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Long-term survival benefit of liver transplantation with respect to nontransplant options opens new perspectives for properly selected patients with neuroendocrine tumors

PRESS RELEASE (11/15/2013) – Up to now, liver transplantation for patients with neuroendocrine tumors of gastroentero-pancreatic (GEP-NET) has been controversial because accepted selection criteria are lacking, benefit in survival is unproven, and allocation of donated liver grafts for these patients still does not rely on reproducible results. Nontransplant treatment options for these patients may prolong survival but are palliative.

Researchers at the Italian National Cancer Institute in Milan examined data from 280 patients who were referred for liver transplant. Of those, 192 were assigned to nontransplant options while 88 were eligible for transplant according to predetermined restrictive criteria. Of those, 42 patients underwent transplantation (mostly with suboptimal grafts) while 46 did not get a liver because of graft unavailability, competitive indications, patients’ reasons, etc. The two groups carrying the same tumor burden were compared after being followed-up for 10 years.

Statistical analysis demonstrated a significant propensity for survival advantage for those who had transplantation vs. who had not. Overall survival 5 and 10 years after transplantation was 96.8 and 88.9 percent, respectively. Notably, the survival benefit associated with transplantation in patients receiving a new liver a decade before exceeded 3 years of life gained (38.4 months) with respect to any alternative treatment. According to the authors, this could justify enlisting NET patients for transplantation as Model for End-Stage Liver Disease (MELD) exceptions.

According to Vincenzo Mazzaferro, “The most surprising result was the unprecedented quantification of the magnitude of the transplant benefit in patients with NET selected according to restrictive criteria. Interestingly, the gain in life measured at 5 years from transplant was suboptimal – about 7 months – and barely justified transplant consideration. Conversely, the 3 years and a half gained at the 10 years follow-up interval tells us that the long natural history of liver metastases from NET has impeded until now the appreciation of the proper time interval needed to assess treatment success of a complex therapy such as transplantation.”

“Our investigation represents the best possible evidence in favor of transplantation for NET,”  Dr. Mazzaferro concludes, “I believe that the study from Milan gives the best evidence to date supporting the principle that liver replacement may benefit certain categories of patients with liver metastases from NETs. This is unprecedented and has potential for radical changes in the treatment algorithm of patients with this disease and for the liver transplant organization and allocation authorities.”

The abstract entitled “Magnitude of survival benefit after Liver Transplantation (LT) for metastatic neuroendocrine tumors (NET) of gastroentero-pancreatic (GEP) origin” has been recently presented at the Plenary Session of the Liver Meeting® (AASLD Annual Meeting, Washington, November 3, 2013) which brought together more than 9,000 researchers from 55 countries.