Outcomes of repeat conventional transarterial chemoembolization in patients with liver metastases.

INTRODUCTION AND OBJECTIVES: Although unlimited sessions of conventional transarterial chemoembolization (cTACE) may be performed for liver metastases, there is no data indicating when treatment becomes ineffective. This study aimed to determine the optimal number of repeat cTACE sessions for nonresponding patients before abandoning cTACE in patients with liver metastases. MATERIALS AND METHODS: In this retrospective, single-institutional analysis, patients with liver metastases from neuroendocrine tumors (NET), colorectal carcinoma (CRC), and lung cancer who underwent consecutive cTACE sessions from 2001 to 2015 were studied. Quantitative European Association for Study of the Liver (qEASL) criteria were utilized for response assessment. The association between the number of cTACE and 2-year, 5-year, and overall survival was evaluated to estimate the optimal number of cTACE for each survival outcome. RESULTS: Eighty-five patients underwent a total of 186 cTACE sessions for 117 liver metastases, of which 30.7 % responded to the first cTACE. For the target lesions that did not respond to the first, second, and third cTACE sessions, response rates after the second, third, and fourth cTACE sessions were 33.3 %, 23 %, and 25 %, respectively. The fourth cTACE session was the optimal number for 2-year survival (HR 0.40; 95 %CI: 0.16-0.97; p = 0.04), 5-year survival (HR 0.31; 95 %CI: 0.11-0.87; p = 0.02), and overall survival (HR 0.35; 95 %CI: 0.13-0.89; p = 0.02). CONCLUSIONS: Repeat cTACE in the management of liver metastases from NET, CRC, and lung cancer was associated with improved patient survival. We recommend at least four cTACE sessions before switching to another treatment for nonresponding metastatic liver lesions.

Síndrome de feocromocitoma-paraganglioma de tipo 5 como causa de hipertensión arterial en una paciente colombiana: reporte de caso / Pheochromocytoma/paraganglioma type 5 syndrome as a cause of secondary hypertension in a Colombian patient: case report

El feocromocitoma es un tumor derivado de las células de la cresta neural con la capacidad de producir sustancias simpaticomiméticas y, por ende, un cuadro clínico particular. Causa menos del 1 % de los casos de hipertensión arterial sistémica y su incidencia se estima entre 0,4 y 0,6 casos por 100.000 personas cada año, con una supervivencia media de siete años. De todos los tumores sólidos, el feocromocitoma tiene un mayor componente genético, que puede heredarse hasta en el 40 % de los casos. Una vez diagnosticada la enfermedad, se debe definir el tratamiento y el pronóstico, en parte condicionados por las variantes genéticas asociadas, en especial RET, SDHx, VHL y NF1.Se presenta el caso de una mujer joven con dolor abdominal e hipertensión arterial sistémica, a quien se le diagnosticó feocromocitoma. Al secuenciar el exoma, se identificó una variante patogénica extremadamente rara y de reciente descubrimiento: SDHA: c.1A>C (p.Met1Leu). La paciente respondió adecuadamente al tratamiento quirúrgico y continuó en seguimiento sin recurrencias.El abordaje diagnóstico de los pacientes con feocromocitoma comienza con la sospecha clínica, seguida de la medición de determinados metabolitos en sangre y orina, y, finalmente, los estudios de imagenología. Los desarrollos tecnológicos actuales permiten la aplicación de la medicina de precisión en este campo. En este caso de feocromocitoma, se identificó un componente genético importante que no solo afecta al paciente, sino también, a sus familiares. La tamización adecuada del caso índice permite identificar mutaciones y caracterizar mejor la enfermedad.

