Predicted number of hospital and home IV antibiotic days in the next year for example patients
| (A) Sets of patient characteristics A, B, C. All are aged 30 and female. | |||
| Predictor | A | B | C |
| FEV1% | 40 | 60 | 80 |
| FEV1% previous year | 40 | 60 | 80 |
| Body mass index | 20 | 22 | 25 |
| Pseudomonas aeruginosa | Yes | Yes | No |
| Staphylococcus aureus | Yes | Yes | No |
| Burkholderia cepacia | Yes | Yes | No |
| CF-related diabetes | Yes | Yes | No |
| Hospital-IV-days, past year | 28 | 14 | 0 |
| Home-IV-days, past year | 28 | 14 | 0 |
| (B) Predicted number of hospital and home IV antibiotic days in the next year for nine example patients under no treatment effects, and imposing the effects of TEZ/IVA and ELEX/TEZ/IVA using approaches 1 and 2. Each set of characteristics A, B, C is considered in combination with each genotype category (1), (2), (3). | ||||||||||
| Genotype (1): F508del homoz (eligible for TEZ/IVA and ELEX/TEZ/IVA) | Genotype (2): F508del heteroz+minimal (eligible for ELEX/TEZ/IVA) | Genotype (3): Gating (eligible for IVA) | ||||||||
| No treatment effects | +TEZ/IVA* | +ELEX/TEZ/IVA+ TEZ/IVA† | No treatment effects | +TEZ/IVA* | +ELEX/TEZ/IVA+ TEZ/IVA† | No treatment effects | +TEZ/IVA* | +ELEX/TEZ/IVA+ TEZ/IVA† | ||
| Hospital-IV-days | ||||||||||
| Approach 1 | A | 34.2 | 31.8 | 25.3 | 34.6 | – | 25.6 | 28.6 | – | – |
| B | 14.5 | 13.4 | 10.5 | 14.7 | – | 10.6 | 11.0 | – | – | |
| C | 1.8 | 1.6 | 1.2 | 1.8 | – | 1.2 | 1.2 | – | – | |
| Approach 2 | A | 34.2 | 22.2 | 12.7 | 34.6 | – | 12.8 | 28.6 | – | – |
| B | 14.5 | 9.4 | 5.4 | 14.7 | – | 5.4 | 11.0 | – | – | |
| C | 1.8 | 1.2 | 0.7 | 1.8 | – | 0.7 | 1.2 | – | – | |
| Home-IV-days | ||||||||||
| Approach 1 | A | 25.2 | 24.1 | 21.1 | 24.0 | – | 20.1 | 18.1 | – | – |
| B | 13.2 | 12.7 | 11.4 | 12.5 | – | 10.7 | 8.7 | – | – | |
| C | 5.2 | 5.1 | 4.7 | 4.9 | – | 4.4 | 3.2 | – | – | |
| Approach 2 | A | 25.2 | 16.4 | 9.3 | 24.0 | – | 8.9 | 18.1 | – | – |
| B | 13.2 | 8.6 | 4.9 | 12.5 | – | 4.6 | 8.7 | – | – | |
| C | 5.2 | 3.4 | 1.9 | 4.9 | – | 1.8 | 3.2 | – | – | |
Values of model predictors were chosen for nine example patients defined by three sets of patient characteristics (A, B, C), each combined with three different genotypes: (1) F508del homozygous (eligible for TEZ/IVA and ELEX/TEZ/IVA), (2) F508del heterozygous with a minimal function mutation (eligible for ELEX/TEZ/IVA only), (3) any gating mutation (assumed to be using ivacaftor, but not eligible for TEZ/IVA or ELEX/TEZ/IVA).
*With TEZ/IVA effect applied to eligible individuals.
†With ELEX/TEZ/IVA effect applied to eligible individuals, including those assumed to switch from TEZ/IVA.
ELEX, elexacaftor; IVA, ivacaftor; TEZ, tezacaftor.