IVA | | | | | | | | |
Ramsey [6] | G551D/any; RCT | 10.6%# | 55%# | NA | NA | NA | NA | NA |
De Boeck [13] | Non-G551D gating/any; RCT | 10.7%# | NA | 0 | NA | NA | NA | NA |
Hebestreit [10] | G551D/any; open, EAP | NA | NA | 14 (100%) | 5.2±5.6¶; median 3.9 (−4.1–16.8)+ | NA | 2.1±2.4 kg¶; median 1.1 (−0.3–6.3) kg+ | NA |
Barry [9] | G551D/any; open, EAP | NA | NA | 21 (100%) | 4.2§ | 49%§,f | 1.8 kg§; median 2.3 (−0.4–4.2) kg+ | NA |
Taylor-Cousar [11] | G551D/any; open, EAP | NA | NA | 44 (100%) | 4.8±0.5¶; (−13.1–22.7)+ | NA | 3.3±4.0 kg¶; (−2.3–14.4) kg+ | NA |
Salvatore [12] | Non-G551D gating/any; open, EAP | NA | NA | 13 (100%) | 11.5§ | 51%§,¶¶ | 3.0 kg§ | NA |
LUM/IVA | | | | | | | | |
Wainright [14] and Elborn [15] | F508del/F508del; RCT, subgroup analysis | 2.6–4.0%# | 39–61%# | 81 (7.3%) | 3.3 (0.2–6.4)– 3.7 (0.5–6.9)#,++ | 53–41%#,§ | BMI 0.3 (−0.2–0.8) and 0.6 (0.1–1.2) kg·m−2#,++ | 3.3 (5.2–11.7) and −4.2 (−12–3.7)#,++ |
Hubert [16] | F508del/F508del; open | NA | NA | 53 (37 completed 3 months) | 3.19§ | NA | BMI 0.03 kg·m−2§ | NA |
Taylor-Cousar [17] | F508del/F508del; open | NA | NA | 46 (35 completed 24 weeks) | −0.4 (−1.9–1.1)#,++ | 64%§,¶¶ | BMI 0.29 (0.17) kg·m−2§,++ | 2.5 (−1.0–5.9)++ |
Murer [18] | F508del/F508del; open | NA | NA | 20 (10 completed) | +2.5## | 60%§,¶¶ | BMI 0.9 kg·m−2## | NA |
Diab-Cáceres [19] | F508del/F508del; open | NA | NA | 20 | | 61%§,¶¶ | | |
TEZ/IVA | | | | | | | | |
Taylor-Cousar [21] | F508del/F508del; RCT | 4.0 (3.1–4.8)#,++ | 36%#,++ | 27 (9.4%) | 3.5 (1.0–6.1)#,++ | NA | NA | NA |
Rowe [22] | F508del/RF; RCT (crossover) | 6.8 (5.7–7.8)#,++ | 46% | 22 (9%) | 4.4 (1.1–7.8)#,++ | NA | NA | NA |
ELX/TEZ/IVA | | | | | | | | |
Middleton [23] | F508del/MF; RCT | 13.8 (12.1–15.4)#,++ | 63% | 18 (9%) | 15.2 (7.3–23.1)#,++ | NA | NA | NA |