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.