Eculizumab Is Safe and Effective as a Long-term Treatment for Protein-losing Enteropathy Due to CD55 Deficiency

@article{Kurolap2019EculizumabIS,
  title={Eculizumab Is Safe and Effective as a Long-term Treatment for Protein-losing Enteropathy Due to CD55 Deficiency},
  author={Alina Kurolap and Orly Eshach Adiv and Tova Hershkovitz and Adi Tabib and Netanel Karbian and Tamar Paperna and Adi Mory and Arcadi Vachyan and Nadav Slijper and Ran Steinberg and Yaniv Zohar and Dror Mevorach and Hagit Baris Feldman},
  journal={Journal of Pediatric Gastroenterology and Nutrition},
  year={2019},
  volume={68},
  pages={325–333}
}
Objectives: Loss of the complement inhibitor CD55 leads to a syndrome of early-onset protein-losing enteropathy (PLE), associated with intestinal lymphangiectasia and susceptibility to large-vein thrombosis. The in vitro and short-term treatment benefits of eculizumab (C5-inhibitor) therapy for CD55-deficiency have been previously demonstrated. Here we present the 18-months treatment outcomes for 3 CD55-deficiency patients with sustained therapeutic response. Methods: Three CD55-deficiency… 
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