TABLE 2.
Patient clinical characteristics
| Fosmetpantotenate (n = 41) | Placebo (n = 43) | |
|---|---|---|
| Age at PKAN onset, years, mean (SD) | 7.8 (7.1) | 8.5 (6.6) |
| Duration of PKAN, years, mean (SD) | 14.8 (8.2) | 14.6 (10.5) |
| First PKAN‐related problem, n (%) | ||
| Walking | 28 (68.3) | 32 (74.4) |
| Speech | 29 (70.7) | 32 (74.4) |
| Swallowing | 11 (26.8) | 10 (23.3) |
| Writing | 18 (43.9) | 20 (46.5) |
| Emotional/behavioral problems | 11 (26.8) | 13 (30.2) |
| Vision | 9 (22.0) | 9 (20.9) |
| Dressing self | 10 (24.4) | 12 (27.9) |
| Coordination | 23 (56.1) | 24 (55.8) |
| Dystonia | 25 (61.0) | 33 (76.7) |
| Mouth/tongue | 16 (39.0) | 21 (48.8) |
| Neck | 13 (31.7) | 12 (27.9) |
| Hand | 15 (36.6) | 27 (62.8) |
| Foot | 19 (46.3) | 24 (55.8) |
| Back/trunk | 15 (36.6) | 15 (34.9) |
| Other dystonia | 2 (4.9) | 7 (16.3) |
| Other problem | 10 (24.4) | 12 (27.9) |
| Time from first symptom to diagnosis, years, mean (SD) | 5.8 (6.9) | 6.3 (5.4) |
| Age at first MRI, years, mean (SD) | 11.4 (6.7) | 14.0 (8.4) |
| Number of doctors seen before diagnosis, mean (SD) a | 3.9 (3.3) | 5.1 (4.4) |
| Number of medical visits in last year, mean (SD) b | 7.8 (6.5) | 9.3 (11.4) |
| Number of therapy visits in last year, mean (SD) c | 44.4 (83.8) | 57.5 (55.4) |
| Baseline PKAN‐ADL total score, mean (SD) | 28.2 (11.4) | 27.4 (11.5) |
| Baseline UPDRS Part III total score, mean (SD) | 45.3 (21.2) | 43.5 (21.1) |
| Baseline BAD total score, mean (SD) | 19.5 (7.8) | 17.2 (9.1) |
a
Data are not available for five patients receiving fosmetpantotenate and five patients receiving placebo.
b
Therapy visits (eg, physical, speech, and occupational therapies) data were not available for two patients receiving fosmetpantotenate and two patients receiving placebo.
c
Data are not available for four patients receiving fosmetpantotenate and five patients receiving placebo.
Abbreviations: PKAN‐ADL, Pantothenate Kinase–Associated Neurodegeneration‐Activities of Daily Living; SD, standard deviation; UPDRS, Unified Parkinson's Disease Rating Scale; BAD, Barry–Albright Dystonia.