SAT0048 Analysis of different therapeutic regimes in patients with hemophilic arthropathy
Authors: L. Mendez Diaz, C. aguilera cros, M.D. Arcila Duran, A. Ruiz Roman, N. Garrido Puñal, M. Lisbona Muñoz, R.J. Nuñez Vazquez, J. Povedano Gomez
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> The greater morbidity of the patient with haemophilia is due to hemarthrosis. The treatment is based on the administration of deficient coagulation factor (FC). The treatment is divided into prophylactic (TP) and on demand (AD). Prophylaxis consists in the administration of FC in order to maintain adequate levels of factor to prevent or reduce spontaneous bleeding and AD is the application of the factor when there is clinical evidence of bleeding. The TP is the recommended treatment in severe haemophilia, plasma and recombinant concentrates are used, safe and effective, but with a short half-life, which requires frequent intravenous infusions, being a barrier to compliance. Another drawback of the current treatment in haemophilia A (HA) is that up to 30% develop inhibitor (antibodies that neutralise the activity of a CF). New subcutaneous drugs (NSD) have begun to be used in clinical trials, such as: Emicizumab: Bispecific anti–IXa/X monoclonal antibody. Concizumab: Anti–TFPI antibody. This new therapeutic strategy can have implications both from a clinical and economic point of view. <h3>Objectives</h3> To analyse the different treatment regimens and their economic implications in a cohort of patients with hemophilic arthropathy (HA). Evolution of hemarthroses in patients with AH after starting treatment with NSD in the clinical trial phase. <h3>Methods</h3> Retrospective descriptive study, in the Haemophilia Unit of our hospital(regional reference), in patients with AH (Haemophilia A and moderate-severe B), followed in consultation with episodes of joint bleeding, from January 2007 to October 2017. Gravity of the haemophilia determined by the percentage of FC activity (VIII and IX), moderate from 1% to 5%, severe &lt;1%. The number of joint bleeds was analysed 6 months before and after the start of treatment with the NFS in patients who have participated in a multicenter phase III study and continue with the treatment <h3>Results</h3> We included 89 patients (88 men and 1 carrier woman), mean age 31±17 years, HA (severe 56%, moderate 26%), HB (severe 15%, moderate 1%). 17% develop inhibitor (10 severe HA, 3 moderate HA, 3 severe HB). Treatment on demand 44% and prophylaxis 38%. Replacement therapy: FC VIII (44% severe HA, 22% moderate HA), FC IX (11% severe HB, 1% moderate HB), recombinant active factor VII (rFVIIa) (2 patients with severe HB with inhibitor). In treatment with monoclonal antibody (patients currently in Clinical Trial): 15% Emicizumab, of these 4 are with inhibitor (2 severe HA, 1 moderate HA, 1 severe HB and 1% Concizumab (severe 1HB with inhibitor). The number of hemarthros was analysed 1 year before the administration of NFS until now, achieving a statistically significant reduction in the rate of joint bleeding of 86% (p&lt;0.005) compared to conventional pre-treatment. <h3>Conclusions</h3> The majority of patients with severe and moderate haemophilia are on demand treatment, despite existing recommendations. It can also be said that there are drugs in the research phase that may involve a paradigm shift in the therapeutic approach of patients with haemophilia, which will allow more personalised treatment, to achieve better joint protection and a better quality of life. <h3>Disclosure of Interest</h3> None declared