Table 4.
Care for individuals with SMA: providers and caretakers, among living individuals with SMA types I, II, and III*
| Characteristics | 2018 Survey (living individuals) | 2017 Survey (living individuals) | ||||
| Type I | Type II | Type III | Type I | Type II | Type III | |
| (n = 157) | (n = 275) | (n = 171) | (n = 105) | (n = 265) | (n = 187) | |
| Number of specialists | ||||||
| Mean (SD) | 4.1 (1.1) | 4.9 (3.3) | 3.2 (2.9) | 3.7 (4.2) | 4.7 (3.0) | 3.0 (2.7) |
| Median (range) | 4 (0–14) | 5 (0–15) | 2 (0–16) | 1 (0–13) | 5 (0–11) | 2 (0–10) |
| Types of specialists seen, n (%) | ||||||
| Neurologist | 135 (87.1) | 233 (92.5) | 141 (91.6) | 89 (82.4) | 202 (81.8) | 137 (86.7) |
| Pulmonologist | 143 (92.3) | 217 (86.1) | 67 (43.5) | 100 (92.6) | 198 (80.2) | 64 (40.5) |
| Rehab medicine/physiatrist | 40 (25.8) | 66 (26.2) | 25 (16.3) | 30 (27.8) | 60 (24.3) | 33 (20.9) |
| Geneticist | 44 (28.4) | 51 (20.2) | 32 (20.8) | 28 (25.9) | 28 (11.3) | 26 (16.5) |
| Orthopedist | 100 (64.5) | 151 (59.9) | 39 (25.3) | 72 (66.7) | 146 (59.1) | 54 (34.2) |
| Cardiologist | 40 (25.8) | 49 (19.4) | 23 (14.9) | 39 (36.1) | 46 (18.6) | 21 (13.3) |
| Speech therapist | 93 (60.0) | 50 (19.8) | 5 (3.3) | 70 (64.8) | 31 (12.6) | 9 (5.7) |
| Occupational therapists | 98 (63.2) | 136 (54.0) | 30 (19.5) | 69 (63.9) | 119 (48.2) | 41 (26.0) |
| Nutritionist | 103 (66.5) | 100 (39.7) | 34 (22.1) | 76 (70.4) | 90 (36.4) | 30 (19.0) |
| Orthotist | 49 (31.6) | 67 (26.6) | 23 (14.9) | 35 (32.4) | 61 (24.7) | 18 (11.4) |
| Vocational rehabilitation counselor | 4 (2.6) | 21 (8.3) | 6 (3.9) | 3 (2.8) | 12 (4.9) | 7 (4.4) |
| Psychologist/therapist | 7 (4.5) | 30 (11.9) | 18 (11.7) | 9 (8.3) | 22 (8.9) | 14 (8.9) |
| Nephrologist | 17 (11.0) | 10 (4.0) | 7 (4.6) | 7 (6.5) | 11 (4.5) | 2 (1.3) |
| Social worker | 57 (36.8) | 53 (21.0) | 21 (13.6) | 49 (45.4) | 50 (20.2) | 20 (12.7) |
| Physical therapist | 125 (80.7) | 180 (71.4) | 86 (55.8) | 84 (77.8) | 169 (68.4) | 78 (49.4) |
| Palliative care | 32 (20.7) | 12 (4.8) | 5 (3.3) | 28 (25.9) | 11 (4.5) | 2 (1.3) |
| Have a caregiver/caretaker, n (%) | 138 (86.8) | 240 (87.3) | 128 (74.4) | 99 (90.8) | 244 (91.7) | 155 (82.9) |
| Of those with caregiver, caregiver is family member, n (%) | 137 (99.3) | 223 (92.9) | 100 (78.1) | 99 (100.0) | 227 (93.0) | 128 (82.1) |
| Of those with caregiver, non-family/paid caretaker, n (%) | 54 (39.1) | 114 (47.5) | 56 (43.8) | 25 (25.3) | 116 (47.4) | 76 (48.7) |
| Of those with a non-family/paid caretaker, number hours per week, n (%) | ||||||
| 0–20 | 7 (13.0) | 34 (29.8) | 8 (14.3) | 2 (8.0) | 33 (28.5) | 30 (39.5) |
| 21–40 | 14 (25.9) | 18 (15.8) | 13 (23.2) | 7 (28.0) | 26 (22.4) | 17 (22.4) |
| ≥40 | 18 (33.3) | 43 (37.7) | 6 (10.7) | 10 (40.0) | 40 (34.5) | 12 (15.8) |
| Missing | 15 (27.8) | 19 (16.7) | 29 (51.8) | 6 (24.0) | 17 (14.7) | 17 (22.4) |
| Nusinersen | ||||||
| Yes, I have been treated† | 127 (80.9) | 148 (53.8) | 87 (50.9) | – | – | – |
| No, never been treated | 21 (13.4) | 114 (41.5) | 78 (45.6) | – | – | – |
| Don’t know | 0 | 1 (0.4) | 0 | – | – | – |
| Missing | 9 (5.7) | 12 (4.4) | 6 (3.5) | – | – | – |
SD, standard deviation; SMA, spinal muscular atrophy. *Questions were not asked of caregivers of deceased individuals. †Includes those who have been treated commercially and/or in a clinical trial.