Table 4.
Rates of progression in disease severity by clinical trial eligibility
| Clinical trial eligibility |
||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Baseline Mean ± SD (n = 227) | All patients SPE, 95% CI (n = 227) | Trial SPE, 95% CI | Non-trial SPE, 95% CI (n = 98) | Non-trial RS SPE, 95% CI (n = 45) | Non-trial variant SPE, 95% CI (n = 53) | Trial vs non-trial P-value | Trial vs non-trial RS P-value | Trial vs non-trial variant P-value | Non-trial variant vs non-trial RS P-value | |
| PSPRS | ||||||||||
| History | 7.9 ± 4.4 | 1.8 (1.5–2.2) | 2.3 (1.9–2.8) | 1.0 (0.5–1.5) | 0.4 (−0.3 to 1.1) | 1.3 (0.7–1.9) | 0.0004 | 0.4 | 0.3 | 1 |
| Mentation | 3.4 ± 2.9 | 0.8 (0.5–1.1) | 0.9 (0.5–1.2) | 0.7 (0.3–1.1) | 0.1 (−0.6 to 0.9) | 1.0 (0.4–1.5) | 0.4 | 1 | 1 | 1 |
| Bulbar | 2.3 ± 1.7 | 0.9 (0.7–1.1) | 1.0 (0.8–1.3) | 0.7 (0.5–0.9) | 0.7 (0.3–1.1) | 0.7 (0.5–1.0) | 0.2 | 1 | 1 | 1 |
| Ocular | 7.8 ± 3.6 | 1.6 (1.2–1.9) | 1.7 (1.3–2.1) | 1.3 (0.8–1.8) | 0.7 (0.2–1.2) | 1.7 (1.0–2.4) | 0.1 | 0.1 | 1 | 1 |
| Limb | 4.3 ± 2.8 | 0.9 (0.7–1.2) | 1.2 (0.8–1.5) | 0.7 (0.3–1.0) | 1.0 (0.2–1.8) | 0.6 (0.2–0.9) | 0.1 | 1 | 1 | 1 |
| Gait | 11.1 ± 4.9 | 2.4 (2.0–2.7) | 3.0 (2.4–3.5) | 1.5 (1.0–2.0) | 1.0 (0.4–1.7) | 1.7 (1.0–2.5) | 0.0006 | 0.4 | 0.1 | 1 |
| Total | 36.8 ± 14.8 | 8.4 (7.3–9.6) | 10.1 (8.6–11.5) | 6.1 (4.3–7.8) | 3.9 (1.3–6.5) | 7.1 (4.9–9.4) | 0.001 | 0.1 | 0.2 | 1 |
| Total (>6 months follow-up) | (n = 143) | (n = 143) | (n = 85) | (n = 58) | (n = 21) | (n = 37) | ||||
| 34.6 ± 13.6 | 8.3 (7.2–9.5) | 9.9 (8.5–11.3) | 6.0 (4.3–7.7) | 4.7 (2.3–7.1) | 6.8 (4.6–9.1) | 0.0005 | 0.005 | 0.04 | 1 | |
| Total (>12 months follow-up) | (n = 109) | (n = 109) | (n = 64) | (n = 45) | (n = 16) | (n = 29) | ||||
| 34.7 ± 13.6 | 8.1 (6.9–9.2) | 9.6 (8.2–11.1) | 5.8 (4.2–7.4) | 5.0 (2.6–7.4) | 6.3 (4.1–8.4) | 0.0007 | 0.01 | 0.03 | 1 | |
| mPSPRS | ||||||||||
| History | 3.7 ± 1.8 | 0.7 (0.5–0.9) | 0.9 (0.7–1.2) | 0.4 (0.2–0.6) | 0.1 (−0.5 to 0.6) | 0.5 (0.3–0.8) | 0.006 | 0.7 | 0.3 | 1 |
| Mentation | 0.9 ± 0.5 | 0.2 (0.1–0.2) | 0.3 (0.2–0.3) | 0.1 (0.06–0.2) | 0.1 (−0.1 to 0.2) | 0.1 (0.1–0.2) | 0.4 | 1 | 1 | 1 |
| Bulbar | 2.0 ± 1.0 | 0.5 (0.4–0.6) | 0.5 (0.4–0.6) | 0.5 (0.3–0.7) | 0.3 (0.2–0.5) | 0.6 (0.4–0.9) | 0.3 | 1 | 1 | 1 |
| Ocular | 0.9 ± 0.6 | 0.1 (0.1–0.2) | 0.1 (0.1–0.2) | 0.1 (0.1–0.2) | 0.1 (−0.01 to 0.2) | 0.2 (0.1–0.2) | 0.6 | 1 | 1 | 1 |
| Limb | 1.1 ± 0.6 | 0.2 (0.1–0.2) | 0.3 (0.2–0.3) | 0.1 (−0.03 to 0.1) | 0.04 (−0.1 to 0.2) | 0.1 (−0.04 to 0.2) | 0.01 | 1 | 0.04 | 0.4 |
| Gait | 5.7 ± 2.0 | 0.9 (0.7–1.0) | 1.1 (0.9–1.3) | 0.5 (0.3–0.8) | 0.3 (0.1–0.5) | 0.7 (0.4–1.0) | 0.006 | 0.3 | 0.4 | 1 |
| Total | 14.4 ± 4.7 | 2.6 (2.2–3.0) | 3.1 (2.6–3.6) | 1.9 (1.2–2.5) | 0.7 (−0.4 to 1.9) | 2.3 (1.5–3.1) | 0.005 | 0.2 | 0.4 | 1 |
mPSPRS = modified Progressive Supranuclear Palsy Rating Scale; PSP = progressive supranuclear palsy; PSPRS = Progressive Supranuclear Palsy Rating Scale; RS = Richardson’s Syndrome; SD = standard deviation; Trial cases refers to the subset deemed likely eligible to recent Phase II clinical trial; SPE = 1-year progression estimate.