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

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...

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Published in:Research and practice in thrombosis and haemostasis 2022-07, Vol.6 (5), p.e12781-n/a, Article e12781
Main Authors: Shah, Sangam, Chamlagain, Rajan, Musalman, Ziyaul Haq, Raj Adhikari, Yagya, Chhetri, Santosh, Paudel, Sujan, Gundabolu, Krishna, Dhakal, Prajwal
Format: Article
Language:English
Subjects:
Anemia
Blood
Confidence intervals
Dehydrogenases
eculizumab
efficacy
Hemoglobin
Monoclonal antibodies
Original
paroxysmal nocturnal hemoglobinuria
Patients
pegcetacoplan
Proteins
safety
Systematic review
Thrombosis
Urine
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Summary: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 clinicaltrials.gov. 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
ISSN:2475-0379
2475-0379
DOI:10.1002/rth2.12781