Clinical pharmacology and pharmaceutical medicine - creation of a new specialist title for physicians in Belgium.

Belgium has a vibrant health eco-system, with world-class universities and hospitals, and a strong presence of pharmaceutical companies, resulting in a substantial contribution to drug development and a high number of clinical trials. Talent development is pivotal for further growth of this eco-system and to attract future professionals. Although physicians play an important role in this complex eco-system, with an estimated 400-450 Belgian physicians presently active in the field of the drug life cycle, Pharmaceutical Medicine and Clinical Pharmacology were not recognized as a speciality in Belgium until recently.

Harmonizing measurement tools: examining the concurrent validity of the Daily Activity Behaviors Questionnaire compared to the ActiGraph to assess 24-hour movement behaviors among adults.

Purpose: An accurate assessment of time spent in 24-hour movement behaviors (24 h-MBs) is crucial in exploring health related associations. This study aims to evaluate the concurrent validity of the Daily Activity Behavior Questionnaire (DABQ) compared to the ActiGraph using absolute and relative indicators of validity.

Methods: This cross-sectional observational study included 105 adults (45 ± 13 y/o, 54% female).

A comparative analysis of 24-hour movement behaviors features using different accelerometer metrics in adults: Implications for guideline compliance and associations with cardiometabolic health.

Background: Movement behavior features such as time use estimates, average acceleration and intensity gradient are crucial in understanding associations with cardiometabolic health. The aim of this study was to 1) compare movement behavior features processed by commonly used accelerometer metrics among adults (i.e.

Long-term evaluation of anterior thalamic deep brain stimulation for epilepsy in the European MORE registry.

Objective: Short-term outcomes of deep brain stimulation of the anterior nucleus of the thalamus (ANT-DBS) were reported for people with drug-resistant focal epilepsy (PwE). Because long-term data are still scarce, the Medtronic Registry for Epilepsy (MORE) evaluated clinical routine application of ANT-DBS.

Methods: In this multicenter registry, PwE with ANT-DBS were followed up for safety, efficacy, and battery longevity.

Explanatory variables of objectively measured 24-h movement behaviors in people with prediabetes and type 2 diabetes: A systematic review.

Aim: Physical activity (PA), sedentary behavior (SB) and sleep (i.e. 24-h movement behaviors) are associated with health indicators in people with prediabetes and type 2 diabetes (T2D).

Cross-sectional associations between 24-hour movement behaviors and cardiometabolic health among adults with type 2 diabetes mellitus: A comparison according to weight status.

Objectives: Type 2 diabetes mellitus (T2DM) is a chronic disease associated with overweight and obesity. Evidence suggests that 24-hour movement behaviors (24 h-MBs) play a crucial role in cardiometabolic health. However, it is not yet known if 24 h-MBs differ between weight status groups among people with T2DM (PwT2DM) and how 24 h-MBs are associated with their cardiometabolic health.

Somatic symptoms, pain, catastrophizing and the association with disability among children with heritable connective tissue disorders.

The aim of the present study was to investigate the nature and prevalence of nonspecific somatic symptoms, pain and catastrophizing in children with Heritable Connective Tissue Disorders (HCTD), and to determine their association with disability. This observational, multicenter study included 127 children, aged 4-18 years, with Marfan syndrome (MFS) (59%), Loeys-Dietz syndrome (LDS) (8%), Ehlers-Danlos syndromes (EDS) (12%) and hypermobile Ehlers-Danlos syndrome (hEDS) (23%). The assessments included the Children's Somatization Inventory or parent proxy (CSI, PCSI), pain visual-analogue scale (VAS), SUPERKIDZ body diagram, Pain Catastrophizing Scale Child or parent proxy (PCS-C, PCS-P) and Childhood Health Assessment Questionnaire (CHAQ-30).

Physical activity and physical fitness in children with heritable connective tissue disorders.

Objectives: Health problems in patients with heritable connective tissue disorders (HCTD) are diverse and complex and might lead to lower physical activity (PA) and physical fitness (PF). This study aimed to investigate the PA and PF of children with heritable connective tissue disorders (HCTD).

Methods: PA was assessed using an accelerometer-based activity monitor (ActivPAL) and the mobility subscale of the Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT).

RNA-based therapies in inherited retinal diseases.

