Search

Background: Efmoroctocog alfa (rFVIIIFc) is an extended half‐life FVIII used notably in surgery for patients with haemophilia A. More information is needed of its usage in real‐life. Methods: Adult patients with HA followed at the Lyon Comprehensive Hemophilia Care Center who underwent a surgery with rFVIIIFc were included in this retrospective analysis. The pharmacokinetics of rFVIIIFc was assessed by plasma factor VIII clotting activity (FVIII:C) using both one‐stage (OSA) and chromogenic substrate (CSA) assays. Results: A total of 39 major and 31 minor surgeries were performed in 49 patients treated with rFVIIIFc. The median dose of rFVIIIFc infused before major and minor surgeries respectively was 67.5 ((interquartile range [IQR] 52.6‐76.9) and 48.0 (38.5‐51.8) IU/kg. For major surgeries, during the first postoperative week, the median residual FVIII:C was 78 (64.5‐101.5) IU/dL with OSA and 99 (71‐118) IU/dL with CSA (p <.0001). After surgery, rFVIIIFc doses were adjusted according to CSA results. This led to a significant decrease of rFVIIIFc consumption compared to what would have been proposed according to the OSA assay, without unusual bleeding or appearance of inhibitor. Considering the high price of the molecule, this was also associated with a significant cost reduction. Conclusion: Dose adjustment of rFVIIIFc according to FVIII: C measured by CSA is effective, safe and well tolerated in patients with haemophilia A undergoing invasive surgery. [ABSTRACT FROM AUTHOR]

This paper presents a practical and efficient workflow for deformation monitoring of transport infrastructure. We propose using commercially available mobile laser scanning (MLS) systems to scan civil infrastructure while driving by in a car or rail vehicle. Our processing pipeline corrects for MLS-specific systematic deviations and models deformations from point clouds of two epochs. Following the concept of rigorous deformation analysis, we statistically test the deformations for significance. The required point cloud uncertainty may be obtained in two ways. First option is empirically by multiple passes and, secondly, by prediction with a learned stochastic model. We apply the method to three retaining structures and evaluate results based on ground truth geodetic surveys. The deviations did not exceed 10 mm, even for complex object surfaces or when traveling at 80 km/h. We demonstrate that the method is capable of revealing displacements in the centimeter range without relying on any installations on the structure. The approach shows great potential as a novel, efficient tool for detecting and quantifying defective structures in a road and railway network. [ABSTRACT FROM AUTHOR]

Introduction: In patients with haemophilia, repeated bleeding in large joints leads to chronic haemophilic arthropathy, a rare disease that can be managed surgically with ankle arthrodesis or with total ankle replacement (TAR). TAR has been reported to provide good surgical results in the medium/long‐term and allow preservation of joint mobility but the medical therapeutic management of the patients has not been described. Aim: To describe the medical therapeutic management of TAR. Methods: All patients with haemophilia A/B, with haemophilic ankle arthropathy, and who underwent TAR between April 2006 and October 2019 were retrospectively included. Factor consumption, perioperative and early complications, volume of blood lost, and orthopaedic data were collected. Results: A total of 25 patients underwent 29 TAR (mean age was 44.7 years [range: 26–65]). In the 17 patients with HA without history of anti‐FVIII inhibitor, the mean ± SD consumption the day of surgery was 116 ± 16 UI/kg when clotting factors were administered by continuous infusion, 106 ± 13 UI/kg when SHL factors were administered by bolus infusion, and 75 ± 22 UI/kg when EHL factors were administered by bolus infusion. During hospitalisation, the mean factor cost was €38,073 (83.7% of the total cost of surgery). Mean blood loss was significantly lower in patients treated with tranexamic acid (164 mL, range: 40–300) than in those not (300 mL, range: 70–800; p =.01). Six patients had haematoma. The 10‐year survival free of any prosthesis removal/arthrodesis was estimated to be 92.2% (95% CI [83; 100]). Conclusion: The medical therapeutic management of TAR is complex, carried out by a multidisciplinary team but effective in avoiding the occurrence of complications. [ABSTRACT FROM AUTHOR]

Introduction and aim: A national survey was initiated by representatives of French patients with haemophilia (AFH) and the French reference centre for haemophilia, in order to appreciate the awareness and knowledge of these patients regarding haemophilia gene therapy (HGT) and understand better their position about this innovative treatment that will soon become available. Results: Of 143 answers received, 137 could be analysed, representing about 3.5% of patients with severe or moderate haemophilia over 16year‐old. They were 80.3% with haemophilia A and 19.7 % with haemophilia B, with a severe form of the disease for 80.3 % of them. Curiosity for HGT was formulated by 64.2% of the participants, 33.6 % being interested by this approach as soon as it will be available and 38.7 % preferring to wait until more patients have been treated. Only 3.6 % of the participants would never consider receiving HGT. The level of awareness and knowledge was estimated to be limited by 39.5 % of the patients. More than 60 % of them declared having never or almost never discussed HGT with the team of their haemophilia centre. Before deciding to get HGT, 54.4 % of the participants considered that it will be very important to compare it with their current treatment and 53.7 % would like to be better informed by their care providers. Conclusions: These results highlight the need for training and education for patients, but also for professionals at haemophilia centres, about HGT and the shared decision‐making process. Objective, unbiased and transparent information must be available for patients about this very promising therapy which nonetheless carries more uncertainty and unknowns compared to other haemophilia treatments. [ABSTRACT FROM AUTHOR]