A Mixed Methods Study of Medication Adherence in Adults with Neurofibromatosis Type 1 (NF1) on a Clinical Trial of Selumetinib.

Simple Summary: People with neurofibromatosis type 1 (NF1) may develop tumors called plexiform neurofibromas (PNs). Selumetinib was the first oral medication to gain FDA approval to treat PNs in children, and the drug also has activity in adults. Clinical observations suggest that people must continue taking selumetinib to maintain its effects. Therefore, it is important to research how well people take selumetinib as prescribed over a long period of time. We used electronic pill caps that record when the bottle is opened, pill counts, and self-report diaries to measure adherence over eighteen 28-day treatment cycles. We found that using the caps is feasible but presents some challenges. We also found evidence that depression and stress were related to lower adherence in our small sample. We also interviewed patients, who talked about things that make adherence easier (consistency, reminders, and social support) and more difficult (forgetting and dose timing). Background: Oral therapeutic options for plexiform neurofibromas (PNs) in individuals with neurofibromatosis type 1 (NF1) are receiving attention in clinical research. The MEK inhibitor (MEKi) Selumetinib is FDA-approved in children ages 2+ years with inoperable PNs, and shows activity in adults. Prolonged therapy with selumetinib is necessary to maintain tumor reduction. Therefore, investigating long-term adherence is vital to understand patterns of adherence over time and its impact on clinical outcomes. Mixed methods research offers rich information about adherence that can inform future intervention trials, and can assist practitioners in addressing medication adherence concerns. Methods: This mixed-method pilot study is the first examination of the feasibility of a technology-based adherence assessment method, the medication events monitoring system (MEMS^TM), among individuals with NF1-PN. Adherence was monitored in a small sample of patients (N = 12; mean age = 34.36 years; 58% male) with NF1 and PN across eighteen 28-day treatment cycles. Qualitative data were obtained from individual interviews using inductive and deductive techniques for thematic analysis. Results: The predetermined criterion was met, suggesting that using MEMS^TM is feasible despite some challenges with the caps. Depression and overall stress were significantly related to reduced adherence, although these results should be considered hypothesis-generating. Barriers to medication adherence included forgetting and the timing of doses related to eating. Facilitators included consistency, reminders, and social support. Conclusions: This study highlights patient characteristics that may be related to increased risk for nonadherence, as well as challenges with electronic pill caps that should be considered in future clinical trials for NF1-related PN. Results can inform future adherence interventions for adults with NF1 and PNs. Future research with larger samples is needed to fully explore factors related to long-term medication adherence among individuals with NF1. [ABSTRACT FROM AUTHOR]