Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews.

Purpose: Hemophilia A is a rare bleeding disorder that leads to recurrent hemarthrosis, which can ultimately result in reduced mobility and poor quality of life. Qualitative exit interviews provide insights into patient perspectives and support the interpretation of quantitative trial data, such as patient-reported outcome measures. In the Phase 3 XTEND-1 study (NCT04161495) of efanesoctocog alfa in participants with severe hemophilia A, exit interviews were conducted to understand pre- and post-study experiences with pain and physical functioning and to evaluate participants' treatment experiences.

Comprehensive genomic filtering algorithm to expose the cause of skewed X chromosome inactivation. The proof of concept in female haemophilia expression.

Background: Exploring the expression of X linked disorders like haemophilia A (HA) in females involves understanding the balance achieved through X chromosome inactivation (XCI). Skewed XCI (SXCI) may be involved in symptomatic HA carriers. We aimed to develop an approach for dissecting the specific cause of SXCI and verify its value in HA.

Evidence-based management of adult traumatic brain injury with raised intracranial pressure in intensive critical care unit at resource-limited settings: a literature review.

Background: In underdeveloped countries, there is a greater incidence of mortality and morbidity arising from trauma, with traumatic brain injury (TBI) accounting for 50% of all trauma-related deaths. The occurrence of elevated intracranial pressure (ICP), which is a common pathophysiological phenomenon in cases of TBI, acts as a contributing factor to unfavorable outcomes. The aim of this systematic review is to analyze the existing literature regarding the management of adult TBI with raised ICP in an intensive critical care unit, despite limited resources.

The effect of preoperative anemia on perioperative outcomes among patients undergoing emergency surgery: A multicenter prospective cohort study.

Introduction: Preoperative anemia is a common finding among surgical patients. It is associated with an increased risk of perioperative morbidity and mortality. Outcomes among emergency surgical patients are not established.

The hidden joint in children with haemophilia on prophylaxis.

Unlabelled: Prophylaxis is the gold standard treatment for children with haemophilia (CWH). MRI studies revealed joint damage, even with this treatment; this suggests the presence of subclinical bleeding. In the case of children with haemophilia, it is relevant to detect early signs of joint damage, as this allows the medical team to provide the appropriate treatment and follow-up, in order to avoid arthropathy development and its consequences.