A core outcome set for best care for the dying person: Results of an international Delphi study and consensus meeting.

Background: Outcome measurement is essential to progress clinical practice and improve patient care.

Aim: To develop a Core Outcome Set for best care for the dying person.

Design: We followed the Core Outcome Measures in Effectiveness Trials (COMET) Initiative guidelines, which involved identifying potential outcomes via a systematic literature review (n = 619 papers) and from participants in the "iLIVE" project (10 countries: 101 patients, 37 family members, 63 clinicians), followed by a two-round Delphi study, and a consensus meeting.

Enhanced thrombin and plasmin generation profiles in alpha-2-antiplasmin-deficient patients: Data from the Rare Bleeding disorders in the Netherlands study.

Background: α2-Antiplasmin (A2AP) deficiency is a rare and often unidentified disorder characterized by increased fibrinolysis and subsequent bleeding. Global hemostasis assays may increase insight into the altered coagulation and fibrinolysis in these patients.

Objectives: To explore thrombin and plasmin generation profiles in A2AP-deficient patients, corresponding A2AP activity levels and associated bleeding phenotypes.

Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.

Current management guidelines for urea cycle disorders (UCDs) offer clear strategies, incorporating both authorized and non-authorized medicinal products (including intravenous formulations and products regulated as food). These varying product categories are subject to specific accessibility challenges related to availability, reimbursement, and pricing. The aim of this study is to identify potential obstacles to optimal UCD treatment implementation in European clinical practice.

Interferon gamma-mediated prevention of tumor progression in a mouse model of multiple myeloma.

Malignant plasma cells in multiple myeloma patients reside in the bone marrow and continuously interact with local immune cells. Progression and therapy response are influenced by this immune environment, highlighting the need for a detailed understanding of endogenous immune responses to malignant plasma cells. Here we used the 5TGM1 murine transfer model of multiple myeloma to dissect early immune responses to myeloma cells.

Glucocorticoids and HPA axis regulation in the stress-obesity connection: A comprehensive overview of biological, physiological and behavioural dimensions.

Chronic stress, characterized by increased long-term exposure to the glucocorticoid hormone cortisol, is increasingly linked to obesity development. Still, various knowledge gaps persist, including on underlying pathophysiological mechanisms. The aim of the current review is to provide the latest insights on the connection between stress and obesity.

A phase I dose-escalation study of LRP5/6 antagonist BI 905677 in patients with advanced solid tumors.

Background: Aberrant Wnt pathway signaling has been implicated in the development of many cancers. Targeting of low-density lipoprotein receptor-related protein 5/6 (LRP5/6) co-receptors inhibits Wnt signaling and may be a novel therapy. BI 905677 is an LRP5/6 antagonist that has demonstrated preclinical antitumor activity.

High iASPP (PPP1R13L) expression is an independent predictor of adverse clinical outcome in acute myeloid leukemia (AML).

Apoptosis-stimulating proteins of p53 (ASPPs) are a family of proteins that modulate key tumor suppressor pathways via direct interaction with p53. Deregulation of these proteins promotes cancer development and impairs sensitivity to systemic (chemo)therapy and radiation. In this study, we describe that the inhibitor of ASPP (iASPP) is frequently highly expressed in acute myeloid leukemia (AML) and that overexpression correlates with a poor clinical outcome.

The association between childhood maltreatment and pain sensitivity in a high-risk adolescent population.

The risk of developing chronic pain is twice as high among people with a history of childhood maltreatment compared to those without these experiences. It is unclear, however, whether childhood maltreatment might lead to lower or higher perception of pain. In this paper, we investigate the association between childhood maltreatment and pain sensitivity.

Determinants of sustainable work participation after spinal cord injury in The Netherlands.

Purpose: Work participation of persons with spinal cord injury (SCI) is lower compared to the overall Dutch population, with determinants largely unknown.

Aim: To identify the determinants of sustainable work participation for persons with SCI.

Methods: A cross-sectional multicenter study was conducted in eight Dutch rehabilitation centers.

