Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study

In this study, we collected real‐world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti‐C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the...

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Published inBritish journal of haematology Vol. 206; no. 4; pp. 1246 - 1249
Main Authors Metafuni, Elisabetta, Brioschi, Filippo Achille, Patriarca, Andrea, Leoni, Claudia, Battipaglia, Giorgia, Carlino, Daniela, Condorelli, Annalisa, Di Giacomo, Valeria, Marktel, Sarah, Marzilli, Maria Antonietta, Miglino, Maurizio, Oliva, Esther Natalie, Sau, Antonella, Ricco, Alessandra, Sanpaolo, Grazia, Trolese, Annarita, Iori, Anna Paola, Sica, Simona, Barcellini, Wilma, Fattizzo, Bruno
Format Journal Article
LanguageEnglish
Published England Blackwell Publishing Ltd 01.04.2025
Subjects
Adult
Aged
Antibodies, Monoclonal, Humanized - administration & dosage
Antibodies, Monoclonal, Humanized - adverse effects
Antibodies, Monoclonal, Humanized - therapeutic use
Bone marrow transplantation
Female
Hemoglobin
Hemoglobinuria, Paroxysmal - blood
Hemoglobinuria, Paroxysmal - drug therapy
Hemoglobinuria, Paroxysmal - epidemiology
Humans
Italy - epidemiology
Male
Middle Aged
Italy
Online AccessGet full text
ISSN0007-1048
1365-2141
1365-2141
DOI10.1111/bjh.20025

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Abstract In this study, we collected real‐world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti‐C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6‐month follow‐up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.
AbstractList In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6-month follow-up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6-month follow-up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.
In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6-month follow-up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.
In this study, we collected real‐world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti‐C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6‐month follow‐up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.
Author Marzilli, Maria Antonietta
Miglino, Maurizio
Sica, Simona
Di Giacomo, Valeria
Ricco, Alessandra
Fattizzo, Bruno
Metafuni, Elisabetta
Marktel, Sarah
Leoni, Claudia
Battipaglia, Giorgia
Condorelli, Annalisa
Sau, Antonella
Iori, Anna Paola
Patriarca, Andrea
Trolese, Annarita
Oliva, Esther Natalie
Carlino, Daniela
Sanpaolo, Grazia
Brioschi, Filippo Achille
Barcellini, Wilma
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10.3389/fimmu.2019.01157
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Snippet In this study, we collected real‐world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing...
In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing...
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SubjectTerms Adult
Aged
Antibodies, Monoclonal, Humanized - administration & dosage
Antibodies, Monoclonal, Humanized - adverse effects
Antibodies, Monoclonal, Humanized - therapeutic use
Bone marrow transplantation
Female
Hemoglobin
Hemoglobinuria, Paroxysmal - blood
Hemoglobinuria, Paroxysmal - drug therapy
Hemoglobinuria, Paroxysmal - epidemiology
Humans
Italy - epidemiology
Male
Middle Aged
Title Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study
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