Remission of longstanding metastatic paraganglioma in a patient after use of zoledronic acid.

A patient with a long history of bone predominant, metastatic paraganglioma who had multiple episodes of progressive disease despite prior treatments demonstrated a remarkable disease response to zoledronic acid. After 1 year of treatment, there was a complete resolution of lymphadenopathy and disappearance of all somatostatin receptor avid lesions by positron emission tomography-CT and radiopharmaceutical Gallium Ga 68 Dotatate. Stability of disease was further demonstrated by CT over several years.

N-Linked Fucosylated Glycans Are Biomarkers for Prostate Cancer with a Neuroendocrine and Metastatic Phenotype.

Prostate cancer is a heterogeneous disease with a spectrum of pathology and outcomes ranging from indolent to lethal. Although there have been recent advancements in prognostic tissue biomarkers, limitations still exist. We leveraged matrix-assisted laser desorption/ionization imaging of formalin-fixed, paraffin embedded prostate cancer specimens to determine if N-linked glycans expressed in the extracellular matrix of lethal neuroendocrine prostate cancer were also expressed in conventional prostate adenocarcinomas that were associated with poor outcomes.

Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors.

Background: Treatment options for patients with advanced neuroendocrine tumors are limited. The efficacy of cabozantinib in the treatment of previously treated, progressive extrapancreatic or pancreatic neuroendocrine tumors is unclear.

Methods: We enrolled two independent cohorts of patients - those with extrapancreatic neuroendocrine tumors and those with pancreatic neuroendocrine tumors - who had received peptide receptor radionuclide therapy or targeted therapy or both.

A Phase II Trial of the WEE1 Inhibitor Adavosertib in SETD2-Altered Advanced Solid Tumor Malignancies (NCI 10170).

Unlabelled: We sought to evaluate the efficacy of WEE1 inhibitor adavosertib in patients with solid tumor malignancies (cohort A) and clear cell renal cell carcinoma (ccRCC; cohort B). NCT03284385 was a parallel cohort, Simon two-stage, phase II study of adavosertib (300 mg QDAY by mouth on days 1-5 and 8-12 of each 21-day cycle) in patients with solid tumor malignancies harboring a pathogenic SETD2 mutation. The primary endpoint was the objective response rate.

Treatment of metastatic rectal squamous cell carcinoma in a pregnant patient.

Rectal squamous cell carcinoma is an exceedingly rare form of rectal cancer, with limited data available regarding its presentation and effective treatment. Rectal cancer occurring during pregnancy is uncommon as well. This is a case of metastatic rectal squamous cell carcinoma presenting in a 22-week pregnant, female patient in her early 30s.

Race and Odds of Surgery Offer in Small Bowel and Pancreas Neuroendocrine Neoplasms.

Background: Despite existing society guidelines, management of pancreatic (PanNEN) and small bowel (SBNEN) neuroendocrine neoplasms remains inconsistent. The purpose of this study was to identify patient- and/or disease-specific characteristics associated with increased odds of being offered surgery for PanNEN and SBNEN.

Patients And Methods: The Surveillance, Epidemiology, and End Results (SEER) Program database and the National Cancer Database (NCDB) were queried for patients with PanNEN/SBNEN.

A Pilot Study of Paricalcitol plus Nanoliposomal Irinotecan and 5-FU/LV in Advanced Pancreatic Cancer Patients after Progression on Gemcitabine-Based Therapy.

Purpose: Vitamin D analogues remodel the desmoplastic stroma, and improve vascularity and efficacy of chemotherapy in preclinical pancreas cancer models.

Patients And Methods: We conducted a pilot study to evaluate the safety and preliminary efficacy of the vitamin D analogue paricalcitol in combination with nanoliposomal irinotecan (Nal-iri) plus 5-fluorouracil/leucovorin (5-FU/LV) in patients with advanced pancreatic cancer who had progressed on gemcitabine-based therapy. Two dose levels (DL) of paricalcitol were tested: fixed dose weekly (75 mcg, DL1) and weight-based weekly (7 mcg/kg, /DL2).

Tuberous sclerosis complex mutations in patients with pancreatic neuroendocrine tumors. Observations on phenotypic and treatment-related associations.

Pancreatic neuroendocrine tumors (PanNETs) in familial tuberous sclerosis (TSC1 and TSC2 mutations) have been known and studied. However, little is known about PanNET patients harboring the very rare (less than 2%) sporadic TSC mutations. Some renal tumors have been shown to harbor sporadic TSC mutations, with a distinctive morphological correlate.

