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 (MEMSTM), 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 MEMSTMis 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.
背景：针对1型神经纤维瘤病（NF1）患者丛状神经纤维瘤（PNs）的口服治疗选择在临床研究中备受关注。MEK抑制剂（MEKi）司美替尼已获美国食品药品监督管理局批准用于治疗2岁及以上无法手术的PNs患儿，并在成人患者中显示出疗效。为维持肿瘤缩小效果，需长期使用司美替尼治疗。因此，研究长期用药依从性对于理解随时间变化的依从性模式及其对临床结果的影响至关重要。混合方法研究能提供关于依从性的丰富信息，可为未来干预试验提供参考，并帮助临床医生解决用药依从性问题。 方法：本项混合方法先导研究首次探讨了基于技术的依从性评估方法——药物事件监测系统（MEMSTM）在NF1-PN患者中的可行性。研究对12例NF1伴PN患者（平均年龄34.36岁；58%为男性）进行了18个28天治疗周期的依从性监测。通过个体访谈获取定性数据，采用归纳与演绎技术进行主题分析。 结果：研究达到了预设标准，表明尽管药瓶盖存在一些技术问题，使用MEMSTM仍具可行性。抑郁和整体压力与依从性降低显著相关，但该结果应视为假设性发现。用药依从性障碍包括遗忘和与进食相关的服药时间安排；促进因素则包括规律作息、用药提醒和社会支持。 结论：本研究揭示了可能与NF1-PN患者用药不依从风险增加相关的患者特征，以及电子药瓶盖存在的技术挑战，这些发现应在未来NF1相关PN的临床试验中予以考虑。研究结果可为未来针对成人NF1-PN患者的依从性干预提供参考。未来需要更大样本的研究来全面探索影响NF1患者长期用药依从性的相关因素。
肿瘤(癌症)患者之家