Curlee Millicent S, Toledo-Tamula Mary Anne, Baker Melissa, Wikstrom Daniel, Harrison Cynthia, Rhodes Amanda, Fagan Margaret, Tibery Cecilia, Wolters Pamela L, Widemann Brigitte C, Gross Andrea M, Martin Staci
Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, 9030 Old Georgetown Road, Bethesda, MD 20892-8200, USA.
Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD 21702, USA.
Cancers (Basel). 2025 Jan 17;17(2):295. doi: 10.3390/cancers17020295.
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.
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), 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.
The predetermined criterion was met, suggesting that using MEMS 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.
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.
1型神经纤维瘤病（NF1）患者丛状神经纤维瘤（PNs）的口服治疗方案正在临床研究中受到关注。MEK抑制剂（MEKi）司美替尼已获美国食品药品监督管理局（FDA）批准，用于治疗2岁及以上患有无法手术切除的PNs的儿童，并在成人中显示出活性。长期使用司美替尼治疗对于维持肿瘤缩小是必要的。因此，研究长期依从性对于了解随时间变化的依从模式及其对临床结果的影响至关重要。混合方法研究提供了有关依从性的丰富信息，可为未来的干预试验提供参考，并可帮助从业者解决药物依从性问题。
这项混合方法的试点研究首次检验了基于技术的依从性评估方法——药物事件监测系统（MEMS）在NF1-PN患者中的可行性。在12名患有NF1和PN的患者（平均年龄 = 34.36岁；58%为男性）的小样本中，跨越18个28天的治疗周期监测依从性。通过使用归纳和演绎技术进行主题分析，从个体访谈中获得定性数据。
达到了预定标准，表明尽管在药帽方面存在一些挑战，但使用MEMS是可行的。抑郁和总体压力与依从性降低显著相关，不过这些结果应被视为生成假设。药物依从性的障碍包括遗忘以及与饮食相关的服药时间。促进因素包括一致性、提醒和社会支持。
本研究突出了可能与不依从风险增加相关的患者特征，以及电子药帽方面的挑战，这些在未来针对NF1相关PN的临床试验中应予以考虑。研究结果可为未来针对患有NF1和PN的成人的依从性干预提供参考。需要进行更大样本的未来研究，以充分探索与NF1患者长期药物依从性相关的因素。
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