<p><strong>BACKGROUND/PURPOSE: </strong>Prostaglandin E1 (PGE) has been used to maintain ductus arteriosus patency and unload the suprasystemic right ventricle (RV) in neonates with congenital diaphragmatic hernia (CDH) and severe pulmonary hypertension (PH). Here we evaluate the PH response in neonates with CDH and severe PH treated with PGE.</p>

<p><strong>METHODS: </strong>We performed a retrospective chart review of CDH infants treated at our center between 2011 and 2016. In a subset, PGE was initiated for echocardiographic evidence of severe PH, metabolic acidosis, or hypoxemia. To assess PH response, we evaluated laboratory data, including B-type natriuretic peptide (BNP) and echocardiograms before and after PGE treatment. Categorical and continuous data were analyzed with Fisher's exact tests and Mann-Whitney t-tests, respectively.</p>

<p><strong>RESULTS: </strong>Fifty-seven infants were treated with PGE a mean 17 ± 2 days. BNP levels declined after 1.4 ± 0.2 days of treatment and again after 5.2 ± 0.6 days. After 6 ± 0.8 days of treatment, echocardiographic estimates of severe PH by tricuspid regurgitation jet velocity, ductus arteriosus direction, and ventricular septum position also improved significantly. Treatment was not associated with postductal hypoxemia or systemic hypoperfusion.</p>

<p><strong>CONCLUSIONS: </strong>In patients with CDH and severe PH, PGE is well tolerated and associated with improved BNP and echocardiographic indices of PH, suggesting successful unloading of the RV.</p>

<p><strong>TYPE OF STUDY: </strong>Treatment study.</p>

<p><strong>LEVEL OF EVIDENCE: </strong>Level III.</p>

<p>The success of rare disease research relies heavily on robust partnerships with clinicians to help identify new patients and collect samples. Many studies for paediatric rheumatic diseases requiring pretreatment samples have suffered from slow enrolment rates due to the low incidence of disease and relative urgency to treat. Therefore, timely identification of all potentially eligible patients is crucial. The objective of this project was to apply quality improvement methods to increase the frequency and timeliness of identification of eligible patients with new paediatric rheumatic diagnoses to approach for research studies. A retrospective chart review was undertaken in our paediatric rheumatology clinic to measure the number of eligible patients identified for potential research recruitment between missed recruitment opportunities. Improvement methodology was used to integrate standardised communication between clinicians and the research team into clinic workflow, to leverage social feedback as positive reinforcement for good communication and to measure change in response to the interventions. The number of eligible patients identified between missed recruitment opportunities increased from every 0-1 patient to every 14 patients during the project period, corresponding to an increase in the overall identification rate from 32% to 91% of all eligible patients. Quality improvement methods can be used to successfully integrate research recruitment into routine clinical care and accelerate advances necessary to improve health outcomes.</p>