Effects of the factor V G1691A mutation and the factor II G20210A variant on the clinical expression of severe hemophilia A in children--results of a multicenter studys.

The present multicenter cohort study of 107 pediatric PUPs was performed to determine whether the concomitant inheritance of the factor (F) V G1691A or the F II G20210A mutation influences the clinical expression of severe hemophilia A (HA). Carriers of the FV and FII mutations had a significantly lower annual bleeding frequency (ABF) than non-carriers (p=0.012). Joint damage (Pettersson score) was significantly less severe in patients with thrombophilia (p=0.022). A protective effect of thrombophilic risk factors was shown for ABF (OR [CIs]: 0.7[0.5-0.9]; p=0.02) and the severity of the hemophilic arthropathy (OR [CIs]: 0.06[0.01-0.3]; p=0.0009).

Long-term safety and efficacy data on childhood venous thrombosis treated with a low molecular weight heparin: an open-label pilot study of once-daily versus twice-daily enoxaparin administration.

In this open label pilot safety study 80 children over 3 months old with deep venous thrombosis were treated with enoxaparin with a target 4 h anti-factor Xa activity between 0.5-0.8 IU/mL. The children were stratified to receive once daily or twice daily doses. The study end-points were post-thrombotic syndrome, re-thrombosis, bleeding, and therapy-related death. The median duration of treatment was 5 months and the median follow-up was 24 months. No significant differences were found between the two groups of patients. No bleeding or therapy-related deaths occurred. These safety and efficacy data may serve as a basis to initiate an international multicenter study on enoxaparin treatment.