Pulmonary exacerbations are clinically debilitating and expensive to treat.
Ideal duration of treatment with antimicrobial medication is unknown.
Clinical outcomes (lung function) were similar between treatment duration groups.
Thirty-day costs were lower in groups that were randomized to receive IV treatment for shorter durations.
The purpose of these analyses was to determine whether overall costs were reduced in cystic fibrosis (CF) patients experiencing pulmonary exacerbation (PEx) who received shorter versus longer durations of treatment.
Among people with CF experiencing PEx, we calculated 30-day inpatient, outpatient, emergency room, and medication costs and summed these to derive total costs in 2020 USD. Using the Kaplan-Meier sample average (KMSA) method, we calculated adjusted costs and differences in costs within two pairs of randomized groups: early robust responders (ERR) randomized to receive treatment for 10 days (ERR-10 days) or 14 days (ERR-14 days), and non-early robust responders (NERR) randomized to receive treatment for 14 days (NERR-14 days) or 21 days (NERR-21 days).
Patients in the shorter treatment duration groups had shorter lengths of stay per hospitalization (mean ± standard deviation (SD) for ERR-10 days: 7.9 ± 3.0 days per hospitalization compared to 10.1 ± 4.2 days in ERR-14 days; for NERR-14 days: 8.7 ± 4.9 days per hospitalization compared to 9.6 ± 6.5 days in NERR-21 days). We found statistically significantly lower adjusted mean costs (95% confidence interval) among those who were randomized to receive shorter treatment durations (ERR-10 days: $60,800 ($59,150 - $62,430) vs $74,420 ($72,610 - $76,450) in ERR-14 days; NERR-14 days: $66,690 ($65,960-$67,400) versus $74,830 ($73,980-$75,650) in NERR-21 days).
Tied with earlier evidence that shorter treatment duration was not associated with worse clinical outcomes, our analyses indicate that treating with shorter antimicrobial durations can reduce costs without diminishing clinical outcomes.
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- Cystic fibrosis transmembrane conductance regulator modulators for cystic fibrosis: a new dawn?.Arch Dis Child. 2021;
- Temporal trends in healthcare resource use and associated costs of patients with cystic fibrosis.J Cyst Fibros. 2021;
- Hospitalization rates among patients with cystic fibrosis using pancreatic enzyme replacement therapy.Chron Respir Dis. 2020; 171479973119900612
- Recent advances in the understanding and management of cystic fibrosis pulmonary exacerbations.F1000Res. 2018; 7
- Frequency and costs of pulmonary exacerbations in patients with cystic fibrosis in the United States.Curr Med Res Opin. 2017; 33: 667-674
- Probability of IV antibiotic retreatment within thirty days is associated with duration and location of IV antibiotic treatment for pulmonary exacerbation in cystic fibrosis.J Cyst Fibros. 2016; 15: 783-790
- A randomized trial of antimicrobial duration for cystic fibrosis pulmonary exacerbation treatment.Am J Respir Crit Care Med. 2021;
- Study design considerations for the standardized treatment of pulmonary exacerbations 2 (STOP2): a trial to compare intravenous antibiotic treatment durations in CF.Contemp Clin Trials. 2018; 64: 35-40
- Pharmaceutical medicine and translational clinical research.2018
- Rationalizing endpoints for prospective studies of pulmonary exacerbation treatment response in cystic fibrosis.J Cyst Fibros. 2017; 16: 607-615
Bureau of Labor Statistics. Databases, Tables, & Calculators by Subject. In: Statistics USBoL, editor. 2021.
- Estimating the costs attributable to a disease with application to ovarian cancer.J Clin Epidemiol. 1996; 49: 95-103
- On the use of survival analysis techniques to estimate medical care costs.J Health Econ. 1999; 18: 365-380
- Regression analysis of incomplete medical cost data.Stat Med. 2003; 22: 1181-1200
- The burden of cystic fibrosis in the Medicaid population.Clinicoecon Outcomes Res. 2018; 10: 423-431
- Treatment of pulmonary exacerbations in cystic fibrosis.Curr Opin Pulm Med. 2020; 26: 679-684
- Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele.N Engl J Med. 2019; 381: 1809-1819
- Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del.N Engl J Med. 2017; 377: 2013-2023
- Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR.N Engl J Med. 2015; 373: 1783-1784
- Effects of hospital practice patterns for antibiotic administration for pneumonia on hospital lengths of stay and costs.J Pediatr Infect Dis Soc. 2019; 8: 115-121
- Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre.Pharmacoeconomics. 2012; 30: 763-777
- Resource use, costs, and utility estimates for patients with cystic fibrosis with mild impairment in lung function: analysis of data collected alongside a 48-week multicenter clinical trial.Value Health. 2012; 15: 277-283
Published online: March 14, 2022
Accepted: March 6, 2022
Received in revised form: March 1, 2022
Received: October 8, 2021
© 2022 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.