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Multiple Endocrine Neoplasia Type 1

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201. Regorafenib (Stivarga) indicated as monotherapy for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib treatment

as monotherapy for the treatment of adult patients with hepatocellular carcinoma who have been previously treated with Sorafenib Version 1.2, October 2017 EUnetHTA Joint Action 3 WP4 7 LIST OF ABBREVIATIONS AASLD American Association for the Study of Liver Diseases AE Adverse event AFP alpha-fetoprotein ALT Alanine transaminase AST Aspartate transaminase ATC Anatomical therapeutic chemical AUC Area under the curve BCLC Barcelona Clinic Liver Cancer BSC Best supportive care CDSR Cochrane Database (...) antigen HBV Hepatitis B virus HCC Hepatocellular carcinoma PTJA02 - Regorafenib indicated as monotherapy for the treatment of adult patients with hepatocellular carcinoma who have been previously treated with Sorafenib Version 1.2, October 2017 EUnetHTA Joint Action 3 WP4 8 HCS Hepatobiliary cancer subscale HCV Hepatitis C virus HFSR Hand–foot skin reaction HR Hazard ratio HRQoL Health-related quality of life ICD International classification of diseases ILD Interstitial lung disease IVRS Interactive

2018 EUnetHTA

202. Management of adrenal incidentalomas: European Society of Endocrinology Clinical Practice Guideline in collaboration with the European Network for the Study of Adrenal Tumours Full Text available with Trip Pro

is usually higher than in series including all patients presenting with an adrenal mass. There is, however, clear evidence that the vast majority of adrenal incidentalomas are benign adrenocortical adenomas. Table 1 Adrenal incidentalomas – frequency of the different underlying tumor types (adapted according ( )). Due to the nature of these studies, a selection bias is very probable (the populations studied not reflecting a random sample of all patients with an adrenal incidentalomas) and most likely (...) tumors), or histology after surgery or biopsy (for benign or malignant adrenal tumors) •Reporting 2×2 contingency table data or at least two indices of diagnostic accuracy (sensitivity, specificity, negative or positive predictive value) and disease prevalence •5496 abstracts b •525 potentially relevant articles •37 studies included in systematic review, 18 in meta-analysis •Major reasons for exclusion of articles were lack of test accuracy data, inadequate or unclear reference standard

2016 European Society of Endocrinology

203. Can exercise suppress tumour growth in advanced prostate cancer patients with sclerotic bone metastases? A randomised, controlled study protocol examining feasibility, safety and efficacy. Full Text available with Trip Pro

formation, growth and distribution by virtue of altered epigenetics and endocrine-paracrine activity. Given the impressive ability of targeted mechanical loading to interfere with metastasis-driven tumour formation in human osteolytic tumour cells, it is of equal interest to determine whether a similar effect is observed in sclerotic tumour cells. The study aims to (1) establish the feasibility and safety of a combined modular multimodal exercise programme with spinal isometric training in advanced (...) prostate cancer patients with sclerotic bone metastases and (2) examine whether targeted and supervised exercise can suppress sclerotic tumour growth and activity in spinal metastases in humans.A single-blinded, two-armed, randomised, controlled and explorative phase I clinical trial combining spinal isometric training with a modular multimodal exercise programme in 40 men with advanced prostate cancer and stable sclerotic spinal metastases. Participants will be randomly assigned to (1) the exercise

2017 BMJ open Controlled trial quality: uncertain

205. Excess mortality and life expectancy of individuals with type 1 diabetes: a rapid review

%). This peaked at 70-79 years. Another important contributor to YLL was death from neoplasm between the ages of 10 and 50 y. After the age of 50 years, death from neoplasm had a much lower contribution to the total YLL. This finding is in contrast with the SR by Huxley et al. where no increased risk of mortality from cancer was found, neither in women or men vs. the general population. KCE Report 314 Excess mortality and life expectancy of individuals with type 1 diabetes 17 In general (for all age groups (...) combined) the order of contribution to YLL is: 1 st circulatory/CV-disease, 2 nd endocrine/metabolic (acute complications: like hypoglycaemic coma or severe diabetic keto acidosis (DKA)), and 3 th neoplasm. The LE obtained is an estimate based on abridged period life tables. The total period covering June 2002 and November 2007 is a considerable period though still limited so that the database did not allow cohort ‘additional’ LE estimates. In principle, the collected data would have made it possible

