Current products used to replace FVIII or FIX are effective and safe. Nevertheless, gene therapy offers these patients the possibility of achieving a sustained correction PKC412 cost of the coagulation defect for their lifetime. Hemophilia has been considered one of the best candidates for a variety of novel gene therapies due to four main factors. First, it is a monogenic disease involving a single protein.

Second, small increments of clotting factor levels (2–3%) have shown to have a substantial reduction in the clinical manifestations of the disease. Third, it is easy to measure the activity of transgene (clotting factor activity) delivery through well-defined coagulation selleck compound assays and finally, there are excellent animal models available. These four factors make hemophilia an excellent disease to investigate gene therapy. Initial clinical trials examining the safety and efficacy of gene transfer in hemophilia have been completed and have demonstrated that gene therapy is feasible; however, there are some obstacles to overcome prior to clinical application. “
“This chapter contains sections titled: Introduction Internal diseases Cardiovascular disease Malignancy and surgical interventions Sexuality Psychological problems Quality of life Balance dysfunctions and risk of falls Conclusion References “
“Summary.  There is a lack of publications concerning the use

of primary prophylaxis in developing countries. The aim of this study was to evaluate the effectiveness of primary prophylaxis therapy in preventing the development of learn more arthropathy in children with severe haemophilia A or B. From January 1999 to April 2009, a prospective study was carried out involving 39 patients with severe haemophilia A or B. These haemophilia A and haemophilia B patients received 20–40 UI kg−1 of factors VIII and IX, three and two times per week, respectively. The patients were followed up by a multidisciplinary team. The analysis was carried out in 23 patients who had been on prophylaxis therapy for at least 12 months. The

orthopaedic evaluation was performed according to the recommendations of the Orthopedic Advisory Committee of the World Federation of Hemophilia, by evaluating pain and bleeding, and by conducting physical examination and radiological assessment (Pettersson’s Joint Score and magnetic resonance): 82.6% of patients who had used the factor regularly did not present any clinical or radiographic changes in the studied joints; 17.4% used the factor irregularly at the beginning of the treatment and of those, most patients presented mild changes in the joints; and 4.3% presented transient knee and ankle pain in spite of regular factor use. The preliminary results of primary prophylaxis confirm its effectiveness in preventing haemophilic arthropathy.