Eculizumab as an adjunctive rescue treatment for life-threatening childhood onset of systemic lupus erythematosus

Childhood-onset systemic lupus erythematosus (cSLE) often presents with aggressive manifestations that may not respond to standard immunosuppressive therapy. We describe two paediatric cSLE cases with severe life-threatening complications-autoimmune hemolytic anemia in one patient and macrophage activation syndrome with acute respiratory distress syndrome in the other-successfully managed with Eculizumab as adjunctive treatment. Both patients demonstrated rapid stabilization after Eculizumab initiation and showed favorable outcomes at their latest follow-up. These cases highlight the potential role of complement inhibition in select cSLE complications and suggest that Eculizumab may serve as a rescue therapy beyond its established use in thrombotic microangiopathy. Further studies are needed to better define its optimal use and target patient population.

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Publication Details

Journal
Lupus
Published
2025-11-24
DOI
https://doi.org/10.1177/09612033251399945
Primary Topic
Complement system in diseases
Type
article
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article

Eculizumab as an adjunctive rescue treatment for life-threatening childhood onset of systemic lupus erythematosus

Teresa Giani, Gabriele Simonini, Ilaria Pagnini, Ilaria Maccora et al.
Lupus
Complement system in diseases
article

Eculizumab as an adjunctive rescue treatment for life-threatening childhood onset of systemic lupus erythematosus

Teresa Giani, Gabriele Simonini, Ilaria Pagnini, Ilaria Maccora, Maria Vincenza Mastrolia, Edoardo Marrani, Antonio Tiezzi, Linda Zollo
article en

Abstract

Childhood-onset systemic lupus erythematosus (cSLE) often presents with aggressive manifestations that may not respond to standard immunosuppressive therapy. We describe two paediatric cSLE cases with severe life-threatening complications-autoimmune hemolytic anemia in one patient and macrophage activation syndrome with acute respiratory distress syndrome in the other-successfully managed with Eculizumab as adjunctive treatment. Both patients demonstrated rapid stabilization after Eculizumab initiation and showed favorable outcomes at their latest follow-up. These cases highlight the potential role of complement inhibition in select cSLE complications and suggest that Eculizumab may serve as a rescue therapy beyond its established use in thrombotic microangiopathy. Further studies are needed to better define its optimal use and target patient population.

LupusVol. 35(1)
Meyer Children's Hospital (IL), Meyer Children's Hospital (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), University of Florence (IT)
Openalex Percentile: Top 59%
Complement system in diseases
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Eculizumab as an adjunctive rescue treatment for life-threatening childhood onset of systemic lupus erythematosus — Teresa Giani, Gabriele Simonini, et al. · Lupus (2025) | TGRS Research Map | TGRS