Patient-reported outcome measures after 8 weeks of mepolizumab treatment and long-term outcomes in patients with severe asthma: an observational study

Abstract

Background The novel anti-IL-5 drug mepolizumab improves asthma outcomes in the majority but not all patients with severe eosinophilic asthma. Currently it is difficult to predict an individuals' chance of being a responder. Early changes in patient-reported outcome measures may contribute to the prediction of long-term outcomes. Aim To compare early changes in patient-reported outcome measures after 8 weeks and long-term response to mepolizumab treatment. Method 22 severe eosinophilic asthma patients starting mepolizumab therapy in a severe asthma centre in the Netherlands were evaluated on baseline, 8 weeks and 52 weeks, collecting questionnaire scores and asthma-related parameters. Well-controlled asthma was defined as an asthma control questionnaire score ≤ 0.75. Long-term treatment response was defined as continuing mepolizumab therapy at 52 weeks. Results Nine patients (41%) had well-controlled asthma at 8 weeks and all were mepolizumab responders at 52 weeks (positive predictive value = 100%, 95%CI 66-100), versus only 5 responders out of 13 patients with not well-controlled asthma at 8 weeks (negative predictive value = 62%, 95%CI 32-86). Conclusion The results in this study suggest that patients receiving mepolizumab therapy with an ACQ-score ≤ 0.75 at 8 weeks are unlikely to need extensive monitoring, for they are very likely to be long-term responders. KeywordsSevere asthma • Mepolizumab • Biologics • Patient-reported outcome measures • Personalized medicine • Treatment evaluation Impacts on practice• Therapy evaluation of mepolizumab treatment based on PROMs could be expedited to as early as 8 weeks after treatment initiation. • The findings in this study suggest the selection of patients requiring less extensive follow-up after initiating mepolizumab treatment.

Clinical response to benralizumab can be predicted by combining clinical outcomes at 3 months with baseline characteristics

“…[13,16] Next to baseline characteristics, early treatment effects may contribute to the prediction of long-term outcomes, as shown by a few studies that focused on predicting future asthma exacerbations or therapy response. [17][18][19] Whether the prediction of long-term response to benralizumab improves with the addition of early treatment outcomes to baseline characteristics is not yet known. Therefore, we assessed the effects of benralizumab treatment using real-world patient data from the Dutch Severe Asthma Registry RAPSODI.…”

“…Certain baseline characteristics, such as higher exacerbation frequency or higher blood eosinophil counts, are associated with more favourable benralizumab-induced outcomes, but it remains difficult to predict an individuals’ probability of being a responder [ 13 , 16 ]. Next to baseline characteristics, early treatment effects may contribute to the prediction of long-term outcomes, as shown by a few studies that focused on predicting future asthma exacerbations or therapy response [ 17 – 19 ]. Whether the prediction of long-term response to benralizumab improves with the addition of early treatment outcomes to baseline characteristics is not yet known.…”