The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.

Hereditary Angioedema (HAE) is a rare and disabling disease for which early diagnosis and effective therapy are critical. This revision and update of the global WAO/EAACI guideline on the diagnosis and management of HAE provides up-to-date guidance for the management of HAE. For this update and revision of the guideline, an international panel of experts reviewed the existing evidence, developed 28 recommendations, and established consensus by an online DELPHI process.

Reversing direct factor Xa or thrombin inhibitors: Factor V addition to prothrombin complex concentrate is beneficial in vitro.

Background: Prothrombin complex concentrate (PCC) is a human plasma-derived mixture of partially purified vitamin K-dependent coagulation factors (VKCF). Current therapeutic indication is treatment and perioperative prophylaxis of bleeding in acquired VKCF deficiency. Off-label uses include treatment of direct factor Xa- or thrombin inhibitor-associated bleeds, treatment of trauma-induced coagulopathy, and hemorrhagic complications in patients with liver disease.

Clinical impact of the worldwide shortage of verteporfin (Visudyne®) on ophthalmic care.

Introduction: Since July 2021, a worldwide shortage of verteporfin (Visudyne®) occurred: an essential medicine required for photodynamic therapy (PDT). PDT with verteporfin has a broad range of indications in ophthalmology, including chronic central serous chorioretinopathy, polypoidal choroidal vasculopathy and choroidal haemangioma. For these disorders, PDT is either the first-choice treatment or regarded as a major treatment option.

The international WAO/EAACI guideline for the management of hereditary angioedema-The 2021 revision and update.

Hereditary angioedema (HAE) is a rare and disabling disease for which early diagnosis and effective therapy are critical. This revision and update of the global WAO/EAACI guideline on the diagnosis and management of HAE provides up-to-date guidance for the management of HAE. For this update and revision of the guideline, an international panel of experts reviewed the existing evidence, developed 28 recommendations, and established consensus by an online DELPHI process.

Performance of risk scores for coronary artery disease: a retrospective cohort study of patients with chest pain in urgent primary care.

Objective: To evaluate the diagnostic performance of the Marburg Heart Score (MHS), INTERCHEST, Gencer rule, Bruins Slot rule and compare these with unaided clinical judgement in patients with chest pain in urgent primary care.

Design: Retrospective, cohort study.

Setting: Regional primary care facility responsible for out-of-hours primary care for a quarter-million people in the Netherlands.

Drug Repurposing for Rare Diseases: A Role for Academia.

The European Commission highlights in its Pharmaceutical Strategy the role of academic researchers in drug repurposing, especially in the development of orphan medicinal products (OMPs). This study summarizes the contribution of academia over the last 5 years to registered repurposed OMPs and describes barriers to success, based upon three real world cases. OMPs granted marketing authorization between January 2016 and December 2020 were reviewed for repurposing and whether the idea originated from academia or industry.

Manual QT interval measurement with a smartphone-operated single-lead ECG versus 12-lead ECG: a within-patient diagnostic validation study in primary care.

Objective: To determine the accuracy of QT measurement in a smartphone-operated, single-lead ECG (1L-ECG) device (AliveCor KardiaMobile 1L).

Design: Cross-sectional, within-patient diagnostic validation study.

Setting/participants: Patients underwent a 12-lead ECG (12L-ECG) for any non-acute indication in primary care, April 2017-July 2018.

Intramuscular corticosteroid injections in seasonal allergic rhinitis: A systematic review.

Objective: Seasonal allergic rhinitis (SAR) is an exaggerated immunological reaction to allergens (pollen) in the air. In a small subgroup of patients, SAR can be difficult to control with first-line therapy. Intramuscular corticosteroid injections (IMCIs) are an additional treatment in this subgroup of SAR patients.

Child neurocognitive functioning influences the effectiveness of specific techniques in behavioral teacher training for ADHD: Moderator analyses from a randomized controlled microtrial.

Background: Childhood attention-deficit/hyperactivity disorder (ADHD) is associated with several neurocognitive impairments. Whether these impairments influence the effectiveness of techniques that are commonly used in behavioral teacher training for ADHD has not been investigated so far.

Method: In this microtrial, teachers of 90 children with ADHD symptoms (6-12 years) were randomly assigned to a short intervention consisting of either antecedent-based (stimulus-control) techniques or consequent-based (contingency management) techniques, or to a waitlist control condition.

Do clinical practice guidelines consider evidence about diagnostic test consequences on patient-relevant outcomes? A critical document analysis.

Rationale, Aims And Objectives: Supporting evidence for diagnostic test recommendations in clinical practice guidelines (CPGs) should not only include diagnostic accuracy, but also downstream consequences of the test result on patient-relevant outcomes. The aim of this study is to assess the extent to which evidence-based CPGs about diagnostic tests cover all relevant test-treatment pathway components.

Methods: We performed a systematic document analysis and quality assessment of publicly accessible CPGs about three common diagnostic tests: C-reactive protein, colonoscopy and fractional exhaled nitric oxide.

ACTION-1: study protocol for a randomised controlled trial on ACT-guided heparinization during open abdominal aortic aneurysm repair.

Background: Heparin is used worldwide for 70 years during all non-cardiac arterial procedures (NCAP) to reduce thrombo-embolic complications (TEC). But heparin also increases blood loss causing possible harm for the patient. Heparin has an unpredictable effect in the individual patient.

Results of an International Survey on Feeding Management in Infants With Short Bowel Syndrome-Associated Intestinal Failure.

Objectives: Short bowel syndrome (SBS) is a complex and rare condition (incidence 1200/100,000 live births) that requires a multidisciplinary team approach to management. In January 2019, the first European Reference Network on Rare and Inherited Congenital Anomalies (ERNICA) Intestinal Failure (IF) workshop was held. Several questions about the strategies used in managing IF associated with SBS were devised.

