Table 1.
Characteristics of participants (n = 18, homemade self-reported questionnaire).
| All parents | Total n = 18 | Non-hesitant n = 11 | Hesitant n = 7 | GT-treated n = 6 | Other therapy n = 12 |
|---|---|---|---|---|---|
| Gender of parent | |||||
| Male | 6 (33.3%) | 3 (27.3%) | 3 (42.9%) | 1 (16.7%) | 5 (41.7%) |
| Female | 12 (66.7%) | 8 (72.7%) | 4 (57.1%) | 5 (83.3%) | 7 (58.3%) |
| Age (years) | 34.7 (±5.2) | 32.6 (±5.1) | 38.0 (±3.3) | 32.8 (±6.8) | 35.7 (±4.1) |
| Marital status | |||||
| Married | 15 (83.3%) | 8 (72.7%) | 7 (100%) | 6 (100%) | 9 (75.0%) |
| Separated and in couple | 2 (11.1%) | 2 (18.2%) | - | - | 2 (16.7%) |
| Separated and single | 1 (5.6%) | 1 (9.1%) | - | - | 1 (8.3%) |
| Participation in couple | |||||
| No | 8 (44.4%) | 5 (45.5%) | 3 (42.9%) | 4 (66.7%) | 4 (33.3%) |
| Yes | 10 (55.6%) | 6 (54.5%) | 4 (57.1%) | 2 (33.3%) | 8 (66.7%) |
| Primiparous | |||||
| No | 8 (44.4%) | 2 (18.2%) | 6 (85.7%) | 1 (16.7%) | 7 (53.8%) |
| Yes | 10 (55.6%) | 9 (81.8%) | 1 (14.3%) | 5 (83.3%) | 5 (41.7%) |
| Economic status | |||||
| Poor | 2 (11.1%) | 2 (18.2%) | - | 1 (16.7%) | 1 (8.3%) |
| Popular | 9 (50.0%) | 6 (54.5%) | 3 (42.9%) | 4 (66.7%) | 5 (41.7%) |
| Medium | 5 (27.8%) | 3 (27.3%) | 2 (28.6%) | 1 (16.7%) | 4 (33.3%) |
| Easy | 2 (11.1%) | - | 2 (28.6%) | - | 2 (16.7%) |
| Parent's working | |||||
| No | 5 (27.8%) | 2 (18.2%) | 3 (42.9%) | 2 (33.3%) | 3 (25.0%) |
| Yes | 13 (72.2%) | 9 (81.8%) | 4 (57.1%) | 4 (66.7%) | 9 (75.0%) |
| Social benefits | |||||
| No | 3 (16.7%) | 1 (9.1%) | 2 (28.6%) | 1 (16.7%) | 2 (16.7%) |
| Yes | 15 (83.3%) | 10 (90.9%) | 5 (71.4%) | 5 (83.3%) | 10 (83.3%) |
| Age of child (months) | 44.3 (±38.0) | 46.5 (±48.0) | 40.7 (±15.2) | 25.2 (±3.4) | 53.8 (±44.0) |
| Type of SMA | |||||
| Type 1 | 10 (55.6%) | 7 (63.6%) | 3 (42.9%) | 3 (50.0%) | 7 (58.3%) |
| Type 1 c | 8 (44.4%) | 4 (36.4%) | 4 (57.1%) | 3 (50.0%) | 5 (41.7%) |
| Age of symptoms’ onset (months) | 3.5 (±2.3) | 3.5 (±2.7) | 3.6 (±1.8) | 2.8 (±1.5) | 3.9 (±2.7) |
| Age of diagnostic (months) | 5.4 (±3.2) | 5.5 (±3.7) | 5.3 (±2.4) | 5.3 (±2.5) | 5.5 (±3.6) |
| Treated with innovative therapy | |||||
| Answer no, but it was yes | 2 (11.1%) | 2 (18.2) | - | 2 (33.3%) | - |
| Yes | 16 (88.9%) | 9 (81.8%) | 7 (100%) | 4 (66.7%) | 12 (100%) |
| Type of innovative therapy | |||||
| Gene therapy | 6 (33.3%) | 5 (45.5%) | 1 (14.3%) | - | - |
| Other therapy | 12 (66.7%) | 6 (54.5%) | 6 (85.7%) | - | - |
| Time from research participation to start of treatment (days) | 813.6 (±455.2) | 666.8 (±375.9) | 1002.3 (±505.7) | 586.0 (±118.6) | 889.4 (±503.6) |