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Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis
Sonya L Heltshe1, Christopher H Goss2, Valeria Thompson3
1Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA Department of Pediatrics, University of Washington, School of Medicine, Seattle, Washington, USA.
Insights
Pulmonary exacerbations (PEx) in cystic fibrosis (CF) management lacks clear indicators of success. Symptom improvement alone does not predict better lung function or longer antibiotic-free periods in CF patients.
Area of Science:
- Pulmonary Medicine
- Cystic Fibrosis Research
- Clinical Trial Analysis
Background:
- Treatment for pulmonary exacerbations (PEx) in cystic fibrosis (CF) lacks standardized protocols and outcome measures.
- Significant heterogeneity exists in PEx care for both pediatric and adult CF patients.
- Clinical trial registration: NCT00788359.
Purpose of the Study:
- To characterize the variability in PEx management among CF patients.
- To correlate PEx treatment factors with clinical response indicators.
- To identify reliable measures for assessing therapeutic success in CF PEx.
Main Methods:
- Prospective observational study of 123 CF patients (≥10 years) receiving intravenous antibiotics for PEx.
- Data collected between 2007 and 2010 across six clinical sites.
- Statistical analyses included ANOVA, logistic, and Cox regression to assess treatment associations with outcomes.
Main Results:
- 33% of patients showed <10% improvement in FEV1, linked to failure to regain baseline lung function and shorter antibiotic-free intervals.
- Symptom improvement was not associated with subsequent lung function or time to next antibiotic therapy.
- Median time to recurrent PEx requiring intravenous antibiotics was 143 days.
Conclusions:
- Short-term symptomatic or respiratory response to PEx treatment does not reliably predict long-term outcomes like lung function or antibiotic-free duration.
- Current PEx outcome measures may not adequately reflect treatment efficacy.
- Findings can guide future research in CF PEx interventions and outcome assessment.
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 (www.clinicaltrials.gov).
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