Table II.
Outcomes
| Adjusted Intent to treat analyses | |||||
|---|---|---|---|---|---|
| Placebo (17) | Drug (35) | Treatment Effect | 95%CI | P value | |
| Patient-reported outcomes | |||||
| Pain score over time † | |||||
| Mean (SD) | 2.82 (2.21) | 2.09 (2.55) | −1.42 | −0.61 – −2.21 | 0.001 |
| LS mean (SE)*** | 3.09 (0.23) | 1.67 (0.34) | |||
| Δ ASCQ-Me Pain domain †† | |||||
| Mean (SD) | 6.9 (24.0) | 2.8 (22.7) | −5.2 | −19.7 – 9.29 | 0.47 |
| LS mean (SE)*** | 6.9 (6.80) | 2.8 (4.86) | |||
| Asthma control test (0–25) †† | |||||
| • Total score | 17.1 (1.78) | 17.7 (2.25) | 0.20 | −1.13 – 1.53 | 0.3 |
| • Respiratory symptoms interfering with work? | 7 (41.2%) | 9 (26.5%) | |||
| • Shortness of breath? | 6 (35.3%) | 5 (14.7%) | |||
| • Nighttime symptoms? | 6 (35.3%) | 7 (20.6%) | |||
| • Wheezing controlled? | 12 (75%) | 22 (66.7%) | |||
| Clinical outcomes | |||||
| ED Visits ††† | |||||
| Mean (SD) | 1.12 (1.87) | 0.97 (1.52) | 0.80* | 0.35 – 1.84 | 0.60 |
| Predicted count (SE)** | 1.05 (0.44) | 1.00 (0.31) | |||
| Observation admits ††† | |||||
| Mean (SD) | 0.59 (1.18) | 0.37 (0.77) | 0.58* | 0.21 – 1.62 | 0.30 |
| Predicted count (SE)** | 0.57 (0.52) | 0.38 (0.41) | |||
| Admissions ††† | |||||
| Mean (SD) | 0.47 (1.23) | 0.37 (0.77) | 0.73* | 0.25 – 2.13 | 0.56 |
| Predicted count (SE)** | 0.45 (0.54) | 0.38 (0.41) | |||
| Overnight stays (admit or obs) ††† | |||||
| Mean (SD) | 0.82 (1.67) | 0.60 (1.09) | 0.69* | 0.28 – 1.71 | 0.43 |
| Predicted count (SE)** | 0.79 (0.45) | 0.61 (0.34) | |||
| Admit rate †††† | |||||
| Mean (SD) | 0.42 (0.39) | 0.38 (0.36) | 0.85* | 0.33 – 2.15 | 0.74 |
| Predicted rate (SE) | 0.42 (0.46) | 0.36 (0.31) | |||
| Admit/obs rate †††† | |||||
| Mean (SD) | 0.74 (0.36) | 0.62 (0.38) | 0.79* | 0.45 – 1.38 | 0.40 |
| Predicted rate (SE) | 0.73 (0.25) | 0.58 (0.21) | |||
| Inpatient days ††† | |||||
| Mean (SD) | 4.09 (9.25) | 2.67 (5.11) | −1.88 | −5.95 – 2.19 | 0.36 |
| LS Mean (SE)*** | 3.95 (1.91) | 2.68 (1.38) | |||
| Biological outcomes | |||||
| Δ Hemoglobin g/dL †† | |||||
| Mean (SD) | −0.01 (1.31) | 0.30 (0.96) | 0.50 | −0.21 – 1.20 | 0.16 |
| LS mean (SE)*** | 0.00 (0.32) | 0.29 (0.23) | |||
| Δ Reticulocytes % †† | |||||
| Mean (SD) | 0.39 (2.85) | −1.16 (2.63) | −1.86 | −3.66 – −0.06 | 0.04 |
| LS mean (SE)*** | 0.37 (0.82) | −1.12 (0.62) | |||
| Δ Reticulocytes × 103/μL †† | |||||
| Mean (SD) | 0.07 (0.52) | −0.15 (0.39) | −0.21 | −0.52 – 0.10 | 0.18 |
| LS mean (SE)*** | 0.07 (0.14) | −0.15 (0.10) | |||
| Δ WBC × 103/μL †† | |||||
| Mean (SD) | −0.61 (3.70) | −0.55 (2.80) | 0.15 | −1.94 – 2.23 | 0.89 |
| LS mean (SE)*** | −0.54 (0.94) | −0.57 (0.67) | |||
| Δ Platelets †† | |||||
| Mean (SD) | 4.33 (127.75) | −41.36 (88.14) | −27.44 | −93.79 – 38.92 | 0.41 |
| LS mean (SE)*** | 2.50 (29.98) | −41.09 (21.28) | |||
| Δ Neutrophils × 103/μL †† | |||||
| Mean (SD) | −0.82 (3.08) | −0.31 (2.92) | 0.51 | −1.54 – 2.56 | 0.50 |
| LS mean (SE)*** | −0.77 (0.93) | −0.33 (0.64) | |||
| Δ LDH †† | |||||
| Mean (SD) | 47.5 (117.15) | −64.81 (132.37) | −111.99 | −272.49 – 48.51 | 0.16 |
| LS mean (SE)*** | 47.39 (76.06) | −65.19 (43.30) | |||
| Δ sVCAM ng/mL †† | |||||
| Mean (SD) | 170.25 (814.74) | −182.47 (785.21) | −526.94 | −1,003.23 – −50.66 | 0.03 |
| LS mean (SE)*** | 169.88 (215.59) | −182.39 (158.85) | |||
| Percent change (SD) | 8.82% (34.60) | −4.86% (29.17) | 13.67 | ||
| Δ eNO (ppb) †† | |||||
| Mean (SD) | 2.71 (6.92) | 0.63 (9.63) | −2.47 | −8.06 – 3.12 | 0.38 |
| LS mean (SE)*** | 2.73 (2.52) | 0.58 (1.84) | |||
| Δ FEV1 %predicted †† | |||||
| Mean (SD) | −2.94 (5.15) | 0.58 (7.69) | 2.67 | −1.70 – 70.4 | 0.23 |
| LS mean (SE)*** | −2.99 (1.96) | 0.66 (1.50) | |||
| Δ FVC % predicted †† | |||||
| Mean (SD) | −1.29 (4.37) | 0.48 (6.53) | 1.39 | −2.37 – 5.15 | 0.46 |
| LS mean (SE)*** | −1.32 (1.69) | 0.60 (1.29) | |||
| Δ FEV1/FVC †† | |||||
| Mean (SD) | −1.41 (3.39) | −0.71 (3.42) | 0.39 | −1.78 – 2.56 | 0.72 |
| LS mean (SE)*** | −1.44 (0.97) | −0.73 (0.74) | |||
All values adjusted for participant age
*
RR – rate ratio
**
Predicted counts obtained via negative binomial regression
***
Least squares mean obtained via linear regression
†
Random-intercepts, mixed effect model with patient as a random effect
††
Linear Regression
†††
Negative Binomial Regression
††††
Generalized estimating equation with “REPEATED” statement for subject ID and binary logit link