Cystic fibrosis · Duration of exacerbation treatment
A Randomized Clinical Trial of Antimicrobial Duration for Cystic Fibrosis Pulmonary Exacerbation Treatment
| Design | Randomised controlled trial with response-adaptive allocation to non-inferiority or superiority comparisons |
|---|---|
| Treatment | 10 days of intravenous antimicrobials for early responders, or 21 days for slower responders |
| Control | 14 days of intravenous antimicrobials |
| Population | 982 adults with cystic fibrosis treated for a pulmonary exacerbation; 277 early responders and 705 with lesser improvement |
| Follow-up | Through treatment and follow-up |
| Primary endpoint | Change in percentage predicted FEV1 |
| Result | Among early responders, mean ppFEV1 change was 12.8 with 10 days and 13.4 with 14 days — a difference of -0.65 (95% CI -3.3 to 2.0), excluding the predefined non-inferiority margin. Among those with less improvement, 21 and 14 days gave changes of 3.3 and 3.4, a difference of -0.10 (-1.3 to 1.1). Secondary endpoints and sensitivity analyses were supportive. |
| Secondary endpoints | Symptoms, weight, adverse events |
Among adults with CF with early treatment improvement during exacerbation, ppFEV1 after 10 days of intravenous antimicrobials is not inferior to 14 days. For those with less improvement after one week, 21 days is not superior to 14 days.
The trial authors, in the published abstract
Answers both halves of the duration question in one adaptive design: shorten treatment for those who respond, and do not extend it for those who do not. The same short-course logic that PneumA established in ventilator-associated pneumonia, arrived at two decades later in cystic fibrosis.
Goss CH, Heltshe SL, West NE, et al. A Randomized Clinical Trial of Antimicrobial Duration for Cystic Fibrosis Pulmonary Exacerbation Treatment. Am J Respir Crit Care Med 2021 Dec 1;204(11):1295-1305.