Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study.
СтатьяНаблюдательное исследованиеМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Iori Anna Paola, De Vivo Antonio, Di Bona Eros, Caocci Giovanni, Fioritoni Francesca, Ciceri Fabio, Beggiato Eloise, Rapezzi Davide, Amendola Angela, Figuera Amalia, Selleri Carmine, Longu Francesco, Fattizzo Bruno, Tucci Alessandra, Cignetti Alessandro, Amico Valeria, Sica Simona, Metafuni Elisabetta, Raso Simona, Urbano Tiziana Anna, Marano Luana, Di Renzo Nicola, Spedini Pierangelo, Rambaldi Alessandro, Lanza Francesco, Clissa Cristina, Danesin Cristina, Greve Maria Bruna, Cabibbo Sergio, Ori Alessandra, Cassanelli Francesca, Sottana Federica, Campolo Benedetta, Gasparri Giulia, Carini Fabio, Barcellini Wilma
Аннотация
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.