Ara h 2-expressing cucumber mosaic virus-like particle (VLP Peanut) induces in vitro tolerogenic cellular responses in peanut-allergic individuals.

Background: Peanut allergy (PA) is one of the most prevalent food allergies with a lack of favorable safety/efficacy treatment. A cucumber mosaic virus-like particle expressing peanut allergen component Ara h 2 (VLP Peanut) has been developed as a novel therapeutic approach for PA.

Objective: We assessed the tolerogenic properties and reactivity of VLP Peanut.

Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies.

Introduction: People with haemophilia A (PwHA) experience acute and chronic pain associated with reduced quality of life (QoL).

Aims: This post hoc analysis of pooled data from the HAVEN 1 (NCT02622321), 3 (NCT02847637), 4 (NCT03020160) and STASEY (NCT0319179) studies assessed the impact of emicizumab prophylaxis on pain-related QoL in PwHA.

Methods: PwHA received emicizumab during the four studies.

Potential of Japanese Macaques for Understanding Etiology and Seasonality of Repetitive Linear Enamel Hypoplasia in Nonhuman Primates.

Japanese macaques are ideal to advance understanding of a wide-spread pattern of recurrent developmental distress in great apes, preserved as repetitive linear enamel hypoplasia (rLEH). Not only are they numerous, unendangered, and well-studied, but they are distributed from warm-temperate evergreen habitats in southern Japan to cool-temperate habitats in the north, where they are adapted behaviorally and phenotypically to winter cold and seasonal undernutrition. We provide a pilot study to determine if enamel hypoplasia exists in Japanese macaques from the north and, if temporal patterns of enamel hypoplasia are consistent with seasonal cold, undernutrition and/or exposure to secondary plant compounds.

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease.

Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.

Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases.

Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE.

International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal.

Introduction: Evidence-based clinical practice guidelines drive optimal patient care and facilitate access to high-quality treatment. Creating guidelines for rare diseases such as haemophilia, where evidence does not often come from randomized controlled trials but from non-randomized and well-designed observational studies and real-world data, is challenging. The methodology used for assessing available evidence should consider this critical fact.