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Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria.
Expert Opin Biol Ther. 2020 Mar; 20(3):227-237.EO

Abstract

Introduction: Eculizumab, which is indicated to treat patients with paroxysmal nocturnal hemoglobinuria (PNH), is a life-changing, life-saving therapy that decreases intravascular hemolysis and thrombosis and improves survival. Some eculizumab-treated patients, however, experience breakthrough hemolysis; and overall, the burden of the treatment schedule (intravenous infusions every 2 weeks) is substantial. Ravulizumab is a long-acting, second-generation complement component 5 (C5) inhibitor that is administered intravenously every 8 weeks. It is approved in the United States (December 2018), Japan (June 2019), Europe (July 2019), and Canada and Brazil (September 2019).Areas covered: This article reviews data presented in journal articles identified on Medline/PubMed, abstracts presented at hematology meetings, and information posted on ClinicalTrials.gov and Alexion.com. Emphasis is placed on the non-inferiority of ravulizumab compared to eculizumab and the advantages of the 8-week, weight-based, dosing regimen.Expert opinion: In phase 3 trials, ravulizumab has been shown to be as safe and efficacious as eculizumab, to be associated numerically with lower rates of breakthrough hemolysis (p for non-inferiority <0.0004), and to be preferred over eculizumab by most patients. Ravulizumab is likely to replace eculizumab as the first-line treatment for PNH both in patients who are naive to eculizumab treatment and in patients who are clinically stable on eculizumab.

Authors+Show Affiliations

Division of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.Department of Haematological Medicine, King's College Hospital, NHS Foundation Trust and King's College, London, UK.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32011183

Citation

Lee, Jong Wook, and Austin G. Kulasekararaj. "Ravulizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria." Expert Opinion On Biological Therapy, vol. 20, no. 3, 2020, pp. 227-237.
Lee JW, Kulasekararaj AG. Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Expert Opin Biol Ther. 2020;20(3):227-237.
Lee, J. W., & Kulasekararaj, A. G. (2020). Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Expert Opinion On Biological Therapy, 20(3), 227-237. https://doi.org/10.1080/14712598.2020.1725468
Lee JW, Kulasekararaj AG. Ravulizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Expert Opin Biol Ther. 2020;20(3):227-237. PubMed PMID: 32011183.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria. AU - Lee,Jong Wook, AU - Kulasekararaj,Austin G, Y1 - 2020/02/14/ PY - 2020/2/6/pubmed PY - 2020/2/6/medline PY - 2020/2/4/entrez KW - Breakthrough hemolysis KW - eculizumab KW - paroxysmal nocturnal hemoglobinuria KW - quality of life KW - ravulizumab KW - terminal complement inhibition SP - 227 EP - 237 JF - Expert opinion on biological therapy JO - Expert Opin Biol Ther VL - 20 IS - 3 N2 - Introduction: Eculizumab, which is indicated to treat patients with paroxysmal nocturnal hemoglobinuria (PNH), is a life-changing, life-saving therapy that decreases intravascular hemolysis and thrombosis and improves survival. Some eculizumab-treated patients, however, experience breakthrough hemolysis; and overall, the burden of the treatment schedule (intravenous infusions every 2 weeks) is substantial. Ravulizumab is a long-acting, second-generation complement component 5 (C5) inhibitor that is administered intravenously every 8 weeks. It is approved in the United States (December 2018), Japan (June 2019), Europe (July 2019), and Canada and Brazil (September 2019).Areas covered: This article reviews data presented in journal articles identified on Medline/PubMed, abstracts presented at hematology meetings, and information posted on ClinicalTrials.gov and Alexion.com. Emphasis is placed on the non-inferiority of ravulizumab compared to eculizumab and the advantages of the 8-week, weight-based, dosing regimen.Expert opinion: In phase 3 trials, ravulizumab has been shown to be as safe and efficacious as eculizumab, to be associated numerically with lower rates of breakthrough hemolysis (p for non-inferiority <0.0004), and to be preferred over eculizumab by most patients. Ravulizumab is likely to replace eculizumab as the first-line treatment for PNH both in patients who are naive to eculizumab treatment and in patients who are clinically stable on eculizumab. SN - 1744-7682 UR - https://www.unboundmedicine.com/medline/citation/32011183/Ravulizumab_for_the_treatment_of_paroxysmal_nocturnal_hemoglobinuria_ L2 - http://www.tandfonline.com/doi/full/10.1080/14712598.2020.1725468 DB - PRIME DP - Unbound Medicine ER -
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