Pheochromocytoma is a tumor derived from neural crest cells able to produce sympathomimetic substances and, hence, a particular clinical picture. It is responsible for less than 1% of high blood pressure cases, with an estimated incidence between 0.4 and 0.6 cases per 100,000 people each year, and an average survival of seven years. Pheochromocytoma is a solid tumor with a high genetic component, as heritability can reach 40%. Once diagnosed, its treatment and prognosis are partly conditioned by the associated pathogenic variants that can be documented, especially those related to RET, SDHx, VHL, and NF1 genes.We present the case of a young woman with abdominal pain and high blood pressure, who was found to have a pheochromocytoma. Genetic testing detected a rare and recently discovered pathogenic variant: the SDHA:c.1A>C (p.Met1Leu). The patient responded adequately to the surgical treatment and continued the follow-up without documented recurrences.The diagnostic approach for pheochromocytoma patients must start with a clinical suspicion, followed by metabolite measurement in blood and urine, and finally, imaging. Currently, technology development allows precision medicine applicability. In this case of pheochromocytoma, recent developments in precision medicine resulted in the detection of associated genetic components involving the patient and her family. Adequate screening of the index patient is required for documenting pathogenic variants and better characterizing the disease.

Nodular image in the appendix observed on ultrasound: endometriosis or neuroendocrine neoplasia?

Objective: To evaluate the association between clinical and imaging with surgical and pathological findings in patients with suspected neuroendocrine tumor of appendix and/or appendix endometriosis. Methods: Retrospective descriptive study conducted at the Teaching and Research Institute of Hospital Israelita Albert Einstein, in which medical records and databases of patients with suspected neuroendocrine tumor of appendix and/or endometriosis of appendix were analyzed by imaging. Results: Twenty-eight patients were included, all of which had some type of appendix alteration on the ultrasound examination. The pathological outcome of the appendix found 25 (89.3%) lesions compatible with endometriosis and three (10.7%) neuroendocrine tumors. The clinical findings of imaging and surgery were compared with the result of pathological anatomy by means of relative frequency. Conclusion: It was possible to observe a higher prevalence of appendix endometriosis when the patient presented more intense pain symptoms. The image observed on ultrasound obtained a high positive predictive value for appendicular endometriosis.

The Immune Microenvironment Landscape of Pituitary NeuroEndocrine Tumors, a Transcriptomic Approach.

Pituitary neuroendocrine tumors (PitNET) are known to be variably infiltrated by different immune cells. Nonetheless, their role in pituitary oncogenesis has only begun to be unveiled. The immune microenvironment could determine the biological and clinical behavior of a neoplasm and may have prognostic implications. To evaluate the expression of immune-related genes and to correlate such expression with the presence of infiltrating immune cells in forty-two PitNETs of different lineages, we performed whole transcriptome analysis and RT-qPCR. Deconvolution analysis was carried out to infer the immune cell types present in each tumor and the presence of immune cells was confirmed by immunofluorescence. We found characteristic expression profiles of immune-related genes including those encoding interleukins and chemokines for each tumor lineage. Genes such as IL4-I1, IL-36A, TIRAP, IL-17REL, and CCL5 were upregulated in all PitNETS, whereas IL34, IL20RA, and IL-2RB characterize the NR5A1-, TBX19-, and POU1F1-derived tumors, respectively. Transcriptome deconvolution analysis showed that M2 macrophages, CD4+ T cells, CD8+ T cells, NK cells, and neutrophils can potentially infiltrate PitNET. Furthermore, CD4+ and CD8+ T cells and NK cells infiltration was validated by immunofluorescence. Expression of CCL18, IL-5RA, and HLA-B as well as macrophage tumor infiltration could identify patients who can potentially benefit from treatment with immune checkpoint inhibitors.

Epidemiological profile of neuroendocrine tumors in adults in Brazil.

