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This is the study that measured what taking the basal cell carcinoma pill is actually like, in more than a thousand ordinary patients rather than a hundred selected ones. It shrank most tumours. Almost everybody had side effects, and the average person stopped after about eight months.
ERIVANCE licensed vismodegib on 104 patients. STEVIE was built to find out what happens when the same drug is given to everyone who might need it: 1,215 evaluable adults in 36 countries, 1,119 with locally advanced and 96 with metastatic basal cell carcinoma, treated with 150 mg daily until progression or intolerance. Safety was the primary objective, which is unusual and is the point.
The efficacy held. Investigator-assessed objective response was 68.5 per cent in locally advanced disease (95 per cent confidence interval 65.7 to 71.3) and 36.9 per cent in metastatic disease (26.6 to 48.1).
The tolerability is the finding. Ninety-eight per cent of patients had at least one treatment-emergent adverse event and 289 (23.8 per cent) had a serious one. The median treatment duration was 8.6 months, with a range from zero to 44, and only 147 patients (12 per cent) were still on the drug when the analysis was done. The characteristic events are not dangerous but are relentless and affect everything a person does: muscle spasms, loss of taste, hair loss and weight loss. Exposure beyond twelve months did not make them worse or more frequent, and most of the common events that were ongoing when treatment stopped had resolved within twelve months afterwards, which matters for anyone considering a planned break.
This is why drug holidays and intermittent schedules were tested, why a topical hedgehog inhibitor was developed, and why the second line for basal cell carcinoma is a PD-1 antibody rather than another pill down the same pathway.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
1,215 treated.
95 per cent confidence interval 65.7 to 71.3
Source289 of 1,215 patients; any treatment-emergent adverse event in 98 per cent
Source95 per cent confidence interval 26.6 to 48.1
Sourcerange 0 to 44; 147 patients (12 per cent) still on study at the analysis
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Objective response rate, locally advanced basal cell carcinoma (investigator assessed)primary | Vismodegib 150 mg daily | 1,119 | 68.5% | - | - | link |
| Objective response rate, metastatic basal cell carcinoma (investigator assessed) | Vismodegib 150 mg daily | 96 | 36.9% | - | - | link |
| Serious treatment-emergent adverse eventsprimary | Vismodegib 150 mg daily | 1,215 | 23.8% | - | - | link |
| Median duration of treatment | Vismodegib 150 mg daily | 1,215 | 8.6 months | - | - | link |
Shares Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome, Patidegib gel in Gorlin syndrome (phase 2A), Why people stop taking hedgehog inhibitors, Smoothened (hedgehog pathway).
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