Short communication, Note

Eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria

Zinaida Perić ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska
Klara Dubravčić ; Zavod za imunologiju, Klinički zavod za laboratorijsku dijagnostiku, KBC Zagreb, Zagreb, Hrvatska
Sandra Bašić-Kinda ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska
Nadira Duraković ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska
Ana Boban ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska
Mirta Mikulić ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska
Drago Batinić ; Zavod za imunologiju, Klinički zavod za laboratorijsku dijagnostiku, KBC Zagreb, Zagreb, Hrvatska
Boris Labar ; Zavod za hematologiju, Klinika za unutarnje bolesti, KBC Zagreb, Zagreb, Hrvatska

Abstract

Aim: A case of a patient with paroxysmal nocturnal hemoglobinuria (PNH) treated with exulicumab, monoclonal antibody which blocks the complement is presented. Case report: A dignosis of PNH was established in a young female patient with a previous history of aplastic anemia, after a thrombotic event in the CNS. Hemolysis started one year later and markedly progressed until the beginning of eculizumab treatment, when she had severe hemolysis, thrombocytopenia and anemia with need for blood transfusions every 2-3 weeks and sceondary hemochromatosis. In January 2010, treatment with eculizumab was started. She received induction treatment without severe side-effects and continued with maintenance treatment for over 9 months. A significant decrease in hemolysis was observed which lead to diseappearance of symptoms and consequently the reduced need for transfusions. All this lead to a dramatic improvement of her quality of life. Discussion and conclusion: Until recently, allogenic stem cell transplantation (allo-SCT), a therapeutic procedure linked with significant morbidity and mortality, was the only treatment for PNH. Two prospective studies have showed that eculizumab decreases intravascular hemolysis and the need for red cell transfusions, improves quality of life, and markedly decreases the risk for thrombosis. We confirmed these findings in our patient. However allo-SCT remains the only curative treatment of PNH and should still be considered as the preferred therapeutic option in young patients with an underlying bone marrow disorder.

Keywords

allo-SCT; eculizumab; PNH; treatment

Hrčak ID:

76676

URI

https://hrcak.srce.hr/76676

Publication date:

1.12.2011.

Article data in other languages: croatian

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