Introduction: Haemoassist TM 2 is an electronic system designed for people with bleeding disorders and their physicians to record prophylactic infusions and treatment of bleeds. It aims to improve adherence by permitting reminders and accuracy of documentation by facilitating real-time reporting. Aim: To assess documentation quality and adherence to prophylactic regimens in patients with haemophilia A, haemophilia B or von Willebrand disease who are using Haemoassist TM 2. Methods: Ten centres enrolled consecutive patients, who had been using Haemoassist TM 2 for ≥ 3 months (Cohort 1, 'quality of documentation'). Of these, patients who had a specified prophylactic regimen in Haemoassist TM 2 for ≥ 3 months were eligible for inclusion in Cohort 2 ('adherence to prophylaxis'). Results: Cohort 1 comprised 796 patients (71% with severe haemophilia A; median 20.5 months of Haemoassist TM 2 use). The most common method of documentation for patients was using the mobile app; the median time between infusion and documentation was 4 hours using the app, compared with 85 hours using a web portal on a stationery device. The median total annualised number of infusions was consistent in the first and last 3 months of documentation (128; IQR: 70-184 and 120; IQR 64-176, respectively). Cohort 2 comprised 202 patients (79% severe haemophilia A; median of 13 months on prophylactic regimen in Haemoassist TM 2). The rate of adherence to prophylaxis was 83%; median deviation between planned and actual infusion time was ± 2 hours. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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Objective: Activated prothrombin complex concentrate (aPCC) is a bypassing agent indicated to treat bleeds in patients with acquired hemophilia A (AHA). Nevertheless, its efficacy and safety in the real-world setting have not often been addressed. Methods:We report the experience of Spanish reference centers for coagulation disorders and from acquired hemophilia Spanish Registry (AHASR) from August 2012 to February 2021. Follow-up period of 30 days after aPCC withdrawal.Results: Thirty patients with a median age of 70 years old, suffering from 51 bleeds treated with aPCC were finally evaluated. As first-line treatment, aPCC stopped bleeding in 13 of 14 (92.9%) cases. aPCC as the second line after recombinant factor VIIa failure, stopped bleeding in all cases. In 17 patients, aPCC was used far from initial bleed control as prophylaxis of rebleeding with 94% effectiveness. No thromboembolic episodes were communicated. One patient developed hypofibrinogenemia,

Background: Immune tolerance therapy (ITT) is the only available therapy for eliminating the inhibitor in patients with haemophilia and high-responding inhibitors. However, it is a very expensive treatment with an efficacy of up to 60%-75%. Factors influencing the outcome of ITT are not completely understood. Some studies have suggested that plasma derived factor VIII (FVIII) concentrates might offer certain advantages in the elimination of the inhibitor when compared with recombinant derivatives. The presence of von Willebrand factor (Auerswald et al,Haematologica 2003;88:EREP05; Kreuz et al,Haematologica 2001;86:16-20) and some molecules with immunomodulatory effect like anti-idiotype antibodies, transforming growth factor-β, HLA type I molecules and soluble FAS ligand in plasma derived concentrates may be involved in such effect (Berntorp et al,Haemophilia 2001;7:109-13; Hodge et al,Br J Haematol 2001;115:376-81; Hodge et al, Haemophilia 2006;12:133-9; Ghio et al, Thromb Haemost 2003;89:365-73). The activated prothrombin complex concentrate (FEIBA®) is a plasma derived product used for bleeding control in patients with haemophilia A and high-responding inhibitors. We hypothesize that FEIBA® may have an immunomodulatory activity due to its plasmatic origin. This effect might be observed in patients with intense treatment with FEIBA® (e.g. prophylaxis treatment) by the reduction in levels of antibodies against FVIII compared with patients on demand treatment with this product. Objective: The aim of this study was to compare levels of inhibitors in patients with severe haemophilia A and inhibitors on demand or under prophylactic treatment with FEIBA®. Methods and results: This was a prospective, observational and multicenter study. A total of 12 adult patients with severe haemophilia A and high-responding inhibitors (6 patients on demand and 6 patients under prophylaxis treatment with FEIBA®) were included. All patients had normal levels of serum creatinine, serum alanine aminotransferase and CD4+ lymphocyte counts at inclusion. Levels of inhibitors were determined by Bethesda assay. Levels of inhibitors in patients on prophylaxis with FEIBA® were lower than those observed in patients on demand treatment but differences were not statistical significant. In most of the patients on prophylaxis, levels of inhibitors went down along the prophylaxis regimen. Table. Medical history of patients. Patient Nº Age Type of treatment Usual dose (IU/kg) Frecuency Titer (BU) Time frame with start of prophylaxis (months)** 480101 53 Prophylaxis 60 Three times a week 64* 4.4 80 4.1 96 1.3 250 -1.3 480102 58 Prophylaxis 55 Three times a week 5* 3.3 12 2.1 7 -3.8 12 -6.1 150101 UNK Prophylaxis 60 Three times a week 4* 3.0 5 -6.0 6 -8.0 280101 28 Prophylaxis 56 Every other day 109* 7.3 77 4.1 101 -8.3 101 -9.7 280102 57 On Demand 56 x 8 doses N/A 174* N/A 37 N/A 83 N/A 21 N/A 25 N/A 184 N/A 280103 57 On Demand 55 x 6 doses N/A 8000* N/A 64 N/A 5 N/A 21 N/A 280104 58 On Demand 68 x 6 doses N/A 152* N/A 280105 23 On Demand 68 x 3 doses 40 x 6 doses N/A 30* N/A 080101 28 On Demand 50 x 5 doses N/A 5.2* N/A 7.0 7.2 4.0 7.8 460101 37 Prophylaxis 66 every 48h Negative 21,0 460102 57 On demand 70 2 doses/ bleeding usually 66.5* More than 20 years 300001 50 Prophylaxis 85 Three times a week 8* 3 *Titer at inclusion. **Positive values represent number of months after starting prophylaxis and negative values are referred to number of months before starting prophylaxis. For example, the patient number 480101 had 250 BU at 1.3 months before starting prophylaxis (in the table= -1.3 months) and 64 UB at month 4.4 after initiation of prophylaxis (in the table= 4.4 months). N/A: not applicable. Conclusion: Prophylaxis with FEIBA® might decrease levels of inhibitors in patients with severe haemophilia A and high-responding inhibitors. However, a broader study is needed to confirm this result. Disclosures Iruin: Baxalta: Research Funding. Bonanad:Baxalta: Research Funding. López Fernández:Baxalta: Research Funding. Altisent:Baxalta: Research Funding. García Candel:Baxalta: Research Funding. Rivas Pollmar:Baxalta: Research Funding. Canales:Baxalta: Research Funding.