Therapy Sequencing in Patients With Advanced Neuroendocrine Neoplasms.

Neuroendocrine neoplasms (NENs) comprise a beautifully complicated, exciting landscape of histologies and clinical behaviors. However, the nuanced complexity of low- and high-grade variants can easily overwhelm both patients and providers. In this chapter, we review the ever-expanding literature on both functioning and nonfunctioning small bowel and pancreatic NENs, touching on somatostatin analogs, hepatic-directed therapies, small molecules, radiopharmaceuticals, immunotherapy, cytotoxic chemotherapy, and new promising agents.

Pancreatic neuroendocrine tumors: tailoring imaging to specific clinical scenarios.

The clinical and imaging presentation of pancreatic neuroendocrine tumors (PanNETs) is variable and depends on tumor grade, stage, and functional status. This degree of variability combined with a multitude of treatment options and imaging modalities results in complexity when choosing the most appropriate imaging studies across various clinical scenarios. While various guidelines exist in the management and evaluation of PanNETs, there is an overall lack of consensus and detail regarding optimal imaging guidelines and protocols.

Acinar cell carcinoma with PRKAR1A and PTEN alterations and paraneoplastic panniculitis.

Pancreatic acinar cell carcinoma is a rare type of pancreatic malignancy, which can be confused with pancreatic neuroendocrine neoplasm. Here, we describe a woman in her 80s who presented with abdominal pain and bilateral lower extremity panniculitis. She underwent surgery for a presumed diagnosis of neuroendocrine tumour with PTEN and PRKAR1A alterations; 19 months, later, a recurrence of her pancreatic malignancy was discovered.

Preliminary experience with a new institutional tumor board dedicated to patients with neuroendocrine neoplasms.

Purpose: To determine the decision patterns of a neuroendocrine neoplasm (NEN) tumor board (TB) and the factors behind those.

Methods: We retrospectively reviewed all NEN-TB recommendations from 07/2018 to 12/2021 and recorded patient characteristics, TB outcomes and associations between them.

Results: A total of 652 patient entries were identified.

Synchronous AML and pancreatic neuroendocrine neoplasm, both successfully treated with somatostatin analogs and decitabine.

Summary: Downregulation of tumor suppression genes by DNA hypermethylation has been proposed as a potential cause of neuroendocrine neoplasm (NEN) formation. In this report, we present a patient simultaneously diagnosed with acute myeloid leukemia (AML) and a metastatic nonfunctioning pancreatic NEN. Because of the two competing diagnoses, he was treated with lanreotide, venetoclax and a long course of the hypomethylating agent decitabine.

Neuroendocrine and Rare Tumor Advances: A New and Promising TRAIL Emerges.

ONC201 is an oral selective antagonist of the dopamine D2 receptor and direct activator of caseinolytic protease P. In a phase II study, ONC021 was shown to be well tolerated with notable efficacy in patients with the rare neuroendocrine neoplasms pheochromocytomas-paragangliomas, although biomarkers for activity remain to be elucidated. See related article by Anderson et al.

Qualitative imaging features of pancreatic neuroendocrine neoplasms predict histopathologic characteristics including tumor grade and patient outcome.

Objectives: To identify PanNEN imaging features associated with tumor grade and aggressive histopathological features.

Methods: Associations between histopathological and imaging features of resected PanNEN were retrospectively tested. Histopathologic features included WHO grade, lymphovascular invasion (LVI), growth pattern (infiltrative, circumscribed), and intratumoral fibrosis (mature, immature).

Total Neoadjuvant Therapy With Short-Course Radiation: US Experience of a Neoadjuvant Rectal Cancer Therapy.

Background: Short-course radiation followed by chemotherapy as total neoadjuvant therapy has been investigated primarily in Europe and Australia with increasing global acceptance. There are limited data on this regimen's use in the United States, however, potentially delaying implementation.

Objective: This study aimed to compare clinical performance and oncologic outcomes of 2 rectal cancer neoadjuvant treatment modalities: short-course total neoadjuvant therapy versus standard chemoradiation.

Common Pitfalls in Ewing Sarcoma and Desmoplastic Small Round Cell Tumor Diagnosis Seen in a Study of 115 Cases.

Ewing sarcoma (ES), "Ewing-like sarcoma" (ELS) and desmoplastic small round cell tumors (DSRCT) can masquerade as other tumor types, particularly neuroendocrine neoplasms and receive inappropriate treatment. We retrieved 115 cases of ES, ELS and DSRCT seen over 17 years in a tertiary center. An initial misdiagnosis or incomplete diagnosis occurred in 6/93 (6.