PRRT achieves disease control in 65% of patients with metastatic phaeochromocytoma/paraganglioma | Research

The takeaway

In 20 patients with metastatic phaeochromocytoma/paraganglioma, 177Lu-DOTATATE PRRT achieved 60% stable disease and 5% partial response over median 29-month follow-up, with no grade 3/4 toxicity and improved quality of life.

65% disease control
60% stable disease + 5% partial response with PRRT in heavily pretreated metastatic phaeochromocytoma/paraganglioma patients

What the researchers found

60% stable disease, 5% partial response, 35% progressive disease. Median PFS 24 months (overall). No grade 3/4 cytopenia or nephrotoxicity. Quality of life improved (declining symptom scores on QLQ-GINET21). 65% had ≥2 prior treatment lines.

Why it matters

Metastatic phaeochromocytomas and paragangliomas have limited treatment options. PRRT offers a targeted approach with a favorable safety profile, achieving disease control in most patients and improving quality of life even in heavily pretreated cases.

How the study worked

Retrospective single-center analysis of 20 patients with metastatic PCC/PGL receiving ≥2 cycles of 177Lu-DOTATATE at an ENETS Centre of Excellence. Assessed radiological, biochemical, clinical response, PFS/OS by Kaplan-Meier, and HRQoL by EORTC QLQ-GINET21.

What this study cannot tell us

Retrospective single-center study. Small sample (n=20). No control group. Heterogeneous prior treatments. SDHx stratification limited by small subgroups.

How to read the evidence

Small retrospective single-center study. Provides meaningful clinical data for a rare tumor type but limited by sample size and design.

When this study was published

Published in 2025.

The bigger picture

This extends PRRT evidence into another rare tumor type beyond traditional neuroendocrine tumors. The 65% disease control rate in heavily pretreated patients highlights PRRT's value as a later-line therapy for SSTR-positive tumors.

Questions still open

Should PRRT be moved earlier in the treatment sequence for mPPGL?
Do SDHx-positive patients genuinely respond better or is this due to different natural history?
Could combination PRRT with other therapies improve outcomes?

Common questions

What are phaeochromocytomas and paragangliomas?

These are rare tumors that arise from cells of the adrenal glands (phaeochromocytomas) or nerve tissue along the spine and abdomen (paragangliomas). When metastatic (spread to other organs), they are difficult to treat and have limited therapeutic options.

How well does PRRT work for these tumors?

In this study, PRRT controlled the disease in 65% of patients (mostly stable disease), with a median time before progression of 24 months. Importantly, it was well-tolerated with no severe side effects and improved patients' quality of life, even in those who had failed multiple prior treatments.

Read the original research

177Lu-DOTATATE peptide receptor radionuclide therapy (PRRT) in metastatic phaeochromocytomas and paragangliomas (mPPGL): a single centre retrospective analysis of experience at an ENETS Centre of Excellence.

Endocrine oncology (Bristol, England), 5(1), e250019

View on PubMed ↗Publisher’s paper ↗

Citation

Shekhda, Kalyan Mansukhbhai; Armeni, Eleni; Xu, Yiwang; D'afflitto, Manfredi; Hayes, Aimee; Mandair, Dalvinder; Yu, Dominic; Quigley, Ann-Marie; Navalkissoor, Shaunak; Gnanasegaran, Gopinath; Grossman, Ashley B; Caplin, Martyn; Toumpanakis, Christos; Khoo, Bernard. (2025). 177Lu-DOTATATE peptide receptor radionuclide therapy (PRRT) in metastatic phaeochromocytomas and paragangliomas (mPPGL): a single centre retrospective analysis of experience at an ENETS Centre of Excellence.. Endocrine oncology (Bristol, England), 5(1), e250019. https://doi.org/10.1530/EO-25-0019