Background: While the Randomized Intervention for children with Vesicoureteral Re ux (RIVUR) trial found that long-term antimicrobial prophylaxis reduced the risk of urinary tract infection (UTI) recurrences by 50%, 10 children had to be treated with long-term antimicrobial prophylaxis for one to bene t (i.e., observed number needed to treat (NNT) of 10). Accordingly, we re-analyzed RIVUR data to systematically identify subgroups of children with vesicoureteral re ux (VUR) with a smaller NNT.Methods: Using patient-level data from the RIVUR trial, we applied penalized regression methods including the baseline age, VUR, and bowel-bladder dysfunction (BBD) as covariates to identify subgroups that consider the trade-off between absolute risk difference and size.Results: We identi ed three relevant subgroups of children that appear to bene t from long-term antimicrobial prophylaxis, all with NNTs smaller than the NNT of 10. Children with grade IV VUR and BBD, 1% of the RIVUR sample, had a NNT of 2; children with BBD, 12% of the RIVUR sample, had a NNT of 4; children with BBD (and any grade VUR) or with grade IV VUR (regardless of BBD status), which was the combination of the rst two subgroups and included 19% of children in the RIVUR sample, had a NNT of 4.Conclusions: Use of long-term antimicrobial prophylaxis appears to be particularly relevant for children with BBD (and any grade of VUR) or those with grade IV VUR (regardless of BBD status) who were at high risk of UTI recurrences.
Key PointsQuestion: In children with vesicoureteral re ux, what combination of clinical and laboratory ndings best predict children who bene t most from long-term antimicrobial prophylaxis? Findings: Children with bowel-bladder dysfunction (irrespective of the grade of vesicoureteral re ux) or those with grade IV vesicoureteral re ux (irrespective of the presence of bowel-bladder dysfunction) appear to bene t the most from long-term antimicrobial prophylaxis.Meaning: This study provides an evidence-based method of selecting children who bene t the most from long-term antimicrobial prophylaxis.
BACKGROUNDThe Randomized Intervention for children with Vesicoureteral Re ux (RIVUR) trial was a multisite, randomized, placebo-controlled trial aimed at evaluating the e cacy of antimicrobial prophylaxis in preventing recurrences of urinary tract infection (UTI) in children with vesicoureteral re ux (VUR) diagnosed after an index UTI [1]. A total of 607 children 2 to 71 months of age were randomized in the ratio of 1:1 to receive either antimicrobial prophylaxis or placebo daily and were followed for 2 years. The