Multicenter Analysis of Presacral Neuroendocrine Neoplasms—Clinicopathological Characterization and Treatment Outcomes of a Rare Disease

Frontiers in Endocrinology

卷:12

Simon Kreutzfeldt¹  Sebastian Krug²  Anke Kröcher³  Harald Lahner⁴  Sami Matrood⁵  Anja Rinke⁵  Thomas Matthias Gress⁵  Zoltan Kender⁶  Leonidas Apostolidis⁷  Damiano Librizzi⁸  Jörg Schrader⁹  Annette Ramaswamy¹⁰ 

[1] 0Department of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany;

[2] Clinic for Internal Medicine I, Martin-Luther University Halle/Wittenberg, Halle, Germany;

[3] Clinic for Internal Medicine I, University Hospital Carl Gustav Carus Dresden, Dresden, Germany;

[4] Department of Endocrinology and Metabolism, University Hospital Essen, Essen, Germany;

[5] Department of Gastroenterology and Endocrinology, UKGM Marburg and Philipps University, Marburg, Germany;

[6] Department of Internal Medicine I and Clinical Chemistry, Heidelberg University Hospital, Heidelberg, Germany;

[7] Department of Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg, Heidelberg University Hospital, Heidelberg, Germany;

[8] Department of Nuclear Medicine, UKGM Marburg and Philipps University, Marburg, Germany;

[9] I. Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, Germany;

[10] Institute of Pathology, UKGM Marburg and Philipps University, Marburg, Germany;

关键词: presacral;    retrorectal;    CUP-NET;    neuroendocrine tumor;    neuroendocrine carcinoma;    carcinoid;   

DOI  :  10.3389/fendo.2021.709256

来源: DOAJ

【 摘 要 】

Background and AimsNeuroendocrine neoplasms (NENs) of the presacral space are an extremely rare disease entity with largely unknown outcome and no established standard of care treatment. Therefore, we wanted to analyze clinical presentation, histopathological findings, treatment outcomes, and prognosis in a multicentric patient cohort.MethodsWe searched local databases of six German NEN centers for patients with presacral NEN. Retrospective descriptive analyses of age, sex, stage at diagnosis, symptoms, grade, immunohistochemical investigations, biomarkers, treatment, and treatment outcome were performed. Kaplan–Meier analysis was used to determine median overall survival.ResultsWe identified 17 patients (11 female, 6 male) with a median age of 50 years (range, 35–66) at diagnosis. Twelve cases presented initially with distant metastases including bone metastases in nine cases. On pathological review the majority of patients had well-differentiated G2 tumors. Immunohistochemical profile resembled rectal NENs. All but one patient had non-functioning tumors. Somatostatin receptor imaging was positive in 14 of 15 investigated cases. Eight patients were treated surgically including palliative resections; 14 patients received somatostatin analogs with limited efficacy. With 14 PRRTs completed, 79% showed clinical benefit, whereas only one patient with neuroendocrine carcinoma (NEC) responded to chemotherapy. Treatment with everolimus in three patients was not successful, whereas cabozantinib resulted in a disease stabilization in a heavily pretreated patient. During a median observation period of 44.5 months, 6 patients died. Median overall survival was not reached.ConclusionPresacral NEN are histopathologically similar to rectal NENs. Presacral NEN should be considered as possible primary in NEN of unknown primary. The majority of tumors is non-functioning and somatostatin receptor positive. PRRT demonstrated promising activity; tyrosine kinase inhibitors warrant further investigations. Further molecular characterization and prospective evaluation of this rare tumor entity are needed.

【 授权许可】

Unknown