Leveraging real-world Data from administrative claims and medical records to inform safety and effectiveness of piperacillin-tazobactam in the Management of Pediatric Hospital Acquired Pneumonia

Abstract Pediatric-specific safety data are required during development of pharmaceutical agents. Retrospective studies can leverage real-world data to assess safety and effectiveness in children where prospective, controlled studies are not feasible. A retrospective cohort study combined data from Pediatric Health Information Systems (PHIS) and medical records to evaluate the safety and effectiveness of piperacillin/tazobactam (P/T) in pediatric patients with hospital-acquired pneumonia (HAP). After identifying 407 patients diagnosed with HAP receiving P/T (n=140) or Comparator (n=267) HAP-appropriate antibiotics between 2003-2016 across seven pediatric institutions, we evaluated comparative risk of a serious adverse event (SAE). Clinical improvement 14 days after therapy initiation was studied as a secondary outcome. Incidence rate ratios (IRRs) were calculated to compare between exposure groups using inverse probability-weighted Poisson regression models. The unadjusted and adjusted IRRs with 95% CIs for SAEs were 1.26(0.66-2.39) and 1.24(0.65-2.35). The unadjusted and adjusted ORs with 95% CIs for clinical improvement were 1.14(0.56-2.34) and 1.50(0.67-3.38). Point estimates from this retrospective analysis suggest similar safety and clinical effectiveness of P/T and comparator antibiotics for treating HAP. However, due to wide CIs, actual between-group differences cannot be excluded. Existing real-world data can be utilized to inform pediatric-specific safety and effectiveness of medications used in off-label settings.