Pilot study success with a new cystic fibrosis treatment adherence tool may augur similar positive results from current RCT. Outcomes are due, soon.
Poor medication adherence is a well-known problem in cystic fibrosis treatment, and has been linked to poorer health outcomes, but has not been well studied. Results from a recent pilot study published in BMC Pulmonary Medicine suggest that a high-quality randomized controlled trial combining real-time data collection of medication adherence with patient feedback and behavior change techniques is feasible, and could inform how to effectively raise adherence in these patients.
“The findings reported in this paper give high levels of confidence that, with improved trial procedures, a full-scale trial is feasible,” Daniel Hind, PhD, of the Clinical Trials Research Unit at the University of Sheffield in the United Kingdom, and colleagues said.
The researchers recruited 430 people with cystic fibrosis (PWCF) from 2 CF units between June and September 2016; of 135 eligible patients, 33 participants were assigned to the intervention group and 31 patients to the control group.
Patients in both the intervention and the control groups received nebulizer devices that recorded and transferred timestamped inhalation data to a secure server where it could be accessed by both patients and researchers through a portal.
“The devices could not distinguish between different drugs administered through the nebuliser; whilst it is theoretically possible for patients to press different buttons depending on the drug, our experience is that any additional step in nebuliser therapy decreases the probability that it will be completed,” the researchers said.
Therapists were trained through a 2-day, face-to-face workshop, online learning modules, and a structured 4-week training program, with a competency assessment of the intervention delivery within the first 5 sessions.
Dr. Hind and colleagues collected data on exacerbations, percent change in medication adherence, and percent predicted forced expiratory volume in 1 second (FEV1).
At the end of the study, exacerbation data was available for 60 participants, percent predicted FEV1 for 57 participants, and medication adherence data for 48 participants, with 2 patients dying during the study period, 2 patients lost to follow-up, 2 patients withdrawing consent for adherence data collection and 1 patient withdrawing consent for research.
The researchers found a 10% difference in adherence between groups favoring the intervention group (95% confidence interval, -5.2 to 25.2), and a between-group difference of 5% in percent predicted FEV1 (95% CI, -2 to 12%).
Overall, there were 8 adverse events, with 7 participants having at least one adverse events. There were also five serious adverse events, with 3 participants (9%) in the intervention group and 2 participants (7%) in the control group. The control group serious adverse events were deaths unrelated to the study, while the intervention group experienced two hospitalizations and 1 event characterized as a “persistent or significant disability or incapacity.”
The researchers concluded by noting that there is poor evidence that patient access to data improves health outcomes or is cost effective, lacking “information about context and implementation.” Dr. Hind and colleagues noted a full-scale version of their RCT, which is underway, will aid in developing theory and practice guidelines on how to improve patient care and use resources more efficiently.
Source: Hind D, Drabble SJ, Arden MA, et al. Supporting medication adherence for adults with cystic fibrosis: a randomised feasibility study. BMC Pulm Med. 2019;19:77.