Table 1.
Baseline Demographics and Characteristics (Modified Intention-to-Treat Population)*
| LAI (n = 44) | Placebo (n = 45) | Overall (n = 89) | P Value | |
|---|---|---|---|---|
| Race/ethnicity, n (%) | 0.386 | |||
| White (not of Hispanic origin) | 42 (95.5) | 40 (88.9) | 82 (92.1) | |
| Hispanic | 0 | 2 (4.4) | 2 (2.2) | |
| African | 0 | 1 (2.2) | 1 (1.1) | |
| Asian | 2 (4.5) | 2 (4.4) | 4 (4.5) | |
| Sex, n (%) | 0.717 | |||
| Male | 6 (13.6) | 5 (11.1) | 11 (12.4) | |
| Female | 38 (86.4) | 40 (88.9) | 78 (87.6) | |
| Height, cm, mean (SD) | 165.4 (7.63) | 164.2 (9.12) | 164.8 (8.39) | 0.503 |
| Weight, kg, mean (SD) | 59.71 (10.132) | 60.15 (15.373) | 59.93 (12.976) | 0.874 |
| BMI, kg/m2, mean (SD) | 21.76 (2.705) | 22.10 (3.941) | 21.93 (3.372) | 0.637 |
| Age, yr, mean (SD) | 58.0 (16.61) | 59.1 (15.20) | 58.5 (15.83) | 0.745 |
| FEV1 % predicted, mean (SD) | 63.56 (21.339) | 62.56 (17.168) | 63.06 (19.239) | 0.808 |
| Stratification at screening, n (%) | ||||
| Predominantly Mycobacterium avium complex | 29 (65.9) | 28 (62.2) | 57 (64.0) | 0.717 |
| Predominantly Mycobacterium abscessus | 15 (34.1) | 17 (37.8) | 32 (36.0) | 0.717 |
| CF | 8 (18.2) | 9 (20.0) | 17 (19.1) | 0.827 |
| M. avium complex and CF | 2 (4.5) | 1 (2.2) | 3 (3.4) | 0.544 |
| M. abscessus and CF | 6 (13.6) | 8 (17.8) | 14 (15.7) | 0.592 |
| Mycobacterial load,† n (%) | 0.761 | |||
| Culture negative | 3 (6.8) | 3 (6.7) | 6 (6.7) | |
| Growth in liquid medium only | 3 (6.8) | 3 (6.7) | 6 (6.7) | |
| 1–49 colonies | 17 (38.6) | 10 (22.2) | 27 (30.3) | |
| 1+ (50–100 colonies) | 2 (4.5) | 4 (8.9) | 6 (6.7) | |
| 2+ (>100–200 colonies) | 2 (4.5) | 2 (4.4) | 4 (4.5) | |
| 3+ (>200–500 colonies) | 3 (6.8) | 4 (8.9) | 7 (7.9) | |
| 4+ (>500 colonies) | 14 (31.8) | 19 (42.2) | 33 (37.1) | |
| Cavitary disease‡ | 33 (75.0) | 35 (77.8) | 68 (76.4) | 0.807 |
| Macrolide resistant | 8 (18.0) | 15 (33.3) | 23 (25.8) | 0.146 |
| Amikacin mutation positive | 2 (4.5) | 6 (13.3) | 8 (8.9) | 0.266 |
| Macrolide resistant or amikacin mutation positive | 9 (20.5) | 16 (35.6) | 25 (28.1) |
Definition of abbreviations: BMI = body mass index; CF = cystic fibrosis; LAI = liposomal amikacin for inhalation.
*
Baseline is defined as the measurement prior and closest to the administration of study drug.
†
At least two sputum samples (preferably three sputum samples) were obtained.
‡
This designation included patients with primarily nodular bronchiectasis.