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

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 &lt;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.

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Journal
Journal of Clinical Oncology
Published
2026-05-27
DOI
https://doi.org/10.1200/jco.2026.44.16_suppl.10027
Primary Topic
Neuroendocrine Tumor Research Advances
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article
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article

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.

Aurora Castellano, Claudio Altini, Maria Debora De Pasquale, Maria Felicia Villani et al.
Journal of Clinical Oncology
Neuroendocrine Tumor Research Advances
article

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.

Aurora Castellano, Claudio Altini, Maria Debora De Pasquale, Maria Felicia Villani, Maria Carmen Garganese, Milena Pizzoferro, Saadi Sollaku, Alessandro Crocoli, Evelina Miele
article en

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 &lt;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.

Journal of Clinical OncologyVol. 44(16_suppl)
Bambino Gesù Children's Hospital (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT)
Good health and well-being
Openalex Percentile: Top 7%
Neuroendocrine Tumor Research Advances
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