Table 1.
Completed prospective immune therapy clinical trials involving previously untreated brain metastases
| Study name or ClinicalTrials.gov identifier | Phase | Disease | Intervention | Neurologic symptoms | Steroid | Intracranial response (CR and PR) n (percentage) | Intracranial PFS (months) | Extracranial PFS (months) | Global PFS (months) | Median overall survival (months) | Citation |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Immunotherapy | |||||||||||
| CA184–042 NCT00623766 | 2 | Melanoma | Ipilimumab | Asymptomatic | Prohibited | 8 (16%) | 1.9 | 3.3 | 2.7 | 7 | Margolin et al. |
| Symptomatic | Allowed | 1 (5%) | 1.2 | 1.3 | 1.3 | 3.7 | |||||
| NIBIT-M1 NCT01654692 | 2 | Melanoma | Ipilimumab/Fotemustine | Asymptomatic | Prohibited | 8 (62%) | 3a | N.R. | 3.4a | 12.7a | Di Giacomo et al. |
| NCT02085070 | 2 | Melanoma and NSCLC | Pembrolizumab | Asymptomatic | Prohibited | 6 (26%) | 4 | 6 | 2 | 17 | Goldberg et al. and Kluger et al. |
| CheckMate-204 NCT02320058 | 2 | Melanoma | Ipilimumab/Nivolumab | Asymptomatic | Prohibited | 52 (55%) | 64.2% at 6 months; 59.5% at 9 months | 75.9% at 6 months; 70.4% at 9 months | 61.1% at 6 months; 56.6% at 9 months | 92.3% at 6 months; 82.8% at 9 months; 81.5% at 12 months | Tawbi et al. |
| ABC NCT02374242 | 2 | Melanoma | Ipilimumab/Nivolumab | Asymptomatic | Prohibited | 16 (46%) | N.R. | 13.8 | N.R. | Not Reached at median cutoff 14 months | Long et al. |
| Nivolumab | Asymptomatic | Prohibited | 5 (20%) | 2.5 | 2.6 | N.R. | 18.5 | ||||
| Nivolumab (after local therapy failure or symptoms) | 63% Symptomatic | Prohibited | 1 (6%) | 2.3 | 2.6 | N.R. | 5.1 | ||||
| Immunotherapy with radiation | |||||||||||
| NCT01703507 | 1 | Melanoma | Ipilimumab and WBRT | N.R. | N.R. | 5 Enrolled (no group-specific breakdown provided for response) | 2.53 | N.R. | 2.5 | 8 | Williams et al. |
| Ipilimumab and SRS | N.R. | N.R. | 11 Enrolled (no group-specific breakdown provided for response) | 2.45 | N.R. | 2.1 | Not Reached by median of 10.5 months | ||||
CR, complete response; N.R., not reported; NSCLC, non-small cell lung cancer; PFS, progression free survival; PR, partial response; SRS, stereotactic radiosurgery; WBRT, whole brain radiotherapy.
a
Includes patients with and without prior brain radiotherapy regardless of intracranial progression at enrollment. Data for the untreated brain metastasis cohort was not specifically reported.