Outcomes at 12 months, by randomised treatment group
| Usual care | Intervention | Usual care versus intervention | |||||
| N | Exacerbation rate (no. of exacerbations, person years) | N | Exacerbation rate (no. of exacerbations, person years) | Adjusted difference in means (95% CI)* | Direction of positive effect (possible range) | Standardised effect size | |
| Exacerbations | 303 | 1.77 (526, 297.2) | 304 | 1.63 (482, 294.9) | 0.96 (0.83 to 1.12) | Decrease (not applicable) | Not applicable |
| Usual care | Intervention | Usual care versus intervention | |||||
| N | Mean±SD | N | Mean±SD | Adjusted difference in means (95% CI)† |
Direction of positive effect
(possible range) | Standardised effect size | |
| Objectively measured effective adherence (weekly) – %‡ | 295 | 34.9±31.7 | 293 | 52.9±31.4 | 9.5 (8.6 to 10.4) | Increase (0 to 100) | 0.29 |
| FEV1 % predicted | 282 | 56.9±23.0 | 274 | 60.6±24.2 | 1.4 (−0.2 to 3.0) | Increase (0 to 100) | 0.06 |
| Body mass index – kg/m2 | 282 | 22.6±4.1 | 273 | 23.1±4.4 | 0.3 (0.1 to 0.6) | Increase (not applicable) | 0.07 |
| Patient-reported outcomes§ | |||||||
| CFQ-R (quality of life): | Increase (each 0 to 100) | ||||||
| Physical | 274 | 52.6±30.6 | 264 | 55.8±30.2 | 2.3 (−1.0 to 5.6) | 0.08 | |
| Emotional | 274 | 66.5±24.7 | 264 | 66.6±22.9 | 0.2 (−2.9 to 3.2) | 0.01 | |
| Social | 274 | 59.6±20.0 | 264 | 60.5±20.0 | 0.3 (−2.2 to 2.7) | 0.01 | |
| Eating | 274 | 81.0±23.2 | 264 | 84.0±21.5 | 1.9 (−1.3 to 5.2) | 0.09 | |
| Body image | 274 | 65.1±29.3 | 264 | 67.2±27.3 | 1.7 (−1.4 to 4.8) | 0.06 | |
| Treatment burden | 274 | 51.5±19.7 | 265 | 56.6±19.5 | 3.9 (1.2 to 6.7) | 0.20 | |
| Respiratory | 271 | 56.6±21.9 | 263 | 58.0±22.5 | 0.7 (−2.4 to 3.8) | 0.03 | |
| Digestion | 272 | 80.2±21.6 | 263 | 80.4±19.4 | 1.1 (−1.7 to 3.9) | 0.05 | |
| BMQ-Specific (beliefs about medication): | |||||||
| Concerns | 271 | 2.1±0.5 | 271 | 2.0±0.5 | −0.2 (−0.2 to –0.1) | Decrease (1 to 5) | 0.29 |
| Necessities | 271 | 3.5±0.7 | 271 | 3.7±0.8 | 0.1 (0.0 to 0.2) | Increase (1 to 5) | 0.18 |
| SRBAI (habit strength for using nebuliser) | 271 | 11.7±4.9 | 261 | 12.9±4.9 | 1.2 (0.5 to 1.8) | Increase (4 to 20) | 0.24 |
| Perceptions of treatment adherence (three-item scale) | 245 | 9.9±3.6 | 237 | 10.8±3.3 | 0.7 (0.2 to 1.2) | Increase (3 to 15) | 0.20 |
| Effort of nebuliser treatments (one item) | 270 | 3.0±1.2 | 260 | 3.3±1.3 | 0.3 (0.1 to 0.5) | Increase (1 to 5) | 0.22 |
| Subjective adherence question – % (self-report estimate of adherence) | 267 | 65.6±32.8 | 258 | 68.6±31.3 | 1.9 (−2.8 to 6.6) | Increase (0% to 100%) | 0.06 |
| CHAOS-6 (life chaos or routine) | 272 | 9.6±3.2 | 263 | 9.4±3.4 | −0.2 (−0.6 to 0.3) | Decrease (0 to 24) | 0.05 |
| PAM-13 (health style assessment) | 274 | 64.9±13.0 | 265 | 68.1±15.6 | 3.4 (1.3 to 5.4) | Increase (0 to 100) | 0.23 |
| EQ-5D-5L (generic health status) | 272 | 0.81±0.18 | 264 | 0.84±0.15 | 0.01 (−0.01 to 0.04) | Increase (−0.224 to 1) | 0.09 |
| Patient-reported outcomes – safety measures§ | |||||||
| PHQ-8 (depression) | 272 | 6.4±5.0 | 262 | 6.3±5.6 | −0.1 (−0.8 to 0.7) | Decrease (0 to 24) | 0.01 |
| GAD-7 (anxiety) | 273 | 4.5±4.8 | 262 | 4.9±5.3 | 0.3 (−0.4 to 1.0) | Decrease (0 to 21) | 0.05 |
Full details and references for all patient-reported outcomes are available in the SAP (provided in online supplemental material).
*Exacerbations analysis adjusted for centre and past year intravenous days.
†All other analyses adjusted for past year intravenous days, centre and outcome measure at baseline.
‡Weekly objectively measured effective adherence (sum of doses taken/sum of doses prescribed) averaged over weeks 3–52 postrandomisation.
§All patient-reported outcomes based on points, unless otherwise stated.
BMQ, Beliefs About Medicines Questionnaire; CFQ-R, CF Questionnaire-Revised; CHAOS-6, Confusion, Hubbub and Order 6-item Scale; EQ-5D-5L, EuroQol 5-dimension and 5-level; GAD-7, Generalised Anxiety Disorder seven-item scale; PAM-13, Patient Activation 13-item Measure; PHQ-8, Patient Health Questionnaire eight-item depression scale; SAP, statistical analysis plan; SRBAI, Self-Report Behavioural Automaticity Index.