Study design considerations for the Standardized Treatment of Pulmonary Exacerbations 2 (STOP2): A trial to compare intravenous antibiotic treatment durations in CF

dc.contributor.authorHeltshe, Sonya L.
dc.contributor.authorWest, Natalie E.
dc.contributor.authorVanDevanter, Donald R.
dc.contributor.authorSanders, D. B.
dc.contributor.authorBeckett, Valeria V.
dc.contributor.authorFlume, Patrick A.
dc.contributor.authorGoss, Christopher H.
dc.contributor.departmentPediatrics, School of Medicineen_US
dc.date.accessioned2019-06-26T20:00:21Z
dc.date.available2019-06-26T20:00:21Z
dc.date.issued2018-01
dc.description.abstractBACKGROUND: Pulmonary exacerbations (PEx) in cystic fibrosis (CF) are common and contribute to morbidity and mortality. Duration of IV antibiotic therapy to treat PEx varies widely in the US, and there are few data to guide treatment decisions. METHODS: We combined a survey of CF stakeholders with retrospective analyses of a recent observational study of CF PEx to design a multicenter, randomized, prospective study comparing the efficacy and safety of different durations of IV antibiotics for PEx to meet the needs of people with CF and their caregivers. RESULTS: IV antibiotic duration was cited as the most important PEx research question by responding CF physicians and top concern among surveyed CF patients/caregivers. During PEx, forced expiratory volume in 1s (FEV1% predicted) and symptom responses at 7-10days of IV antibiotics identified two distinct groups: early robust responders (ERR) who subsequently experienced greater FEV1 improvements compared to non-ERR (NERR). In addition to greater FEV1 and symptom responses, only 14% of ERR patients were treated with IV antibiotics for >15days, compared with 45% of NERR patients. CONCLUSIONS: A divergent trial design that evaluates subjects' interim improvement in FEV1 and symptoms to tailor randomization to IV treatment duration (10 vs. 14days for ERR, 14 vs. 21days for NERR) may alleviate physician and patient concerns about excess or inadequate treatment. Such a study has the potential to provide evidence necessary to standardize IV antibiotic duration in CF PEx care -a first step to conducting PEx research of other treatment features.en_US
dc.eprint.versionAuthor's manuscripten_US
dc.identifier.citationHeltshe, S. L., West, N. E., VanDevanter, D. R., Sanders, D. B., Beckett, V. V., Flume, P. A., … STOP Study Group (2018). Study design considerations for the Standardized Treatment of Pulmonary Exacerbations 2 (STOP2): A trial to compare intravenous antibiotic treatment durations in CF. Contemporary clinical trials, 64, 35–40. doi:10.1016/j.cct.2017.11.012en_US
dc.identifier.urihttps://hdl.handle.net/1805/19701
dc.language.isoen_USen_US
dc.publisherElsevieren_US
dc.relation.isversionof10.1016/j.cct.2017.11.012en_US
dc.relation.journalContemporary Clinical Trialsen_US
dc.rightsPublisher Policyen_US
dc.sourcePMCen_US
dc.subjectCystic fibrosisen_US
dc.subjectFEV1en_US
dc.subjectPulmonary exacerbationen_US
dc.subjectSymptomsen_US
dc.titleStudy design considerations for the Standardized Treatment of Pulmonary Exacerbations 2 (STOP2): A trial to compare intravenous antibiotic treatment durations in CFen_US
dc.typeArticleen_US
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