Table 2.
Measures of Disease Activity and Improvement from Baseline.*
| Measure | Phase of Open-Label Treatment | P Value† | P Value‡ | |||
|---|---|---|---|---|---|---|
| Baseline | One Month | Three Months | Six Months | |||
| Primary measure of clinical response | ||||||
|
| ||||||
| Global diary score§ | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 3.70 | 0.79 | 0.29 | 0.26 | ||
|
| ||||||
| Interquartile range | 2.16–4.84 | 0.26–1.25 | 0.08–0.84 | 0.12–0.70 | ||
|
| ||||||
| Primary measures of laboratory response | ||||||
|
| ||||||
| Erythrocyte sedimentation rate (mm/hr) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 57.5 | 12.5 | 18.0 | 16.0 | ||
|
| ||||||
| Interquartile range | 35.0–73.0 | 11.0–24.0 | 9.0–25.0 | 11.0–29.0 | ||
|
| ||||||
| C-reactive protein (mg/dl) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 5.29 | 0.93 | 0.34 | 0.40 | ||
|
| ||||||
| Interquartile range | 4.00–10.50 | 0.49–1.94 | 0.16–0.89 | 0.10–0.91 | ||
|
| ||||||
| Serum amyloid A (mg/liter) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 174 | 25 | 8 | 6 | ||
|
| ||||||
| Interquartile range | 131–436 | 9–97 | 3–34 | 3–16 | ||
|
| ||||||
| Secondary measures of clinical response | ||||||
|
| ||||||
| CHAQ score¶ | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 1.30 | 0.64 | 0.37 | 0.34 | ||
|
| ||||||
| Interquartile range | 0.65–1.57 | 0.31–1.03 | 0.12–0.72 | 0.13–0.68 | ||
|
| ||||||
| Physician’s global assessment (mm)|| | 0.001 | <0.001 | ||||
|
| ||||||
| Median | 16.5 | 9.0 | 4.5 | 4.5 | ||
|
| ||||||
| Interquartile range | 8.0–32.0 | 7.0–14.0 | 4.0–10.0 | 2.0–8.0 | ||
|
| ||||||
| Parent’s global assessment (mm)|| | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 48.5 | 10.0 | 5.5 | 5.5 | ||
|
| ||||||
| Interquartile range | 23.5–52.0 | 4.0–28.0 | 2.0–16.0 | 2.0–8.5 | ||
|
| ||||||
| Visual-analogue scale for pain (mm)|| | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 38.0 | 12.0 | 3.0 | 5.5 | ||
|
| ||||||
| Interquartile range | 22.0–60.0 | 6.0–20.0 | 2.0–10.0 | 2.0–13.0 | ||
|
| ||||||
| Dose of prednisone or prednisone equivalent dose (mg/kg/day)** | 0.002 | 0.001 | ||||
|
| ||||||
| Median | 0.46 | ND | 0.30 | 0.17 | ||
|
| ||||||
| Interquartile range | 0.21–0.96 | 0.20–0.38 | 0.08–0.24 | |||
|
| ||||||
| Secondary measures of laboratory response | ||||||
|
| ||||||
| White-cell count (×10−3/mm3) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 17.2 | 8.9 | 9.3 | 8.4 | ||
|
| ||||||
| Interquartile range | 13.6–21.5 | 7.1–13.9 | 7.5–11.2 | 6.8–12.1 | ||
|
| ||||||
| Absolute neutrophil count (×10−3/mm3) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 12.4 | 5.0 | 5.4 | 5.1 | ||
|
| ||||||
| Interquartile range | 9.9–15.5 | 3.9–9.8 | 3.0–7.4 | 2.8–7.2 | ||
|
| ||||||
| Hemoglobin (g/dl) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 11.2 | 12.5 | 13.3 | 13.4 | ||
|
| ||||||
| Interquartile range | 10.4–11.8 | 12.0–13.1 | 12.5–14.4 | 12.4–14.1 | ||
|
| ||||||
| Platelets (×10−3/mm3) | <0.001 | <0.001 | ||||
|
| ||||||
| Median | 423 | 326 | 302 | 296 | ||
|
| ||||||
| Interquartile range | 380–531 | 249–417 | 219–368 | 269–409 | ||
|
| ||||||
| Height (cm)†† | 112.9±24.8 | 113.3±24.6 | 115.0±25.1 | 116.0±25.2 | <0.001 | <0.001 |
|
| ||||||
| Weight (kg) | 28.7±15.8 | 30.0±16.2 | 31.4±17.3 | 32.7±18.3 | <0.001 | 0.001 |
|
| ||||||
| Cerebrospinal fluid pressure (mm of water)‡‡ | <0.001 | NA | ||||
|
| ||||||
| Median | 287 | ND | 197 | ND | ||
|
| ||||||
| Interquartile range | 250–325 | ND | 167–222 | ND | ||
|
| ||||||
| Cerebrospinal fluid protein (mg/dl)§§ | 0.05 | NA | ||||
|
| ||||||
| Median | 35 | ND | 33 | ND | ||
|
| ||||||
| Interquartile range | 24–51 | ND | 23–40 | ND | ||
|
| ||||||
| White-cell count in cerebrospinal fluid (cells/mm3)§§ | 0.05 | NA | ||||
|
| ||||||
| Median | 19 | ND | 9 | ND | ||
|
| ||||||
| Interquartile range | 6–49 | ND | 6–12 | ND | ||
|
| ||||||
| Neutrophil count in cerebrospinal fluid (cells/mm3)§§ | 0.04 | NA | ||||
|
| ||||||
| Median | 10.2 | ND | 3.8 | ND | ||
|
| ||||||
| Interquartile range | 4.0–25.5 | ND | 1.8–6.7 | ND | ||
Plus–minus values are means ±SD. NA denotes not applicable, and ND not done.
P values are for the comparison of baseline values with values obtained at three months.
P values are for the comparison of baseline values with values obtained at six months.
Median daily scores of five symptoms (fever, rash, headache, joint pain, and vomiting) were evaluated daily with the use of a scale that ranged from 0 (no symptoms) to 4 (severe symptoms) (possible total range, 0 to 20). The maximal daily score measured was 14; the minimal score was 0.
Scores for the Childhood Health Assessment Questionnaire (CHAQ), a standardized test for the assessment of disability, range from 0 to 3, with higher scores indicating more severe impairment.
A visual-analogue scale was used in which a value of 100 mm indicates the worst possible measure for the condition assessed by the test.
Values are for 11 patients who were receiving corticosteroids at study entry.
Values are for 15 patients with open growth plates only. At six months, the growth velocity in percentile was 74; the heights of 12 of these patients fell below the 3rd percentile for age at the beginning of the study.
Cerebrospinal fluid pressures could be evaluated in 12 patients (i.e., could be obtained on both visits, and patients did not cry during the procedure).
Cerebrospinal fluid could be evaluated in 14 patients (i.e., could be obtained on both visits, and patients had a red-cell count of less than 50 cells per cubic millimeter in the cerebrospinal fluid).