Rizio Avery A, White Michelle K, McCausland Kristen L, Quock Tiffany P, Guthrie Spencer D, Yokota Miyo, Bayliss Martha S
Scientist, Optum, Johnston, RI.
Senior Scientist, Optum.
Am Health Drug Benefits. 2018 Nov;11(8):430-437.
Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment.
To explore the prevalence of AEs and treatment tolerability problems as reported by patients who received therapies for AL amyloidosis, and to examine the effects of AEs on treatment continuation and on health-related quality of life (HRQOL).
Patients with AL amyloidosis were recruited for this noninterventional, longitudinal, online survey. The patients responded to survey items regarding demographics, disease characteristics, most recent AL amyloidosis treatment, and HRQOL. The study analyses are based on data collected during the 6-month follow-up survey and are restricted to patients who completed the baseline and 6-month surveys and received treatment for AL amyloidosis within 6 months before the follow-up survey.
A total of 100 patients met the inclusion criteria and were included in the study. The patients self-reported having a variety of AEs, which ranged in severity. Overall, 69.4% of patients had problems tolerating their treatment in the past 6 months, of whom 22% discontinued at least 1 therapy. In addition, approximately 33% of patients reduced their AL amyloidosis treatment because of AEs. Most often reported AEs included fatigue (83%), shortness of breath (53%), nausea (52%), and diarrhea (51%). Overall, 50% of the patients reported that their treatment was moderately well-tolerated and 41% said it was very well-tolerated. Those whose treatment was not well-tolerated had significantly worse HRQOL than patients whose treatment was well-tolerated.
Patient-reported experiences should be considered by clinicians when making treatment-related decisions. More research is needed to explore additional factors that may contribute to treatment discontinuation in patients with AL amyloidosis.
免疫球蛋白轻链淀粉样变性（AL淀粉样变性）是一种罕见且往往致命的疾病，目前美国食品药品监督管理局或欧洲药品管理局尚未批准任何治疗方法。治疗方案通常基于多发性骨髓瘤的疗法且为非适应证用药，与大量不良事件（AE）相关。由于AE的严重程度通常由临床医生判定，对治疗耐受性的评估可能无法充分考虑患者自身的治疗体验。
探讨接受AL淀粉样变性治疗的患者报告的AE患病率和治疗耐受性问题，并研究AE对治疗持续进行及健康相关生活质量（HRQOL）的影响。
招募AL淀粉样变性患者参与这项非干预性、纵向在线调查。患者回答有关人口统计学、疾病特征、最近的AL淀粉样变性治疗及HRQOL的调查项目。研究分析基于6个月随访调查期间收集的数据，且仅限于完成基线和6个月调查并在随访调查前6个月内接受过AL淀粉样变性治疗的患者。
共有100名患者符合纳入标准并被纳入研究。患者自我报告出现了各种严重程度不一的AE。总体而言，69.4%的患者在过去6个月中存在治疗耐受性问题，其中22%的患者至少停用了1种疗法。此外，约33%的患者因AE减少了AL淀粉样变性治疗。最常报告的AE包括疲劳（83%）、呼吸急促（53%）、恶心（52%）和腹泻（51%）。总体而言，50%的患者报告其治疗耐受性中等良好，41%的患者表示耐受性非常良好。治疗耐受性不佳的患者的HRQOL明显差于治疗耐受性良好的患者。
临床医生在做出与治疗相关的决策时应考虑患者报告的体验。需要更多研究来探索可能导致AL淀粉样变性患者停药的其他因素。
