Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study

Ravulizumab is a second-generation C5i engineered from eculizumab to achieve immediate, complete, and sustained inhibition of terminal complement activity in PNH. The REACTION observational cohort study describes the effectiveness and tolerability of ravulizumab in Italian patients who were previously treated with eculizumab. Eighty-one PNH patients were enrolled in this study. The primary endpoint was the percentage change in lactate dehydrogenase (LDH) from baseline to the end of observation (52 weeks follow-up). Among secondary endpoints, transfusion avoidance, breakthrough hemolysis (BTH) and patients' quality of life (QoL) were evaluated. The median (25-75 percentiles) percentage change in LDH at 52 weeks follow-up was -2.6 (-11.5-13.4) U/L, with 92.3% of the patients presenting LDH within or < 1.5 × upper limit of normal (ULN). Overall, 20 (25.0%) patients required transfusion during the eculizumab period and 15 (18.8%) during the ravulizumab. Seven BTH events were observed, 5 during eculizumab period and 2 (triggered by other medical conditions) during ravulizumab, suggesting the reduction of pharmacokinetic BTH during ravulizumab treatment. EORTC-QLQ-C30 and FACIT-Fatigue scores were similar to the general population, and patients' preference indicated ravulizumab as the favorite treatment. The REACTION study confirmed the effectiveness of ravulizumab in maintaining stable disease and hemolysis control in the real-world setting. Clinical trial registration. NCT05274633, 02-Mar-2022.

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Journal
Annals of Hematology
Published
2026-01-22
DOI
https://doi.org/10.1007/s00277-026-06792-w
Primary Topic
Complement system in diseases
Type
article
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article

Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study

Tiziana Urbano, Luana Marano, A. Rambaldi, Alessandro Cignetti et al.
Annals of Hematology
Complement system in diseases
article

Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study

Tiziana Urbano, Luana Marano, A. Rambaldi, Alessandro Cignetti, S Cabibbo, N. Di Renzo, Simona Raso, Francesca Fioritoni, Alessandra Ori, Amalia Figuera, Benedetta Campolo, Eloise Beggiato, Giulia Gasparri, Antonio De Vivo, Mark Greve, C. Selleri, F. Cassanelli, Cristina Danesin, Cristina Clissa, Francesco Longu, Fabio Carini, Bruno Fattizzo, Eros Di Bona, Anna Paola Iori, Valeria Amico, Francesco Lanza, Pierangelo Spedini, Elisabetta Metafuni, Davide Rapezzi, Fabio Ciceri, Federica Sottana, Simona Sica
article en

Abstract

Ravulizumab is a second-generation C5i engineered from eculizumab to achieve immediate, complete, and sustained inhibition of terminal complement activity in PNH. The REACTION observational cohort study describes the effectiveness and tolerability of ravulizumab in Italian patients who were previously treated with eculizumab. Eighty-one PNH patients were enrolled in this study. The primary endpoint was the percentage change in lactate dehydrogenase (LDH) from baseline to the end of observation (52 weeks follow-up). Among secondary endpoints, transfusion avoidance, breakthrough hemolysis (BTH) and patients' quality of life (QoL) were evaluated. The median (25-75 percentiles) percentage change in LDH at 52 weeks follow-up was -2.6 (-11.5-13.4) U/L, with 92.3% of the patients presenting LDH within or < 1.5 × upper limit of normal (ULN). Overall, 20 (25.0%) patients required transfusion during the eculizumab period and 15 (18.8%) during the ravulizumab. Seven BTH events were observed, 5 during eculizumab period and 2 (triggered by other medical conditions) during ravulizumab, suggesting the reduction of pharmacokinetic BTH during ravulizumab treatment. EORTC-QLQ-C30 and FACIT-Fatigue scores were similar to the general population, and patients' preference indicated ravulizumab as the favorite treatment. The REACTION study confirmed the effectiveness of ravulizumab in maintaining stable disease and hemolysis control in the real-world setting. Clinical trial registration. NCT05274633, 02-Mar-2022.

Annals of HematologyVol. 105(2)
University of Verona (IT), AOL (United States) (US), Vita-Salute San Raffaele University (IT), University of Sassari (IT), University of Milan (IT), Policlinico Umberto I (IT), Agostino Gemelli University Polyclinic (IT), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), IQVIA (United Kingdom) (GB), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Ospedale "Santa Maria delle Croci" di Ravenna (IT), IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola (IT), Azienda Ospedaliera G.Rummo (IT), Azienda ospedaliera "Bianchi-Melacrino-Morelli" (IT), Ospedale Vincenzo Cervello (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedale Papa Giovanni XXIII (IT), Ospedale di Santo Spirito (IT), Azienda Ospedaliera S.Giuseppe Moscati (IT), Regione Puglia (IT), Ospedale San Bassiano (IT), A. O. Ordine Mauriziano di Torino (IT), Policlinico Universitario di Catania (IT), IRCCS Ospedale San Raffaele (IT), Ospedali Riuniti San Giovanni di Dio e Ruggi d'Aragona (IT), Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo (IT), Ca' Foncello Hospital (IT), Ospedale Vito Fazzi (IT), Gruppo Italiano per il Trapianto di Midollo Osseo (IT), Istituti Ospitalieri di Cremona (IT), Alexion Pharma (Switzerland) (CH)
Alexion Pharmaceuticals
Openalex Percentile: Top 81%
Complement system in diseases
DOIOpen Access PDFOpenAlex
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