Haemophilia : the official journal of the World Federation of Hemophilia
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Standard treatment of haemophilia A is based on replacing the missing coagulation factor VIII (FVIII) to treat and prevent bleeding episodes. The most challenging complication of FVIII therapy is the development of neutralizing antibodies (inhibitors) that can render treatment ineffective. Eradication of the inhibitor through immune tolerance induction (ITI) remains the most effective strategy for managing these patients. ⋯ As a consequence the United Kingdom Haemophilia Centres Doctors' Organisation (UKHCDO) issued the first guidance on the treatment of bleeding episodes in patients receiving emicizumab. To build on and extend this work, a panel of German haemophilia specialists met to discuss the UK guidance, review current evidence and provide additional guidance for German healthcare professionals on how to optimize the management of patients with haemophilia A receiving emicizumab. Recommendations are provided on the use of bypassing and other agents to manage breakthrough bleeding, ITI in the emicizumab era, haemostatic support during surgery and issues relating to laboratory monitoring.
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The treatment options for the haemostatic disorders, haemophilia A and haemophilia B, have progressed rapidly over the last decade. The introduction of extended half-life recombinant factor VIII (FVIII) and factor IX (FIX) concentrates to replace these missing clotting factors highlighted discordance between one-stage activated partial thromboplastin time (APTT)-based clotting factor assays and chromogenic factor assays with some products. This raised awareness of the importance of investigation of potential reagent or assay differences by pharmaceutical companies. ⋯ Chromogenic assays containing human factor IXa (FIXa) and factor X (FX) are sensitive to the presence of emicizumab but those containing bovine FIXa and FX are unaffected. Many haemostasis laboratories have been required to evaluate new assays to enable accurate monitoring of emicizumab in patient plasma. A number of gene therapy approaches have been trialled in both haemophilia A and haemophilia B but there are scant data published on the measurement of FVIII and FIX in these patients and whether there are discrepancies between reagents or assay methodologies.
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Meta Analysis
Mental health disorders in haemophilia: Systematic literature review and meta-analysis.
Despite significant advances in morbidity and mortality outcomes, quality of life for people with haemophilia (PWH) remains compromised. Underrecognized and undertreated mental health disorders decrease quality of life; however, reports are inconsistent regarding the true prevalence of mental health disorders in PWH. ⋯ Overall, our meta-analysis suggests that the prevalence of depression, anxiety and ADHD across decades is significantly increased in PWH compared to the general population.
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Due to the global prevalence of oral disease, tooth extraction is the most common surgical procedure required in general population thus likely to be similarly common in patients with haemophilia, especially those in older age and those living in countries with restricted resources. There are little or no consensus about optimal level and duration of factor replacement (FRP) therapy required to prevent bleeding complication following surgery and low levels of evidence to inform protocols and guidelines. The goal of this article was to review the literature regarding haematological treatment protocols and to assess their effectiveness in prevention of bleeding complications during and after tooth extractions in people with haemophilia. ⋯ Based on the haematological regimens, all studies were classified into one of three groups: pre- and postoperative FRP or DDAVP, single preoperative FRP or DDAVP, and no FRP treatment. The overall reported bleeding rate in case of both pre- and postoperative FRP and single dose FRP preoperative is similar, 11.9% and 11.4%, respectively, indicating that minimizing the use of clotting factor concentrate is possible if proper local haemostatic measures are provided. Strictly designed prospective studies with higher number of patients are necessary to get firm conclusions about optimal FRP treatment required to prevent bleeding complications during and after oral surgery in patients with haemophilia.
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In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry representatives, were sought in the identification of the key issues we expect to face. ⋯ These were followed by small group work. This manuscript outlines the key issues identified and the path forward.