Eculizumab in secondary atypical haemolytic uraemic syndrome
AuthorCavero, Teresa; Rabasco, Cristina; López, Antía; Román, Elena; Ávila, Ana; Sevillano, Ángel; Huerta, Ana; Rojas-Rivera, Jorge; Fuentes, Carolina; Blasco, Miquel; Jarque, Ana; García, Alba; Mendizabal, Santiago; Gavela, Eva; Macía, Manuel; Quintana, Luis F.; Romera, Ana María; Borrego, Josefa; Arjona, Emi; Espinosa, Mario; Portolés Pérez, José María; Gracia-Iguacel, Carolina; González-Parra, Emilio; Aljama, Pedro; Morales, Enrique; Cao, Mercedes; Rodríguez de Córdoba, Santiago; Praga, Manuel
EntityUAM. Departamento de Medicina; Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD)
PublisherOxford University Press on behalf of ERA-EDTA.
10.1093/ndt/gfw453Nephrology Dialysis Transplantation 32.3 (2017): 466-474
ISSN0931-050 (print); 1460-2385 (online)
Funded byWork in this report was funded by the Instituto de Salud Carlos III: REDinREN (RD 016/009 Feder Funds), the Fondo de Investigaciones Sanitarias (13/02502 and ICI14/00350), the Ministerio de Economia y Competitividad (SAF2015-66287R) and the Autonomous Region of Madrid (S2010/BMD-2316; Grupo de Investigación Complemento-CM). SRdeC is funded by the Seventh Framework Programme European Union Project EURenOmics (305608)
ProjectGobierno de España. RD 016/009; Gobierno de España. ICI14/00350; Gobierno de España. SAF2015-66287R; Comunidad de Madrid. S2010/BMD-2316/COMPLEMENTO; info:eu-repo/grant/Agreement/EC/FP7/305608; Gobierno de España.13/02502
SubjectsAtypical haemolytic uraemic syndrome; Complement activation; Eculizumab; Thrombotic microangiopathies; Medicina
Rights© The Author 2017
Esta obra está bajo una licencia de Creative Commons Reconocimiento-NoComercial 4.0 Internacional.
Background. Complement dysregulation occurs in thrombotic microangiopathies (TMAs) other than primary atypical haemolytic uraemic syndrome (aHUS). A few of these patients have been reported previously to be successfully treated with eculizumab. Methods. We identified 29 patients with so-called secondary aHUS who had received eculizumab at 11 Spanish nephrology centres. Primary outcome was TMA resolution, defined by a normalization of platelet count (>150 × 109/L) and haemoglobin, disappearance of all the markers of microangiopathic haemolytic anaemia (MAHA), and improvement of renal function, with a ≥25% reduction of serum creatinine from the onset of eculizumab administration. Results. Twenty-nine patients with secondary aHUS (15 drug-induced, 8 associated with systemic diseases, 2 with postpartum, 2 with cancer-related, 1 associated with acute humoral rejection and 1 with intestinal lymphangiectasia) were included in this study. The reason to initiate eculizumab treatment was worsening of renal function and persistence of TMA despite treatment of the TMA cause and plasmapheresis. All patients showed severe MAHA and renal function impairment (14 requiring dialysis) prior to eculizumab treatment and 11 presented severe extrarenal manifestations. A rapid resolution of the TMA was observed in 20 patients (68%), 15 of them showing a ≥50% serum creatinine reduction at the last follow-up. Comprehensive genetic and molecular studies in 22 patients identified complement pathogenic variants in only 2 patients. With these two exceptions, eculizumab was discontinued, after a median of 8 weeks of treatment, without the occurrence of aHUS relapses. Conclusion. Short treatment with eculizumab can result in a rapid improvement of patients with secondary aHUS in whom TMA has persisted and renal function worsened despite treatment of the TMA-inducing condition
Google Scholar:Cavero, Teresa - Rabasco, Cristina - López, Antía - Román, Elena - Ávila, Ana - Sevillano, Ángel - Huerta, Ana - Rojas-Rivera, Jorge - Fuentes, Carolina - Blasco, Miquel - Jarque, Ana - García, Alba - Mendizabal, Santiago - Gavela, Eva - Macía, Manuel - Quintana, Luis F. - Romera, Ana María - Borrego, Josefa - Arjona, Emi - Espinosa, Mario - Portolés Pérez, José María - Gracia-Iguacel, Carolina - González-Parra, Emilio - Aljama, Pedro - Morales, Enrique - Cao, Mercedes - Rodríguez de Córdoba, Santiago - Praga, Manuel
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