Table 1.
Caregiver and patient demographics
| Total (N = 34) | US (n = 10) | Germany (n = 6) | UK (n = 5) | FR (n = 10) | ||
|---|---|---|---|---|---|---|
| Caregivers | ||||||
| Gender, female | 28 (82.4) | 8 (80) | 4 (66.7) | 5 (100) | 9 (90) | |
| Age | ||||||
| 20–29 years | 5 (14.7) | 1 (10) | 1 (16.7) | 1 (20) | 1 (10) | |
| 30–39 years | 11 (32.4) | 5 (50) | 1 (16.7) | 2 (40) | 3 (30) | |
| 40–49 years | 11 (32.4) | 2 (20) | 2 (33.3) | 2 (40) | 4 (40) | |
| 50–59 years | 5 (14.7) | 1 (10) | 2 (33.3) | 0 (0) | 1 (10) | |
| 60–69 years | 2 (5.9) | 1 (10) | 0 (0) | 0 (0) | 1 (10) | |
| Relationship status | ||||||
| Married | 25 (73.5) | 1 (10) | 6 (100) | 2 (40) | 7 (70) | |
| Living with a partner | 8 (23.5) | 7 (70) | 0 (0) | 3 (60) | 3 (30) | |
| Single | 1 (2.9) | 2 (20) | 0 (0) | 0 (0) | 0 (0) | |
| Employment status | ||||||
| Full-time | 8 (23.5) | 4 (40) | 2 (33.3) | 1 (20) | 0 (0) | |
| Part-time | 12 (35.3) | 3 (30) | 2 (33.3) | 0 (0) | 6 (60) | |
| Unemployed | 14 (41.2) | 3 (30) | 2 (33.3) | 4 (80) | 4 (40) | |
| Household income* | ||||||
| < 25,000 | 4 (12.5) | 1 (10) | 0 (0) | 2 (40) | 1 (10) | |
| 25,000–49,999 | 11 (34.4) | 4 (40) | 0 (0) | 1 (20) | 6 (60) | |
| 50,000–74,999 | 7 (21.9) | 1 (10) | 2 (50) | 1 (20) | 2 (20) | |
| 75,000–99,999 | 6 (18.8) | 2 (20) | 2 (50) | 0 (0) | 1 (10) | |
| 100,000–149,999 | 4 (12.5) | 2 (20) | 0 (0) | 1 (20) | 0 (0) | |
| MLD Patients | ||||||
| Number of MLD Patients | 35 | 10 | 6 | 5 | 11 | |
| Gender, female | 22 (62.9) | 5 (50) | 5 (83.3) | 5 (100) | 6 (54.5) | |
| Age (years)** | ||||||
| Mean (SD) | 9.9 (7.5) | 10.2 (8.4) | 7.8 (6.2) | 9.0 (4.4) | 9.1 (5.9) | |
| Median (range) | 8.0 (2.3 – 33.3) | 8.3 (3 – 30.3) | 4.6 (3.2 – 18.4) | 6.8 (4.6 – 15.4) | 9.0 (2.3 – 17.0) | |
| Time since MLD Diagnosis (years)** | ||||||
| Mean (SD) | 5.1 (4.4) | 5.1 (4.8) | 2.8 (1.9) | 4.9 (2.9) | 5.1 (4.7) | |
| Median (range) | 3.3 (0.3 – 16.1) | 3.2 (0.8- 16.1) | 2.5 (0.8 -5.9) | 3.9 (1.9- 9.3) | 4.3 (0.3- 12.5) | |
| Time of symptom onset | ||||||
| Late infantile (≤ 30 months old) | 21 (60) | 6 (60) | 4 (66.7) | 3 (60) | 7 (63.6) | |
| Juvenile (between 30 months and 17 years old) | 13 (37.1) | 3 (30) | 2 (33.3) | 2 (40) | 4 (36.4) | |
| Borderline late infantile and juvenile# | 1 (2.9) | 1 (10) | 0 (0) | 0 (0) | 0 (0) | |
*Currencies were USD for US, Euros for Germany, France, Pound Sterling for UK; 2 Germany respondents elected not to share income; **4 deceased individuals with MLD were excluded from calculation; #One individual with MLD was diagnosed by a physician as an unusual case of borderline late infantile and juvenile MLD