Table 3.
Healthcare utilization
| All patients N = 39 |
Lowest n = 9 |
Second Lowest n = 10 |
Third Lowest n = 10 |
Highest n = 10 |
P-value | |
|---|---|---|---|---|---|---|
| PKAN treatments received | ||||||
| Medication from doctor, n (%) | 39 (100%) | 9 (100%) | 10 (100%) | 10 (100%) | 10 (100%) | |
| Vitamins or OTC, n (%) | 35 (89.7%) | 8 (88.9%) | 7 (70.0%) | 10 (100%) | 10 (100%) | 0.0886 |
| Injections (botulinum toxin, etc), n (%) | 25 (64.1%) | 4 (44.4%) | 7 (70.0%) | 5 (50.0%) | 9 (90.0%) | 0.1422 |
| Therapies (physical, speech, occupational, etc), n (%) | 36 (92.3%) | 7 (77.8%) | 10 (100%) | 9 (90.0%) | 10 (100%) | 0.2198 |
| Surgery, n (%) | 16 (41.0%) | 2 (22.2%) | 2 (20.0%) | 3 (30.0%) | 9 (90.0%) | 0.0036 |
| DBS device | 9 (23.1%) | 2 (22.2%) | 2 (20.0%) | 2 (20.0%) | 3 (30.0%) | 0.9443 |
| Other surgery | 14 (35.9%) | 2 (22.2%) | 1 (10.0%) | 2 (20.0%) | 9 (90.0%) | 0.0006 |
| Other treatment, n (%)a | 14 (35.9%) | 1 (11.1%) | 5 (50.0%) | 4 (40.0%) | 4 (40.0%) | 0.3322 |
| PKAN healthcare utilization past year | ||||||
| Medical visits | 0.3446 | |||||
| Mean (SD) | 13.0 (13.1) | 12.2 (16.8) | 7.6 (5.3) | 13.9 (15.6) | 18.3 (11.9) | |
| Median | 8.0 | 6.0 | 5.0 | 7.0 | 15.0 | |
| Range (min, max) | (1.0–56.0) | (2.0–56.0) | (1.0–15.0) | (3.0–50.0) | (1.0–40.0) | |
| Therapy visits | n = 38/39 | n = 9/10 | 0.2188 | |||
| Mean (SD) | 55.2 (78.5) | 23.1 (50.2) | 33.7 (32.1) | 85.1 (126.5) | 78.7 (69.1) | |
| Median | 22.5 | 6.0 | 26.5 | 20.0 | 46.0 | |
| Range (min, max) | (0.0–384.0) | (0.0–156.0) | (0.0–100.0) | (0.0–384.0) | (1.0–208.0) | |
| Caregiving required | ||||||
| Part-time caregiver, n (%) | 25 (64.1%) | 3 (33.3%) | 6 (60.0%) | 8 (80.0%) | 8 (80.0%) | 0.1656 |
| Age at onset, y | n = 23/25 | n = 1/3 | ||||
| Mean (SD) | 11.5 (7.6) | 12.0 (NA) | 17.7 (8.0) | 10.8 (7.9) | 7.6 (4.8) | 0.0975 |
| Median | 11.0 | 12.0 | 19.0 | 11.0 | 7.0 | |
| Range (min, max) | (< 1.0–28.0) | (12.0–12.0) | (6.0–28.0) | (< 1.0–20.0) | (< 1.0–14.0) | |
| Full-time caregiver, n (%) | 21 (53.8%) | 1 (11.1%) | 3 (30.0%) | 7 (70.0%) | 10 (100%) | 0.0021 |
| Age at onset, y | n = 20/21 | n = 2/3 | ||||
| Mean (SD) | 10.3 (6.6) | 19.0 (NA) | 16.5 (9.2) | 9.7 (7.3) | 8.5 (5.1) | 0.2379 |
| Median | 10.0 | 19.0 | 16.5 | 12.0 | 8.5 | |
| Range (min, max) | (< 1.0–23.0) | (19.0–19.0) | (10.0–23.0) | (< 1.0–20.0) | (< 1.0–17.0) | |
aExamples of other treatments for PKAN included leg braces, hippotherapy (horseback riding), and participating in a clinical trial (eg, deferiprone)
DBS deep brain stimulation; OTC over the counter; PKAN pantothenate kinase-associated neurodegeneration; SD standard deviation