Objective: Neuroendocrine tumors (NETs) are a set of diseases that originate from neuroendocrine cells, which comprises a diffuse endocrine system present in various organs of the body. These tumors are more frequent in the gastrointestinal tract (70%) and the bronchopulmonary system (20%-30%). A NET incidence rate of 1-5 per 100,000 inhabitants has been estimated for several European countries and the USA employing 20 years of data. However, no comprehensive studies on this rare neoplasm are available in Brazil. In this context, the aim of this study was to characterize the epidemiological NET profile in the country. Materials and methods: This is a retrospective descriptive observational study based on data from Hospital Cancer Records available at the Brazilian National Cancer Institute and the São Paulo Oncocentro Foundation. Demographic, clinical and treatmentrelated variables were analyzed from selected cases employing descriptive statistics. Results and Conclusion: A total of 15,859 cases were identified, most occurring in males (53.4%) and in individuals under 65 years old (63.3%). Small cell carcinoma was the most frequent histological type (46.7%). Bronchopulmonary tumors were the most frequent NETs, followed by pancreatic tumors, with cases mostly concentrated in high complexity centers in the Brazilian Southeast and treated mainly with surgery and chemotherapy, with over half of the patients diagnosed in advanced stages of the disease.

The molecular biology of sporadic acromegaly.

GH-secreting tumors represent 15 % to 20 % of all pituitary neuroendocrine tumors (pitNETs), of which 95 % occur in a sporadic context, without an identifiable inherited cause. Recent multi-omic approaches have characterized the epigenomic, genomic, transcriptomic, proteomic and kynomic landscape of pituitary tumors. Transcriptomic analysis has allowed us to discover specific transcription factors driving the differentiation of pituitary tumors and gene expression patterns. GH-secreting, along with PRL- and TSH-secreting pitNETs are driven by POU1F1; ACTH-secreting tumors are determined by TBX19; and non-functioning tumors, which are predominantly of gonadotrope differentiation are conditioned by NR5A1. Upregulation of certain miRNAs, such as miR-107, is associated with tumor progression, while downregulation of others, like miR-15a and miR-16-1, correlates with tumor size reduction. Additionally, miRNA expression profiles are linked to treatment resistance and clinical outcomes, providing insights into potential therapeutic targets. Specific somatic mutations in GNAS, PTTG1, GIPR, HGMA2, MAST and somatic variants associated with cAMP, calcium signaling, and ATP pathways have also been associated with the development of acromegaly. This review focuses on the oncogenic mechanisms by which sporadic acromegaly can develop, covering a complex series of molecular alterations that ultimately alter the balance between proliferation and apoptosis, and dysregulated hormonal secretion.

Clinical features and prognosis of malignant small bowel tumors: Experience from a university hospital in Chile.

BACKGROUND: Small bowel tumors (SBT) are infrequent and represent a small proportion of digestive neoplasms. There is scarce information about SBT in Latin America. AIM: To describe the epidemiology, clinical characteristics, diagnostic methods, and survival of malignant SBTs. METHODS: Retrospective observational study of adult patients with histopathological diagnosis of SBT between 2007 and 2021 in a university hospital in Chile. RESULTS: A total of 104 patients [51.9% men; mean age 57 years] with SBT. Histological type: neuroendocrine tumor (NET) (43.7%, n=38), gastrointestinal stromal tumors (GIST) (21.8%, n=19), lymphoma (17.2%, n=15) and adenocarcinoma (AC) (11.5%, n=10). GIST was more frequent in duodenum (50%; n=12) and NET in the ileum (65.8%; n=25). Metastasis was observed in 17 cases, most commonly from colon and melanoma. Nausea and vomiting were significantly more often observed in AC (p=0.035), as well as gastrointestinal bleeding in GIST (p=0.007). The most common diagnostic tools were CT and CT enteroclysis with an elevated diagnostic yield (86% and 94% respectively). The 5-year survival of GIST, NET, lymphoma and AC were 94.7% (95%CI: 68.1-99.2), 82.2% (95%CI: 57.6-93.3), 40.0% (95%CI: 16.5-82.8) and 25.9% (95%CI: 4.5-55.7%), respectively. NET (HR 6.1; 95%CI: 2.1-17.2) and GIST (HR 24.4; 95%CI: 3.0-19.8) were independently associated with higher survival compared to AC, adjusted for age and sex. CONCLUSIONS: Malignant SBT are rare conditions and NETs are the most common histological subtype. Clinical presentation at diagnosis, location or complications may suggest a more probable diagnosis. GIST and NET are associated with better survival compared to other malignant subtypes.

