Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value-based healthcare approach and systematic benefit-risk assessment.

Off-label repurposing of empagliflozin allows pathomechanism-based treatment of neutropenia/neutrophil-dysfunction in glycogen storage disease type Ib (GSDIb). From a value-based healthcare (VBHC) perspective, we here retrospectively studied patient-reported, clinical and pharmacoeconomic outcomes in 11 GSDIb individuals before and under empagliflozin at two centers (the Netherlands [NL], Austria [AT]), including a budget impact analysis, sensitivity-analysis, and systematic benefit-risk assessment. Under empagliflozin, all GSDIb individuals reported improved quality-of-life-scores.

Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.

Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.

Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables.

Fast assessment and management of chest pain patients without ST-elevation in the pre-hospital gateway (FamouS Triage): ruling out a myocardial infarction at home with the modified HEART score.

Background: The first study of the FamouS Triage project investigates the feasibility of ruling out a myocardial infarction in pre-hospital chest pain patients without electrocardiographic ST-segment elevation by using the modified HEART score at the patient's home, incorporating only a single highly sensitive troponin T measurement.

Methods: A venous blood sample was drawn in the ambulance from 1127 consecutive chest pain patients for measurement of the pre-hospital highly sensitive troponin T levels, in order to establish a pre-hospital HEART score (i.e.

A nurse-initiated pain protocol in the ED improves pain treatment in patients with acute musculoskeletal pain.

While acute musculoskeletal pain is a frequent complaint, its management is often neglected. An implementation of a nurse-initiated pain protocol based on the algorithm of a Dutch pain management guideline in the emergency department might improve this. A pre-post intervention study was performed as part of the prospective PROTACT follow-up study.

Prehospital paths and hospital arrival time of patients with acute coronary syndrome or stroke, a prospective observational study.

Background: Patients with a presumed diagnosis of acute coronary syndrome (ACS) or stroke may have had contact with several healthcare providers prior to hospital arrival. The aim of this study was to describe the various prehospital paths and the effect on time delays of patients with ACS or stroke.

Methods: This prospective observational study included patients with presumed ACS or stroke who may choose to contact four different types of health care providers.

Pain management in the emergency chain: the use and effectiveness of pain management in patients with acute musculoskeletal pain.

Objective: While acute musculoskeletal pain is a frequent complaint in emergency care, its management is often neglected, placing patients at risk for insufficient pain relief. Our aim is to investigate how often pain management is provided in the prehospital phase and emergency department (ED) and how this affects pain relief. A secondary goal is to identify prognostic factors for clinically relevant pain relief.

Fast assessment and management of chest pain without ST-elevation in the pre-hospital gateway: rationale and design.

Background: For chest pain patients without ST-segment elevation in the pre-hospital setting, current clinical guidelines merely offer in-hospital risk stratification and management, as opposed to chest pain patients with ST-segment elevation for whom there is a straightforward pre-hospital strategy for diagnosis, medication regimen and logistics. The FAMOUS TRIAGE study will assess the effects of introducing a pre-hospital triage system that reliably stratifies chest pain patients without ST-segment elevation into 1) patients at high risk for NSTEMI requiring a direct transfer to a PCI-hospital; 2) patients at intermediate risk for a major adverse cardiac event (MACE) who could be evaluated at the nearest non-PCI hospital; and 3) patients at low risk for MACE (benign non-cardiac chest pain) who could have further evaluation at home or in a primary care setting.

Methods: The FAMOUS TRIAGE study will be performed in three phases.