[The hopes and stakes of a public health reform. The example of New Caledonia].

At a time when many of us desire fundamental reform of our health system, we return to the case of the New Caledonian Do-Kamo project. The proposed model provides interesting elements of reflection, due to it being person-centered and favoring a cultural approach to disease.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.

Background: Decades of research have transformed hemophilia from severely limiting children's lives to a manageable disorder compatible with a full, active life, for many in high-income countries. The direction of future research will determine whether exciting developments truly advance health equity for all people with hemophilia (PWH). National Hemophilia Foundation (NHF) and American Thrombosis and Hemostasis Network conducted extensive inclusive all-stakeholder consultations to identify the priorities of people with inherited bleeding disorders and those who care for them.

Gene Therapy and Hemophilia: Where Do We Go from Here?

Gene therapy for hemophilia using adeno-associated virus (AAV) derived vectors can reduce or eliminate patients' disease-related complications and improve their quality of life. Broad implementation globally will lead to societal gains and foster health equity. Several vector products each for factor IX (FIX) or factor VIII (FVIII) deficiency are in advanced clinical development.

Screening for inattention, hyperactivity and impulsivity in children with haemophilia: A quality improvement intervention.

Introduction: Children with haemophilia have been reported with increased rates of inattention (IN) and hyperactivity/impulsivity (HI) and, therefore, are particularly vulnerable to poor social and academic outcomes.

Aim: To examine the benefit of utilizing a formal screening process for IN/HI in children with haemophilia during comprehensive clinic visits using a quality improvement approach.

Methods: At a single haemophilia treatment centre, screening for psychosocial issues was expanded and formalised to include (1) the Conners 3 Edition (Conners3) screening tool for IN/HI symptoms administered during the standard psychosocial assessment (SPA) by the social worker and school advocacy coordinator, (2) formal pathways to diagnosis and intervention as indicated including psychology consultation, psychological testing, or referral to community-based mental health professionals, and in-person advocacy assistance in the patient's community school.

Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.

Background: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.

Methods: In this multicenter, open-label, phase 1-2 trial, we assessed the safety and efficacy of varying doses of FLT180a in patients with severe or moderately severe hemophilia B (factor IX level, ≤2% of normal value). All the patients received glucocorticoids with or without tacrolimus for immunosuppression to decrease the risk of vector-related immune responses.