Eculizumab: another breakthrough

Eculizumab: another breakthrough.

PNH is a clonal hematopoietic stem cell disease associated with a complementdependent hemolytic anemia. The disease manifests after the expansion of a multipotent hematopoietic stem cell that acquires a mutation of the PIGA gene. The PIGA gene product is required for the biosynthesis of glycophosphatidylinositol anchors, a glycolipid moiety that attaches dozens of proteins to the plasma membran...

Another horizon and breakthrough for the Sudanese Journal of Paediatrics.

Successful pregnancy outcome in paroxysmal nocturnal hemoglobinuria (PNH) following escalated eculizumab dosing to control breakthrough hemolysis

Pregnancy in women with paroxysmal nocturnal hemoglobinuria (PNH) is associated with increased maternal and fetal morbidity and mortality. There is limited published experience regarding therapy of PNH during pregnancy. We describe a case of a 30 year old female with hypoplastic myelodysplastic syndrome and PNH. After two years of treatment with eculizumab, she became pregnant. She developed br...

Treating C3 glomerulopathy with eculizumab

BACKGROUND C3 glomerulopathy (C3G) is a rare, but severe glomerular disease with grim prognosis. The complex pathogenesis is just unfolding, and involves acquired as well as inherited dysregulation of the alternative pathway of the complement cascade. Currently, there is no established therapy. Treatment with the C5 complement inhibitor eculizumab may be a therapeutic option. However, due to ra...

Paroxysmal nocturnal hemoglobinuria and eculizumab.