Table 1.
Patients’ clinical information and in vitro response to modulators.
| Subject (Gender) |
Genotype | Functional class | Age at sampling procedure | Pancreatic function |
FEV1% | Bronchiectasis | Chronic colonization | In vitro response to modulators |
|---|---|---|---|---|---|---|---|---|
| CF1 (M) | F508del/2183AA>G | II/I | 15 | Insufficiency | 45 | Yes | SA-PA | + + |
| CF2 (F) | F508del/2183AA>G | II/I | 17 | Insufficiency | 85 | Yes (mild) | No | + |
| CF3 (M) | F508del/3849 + 10KbC>T | II/V | 6 | Sufficiency | 110 | No | No | + + * |
| CF4 (M) | L227R/L227R | Undetermined | 19 | Insufficiency | 80 | Yes | MRSA | - |
| CF5 (F) | G542X/R117H-7H | I/IV | 12 | Sufficiency | 116 | No | No | - |
| CF6 (M) | F508del/F508del | II | 18 | Insufficiency | 57 | Yes | SA-PA | + + |
| CF7 (M) | F508del/1812-1G>A | II/I (canonical splicing) | 18 | Insufficiency | 86 | Yes | SA | - |
CF1 and CF2 subjects share the same genotype but are unrelated. Pathogenic variants are indicated with their legacy names.
Gender: M (male) F (female); Chronic colonization: SA (Staphylococcus aureus), MRSA (methicillin resistant Staphylococcus aureus), SA-PA (Staphylococcus aureus and Pseudomonas aeruginosa). Last column describes in vitro nasospheroid response to VX-770 + VX-809 and/or VX-770 + VX-661 assessing FR and AUC analysis in IFRA after live-cell imaging assay (t = 60 min). Responses were categorized according to the statistical differences found between FSK and VX treatment: + + notable response = observed in both, FR and AUC values; + moderate response = significant differences in FR or AUC values; − no response = no significant differences. * Only responder to VX-770 + VX-661.