Ali Juzar, Wu Jasmanda, Hassan Mariam, Tsai Jui-Hua, Touba Nancy, McCarrier Kelly, Ballard Mark, Chatterjee Anjan
Louisiana State University Health Sciences Center-New Orleans (LSU HSC), New Orleans, LA, USA.
Insmed Incorporated, 700 US Highway 202/206, Bridgewater, NJ, USA.
Pulm Ther. 2024 Sep;10(3):315-330. doi: 10.1007/s41030-024-00263-1. Epub 2024 Jul 16.
This study aimed to gain insight from patients with refractory Mycobacterium avium complex lung disease (MAC-LD) into strategies used to manage adverse events (AEs) associated with amikacin liposome inhalation suspension (ALIS).
We conducted semi-structured interviews with US patients with refractory MAC-LD prescribed ALIS in a real-world setting. Interview transcripts were analyzed and coded to identify patterns in participants' descriptions of their ALIS treatment experiences, including AEs and their disruptiveness, and AE mitigation strategies, including participants' ratings of strategies' effectiveness. Concept saturation was also assessed.
Twenty participants (mean age 48.7 years; 80% women; mean ALIS duration 5.45 months) were interviewed. At the time of the interview, 15 participants (75%) had received ALIS for > 1 month and 13 (65%) were currently receiving ALIS. Participants described 44 unique AE mitigation strategies, which can be categorized into three groups: prepare for treatment; prevent increased emergence of AEs; and persist on treatment by mitigating AEs. Common strategies (reported by ≥ 50% of participants) included use of educational materials from the patient support program, localized management of throat irritation, and symptom management to reduce fatigue. Evidence of concept saturation was observed: no new strategies were identified in the last five interviews, which suggests the sample was robust enough to identify all mitigation strategies likely to be used by the broader patient population.
This real-world study identified a diverse set of potential AE mitigation strategies intended to help individual patients prepare for ALIS treatment, prevent the increased emergence of certain AEs, and mitigate the impact of AEs on treatment persistence. Developing a comprehensive accounting of the types of mitigation strategies in use among patients in real-world settings can inform future investigation of the effectiveness of such strategies, and support evidence-based recommendations for treatment management.
本研究旨在从难治性鸟分枝杆菌复合群肺病（MAC-LD）患者中了解用于管理与阿米卡星脂质体吸入混悬液（ALIS）相关不良事件（AE）的策略。
我们在现实环境中对美国开具ALIS处方的难治性MAC-LD患者进行了半结构化访谈。对访谈记录进行分析和编码，以识别参与者对其ALIS治疗经历的描述模式，包括不良事件及其干扰性，以及不良事件缓解策略，包括参与者对策略有效性的评分。还评估了概念饱和度。
共访谈了20名参与者（平均年龄48.7岁；80%为女性；平均ALIS治疗时长5.45个月）。在访谈时，15名参与者（75%）接受ALIS治疗超过1个月，13名（65%）目前正在接受ALIS治疗。参与者描述了44种独特的不良事件缓解策略，可分为三组：为治疗做准备；预防不良事件增加出现；通过缓解不良事件坚持治疗。常见策略（≥50%的参与者报告）包括使用患者支持项目的教育材料、局部处理咽喉刺激以及进行症状管理以减轻疲劳。观察到概念饱和的证据：在最后五次访谈中未发现新策略，这表明该样本足够强大，能够识别更广泛患者群体可能使用的所有缓解策略。
这项现实世界研究确定了一系列不同的潜在不良事件缓解策略，旨在帮助个体患者为ALIS治疗做准备、预防某些不良事件增加出现，并减轻不良事件对治疗持续性的影响。全面了解现实环境中患者使用的缓解策略类型，可为未来此类策略有效性的研究提供信息，并支持基于证据的治疗管理建议。