FLNA expression modulates pathological markers of pituitary neuroendocrine tumours.

Due to the current limited knowledge about the role of filamin A (FLNA) in pituitary tumour progression, we aimed to analyse FLNA expression levels and its impact on aggressive markers of pituitary neuroendocrine tumours (PitNETs), using an integrative approach of in vivo and in vitro models and human samples. An increase in the expression levels of FLNA was observed in the advanced tumoural stages of the hyperplastic adenomatous pituitary model, concomitant with a decrease in cell proliferation and with a modification in the subcellular localisation of this protein. Similarly, overexpression of FLNA in the somatolactotropic GH3 cell line induced a decrease in the cell proliferation, promoted a migratory phenotype, increased invasion activity, and decreased the prolactin secretion. Cyclin D1 (CCND1) and cyclin-dependent kinase 4 (CDK4) expression increased in both models in correlation with the increase observed in FLNA levels. When human tissues were analysed a significant increase of FLNA was observed in PitNETs compared to normal pituitary gland, with heterogeneous intracellular localisation. Higher levels of FLNA expression were observed in tumours with invasive characteristics. These results underline the crucial roles of FLNA as a modulator of pathological markers and as a potential prognostic marker in pituitary tumours.

GASTRIC NEUROENDOCRINE TUMOR: WHEN SURGICAL TREATMENT IS INDICATED?

BACKGROUND: Gastric neuroendocrine tumors are a heterogeneous group of neoplasms that produce bioactive substances. Their treatment varies according to staging and classification, using endoscopic techniques, open surgery, chemotherapy, radiotherapy, and drugs analogous to somatostatin. AIMS: To identify and review cases of gastric neuroendocrine neoplasia submitted to surgical treatment. METHODS: Review of surgically treated patients from 1983 to 2018. RESULTS: Fifteen patients were included, predominantly female (73.33%), with a mean age of 55.93 years. The most common symptom was epigastric pain (93.3%), and the mean time of symptom onset was 10.07 months. The preoperative upper digestive endoscopy (UDE) indicated a predominance of cases with 0 to 1 lesion (60%), sizing ≥1.5 cm (40%), located in the gastric antrum (53.33%), with ulceration (60%), and Borrmann III (33.33%) classification. The assessment of the surgical specimen indicated a predominance of invasive neuroendocrine tumors (60%), with angiolymphatic invasion in most cases (80%). Immunohistochemistry for chromogranin A was positive in 60% of cases and for synaptophysin in 66.7%, with a predominant Ki-67 index between 0 and 2%. Metastasis was observed in 20% of patients. The surgical procedure most performed was subtotal gastrectomy with Roux-en-Y reconstruction (53.3%). Tumor recurrence occurred in 20% of cases and a new treatment was required in 26.67%. CONCLUSIONS: Gastric neuroendocrine tumors have a low incidence in the general population, and surgical treatment is indicated for advanced lesions. The study of its management gains importance in view of the specificities of each case and the need for adequate conduct to prevent recurrences and complications.

Divertículo de Meckel con tumor neuroendocrino: presentación inusual / Meckel's diverticulum with neuroendocrine tumor: unusual presentation

El divertículo de Meckel es una malformación congénita que suele presentarse como un hallazgo incidental asintomático. Puede complicarse por procesos inflamatorios o tumores, cursando con sintomatología abdominal sumamente inespecífica, lo que complica su diagnóstico oportuno. Aunque la incidencia de neoplasias malignas en estos divertículos es baja, los tumores neuroendocrinos son los más representativos. Presentamos el caso de una paciente de 72 años que consultó por dolor abdominal y deposiciones melénicas, con múltiples nódulos intrahepáticos sugestivos de tumores neuroendocrinos y hallazgo intraoperatorio incidental de diverticulitis aguda de Meckel con metástasis peridiverticular de un tumor neuroendocrino. (AU)

