Summary of the frequency of adverse events (dyspnoea and “abnormal respiration”) in clinical trials and case series of lumacaftor (LUM)/ivacaftor (IVA) in people with advance cystic fibrosis lung disease
| First author [ref.] | Type of trial | Patients | Patients with FEV1 <40% pred | Adverse event frequency in FEV1 <40%: dyspnoea/abnormal respiration | Discontinued LUM/IVA |
| Elborn [15] (TRAFFIC AND TRANSPORT) | RCT | 1072 | 81 | 14 (26)/4 (8)# | 3 (3.4) |
| Popowicz [25] | Open label | NA | 12 | 8 (75) | 3 (25) |
| Hubert [16] | Open label | NA | 53 | 27 (51) | 13 (24) |
| Jennings [20] | Open label | 116 | 19 | 11 (58) | 6 (32) |
| Taylor-Cousar [17] | Open label | NA | 48 | 20 (43)/26 (57) | 8 (17) |
| Murer [18] | Open label | NA | 20 | 1 (5)/1 (5)# | 1 (5) |
| Diab-Cáceres [19] | Open label | NA | 20 | 15 (75) | 7 (35) |
Data are presented as n or n (%). RCT: randomised controlled trial; FEV1: forced expiratory volume in 1 s; NA: not available. #: dyspnoea/chest tightness only.