Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis

Nicole Mayer-Hamblett, Margaret Rosenfeld, Miriam Treggiari, Michael W. Konstan, George Retsch-Bogart, Wayne Morgan, Jeff Wagener, Ronald L. Gibson, Umer Khan, Julia Emerson, Valeria Thompson, Eric P. Elkin, Bonnie W. Ramsey

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Rationale The Early Pseudomonal Infection Control (EPIC) randomized trial rigorously evaluated the efficacy of different antibiotic regimens for eradication of newly identified Pseudomonas (Pa) in children with cystic fibrosis (CF). Protocol based therapy in the trial was provided based on culture positivity independent of symptoms. It is unclear whether outcomes observed in the clinical trial were different than those that would have been observed with historical standard of care driven more heavily by respiratory symptoms than culture positivity alone. We hypothesized that the incidence of Pa recurrence and hospitalizations would be significantly reduced among trial participants as compared to historical controls whose standard of care preceded the widespread adoption of tobramycin inhalation solution (TIS) as initial eradication therapy at the time of new isolation of Pa. Methods Eligibility criteria from the trial were used to derive historical controls from the Epidemiologic Study of CF (ESCF) who received standard of care treatment from 1995 to 1998, before widespread availability of TIS. Pa recurrence and hospitalization outcomes were assessed over a 15-month time period. Results As compared to 100% of the 304 trial participants, only 296/608 (49%) historical controls received antibiotics within an average of 20 weeks after new onset Pa. Pa recurrence occurred among 104/298 (35%) of the trial participants as compared to 295/549 (54%) of historical controls (19% difference, 95% CI: 12%, 26%, P <0.001). No significant differences in the incidence of hospitalization were observed between cohorts. Conclusions Protocol-based antimicrobial therapy for newly acquired Pa resulted in a lower rate of Pa recurrence but comparable hospitalization rates as compared to a historical control cohort less aggressively treated with antibiotics for new onset Pa.

Original languageEnglish (US)
Pages (from-to)943-953
Number of pages11
JournalPediatric Pulmonology
Volume48
Issue number10
DOIs
StatePublished - Oct 2013
Externally publishedYes

Fingerprint

Pseudomonas
Cystic Fibrosis
Pseudomonas aeruginosa
Hospitalization
Standard of Care
Recurrence
Tobramycin
Therapeutics
Anti-Bacterial Agents
Inhalation
Incidence
Infection Control
Epidemiologic Studies
Clinical Trials

Keywords

  • cystic fibrosis
  • early intervention
  • historical controls
  • Pseudomonas aeruginosa
  • randomized trial

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine

Cite this

Mayer-Hamblett, N., Rosenfeld, M., Treggiari, M., Konstan, M. W., Retsch-Bogart, G., Morgan, W., ... Ramsey, B. W. (2013). Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis. Pediatric Pulmonology, 48(10), 943-953. https://doi.org/10.1002/ppul.22693

Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis. / Mayer-Hamblett, Nicole; Rosenfeld, Margaret; Treggiari, Miriam; Konstan, Michael W.; Retsch-Bogart, George; Morgan, Wayne; Wagener, Jeff; Gibson, Ronald L.; Khan, Umer; Emerson, Julia; Thompson, Valeria; Elkin, Eric P.; Ramsey, Bonnie W.

In: Pediatric Pulmonology, Vol. 48, No. 10, 10.2013, p. 943-953.

Research output: Contribution to journalArticle

Mayer-Hamblett, N, Rosenfeld, M, Treggiari, M, Konstan, MW, Retsch-Bogart, G, Morgan, W, Wagener, J, Gibson, RL, Khan, U, Emerson, J, Thompson, V, Elkin, EP & Ramsey, BW 2013, 'Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis', Pediatric Pulmonology, vol. 48, no. 10, pp. 943-953. https://doi.org/10.1002/ppul.22693
Mayer-Hamblett, Nicole ; Rosenfeld, Margaret ; Treggiari, Miriam ; Konstan, Michael W. ; Retsch-Bogart, George ; Morgan, Wayne ; Wagener, Jeff ; Gibson, Ronald L. ; Khan, Umer ; Emerson, Julia ; Thompson, Valeria ; Elkin, Eric P. ; Ramsey, Bonnie W. / Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis. In: Pediatric Pulmonology. 2013 ; Vol. 48, No. 10. pp. 943-953.
@article{59a147fd42de4eb780d6ab35bcefc61c,
title = "Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis",
abstract = "Rationale The Early Pseudomonal Infection Control (EPIC) randomized trial rigorously evaluated the efficacy of different antibiotic regimens for eradication of newly identified Pseudomonas (Pa) in children with cystic fibrosis (CF). Protocol based therapy in the trial was provided based on culture positivity independent of symptoms. It is unclear whether outcomes observed in the clinical trial were different than those that would have been observed with historical standard of care driven more heavily by respiratory symptoms than culture positivity alone. We hypothesized that the incidence of Pa recurrence and hospitalizations would be significantly reduced among trial participants as compared to historical controls whose standard of care preceded the widespread adoption of tobramycin inhalation solution (TIS) as initial eradication therapy at the time of new isolation of Pa. Methods Eligibility criteria from the trial were used to derive historical controls from the Epidemiologic Study of CF (ESCF) who received standard of care treatment from 1995 to 1998, before widespread availability of TIS. Pa recurrence and hospitalization outcomes were assessed over a 15-month time period. Results As compared to 100{\%} of the 304 trial participants, only 296/608 (49{\%}) historical controls received antibiotics within an average of 20 weeks after new onset Pa. Pa recurrence occurred among 104/298 (35{\%}) of the trial participants as compared to 295/549 (54{\%}) of historical controls (19{\%} difference, 95{\%} CI: 12{\%}, 26{\%}, P <0.001). No significant differences in the incidence of hospitalization were observed between cohorts. Conclusions Protocol-based antimicrobial therapy for newly acquired Pa resulted in a lower rate of Pa recurrence but comparable hospitalization rates as compared to a historical control cohort less aggressively treated with antibiotics for new onset Pa.",
keywords = "cystic fibrosis, early intervention, historical controls, Pseudomonas aeruginosa, randomized trial",
author = "Nicole Mayer-Hamblett and Margaret Rosenfeld and Miriam Treggiari and Konstan, {Michael W.} and George Retsch-Bogart and Wayne Morgan and Jeff Wagener and Gibson, {Ronald L.} and Umer Khan and Julia Emerson and Valeria Thompson and Elkin, {Eric P.} and Ramsey, {Bonnie W.}",
year = "2013",
month = "10",
doi = "10.1002/ppul.22693",
language = "English (US)",
volume = "48",
pages = "943--953",
journal = "Pediatric Pulmonology",
issn = "8755-6863",
publisher = "Wiley-Liss Inc.",
number = "10",

