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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
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172