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Table 5 Preferences for SMA treatment by sub-groups

From: Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

  SMA types Age groups Currently under gene therapy Currently under Spinraza® (nusinersen) treatment
Type I
(n = 21)
Type II
(n = 48)
Type III
(n = 29)
 ≤ 2 years
(n = 16)
3–17 years
(n = 10)
 ≥ 18 years
(n = 75)
Yes
(n = 10)
No
(n = 91)
Yes
(n = 59)
No
(n = 42)
Level of change in motor function (ref: stable)           
 Better 0.80* 0.41* 0.92* 0.68* 0.95* 0.61* 0.46 0.67* 0.79* 0.45*
Change in breathing function (ref: stable)           
 Better 1.16* 0.75* 0.58* 1.71* − 0.12 0.80* 1.47* 0.75* 0.77* 0.78*
Drug is approved for: (ref: pediatric and adult patients with SMA)           
 Pediatric patients with SMA under 2 years of age − 0.76* − 1.51* − 1.35* − 0.10 − 0.28 − 1.68* 0.30 − 1.44* − 1.33* − 1.16*
Route and frequency of treatments (ref: intrathecal spinal injection—3–6 times per year)           
 Intravenous injection—one-time treatment 0.82* 0.42* 0.44* 1.69* 0.19 0.38* 2.03* 0.39* 0.39* − 0.65*
 Oral—daily medication 0.98* 0.82* 0.76* 1.01* 0.33 0.92* 0.65 0.87* 0.87* 0.05
Treatments potential risk (ref: mild risk)           
 Moderate risk − 0.73* − 0.81* − 0.70* − 0.79* − 0.68 − 0.84* − 0.34 − 0.84* − 0.73* − 0.81*
 Serious, or life-threatening risk − 1.91* − 2.11* − 1.43* − 3.03* − 1.55* − 1.85* − 2.83* − 1.85* − 1.61* − 2.22*
  1. *p < 0.05; **p < 0.01; ***p < 0.001