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Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: A systematic review on efficacy and safety
Journal article   Open access   Peer reviewed

Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: A systematic review on efficacy and safety

Sangam Shah, Rajan Chamlagain, Ziyaul Haq Musalman, Yagya Raj Adhikari, Santosh Chhetri, Sujan Paudel, Krishna Gundabolu and Prajwal Dhakal
Research and practice in thrombosis and haemostasis, Vol.6(5), pp.e12781-n/a
07/01/2022
DOI: 10.1002/rth2.12781
PMCID: PMC9357886
PMID: 35949886
url
https://doi.org/10.1002/rth2.12781View
Published (Version of record) Open Access

Abstract

Introduction Pegcetacoplan, a pegylated penta-decapeptide, targets complement C3 to control both intravascular and extravascular hemolysis. This systematic review aims to study the efficacy and safety of pegcetacoplan in paroxysmal nocturnal hemoglobinuria (PNH). Methods We performed a comprehensive and systematic literature search for all studies on PubMed, Google Scholar, Cochrane Library, and . The studies were searched using keywords "paroxysmal nocturnal hemoglobinuria" or "PNH," "Pegcetacoplan" or "Empaveli." The primary outcomes included change in hemoglobin level, transfusion independence, absolute reticulocyte count, and lactate dehydrogenase (LDH) level after pegcetacoplan therapy. The safety outcomes included the proportion of deaths and adverse effects. Results We included a total of three studies. The total number of patients with PNH was112. 59.83% were female. In the PADDOCK study and study by Hillmen et al., the average increase in hemoglobin was 3.68 g/L and 2.37 g/L, respectively. In the study by de Castro et al., the hemoglobin level increased from below the lower limit of normal and stayed in the normal range (11.1-15.9 g/L). Absolute reticulocyte count and LDH levels decreased in all patients receiving pegcetacoplan. In the study by de Castro et al., LDH level remained stable, and within <1.5x upper limit of normal, whereas in the study by Hillman, the mean change of LDH from baseline was -15 +/- 43 U/L. Two of six, seven of 23, and seven of 41 patients reported adverse events in the study by de Castro et al., PADDOCK, and Hillmen et al., respectively. Conclusion Pegcetacoplan effectively improves hemoglobin level and transfusion requirements in patients with PNH, including those unresponsive to eculizumab.
 Cardiovascular System & Cardiology Hematology Life Sciences & Biomedicine Peripheral Vascular Disease Science & Technology

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