Clinical characteristics of quartiles of ΔFEV1 response to rapamycin
| ΔFEV1 response quartile | Q1 | Q2 | Q3 | Q4 | Q1 vs Q4 (p) |
| n | 11 | 12 | 12 | 12 | |
| Rapamycin-treated ΔFEV1 | |||||
| Range (mL/year) | 254 to 56 | 50 to 0 | −1 to −30 | −31 to −148 | − |
| Mean (mL/year) | 111 (58) | 28 (15) | −17 (11) | −71 (41) | <0.0001 |
| Duration of treatment (years) | 2.4 (1.3) | 3.4 (1.6) | 2.8 (1.1) | 2.4 (2.1) | 0.09 |
| Clinical characteristics at start of treatment | |||||
| Pretreatment ΔFEV1 (mL/year)* | −186 (238) | −100 (69) | −152 (77) | −53 (202) | 0.20 |
| Age (years) | 41 (9) | 45 (11) | 44 (8) | 45 (6) | 0.13 |
| Disease duration (years) | 6.1 (5.3) | 13.0 (10.2) | 8.2 (5.0) | 11.3 (5.9) | 0.02 |
| FEV1 (% predicted) | 67 (23) | 42 (10) | 52 (17) | 48 (20) | 0.02 |
| DLCO (% predicted) | 46 (11) | 40 (10) | 45 (14) | 40 (15) | 0.13 |
| Disease features present | |||||
| Angiomyolipoma | 6 | 5 | 6 | 3 | NS |
| Lymphatic disease | 3 | 3 | 1 | 2 | NS |
| TSC | 3 | 2 | 2 | 2 | NS |
| Ever had pneumothorax | 5 | 4 | 6 | 3 | NS |
| Post-treatment laboratory values | |||||
| Trough rapamycin level (ng/mL)† | 4.2 (1.2) | 5.2 (1.2) | 5.6 (1.7) | 5.2 (1.5) | 0.07 |
| Urine protein:creatinine ratio‡ | 8 (5–366) | 9 (6–43) | 11 (5–79) | 8 (6–15) | 0.84 |
*For 21 subjects only.
†Mean value over 12 months (p for Student’s t-test).
‡Median (range) value after 12 months (p for Mann-Whitney test). NS for non-parametric data analysed by χ2 and Fischer’s exact test.