Pragmatic Research on Diuretic Management in Early BPD Pilot

Percent of enrolled infants who completed the full N-of-1 trial, remain on respiratory support at the conclusion of the N-of-1 trial, and were identified as a responder,Percent of providers willing to support randomizing a responder infant

Babies who are born prematurely often develop a chronic lung disease called bronchopulmonary
 dysplasia (BPD). BPD puts babies at higher risk for problems with growth and development.
 Diuretics, such as furosemide, are frequently used in the management of early BPD). Many
 clinicians use informal trials of therapy to see if a baby responds to diuretics in the
 short-term before starting chronic diuretic therapy. Despite frequent use of diuretics, it is
 unclear how many babies truly respond to therapy and if there are long-term benefits of
 diuretic treatment. Designing research studies to figure this out has been challenging. The
 Pragmatic Research on Diuretic Management in Early BPD (PRIMED) study is a feasibility pilot
 study to help us get information to design a larger trial of diuretic management for BPD. Key
 questions this study will answer include: (1) Can we use an N-of-1 trial to determine whether
 a particular baby responds to furosemide? In an N-of-1 trial, a baby is switched between
 furosemide and placebo to compare that particular infant's response on and off diuretics. It
 is a more rigorous approach to the informal trials of therapy that are often conducted in
 clinical care. We hope to learn how many babies have a short-term response to furosemide
 (responders); (2) how many babies will still be on respiratory support at the end of the
 N-of-1 trial? This will help us determine how many patients would be eligible to randomize to
 chronic diuretic therapy in the second phase of the larger trail, and (3) if a baby is
 identified as a short-term responder, how many parents and physicians would be willing to
 randomize the baby to chronic diuretics (3 months) versus placebo in the longer trial?