Identification of predictive factors for reversal of cerebral vasculopathy in an original longitudinal cohort study in newborns with sickle cell anaemia
Julie Sommet, Priscilla Boizeau, Zinedine Haouari, Berengère Koehl, Monique Elmaleh, Emmanuelle Lesprit, Florence Missud, Ghislaine Ithier, Laurent Holvoet, Valentine Brousse, Christophe Delclaux, André Denjean, Suzanne Verlhac, Corinne Alberti, André Baruchel, Nathalie Couque, Malika BenkerrouSummary
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.