Efficacy and safety of current treatments for paroxysmal nocturnal hemoglobinuria: A systematic review

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a non-malignant clonal disorder of the pluripotent hematopoietic stem cell. Currently, Eculizumab, Ravulizumab, and Pegcetacoplan are the approved drugs to treat PNH. In order to assess the efficacy and safety of different medications available for PNH, we performed a systematic search. The primary efficacy endpoint was the percentage change in lactate dehydrogenase, transfusion avoidance, and stabilized hemoglobin. Key secondary endpoints included the proportion of patients with breakthrough hemolysis, anemia, adverse events, number of deaths, and discontinuation of treatment. From 2526 articles retrieved from electronic databases and manual searches, a total of five studies were included in this review: 1 observational study and 4 randomized clinical trials. For all efficacy and safety endpoints, Ravulizumab and Pegcetacoplan achieved noninferiority compared with the first standardized treatment Eculizumab. The use of complement inhibition therapy can further improve hematological outcomes in PNH patients.

Keywords

• Paroxysmal nocturnal hemoglobinuria
• Eculizumab
• Ravulizumab
• Pegcetacoplan