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Palivizumab and Long-term Outcomes in Cystic Fibrosis
Aliza K Fink1, Gavin Graff2, Carrie L Byington3
1Cystic Fibrosis Foundation, Bethesda, Maryland; afink@cff.org.
Insights
Palivizumab prophylaxis did not improve longer-term outcomes for infants with cystic fibrosis (CF). This study found no significant differences in lung function, Pseudomonas aeruginosa infections, or hospitalizations by age seven.
Area of Science:
- Pediatric Pulmonology
- Infectious Disease Management
Background:
- Current guidelines suggest palivizumab for high-risk infants with cystic fibrosis (CF), but its long-term benefits remain unclear.
- Uncertainty exists regarding the impact of palivizumab on sustained health outcomes in young children with CF.
Purpose of the Study:
- To evaluate the association between palivizumab use in the first two years of life and longer-term outcomes in children with CF.
- Assessed lung function, time to first Pseudomonas infection, and hospitalization rates up to age seven.
Main Methods:
- Utilized data from the Cystic Fibrosis Foundation Patient Registry for infants born between 2008-2015 diagnosed with CF within six months.
- Employed propensity score adjustment to control for confounding factors, including indication for treatment.
- Performed regression analyses to assess the relationship between palivizumab receipt and key health outcomes.
Main Results:
- Analysis included 4267 infants; 37% received palivizumab.
- No significant difference in mean lung function (percent predicted FEV1) at age seven was observed between palivizumab recipients and non-recipients.
- Time to first positive Pseudomonas aeruginosa culture and annual hospitalization risk were similar in both groups.
Conclusions:
- Palivizumab prophylaxis was not associated with improved longer-term health outcomes in the general population of children with CF.
- Findings suggest that routine palivizumab use may not confer sustained benefits for lung function or infection rates in CF patients.
Background:
The American Academy of Pediatrics does not recommend routine use of palivizumab prophylaxis for infants with cystic fibrosis (CF) but recommends consideration in infants with clinical evidence of chronic lung disease or nutritional compromise. However, the beneficial impact of palivizumab on longer-term outcomes is uncertain.
Methods:
We used Cystic Fibrosis Foundation Patient Registry data to assess the association of receiving palivizumab during the first 2 years of life with longer-term outcomes, including lung function at 7 years old, time to first positive Pseudomonas respiratory culture, and pulmonary-related hospitalizations during the first 7 years of life. Eligible infants were born from 2008 to 2015 and diagnosed with CF during the first 6 months of life. Demographic and clinical confounders of association between palivizumab receipt and outcomes were explored. We created propensity scores to adjust for potential confounding by indication (ie, sicker infants were more likely to receive palivizumab). For each outcome, we performed regression analyses adjusted by propensity scores.
Results:
The sample included 4267 infants; 1588 (37%) received palivizumab. Mean percent forced expiratory volume in 1 second predicted at 7 years old was similar among those who did (98.2; 95% confidence interval: 96.9-99.5) and did not (97.3; 95% confidence interval: 96.1-98.5) received palivizumab, adjusting for propensity scores. Time to first positive Pseudomonas aeruginosa culture and annual risk of hospitalization were similar among those who did and did not receive palivizumab.
Conclusions:
At the population level, palivizumab receipt was not associated with improved longer-term outcomes in children with CF.
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