PTCH 1 staining of pancreatic neuroendocrine tumor (PNET) samples from patients with and without multiple endocrine neoplasia (MEN-1) syndrome reveals a potential therapeutic target

Figures & data

Table 1. Demographic and clinical data for the study cohort

Mean Age at Biopsy (years ±SD)	50.8 ± 13.7
Duration of Disease at Biopsy (years ± SD)	2.0 ± 5.6
Male (Number [%])	8 (36)
Ethnicity (Number [%])
White	20 (91)
Asian	1 (4.5)
African American	1 (4.5)
Functional Syndrome (Number [%])
Absent	9 (41)
Present	13 (59)
Gastrinoma	7 (32)
Insulinoma	4 (18)
Other (1 VIPoma; 1 Somatostatinoma)	2 (9)
Inherited Syndrome (Number [%])
Absent	17 (77)
Present (all MEN-1)	5 (23)*
Chromogranin A at Biopsy (% above upper level limit)	3686**
Date range from CgA result to Biopsy (days)	0-64
Somatostatin Analog therapy at Biopsy (Number [%])	3 (13)
Duration of therapy at Biopsy (months ± SD)	5 ± 1
Prior Chemotherapy (Number [%])	1 (5)
Prior Liver directed therapy (Number [%])	2 (9)
Prior PRRT (number [%])	1 (5)
Prior surgical resections (Number [%])
None	19 (86)
One	2 (9)
Two	0 (0)
Three or more	1 (5)

* 4 confirmed by genetic testing, 1 unconfirmed clinical diagnosis.

* *11 with no CgA results at Biopsy.

Table 2. Pathology, clinical features and PTCH1 staining results

Subject #	Specimen Size(s) (cm)	Primary Tumor site(s)	Tumor Grade	Differentiation	Lymph Node Metastases (Y/N)	Liver Metastases (Y/N)	Syndrome	CgA [pg/ml] (normal range)	MEN 1 (Y/N)	PTCH1
1	1.4	Duodenum	1	Well	Y	N	Gastrinoma	7663 (0–50 )	N	+++
2	8	Pancreas	2	Moderate	Y	Y	none	258 (0–95)	N	++
3	8	Pancreas	1	Well	Y	N	none	174 (0–95)	N	−
4	5	Pancreas	2	Moderate	Y	N	none	n/a	N	+++
5	0.8	Duodenum	1	Well	Y	N	Gastrinoma	432 (0-375)	N	++
6	7.5	Pancreas	2	Moderate	N	N	VIPoma	n/a	N	+
7	1.3	Pancreas	2	Well	N	N	Somato statinoma	n/a	N	+
8	2	Pancreas	1	Well	N	N	Insulinoma	n/a	N	−
9	1.5	Pancreas	2	Moderate	Y	N	none	n/a	N	++
10	0.6/1.0	Pancreas, Duodenum	1	Well	Y	Y	none	n/a	N	−
11	4.5	Pancreas	2	Well	Y	N*	none	n/a	N	+++
12	1.6	Pancreas	1	Well	Y	N	none	<200 (0–95)	Y	+
13	4.5/0.7	Pancreas, Duodenum	1	Well	Y	N	Gastrinoma	2725 (0–50)	N	−
14	2.5	Pancreas	3	Poor	Y	Y	Insulinoma	15600 (0–95)	N	−
15	1.5	Duodenum	2	Moderate	Y	N	Insulinoma	5.4 (1.9–15.0)	N	−
16	4	Pancreas	2	Moderate	Y	N	none	n/a	N	+++
17	3.1	Unknown	1	Well	Y	N	Gastrinoma	96.0 (n/a)	N	+
18	1.1	Duodenum	1	Well	Y	N	Gastrinoma	1625 (0–115)	Y	++
19	5.5/3.2	Pancreas	2	Well	n/a**	Y	none	48 (0–50)	N	+++
20	n/a	Pancreas, Duodenum	1	Well	Y	N	Gastrinoma	n/a	Y	+++
21	n/a	Pancreas	1	Well	n/a	N	none	n/a	Y	+++
22	1.1/2.2	Pancreas	1	Well	n/a***	N	Insulinoma	n/a	Y	+++

Abbreviations: CgA = chromogranin A; n/a = not available.

* This patient had a perihepatic lymph node identified at surgery but no liver metastases were identified.

* *Away from operative site.

* **Local enucleation.

Figure 1. Immunofluorescence for PTCH 1 in a normal pancreatic islet (A), a tumor not expressing PTCH 1 (B), and a tumor expressing PTCH-1 (C).