Table 2.
Response to Treatment in Symptomatic Patients
| Patients (n = 18) | |||
|---|---|---|---|
| Intracranial | Extracranial | Global | |
| Best overall response, No. (%)a | |||
| Complete response | 2 (11.1) | 0 | 0 |
| Partial response | 2 (11.1) | 4 (22.2) | 4 (22.2) |
| Stable disease ≥ 6 months | 0 | 0 | 0 |
| Progressive disease | 10 (55.6) | 6 (33.3) | 8 (44.4) |
| Not evaluable for CBR: | 4 (22.2) | 8 (44.4) | 6 (33.3) |
| Death prior to first on-study assessment | 2 (11.1) | 1 (5.6) | 1 (5.6) |
| Early discontinuation due to toxicity | 0 | 0 | 0 |
| Stable disease <6 months | 2 (11.1) | 3 (16.7) | 2 (11.1) |
| No extracranial disease at baseline | NA | 1 (5.6) | NA |
| Otherb | 0 | 3 (16.7) | 3 (16.7) |
| ORR, no./No. (%; 95% CI) | 4/18 (22.2; 6.4-47.6) | 4/18 (22.2; 6.4-47.6) | 4/18 (22.2; 6.4-47.6) |
| CBRc, no./No. (%; 95% CI) | 4/18 (22.2; 6.4-47.6) | 4/18 (22.2; 6.4-47.6) | 4/18 (22.2; 6.4-47.6) |
| Median time to response, months (range) | 4.1 (1.0-6.9) | 1.3 (1.0-2.8) | 2.0 (1.0-4.2) |
| Median duration of response, months (95% CI)d | Not reached (0-not reached) | Not reached | Not reached |
| Ongoing response, no./No. (%) | 3/4 (75.0) | 4/4 (100) | 4/4 (100) |
Abbreviations: CBR, clinical benefit rate; CI, confidence interval; CR, complete response; ORR, objective response rate; PD, progressive disease; PR, partial response; SD, stable disease.
aOne patient had a response preceded by initial progressive disease.
bPD not confirmed (n = 1) and insufficient radiographic scan data (n = 2).
cClinical benefit rate = complete response + partial response + stable disease ≥ 6 months.
dOne patient had a response but was not included in the duration of response because no response date was recorded by the investigator.