Meckel's diverticulum is a congenital malformation that usually presents as an incidental finding. It can be complicated by inflammatory processes or tumors, with non-specific abdominal symptoms which delay its timely diagnosis. Although the incidence of malignant neoplasms in these diver-ticula is low, neuroendocrine tumors are the most representative. We present the case of a 72-year-old female patient who consulted for abdominal pain and melenic bowel movements, with multiple intrahepatic nodules suggestive of neuroendocrine tumors and an incidental intraoperative finding of acute Meckel's diverticulitis with peridiverticular metastasis of a neuroendocrine tumor. (AU)

Belzutifan (WeliregTM) en tumores neuroendocrinos asociados a la enfermedad de von Hippel-Lindau / Belzutifan (WeliregTM) in neuroendocrine tumors associated with von Hippel-Lindau disease

INTRODUCCIÓN: Los tumores neuroendocrinos (TNE) constituyen un grupo heterogéneo de neoplasias que se originan de las células de la cresta por lo que pueden presentarse en múltiples localizaciones del organismo, aunque la mayoría aparecen en el eje gastroenteropancreático (TNE-GEP). Su comportamiento clínico es muy variable: pueden ser hormonalmente activos o no funcionantes y pueden comportarse con un crecimiento muy lento o altamente agresivo. La resección quirúrgica es el único tratamiento curativo. Debido a que la mayoría de los TNE (aproximadamente el 80%) expresan una alta densidad de receptores de somatostatina en aquellos pacientes con tumores inoperables o enfermedad metastásica, los análogos de somatostatina (como el octreotide o el lanreotide) se consideran la primera línea de tratamiento, al igual que los análogos radiomarcados. TECNOLOGÍA: Belzutifan es un inhibidor del HIF-2α, donde al bloquear la posibilidad de interacción con otros factores provoca una reducción de la transcripción y expresión de los genes que estarían implicados en la proliferación tumoral. OBJETIVO: El objetivo del presente informe es evaluar rápidamente los parámetros de eficacia, seguridad, costos y recomendaciones disponibles acerca del empleo del uso de belzutifan en tumores en tumores neuroendocrinos asociados con la enfermedad de Von Hippel-Lindau. MÉTODOS: Se realizó una búsqueda bibliográfica en las principales bases de datos tales como PUBMED, LILACS, BRISA, COCHRANE, SCIELO, EMBASE, TRIPDATABASE como así también en sociedades científicas, agencias reguladoras, financiadores de salud y agencias de evaluación de tecnologías sanitarias. Se priorizó la inclusión de revisiones sistemáticas, ensayos clínicos controlados aleatorizados, evaluación de tecnología sanitaria y guías de práctica clínica de alta calidad metodológica. EVIDENCIA CLÍNICA: Jonasch y col. publicaron en el año 2021 los resultados de un ensayo clínico abierto y no aleatorizado con el objetivo de evaluar la eficacia y la seguridad belzutifan en pacientes con diagnóstico de carcinoma renal asociados a la enfermedad de VHL (estudio de fase 2 denominado 004; NCT03401788). Para su enrolamiento los pacientes requerían poseer la confirmación molecular en línea germinal del VHL. Los pacientes enrolados podían tener otros tumores asociados a la enfermedad de VHL, incluidos hemangioblastomas del sistema nervioso central y tumores neuroendocrinos gastroenteropancreaticos. Fueron excluidos aquellos pacientes con enfermedad metastásica o con requerimiento de cirugía inmediata. RECOMENDACIONES: Las guías de la Red Nacional de Centros para el Tratamiento Integral del cáncer de los Estados Unidos (NCCN, su sigla del inglés National Comprehensive Cáncer Network) recomienda considerar el uso de belzutifan para tumores neuroendocrinos resecables solo en el contexto de pacientes con mutaciones en la línea germinal del gen VHL demostrada. Los datos sobre su uso para tumores grandes, localmente avanzados considerados irresecables o con enfermedad a distancia son extremadamente limitados. La valoración del beneficio clínico según la escala de la Sociedad Europea de Oncología Médica (ESMO, su sigla del inglés European Society for Medical Oncology) fue de 3 puntos sobre 5. No clasificando como una magnitud sustancial del beneficio clínico en el ámbito no curativo. En Argentina, la Asociación Argentina de Oncología Clínica no menciona su utilización dentro de sus recomendaciones en oncología. La guía de recomendaciones elaborada por la Sociedad Española de Oncología Médica (SEOM) y Grupo Español de Tumores Neuroendocrinos y Endocrinos (GETNE) no menciona la utilización el uso de belzutifan para tumores neuroendocrinos. Actualmente el Instituto Nacional de Salud y Cuidados de Excelencia del Reino Unido (NICE, su sigla del inglés National Institute for Health and Care Excellence) se encuentra evaluándo su utilización para el tratamiento de tumores asociados con la enfermedad de VHL. CONCLUSIONES: La evidencia que sustenta la aprobación de comercialización de belzutifan en pacientes con enfermedad de von Hippel-Lindau y diagnóstico de tumores neuroendocrinos se basa en un análisis de subgrupo reportado en un único ensayo clínico no aleatorizado con escaso número de pacientes. En este estudio pacientes con tumores neuroendocrinos de páncreas localizados y que no requieren a tratamiento quirúrgico inmediato recibieron belzutifan. El impacto en la sobrevida global de los pacientes y el periodo libre de progresión, como también el potencial beneficio en la calidad de vida de los pacientes no fueron reportados. El estudio reportó que aquellos pacientes que lo utilizaron reportaron tasas de respuesta al 90%. La seguridad y eficacia frente a otras opciones terapéuticas actualmente disponibles y recomendadas para el tratamiento no puede ser establecida debido a que no se encontraron estudios comparativos. En Argentina, la Asociación Argentina de Oncología Clínica no lo menciona dentro de las opciones actuales de tratamiento. Una guía de recomendaciones de los Estados Unidos lo mencionan como una opción terapéutica a considerar, solo en estadios localizados. Utilizando precios de referencia internacionales el costo de adquisición para un mes de tratamiento fue estimado en aproximadamente 10,4 millones de ARS.

