Article Text

Original article
Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis
  1. Sonya L Heltshe1,2,
  2. Christopher H Goss1,3,
  3. Valeria Thompson1,
  4. Scott D Sagel4,
  5. Don B Sanders5,
  6. Bruce C Marshall6,
  7. Patrick A Flume7
  1. 1Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA
  2. 2Department of Pediatrics, University of Washington, School of Medicine, Seattle, Washington, USA
  3. 3Division of Pulmonary and Critical Care Medicine, University of Washington School of Medicine, Seattle, Washington, USA
  4. 4Department of Pediatrics, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora, Colorado, USA
  5. 5Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA
  6. 6Cystic Fibrosis Foundation, Bethesda, Maryland, USA
  7. 7Department of Medicine and Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA
  1. Correspondence to Dr Sonya L Heltshe, Seattle Children's Research Institute M/S CW8-5B, 2001 Eighth Ave, Suite 400, Seattle, WA 98121, USA; sonya.heltshe{at}


Background Treatment of pulmonary exacerbations (PEx) in cystic fibrosis (CF) varies widely with no consensus on management practices or best indicators of therapeutic success. To design trials evaluating PEx treatment factors, we characterise the heterogeneity of PEx care in adults and paediatrics, and correlate it with measures of clinical response including short-term and long-term lung function changes, change in symptom severity score and time to next intravenous antibiotic therapy.

Methods Data were used from a prospective observational study of patients with CF ≥10 years of age enrolled at six sites between 2007 and 2010. All were started on intravenous antibiotics for a clinically diagnosed PEx. Analysis of variance, logistic and Cox regression were used to examine the association of treatment factors with short-term and long-term clinical response.

Results Of 123 patients with CF (60% women, aged 23.1±10.2 years), 33% experienced <10% relative improvement in FEV1 during treatment, which was associated with failing to recover baseline lung function 3 months after treatment (OR=7.8, 95% CI 1.9 to 31.6, p=0.004) and a longer time to next intravenous antibiotic (HR=0.48, 95% CI 0.27 to 0.85, p=0.011). Symptom improvement was observed but was not associated with subsequent lung function or time to next antibiotic therapy, which had a median recurrence time of 143 days.

Conclusions Immediate symptomatic or respiratory response to PEx treatment did not have a clear relationship with subsequent outcomes such as lung function or intravenous antibiotic-free interval. These results can inform future research of treatment regimens for PEx in terms of interventions and outcome measures.

Trial registration NCT00788359 (

  • Cystic Fibrosis

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