Table 1.
Characteristics at enrolment: overall and according to initial choice of quinacrine or not
| All patients* | Chose quinacrine at enrolment | Chose no quinacrine at enrolment† | p value‡ | Chose quinacrine vs no quinacrine OR (95% CI; p)§ | ||
|---|---|---|---|---|---|---|
| Enrolled | 107 (100%) | 32 (100%) | 69 (100%) | |||
| Studies | 0·16 | |||||
| Pilot study | 23 (22%) | 8 (25%) | 9 (13%) | |||
| Main study | 84 (78%) | 24 (75%) | 60 (87%) | |||
| Type of prion disease | 0·06 | .. | ||||
| Sporadic | 45 (42%) | 8 (25%) | 36 (52%) | |||
| Iatrogenic | 2 (2%) | 1 (3%) | 1 (1%) | |||
| Variant | 18 (17%) | 6 (19%) | 10 (14%) | |||
| Inherited | 42 (39%) | 17 (53%) | 22 (32%) | |||
| Median age (years; range) | 56 (14–82) | 55 (17–75) | 58 (19–82) | 0·18 | .. | |
| Non-inherited disease | 60 (14–82) | 60 (17–75) | 62 (19–82) | |||
| Inherited disease | 43 (32–72) | 53 (32–65) | 41 (32–72) | |||
| Median time (months) since first symptoms¶ (range) | 10 (1–140) | 13 (2–118) | 8 (1–140) | 0·04 | .. | |
| Non-inherited disease | 7 (1–50) | 9 (2–19) | 7 (1–50) | |||
| Inherited disease | 26 (3–140) | 25 (4–118) | 27 (3–140) | |||
| Barthel index | 1·89 (0·97–3·62; 0·06) | |||||
| Number assessed | 95 (89%) | 28 (88%) | 67 (97%) | 0·08 | ||
| Median (IQR) | 4 (11–17) | 14 (5–18) | 2 (0–12) | 0·0007 | ||
| MMSE | 1·29 (1·06–1·57; 0·01) | |||||
| Number assessed | 58 (54%) | 26 (81%) | 27 (39%) | 0·0001 | ||
| Median (IQR) | 20 (15–25) | 23 (18–26) | 18 (14–25) | 0·20 | ||
| Median observed/imputed value (IQR)‖ | 7 (0–21) | 22 (7–25) | 0 (0–15) | <0·0001 | ||
| CDR | 0·64 (0·44–0·92; 0·02) | |||||
| Number assessed | 75 (70%) | 27 (84%) | 42 (61%) | 0·02 | ||
| Median (IQR) | 8 (4–12) | 6 (2–8) | 9 (6–13) | 0·01 | ||
| Median observed/imputed value (IQR)‖ | 11 (6–18) | 7 (3–10) | 16 (8–18) | 0·0007 | ||
| GCS | .. | |||||
| Number assessed | 74 (69%) | 19 (59%) | 54 (78%) | 0·06 | ||
| Median (IQR) | 12 (10–14) | 15 (11–15) | 12 (9–14) | 0·001 | ||
| Median observed/imputed value (IQR)‖ | 14 (11–15) | 15 (14–15) | 13 (10–15) | <0·0001 | ||
| ADAS-cog | ||||||
| Number assessed | 37 (35%) | 20 (62%) | 17 (25%) | 0·0004 | ||
| Median (IQR) | 17 (8–29) | 18 (8–26) | 16 (8–32) | 0·95 | .. | |
| BPRS | .. | |||||
| Number assessed | 37 (35%) | 19 (59%) | 18 (26%) | 0·002 | ||
| Median (IQR) | 33 (30–40) | 37 (30–40) | 32 (30–40) | 0·46 | ||
| Rankin scale | ||||||
| Number assessed | 105 (98%) | 32 (100%) | 67 (97%) | 1·00 | ||
| No or slight symptoms (1/2)** | 13 (12%) | 5 (16%) | 8 (12%) | 0·0007 | 1·00 (0·09) | |
| Moderate disability (3) | 21 (20%) | 11 (34%) | 9 (13%) | 3·21 (0·71–14·5) | ||
| Moderate to severe disability (4) | 31 (30%) | 12 (38%) | 15 (22%) | 5·20 (0·90–30·0) | ||
| Severe disability (5) | 40 (38%) | 4 (12%) | 35 (52%) | 1·52 (0·17–13·5) | ||
| CIBIC-P | ||||||
| Number assessed | 105 (98%) | 31 (97%) | 68 (99%) | 0·54 | ||
| Normal or borderline (1/2)** | 7 (7%) | 1 (3%) | 6 (9%) | 0·0001 | 1·00 (0·07) | |
| Mildly ill (3) | 11 (10%) | 7 (23%) | 4 (6%) | 12·8 (1·06–155) | ||
| Moderately ill (4) | 23 (22%) | 11 (35%) | 10 (15%) | 11·6 (1·04–130) | ||
| Markedly ill (5) | 26 (25%) | 9 (29%) | 14 (21%) | 14·3 (0·91–222) | ||
| Severely ill or the most ill (6/7) | 38 (36%) | 3 (10%) | 34 (50%) | 3·39 (0·13–85·5) | ||
Data are number (%) unless otherwise stated. MMSE=mini-mental state examination. CDR=clinical dementia rating. GCS=Glasgow coma scale. ADAS-cog= Alzheimer's Disease Assessment scale cognitive component. BPRS=brief psychiatric rating scale. CIBIC-P=clinician interview-based impression of change plus carer input.
Includes the first six patients who received quinacrine in a pilot study without the option of no quinacrine who are excluded from comparisons according to choice.
One patient who chose to be randomised at enrolment is included in the chose no quinacrine group (the other randomised patient chose no quinacrine at enrolment, see Results).
Univariate p values from exact tests (categorical) or ranksum (continuous) assessing the effect of each factor on choice of quinacrine or no quinacrine.
Multivariate independent predictors; the best multivariate logistic models adjusted for baseline Rankin score or CIBIC-P and one of Barthel index, MMSE, and CDR; numbers were too small to discriminate further between these predictors; effect on choice of quinacrine versus no quinacrine shown for Rankin and MMSE (OR per 3 units higher) from model including Rankin and MMSE, for CDR and Barthel index (OR per 3 units higher) from model including Rankin and CDR or Barthel index, and for CIBIC-P from a model including CIBIC-P and MMSE (similar results were obtained for other models combining these factors), absence of OR means no evidence of a independent contribution of this factor (p>0·10).
Excluding six patients asymptomatic at enrolment (one chose quinacrine, five chose no quinacrine).
Methods for imputation strategy; number of imputed baseline values: MMSE 48, CDR 30, GCS 32.
Percentages of non-missing values.