôpitaux de Paris, Department of Biological Immunology, Hôpital Européen Georges Pompidou, and INSERM UMR S1138, Complément et Maladies, Centre de Recherche des Cordeliers, Paris, France., Kwon T; Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France., Quadri L; Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Ranica, Bergamo, Italy., Pasini A; Paediatric Clinic, IRCCS Ospedale Maggiore Policlinico Sant'Orsola, Department of Medicine and Surgery, University of Bologna, Bologna, Italy., Sellier-Leclerc AL; Department of Pediatric Nephrology, Centre de Référence des Maladies Rénales Rares Néphrogones, Filières Maladies Rares ORKID et ERK-Net, CHU de Lyon, Bron, France., Vivarelli M; Division of Nephrology, Laboratory of Nephrology, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy., Hogan J; Pediatric Nephrology Department, Centre de référence des Maladies Rénales Rares MARHEA, Filières Maladies Rares ORKID et ERK-Net, Robert Debré Hospital, APHP, 48 bd Sérurier, 75019, Paris, France. Julien.hogan2@aphp.fr.; INSERM, UMR-U970, PARCC, Paris Institute for Transplantation and Organ Regeneration, Université Paris Cité, Paris, France. Julien.hogan2@aphp.fr.
Jazyk: angličtina
Zdroj: Pediatric nephrology (Berlin, Germany) [Pediatr Nephrol] 2024 Nov; Vol. 39 (11), pp. 3263-3269. Date of Electronic Publication: 2024 Apr 17.
DOI: 10.1007/s00467-024-06373-x
Abstrakt: Background: Between 5 and 50% of atypical hemolytic uremic syndrome (aHUS) cases in children are caused by autoantibodies against complement factor H (CFH). Given the acquired autoimmune nature of the disease, plasma exchange (PE) and various immunosuppressive treatments have been used. More recently, eculizumab has been proposed.
Methods: In this multicenter, retrospective study, we report outcomes of 12 children with anti-FH antibody-associated HUS treated with eculizumab associated with various immunosuppressive regimens.
Results: Patients were treated with eculizumab for 15.5 [9.5;23.0] months and 3 received PE or IgG adsorption. Three patients received mycophenolate mofetil (MMF) alone, 1 patient received MMF and steroids, 1 patient received MMF and rituximab, 3 patients received MMF/steroids and rituximab, and 4 patients did not receive any immunosuppression. Anti-FH antibody levels significantly decreased but no difference was observed based on the immunosuppressive regimen. Eculizumab was discontinued in 7/10 patients after 11 [7.5;15.5] months and MMF in 6/8 patients after 36 [35;40] months. Anti-FH titers at MMF discontinuation ranged from 257 to 3425 UI/L. None of these patients relapsed and eGFR at last follow-up was above 70 mL/min/1.73 m 2 in all patients.
Conclusions: Eculizumab is effective and safe in inducing and maintaining remission in aHUS secondary to anti-FH antibodies and renders reduction of anti-FH titers less urgent. Anti-FH antibody titers decreased in most patients irrespective of the immunosuppressive treatment chosen, so that a strategy consisting of combining eculizumab with MMF monotherapy seems sufficient at least in non-Indian or less severe forms of anti-FH antibody-associated HUS.
(© 2024. The Author(s), under exclusive licence to International Pediatric Nephrology Association.)
Databáze: MEDLINE
Externí odkaz: