Marc Trossaërt

Background The bleeding risk of patients with mild platelet function disorders is difficult to assess and their phenotype remains ill‐explored. Aim This study was designed to establish a comprehensive biological phenotype of patients with mild platelet function disorders. Methods Twenty patients were included with persistent abnormal light transmission aggregometry (LTA). The ISTH bleeding assessment tool (ISTH‐BAT) was assessed to identify laboratory analyses associated with an abnormal hemorrhagic score. Results The majority of patients had defects that might affect Gαi protein signaling pathways or minor abnormalities. No LTA nor flow cytometry parameters were associated with an above‐normal hemorrhagic score. However, prothrombin consumption, which corresponds to the ratio of serum residual factor II to plasma residual factor II, was significantly higher ( p = .006) in the abnormal ISTH‐BAT group (mean = 14%, SD = 6) compared with the normal ISTH‐BAT group (mean = 8%, SD 4). Prothrombin consumption was significantly associated with ISTH‐BAT score ( r = .5287, IC 95% 0.0986–0.7924, p = .0165). Conclusion In this group of patients, there was an association between a pathological bleeding score and increased prothrombin consumption. This test could be used as an additional indicator of platelet function abnormality liable to be related to bleeding risk.

Background Efmoroctocog alfa, the first recombinant factor VIII fusion protein with extended half‐life (rFVIII‐Fc), has been hypothesized to lower FVIII consumption in patients with severe Haemophilia A (pwSHA), without reducing clinical efficacy. What about real life? Method MOTHIF‐II was a noninterventional, multicentre, before/after study, via the collection of retrospective data from July 2015 to June 2016 (called T1), and from July 2017 to June 2018 (called T2), in 7 French haemophilia treatment centres. We examined the prescriptions and dispensations of factor VIII and the Annual Bleeding Rate (ABR), in pwSHA without current inhibitors on prophylaxis, before and after the introduction of rFVIII‐Fc. The data gathered from the BERHLINGO research database and from the French Healthcare claims database with a determinist pairing process based on the national unique identification number. Results A total of 156 pwSHA were included in the prescription cohort and 83 in the ABR cohort. For switched patients, the mean amounts of prescribed FVIII were significantly higher during T1 compared to T2 (4333 (2052) vs. 3921 (2029) IU/kg/year/patient, p : 0.028); a significant decrease in their ABR was also observed between T1 and T2 (6.3 (6.0) vs. 4.4 (5.4), p : 0.047). These patients had a more severe bleeding profile centred on haemarthrosis. Conclusion The results are related to those of the pivotal clinical trials for the reduction in FVIII consumption following the switch to rFVIII‐Fc, with a significant improvement in the haemorrhagic phenotype for pwSHA.