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Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies

OBJECTIVES: The complement component 5 (C5) inhibitor ravulizumab demonstrated non‐inferiority to eculizumab following 26 weeks of treatment in complement inhibitor‐naïve and complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria (PNH; studies 301 and 302, respectively)....

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Autores principales: Kulasekararaj, Austin G., Griffin, Morag, Langemeijer, Saskia, Usuki, Kensuke, Kulagin, Alexander, Ogawa, Masayo, Yu, Ji, Mujeebuddin, Arshad, Nishimura, Jun‐ichi, Lee, Jong Wook, Peffault de Latour, Régis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9546219/
https://www.ncbi.nlm.nih.gov/pubmed/35502600
http://dx.doi.org/10.1111/ejh.13783
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author Kulasekararaj, Austin G.
Griffin, Morag
Langemeijer, Saskia
Usuki, Kensuke
Kulagin, Alexander
Ogawa, Masayo
Yu, Ji
Mujeebuddin, Arshad
Nishimura, Jun‐ichi
Lee, Jong Wook
Peffault de Latour, Régis
author_facet Kulasekararaj, Austin G.
Griffin, Morag
Langemeijer, Saskia
Usuki, Kensuke
Kulagin, Alexander
Ogawa, Masayo
Yu, Ji
Mujeebuddin, Arshad
Nishimura, Jun‐ichi
Lee, Jong Wook
Peffault de Latour, Régis
author_sort Kulasekararaj, Austin G.
collection PubMed
description OBJECTIVES: The complement component 5 (C5) inhibitor ravulizumab demonstrated non‐inferiority to eculizumab following 26 weeks of treatment in complement inhibitor‐naïve and complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria (PNH; studies 301 and 302, respectively). This study aims to describe the results of both studies from 27 weeks to 2 years. METHODS: Patients (N = 441) continued to receive ravulizumab throughout the extension period. Efficacy endpoints included lactate dehydrogenase (LDH) normalization, transfusion avoidance and fatigue score (FACIT‐F). Safety analyses were also performed. RESULTS: From 27 weeks to 2 years, improvements in LDH levels were maintained in both study populations. Transfusion avoidance was maintained in 81.9% (study 301) and 85.6% (study 302) of patients, and FACIT‐F scores remained stable. Ravulizumab was well tolerated, and the incidence of adverse events (AEs) were similar between patients of both studies. Incidence of serious AEs deemed related to ravulizumab treatment was low (<3%). CONCLUSIONS: This study reports, to date, the longest period of follow‐up in over 400 patients with PNH treated with ravulizumab (662 patient‐years). Long‐term, ravulizumab demonstrated durable efficacy and was well tolerated, highlighting the importance of C5 inhibitors as the mainstay of PNH treatment.
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spelling pubmed-95462192022-10-14 Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies Kulasekararaj, Austin G. Griffin, Morag Langemeijer, Saskia Usuki, Kensuke Kulagin, Alexander Ogawa, Masayo Yu, Ji Mujeebuddin, Arshad Nishimura, Jun‐ichi Lee, Jong Wook Peffault de Latour, Régis Eur J Haematol Original Articles OBJECTIVES: The complement component 5 (C5) inhibitor ravulizumab demonstrated non‐inferiority to eculizumab following 26 weeks of treatment in complement inhibitor‐naïve and complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria (PNH; studies 301 and 302, respectively). This study aims to describe the results of both studies from 27 weeks to 2 years. METHODS: Patients (N = 441) continued to receive ravulizumab throughout the extension period. Efficacy endpoints included lactate dehydrogenase (LDH) normalization, transfusion avoidance and fatigue score (FACIT‐F). Safety analyses were also performed. RESULTS: From 27 weeks to 2 years, improvements in LDH levels were maintained in both study populations. Transfusion avoidance was maintained in 81.9% (study 301) and 85.6% (study 302) of patients, and FACIT‐F scores remained stable. Ravulizumab was well tolerated, and the incidence of adverse events (AEs) were similar between patients of both studies. Incidence of serious AEs deemed related to ravulizumab treatment was low (<3%). CONCLUSIONS: This study reports, to date, the longest period of follow‐up in over 400 patients with PNH treated with ravulizumab (662 patient‐years). Long‐term, ravulizumab demonstrated durable efficacy and was well tolerated, highlighting the importance of C5 inhibitors as the mainstay of PNH treatment. John Wiley and Sons Inc. 2022-06-16 2022-09 /pmc/articles/PMC9546219/ /pubmed/35502600 http://dx.doi.org/10.1111/ejh.13783 Text en © 2022 Alexion, AstraZeneca Rare Disease. European Journal of Haematology published by John Wiley & Sons Ltd. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
spellingShingle Original Articles
Kulasekararaj, Austin G.
Griffin, Morag
Langemeijer, Saskia
Usuki, Kensuke
Kulagin, Alexander
Ogawa, Masayo
Yu, Ji
Mujeebuddin, Arshad
Nishimura, Jun‐ichi
Lee, Jong Wook
Peffault de Latour, Régis
Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title_full Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title_fullStr Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title_full_unstemmed Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title_short Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
title_sort long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9546219/
https://www.ncbi.nlm.nih.gov/pubmed/35502600
http://dx.doi.org/10.1111/ejh.13783
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