Peptide receptor radionuclide therapy with ¹⁷⁷ Lu-DOTATATE in pediatric, adolescent, and young adult neuroendocrine tumors: A single-center experience from Bambino Gesù Children’s Hospital.

Abstract

10027 Background: Neuroendocrine tumors (NETs) in pediatric, adolescent, and young adult (AYA) patients are rare and often associated with hereditary syndromes such as Von Hippel Lindau (VHL) and type 1 neurofibromatosis (NF1). Peptide receptor radionuclide therapy (PRRT) with ^177Lu-DOTATATE is well established in adults with somatostatin receptor–positive NETs, but data in younger populations remain limited. We report our single-center experience evaluating feasibility, safety, and efficacy of PRRT in this age group. Methods: We retrospectively analyzed patients <20 years treated with ^177Lu-DOTATATE at Bambino Gesù Children’s Hospital between 2019 and 2025. Eligibility required somatostatin receptor expression confirmed by PET-DOTA imaging (Krenning score ≥2). Data included demographics, age at diagnosis and PRRT, tumor characteristics, prior therapies, PRRT cycles, cumulative dose, toxicity, and best response per RECIST. Follow-up status was assessed at last visit. Results: Six patients (median age at diagnosis 11 years, range 7–16; median age at PRRT 15 years, range 10–19) received PRRT for metastatic or unresectable NETs (pheochromocytoma/paraganglioma, n=5; bronchial carcinoid, n=1). All completed 4 cycles of ^177Lu-DOTATATE (median cumulative dose ~30 GBq). Prior treatments included surgery, chemotherapy (temozolomide), and MIBG therapy. No grade ≥3 hematologic or renal toxicity occurred; treatment was well tolerated. Best responses: 4 partial responses (PR), and 2 stable disease (SD). At a median follow-up of 18 months, all patients were alive: one in complete remission after surgery, others with stable disease or ongoing therapy. Genetic predisposition was present in 3 patients (VHL, NF1). SUVmax on baseline PET ranged from 1.2 to 43.8, correlating with Krenning score ≥2 in all cases. Conclusions: PRRT with ^177Lu-DOTATATE is feasible and well tolerated in pediatric, adolescent, and young adult patients with somatostatin receptor–positive NETs, achieving meaningful disease control with minimal toxicity. This single-center experience supports PRRT as a valuable option in selected cases and highlights the need for prospective studies and age-specific guidelines.