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

on behalf of the STOP Study Group

Research output: Contribution to journalArticle

Abstract

Background 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 1 s (FEV1% predicted) and symptom responses at 7–10 days 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 > 15 days, 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. 14 days for ERR, 14 vs. 21 days 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.

Original languageEnglish (US)
Pages (from-to)35-40
Number of pages6
JournalContemporary Clinical Trials
Volume64
DOIs
StatePublished - Jan 2018

Keywords

  • Cystic fibrosis
  • FEV1
  • Pulmonary exacerbation
  • Symptoms

ASJC Scopus subject areas

  • Pharmacology (medical)

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