Table 2.
Response and adverse reactions to targeted molecular therapy in BRAF-V600E–positive papillary craniopharyngiomas
| Case | Age | Gender | Previous Treatments + Treatment After Targeted Therapy | Drugs and Total Duration of Treatment | Response to Treatment | Adverse Reactions |
|---|---|---|---|---|---|---|
| Aylwin et al [5] | 57 (27 at diagnosis) | Female | Surgery × 2, RT + surgical repair for CSF leak | Vemurafenib 960 mg bid; total: 10 m, 3-m interruption after 3 m | Initial—PR (MRI—near total); after interruption, PR with tumor regrowth after 7 m | CSF leak with meningitis from tumor reduction |
| Brastianos et al [6] | 39 | Male | Surgery × 5 + surgery and RT at day 38 | Dabrafenib 150 mg bid; trametinib 2 mg bid after 21 days; total: 1.7 m | PR (MRI—85% and 81% reduction in solid and cystic component at 35 days stable disease after 18 m. | Low-grade fever |
| Roque et al [7] | 47 | Female | Surgery, ommaya for cyst aspiration and RT | Dabrafenib 150mg bid + trametinib 2 mg bid; total: 7 m | CR (MRI—disappearance of the tumor) clinical improvement | Intermittent fever |
| Rostami et al [8] | 65 | Male | Surgery | Dabrafenib 150 mg bid; trametinib 2 mg bid after 21 days; total: 3.5 m | PR (MRI—near total 91% reduction) clinical improvement | Fever |
| Himes et al [9] | 52 (47 at diagnosis) | Male | Surgery, RT | Dabrafenib 150 mg bid; dose reduction after several weeks; total: 12 m | PR (decrease in tumor size at 6 m); stable disease 1 year off therapy | Arthralgia |
| Juratli et al [10] | 21 | Male | Surgery (biopsy) | Dabrafenib 150 mg bid; trametinib 2 mg daily; total: 6 m (ongoing) | PR (MRI > 80% response at 6 m) | |
| Berstein et al [11] | 60 | Male | Surgery × 4, RT | Dabrafenib 150 mg bid; trametinib 2 mg daily after 14 days; total: 28 m (ongoing) | CR (100% tumor reduction at 2 m); best clinical response after 3 m | Verrucal keratosis |
| Rao et al [12] | 35 | Male | Shunt for hydrocephalus Craniotomy | Dabrafenib 150 mg bid; total: over 24 m | PR (MRI at 2 m); CR of solid component at 24 m | None |
| Chik et al | 37 (10 at diagnosis) | Male | 4 prior surgeries + 2 surgeries followed by RT and gamma knife | Vemurafenib 960 mg bid; intermittent dose reduction after 3.7 m, 1.5 m interruption after 14.7 m; total: 4 0m (ongoing) | PR (MRI, 55% tumor reduction at 15 m); after interruption, similar reduction after 0.5 m | Arthralgia, myalgia, elevated liver enzymes, severe sun sensitivity |
Abbreviations: CR, complete response; CSF, cerebrospinal fluid; m, months; PR, partial response; RT, radiotherapy.