Development of a haemophilia A gene therapy shared decision-making tool for clinicians.

Introduction: As gene therapies are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation.

Aim: To inform development of a clinician SDM tool for haemophilia A gene therapy.

Methods: Clinicians at US Hemophilia Treatment Centers completed semi-structured interviews about their experience with SDM and provided feedback on a clinician SDM tool prototype.

Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.

Purpose: As gene therapies for hemophilia are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation. SDM tools may facilitate informed decision-making for gene therapy and other novel therapies.

Objective: To inform the development of SDM tools for hemophilia gene therapy.

Gene therapy preferences and informed decision-making: Results from a National Hemophilia Foundation Community Voices in research survey.

Introduction: To inform education and treatment discussions, it is important to understand how persons with haemophilia prefer to learn about and discuss new therapies and to identify variables that influence decision-making.

Aim: The aim of this study was to evaluate preferences and variables which influence decision-making related to gene therapy and other novel haemophilia therapies.

Methods: An online survey was sent to men with severe haemophilia enrolled in the National Hemophilia Foundation Community Voices in Research online platform for patient-powered research.

The odds and implications of coinheritance of hemophilia A and B.

We report 2 patients with coinheritance of the X-linked bleeding disorders hemophilia A and hemophilia B. We describe the family pedigrees, clinical features, and genotyping. The case report addresses the key clinical questions of how to manage patients with both hemophilia A and B and how to counsel families regarding recurrence risk.

Risk factors for cardiovascular disease in children and young adults with haemophilia.

Introduction: The origins of cardiovascular disease (CVD) begin in childhood. The primary objective of this cross-sectional cohort study was to determine the prevalence of cardiovascular risk factors in patients with congenital haemophilia A or B followed at Rady Children's Hospital San Diego Hemophilia and Thrombosis Treatment Center (HTC). We hypothesized that cardiovascular risk factors could be identified as part of a comprehensive clinic visit.