2019 Belgian Health Care Knowledge Centre

206. Study to Evaluate Safety, PK, PD, Immunogenicity & Antitumor Activity of MSC-1 in Patients With Adv Solid Tumors

), and is intended to treat adult patients with Advanced Solid Tumors. In part 1, multiple dose levels of MSC-1 in patients with Advanced Solid Tumors will be studied to determine the recommended dose for further evaluation of safety and efficacy in Part 2. Condition or disease Intervention/treatment Phase Advanced Solid Tumors Pancreatic Cancer Non Small Cell Lung Cancer Ovarian Cancer Biological: MSC-1 Phase 1 Detailed Description: MSC-1 is a first-in-class, humanized monoclonal antibody (IgG1) which binds (...) in a subset of tumors across multiple solid tumor types. During dose escalation, patients with Advanced Solid Tumors will be treated with MSC-1 with the primary objective of determining the safety and tolerability of MSC-1 and defining an appropriate dose for further evaluation in dose expansion. MSC-1 will be administered intravenously (IV) until disease progression, unmanageable toxicity, withdrawal of consent or study termination. In dose expansion, up to 4 parallel cohorts of patients with LIF-High

2018 Clinical Trials

207. A Phase I Clinical Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Anti-PD-1 Antibody (HLX10) in Combination With Avastin Biosimilar (HLX04) in Patients With Advanced Solid Tumors

Details Study Description Go to Brief Summary: This is a single-center, open-label, dose-escalation Phase I clinical trial to evaluate the safety and the tolerability of HLX10-HLX04 combination therapy in patients with advanced solid tumors after failure of standard of care. Condition or disease Intervention/treatment Phase Solid Tumor Drug: HLX04 Drug: HLX10 Phase 1 Study Design Go to Layout table for study information Study Type : Interventional (Clinical Trial) Estimated Enrollment : 24 (...) of treatment up to 2 years ] Area under the plasma concentration versus time curve (AUC) for single dose and multiple doses Objective Response Rate (ORR) of HLX04 plus HLX10 in patients with advanced solid tumors [ Time Frame: Day 1 of treatment up to 2 years ] ORR determined by RECIST criteria Disease Control Rate (DCR) of HLX04 plus HLX10 in patients with advanced solid tumors [ Time Frame: Day 1 of treatment up to 2 years ] DCR determined by RECIST criteria Duration of Response (DOR) of HLX04 plus HLX10

2018 Clinical Trials

208. Shortness: an unknown phenotype of multiple endocrine neoplasia type 1. (Abstract)

Shortness: an unknown phenotype of multiple endocrine neoplasia type 1. An observation of shortness among the female participants of a regular screening program in multiple endocrine neoplasia type 1 (MEN1) patients has raised the question as to whether shortness represents a phenotype characteristic of the disease.The body height (cm) of genetically confirmed MEN1 patients at the time of diagnosis was compared with the body height of their unaffected relatives (parents, siblings, and children

2013 European Journal of Endocrinology

209. Tanycytic ependymoma of the filum terminale associated with multiple endocrine neoplasia Type 1: first reported case. (Abstract)

Tanycytic ependymoma of the filum terminale associated with multiple endocrine neoplasia Type 1: first reported case. Ependymoma associated with multiple endocrine neoplasia type 1 (MEN-1) is an extremely rare clinical entity. To the best of our knowledge, only five cases of ependymoma associated with MEN-1 have been previously described. Furthermore, there has been no case of tanycytic ependymoma of the filum terminale associated with MEN-1.The present case report illustrates a 53-year-old man (...) with tanycytic ependymoma of the filum terminale associated with MEN-1. We review the literature on ependymoma with MEN-1 and tanycytic ependymoma of the cauda equina region and also discuss the risk of recurrence.A case report.The patient presented with complaints of nocturnal pain in the lower back, accompanied by numbness around the anus and intermittent claudication for approximately 1 year. Magnetic resonance imaging (MRI) identified an intradural-enhancing, large mass lesion at the level from Th12

2013 The Spine Journal

211. Utility of Intraoperative Parathyroid Hormone Monitoring in Patients with Multiple Endocrine Neoplasia Type 1-Associated Primary Hyperparathyroidism Undergoing Initial Parathyroidectomy. (Abstract)

Utility of Intraoperative Parathyroid Hormone Monitoring in Patients with Multiple Endocrine Neoplasia Type 1-Associated Primary Hyperparathyroidism Undergoing Initial Parathyroidectomy. Intraoperative parathyroid hormone monitoring (IOPTH) is a widely used adjunct for primary hyperparathyroidism (pHPT). However, the benefit of IOPTH in familial pHPT, such as in multiple endocrine neoplasia type I (MEN1), remains unclear.We performed a retrospective analysis of 52 patients with MEN1-associated

