Table 1.

Summary of targeted molecular agents trialed in skull-base chordomas

Agent(s)	Study	Mechanism of action	Type of study	N	Tumor location	Results	Comments
Cetuximab and gefitinib	Hof et al110	EGFR inhibitor	Case report	1	Sacral	Partial response over 9 months
Cetuximab and gefitinib	Lindén et al93	EGFR inhibitor	Case report	1	Cervical	Initial symptomatic improvement; partial response over 4 months
Erlotinib	Singhal et al66	EGFR inhibitor	Case report	1	Sacral	Initial symptomatic improvement; 30% reduction in tumor bulk at 3 months
Erlotinib	Launay et al94	EGFR inhibitor	Case report	1	Sacral	Initial symptomatic improvement; partial response or stable metastatic disease at 12 months
Lapatinib	Stacchiotti et al111	EGFR inhibitor	Phase II study	18	Sacral (n=12), clivus (n=2), and mobile spine (n=4)	Six of 18 (33.3%) partial response Seven of 18 (38.9%) stable disease PFS 6 months
Imatinib	Casali et al97	PDGFR inhibitor	Case series	6	Sacral (n=5) and clival (n=1)	Five of six (83.3%) symptomatic improvement; six of six (100%) had radiologic effect over weeks to months
Imatinib	Stacchiotti et al100	PDGFR inhibitor	Phase II study	56	Sacral (n=33), mobile spine (n=14), and skull base (n=9)	One of 50 (2%) with partial response, and 35 of 50 (70%) with stable disease at 6 months
Rapamycin	Stacchiotti et al99	mTOR inhibitor	Case series	10	Sacral (n=9) and skull base (n=1)	One of nine (33.3%) had partial response, seven of nine (77.8%) had stable disease, and one of nine (11.1%) had progressive disease at 3 months	Rapamycin used in combination with imatinib
Rapamycin	Ricci-Vitiani et al106	mTOR inhibitor	Case report	1	Skull base	Sixfold decrease in tumor-growth rate over 10 months compared to pretreatment
Bevacizumab	Asklund et al108	VEGF inhibitor	Case series	3	Sacral (n=1) and skull base (n=2)	Stable disease over 4.5 years (n=1), 27 months (n=1), and 24 months (n=1)	Bevacizumab used in combination with erlotinib

Abbreviation: PFS, progression-free survival.