Clinical trial NCT03084770: Asymptomatic Small Pancreatic Endocrine Neoplasms.
Official title: A Prospective Evaluation of the Management of Sporadic Asymptomatic Nonfunctioning Pancreatic Neuroendocrine Neoplasms ≤ 2 cm
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Non Functioning Pancreatic Endocrine Tumor
Keywords: NF-PNEN
Lead sponsor: IRCCS San Raffaele
Sponsor class: OTHER
First posted: 2017-03-21
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with a newly diagnosis (less thn 12 months) of non- functioning pancreatic endocrine neoplasia (NF-PNEN) ≤ 2 cm of diameter. Either patients submitted to surgery or in active surveillance can be enrolled. Patients with sindrome or genetics disorders are excluded from the study.

Brief summary: The aim of the study is to evaluate the most appropriate management of sporadic asymptomatic non-functioning pancreatic neuroendocrine neoplasms (NF-PNEN) ≤ 2 cm. P NF-PNEN management will be decided at the hospital and all therapeutics decision will be decided/coordinated by the treating physician.

Patients will be either submitted to surgical resection or to active surveillance.

Detailed description: In the last decade a dramatic increase in diagnosis of small, incidentally discovered, NF-PNEN was observed. Various study indicates the safety of a conservative management for this lesion and the The European Neuroendocrine Tumor Society (ENETS) proposed a "wait and see" approach for small NF-PNEN.

Indications for surgery include the presence of a localized NF-PNEN in the absence of distant metastases as curative resection of these tumors is associated with favourable prognosis especially for low grade.

In the last decade a dramatic increase in diagnosis of small, incidentally discovered, NF-PNEN was observed.Moreover, other investigators observed a clear relationship between the tumor diameter and low risk of malignancy and systemic progression.

In particular, a tumor size ≤ 2 cm seems to be associated with a negligible risk of disease recurrence and with a very low incidence of aggressive features such as lymph node involvement.On this basis, the European Neuroendocrine Tumor Society (ENETS) proposed a "wait and see" approach for small NF-PNEN when incidentally discovered. Since then, various series evaluated the safety of a conservative management for small, sporadic, incidentally diagnosed, NF-PNEN.

After a median follow-up of 28-45 months, all the studies confirmed that an intensive surveillance for incidental and small NF-PNEN is safe in selected cases.

Nevertheless, available data are based only on retrospective series with a significant heterogeneity of inclusion criteria and different tumor diameter cut-off and the appropriate management of this entities (surveillance versus surgery) is still a matter of debate.

Eligibility criteria: Inclusion Criteria:

* Age \> 18 years
* Individuals with asymptomatic sporadic NF-PNEN ≤ 2 cm
* Diagnosis has to be proven by a positive fine-needle aspiration (FNA) or by the presence of a measurable nodule on high-quality imaging technique (CT or MR) that is positive at 68Gallium DOTATOC-PET scan or Octreoscan.
* Patients who undergo surgery for NF-PNEN\<2cm within 12 months. In these cases, diagnosis has to be proven by histological confirmation of NF-PNEN
* Informed consent

Exclusion Criteria:

* NF-PNEN \> 2 cm of maximum diameter
* Presence of genetic syndrome (MEN1, VHL, NF)
* Presence of symptoms (specific symptoms suspicious of a clinical syndrome related to hypersecretion of bioactive compounds) or unspecific symptoms

Primary outcomes:
measure: Disease/progression-free survival of NF-PNEN ≤ 2 cm
timeFrame: From date of enrolment until the date of first documented progression or first evidence of recurrence, from 6 months up to 6 years.
Secondary outcomes:
measure: Frequency of NF-PNEN ≤ 2 cm
timeFrame: 6 years
measure: Outcome of surgical intervention of NF-PNEN ≤ 2 cm
timeFrame: from the date of surgery to 1 months later the surgery
measure: Epidemiology of patients submitted to surgical intervention for NF-PNEN ≤ 2 cm
timeFrame: from the date of surgery, up to 6 years
measure: Evolution of NF-PNEN ≤ 2 cm
timeFrame: From date of enrolment until the date of first documented radiological evolution, from 6 months up to 6 years.
measure: Quality of Life of NF-PNEN ≤ 2 cm
timeFrame: from 6 months up to 6 years.
Other outcomes:
Locations:
city: Milan
country: Italy
facility: IRCCS San Raffaele Hospital
Study officials:
affiliation: IRCCS San Raffaele
name: Massimo Falconi, Professor
role: PRINCIPAL_INVESTIGATOR
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