Inherited retinal diseases (IRDs) are a genetically and phenotypically heterogeneous group of genetic eye disorders. There are more than 300 disease entities, and together this group of disorders affects millions of people globally and is a frequent cause of blindness or low-vision certification. However, each type is rare or ultra-rare.

An assessment of the proportion of LGB+ persons in the Belgian population, their identification as sexual minority, mental health and experienced minority stress.

Background: Previous studies report vast mental health problems in sexual minority people. Representative national proportion estimates on self-identifying LGB+ persons are missing in Belgium. Lacking data collection regarding sexual orientation in either census or governmental survey data limits our understanding of the true population sizes of different sexual orientation groups and their respective health outcomes.

Gene Therapy for Inherited Retinal Disease: Long-Term Durability of Effect.

The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65 mutation-associated inherited retinal dystrophy with viable retinal cells represents an important step in the development of ocular gene therapies. Herein, we review studies investigating the episomal persistence of different recombinant adeno-associated virus (rAAV) vector genomes and the preclinical and clinical evidence of long-term effects of different RPE65 gene replacement therapies. A targeted review of articles published between 1974 and January 2021 in Medline®, Embase®, and other databases was conducted, followed by a descriptive longitudinal analysis of the clinical trial outcomes of voretigene neparvovec.

Use and safety of aprotinin in routine clinical practice: A European postauthorisation safety study conducted in patients undergoing cardiac surgery.

Background: Aprotinin has been used to reduce blood loss and blood product transfusions in patients at high risk of major blood loss during cardiac surgery. Approval by the European Medicines Agency (EMA) for its current indication is limited to patients at high risk of major blood loss undergoing isolated coronary artery bypass graft surgery (iCABG).

Objective: To report current real-world data on the use and certain endpoints related to the safety of aprotinin in adult patients.

International Perspectives on Joint Hypermobility: A Synthesis of Current Science to Guide Clinical and Research Directions.

There is exponential clinical and research interest in joint hypermobility due to recognition of the complexity of identification, assessment, and its appropriate referral pathways, ultimately impacting management. This state-of-the-science review provides an international, multidisciplinary perspective on the presentation, etiology, and assessment of joint hypermobility, as it presents in those with and without a systemic condition. We synthesize the literature, propose standardizing the use of terminology and outcome measures, and suggest potential management directions.

The European Code of Cancer Practice.

There are considerable disparities between the quality of cancer care and clinical outcomes for cancer patients in different European countries, regions, hospitals and communities. These have persisted despite the introduction of many European and National Cancer Plans, an extensive portfolio of clinical guidelines and the existence of evidence based guidelines for the good practice in planning cancer healthcare systems. We describe the European Code of Cancer Practice which is a citizen and patient-centred accessible widely disseminated statement of the core requirements for good clinical cancer practice.

The Natural History of Leber Congenital Amaurosis and Cone-Rod Dystrophy Associated with Variants in the GUCY2D Gene.

Objective: To describe the spectrum of Leber congenital amaurosis (LCA) and cone-rod dystrophy (CORD) associated with the GUCY2D gene and to identify potential end points and optimal patient selection for future therapeutic trials.

Design: International, multicenter, retrospective cohort study.

Subjects: Eighty-two patients with GUCY2D-associated LCA or CORD from 54 families.

X-Linked Retinoschisis: Novel Clinical Observations and Genetic Spectrum in 340 Patients.

Purpose: To describe the natural course, phenotype, and genotype of patients with X-linked retinoschisis (XLRS).

Design: Retrospective cohort study.

Participants: Three hundred forty patients with XLRS from 178 presumably unrelated families.

Innovation, value and reimbursement in radiation and complex surgical oncology: Time to rethink.

Background And Purpose: Complex surgery and radiotherapy are the central pillars of loco-regional oncology treatment. This paper describes the reimbursement schemes used in radiation and complex surgical oncology, reports on literature and policy reviews.

Material And Methods: A systematic review of the literature of the reimbursement models has been carried out separately for radiotherapy and complex cancer surgery based on PRISMA guidelines.

Innovation, value and reimbursement in radiation and complex surgical oncology: Time to rethink.

Background And Purpose: Complex surgery and radiotherapy are the central pillars of loco-regional oncology treatment. This paper describes the reimbursement schemes used in radiation and complex surgical oncology, reports on literature and policy reviews.

Material And Methods: A systematic review of the literature of the reimbursement models has been carried out separately for radiotherapy and complex cancer surgery based on PRISMA guidelines.