LIVER TRANSPLANT FOR METASTATIC NEUROENDOCRINE TUMORS: A SINGLE-CENTER REPORT.

BACKGROUND: Neuroendocrine tumors are rare neoplasms of uncertain biological behavior. The liver is one of the most common sites of metastases, occurring in 50% of patients with metastatic disease. AIMS: To analyze a clinical series in liver transplant of patients with neuroendocrine tumors metastases. METHODS: A retrospective descriptive study, based on the review of medical records of patients undergoing liver transplants due to neuroendocrine tumor metastases in a single center in northeast Brazil, over a period of 20 years (January 2001 to December 2021). RESULTS: During the analyzed period, 2,000 liver transplants were performed, of which 11 were indicated for liver metastases caused by neuroendocrine tumors. The mean age at diagnosis was 45.09±14.36 years (26-66 years) and 72.7% of cases were females. The most common primary tumor site was in the gastrointestinal tract in 64% of cases. Even after detailed investigation, three patients had no primary tumor site identified (27%). Overall survival after transplantation at one month was 90%, at one year was 70%, and five year, 45.4%. Disease-free survival rate was 72.7% at one year and 36.3% at five years. CONCLUSIONS: Liver transplantation is a treatment modality with good overall survival and disease-free survival results in selected patients with unresectable liver metastases from neuroendocrine tumors. However, a rigorous selection of patients is necessary to obtain better results and the ideal time for transplant indication is still a controversial topic in the literature.

[Primary neuroendocrine tumor of the common bile duct] / Tumor neuroendocrino primario del conducto hepático común.

