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Background: Deficiency of factor VIII (FVIII) (Hemophilia A) and factor IX (Hemophilia B) are the most frequent coagulation defects. The incidence of hemophilia A varies in different regions of India. Aims and Objectives: The present study was done to classify hemophilia A patients by estimating FVIII levels in them and to describe the clinical profile of the patients attending a tertiary care hospital in southern part of India, Kerala. Materials and Methods: A hospital-based cross-sectional study was done on patients with hemophilia A, attending Department of Medicine and Department of Pediatrics from March 2012 to September 2013. Demographic and clinical details were collected using per forma and quantitative assessment of FVIII levels were done using semi-automated blood coagulation analyzer. Results: Out of 90 cases studied, majority of the patients had severe hemophilia (85.6%), followed by mild (10%) and moderate hemophilia (4.4%). Positive family history was seen in 67.8% patients. Median age of the patients at diagnosis was 1 years of age. Most common clinical presentation was found to be hemarthrosis (84.4%) followed by bleeding into muscle (56.7%). Knee joint (75.6%) was the predominantly affected joint. Bony ankylosis (30%), intracranial bleed (14.4%), and retroperitoneal bleed (24.4%) were the most common complications noted. Bleeding manifestations such as gum bleed (24.4%), epistaxis (12.2%), hematuria (37.8%), and ecchymosis (15.6%) were also reported among them. Conclusion: Proportion of severe hemophilia among hemophilia A patients was found to be higher in number than in other parts of the country. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Therefore, promotion of low cost, regular availability of factor concentrates, prophylactic factor replacement, establishing comprehensive care center, and regular training of medical and paramedical staff will help in reducing the complications due to this disease.
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OBJECTIVE@#To calculate the pharmacokinetic parameters of recombinant human coagulation factor Ⅷ using myPKFiT in patients with severe hemophilia A, and provide an individualized treatment plan for patients.@*METHODS@#A total of 42 patients with severe hemophilia A who were treated with recombinant human coagulation factor Ⅷ were included from January 2021 to December 2021. myPKFiT was used to calculate the pharmacokinetic parameters of FⅧ, and the individualized treatment plan for hemophilia A patients was formulated.@*RESULTS@#The median age of 42 patients with severe hemophilia A was 31（16-50） years old, the average weight was 54.0±9.9 kg, the half-life of FⅧ was 12.05±1.6 h, the time to more than 1% of the baseline was 62.3±15.3 h, and the 0 bleeding rate after the guidance of myPKFiT was significantly increased from 39% to 49%, the Annual bleeding rate was reduced from 3.6±2.5 to 2.1±2.0, and the Annual joint bleeding rate was reduced from 3.2±2.2 to 1.9±0.9, all of which were statistically different (P<0.05).@*CONCLUSION@#Individualized therapy in patients with severe hemophilia A who were guided by myPKFiT assay of pharmacokinetics parameters can significantly reduce the annual bleeding rate and annual joint bleeding rate of patients.
Subject(s)
Adult , Humans , Middle Aged , Adolescent , Young Adult , Blood Coagulation Factors , Factor VIII/pharmacokinetics , Hemophilia A , Hemorrhage , Recombinant Proteins/pharmacokineticsABSTRACT
<p><b>OBJECTIVE</b>To investigate the efficacy of short-term full-dose prophylaxis in adult Chinese patients with severe hemophilia A.</p><p><b>METHODS</b>Thirteen adult Chinese patients with severe hemophilia A receiving on-demand or low-dose prophylaxis underwent ultrasound examination of the target joints and evaluation of Hemophilia Joint Health Score (HJHS). The data of annual bleeding episodes in the period of on-demand or low-dose prophylaxis were collected retrospectively from the patients, and the changes in bleeding and joint condition (ultrasound findings of the target joints and HJHS) were observed during short-term full-dose prophylaxis. The activity intensity of the patients was assessed using the IPAQ questionnaire, and the 72 h FⅧ trough activity was measured during full-dose prophylaxis.</p><p><b>RESULTS</b>The median age of the 13 patients was 26.0 (20.5-29.0) years. For full-dose prophylaxis, the patients received a median therapeutic dose of 31.0 (29.1-33.0) IU/kg, administered for 3 times per week; the median 72 h FⅧ trough activity of patients was 1.7% (1.3-3.4%). During the follow-up period for 3 months, the annual bleeding rates (ABR) and annual joint bleeding rates (AJBR) decreased significantly in all the patients (=0.001 and 0.001, respectively), but zero bleeding was achieved in only 4 patients (30.8%) and zero joint bleeding in 7 patients (53.8%); 9 patients (69.2%) still experienced breakthrough bleeding. The damage severity of target joints assessed by ultrasound and HJHS in 6 patients (46.2%)was worse than before and no obvious progression of target joints damage was found in 7 patients (53.8%). Compared with the patients without progression, the patients with worsened joint damage had poorer baseline joint condition, higher bleeding frequencies before and during the follow-up, a higher intensity of physical activity, and a lower baseline FⅧ activity.</p><p><b>CONCLUSIONS</b>At present, although short-term full-dose prophylaxis can significantly reduce the bleeding and partially prevent the progression of joint damage, it is not yet possible to achieve the goal of zero bleeding for all adult patients with severe hemophilia A in China, nor can it completely prevent further joint damage. For adult patients with different clinical bleeding phenotypes, joint conditions and physical activity intensity, individualized therapy involving additional evaluation methods should be implemented, and physiotherapy and surgical intervention can be considered when necessary.</p>
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An 18-Year-old male hemophiliac with high titer of factorVIII inhibitor, stage V hemophilic arthropathy in right knee joint and a history of hematuria and retroperitoneal hemorrhage was admitted because of acute and massive bleeding of epistaxis, pulmonary hemorrhage and intestinal bleeding. The bleeing was not controolled by massive infusion of factorVIII concentrates but by prothrombin complex concentrates and high dose of factorVIII concentrates. He showned symptoms of sustained fever and diffuse pulmonary infiltration which was diagnosed as pulmonary hemosiderosis by MRI. We suppressed his immune reaction by prednisolne to prevent the formation of factorVIII inhibitor. He has been followed up for 3 years and shown no massive bleeding there-after.