Comparative analysis of treatment modalities for solitary, small (≤3 cm) hepatocellular carcinoma: A systematic review and network meta-analysis of oncologic outcomes.

Background: Solitary hepatocellular carcinoma measuring ≤3 cm represents approximately 30% of hepatocellular carcinoma cases, yet treatment guidelines lack robust evidence. This study compares oncologic outcomes after ablation, liver resection, and liver transplantation for solitary, small hepatocellular carcinoma.

Methods: We systematically searched databases up to 7 February 2022, for studies including adults with solitary hepatocellular carcinoma ≤3 cm treated by any ablation, liver resection, or liver transplantation.

An Application of the Checklist for Health Economic Quality Evaluations in a Systematic Review Setting.

Objective: Quality assessment tools serve an important role in evaluating economic evaluations. This article showcases the first application of the Checklist for Health Economic Quality Evaluations (CHEQUE) tool in a systematic review setting and offers descriptive reflections on its use.

Methods: We applied CHEQUE to 21 diverse economic evaluations in a systematic review of medical education.

Margin and robustness settings for a library-of-plans IMPT strategy for locally advanced cervical cancer.

This study aims to determine a margin and robustness setting for treating locally advanced cervical cancer (LACC) with a library-of-plans (LoP) based online-adaptive intensity-modulated proton therapy (IMPT).We analyzed 13 LACC patients with delineated planning and weekly repeat CT scans (reCTs). For each patient, 120 IMPT treatments of 25 fractions were simulated with a LoPs approach.

Exploring the need for a preload on the estimation of permeability, vessel radius, and relative cerebral blood volume in MR vascular fingerprinting-based dynamic susceptibility contrast perfusion imaging.

Purpose: Dynamic susceptibility contrast (DSC) MRI is commonly part of the clinical brain tumor imaging protocol. Usually, a preload of contrast agent is administered to minimize contrast-leakage T effects. However, recent studies have indicated that with adaptation of scan parameters (in particular, low flip angle), a preload is not required.

Reducing Overtreatment of Prostate Cancer Patients: Revisiting the European Association of Urology Pretreatment Risk Group Classification Using Long-term Follow-up Data from the European Randomized Study of Screening for Prostate Cancer Rotterdam.

Background And Objective: Tailored treatment for prostate cancer (PCa) requires accurate risk stratification. This study examines the effectiveness of the European Association of Urology (EAU) classification in predicting long-term PCa-specific mortality (PCSM) and assesses whether an alternative system can improve the identification of patients with low-risk disease.

Methods: This study included two cohorts of patients with localized PCa: one with screen-detected PCa (n = 1563; S-cohort) and the other with clinically detected PCa (n = 755; C-cohort), all from a population-based, randomized screening study, who underwent primary radical prostatectomy or radiation monotherapy.

Consensus-Based Recommendations for Designing, Delivering, Evaluating, and Reporting Exercise Intervention Research Involving People Living With a Spinal Cord Injury.

Objectives: To establish recommendations for designing, delivering, evaluating, and reporting exercise intervention research to improve fitness-related outcomes in people living with spinal cord injury (PwSCI).

Design: International consensus process.

Setting: (1) An expert panel was established consisting of 9 members of the governing panel of the International Spinal Cord Society Physical Activity Special Interest Group and 9 additional scientists who authored or co-authored ≥1 exercise randomized controlled trial paper involving PwSCI.

Evaluating and reporting LET and RBE-weighted dose in proton therapy for glioma - The Dutch approach.

Background And Purpose: With proton therapy, the relative biological effectiveness (RBE) accounts for increased DNA damage caused by higher linear energy transfer (LET) compared to photons. However, the LET and hence the RBE varies along the proton range, particularly at the Bragg peak, introducing challenges in proton treatment planning for brain tumors. The aim of this paper is to standardize evaluating and reporting LET and RBE in proton therapy for patients with grade 2 and 3 IDH mutant gliomas among the Dutch proton therapy centers.