Hemophilia Patients Research Articles

PB0176 Laboratory Monitoring of Severe Hemophilia Patients during PK at the Hemowab Reference Center: A Retrospective Study

PB0176 Laboratory Monitoring of Severe Hemophilia Patients during PK at the Hemowab Reference Center: A Retrospective Study

PB1303 A Study of Support for Hemophilia Patients Using the “ Questionnaire for Daily Movement and Sensory of PwH”

PB1303 A Study of Support for Hemophilia Patients Using the “ Questionnaire for Daily Movement and Sensory of PwH”

OC 12.2 Decreased Quality of Life in Non-Severe Hemophilia Patients with an Increased Bleeding Phenotype

OC 12.2 Decreased Quality of Life in Non-Severe Hemophilia Patients with an Increased Bleeding Phenotype

Association between the levels of serum vitamin D and trace elements and joint health in children with hemophilia

Hemophilia is defined as X-linked recessive bleeding disorder. Recurrent bleeding episodes lead to hemarthrosis.Objectives: to investigate the levels of serum 25(OH) D and trace elements in children with hemophilia A and B and to identify the possible association of these factors with Hemophilia Joint Health Score (HJHS). This case-control study was conducted among children with hemophilia A and B. A total of 48 cases were recruited from the hematology units at the Menoufia University Hospital (n = 36) and Sohag University Hospital (n = 12) from December 2020 to February 2022. Forty healthy controls were matched to cases on age, sex and socioeconomic status. Serum zinc and magnesium levels in the hemophilia patients were significantly lower than in the controls, while serum alkaline phosphatase levels in the cases were significantly higher than in the controls. Informed consent was obtained from all the children's parents and ethical approval was acquired from the ethical committee (ID: 5/2020PEDI38), Faculty of Medicine, Menoufia University. The levels of phosphorus and calcium were the same in two groups. Serum 25(OH) D levels were deficient in 85.4% of the cases and insufficient in 14.6%. None of the hemophilia patients had sufficient levels of serum 25(OH) D. There was no significant correlation between HJHS and the levels of serum trace elements but there was a significant positive correlation between HJHS and annualized bleeding rate and a significant negative correlation between HJHS and serum vitamin D. There was no significant difference regarding the demographic data except for weight and body mass index. The patients had significantly higher weight and body mass index compared to the control group. The levels of serum vitamin D and trace elements were decreased in hemophilia patients, and these low values were associated with the worst joint health.

The Evaluation of Clot Waveform Analyses for Assessing Hypercoagulability in Patients Treated with Factor VIII Concentrate.

Regular prophylactic therapy has become an increasingly common treatment for severe hemophilia. Therefore, hypercoagulability-a potential risk factor of thrombosis-is a cause for concern in hemophilic patients treated with a high dose of FVIII concentrate. In clot waveform analysis (CWA)-thrombin time (TT), a small amount of thrombin activates clotting factor VIII (FVIII) instead of fibrinogen, resulting in FVIII measurements using CWA-TT with a small amount of thrombin. The coagulation ability of patients treated with FVIII concentrate or emicizumab was evaluated using activated partial thromboplastin time (APTT), TT and a small amount of tissue factor-induced FIX activation assay (sTF/FIXa) using CWA. The FVIII activity based on CWA-TT was significantly greater than that based on the CWA-APTT or chromogenic assay. FVIII or FVIII-like activities based on the three assays in plasma without emicizumab were closely correlated; those in plasma with emicizumab based on CWA-TT and chromogenic assays were also closely correlated. CWA-APTT and CWA-TT showed different patterns in patients treated with FVIII concentrates compared to those treated with emicizumab. In particular, CWA-TT in patients treated with FVIII concentrate showed markedly higher peaks in platelet-rich plasma than in platelet-poor plasma. CWA-APTT showed lower coagulability in hemophilic patients treated with FVIII concentrate than in healthy volunteers, whereas CWA-sTF/FIXa did not. In contrast, CWA-TT showed hypercoagulability in hemophilic patients treated with FVIII concentrate. CWA-TT can be used to evaluate the thrombin bursts that cause hypercoagulability in patients treated with emicizumab. Although routine APTT evaluations demonstrated low coagulation ability in patients treated with FVIII concentrate, CWA-TT showed hypercoagulability in these patients, suggesting that the evaluation of coagulation ability may be useful when using multiple assays.

Intra-articular injection of tranexamic acid in patients with haemophilia arthritis: retrospective controlled study in total knee arthroplasty.

