The effect of volanesorsen treatment on the burden associated with familial chylomicronemia syndrome: the results of the ReFOCUS study

Figures & data

Table 1. Baseline demographics and characteristics.

Patient characteristics	Patients N = 22
Female gender, n (%)	16 (73)
Age at time of survey, median (range), years	51 [26–65]
Age at FCS diagnosis, years, median (range)	24 [0–63]
≤10	9 (41)
11–20	1 (5)
21–30	5 (23)
31–40	2 (9)
41–50	1 (5)
51–60	3 (14)
≥61	1 (5)
Country of residence, n (%)
USA	7 (32)
Canada	5 (23)
Italy	4 (18)
UK	3 (14)
Spain	2 (9)
South Africa	1 (5)
Family history of FCS, n (%)	15 (68)
No. of physicians seen before diagnosis, median [range]	3 [1–15] *
1–3	13 (59)
4–6	5 (23)
7–9	0 (0)
10–12	0 (0)
13–15	1 (5)
Unable to recall	3 (14)
Patients with misdiagnoses, n (%)
No	8 (36)
Yes	12 (55)
Unable to recall	2 (9)

Data are shown as n (%), unless indicated otherwise

* Data from N = 19 who were able to recall this information; N = 3 selected ‘I don’t know’.

Figure 1. Approaches used to manage symptoms of FCS.

N = 20 unless otherwise stated. *P < 0.05 vs. baseline. ^aN for PRE = 21; ^bN = 16; ^cN for PRE = 16; ^dImprovement defined as decrease in mean rank rating in past 3 months versus 3 months prior to starting treatment with volanesorsen; exception: ‘My approach to managing…,’ where improvement is defined as an increase in mean rank rating. Medians for the ratings scales pre- and post-treatment are shown with the interquartile ranges (Q₁–Q₃).

Figure 2. Number of symptoms of FCS experienced before and after being treated with volanesorsen.

Figure 3. (A)(B)(C). Effect of volanesorsen treatment on (A) physical symptoms, (B) emotional symptoms, and (C) cognitive symptoms.

Numbers in parentheses indicate the number of patients in the pretreatment period who experienced each symptom. *P < 0.05

Figure 4. Overall impact of FCS on patients’ lives before and after volanesorsen treatment (N = 22).

Figure 5. Interference of FCS with work/school attendance and responsibilities before and after treatment with volanesorsen (N = 22).

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