Modified Primary Prophylaxis in Previously Untreated Patients With Severe Hemophilia A in Iran.

BACKGROUND: Recently the low-dose tailoring method of primary prophylaxis has been introduced for previously untreated patients with hemophilia A. OBJECTIVE: To evaluate the efficacy and safety of low-dose tailoring method of primary prophylaxis in previously untreated patients with severe hemophilia A. MATERIALS AND METHODS: In this pre-post interventional study, 33 patients with severe hemophilia A who were previously untreated and affiliated to universities in the capital city and southern Iran were evaluated during 2014 to 2015. Modified primary continuous prophylaxis was used for patients with age below 3 years old, after first and before the second episodes of obvious clinical bleeding in large joints or large soft tissue hematoma or large amount of bleeding. Prophylaxis was started by 25 IU/kg once per week and increased to twice or 3 times a week according to defined bleeding events. RESULTS: The median age at diagnosis and age of starting prophylaxis were: 4 months (at birth, 22 mo) and 12 months (1 to 35 mo), respectively. Mean annual bleeding rate of patients after prophylaxis was 1.08±2.21 episodes per year. None of the patients had inhibitors before therapy. Inhibitor was present in 5 patients (15.1%) after prophylaxis. CONCLUSIONS: It seems that modified primary continuous prophylaxis has been successful in reducing bleeding episodes in children with severe hemophilia A and in the texture of lower cost it can improve quality of life of these patients, especially in developing countries where financial resources are limited.

Iranian Low-dose Escalating Prophylaxis Regimen in Children with Severe Hemophilia A and B.

Establishing an appropriate prophylaxis regimen for children with hemophilia is a critical challenge in developing countries. Barriers including availability and affordability, catheter-related complications, and inhibitor development risks have led to the introduction of new tailored prophylaxis regimens in different countries. This study emphasizes on the benefits of the Iranian low-dose escalating prophylaxis regimen in a Hemophilia Comprehensive Care Center in Iran. Referred patients with hemophilia less than 15 years of age, who were subject to prophylaxis regimen, are studied retrospectively. A once-weekly prophylaxis regimen of 25 IU/kg was started for the patients primarily. Their prophylaxis regimen was changed to 25 IU/kg twice a week and then 3 times a week when they experienced 3 joint bleedings, 4 soft tissue bleedings, or a 1 life-threatening bleed without a specific trauma history. Overall, 25 patients with severe hemophilia and at least 6-month history of on-demand (OD) treatment were studied. A mean of 1754 IU/kg/yr of coagulation factors, used for OD and prophylaxis purposes, was sufficient to decrease the mean annual bleeding rate (ABR) to 1.86 after prophylaxis. It also reduced the mean hospitalization days and the mean number of target joints to 0.24 and 0.16, respectively. Overall, 19 (76%) patients were continuing their once-weekly regimen at the end of the follow-up. None of the patients needed 3-times-a-week regimen or central venous catheterization and none developed inhibitors in the follow-up. Benefits of the Iranian low-dose escalating prophylaxis regimen prove equal to some of the previous 3-times-a-week prophylaxis regimens in reducing the ABR and hospitalizations.

Cost-Effectiveness Analysis of Biogeneric Recombinant Activated Factor VII (AryoSeven™) and Activated Prothrombin Complex Concentrates (FEIBA™) to Treat Hemophilia A Patients with Inhibitors in Iran.

Nowadays, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrates (aPCC) are used to treat bleeding episodes in the Hemophilia patients with inhibitors. AryoSeven® is an Iranian biogeneric rFVIIa with homogeneity of efficacy and the nature to NovoSeven in a comparative trial. The current clinical trial aimed to evaluate the cost-effectiveness of FEIBA and AryoSeven® by Decision Analytic Model according to the Iranian healthcare system. An open label, multi-center, cross-over clinical trial was designed. Patients were categorized into 3 groups based on their prior tendency to one or none of the products. To determine the premium therapeutic strategy, the Incremental cost-effectiveness ratio (ICER) was calculated. Protocol F led to more treatment success in group F than the other groups (P= 0.03). Also, there was a significant statistical difference between the mean of effectiveness scores in the groups using protocol F (P = 0.01). The effectiveness of protocol F and A were 89% and 72%, respectively. ICER cost US$ to manage an episode of bleeding to get one more unit of effectiveness using FEIBA VS. AryoSeven. Although the results showed that AryoSeven was more cost-effective compared to FEIBA, the two strategies were undominated. In other words, both medicines can be applied in the first line of the treatment if the cost of FEIBA was reduced. The present clinical trial was registered at IRCT website, under ID No.2013020612380N1.