Abstract
Bone marrow transplantation (BMT) is nowadays used in various hematological disorders including leukemias. Hemophilia A is sex linked bleeding disorder in which there are various genetic abnormalities in factor VIII gene. Among various hematological disorders, bleeding disorders mainly hemophilia in now widely treated using plasma derived and recombinant factor VIII concentrates. Day to day transfusion of coagulation factor VIII in current lifelong disorder is a burden for the patients and decreases their quality of life. Although infection of coagulation products with blood-borne viruses that was major concern in treatment of hemophilia has been eliminated nearly complete at the present time, but appearance of factor VIII inhibitor is huge burden in treatment of hemophilia for both clinicians and patients. In this day and age BMT has gathered new insight and acceptable treatment for many hematological and non-hematological disorders. If BMT will be successful in treatment of hemophilia, it will improve quality of life (by nearly permanent treatment without necessary of daily treatment) and decrease mortality rate (by providing continuous coagulation factor level in blood system).
Highlights
Hemophilia A is X-linked bleeding disorder that mainly affects men
The current hemostasis defect originated from a deficiency or functional defect in factor VIII protein in circulation that terminated to inability to form blood clot and hemorrhage [2]
The burden resulted of current treatment is the appearance of factor VIII inhibitor in plasma of the patients that makes the control process of hemorrhagic episodes unfruitful and challenging [2]
Summary
Hemophilia A is X-linked bleeding disorder that mainly affects men. It affects 1 in 5000 males [1]. Occurrence of vast genetic abnormalities from single point mutation to complete deletion of factor VIII gene have been reported in hemophilia A [3]. These genetic abnormalities reflect in plasma level of factor VIII in affected patients. The burden resulted of current treatment is the appearance of factor VIII inhibitor in plasma of the patients that makes the control process of hemorrhagic episodes unfruitful and challenging [2]. New insights and attractions have been absorbed to novel therapeutic options including gene therapy and BMT to overcoming current problem using manipulation of hematopoietic stem cells [11]. We will review contributions on efforts of bone marrow transplantation in treatment of hemophilia and will pay attention to role of BMT in treatment of hemophilia at present time
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