Table 3 Rating of impact of NPC symptoms on ADLs and HRQOL of patients and family/others
From: Impacts and Burden of Niemann pick Type-C: a patient and caregiver perspective
Patient self-reported (n = 6) | Caregiver reported (n = 42)* | |||||||
|---|---|---|---|---|---|---|---|---|
Impact on patients | Impact on family/others | Impact on patients | Impact on family/others | |||||
ADLs | HRQOL | ADLs | HRQOL | ADLs | HRQOL | ADLs | HRQOL | |
Ambulation, n (%) | ||||||||
Not at all | 1 (16.7) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 5 (11.9) | 6 (14.3) | 6 (14.3) | 10 (23.8) |
Somewhat or moderately | 2 (33.3) | 1 (16.7) | 1 (16.7) | 2 (33.3) | 15 (35.7) | 13 (31.0) | 21 (50.0) | 15 (35.7) |
A lot or extremely | 3 (50.0) | 3 (50.0) | 3 (50.0) | 2 (33.3) | 22 (52.4) | 23 (54.8) | 15 (35.7) | 17 (40.5) |
Speech, n (%) | ||||||||
Not at all | 2 (33.3) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 7 (16.7) | 9 (21.4) | 13 (31.0) | 13 (31.0) |
Somewhat or moderately | 1 (16.7) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 20 (47.6) | 17 (40.5) | 22 (52.4) | 22 (52.4) |
A lot or extremely | 3 (50.0) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 14 (33.3) | 15 (35.7) | 6 (14.3) | 6 (14.3) |
Swallow, n (%) | ||||||||
Not at all | 2 (33.3) | 1 (16.7) | 3 (50.0) | 3 (50.0) | 12 (28.6) | 11 (26.2) | 13 (31.0) | 15 (35.7) |
Somewhat or moderately | 3 (50.0) | 4 (66.7) | 2 (33.3) | 2 (33.3) | 9 (21.4) | 12 (28.6) | 20 (47.6) | 15 (35.7) |
A lot or extremely | 1 (16.7) | 1 (16.7) | 1 (16.7) | 1 (16.7) | 19 (45.2) | 17 (40.5) | 8 (19.0) | 10 (23.8) |
Fine motor skills, n (%) | ||||||||
Not at all | 1 (16.7) | 1 (16.7) | 2 (33.3) | 2 (33.3) | 7 (16.7) | 9 (21.4) | 11 (26.2) | 14 (33.3) |
Somewhat or moderately | 3 (50.0) | 4 (66.7) | 3 (50.0) | 3 (50.0) | 19 (45.2) | 16 (38.1) | 21 (50.0) | 19 (45.2) |
A lot or extremely | 2 (33.3) | 1 (16.7) | 1 (16.7) | 1 (16.7) | 15 (35.7) | 16 (38.1) | 9 (21.4) | 8 (19.0) |
Cognition, n (%) | ||||||||
Not at all | 2 (33.3) | 1 (16.7) | 2 (33.3) | 2 (33.3) | 5 (11.9) | 4 (9.5) | 8 (19.0) | 9 (21.4) |
Somewhat or moderately | 4 (66.7) | 5 (83.3) | 4 (66.7) | 4 (66.7) | 13 (31.0) | 14 (33.3) | 17 (40.5) | 19 (45.2) |
A lot or extremely | 0 | 0 | 0 | 0 | 22 (52.4) | 23 (54.8) | 15 (35.7) | 12 (28.6) |