Table 1 Demographic characteristics and disease history, stratified by treatment status
Characteristic, n (%) | All (N = 280) | Currently treated | Previously treated with ERT (n = 9) | Treatment naïve (n = 28) | Other (n = 9) | ||
|---|---|---|---|---|---|---|---|
Agalsidase beta (n = 189) | Agalsidase alfa (n = 19) | Migalastat (n = 26) | |||||
Gender | |||||||
Female | 191 (68) | 120 (63) | 10 (53) | 19 (73) | 9 (100) | 27 (96) | 6 (67) |
Male | 88 (31) | 68 (36) | 9 (47) | 7 (27) | 0 (0) | 1 (4) | 3 (33) |
Non-conforming | 1 (0) | 1 (1) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
Age group | |||||||
18−44 years | 124 (44) | 82 (43) | 12 (63) | 9 (35) | 5 (56) | 11 (39) | 5 (56) |
45−64 years | 118 (42) | 82 (43) | 6 (32) | 9 (35) | 3 (33) | 14 (50) | 4 (44) |
≥65 years | 38 (14) | 25 (13) | 1 (5) | 8 (31) | 1 (11) | 3 (11) | 0 (0) |
Education level | |||||||
Received masters or above | 52 (19) | 32 (17) | 1 (5) | 6 (23) | 2 (22) | 9 (32) | 2 (22) |
Finished college | 106 (38) | 77 (41) | 3 (16) | 11 (42) | 3 (33) | 8 (29) | 4 (44) |
Finished trade school | 27 (10) | 19 (10) | 4 (21) | 1 (4) | 1 (11) | 1 (4) | 1 (11) |
Finished high school/received GED | 88 (31) | 56 (30) | 11 (58) | 8 (31) | 3 (33) | 9 (32) | 1 (11) |
Did not finish high school | 7 (3) | 5 (3) | 0 (0) | 0 (0) | 0 (0) | 1 (4) | 1 (11) |
Employment status | |||||||
Employed full- or part-time | 152 (54) | 103 (54) | 11 (58) | 11 (42) | 3 (33) | 20 (71) | 4 (44) |
Retired | 82 (29) | 57 (30) | 3 (16) | 13 (50) | 4 (44) | 2 (7) | 3 (33) |
Not employed or retired | 17 (6) | 10 (5) | 3 (16) | 0 (0) | 1 (11) | 2 (7) | 1 (11) |
Student full- or part-time | 15 (5) | 11 (6) | 2 (11) | 1 (4) | 0 (0) | 1 (4) | 0 (0) |
Stay-at-home household manager | 14 (5) | 8 (4) | 0 (0) | 1 (4) | 1 (11) | 3 (11) | 1 (11) |
Health insurance | |||||||
Commercial/private | 157 (56) | 108 (57) | 10 (53) | 12 (46) | 5 (56) | 18 64) | 4 (44) |
Medicare | 71 (25) | 49 (26) | 3 (16) | 8 (31) | 5 (56) | 3 (11) | 3 (33) |
Medicaid | 29 (10) | 19 (10) | 4 (21) | 3 (12) | 2 (22) | 0 (0) | 1 (11) |
Marketplace | 12 (4) | 9 (5) | 1 (5) | 2 (8) | 0 (0) | 0 (0) | 0 (0) |
Military | 8 (3) | 6 (3) | 0 (0) | 1 (4) | 0 (0) | 1 (4) | 0 (0) |
Other | 27 (10) | 19 (10) | 1 (5) | 2 (8) | 1 (11) | 4 (14) | 0 (0) |
Not insured | 4 (1) | 1 (1) | 0 (0) | 0 (0) | 0 (0) | 2 (7) | 1 (11) |
Classic FD | |||||||
Yes | 181 (65) | 136 (72) | 13 (68) | 10 (38) | 6 (67) | 9 (32) | 7 (78) |
No | 49 (18) | 19 (10) | 5 (26) | 11 (42) | 2 (22) | 11 (39) | 1 (11) |
Not sure | 50 (18) | 34 (18) | 1 (5) | 5 (19) | 1 (11) | 8 (29) | 1 (11) |
Time since FD diagnosis | |||||||
<1 year | 5 (2) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 4 (14) | 1 (11) |
1−2 years | 24 (9) | 15 (8) | 6 (32) | 2 (8) | 0 (0) | 1 (4) | 0 (0) |
3−5 years | 58 (21) | 34 (18) | 8 (42) | 7 (27) | 1 (11) | 7 (25) | 1 (11) |
6−10 years | 49 (18) | 37 (20) | 1 (5) | 5 (19) | 2 (22) | 4 (14) | 0 (0) |
>10 years | 144 (51) | 103 (54) | 4 (21) | 12 (46) | 6 (67) | 12 (43) | 7 (78) |
Physician managing FD | |||||||
Geneticist | 128 (46) | 90 (48) | 3 (16) | 15 (58) | 3 (33) | 13 (46) | 4 (44) |
Nephrologist | 63 (23) | 48 (25) | 5 (26) | 2 (8) | 3 (33) | 4 (14) | 1 (11) |
Primary care/family doctor | 45 (16) | 27 (14) | 5 (26) | 5 (19) | 2 (22) | 3 (11) | 3 (33) |
Cardiologist | 20 (7) | 12 (6) | 5 (26) | 2 (8) | 1 (11) | 0 (0) | 0 (0) |
Other | 10 (4) | 7 (4) | 0 (0) | 1 (4) | 0 (0) | 2 (7) | 0 (0) |
Neurologist | 8 (3) | 5 (3) | 1 (5) | 1 (4) | 0 (0) | 1 (4) | 0 (0) |
No one | 6 (2) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 5 (18) | 1 (11) |