Patient-Reported Outcomes to Understand Infant to Early Childhood Transition of Infants with Bronchopulmonary Dysplasia

PROJECT SUMMARY/ABSTRACT The goal of this proposal is to establish patient-reported outcome measures that refine neonatal risk stratification and identify barriers to post-discharge specialty care, to identify hospital-to-home transition strategies that reduce post-prematurity respiratory disease for preterm infants with bronchopulmonary dysplasia (BPD). For infants with BPD, the transition home from the neonatal intensive care unit (NICU) profoundly impacts both children and their parents. Nearly 70% of infants with BPD develop post-prematurity respiratory disease after NICU discharge, defined as a composite of respiratory symptoms, hospitalizations, equipment and medications. In practice, equipment and medications should reduce symptoms and hospitalization, but the effectiveness of specialty care depends on successful interactions between a family and health system. Family perspectives are thus critical to evaluating the complex care that supports infants with BPD after the NICU. However, there are critical gaps in available patient-reported outcome measures for young children. First, there is no validated tool to assess respiratory symptoms for infants with BPD. Respiratory symptom questionnaires currently used for infants with BPD were developed for pediatric asthma, and fail to capture symptoms specific to the transition home from the NICU. Second, barriers to care have not been studied in relationship to pulmonary outcomes of infants with BPD. Since prematurity disproportionately impacts families experiencing socioeconomic disadvantage, and infants with BPD require complex specialty care with wide variation in availability and coordination of needed supports, it is likely that multiple domains of barriers to care exacerbate the impact of post-prematurity respiratory disease. Over the past several years, supported by a NHLBI K23 award, we have developed mobile health (mHealth) strategies to determine how post-discharge specialty care for infants with BPD impacts patient-reported outcomes. We now propose to leverage this experience to recruit at least 750 infants from 15 sites in the Children’s Hospitals Neonatal Consortium (CHNC), a group which offers shared NICU data infrastructure, a collaborative publication record, and large numbers of infants across key disease characteristics. We will link NICU data to mobile-enabled parent-reported outcomes from pre-NICU discharge through two years of age. The first aim seeks to determine how NICU illness characteristics predict respiratory symptom trajectories, using a novel BPD-specific respiratory symptom measure which was developed and tested in our preliminary work. The second aim seeks to determine how barriers to care explain variation in post-prematurity respiratory disease, using parent- reported measures used in other pediatric complex care populations. Our central hypothesis is that patient- reported measures over the transition from NICU to home will identify infants with BPD at risk of prolonged symptom trajectories and barriers to care that worsen post-prematurity respiratory disease. Results will identify actionable targets for system interventions to improve pulmonary outcomes for premature infants.