Prior research has shown that in serious illnesses including breast cancer and HIV, being connected to disease and patient advocacy organizations and other support groups can have a positive effect on patients. However, limited data are available about the potential benefits of patient connectivity for people living with rare diseases, including the rare lipid disorder familial chylomicronemia syndrome (FCS), which affects an estimated 3000 to 5000 people worldwide.
FCS is characterized by extremely high triglycerides that can cause painful, debilitating, and potentially fatal attacks of acute pancreatitis. There are currently no approved treatment options available for patients. Because of its rarity and the fact that symptoms are often associated with other more well-known diseases, delayed diagnosis is common. Also, historically, opportunities for patients and caregivers to connect with others affected by FCS either in person or online have been extremely limited, despite the growing FCS community in recent years. Lack of awareness and connectivity can, in turn, promote feelings of isolation and a lack of support that can be devastating.
To better understand the potential benefits and effect of patient-to-patient connectivity for people living with FCS, I worked with the team at Akcea Therapeutics to collect a range of insights from both patients with FCS and their caregivers in the United States and Canada in an effort known as the CONNECT study. Participants answered a series of questions related to their level of connectivity with FCS-focused organizations, providing many first-time findings that were published in July 2018 in the peer-reviewed journal Expert Opinion on Orphan Drugs.
A total of 50 respondents self-identified as either actively or passively connected or not connected to any patient organizations or communities. Responses showed conclusively that for patients with FCS, any level of connection with other patients provided a number of positive benefits. Among the findings, connected respondents reported a significant increase in positive perceptions of their overall health, disease outlook, and emotional well-being after connecting with other patients compared with the period before making these connections. Patients who were actively connected (regularly taking part in ongoing conversations with 1 or more groups) were also 3 times as likely to report “high” or “extremely high” motivation in managing their own health. As levels of connection increase, patients are also more likely to report higher levels of satisfaction with their primary treating physician.
There are a growing number of resources that clinicians including cardiologists and lipidologists can recommend to their patients with FCS who might be looking for opportunities to connect with other patients, including The FCS Foundation and FCS Focus, as well as in-person meetings and social media channels. These options position all patients to choose the level of engagement that works best for them.
Although there is still a need for an approved treatment, these findings reinforce that efforts to connect both patients and caregivers with others affected by this serious disease should be considered a vital and beneficial element in patient management.
Disclosures: This study was funded by Akcea Therapeutics, Inc. Dr Davidson has received financial support from Sanofi, Regeneron Pharmaceuticals, Amgen, and Akcea Therapeutics.
Salvatore V, Gilstrap A, Williams R, et al. Evaluating the impact of peer support and connection on the quality of life of patients with familial chylomicronemia syndrome [published online August 5, 2018]. Expert Opin Orphan Drugs. doi:10.1080/21678707.2018.1505495