Leukemia Developed During Peptide Receptor Therapy for Neuroendocrine Tumor — Successfully Treated Without Chemotherapy | Research

The takeaway

An elderly woman receiving peptide receptor radionuclide therapy (PRRT) for a pancreatic neuroendocrine tumor developed secondary acute myeloid leukemia, which was successfully treated with a non-chemotherapy regimen that also controlled her original tumor.

Dual remission achieved
Both acute myeloid leukemia and the neuroendocrine tumor responded to a non-chemotherapy regimen of azacitidine and venetoclax

What the researchers found

The patient, an elderly woman with a metastatic pancreatic neuroendocrine tumor (pNET), developed acute myeloid leukemia (AML) while receiving peptide receptor radionuclide therapy (PRRT) with octreotide. The AML diagnosis was complicated by pancytopenia that initially masked the leukemia.

She was treated with a non-chemotherapy regimen of azacitidine and venetoclax, which achieved remission of both the AML and the neuroendocrine tumor simultaneously. This dual response demonstrates the potential for non-intensive therapeutic approaches in managing therapy-related AML in complex oncology patients.

Why it matters

PRRT is an increasingly used treatment for neuroendocrine tumors, and as more patients survive longer on this therapy, the long-term risks — including secondary blood cancers — become more relevant. This case demonstrates that therapy-related AML can develop during PRRT and may be initially obscured by the blood count changes caused by the radiation. Importantly, it shows a successful non-chemotherapy management strategy, which is valuable for patients who may not tolerate aggressive treatment.

How the study worked

This is a clinical case report documenting the sequential management of a metastatic neuroendocrine tumor with PRRT (using radiolabeled octreotide) and the subsequent development and treatment of secondary AML. The case was managed through a multidisciplinary tumor board approach.

What this study cannot tell us

This is a single case report and cannot establish the frequency of secondary AML following PRRT or the generalizability of the non-chemotherapy treatment approach. The causal relationship between PRRT and AML development cannot be definitively established from one case — the leukemia could have developed independently. The long-term durability of the dual remission is not reported.

How to read the evidence

This is a single case report — the lowest tier of clinical evidence. While the successful outcome is noteworthy, it cannot establish treatment recommendations or risk estimates. The value lies in alerting clinicians to a potential PRRT complication and demonstrating a management approach.

When this study was published

Published in 2025, this case report is timely as PRRT usage is expanding globally following positive results from trials like NETTER-1, making awareness of long-term complications increasingly important.

The bigger picture

Peptide receptor radionuclide therapy represents one of the most successful applications of peptide-based targeted therapy in oncology. By attaching radioactive isotopes to peptides that bind somatostatin receptors on tumor cells, PRRT delivers radiation precisely where it's needed. However, the radiation can also affect bone marrow, potentially causing secondary cancers. As PRRT use expands and patients live longer, understanding and managing these long-term complications becomes essential for the field's continued success.

Questions still open

What is the actual incidence of secondary hematological malignancies in patients receiving PRRT for neuroendocrine tumors?
Should routine bone marrow monitoring be implemented for all patients on long-term PRRT?
Is the dual remission (AML + NET) with azacitidine and venetoclax sustained long-term, and could this approach become standard for therapy-related AML in PRRT patients?

Common questions

What is peptide receptor radionuclide therapy (PRRT)?

PRRT is a targeted cancer treatment that attaches radioactive molecules to peptides (small proteins) that specifically bind to receptors on neuroendocrine tumor cells. When injected, these radiolabeled peptides seek out tumor cells throughout the body and deliver radiation directly to them, minimizing damage to healthy tissue. It's most commonly used with somatostatin analogue peptides like octreotide.

How common is leukemia as a side effect of PRRT?

Secondary blood cancers following PRRT are rare, and the exact risk is still being studied as more patients receive this treatment and are followed for longer periods. The radiation from PRRT can affect bone marrow, which is where blood cells are made. This case report highlights the importance of monitoring blood counts in PRRT patients so that any blood-related complications can be caught early.

Read the original research

Metachronous Occurrence of Acute Myeloid Leukaemia in a Case of Neuroendocrine Tumour.

European journal of case reports in internal medicine, 12(4), 005233

View on PubMed ↗Publisher’s paper ↗

Citation

Menon, Abhinav; Harindran Vallathol, Dilip; Charles, Deepak; Nair, Shagos; Kundil Veetil, Karthika; Komaranchath, Ashok S; Warrier, Arun R. (2025). Metachronous Occurrence of Acute Myeloid Leukaemia in a Case of Neuroendocrine Tumour.. European journal of case reports in internal medicine, 12(4), 005233. https://doi.org/10.12890/2025_005233