Primary neuroendocrine tumors (NETs) of the bile duct are extremely rare and represent only 0.2-2% of all gastrointestinal NETs. Within the biliary system, the main bile duct is the most affected site. A 28-year-old man with a 6-month history of intermittent jaundice, pruritus, and choluria. MRCP, PET-CT and endoscopic ultrasound were performed. A well-differentiated neuroendocrine neoplasia was diagnosed. Complete resection of the main bile duct was performed with lymphadenectomy of the hepatic pedicle with Roux-en-Y hepaticojejunostomy, without complications. The patient had an adequate evolution and nowadays he's disease-free. Primary neuroendocrine tumors of the bile duct are extremely rare. They may present clinically and radiologically similar to perihilar cholangiocarcinoma, which makes preoperative diagnosis difficult. Radical resection is indicated. Usually, they are well differentiated tumors, being the Ki-67 labeling index a reliable prognostic marker.

Los tumores neuroendocrinos (TNE) primarios de la vía biliar son extremadamente raros y representan sólo el 0.2-2% de todos los TNE gastrointestinales. Dentro del sistema biliar, la vía biliar principal es el sitio más afectado. Hombre de 28 años con cuadro de 6 meses de evolución caracterizado por ictericia intermitente, prurito y coluria. Se realizó colangiopancreatoresonancia magnética nuclear, PET-TC y ultrasonido endoscópico que concluyeron neoplasia neuroendocrina bien diferenciada. Se realizó resección completa de la vía biliar principal con linfadenectomía del pedículo hepático con hepaticoyeyunoanastomosis en Y de Roux, sin complicaciones. El paciente cursó adecuada evolución y se encuentra libre de enfermedad. Los tumores neuroendocrinos primarios de la vía biliar son extremadamente raros, presentándose clínica y radiológicamente como lesiones similares al colangiocarcinoma perihiliar lo que dificulta el diagnóstico preoperatorio. Está indicado su tratamiento quirúrgico radical. Suelen ser bien diferenciados, siendo el antígeno Ki-67 un marcador pronóstico confiable.

Breakthrough Symptoms Remain an Unmet Need in Symptomatic Patients With Neuroendocrine Tumors and Carcinoid Syndrome.

OBJECTIVES: The aims of the study were to assess the effects of breakthrough carcinoid syndrome symptoms on well-being in neuroendocrine tumor (NET) patients insufficiently controlled on long-acting somatostatin analog (SSA) and to assess patient experience with treatment options, physician communication, and disease information sources. METHODS: This study surveyed US NET patients from 2 online communities, experiencing at least one symptom, by utilizing a 64-item questionnaire. RESULTS: One hundred patients participated: 73% female, 75% age 56 to 75 years, and 93% White. Primary tumor distribution was as follows: gastrointestinal NET (n = 55), pancreatic NET (n = 33), lung NET (n = 11), and other NET (n = 13). All patients were actively treated with one long-acting SSA and experiencing breakthrough symptoms: diarrhea, flushing, or other (13% experienced one, 30% two, 57% greater than two). More than one third of treated patients experienced carcinoid-related symptoms daily. Sixty percent of respondents reported not having short-acting "rescue" treatment available, impacting well-being though anxiety or depression (45%), trouble exercising (65%), sleeping (57%), employment (54%), and maintaining friendships (43%). CONCLUSIONS: Breakthrough symptoms remain an unmet need, even in treated patients with NETs. Though still relying on physicians, NET patients are now also using the Internet. Improved awareness of optimal SSA use may improve syndrome control.

SEOM-GETNE clinical guidelines for the diagnosis and treatment of gastroenteropancreatic and bronchial neuroendocrine neoplasms (NENs) (2022).

Neuroendocrine neoplasms (NENs) are a heterogeneous family of tumors of challenging diagnosis and clinical management. Their incidence and prevalence continue to rise mainly due to an improvement on diagnostic techniques and awareness. Earlier detection, along with steadfast improvements in therapy, has led to better prognosis over time for advanced gastrointestinal and pancreatic neuroendocrine tumors. The aim of this guideline is to update evidence-based recommendations for the diagnosis and treatment of gastroenteropancreatic and lung NENs. Diagnostic procedures, histological classification, and therapeutic options, including surgery, liver-directed therapy, peptide receptor radionuclide therapy, and systemic hormonal, cytotoxic or targeted therapy, are reviewed and discussed, and treatment algorithms to guide therapeutic decisions are provided.