Adoption of emicizumab (Hemlibra®) for hemophilia A in Europe: Data from the 2020 European Association for Haemophilia and Allied Disorders survey.

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.

Real-life experience in switching to new extended half-life products at European haemophilia centres.

The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half-life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real-life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half-life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half-life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.

Scope of practice of haemophilia physiotherapists: A European survey.

INTRODUCTION: European guidelines on the care of haemophilia recommend ready access to a range of services provided by a multidisciplinary team of specialists including physiotherapy. However, the scope of physiotherapy provided is unknown. METHODS: The Physiotherapists Committee of the European Association for Haemophilia and Allied Disorders (EAHAD) conducted a web-based survey to quantify the role and scope of practice of physiotherapists involved in haemophilia care. The survey was sent to more than 200 physiotherapists registered on the EAHAD database. Questions concerned their work practices including assessment and treatment activities and level of autonomy. RESULTS: Eighty physiotherapists from twenty-four European countries responded. Considerable heterogeneity exists in roles, responsibilities, and clinical practice of physiotherapists, particularly in access to and type of physiotherapy treatment provided, as well as the skill set and autonomy of physiotherapists to make independent assessment and treatment decisions. DISCUSSION: This pan-European survey establishes a context to support physiotherapy role development and professional identity. Key recommendations include the following: (a) establishing a pan-European network to support collaboration and education for physiotherapists working in haemophilia, (b) developing a core skills and capability framework to ensure person-centred approaches are central as well as working in partnership with those with the condition to maximize early recovery, support self-management and enablement in remaining active and independent, (c) regular training, standardized validation and maintenance of competency for assessment tools, (d) well-designed randomized clinical studies with larger numbers of participants from multiple sites should be the focus of future research.