Efficacy and Cost-Effectiveness of Immediate Surgery versus a Wait-and-See Strategy for Sporadic Nonfunctioning T1 Pancreatic Endocrine Neoplasms

Abstract

Background: Whether patients with small (<2 cm), sporadic nonfunctioning pancreatic endocrine tumors (NF-PETs) should directly undergo pancreatic surgery or should be followed longitudinally to detect growth and malignancy still has to be defined. Study Design: Based on the pertinent literature of the past decade, a Markov model was developed to investigate this issue. In the wait-and-see strategy arm, surgery was performed if the tumor attained a size ≥2 cm or surpassed 20% of the initial size. In a Monte Carlo probabilistic analysis, 100 hypothetical patients undergoing a wait-and-see strategy were compared to 100 patients directly undergoing surgery, with the aim of investigating the efficacy and cost-effectiveness of the two strategies. Results: During the postdiagnostic lifetime, 63 NF-PETs in the wait-and-see group showed significant growth and underwent surgery: 38 were stage I, 10 were stage II, 15 were stage III and none were stage IV. In the base-case scenario, the mean life expectancy and quality-adjusted life expectancy were found to be superior after immediate surgery [26.1 years and 11.8 quality-adjusted life years (QALYs)] than with the wait-and-see strategy (22.1 years and 8.3 QALYs) as the consequence of ageing during the wait-and-see follow-up which increased mortality due to surgery, when surgery was needed. The model was sensitive to starting age and length of follow-up; in particular, for patients >65 years of age, the two strategies provided similar results but the wait-and-see strategy was more cost-effective. Conclusions: The wait-and-see strategy for NF-PETs <2 cm represents a reasonable approach in patients over 65 years of age; otherwise, immediate surgery is preferable.

Surveillance strategy for small asymptomatic non-functional pancreatic neuroendocrine tumors – a systematic review and meta-analysis

“…Thus, how to best select MEN1 patients for surgery based on age, cut-off size, and/ or location remains to be formally determined. 48 There are several limitations in this systematic review and in the included studies. First, only nine studies were found to meet the criteria for this systematic review.…”

Sporadic non-functioning pancreatic neuroendocrine tumours: multicentre analysis

“…The prognosis of G1 Pan-NET was excellent for all stages and the data confirmed that non-operative management could be considered for stage I disease 21,22 . Indeed, if a conservative strategy in stage I was adopted and an earlier stage shift in stage II or III was observed, the prognosis remained good 23 . Normal lifespan probability and excess death risk histograms (Fig.…”

“…On the contrary, all patients with G1 and G2 Pan-NETs in stage III-IV had a significant risk of recurrence despite surgery, so an adjuvant or neoadjuvant approach might be sensible. A hypothetical shift from stage I to stage III during conservative management of small G1 neoplasms would be dangerous, but the probability of this happening is very low 23 . The G3 Pan-NET category had a RS of over 70 per cent only in stage I, suggesting that adjuvant or neoadjuvant therapy should be considered as a rational approach from stage II onwards.…”

Consensus of clinical diagnosis and treatment for non-functional pancreatic neuroendocrine neoplasms with diameter <2 cm

“…Surgery can be considered if the patient has poor compliance or has significant concerns about long-term follow-up or tumor progression. Since elderly patients with small pNENs benefit little from surgery, active follow-up is preferred for patients over 70 years old at the first visit [48] (or over 65 years old [49] ) and those with more complications, while younger patients should be more actively treated with surgery. [50,51] However, for elderly patients with tumor progression tendency, surgery may also be actively performed to avoid a gradual decrease in surgical tolerance or a change of surgical procedure due to tumor progression.…”