INTRODUCTION
In Cystic Fibrosis (CF) lung disease, inflammation due to bacterial
colonization and neutrophil recruitment increases during an Acute
Pulmonary Exacerbation (APE) 1. Typical treatment of
moderate to severe APE supported by the evidence based CF pulmonary
guidelines include intravenous (IV) antibiotics in the hospital setting,
maintenance of chronic therapies for lung health, and more frequent
administration of airway clearance therapy 2. Despite
these treatments, as many as 25% of patients fail to recover to their
baseline FEV1 after treatment 3.
Corticosteroid therapy and its potent anti-inflammatory property has
long been postulated to be of benefit in CF either by preventing decline
in baseline FEV1 percent predicted (FEV1pp) or reversing decline
associated with APE. CF pulmonary guidelines for maintenance of lung
health report that there is insufficient evidence to recommend routine
use of oral corticosteroids in children with CF due to “net-negative”
effects 4.
In clinical practice, steroid use remains variable. Across 38 CF centers
treating hospitalized pediatric patients with CF, steroid treatment use
ranged from 3-61% during APE 5. In the STOP trial, a
prospective observational study that assessed APE treatment practices at
11 CF centers, 21% of the 220 enrolled patients were given
corticosteroids as adjunct therapy 6. Our pediatric CF
center providers have an informal practice of five to seven day
“rescue” steroid treatment when standard treatment fails to
demonstrate expected improvement in FEV1pp following at least one week
of guideline driven hospital based therapy. We hypothesized that use of
oral corticosteroids during failed inpatient APE management would
increase FEV1pp at discharge and at hospital follow-up visit. We also
wanted to understand when and why providers engage in this approach. To
evaluate our hypotheses, we conducted a retrospective study examining a
cohort of CF patients and recovery to their baseline FEV1pp after
hospital admission for APE, FEV1pp at follow up visit, and time to next
APE compared to routine treatment. We also employed a propensity score
matching scheme to more fairly estimate treatment effect by controlling
specific covariates.