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. Author manuscript; available in PMC: 2019 Jun 25.
Published in final edited form as: Pharmacotherapy. 2017 Oct 26;37(11):e120–e121. doi: 10.1002/phar.2029

Efficacy and effectiveness of RSV immunoprophylaxis in children with cystic fibrosis – an unsolved question with more to be asked

Rees L Lee 1, Rebekah F Brown 2, Tebeb Gebretsadik 3, Tina V Hartert 4, William D Dupont 3, Pingsheng Wu 3,4
PMCID: PMC6592267  NIHMSID: NIHMS1034004  PMID: 28950031

The systematic review of the safety and efficacy of palivizumab in children with cystic fibrosis (CF) by Kua and Lee1 demonstrates the woeful lack of definitive data in this area and correctly points out the critical need for additional well-designed studies. Their review examined 10 scientific reports and concluded that respiratory syncytial virus (RSV) immunoprophylaxis “may have a potential role in reducing RSV hospitalizations in children aged less than 2 years with CF”.1 While we fully agree with the authors’ non-committal conclusions, it is important to highlight the extreme caution that must be exercised in interpreting this literature. Five studies purported to show reduced rates of RSV-related hospitalization by comparing palivizumab prophylaxis with no prophylaxis. One study was a simple survey of CF centers in the United Kingdom in which RSV immunoprophylaxis use was restricted to only 3 of 34 responding centers with no additional details regarding patients or center treatment patterns.2 Two studies used historical controls rather than contemporary comparisons, an important limitation as CF morbidity has decreased drastically with recent therapeutic advancements.3, 4 The largest retrospective study is underpowered due to low incidence of RSV hospitalization, and the results are confounded by more aggressive overall treatment in patients receiving immunoprophylaxis and treatment indication.5

RSV prevention has both short- and long-term potential to reduce respiratory morbidity and improve lung health.6–8 In fact, long-term consequences of RSV prevention may be of greater importance than hospitalization rate reductions in infancy. Unfortunately, adequately powered randomized trials would be unfeasibly large. The best solution is to conduct carefully designed large observational studies examining CF infants stratified by RSV immunoprophylaxis status not restricted to RSV-related hospitalization, but with long-term respiratory and health measures being the primary outcomes. Results from such studies will certainly provide vital data in guiding the healthcare of children with CF.

Acknowledgments

Funding support: None

Footnotes

Disclosure of potential conflicts of interest: The authors declare that they have no conflict of interest.

Disclaimer: The views expressed are those of the authors and do not necessarily reflect the official policy or position of the Department of the Navy, Department of Defense or the United States Government. Copyright Notice: Rees Lee is a military service member. This work was prepared as part of his official duties. Title 17 U.S.C. 105 provides that ‘Copyright protection under this title is not available for any work of the United States Government.’ Title 17 U.S.C. 101 defines a United States Government work as a work prepared by a military service member or employee of the United States Government as part of that person’s official duties.

References

  • 1.Kua KP, Lee SWH. Systematic Review of the Safety and Efficacy of Palivizumab among Infants and Young Children with Cystic Fibrosis. Pharmacotherapy. 2017;37(6):755–69. doi: 10.1002/phar.1936. PubMed PMID: . [DOI] [PubMed] [Google Scholar]
  • 2.McCormick J, Southern KW. A survey of palivizumab for infants with cystic fibrosis in the UK. Arch Dis Child. 2007;92(1):87–8. doi: 10.1136/adc.2006.0105338. PubMed PMID: ; PubMed Central PMCID: . [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 3.Speer ME, Fernandes CJ, Boron M, Groothuis JR. Use of Palivizumab for prevention of hospitalization as a result of respiratory syncytial virus in infants with cystic fibrosis. Pediatr Infect Dis J. 2008;27(6):559–61. doi: 10.1097/INF.0b013e3181673c15. PubMed PMID: . [DOI] [PubMed] [Google Scholar]
  • 4.Groves HE, Jenkins L, Macfarlane M, Reid A, Lynn F, Shields MD. Efficacy and long-term outcomes of palivizumab prophylaxis to prevent respiratory syncytial virus infection in infants with cystic fibrosis in Northern Ireland. Pediatr Pulmonol. 2016;51(4):379–85. doi: 10.1002/ppul.23376. PubMed PMID: . [DOI] [PubMed] [Google Scholar]
  • 5.Winterstein AG, Eworuke E, Xu D, Schuler P. Palivizumab immunoprophylaxis effectiveness in children with cystic fibrosis. Pediatr Pulmonol. 2013;48(9):874–84. doi: 10.1002/ppul.22711. PubMed PMID: . [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 6.Stoltz DA, Meyerholz DK, Welsh MJ. Origins of cystic fibrosis lung disease. N Engl J Med. 2015;372(16):1574–5. doi: 10.1056/NEJMc1502191. PubMed PMID: . [DOI] [PubMed] [Google Scholar]
  • 7.Armstrong D, Grimwood K, Carlin JB, et al. Severe viral respiratory infections in infants with cystic fibrosis. Pediatr Pulmonol. 1998;26(6):371–9. PubMed PMID: . [DOI] [PubMed] [Google Scholar]
  • 8.Van Ewijk BE, Wolfs TF, Aerts PC, et al. RSV mediates Pseudomonas aeruginosa binding to cystic fibrosis and normal epithelial cells. Pediatr Res. 2007;61(4):398–403. doi: 10.1203/pdr.0b013e3180332d1c. PubMed PMID: . [DOI] [PubMed] [Google Scholar]

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