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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)....
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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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. |
format | Online Article Text |
id | pubmed-9546219 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
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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