Wray Sibyl, Jacques Francois, Miller Tamara A, Nicholas Jacqueline A, Arroyo Rafael, Travis Lori, Khatri Bhupendra, Chirieac Magdalena, Gandhi Roopali, Roesch Nora, Rodrigues Amelie, Melas-Melt Lydie, Rawlings Andreea M, Hunter Samuel F
Hope Neurology, Knoxville, TN, USA.
Clinique Neuro-Outaouais, Gatineau, Canada.
Mult Scler J Exp Transl Clin. 2022 Nov 15;8(4):20552173221135888. doi: 10.1177/20552173221135888. eCollection 2022 Oct-Dec.
Patient-reported outcomes are increasingly used in the management of patients with multiple sclerosis to understand the patient's perspective of disease and treatment. These measures provide insights into important factors including treatment satisfaction, physical and psychological function, and quality of life.
To present results from the real-world PRO-ACT study in patients with multiple sclerosis who switched to alemtuzumab from another disease-modifying therapy.
This 24-month, prospective, multicenter, observational study had a primary endpoint of change in overall satisfaction, measured using the Treatment Satisfaction Questionnaire for Medication (TSQM) version 1.4. Secondary endpoints included the Multiple Sclerosis Impact Scale-29 (MSIS-29), Modified Fatigue Impact Scale-5 (MFIS-5), and the Patient-Determined Disease Steps (PDDS). Safety was monitored with adverse events (AEs).
Of 199 enrolled patients, improvements were observed in mean TSQM scores for overall satisfaction (baseline, 50.3; year 2, + 13.2; 0.0001), effectiveness (49.3 and + 12.2; 0.0001), and side effects (77.6 and + 4.5; 0.04). Improvements were also observed in MSIS-29 physical (52.4 and -6.0; 0.0001), MSIS-29 psychological (53.4 and -7.0; 0.0003), and MFIS-5 (12.8 and -1.7; 0.0001). Most (95.0%) patients experienced ≥ 1 AE (88.4% mild, 67.8% moderate).
The primary endpoint was met; the safety of alemtuzumab was consistent with pivotal studies.
患者报告的结局越来越多地用于多发性硬化症患者的管理中，以了解患者对疾病和治疗的看法。这些措施有助于深入了解包括治疗满意度、身体和心理功能以及生活质量等重要因素。
展示在从另一种疾病修饰疗法改用阿仑单抗的多发性硬化症患者中进行的真实世界PRO-ACT研究的结果。
这项为期24个月的前瞻性多中心观察性研究的主要终点是使用药物治疗满意度问卷（TSQM）1.4版测量的总体满意度变化。次要终点包括多发性硬化症影响量表-29（MSIS-29）、改良疲劳影响量表-5（MFIS-5）和患者确定的疾病阶段（PDDS）。通过不良事件（AE）监测安全性。
在199名入组患者中，总体满意度的平均TSQM评分有所改善（基线时为50.3；第2年为+13.2；P<0.0001），有效性（49.3和+12.2；P<0.0001）和副作用（77.6和+4.5；P=0.04）。MSIS-29身体方面（52.4和-6.0；P<0.0001）、MSIS-29心理方面（53.4和-7.0；P<0.0003）和MFIS-5（12.8和-1.7；P<0.0001）也有改善。大多数（95.0%）患者经历了≥1次不良事件（88.4%为轻度，67.8%为中度）。
达到了主要终点；阿仑单抗的安全性与关键研究一致。
