Table 3.

Patients with a rare tumor treated on a targeted agent clinical trial and their best response

DiseaseAberrationTherapyBest response
CholangiocarcinomaBRAFV600EBRAFi (26)PR
ECDBRAFV600EBRAFi (26)PR
Glioblastoma multiformeBRAFV600EBRAFi (26)PR
Adenoid cystic carcinomaPIK3R1AKTi (27)SD × 24 cycles
E515fs*1
Adenoid cystic carcinomaKIT and PDGFRA amplificationKITi + PDGFRAi (28)SD × 7 cycles, continues on trial
Salivary gland adenocarcinomaAKT1 E17KAKTi (27)SD × 6 cycles
Salivary gland adenocarcinomaBRAFV600EBRAFi (26)PR
ECDBRAFV600EBRAFi (26)SD × 3 years
Metaplastic breast carcinomaPIK3R1Chemo + mTORi (29)SD × 7 cycles
Y580fs*19
Metaplastic breast carcinomaPIK3CAChemo + mTORi (29)SD × 8 cycles
H1047R
Ovarian clear cell carcinomaPIK3CA H1047RChemo + mTORi (29)PD
Prostate neuroendocrine carcinomaPTEN lossChemo + mTORi (29)PD
Lung large cell neuroendocrine carcinomaPIK3CA amplificationPI3Ki (30) + MEKi (31)PD
  • Abbreviations: ECD, Erdheim–Chester disease; i, inhibitor; PD, progressive disease; PR, partial response; SD, stable disease.