Identification of predictive factors for reversal of cerebral vasculopathy in an original longitudinal cohort study in newborns with sickle cell anaemia.

Cerebral macrovasculopathy (CV) is a major complication in children with sickle cell anaemia (SCA) and usually requires a long-term transfusion programme (TP) to prevent stroke. This study aimed to identify factors predicting reversal of CV on TP in a single-centre newborn cohort. Among 375 patients, 50 presented CV and received TP. Median age at diagnosis was 4.9 years [3.6-6.1], with median follow-up of 13.3 years (915 patient-years). Transcranial Doppler (TCD) velocities and magnetic resonance angiography (MRA) normalised in 35 patients, that is, they had reversal of CV (RCV group). Fifteen patients had no reversal of CV (No-RCV group). Kaplan-Meier analysis estimated that 48% (95% confidence interval [CI] [33-62]) still had abnormal TCD and/or MRA 5 years after diagnosis. In a multivariate Cox model, three baseline factors independently predicted CV reversal: age at CV onset, initial severity of stenosis and bronchial obstruction. Older age at diagnosis increased the likelihood of CV reversal (hazard ratio [HR] 1.31 per year, 95% CI [1.08-1.59], p = 0.01). In contrast, severe stenosis (HR 0.72 per 10% increase, 95% CI [0.62-0.85], p < 0.0001) and bronchial obstruction (HR 0.14, 95% CI [0.03-0.63], p = 0.001) reduced the likelihood of CV reversal. These results suggest that, beyond TP, managing bronchial obstruction may improve vascular outcomes.