Background: 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.
Objective: 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.
Methods: 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.
Results: 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.
Conclusions: Our study has provided new data on CAD symptoms, particularly data on the importance and type of fatigue and the fluctuation of CAD symptoms.
Keywords: autoimmune diseases; cold agglutinin disease; cold autoimmune hemolytic anemia; fatigue; perception; rare diseases; surveys and questionnaires.
©Florence Joly, Lisa Anne Schmitt, Patricia Ann McGee Watson, Emilie Pain, Damien Testa. Originally published in JMIR Formative Research (https://formative.jmir.org), 22.07.2022.