Total knee arthroplasty is the main method for the treatment of advanced haemophilic knee arthritis. Due to the particularity of hemophilia, the blood management plan is the focus of the perioperative period for haemophilia patients. This study aimed to investigate the clinical effect and safety of intra-articular injection of tranexamic acid in patients with haemophilia. This is a retrospective study. According to whether tranexamic acid is used or not, patients are divided into tranexamic acid group (n=30) and non-tranexamic acid group (n=29). Total blood loss, intraoperative blood loss, complete blood count, total amount of coagulation factor VIII (FVIII) usage, coagulation biomarkers, inflammatory biomarkers, knee range of motion, knee joint function, pain status, complication rate, and patient satisfaction were assessed and compared at a mean follow-up of 16 months. Injecting tranexamic acid into the knee joint cavity can effectively reduce the hidden blood loss and total blood loss (P<0.001), and reduce the patient's early postoperative inflammation biomarkers, pain status, and limb swelling. Therefore, the patient can obtain a better range of motion following total knee arthroplasty. In the long run, in terms of joint function and surgical satisfaction, there are no statistically significant differences. In addition, there are no statistically significant differences between the two groups of patients in terms of the total amount of FVIII usage, length of stay, and hospitalization expenses. In patients with haemophilia, intra-articular injection of tranexamic acid during total knee arthroplasty can effectively reduce postoperative blood loss, early postoperative inflammation levels, pain and limb swelling, and enable patients to receive higher-quality rehabilitation exercises to get better joint function. Previous studies on TKA in haemophilic patients have already demonstrated the efficacy of intra-articular injections of TXA in reducing postoperative blood loss. Our study confirms this efficacy.

Mobilization under anaesthesia for correction of knee flexion deformity in haemophilia.

Haemophilia patients not treated with primary or secondary prophylaxis, often present with recurrent haemarthrosis. Knee is the most frequently involved joint leading to disabling knee flexion deformity (KFD). Here, we present a retrospective study of our experience on the role of mobilization under general anaesthesia in the correction of KFD. To study the effectiveness of mobilization under anaesthesia (MUA) for correction of knee flexion deformity (KFD) in persons with haemophilia (PWH). Outcome of all patients managed with MUA in our multidisciplinary haemophilia clinic from 2008 to 2019 were included for analysis. PWH with KFD>20 degree who underwent MUA were included in the study. Under general anaesthesia and cover of clotting factor replacement, gentle joint mobilization was done to achieve maximal correction in flexion deformity, followed by above knee casting in this position. The outcome measures assessed were reduction in knee flexion deformity following MUA and complications, if any. Thirty patients (34 knees) with knee flexion deformity were included in the study. Mean age of the study population was 14.23 years ± 8.3. Study population was analysed in two groups, Group 1 included patients who underwent single MUA and Group 2, patients who underwent two or more MUA. There was significant improvement in KFD correction in both groups. [Group 1; Mean difference: 22 ± 13.7, p value -0.01, 95% CI (16.4-27.5) and Group 2; Mean difference 48.8 ± 19.8, p value -0.00, 95% CI (34.2-64.5)]. MUA can be effective in the short-term correction of KFD in PWH particularly those below 15 years of age. It should be done judiciously when target correction is not achieved with other physical methods.

Frequency of Inhibitors among known Hemophilia A Patients.

ABSTRACT&#x0D; Introduction: Inhibitors are IgG alloantibodies which are directed against exogenous clotting factor VIII that neutralize the function of infused clotting factor concentrate used for the treatment. The prevalence of inhibitors in Hemophilia A is around 30% worldwide. Inhibitors occurs as a result of natural immune process of the body as factor concentrates used for treatment are foreign to the body and patient’s own body either produces no factor or produces structurally abnormal protein.&#x0D; Objectives: To determine the frequency of inhibitors among known hemophilia A patients.&#x0D; Methods: Cross-sectional survey done including 100 male patients of hemophilia A and on treatment for more than 1 year, from 2 to 60 years of age, were included. All the patients were undergone aPTT based screening for inhibitor, both immediately and two hours after mixing the patients plasma with the normal plasma.&#x0D; Results: Age range in this study was from 2 to 60 years with mean age of 37.21 ± 14.73 years. Frequency of inhibitors among known hemophilia A patients was seen in 18 (18.0%) of cases.&#x0D; Conclusion: This study concluded that frequency of inhibitors among known hemophilia A patients is quite high.&#x0D; Keywords: Hemophilia A, inhibitors, severity.

Molecular Diagnosis of Hemophilia A and Pathogenesis of Novel F8 Variants in Shanxi, China.

