Asking patients what they want could improve adherence and help close the gap between research and clinical practice
When asked about their preferred attributes of an asthma treatment regimen, patients said having no shortness of breath was their most important goal, followed by a reduced risk of flare-ups, a study has shown.
The study, published in Thorax, is the first of its kind to compare a regular versus an as-needed preventer regimen, which the research team – led by the Medical Research Institute of New Zealand – said was particularly relevant given the inclusion of symptom-driven ICS formoterol in the 2019 GINA update.
The cohort of 300 patients with mild-moderate disease was drawn from participants due for their final visit on or after 26 March 2018 in the PRACTICAL study, where they were randomised to receive either budesonide-formoterol reliever therapy or maintenance budesonide plus terbutaline reliever therapy.
About two-thirds (64%) of the 288 patients reported preference for the as-needed combination preventer and reliever therapy over twice-daily preventer and as-needed reliever therapy.
Based on their preferred treatment, they were then given one of two versions of a discrete choice experiment (DCE).
This involved repeatedly answering a series of “trade-off” questions, choosing their preferred treatment from a pair of hypothetical treatments defined on just two attributes each time (including treatment regimen, shortness of breath in an average week, steroid dose, and likelihood of asthma flare-up severe enough that they needed to see a doctor).
Both groups ranked no shortness of breath as the most important attribute of therapy.
The lowest “likelihood of an asthma flare-up”, defined as five out of 100 people having a flare-up in a year, was ranked next highest by both groups.
However, the authors noted that aside from shortness of breath, the importance of the treatment attributes was influenced by whether the patient preferred symptom-driven or maintenance-inhaled corticosteroids.
“Though a lower likelihood of an asthma flare-up was ranked second in both DCEs, for those who preferred combined treatment taken as needed, this regimen and a lower likelihood of an asthma flare-up had similar preference weights, indicating that they were of similar importance,” they wrote in Thorax.
“A very low ‘dose of steroid’ was the lowest-ranked attribute, indicating that it was the least important. In contrast, for participants who preferred maintenance treatment two times a day, this regimen and very low ‘dose of steroid’ were of similar weight and the least important attributes.”
Lead author Dr Christina Baggott told The Medical Republic that while a desire to avoid breathlessness was consistent with previous studies, understanding of patient priorities was particularly helpful given the expanding treatment options available.
“Knowledge of these priorities, together with knowledge the clinical outcomes associated with the different regimens from clinical trials can be used to help choose the most appropriate regimen for the patient,” said Dr Baggott, a respiratory specialist at MRINZ.
“For example, if a patient is very motivated by avoidance of asthma exacerbations then data from the PRACTICAL study would suggest that budesonide-formoterol reliever therapy would be most appropriate for them, and the data from the SYGMA 1&2 and Novel START studies would suggest that this regimen is non-inferior to daily maintenance preventer.”
There is currently little research on how patients view symptom-driven preventer-reliever regimens, but the demonstrated reduction in likelihood of flare-ups with budesonide–formoterol reliever therapy is an important aspect for discussion with patients, Dr Baggott added.
“Our study should reassure clinicians that budesonide-formoterol reliever therapy will be acceptable to patients, as in those who preferred this regimen it was almost as important to them as their risk of an asthma flare-up.
“Many asthma flare-ups or exacerbations could be prevented if patients took their preventer inhalers with the same enthusiasm that they take their SABAs.”
Co-author Dr James Fingleton said it made sense to try to match the regimen to patients’ preferences provided the outcomes would be similar between approaches, and he hoped to see more studies investigating patient preferences alongside clinical trials.
“Whilst we have to be careful about extrapolating from trial participants to the general population, participants have an insight into the experience of being on a medication that goes beyond our normal trial outcome measures,” said Dr Fingleton, the Asthma and COPD program director at MRINZ.
“Historically I think as researchers we have not asked about that experience and patient preferences enough and this knowledge gap probably contributes to the gap between the benefit from a medication in a clinical trial (efficacy) and its benefit in clinical practice (effectiveness), which will at least in part be due to low patient adherence.”
