背景:阵发性睡眠性血红蛋白尿(PNH)是一种罕见的,慢性血液病.疲劳等症状会对患者的身体活动水平产生重大影响,睡眠,生活质量,和工作效率。Ravulizumab治疗可以降低血栓形成的风险,提高生存和生活质量,减少PNH的疲劳,但是关于它如何影响睡眠和身体活动的信息是有限的。这里,静息心率数据,日常体力活动,通过数字可穿戴式活动跟踪设备被动收集接受ravulizumab治疗的PNH患者的睡眠,并通过每周调查收集同一队列中的患者报告结局(PRO)数据.
方法:REVEAL是一项为期32周的前瞻性观察性队列研究,对象是在美国接受ravulizumab的PNH患者。手腕佩戴的Fitbit™收集了静息心率数据,每日步数,和符合条件的患者的睡眠持续时间。患者还完成了以下每周电子调查:慢性疾病治疗的功能评估(FACIT)-疲劳,患者报告结果测量信息系统(PROMIS)全球身体健康,PROMIS全球心理健康,PROMIS睡眠相关损害和睡眠障碍,以及工作生产力和活动障碍问卷-特定健康问题(WPAI-SHP)。从活动追踪器和调查中收集的数据与文献中报告的美国一般人群值进行了比较。
结果:纳入了28名用ravulizumab治疗的患者(中位年龄:34岁;54%为女性)。PRO分数在美国一般人口标准值范围内,包括FACIT-疲劳(40.0),PROMIS全球身体健康(51.0),全球心理健康(51.0)睡眠相关损害(50.0),睡眠障碍(49.0)。同样,平均静息心率(67bpm),每日步数(7476),和睡眠持续时间(7.7h)在美国一般人群值范围内。每日步数与PROMIS全球身心健康得分呈正相关。
结论:这是第一项使用数字监测技术收集PNH患者身体活动和睡眠数据的研究。研究结果表明,ravulizumab治疗使PNH患者能够达到活动水平(心率,睡眠持续时间,步数)和生活质量与美国普通人群相当。患者报告的身心健康与日常身体活动水平之间存在弱正相关。
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients\' physical activity levels, sleep, quality of life, and work productivity. Ravulizumab treatment can reduce thrombosis risk, improve survival and quality of life, and reduce fatigue in PNH, but information is limited on how it impacts sleep and physical activity. Here, data on resting heart rate, daily physical activity, and sleep in ravulizumab-treated patients with PNH were passively collected via a digital wearable activity-tracking device and patient-reported outcome (PRO) data were collected via weekly surveys in the same cohort.
METHODS: REVEAL was a 32-week prospective observational cohort study in individuals with PNH receiving ravulizumab in the USA. A wrist-worn Fitbit™ collected data on resting heart rate, daily step count, and sleep duration from eligible patients. Patients also completed the following electronic weekly surveys: Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health, PROMIS Global Mental Health, PROMIS Sleep-Related Impairment and Sleep Disturbance, and Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP). Data collected from the activity trackers and surveys were compared against US general population values reported in the literature.
RESULTS: Twenty-eight ravulizumab-treated patients were included (median age: 34 years; 54% female). PRO scores were within US general population normative values, including FACIT-Fatigue (40.0), PROMIS Global Physical Health (51.0), Global Mental Health (51.0), Sleep-Related Impairment (50.0), and Sleep Disturbance (49.0). Similarly, mean resting heart rate (67 bpm), daily step count (7476), and sleep duration (7.7 h) were within the range of US general population values. Daily step count was positively correlated with PROMIS Global Physical and Mental Health scores.
CONCLUSIONS: This was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH. The findings indicate that ravulizumab treatment enables patients with PNH to achieve activity levels (heart rate, sleep duration, step count) and quality of life that are comparable to those of the US general population. A weak positive correlation was identified between patient-reported physical and mental health and daily physical activity levels.