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Multicenter Study
. 2013 Sep;12(5):461-7.
doi: 10.1016/j.jcf.2012.12.009. Epub 2013 Jan 24.

Treatment complexity in cystic fibrosis: trends over time and associations with site-specific outcomes

Gregory S Sawicki  1 , Clement L Ren, Michael W Konstan, Stefanie J Millar, David J Pasta, Alexandra L Quittner; Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis
Affiliations
Multicenter Study

Treatment complexity in cystic fibrosis: trends over time and associations with site-specific outcomes

Gregory S Sawicki et al. J Cyst Fibros. 2013 Sep.

Abstract

Background: Patients with cystic fibrosis (CF) have increasing treatment complexity and high treatment burden. We describe trends in treatment complexity and evaluate its relationship with health outcomes.

Methods: Using Epidemiologic Study of Cystic Fibrosis (ESCF) data, we developed a treatment complexity score (TCS) from 37 chronic therapies and assessed change by age group (6-13, 14-17, and 18+ years) over a three year period. Differences in average site TCS were evaluated by quartiles based on FEV1, BMI, or Treatment Burden score on the Cystic Fibrosis Questionnaire-Revised (CFQ-R).

Results: TCS scores were calculated for 7252 individual patients (42% child, 16% adolescent, 43% adult) across 153 sites. In 2003, mean TCS was 11.1 for children, 11.8 for adolescents, and 12.1 for adults. In all 3 age groups, TCS increased over 3 years; the increase in TCS from 2003-2005 for children was 1.25 (95% CI 1.16-1.34), for adolescents 0.77 (0.62-0.93), and for adults 1.20 (1.08-1.31) (all P<0.001 for trend over time). At the site level, there were no significant differences in mean TCS based on FEV1 quartile. Mean TCS was higher in the highest BMI z-score quartile. Across all 3 versions of the CFQ-R, mean TCS was lower at sites in the highest quartiles (lowest burden) for CFQ-R treatment burden scores.

Conclusion: Treatment complexity was highest among adults with CF, although over 3 years, we observed a significant increase in treatment complexity in all age groups. Such increases in treatment complexity pose a challenge to patient self-management and adherence. Future research is needed to understand the associations between treatment complexity and subsequent health outcomes to reduce treatment burden and improve disease management.

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Conflict of interest statement

Disclosure of Conflict of Interest

Gregory Sawicki, Clement Ren, Michael Konstan, and Alexandra Quittner have received honoraria from Genentech for serving as members of the Scientific Advisory Group for the Epidemiologic Study of Cystic Fibrosis (ESCF) and have served as consultants to Genentech. No compensation was provided to these authors in exchange for production of this manuscript. Stefanie Millar and David Pasta are employees of ICON Late Phase & Outcomes Research, which was paid by Genentech for providing analytical services for this study.

Figures

Figure 1
Figure 1. Mean Treatment Complexity Scores for Each Year by Age Group and Age-Adjusted FEV1 Percent Predicted Decile*
* The test for trend for each decile in each age group is statistically significant (P < 0.05) except for decile 5 for age 14-<18 (P=0.89). The statistical significance of differences between deciles varies somewhat by age group. For age groups 6–13 years and 18 years, differences of 1.16 points or more are always significant (P < 0.05) and many differences as small as 0.5 points are significant. For Age group 14–17 years, most differences of 1.7 points or more are significant, but differences involving Decile 1 may need to be as large as 2.5 points to be significant.
Figure 2
Figure 2. Treatment Complexity Scores by Site-specific Quartiles for FEV1 Percent Predicted and BMI Z-score
*p<0.001 for differences between highest and lowest site quartile and for trend over quartiles for BMI z-score.
Figure 3
Figure 3. Treatment Complexity Scores by site-specific quartiles for CFQ-R Treatment Burden Score
*p<0.001 for differences between highest and lowest site quartiles and for trend over quartiles.
See this image and copyright information in PMC

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