Table 1.
Effects table for avapritinib in studies BLU-285-1101 [cut-off date (COD) for efficacy: 17 April 2020, COD for safety: 9 March 2020] and study BLU-285-1303 (COD: 9 March 2020)
| Effect | Unit | Results | Uncertainties/Strength of evidence | ||
|---|---|---|---|---|---|
| PDGFRA D842V-mutated population in BLU-285-1101 study (n = 38) | ORR Median DOR Median PFS Median OS |
% (95% CI) Months (95% CI) Months (95% CI) Months (95% CI) |
95 (82.3-99.4) 22.1 (14.1-NE) 24 (18.4-NE) Not reached [70.6% (55.2-86.0) at 36 months] |
||
| PDGFRA D842V-mutated population in BLU-285-1303 study (n = 13) | ORR Median PFS |
% Months (95% CI) |
Avapritinib (n = 7) | Regorafenib (n = 6) | These data are preliminary and limited as the study is ongoing |
| 43 NE (9.7-NE) |
0 4.5 (1.7-NE) |
||||
| All AEs | Treatment-related AEs | ||||
| General safety profile | AEs overall Grade ≥3 AEs/SAEs AEs leading to death AEs leading to treatment discontinuation |
% | 98.4 75.1/49.8 8.0 18.0 |
93.8 55.5/23.1 0.4a 10.9 |
aIncludes reports of disease progression |
| Gastrointestinal AEs | Overall Grade ≥3 AEs/SAEs Nausea Vomiting Diarrhoea Abdominal pain |
% | 61.5 5.5/3.1 45.1 24.2 26.4 10.9 |
SAEs include gastrointestinal bleeding | |
| Cytopenia AEs | Overall Grade ≥3 AEs/SAEs Grade ≥3 anaemia Grade ≥3 neutropenia |
% | 44.5 27.1/6.4 20.0 8.9 |
||
| Fluid retention AEs | Peripheral oedema Facial oedema Periorbital oedema Pleural effusion |
% | 22.5 26.5 32.9 6 |
||
| Cognitive AEs | Overall Grade ≥3 AEs/SAEs Memory impairment Cognitive disorders Confusional status Encephalopathy |
% | 33.1 2.2/1.3 20.2 11.8 4.7 0.9 |
||
| Haemorrhage | Overall Grade ≥3 AEs/SAEs Intracranial |
% | 3.8 2.0/2.0 1.6 |
||
AEs, adverse events; CI, confidence interval; DOR, duration of response; NE, not estimable; ORR, overall response rate; OS, overall survival; PDGFRA, platelet-derived growth factor receptor alpha; PFS, progression-free survival; SAEs, serious adverse events.