Did Primary Healthcare Patients in Riyadh Experience Their First Migraine Episodes After a Stressful Event, and What Triggers and Relievers Do They Commonly Report?

Objective: This study aimed to investigate whether the first onset of migraine episodes among primary healthcare patients in Riyadh, Saudi Arabia, is preceded by a highly stressful event, as well as to identify common potential triggers and relievers reported by these patients.

Background: Migraine is a prevalent and debilitating neurological disorder. The pathophysiology of migraine involves complex interactions between genetic, neurological, and environmental factors, including the trigeminovascular system and neuropeptides such as calcitonin gene-related peptide (CGRP).

Multi-stakeholder sessions on major innovation topics in rare disease clinical trials.

Background: The European Joint Programme on Rare Diseases aims to enhance the rare diseases research ecosystem by bringing together stakeholders such as research funders, institutions and patient organizations. Work Package 20 focuses on the validation, use and development of innovative methodologies for rare disease clinical trials. This paper reports on the outcomes of a retreat held in April 2023, where areas for innovation and educational needs in rare disease clinical trials were discussed in multi-stakeholder sessions.

Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases-A secondary analysis of a randomized clinical trial.

Background: In clinical research, the most appropriate way to assess the effect of an intervention is to conduct a randomized controlled trial (RCT). In the field of rare diseases, conducting an RCT is challenging, resulting in a low rate of clinical trials, with a high frequency of early termination and unpublished trials. The aim of the EPISTOP trial was to compare outcomes in infants with tuberous sclerosis (TSC) who received vigabatrin preventively before the seizures onset with those who received it conventionally after.

Should the Left Atrial Appendage Closure (LAAC) Technique Be the Main Form of Stroke Prevention in Patients With Long-Standing Persistent or Permanent Atrial Fibrillation?

Currently, oral anticoagulants are considered the gold standard for stroke prevention in patients with atrial fibrillation. Despite the efficacy of oral anticoagulants in reducing stroke incidence, patients are at risk of developing adverse reactions such as excessive bleeding and bruising, and can also have drug-drug interactions. In the early 2000s, a minimally invasive technique called the left atrial appendage closure emerged as an alternative for stroke prevention in atrial fibrillation patients who could not tolerate oral anticoagulants.

Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.

Background: The conduct of rare disease clinical trials is still hampered by methodological problems. The number of patients suffering from a rare condition is variable, but may be very small and unfortunately statistical problems for small and finite populations have received less consideration. This paper describes the outline of the iSTORE project, its ambitions, and its methodological approaches.