Symptomatic haemophilia A: management during pregnancy and the postpartum period

As an X linked disorder, the presence of severe symptomatic haemophilia A is an extremely rare disorder in women. Therefore there are no high-level evidence-based guidelines when it comes to pregnancy. Although there have been advances in the fields of prenatal counselling and maternal-fetal care, the management of these gestations continues to embody a challenge for any medical team. We report the successful management of a pregnant woman with symptomatic haemophilia A, from pregnancy to the postpartum period. Our aim is to enhance knowledge on this topic, and further improve outcomes for these mothers and their offspring.

Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study

Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data are scarce. Here, we report real-world data from a global cohort. Participants were men ≥18 years old with severe HA (FVIII ≤ 1 IU/dL) receiving regular prophylaxis with FVIII. Participants provided 6 months of retrospective data and were prospectively followed for up to 12 months. Annualised bleeding rate (ABR) and FVIII utilisation and infusion rates were calculated. Differences between geographic regions were explored. Of 294 enrolled participants, 225 (76.5%) completed ≥6 months of prospective follow-up. Pre-baseline and on-study, the median (range) ABR values for treated bleeds were 2.00 (0–86.0) and 1.85 (0–37.8), respectively; the median (range) annualised FVIII utilisation rates were 3629.0 (1008.5–13541.7) and 3708.0 (1311.0–14633.4) IU/kg/year, respectively; and the median (range) annualised FVIII infusion rates were 120.0 (52.0–364.0) and 122.4 (38.0–363.8) infusions/year, respectively. The median (range) Haemo-QoL-A Total Score was 76.3 (9.4–100.0) (n = 289), ranging from 85.1 in Australia to 67.7 in South America. Physical Functioning was the most impacted Haemo-QoL-A domain in 4/6 geographic regions. Despite differences among sites, participants reported bleeding requiring treatment and impaired physical functioning. These real-world data illustrate shortcomings associated with FVIII prophylaxis for this global cohort of individuals with severe HA.

EFFICACY AND SAFETY OF MOROCTOCOG ALFA IN AN OPEN, MULTICENTER, PROSPECTIVE, CLINICAL STUDY IN CHILDREN 2 TO 6 YEARS OF AGE WITH SEVERE HEMOPHILIA A

The purpose of the study was to assess the efficacy, safety and pharmacokinetics of the moroctocog alfa (Octofactor) in children aged 2-6 with haemophilia A. Materials and methods of research : six patients between 2 and 6 years of age (average age 4.3±0.8 years) were included in the open multicenter prospective clinical trial. The efficacy of the drug was assessed against the background of the introduction of 30±10 IU/kg every 2–3 days, the safety was assessed by the frequency and causality of adverse reactions. Results: 7 post-traumatic bleeding was registered. The average prophylactic dose of the drug is 37.84±7.13 IU/kg. The dose of the drug for stopping bleeding was 1000 IU. 2 adverse events have been reported that are not related to moroсtocog alfa. Conclusion: the obtained data indicate the efficacy and safety of moroсtocog alfa in the study group of patients.