}

TY - JOUR

T1 - Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis

AU - Mayer-Hamblett, Nicole

AU - Rosenfeld, Margaret

AU - Treggiari, Miriam

AU - Konstan, Michael W.

AU - Retsch-Bogart, George

AU - Morgan, Wayne

AU - Wagener, Jeff

AU - Gibson, Ronald L.

AU - Khan, Umer

AU - Emerson, Julia

AU - Thompson, Valeria

AU - Elkin, Eric P.

AU - Ramsey, Bonnie W.

PY - 2013/10

Y1 - 2013/10

N2 - Rationale The Early Pseudomonal Infection Control (EPIC) randomized trial rigorously evaluated the efficacy of different antibiotic regimens for eradication of newly identified Pseudomonas (Pa) in children with cystic fibrosis (CF). Protocol based therapy in the trial was provided based on culture positivity independent of symptoms. It is unclear whether outcomes observed in the clinical trial were different than those that would have been observed with historical standard of care driven more heavily by respiratory symptoms than culture positivity alone. We hypothesized that the incidence of Pa recurrence and hospitalizations would be significantly reduced among trial participants as compared to historical controls whose standard of care preceded the widespread adoption of tobramycin inhalation solution (TIS) as initial eradication therapy at the time of new isolation of Pa. Methods Eligibility criteria from the trial were used to derive historical controls from the Epidemiologic Study of CF (ESCF) who received standard of care treatment from 1995 to 1998, before widespread availability of TIS. Pa recurrence and hospitalization outcomes were assessed over a 15-month time period. Results As compared to 100% of the 304 trial participants, only 296/608 (49%) historical controls received antibiotics within an average of 20 weeks after new onset Pa. Pa recurrence occurred among 104/298 (35%) of the trial participants as compared to 295/549 (54%) of historical controls (19% difference, 95% CI: 12%, 26%, P <0.001). No significant differences in the incidence of hospitalization were observed between cohorts. Conclusions Protocol-based antimicrobial therapy for newly acquired Pa resulted in a lower rate of Pa recurrence but comparable hospitalization rates as compared to a historical control cohort less aggressively treated with antibiotics for new onset Pa.

AB - Rationale The Early Pseudomonal Infection Control (EPIC) randomized trial rigorously evaluated the efficacy of different antibiotic regimens for eradication of newly identified Pseudomonas (Pa) in children with cystic fibrosis (CF). Protocol based therapy in the trial was provided based on culture positivity independent of symptoms. It is unclear whether outcomes observed in the clinical trial were different than those that would have been observed with historical standard of care driven more heavily by respiratory symptoms than culture positivity alone. We hypothesized that the incidence of Pa recurrence and hospitalizations would be significantly reduced among trial participants as compared to historical controls whose standard of care preceded the widespread adoption of tobramycin inhalation solution (TIS) as initial eradication therapy at the time of new isolation of Pa. Methods Eligibility criteria from the trial were used to derive historical controls from the Epidemiologic Study of CF (ESCF) who received standard of care treatment from 1995 to 1998, before widespread availability of TIS. Pa recurrence and hospitalization outcomes were assessed over a 15-month time period. Results As compared to 100% of the 304 trial participants, only 296/608 (49%) historical controls received antibiotics within an average of 20 weeks after new onset Pa. Pa recurrence occurred among 104/298 (35%) of the trial participants as compared to 295/549 (54%) of historical controls (19% difference, 95% CI: 12%, 26%, P <0.001). No significant differences in the incidence of hospitalization were observed between cohorts. Conclusions Protocol-based antimicrobial therapy for newly acquired Pa resulted in a lower rate of Pa recurrence but comparable hospitalization rates as compared to a historical control cohort less aggressively treated with antibiotics for new onset Pa.

KW - cystic fibrosis

KW - early intervention

KW - historical controls

KW - Pseudomonas aeruginosa

KW - randomized trial

UR - http://www.scopus.com/inward/record.url?scp=84884138505&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84884138505&partnerID=8YFLogxK

U2 - 10.1002/ppul.22693

DO - 10.1002/ppul.22693

M3 - Article

C2 - 23818295

AN - SCOPUS:84884138505

VL - 48

SP - 943

EP - 953

JO - Pediatric Pulmonology

JF - Pediatric Pulmonology

SN - 8755-6863

IS - 10

ER -