Safety and Efficacy of Peptide Receptor Radionuclide Therapy in Multiple Endocrine Neoplasia Syndrome: A

Piyush Aggarwal1, Swayamjeet Satapathy1, Gurjeet Kaur2

  • 1Departments of Nuclear Medicine.

PubMed
Abstract

Insights

Peptide receptor radionuclide therapy (PRRT) with 177 Lu-DOTATATE shows safety and efficacy in advanced neuroendocrine tumors (NETs) within multiple endocrine neoplasia (MEN) syndrome. This treatment offers significant progression-free and overall survival benefits for MEN syndrome patients with inoperable or metastatic NETs.

Area of Science:

  • Endocrinology
  • Nuclear Medicine
  • Oncology

Background:

  • Peptide receptor radionuclide therapy (PRRT) is a standard treatment for advanced neuroendocrine tumors (NETs).
  • Limited data exists on PRRT's efficacy in patients with multiple endocrine neoplasia (MEN) syndrome.
  • This study addresses the need for data on PRRT in MEN syndrome patients with advanced NETs.

Purpose of the Study:

  • To explore the utility and effectiveness of PRRT in patients diagnosed with MEN syndrome and inoperable or metastatic NETs.
  • To evaluate treatment response, survival rates, and adverse events associated with PRRT in this specific patient population.

Main Methods:

  • A retrospective analysis was conducted on MEN syndrome patients who received PRRT.
  • Patients were administered up to 4 cycles of 177 Lu-DOTATATE (5.5–7.4 GBq/cycle) every 8–12 weeks.
  • Treatment response was assessed using RECIST 1.1, and adverse events were graded per CTCAE v5. Progression-free survival (PFS) and overall survival (OS) were calculated.

Main Results:

  • Fifteen MEN syndrome patients (14 MEN-1, 1 MEN-2) with advanced NETs were analyzed.
  • The majority (13/15) had gastroenteropancreatic (GEP) NETs; multifocal primaries were common.
  • Partial response was observed in 43% (6/14) of patients, with an overall disease control rate (DCR) of 86% (12/14). Median PFS was 32.6 months, and the 5-year OS rate was 76.2%.

Conclusions:

  • 177 Lu-DOTATATE PRRT is a safe and effective therapeutic option for advanced NETs in patients with MEN syndrome.
  • The treatment demonstrated favorable response rates and survival outcomes in this cohort.
  • Larger, prospective, multicenter studies are warranted to further validate these findings.

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