Table 3.

Overview on studies with targeted therapies in patients with neuroendocrine tumors.

First Author and Year	patients	Number of Patients	Regimen	PD Prior to Treatment	Design	PR	TTP/PFS	Additional Remarks
Hobday 2007 [45], (abstract)	carcinoid	50	sorafenib 400 mg bid	no	phase II	10%	7.8 months	43% grade 3/4 toxicity
	pNET	43					11.9 months
Yao 2008 [46]	carcinoid	22	octreotide + bevacizumab	no	randomized phase II	18%	95% at week 18
		22	octreotide + PEGIFN			0%	68% at week 18
Kulke 2008 [47]	carcinoid	41	sunitinib 37.5 mg	no	phase II	2.4%	10.2 months
	pNET	66				16.7%	7.7 months
Raymond 2011 [48]	pNET	171	sunitinib 37.5 mg	yes	randomized phase III, placebo-controlled	9.3%	11.4 months versus 5.5 months (placebo)	340 planned patients; survival advantage
Phan 2010 [49], (abstract)	carcinoid	20	octreotide + pazopanib 800 mg	no	phase II	0%	12.7 months	grade 3/4 hypertension 11.7%
	pNET	31				19%	11.7 months
Duran 2006 [40]	GEPNET	37	temsirolimus	yes	phase II	6%	6 months
Yao 2008 [41]	carcinoid	30	5–10 mg everolimus + octreotide	no	phase II	17%	63 weeks	trend to better results at 10 mg dose level
	pNET	30				27%	50 weeks
Yao 2010 [42]	pNET	115	10 mg everolimus	yes	phase II	9.7%	9.7 months	2 strata, no randomization
		45	10 mg everolimus + octreotide			4.4%	17 months
Yao 2011 [43]	pNET	410	10 mg everolimus versus placebo	yes	randomized phase III, placebo-controlled	5%	11.4 months versus 5.4 months (placebo)
Pavel 2010 [44], (abstract)	carcinoid syndrome	429	10 mg everolimus + octreotide versus placebo + octreotide	yes	randomized phase III, placebo-controlled		16.4 months versus 11.3 months	only 50% intestinal primary, mixed population

PD: progressive disease; PR: partial remission; TTP: time to progression; PFS: progression-free survival; GEPNET: gastroenteropancreatic neuroendocrine tumor; pNET: pancreatic neuroendocrine tumor; PEGIFN: pegylated interferon-α.