Orphanet J Rare Dis
January 2025
Background: Clinical research has offered many definitions and fragmented perspectives of joint morbidity in haemophilia. As joint damage, pain and mobility impairment can be present without clinical record of persistent bleeding, a person-centric joint morbidity characterisation remained a priority for the haemophilia community, giving rise to the 'problem joint' concept. As diagnosing and managing joint morbidity is critical, the aim of this study was to analyse the holistic burden of problem joints in people with moderate or severe haemophilia A (HA).
View Article and Find Full Text PDFHematol Oncol Clin North Am
December 2024
Heavy ion radiotherapy is an emerging technology for treating radioresistant solid tumors. Unlike current low-linear energy transfer techniques, heavy ion radiotherapy, such as carbon ion radiotherapy, enhances the biologic effects related to cancer therapy. Prospective clinical evidence has demonstrated feasibility and efficacy in several disease sites, including head and neck, thoracic, central nervous system, gastrointestinal, pelvic tumors, and sarcomas.
View Article and Find Full Text PDFIntroduction: 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.
Significance: Selecting a nerve-specific lead fluorescent agent for translation in fluorescence-guided surgery is time-consuming and expensive. Preclinical fluorescent agent studies rely primarily on animal models, which are a critical component of preclinical testing, but these models may not predict fluorophore performance in human tissues.
Aim: The primary aim of this study was to evaluate and compare two preclinical models to test tissue-specific fluorophores based on discarded human tissues.