Table 1.
Demographic and pain characteristics in 25 UK adult patients with Pompe disease. Data are given either as median (range) or numbers (percentage).
| Characteristic | Pompe patients (n = 25) |
|---|---|
| Median age, years (range) | 47 (20–70) |
| Female, n (%) | 13 (52) |
| Median age at first symptoms, years (range) | 25 (2–54) |
| ERT, n (%) | |
| Currently receiving | 19 (76) |
| Discontinuation | 2 (8) |
| Never | 4 (16) |
| Patients reporting pain in the last 7 days, n (%) | 22 (88) |
| Pain severity score (PSS) (0–10)a | |
| Median PSS (range) | 3.9 (0.5–7.5) |
| Pain severity subgroups | |
| No pain (rating of 0), n (%) | – |
| Mild pain (1–3), n (%) | 10 (45.5) |
| Moderate pain (4–6), n (%) | 10 (45.5) |
| Severe pain (7–10), n (%) | 2 (9) |
| Pain related interference with daily activities | |
| Median pain interference score (range) | 3.9 (0–7.5) |
| General activity, median (range) | 4.0 (0–10) |
| Mood, median, (range) | 3.0 (0–10) |
| Walking ability, median (range) | 5.0 (0–10) |
| Normal work, median (range) | 4.0 (0–10) |
| Relations with other people, median (range) | 3.0 (0–10) |
| Sleep, median (range) | 4.0 (0–10) |
| Enjoyment of life, median (range) | 4.0 (0–10) |
| Quality of life/other patient reported outcomes | |
| Median HADS depression score (range) | 8.0 (0–14) |
| Median HADS anxiety score (range) | 7.0 (1–18) |
| Median SF-36v2 physical component summary score (range) | 27 (14–55) |
| Median SF-36v2 mental component summary score (range) | 48 (24–71) |
| Median Rotterdam Handicap Scale Score (range) | 24 (12–36) |
HADS — Hospital Anxiety and Depression Score, SF-36v2 — Short Form Health Survey 36 version 2.
a
Pain was scored within the last 7 days.