Development and external validation of a prediction model for overall survival after resection of distal cholangiocarcinoma.

Background: Various prognostic factors are associated with overall survival (OS) after resection of distal cholangiocarcinoma (dCCA). The objective of this study was to develop and validate a prediction model for 3-year OS after pancreatoduodenectomy for dCCA.

Methods: The derivation cohort consisted of all patients who underwent pancreatoduodenectomy for dCCA in the Netherlands (2009-2016).

Characterization of HNRNPA1 mutations defines diversity in pathogenic mechanisms and clinical presentation.

Mutations in HNRNPA1 encoding heterogeneous nuclear ribonucleoprotein (hnRNP) A1 are a rare cause of amyotrophic lateral sclerosis (ALS) and multisystem proteinopathy (MSP). hnRNPA1 is part of the group of RNA-binding proteins (RBPs) that assemble with RNA to form RNPs. hnRNPs are concentrated in the nucleus and function in pre-mRNA splicing, mRNA stability, and the regulation of transcription and translation.

Extended criteria for liver transplantation in hepatocellular carcinoma. A retrospective, multicentric validation study in Belgium.

Background: Recent studies indicate that a group of patients with cirrhosis receiving a liver transplantation for hepatocellular cancer (HCC) beyond the Milan Criteria (MC) can achieve a similar outcome compared to patients within these criteria. This study aims to investigate the value of the Asan critera (AC), up-to-7 criteria (UT7), French alpha-foetoprotein (AFP) model and Metroticket 2.0 (MT2.

Risk of advanced lesions in patients with branch-duct IPMN and relative indications for surgery according to European evidence-based guidelines.

Background: European evidence-based guidelines proposed surgery for branch-duct intraductal papillary mucinous neoplasms (BD-IPMNs) based on the presence of 1-2 relative indications, depending on the comorbidity burden.

Aims: To assess the accuracy of the guidelines in patients with relative indications in a surgical cohort of demonstrated BD-IPMNs.

Methods: This report describes a multi-centre, observational, retrospective study.

Efficacy and safety of radiofrequency ablation of Barrett's esophagus in the absence of reimbursement: a multicenter prospective Belgian registry.

Background: Radiofrequency ablation (RFA), combined with endoscopic resection, can be used as a primary treatment for low grade dysplasia, high grade dysplasia, and early esophageal adenocarcinoma (EAC) in Barrett's esophagus (BE). The aim of the Belgian RFA registry is to capture the real-life outcome of endoscopic therapy for BE with RFA and to assess efficacy and safety outside study protocols, in the absence of reimbursement.

Patients And Methods: Between February 2008 and January 2017, data from 7 different expert centers were prospectively collected in the registry.

Technical Aspects of Orthotopic Liver Transplantation-a Survey-Based Study Within the Eurotransplant, Swisstransplant, Scandiatransplant, and British Transplantation Society Networks.

Background: Orthotopic liver transplantation (OLT) has emerged as the mainstay of treatment for end-stage liver disease. However, technical aspects of OLT are still subject of ongoing debate and are widely based on personal experience and local institutional protocols.

Methods: An international online survey was sent out to all liver transplant centers (n = 52) within the Eurotransplant, Swisstransplant, Scandiatransplant, and British Transplant Society networks.

European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.

Background And Purpose: Pompe disease is a rare inheritable muscle disorder for which enzyme replacement therapy (ERT) has been available since 2006. Uniform criteria for starting and stopping ERT in adult patients were developed and reported here.

Methods: Three consensus meetings were organized through the European Pompe Consortium, a network of experts from 11 European countries in the field of Pompe disease.

Hepatic Epithelioid Hemangioendothelioma and Adult Liver Transplantation: Proposal for a Prognostic Score Based on the Analysis of the ELTR-ELITA Registry.

Background: Hepatic epithelioid hemangioendothelioma (HEHE) is a rare vascular tumor which has an intermediate aggressive behavior. Although the value of liver transplantation (LT) is well established, its place in the management of HEHE is still unclear. The aim of this study is to confirm, based on a very large patient cohort, the value of LT in the management of HEHE and to identify risk factors for post-LT recurrence.

Cramp-fasciculation syndrome associated with monofocal motor neuropathy.

Introduction: Cramp-fasciculation syndrome is a peripheral nerve hyperexcitability disorder, which could be caused by inflammatory neuropathy.

Case Report: We describe a 51-year-old woman who presented with a 4- to 5-year history of fasciculations and painful cramping of the right thenar eminence.

Results: Electrophysiological studies showed motor conduction block in the right median nerve between the axilla and the elbow with fasciculation potentials and cramp discharges on electromyography in the right abductor pollicis brevis muscle.

Delta-slope of alpha-fetoprotein improves the ability to select liver transplant patients with hepatocellular cancer.

Objective: The aim of the present study was to investigate the role of different alpha-foetoprotein (AFP) determinations in order to propose a new model aimed at predicting intention-to-treat (ITT) death and post- liver transplantation (LT) recurrence in a cohort of patients with hepatocellular cancer (HCC) enlisted for LT.

Background: Recent studies have increasingly focused on the role of AFP as a useful tool for patient selection in the setting of LT for HCC. However, no definitive AFP model has been definitively validated.

Grip strength comparison in immune-mediated neuropathies: Vigorimeter vs. Jamar.

The Jamar dynamometer and Vigorimeter have been used to assess grip strength in immune-mediated neuropathies, but have never been compared to each other. Therefore, we performed a comparison study between these two devices in patients with immune-mediated neuropathies. Grip strength data were collected in 102 cross-sectional stable and 163 longitudinal (new diagnoses or changing condition) patients with Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), gammopathy-related polyneuropathy (MGUSP), and multifocal motor neuropathy (MMN).