New methodology for measuring intraocular lens performance using acuity reserve.

Purpose: To validate a new methodology of intraocular lens (IOL) assessment using acuity reserve analysis via area under the curve (AUC) to assess intermediate vision.

Setting: Study 1 was conducted at 17 sites in Australia, Chile, and Europe. Study 2 was conducted at 15 sites in Australia, Brazil, and Europe.

Budget Impact of Restrictive Strategy Versus Usual Care for Cholecystectomy (SECURE-Trial).

Objectives: A cost-effectiveness analysis of a multicenter randomized-controlled trial comparing restrictive strategy versus usual care in patients with gallstones showed that savings by restrictive strategy could not compensate for the lower proportion of pain-free patients. However, four subgroups based on combined stratification factors resulted in less cholecystectomies and more pain-free patients in restrictive strategy (female-low volume-BMI > 30, female-low volume-BMI25-30, female-high volume-BMI25-30, and male-low volume-BMI < 25). The aim of this study was to explore the budget impact from a hospital healthcare perspective of implementation of restrictive strategy in these subgroups.

Quality of life in patients with pulmonary embolism treated with edoxaban versus warfarin.

Background: Long-term sequelae of acute pulmonary embolism (PE) include decreased quality of life (QoL). Evidence suggests that adequacy of initial anticoagulant treatment in the acute phase of venous thrombosis has a key impact on late postthrombotic complications. We hypothesize that patients with acute PE treated with edoxaban for acute PE experience have improved QoL compared to those treated with warfarin.

Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia.

Objectives: Mexiletine is a long-known drug used for the treatment of arrhythmias and repurposed in the 1980s for patients with nondystrophic myotonia (NDM). Recently, the price of mexiletine in Europe increased significantly after registration as an orphan drug for NDM. This led to international discussions on affordability and willingness to reimburse mexiletine in the absence of background information that would justify such a price.

Family integrated care: Supporting parents as primary caregivers in the neonatal intensive care unit.

Family integrated care (FICare) is a collaborative model of neonatal care which aims to address the negative impacts of the neonatal intensive care unit (NICU) environment by involving parents as equal partners, minimizing separation, and supporting parent-infant closeness. FICare incorporates psychological, educational, communication, and environmental strategies to support parents to cope with the NICU environment and to prepare them to be able to emotionally, cognitively, and physically care for their infant. FICare has been associated with improved infant feeding, growth, and parent wellbeing and self-efficacy; important mediators for long-term improved infant neurodevelopmental and behavioural outcomes.

Risk Assessment Models for Thrombosis and Anticoagulant-Related Bleeding in Ambulatory Cancer Patients.

Cancer patients have a high risk of developing venous thromboembolism and arterial thrombosis, along with an increased risk of anticoagulant-related bleeding with primary and secondary prophylaxis of cancer-associated thrombosis. Decisions on initiation, dosing, and duration of anticoagulant therapy for prevention and treatment of cancer-associated thrombosis are challenging, as clinicians have to balance patients' individual risk of (recurrent) thrombosis against the risk of bleeding complications. For this purpose, several dedicated risk assessment models for venous thromboembolism in cancer patients have been suggested.

Effectiveness and Policy Determinants of Sugar-Sweetened Beverage Taxes.

Sugar consumption is on the rise globally with detrimental (oral) health effects. There is ample evidence that sugar-sweetened beverage (SSB) taxes can efficiently reduce sugar consumption. However, evidence alone is seldom enough to implement a policy.

Dynamics of antibodies to SARS-CoV-2 in convalescent plasma donors.

Objectives: Characterisation of the human antibody response to SARS-CoV-2 infection is vital for serosurveillance purposes and for treatment options such as transfusion with convalescent plasma or immunoglobulin products derived from convalescent plasma. In this study, we longitudinally and quantitatively analysed antibody responses in RT-PCR-positive SARS-CoV-2 convalescent adults during the first 250 days after onset of symptoms.

Methods: We measured antibody responses to the receptor-binding domain (RBD) of the SARS-CoV-2 spike protein and the nucleocapsid protein in 844 longitudinal samples from 151 RT-PCR-positive SARS-CoV-2 convalescent adults.

Peach allergen Pru p 1 content is generally low in fruit but with large variation in different varieties.

Background: Pru p 1 is a major allergen in peach and nectarine, and the different content in varieties may affect the degree of allergic reactions. This study aimed to quantify Pru p 1 levels in representative peach varieties and select hypoallergenic Pru p 1 varieties.

Methods: To obtain monoclonal and polyclonal antibodies, mice and rabbits, respectively, were immunized with recombinant Pru p 1.

Oral Health-related quality of life after coronectomy for impacted mandibular third molar in the first postoperative week.

Background: Coronectomy of a mandibular impacted third molar is a surgical treatment to minimize the risk for inferior alveolar nerve damage. We aimed to determine whether this procedure affected the oral health-related quality of life (OHRQoL) within the first postoperative week.

Material And Methods: This prospective study included 50 patients that underwent a coronectomy for an impacted mandibular third molar.

Physiology-Based Revascularization: A New Approach to Plan and Optimize Percutaneous Coronary Intervention.

Coronary physiological assessment using fractional flow reserve or nonhyperemic pressure ratios has become a standard of care for patients with coronary atherosclerotic disease. However, most evidence has focused on the pre-interventional use of physiological assessment to aid revascularization decision-making, whereas post-interventional physiological assessment has not been well established. Although evidence for supporting the role of post-interventional physiological assessment to optimize immediate revascularization results and long-term prognosis has been reported, a more thorough understanding of these data is crucial in incorporating post-interventional physiological assessment into daily practice.