Efficacy of omalizumab after discontinuation: a retrospective single-center observational study in children with severe asthma

Simone Foti Randazzese ^1* Cecilia Lugarà ¹ Francesca Galletta ¹ Giovanni Pioggia ² Giuseppe Crisafulli ¹ Lucia Caminiti ¹ Sebastiano Gangemi ³ Paolo Ruggeri ^4* Sara Manti ¹

• ¹ Pediatric Unit, Department of Human Pathology in Adult and Developmental Age “Gaetano Barresi”, University of Messina, Via Consolare Valeria, 1, 98124, Messina, Italy
• ² Institute for Biomedical Research and Innovation (IRIB), National Research Council of Italy (CNR), Via Vincenzo Leanza, 98164, Messina, Italy
• ³ School and Operative Unit of Allergy and Clinical Immunology, Department of Clinical and Experimental Medicine, University of Messina, Via Consolare Valeria, 1, 98124, Messina, Italy
• ⁴ Pulmonology Unit, Department of Biomedical, Dental, Morphological and Functional Imaging Sciences (BIOMORF), University of Messina, Via Consolare Valeria, 1, 98124, Messina, Italy

Introduction: Several trials documented safety and efficacy of omalizumab, but there are a few data about its effects after discontinuation. This study aims to evaluate the maintenance of efficacy of omalizumab in pediatric asthmatic patients one year after its suspension.Methods: A retrospective analysis was conducted on 17 subjects aged 6-18 years, divided into two groups: Group A (9 patients) who discontinued omalizumab after 18 months, and Group B (8 patients) who continued the therapy. Data on respiratory function (FEV1%), the number of exacerbations, need for hospitalizations, use of oral corticosteroids, and Asthma Control Test (ACT) scores were collected and analyzed at three time points: baseline (T0), after 18 months of treatment (T1), and 36 months (T2).Results: In Group A, significant differences were observed between T0 and T1, and T1 and T2, in FEV1% values, the number of exacerbations, the need for oral corticosteroids, and ACT scores. Group B showed significant differences in these parameters over time, with a notable reduction in exacerbations and improvement in ACT scores. The comparative analysis revealed that Group B had a higher number of exacerbations compared to Group A at T0 and greater use of oral cortico-steroids at T1. By T2, Group A had a higher ACT score than Group B at T0, whereas Group B showed higher ACT scores at T2 compared to Group A.Discussion: The study confirmed the efficacy and safety of omalizumab, with its benefits persisting one year after treatment discontinuation in terms of lung function, reduction in exacerbations, decreased need for oral corticosteroids, and improved quality of life. Further research is necessary.