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

Autor: Shah S; Maharagunj Medical Campus Tribhuvan University, Institute of Medicine Maharajgunj Nepal., Chamlagain R; Tribhuvan University Teaching Hospital Maharajgunj Nepal., Musalman ZH; Maharagunj Medical Campus Tribhuvan University, Institute of Medicine Maharajgunj Nepal., Raj Adhikari Y; Tribhuvan University Teaching Hospital Maharajgunj Nepal., Chhetri S; Department of Nephrology and Transplant Medicine Tribhuvan University, Institute of Medicine Maharajgunj Nepal., Paudel S; Division of Research & Academic Affairs Larkin Community Hospital Miami Florida USA., Gundabolu K; Fred and Pamela Buffett Cancer Center, Department of Internal Medicine, Division of Hematology-Oncology University of Nebraska Medical Center Omaha Nebraska USA., Dhakal P; Department of Hematology, Oncology, and Blood & Marrow Transplantation, Department of Internal Medicine University of Iowa Health Care Iowa City Iowa USA.
Jazyk: angličtina
Zdroj: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2022 Aug 07; Vol. 6 (5), pp. e12781. Date of Electronic Publication: 2022 Aug 07 (Print Publication: 2022).
DOI: 10.1002/rth2.12781
Abstrakt: 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 <1.5× 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.
(© 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).)
Databáze: MEDLINE
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