EQOFIX: a combined economic and quality-of-life study of hemophilia B treatments in France.

International audienceEQOFIX is a medicoeconomic study that analyzed the health-related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma-derived Factor IX (pdFIX) or recombinant FIX (rFIX).The primary objectives were evaluations of the impact of hemophilia B on HRQoL and of the costs associated with its management. The secondary objectives were evaluations of the clinical efficacy and costs of care of pdFIX and rFIX. In this observational study we included and followed for 1 year severe and moderate hemophilia B patients without inhibitor. HRQoL was evaluated through generic and disease-specific questionnaires. Information on the health resources consumed was collected every 3 months.The EQOFIX cohort was composed of 155 patients, including 51 children and 104 adults, with 114 having severe disease and 41 having moderate disease. The regimens were prophylactic for 61 and on demand for 94. Altogether, 78 were treated with rFIX and 77 with pdFIX. There was no difference in the QoL between the pdFIX and rFIX treatments. The extra cost of prophylaxis was €22,605 per bleeding event prevented. The consumption of FIX was 1.4-fold higher for the patients treated with rFIX than for the patients treated with pdFIX.Our findings in a cohort composed of 25% of the French population of moderate and severe hemophilia B patients show, with similar clinical and HRQoL results, that treatment with rFIX is more expensive than treatment with pdFIX

Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A.

International audienceSix recombinant factor VIII (rFVIII) products have been marketed worldwide. In 2013, the Research of Determinants of Inhibitor Development (RODIN) study group reported an unexpectedly high risk of inhibitor development with a second-generation full-length rFVIII (Product D) in previously untreated patients (PUPs) with severe hemophilia A (HA). In 1994, French public health authorities established a prospective cohort to monitor hemophilia treatment safety. A PUP subgroup was designed to investigate inhibitor risk factors. We analyzed this subcohort in view of the RODIN findings. After excluding 50 patients who participated in the RODIN study, the primary analysis focused on 303 boys with severe HA first treated with a rFVIII product. A clinically significant inhibitor was detected in 114 boys (37.6%). The inhibitor incidence was higher with Product D vs the most widely used rFVIII product (adjusted hazard ratio [aHR], 1.55; 95% confidence interval [CI], 0.97-2.49). Similar results were found for high-titer inhibitors and in 10 sensitivity analyses. No heterogeneity was observed between RODIN and our results. Combined aHRs were 1.58 (95% CI, 1.17-2.14) for all inhibitors and 1.70 (95% CI, 1.15-2.52) for high-titer inhibitors. Our results confirm the higher immunogenicity of Product D vs other rFVIII products in PUPs with severe HA

Obstetrical complications in hereditary fibrinogen disorders: the Fibrinogest Study.

BACKGROUND Women with hereditary fibrinogen disorders (HFDs) seem to be at increased risk of adverse obstetrical outcomes, but epidemiologic data are limited Patients/methods: We conducted a retrospective and prospective international study to determine the prevalence of pregnancy complications, the modalities and management of delivery, and the postpartum events. RESULTS A total of 425 pregnancies were investigated from 159 women (49 hypofibrinogenemia, 95 dysfibrinogenemia, 15 hypodysfibrinogenemia). Overall, only 55 (12.9%) pregnancies resulted in an early miscarriage, 3 (0.7%) in a late miscarriage and 4 (0.9%) in an intrauterine fetal death. Prevalence of live birth was similar among the types of HFD (p=0.31). Obstetrical complications were observed in 54 (17.3%) of live birth pregnancies, including vaginal bleeding (14, 4.4%), retroplacental hematoma (13, 4.1%), and thrombosis (4, 1.3%). Most 56deliveries were spontaneous (218, 74.1%) with a vaginal non-instrumental delivery (195, 63.3%). A neuraxial anesthesia was performed in 116 (40.4%) pregnancies, while 71 (16.6%) and 129 (44.9%) were under general or no anesthesia, respectively. A fibrinogen infusion was administered in 28 (8.9%) deliveries. Postpartum hemorrhages were observed in 62 (19.9%) of pregnancies. Postpartum venous thrombotic events occurred in 5 (1.6%) pregnancies. Women with hypofibrinogenemia were more at risk of bleeding during the pregnancy (p=0.04). CONCLUSIONS Compared to European epidemiologic data, we did not observe a greater frequency of miscarriage while retroplacental hematoma, postpartum hemorrhage and thrombosis were more frequent. Delivery was often performed without locoregional anesthesia. Our findings highlight the urgent need for guidance on management of pregnancy in HFDs

Age at diagnosis is delayed in women/girls with haemophilia compared to men/boys: a FranceCoag report

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