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. 2017 Mar 1;32(3):466-474.
doi: 10.1093/ndt/gfw453.

Eculizumab in secondary atypical haemolytic uraemic syndrome

Teresa Cavero  1 , Cristina Rabasco  2 , Antía López  3 , Elena Román  4 , Ana Ávila  5 , Ángel Sevillano  1 , Ana Huerta  6 , Jorge Rojas-Rivera  7 , Carolina Fuentes  8 , Miquel Blasco  9 , Ana Jarque  10 , Alba García  3 , Santiago Mendizabal  4 , Eva Gavela  5 , Manuel Macía  10 , Luis F Quintana  9 , Ana María Romera  11 , Josefa Borrego  12 , Emi Arjona  13 , Mario Espinosa  2 , José Portolés  6 , Carolina Gracia-Iguacel  7 , Emilio González-Parra  7 , Pedro Aljama  2 , Enrique Morales  1 , Mercedes Cao  3 , Santiago Rodríguez de Córdoba  13 , Manuel Praga  1   4
Affiliations

Eculizumab in secondary atypical haemolytic uraemic syndrome

Teresa Cavero et al. Nephrol Dial Transplant. .

Abstract

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 × 10 9 /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.

Keywords: atypical haemolytic uraemic syndrome; complement activation; eculizumab; thrombotic microangiopathies.

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Figures

FIGURE 1
FIGURE 1
Changes in (A) serum creatinine, (B) haemoglobin and (C) platelet count.
See this image and copyright information in PMC

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