2013 World Journal of Surgery

212. A systematic review of evidence on malignant spinal metastases: natural history and technologies for identifying patients at high risk of vertebral fracture and spinal cord compression

revealed that about 70% had evidence of metastatic bone disease. 43 High percentages have also been observed for thyroid, kidney and lung carcinomas. 18 Mechanism of metastasis There are three mechanisms by which a cancer can disseminate in the body: (1) direct seeding of body cavities or surfaces, (2) lymphatic spread and (3) haematogenous spread. 44 Direct dissemination of tumour cells is rare. It can, however, occur during surgery. 45 A direct seeding of body cavities and surfaces may occur when (...) is measured using the tumour/node/metastasis (TNM) classification. In this classification, ‘T’ refers to the size of the tumour, ‘N’ refers to spread of the tumour to lymph nodes and ‘M’ refers to distant metastases. 20 The treatment and prognosis of patients with breast cancer depend on the extent of the disease. 20 There are three types of receptors expressed on breast cancer cells, namely oestrogen receptor, progesterone receptor and human epidermal growth factor receptor 2. 20 Treatment of patients

2013 NIHR HTA programme

213. Multiple endocrine neoplasia type 1- presenting multiple lipomas and hypoglycemia onset Full Text available with Trip Pro

Multiple endocrine neoplasia type 1- presenting multiple lipomas and hypoglycemia onset Multiple endocrine neoplasia type 1 (MEN1), also called Wermer syndrome, is an autosomal dominant disorder characterized by tumors of the parathyroid glands, the anterior pituitary, and the endocrine pancreas.Here, we report a case of MEN1. Our patient was a 44-year-old woman who manifested typical features of MEN1, including insulinoma, pituitary tumors, and parathyroidoma, and exhibited multiple lipomas (...) and a gastrinoma with duodenal ulcers. She was admitted to our hospital because of recurrent massive bleeding of the upper gastrointestinal tract and hypoglycemia. The first operation for pituitary tumors was performed when she was 40 years old. According to these examinations and her clinical course, the patient was diagnosed with insulinoma and gastrinoma. She subsequently underwent surgery for the pancreatic tumors. The majority of these tumor cells were immunohistochemically positive for insulin

2012 The American journal of case reports

214. Middle segment-preserving pancreatectomy for metachronous intraductal papillary mucinous neoplasm after pancreatoduodenectomy: a case report Full Text available with Trip Pro

Middle segment-preserving pancreatectomy for metachronous intraductal papillary mucinous neoplasm after pancreatoduodenectomy: a case report Total pancreatectomy has occasionally been performed to treat patients with multiple lesions (such as intraductal papillary mucinous neoplasm (IPMN)) or patients who have undergone repeated pancreatic resection. However, deficiencies of the exocrine and endocrine functions worsen patients' quality of life. Recently, there have been several case reports (...) citing middle segment-preserving pancreatectomy (MSPP) as a safe procedure and beneficial with respect to preservation of the exocrine and endocrine functions. We herein report the case of a patient who underwent MSPP for repeat pancreatectomy for IPMN and in whom a favorable outcome was achieved. The patient, a 70-year-old man, was diagnosed with branch duct-type IPMN (BD-IPMN) with worrisome features in the pancreatic head and a single cyst in the pancreatic tail, during a preoperative examination

2017 Surgical Case Reports

215. Primary hyperparathyroidism in young people. When should we perform genetic testing for multiple endocrine neoplasia 1 (MEN-1)? Full Text available with Trip Pro

Primary hyperparathyroidism in young people. When should we perform genetic testing for multiple endocrine neoplasia 1 (MEN-1)? Multiple endocrine neoplasia (MEN-1) is a rare, autosomal dominant inherited disorder. Primary hyperparathyroidism (pHPT) is the most frequent and usually the earliest expression of MEN-1, with typical age of onset at 20-25 years. Early detection of the disease and correct treatment are therefore of great importance.A 31-year-old woman with osteogenesis imperfecta (...) was incidentally found also to have hypercalcemia and elevated PTH (pHPT). Exploratory neck surgery showed multiglandular parathyroid affection; she turned out to have MEN-1, but she was diagnosed 7 years after her debut of pHPT.The aim was to search literature on indications for performing mutational analysis in young patients with pHPT and no family history of MEN-1. PubMed was searched for English language articles, and words used were: MEN1 OR MEN-1 OR MEN type 1 OR multiple endocrine neoplasia 1

