Joly Florence, Schmitt Lisa Anne, Watson Patricia Ann McGee, Pain Emilie, Testa Damien
Health Economics And Outcomes Research, Sanofi, Chilly-Mazarin, France.
Public Affairs and Patient Advocacy, Sanofi Genzyme, Cambridge, MA, United States.
JMIR Form Res. 2022 Jul 22;6(7):e34248. doi: 10.2196/34248.
Cold agglutinin disease (CAD) is a rare disorder, affecting 15% of patients with autoimmune hemolytic anemia. Few studies have assessed CAD symptoms and their impact on daily life, but these studies did not address the patients' perspectives.
The aims of this study were to increase the knowledge about CAD through a patient-centric survey and to gain a better understanding of the burden of this disease.
We conducted an internet-based survey in September 2020 among American patients registered on the CAD Unraveled website and members of the Cold Agglutinin Disease Foundation.
A total of 50 respondents were included in this study. Totally, 90% (45/50) of the patients reported having experienced fatigue. Fatigue was mainly reported on a daily basis, and approximately one-third of these patients (13/45, 29%) said that their fatigue was constant throughout the day. It has also been shown that CAD has a great impact on patients' physical well-being, emotional well-being, social life, and household finances. The disease varies over time, with or without symptoms. A total of 88% (44/50) of the patients reported previous episodes of the increased intensity or sensitivity of their CAD symptoms, with a mean of 4.5 (SD 5.4) episodes reported during the past year. More than half of the patients (27/50, 54%) considered their disease to be moderate or severe, and 42% (21/50) of the study group reported that their symptoms had worsened since the time of diagnosis.
Our study has provided new data on CAD symptoms, particularly data on the importance and type of fatigue and the fluctuation of CAD symptoms.
冷凝集素病(CAD)是一种罕见疾病,在自身免疫性溶血性贫血患者中占15%。很少有研究评估CAD的症状及其对日常生活的影响,但这些研究未涉及患者的观点。
本研究的目的是通过以患者为中心的调查增加对CAD的了解,并更好地理解这种疾病的负担。
2020年9月,我们对在CAD揭秘网站注册的美国患者以及冷凝集素病基金会的成员进行了一项基于互联网的调查。
本研究共纳入50名受访者。总体而言,90%(45/50)的患者报告曾经历过疲劳。疲劳主要是每天都会出现,其中约三分之一的患者(13/45,29%)表示他们一整天都持续感到疲劳。研究还表明,CAD对患者的身体健康、情绪健康、社交生活和家庭财务都有很大影响。这种疾病会随时间变化,有症状或无症状。共有88%(44/50)的患者报告之前有过CAD症状强度增加或敏感度增加的发作,过去一年中平均报告发作4.5次(标准差5.4)。超过一半的患者(27/50,54%)认为他们的疾病为中度或重度,42%(21/50)的研究组患者报告自确诊以来症状有所恶化。
我们的研究提供了关于CAD症状的新数据,特别是关于疲劳的重要性和类型以及CAD症状波动的数据。