Despite the numerous and groundbreaking therapeutic advances made in the field of hemophilia over the past decades and particularly in recent years, hemophilia remains a disease that has a major impact on the daily lives of our patients, through the multiple complications and burdensome treatments it imposes. The disease burden is not only physical but also psychological and is difficult to evaluate solely by questionnaires and scores. In this article, we propose to examine the absence of psychological burden and of permanent thoughts about the disease and its complications in people with hemophilia as a new ambition that should guide hemophilia care and research in the future.

Background While the number of individuals with hemophilia who are expected to be or have already been included in gene therapy trials has been regularly reported, the number of unscreened or excluded individuals, in addition to the reasons for exclusion, is mostly not reported. Methods We conducted an eligibility assessment of all people with severe hemophilia for gene therapy trials in one large Belgian hemophilia treatment center based on patient selection criteria of gene therapy trials and patients’ profiling. Results Among 87 adult patients with severe hemophilia A and B, 11 aged ≥65 years and two women were excluded from analysis. Six patients were excluded because of inhibitor development. One patient exhibited active hepatitis C infection, one had insufficient exposure to factor VIII, and five had uncontrolled comorbidities, while two were enrolled in other trials and two abused alcohol. Overall, 43 patients were not screened owing to psychosocial factors. Among 14 patients accepting gene therapy, six had adeno‐associated virus type 5 neutralizing antibodies and one had liver fibrosis. The number of patients who would accept gene therapy in the absence of strict clinical trial requirements was estimated at 36 (41.4%), irrespective of any exclusion criteria. Conclusion The majority of individuals with severe hemophilia could not be enrolled in gene therapy trials, almost half of them because of partly modifiable psychosocial reasons (49.4%). The proportion of candidates should substantially increase in the future, as eligibility criteria are likely to change and as more data on long‐term efficacy and safety of gene therapy will become available.

Background Emicizumab, a bispecific monoclonal antibody administered subcutaneously, mimicking the action of activated coagulation factor VIII, has been approved in Europe for use in patients with severe hemophilia of all ages. Aims To assess availability, acceptance, adverse events, efficacy and laboratory monitoring of emicizumab and the effect of the coronavirus disease 2019 (COVID‐19) pandemic on its use. Methods Online questionnaire sent to 144 hemophilia treatment centres (November 2020 to January 2021). Results Forty‐six physicians from 21 countries responded, with a total of 3420 patients with severe HA under their care. Emicizumab was widely available, for 100% of inhibitor patients and 88% of non‐inhibitor patients. No major adverse events were reported. Four reported deaths in patients on emicizumab were not thought to be related to emicizumab. An annualized bleeding rate (ABR) of zero was achieved in 73% of inhibitors patients. Haemostasis was satisfactory in the majority of minor (93.7%) and major (90.7%) surgical procedures performed while on emicizumab. Inhibitor titers were monitored in 78.4% of inhibitor patients on emicizumab, but chromogenic FVIII assay was only available in 73% of centres. The COVID‐19 pandemic did not have a major impact on the adoption of emicizumab in most centres (64.9%). Conclusion Three years after its rollout in Europe, emicizumab is widely available. Clinical efficacy and safety were evaluated to be very good, keeping in mind the inherent limitations of the study. Unmet needs include establishment of treatment guidelines for surgery and breakthrough bleeding, limited expertise, especially in young children, and availability of laboratory assays.

Behçet’s disease is an inflammatory disease, the origin of which still remains unclear, and it has multiple manifestations, one of them being thrombosis. In this report, we describe the case of a 24-year-old Moroccan patient who presented with headache persisting for more than 2 weeks, which was found to be caused by cerebral venous sinus thrombosis. His medical history of recurrent oral and genital ulcerations, epididymitis and one episode of pericarditis led to the diagnosis of Behçet’s disease. We could observe an almost complete relief of symptoms with colchicine therapy, and anticoagulation with warfarin was started for secondary prevention of thrombosis.