2014 Journal of Clinical Endocrinology and Metabolism

216. Long non-coding RNA metastasis associated in lung adenocarcinoma transcript 1 (MALAT1) interacts with estrogen receptor and predicted poor survival in breast cancer Full Text available with Trip Pro

Long non-coding RNA metastasis associated in lung adenocarcinoma transcript 1 (MALAT1) interacts with estrogen receptor and predicted poor survival in breast cancer Metastasis associated in lung adenocarcinoma transcript 1 (MALAT1), a lncRNA that was first recognized as a prognostic parameter for patient survival of stage I lung cancer, is up-regulated in multiple human malignancies, including breast cancer. However, the mechanism of its function remained elusive. In the current study (...) ). MALAT1 overexpression was also associated with poor RFS in tamoxifen treated ER-positive breast cancer patients, which might serve as a potential biomarker to predict endocrine treatment sensitivity.

2016 Oncotarget

217. Longitudinal Effects of Adjuvant Endocrine Therapy on the Quality of Life of Post-menopausal Women with Non-metastatic ER+ Breast Cancer: A Systematic Review. Full Text available with Trip Pro

Longitudinal Effects of Adjuvant Endocrine Therapy on the Quality of Life of Post-menopausal Women with Non-metastatic ER+ Breast Cancer: A Systematic Review. Anti-estrogen (ER) endocrine therapy is an effective treatment strategy in reducing breast cancer mortality. This therapy has a better therapeutic index than chemotherapy but can still affect patients' quality of life (QOL) over time.The objectives of this systematic review were to (1) describe QOL instruments used in ER-positive (ER (...) +) non-metastatic breast cancer trials and (2) document the longitudinal effects of adjuvant endocrine therapy on the QOL of post-menopausal women with ER+ non-metastatic breast cancer.We searched three electronic bibliographic databases for articles published from inception to October 2017 that described (1) a randomized controlled trial (RCT) of non-metastatic breast cancer containing an adjuvant endocrine regimen in at least one arm; (2) the use of a patient self-report measure assessing general

2018 PharmacoEconomics - open

219. Distribution of RET mutations in multiple endocrine neoplasia 2 in Denmark 1994-2014: a nationwide study. Full Text available with Trip Pro

Distribution of RET mutations in multiple endocrine neoplasia 2 in Denmark 1994-2014: a nationwide study. Germline mutations of the REarranged during Transfection (RET) proto-oncogene cause multiple endocrine neoplasia 2 (MEN2). It is unclear whether the distribution of RET mutations varies among populations. The first nationwide study of the distribution of RET mutations was conducted, and the results were compared to those of other populations.This retrospective cohort study included 1583 (...) patients who underwent RET gene testing in one of three centers covering all of Denmark between September 1994 and December 2014. Primary testing method was Sanger sequencing, which included exons 8-11 and 13-16. Mutations were defined according to the ARUP database July 1, 2016.RET mutations were identified in 163 patients from 36 apparently unrelated families. Among the 36 families 13 (36.1%) carried mutations in codon 611, four (11.1%) in codon 618, three (8.3%) in codon 620, one (2.8%) in codon 631

2016 Thyroid

220. When it is not just DKA; diabetic ketoacidosis as a first presentation of pancreatic adenocarcinoma. (Abstract)

. DKA occurs less commonly in patients with type 2 diabetes. Common precipitating factors for DKA in type 2 diabetes include medication noncompliance, infection, or possibly treatment with SGLT2 inhibitors. DKA as a first presentation of pancreatic cancer has been reported more with endocrine islet cell tumors but rarely with adenocarcinoma of the exocrine pancreas. Studies have shown that diabetes is an independent risk factor for development of pancreatic cancer. Other studies suggested (...) When it is not just DKA; diabetic ketoacidosis as a first presentation of pancreatic adenocarcinoma. Pancreatic cancer is the fourth leading cause of cancer related-deaths in the United States. Early symptoms of pancreatic cancer are nonspecific, and most cases are diagnosed at an advanced stage. DKA as a first presentation of pancreatic adenocarcinoma is a seldom reported condition. We present a case of a 59 year old female with type 2 diabetes, who presented to the emergency department

2018 American Journal of Emergency Medicine

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