Table 2.
Demographics and adverse events for PD patients
| Demographics | Placebo | Sargramostim | ||
|---|---|---|---|---|
| N | Mean (SD) | N | Mean (SD) | |
| Age (years) | 10 | 67 (6) | 10 | 62 (7) |
| Time since first symptoms (years) | 9 | 7 (3) | 10 | 7 (2) |
| Time since diagnosis (years) | 10 | 5 (4) | 10 | 6 (3) |
| UPDRS III score | 10 | 24 (10) | 10 | 20 (5) |
| N (Percentage) | N (Percentage) | |
|---|---|---|
| Male sex | 8 (80) | 8 (80) |
| Caucasian race | 10 (100) | 10 (100) |
| Jobs with pesticides | 3 (30) | 0 (0) |
| Exposure to pesticides | 7 (70) | 3 (30) |
| Jobs with chemical solvents | 4 (40) | 4 (40) |
| Jobs with other chemical fumes | 5 (40) | 4 (40) |
| Jobs with heavy metals | 1 (10) | 1 (10) |
| Adverse eventsa | ||
| Any adverse event | 8 (80) | 10 (100) |
| Any severe adverse events | 0 (0) | 3 (30) |
| Any serious adverse events | 0 (0) | 1 (10) |
| Adverse event leading to withdrawal | 0 (0) | 4 (40) |
| Possible relationship to drug | 7 (70) | 10 (100) |
| Definitive relationship to drug | 2 (20) | 7 (70) |
| Categorya | ||
| Injection site reaction | 4 (40) | 10 (100)b |
| Abnormal laboratory values | 3 (30) | 10 (100)b |
| Pain, other than injection site | 3 (30) | 7 (70) |
| Pain, upper torso and extremities | 0 (0) | 7 (70)b,g |
| Pain, lower torso and extremities | 3 (30) | 3 (30) |
| Chest pain or discomfort | 0 (0) | 4 (40) |
| Muscle, soreness, weakness | 4 (40) | 3 (30) |
| Rash, other than injection site | 2 (20) | 4 (40) |
| Shortness of breath, wheezing | 0 (0) | 3 (30) |
| GI tract, nausea, vomiting | 0 (0) | 3 (30) |
| Injury | 3 (30) | 2 (20) |
| Headache | 2 (20) | 2 (20) |
| Fatigue | 2 (20) | 2 (20) |
| Infection, any | 2 (20) | 2 (20) |
| Neurological, psychological, dyskinesia | 2 (20) | 2 (20) |
| Chills, fever | 1 (10) | 2 (20) |
| Itching, other than injection site | 0 (0) | 2 (20) |
| Cardiovascular, hematological | 0 (0) | 2 (20) |
| Skin, not infection | 3 (30) | 1 (10) |
| Equilibrium | 1 (10) | 1 (10) |
| Sleep anomalies | 1 (10) | 1 (10) |
| Edema, other than injection site | 0 (0) | 1 (10) |
| Ophthalmological | 0 (0) | 1 (10) |
| Median (IQR) | Mean (SD) | Median (IQR) | Mean (SD) | |
|---|---|---|---|---|
| Severity of combined adverse eventsc | 1.2 (1.1–1.4) | 1.2 (0.1) | 1.7 (1.4–1.8)e | 1.6 (0.3) |
| Likelihood of events being drug-relatedd | 2.4 (1.9–2.7) | 2.2 (0.6) | 3.8 (3.1–3.9)e | 3.6 (0.6) |
| Severity of injection site reactionc | 1.5 (1.0–2.0) | 1.5 (0.6) | 1.0 (1.0–2.0) | 1.3 (0.5) |
| Severity of pain, other than injection sitec,g | 1.0 (1.0–1.8) | 1.2 (0.5) | 2.0 (2.0–3.0) | 2.0 (0.7) |
| Severity of pain, upper torso and extremitiesc,g | ndf | nd | 2.0 (1.8–2.0) | 2.0 (0.6) |
| Severity of pain, lower torso and extremitiesc,g | 1.0 (1.0–1.8) | 1.2 (0.5) | 2.0 (1.5–3.0) | 2.1 (0.8) |
a Adverse events reported since the initiation of placebo/drug. More than two adverse advents per patient may have been reported; however, patients are only counted once within each category. The same patient may be counted in different categories
b P ≤ 0.01 by Fisher’s exact test
c Scored by attending physician; 1 = mild, 2 = moderate, 3 = severe
d Scored by attending physician; 1 = Unrelated, 2 = Unlikely, 3 = Possibly, 4 = Probably, 5 = Definite
e P ≤ 0.004 by Mann–Whitney U test
f nd no data
g The incidence of upper torso bone and musculoskeletal chest pain were higher in patients treated with sargramostim compared with placebo and was distinct from that associated with PD19–22