View clinical trials related to Hemophilia.
Filter by:Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region. This study aims to 1. To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region. 2. To investigate modifiable risk factors associated with development of inhibitors in both diseases. 3. To correlate the level of inhibitor with the clinical presentation of the patients. 4. To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.
Background Hemophilia is a sex-linked genetic disorders. When the joint or the muscles is bleeding, it may cause haemarthrosis, synovium, cartilage tissue thickening, joint activity (Range of Motion) decreasing and other musculoskeletal and related disorder. Patients will produce pain in the action, compensatory action occurs, thus causing recurrent of bleeding, and joint damage. There is high rate of ankle joint bleeding in hemophilia. The ankle articular joint disease will affect lower limbs activities, and the functional activities will impaired. Review studies, in addition to physical therapy, Kinesio taping is a common intervention to improve other subjects' static balance, proprioception, functional ankle stability, correct poor posture. The main intervention of this study is physical therapy and Kinesio taping, expect to improve the stability and muscular strength of lower extremities, and balance, correcting gait and lower extremity functional activities of subjects with hemophilia.
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.
Hemophilia is caused by a single-gene defect resulting in familial bleeding disorder. Small increase in gene products could transform a severe form of hemophilia into a mild one. Stem cells from extrahepatic sources are being considered for clinical applications in liver cell therapy as they possess high in vitro culture potential and could be used in transplant procedures. We studied the differentiation of bone marrow hematopoietic stem cells (BM-HSCs) from hemophilia patients' relatives into factor 8 (FVIII)-producing hepatocyte-like cells aiming to expand patients' donor options for partial replacement of mutant liver cells by healthy cells in hemophilia A patients which could manage the severity of the bleeding disorder. BM-HSCs from hemophilic families will be cultured in short-liquid hepatic induction medium. Appearance of hepatic phenotype will be evaluated by alpha-fetoprotein expression using immunocytochemistry. Functional evaluation of transdifferentiation will be done through detection of albumin synthesis using microalbumin assay kit, factor VIII activity by one-stage clotting assay and expression of FVIII messenger RNA( mRNA) by reverse transcription ( RT-PCR). Inducing the differentiation of BM-HSCs by in-vitro manipulation may become a valuable tool to provide a cell source for hepatocyte transplant procedures for treatment of hemophilia patients.