The Current Role of Bone Marrow Transplantation in the Treatment of Hemophilia A Hassan Mansouritorghabeh 1* and Ali Ghasemi 2 1 Allergy Research Center, Ghaem Hospital, School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran 2 Department of Pediatrics, School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran * Corresponding author: Hassan Mansouritorghabeh, Allergy Research Center, Ghaem Hospital, School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran, Tel: +98-511-8012827; Fax: +98-511-8422544; E-mail: Mansouritorghabeh@mums.ac.ir Received date: May 23, 2014, Accepted date: June 07, 2014, Published date: June 18, 2014 Copyright:© 2014 Mansouritorghabeh H, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. 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). The aim of current review was to determine current role of BMT in treatment of hemophilia and to evaluate current usage of BMT for treatment of hemophilia. The review of literatures showed that although BMT has been used in a rare cases with hemophilia (mainly hemophilia with another blood disorder), it does not use commonly in treatment of patients with hemophilia A at present time. Literature review showed that scientists now focused on bone marrow transplantation in animal model of hemophilia. Keywords: Hemophilia A; Treatment; Bone marrow transplantation Introduction Hemophilia A is X-linked bleeding disorder that mainly affects men. It affects 1 in 5000 males [1]. 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 finally hemorrhage [2]. 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 severity of hemophilia correlates with plasma level of factor VIII (severe form <1%, moderate 1-5% and mild form 5-30%) [4]. The spectrum of clinical manifestation varies from mild ecchymosis to fatal spontaneous intracranial hemorrhagic episodes [5]. The hallmark of severe type of hemophilia is occurrence of massive bleeding that happen spontaneously or after minor trauma [6]. The main stone of treatment is replacement therapy using various factor VIII containing products including cryoprecipitate, plasma derived factor VIII concentrate, recombinant factor VIII and other products such as recombinant activated factor VII (rFVIIa), prothrombin complex concentrate, anti-fibrinolytic agents (Desmopressin and ε-aminocaproic acid) and activated prothrombin complex concentrates [7,8]. Nowadays the common treatment option comprises infusion of plasma derived and recombinant factor VIII concentrates in many countries. 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]. The life-expectancy in patients with hemophilia who have complete access to modern treatments has been improved and they now encounter age related diseases such as cancers [9]. Bone marrow transplantation (BMT) is an ideal cure for some cancers, inborn errors of metabolism or immune system [10]. 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]. The accessibility and capacity of ex vivo manipulation of hematopoietic stem cells have made them as an attractive target for treatment of hemophilia [12] while a multidisciplinary team work needs to manage the condition [10]. Here 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. Common Treatment Options Cryoprecipitate The first preparation was described widely and introduced by Dr. Pool in 1965. It was prepared using precipitation method from plasma Mansouritorghabeh and Ghasemi, J Hematol Thrombo Dis 2014, 2:4 DOI: 10.4172/2329-8790.1000143 Review Article Open Access J Hematol Thrombo Dis ISSN:2329-8790 JHTD, an open access journal Volume 2 • Issue 4 • 1000143 J o u r n a l o f H e m a t o l o g y & T h r o m b o e m b o l i c D i s e a s e s ISSN: 2329-8790 Journal of Hematology & Thromboembolic Diseases