Outcome measures in neuromuscular disease: is the world still flat?

Valid, responsive, and meaningful outcome measures for the measurement of the impairment, activity limitations, and quality of life in patients with neuromuscular disease are crucial to identify the natural history of disease and benefits of therapy in clinical practice and trials. Although understanding of many aspects of neuromuscular diseases has advanced dramatically, the development of outcome measures has received less attention. The scales developed from Rasch theory by the PeriNomS Group represent the biggest significant shift in thought in neuromuscular outcome measures for decades.

Effects of low frequency filtering on distal compound muscle action potential duration for diagnosis of CIDP: A Japanese-European multicenter prospective study.

Objective: The duration of the distal compound muscle action potential (DCMAP) is a useful index to detect demyelination in the distal nerve segments. However in published electrodiagnostic criteria for chronic inflammatory demyelinating polyneuropathy (CIDP), the cut-off values of DCMAP duration are defined using an EMG low frequency filter of only 20 Hz. We aimed to provide widely-available reference data using several low cut filters.

A different view on predictors of pulmonary hypertension in secundum atrial septal defect.

Background/objectives: Pulmonary arterial hypertension is an important complication in hemodynamically relevant atrial septal defects (ASD) and negatively affects outcome. This retrospective study aimed at (1) estimating the prevalence of pulmonary hypertension (PH) in patients with secundum ASD and (2) identifying predictors of PH development or persistence after ASD closure.

Methods: Consecutive patients with an isolated secundum ASD from the Belgian Registry on Adult Congenital Heart Disease were studied.

Hepatocellular cancer: how to expand safely inclusion criteria for liver transplantation.

Purpose Of Review: The Milan criteria are still considered to be the best ones to select patients with hepatocellular cancer (HCC) for liver transplantation. Although the Milan criteria allowed lowering the incidence of tumor recurrence to a remarkable 10%, there is growing evidence that high numbers of patients were unrightfully excluded from a curative liver transplantation when exceeding these criteria. New strategies have been advocated during recent years with the intent not only to enlarge the number of potential transplant candidates, but also to select recipients with the lowest biological risk of recurrence.

Liver transplantation for metastatic liver malignancies.

Purpose Of Review: Liver transplantation is a validated treatment of primary hepatobiliary tumours. Over the last decade, a renewed interest for liver transplantation as a curative treatment of colorectal liver metastasis (CR-LM) and neuro-endocrine metastasis (NET-LM) has developed.

Recent Findings: The ELTR and UNOS analyses showed that liver transplantation may offer excellent disease-free survival (ranging from 30 to 77%) in case of NET-LM, on the condition that stringent selection criteria are implemented.

Laing early-onset distal myopathy in a Belgian family.

We report the first Belgian family with Laing early-onset distal myopathy (MPD1). The proposita started limping at age 7. Later, there was severe weakness of proximal and distal muscles, including neck flexors.

Neutrophil and platelet-to-lymphocyte ratio as new predictors of dropout and recurrence after liver transplantation for hepatocellular cancer.

There is increasing evidence that systemic inflammation markers like neutrophil (NLR) and platelet-to-lymphocyte ratios (PLR) may play a role in the outcome of hepatocellular cancer (HCC). Between January 1994 and March 2012, 181 patients with HCC were registered on the transplant waiting list: 35 (19.3%) patients dropped out during the waiting period and 146 (80.

Hepatic hemangiosarcoma: an absolute contraindication to liver transplantation--the European Liver Transplant Registry experience.

Background: Liver transplantation (LT) is performed for hemangiosarcoma (HAS) despite disappointing results.

Methods: Retrospective study of 14 males and 8 females reported to the European Liver Transplant Registry. In view of the difficult differential diagnosis between HAS and hemangioendothelioma (HE), the study was deliberately restricted to the period 1986 to 2004 to allow comparison of clinical and biochemical behavior of HAS and HE liver recipients transplanted during the same time period.

Liver transplantation for hepatocellular cancer: UCL experience in 137 adult cirrhotic patients. Alpha-foetoprotein level and locoregional treatment as refined selection criteria.

Liver transplantation (LT) is a validated treatment for selected cirrhotics with hepatocellular cancer (HCC). A retrospective single center study including 137 recipients having proven HCC was done to refine inclusion criteria for LT as well as to look at impact of locoregional treatment (LRT) on outcome. At pre-LT imaging, 42 (30.

Liver transplantation and vascular tumours.

Based on analysis of the literature and of the audited ELITA (European Liver Intestinal Transplant Association)-ELTR (European Liver Transplant Registry) data, the place of liver transplantation (LT) in the treatment of vascular tumours is discussed. Hepatic epithelioid haemangioendothelioma has currently become a good indication for LT with 5- and 10-year post-LT patient survival rates of 83% and 74% respectively and 5- and 10-year recurrence-free survival rates of 82% and 64% respectively. In contrast, the results of LT for haemangiosarcoma (HAS) are disastrous with an universal tumour recurrence within 6 months and no single patient survival after 2 years.

Liver transplantation and neuroendocrine tumors: lessons from a single centre experience and from the literature review.

Neuroendocrine tumor (NET) metastases represent at this moment the only accepted indication of liver transplantation (LT) for liver secondaries. Between 1984-2007, nine (1.1%) of 824 adult LTs were performed because of NET.

The place of liver transplantation in the treatment of hepatic epitheloid hemangioendothelioma: report of the European liver transplant registry.

Background: Hepatic epitheloid hemangioendothelioma (HEHE) is a rare low-grade vascular tumor. Its treatment algorithm is still unclear mainly due to a lack of larger clinical experiences with detailed long-term follow-up.

Material And Methods: Fifty-nine patients, reported to the European Liver Transplant Registry, were analyzed to define the role of liver transplantation (LT) in the treatment of this disease.