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Retrospective multicenter study evaluates crizanlizumab's impact on vaso-occlusive crises in Brazilian sickle cell patients, indicating significant reductions in hospital visits.
Introduction Sickle cell disease (SCD) is a genetic condition characterized by recurrent vaso‐occlusion and chronic hemolysis, which lead to long‐term irreversible complications, reducing patients’ quality of life and survival. Crizanlizumab is a humanized monoclonal antibody that acts in the pathogenesis of vaso‐occlusive crisis (VOCs) preventing their occurrence. The objective of this study is to describe the effectiveness and safety of crizanlizumab in Brazilian patients with SCD. We conducted a retrospective, multicenter study based on medical chart data. Materials and Methods The study period encompassed 1 year preceding the initiation of crizanlizumab treatment and extended through 1 year of therapy. Eligible patients were identified from the Novartis local cohort, classified as Expanded Access Program (EAP). Demographic information, VOC events, and clinical and follow‐up variables were collected. Results Among the 81 patients with at least one VOC prior to crizanlizumab initiation, 61 patients experienced VOC during the posttreatment period. Despite this reduction in the number of patients with VOCs, event‐based analyses showed a decrease in the number of VOCs requiring hospitalization (from 105 to 74) and emergency care (from 213 to 82) after crizanlizumab initiation. The median time between first and second VOCs requiring a healthcare visit increased from 1.66 to 1.84 months, and the median time between VOCs requiring hospitalization increased from 2.22 to 3.73. Conclusions These results suggest that treatment with crizanlizumab is associated with a reduction in VOC occurrence in all genotypes, SCD general complications, duration of hospitalization (in VOCs that required emergency care), and symptoms during VOCs.
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Barros et al. (2026) studied this question.
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