The aim of this study was to perform a molecular diagnosis of hemophilia A (HA) among patients in the Shanxi Province of China. Fifty-two HA patients were tested, including IVS22 (31 samples), IVS1 (3 samples), missense (11 samples), nonsense (3 samples), and 4 cases of frameshift (2 cases of deletion, 1 case of insertion, 1 case of single-base duplication). With the exception of the single-base G duplication variant (p.Ile1213Asnfs*28), this was the hotspot variant reported by research groups at an early stage. The remaining variants were found, for the first time, in the region. The missense variants p.Cys172Ser, p.Tyr404Ser, p.Asp1903Gly, and p.Ser2284Asn, the deletion variant p.Leu2249fs*9, and the insertion variant p.Pro2319fs*97 were novel variants. The application of next-generation sequencing (NGS) molecular diagnosis enriched the variant spectrum of HA, which is greatly significant for individualized genetic counseling, clinical diagnosis, and treatment. NGS and a variety of bioinformatics prediction methods can further analyze the impact of genetic variation on protein structure or function and lay the foundation to reveal the molecular pathogenic mechanism of novel variants.

Factor VIII Level in the Potential Female Carrier Mothers of Patients with Hemophilia A and Its Impact on Joint Health

Background Hemophilia A is an X-linked bleeding disorder. The characteristic bleeding phenotype in patients with severe hemophilia is recurrent spontaneous hemarthrosis with development of hemophilic arthropathy. Heterozygous female carriers with factor VIII (FVIII) activity level lower than 40% may be at risk for bleeding that is usually comparable to that seen in males with mild hemophilia. Aim of the Work To assess factor VIII level in mothers of known patients of hemophilia A correlating it with the frequency of clinically and radiologically documented arthropathy. Patients and Methods A cross sectional study included 40 mothers of patient with hemophilia A during the period from September 2020 to September 2021. Studied participants were subjected to detailed analysis of bleeding history, six major joints range of motion (ROM) examination, functional independence score in hemophilia (FISH), FVIII level one stage assessment and magnetic resonant imaging (MRI) of the reported affected joints. Results Mean age of mothers is 42.25 ± 7.74 years. Eleven mothers (27.5%) had no bleeding symptoms, 16 (40%) had one bleeding symptoms and 13 (32.5%) had two or more bleeding symptoms. As regard joint pain, 13 (32.5%) had severe pain, 3 (7.5%) had ankle pain and 2 (5%) had elbow pain with a mean pain score of 2.55±3.32, and 7 (17.5%) had bilateral pain. Mean FVIII activity was 43.33 IU (± 22.79), ranging from FVIII (0 &amp;lt; 30 IU) in 13 (32.5%), FVIII (30 &amp;lt;60) in 18 (45.0%) and FVIII ≥ 60 IU in 9 (22.5%) among the studied participants. Knees were the most affected joints with decreased mean ROM (±SD) in right and left knees of (123.25 (±12.06) and 120.00 (±15.81)) respectively. The median MRI scores were studied was 5 (2.6%) with median osteochondral changes of 4 (2.5%) and median soft tissue changes of 1 (0-1), their median FISH score of 8 (8-9). By comparing groups at the level of factor VIII, there was no significant difference according to anthropometric bleeding parameters, pain, and MRI score. None of participants needed hospital admission, factor VIII replacement or packed red cells replacement. Conclusion Potential female hemophilia A carriers are at risk of spontaneous bleeding which may entail bleeding in the joints, causing early joint damage. They showed reduced overall joint ROM and structural osteochondral changes in MRI joint examination which mandates further studies about early detection of potential female hemophilia carriers for the possible need of early medical intervention.

Characterization of Immunogenicity and Safety of COVID-19 mRNA-1273 in HIV-Positive Italian Patients with Hemophilia: A Prospective Single-Center Cohort Study.

To characterize the immunogenicity of mRNA-1273 (Moderna, Cambridge, MA, USA) vaccine in HIV-positive hemophilic patients during the third COVID-19 wave in Italy and to investigate biomarkers of coagulation and endothelial perturbation before and after complete vaccination schedule, twenty-three consecutive adult HIV-positive patients with hemophilia were included. Blood was collected before and two weeks after vaccination. We measured anti-SARS-CoV-2 spike protein antibodies to assess immunogenicity; circulating biomarkers of coagulation (protein C and D-dimer), endothelial perturbation (von Willebrand factor (VWF)) and anti-Platelet Factor 4 (PF4) antibodies were analyzed. Flow-based analysis of thrombus formation was performed in nine patients using a flow-chamber device. Two weeks after completing the vaccination schedule, all patients had anti-spike antibodies values consistent with an effective immunization. Mean (±standard deviation) basal values of protein C and VWF (106 ± 21% and 171 ± 45%, respectively) were not significantly different from data obtained two weeks after the second dose (103 ± 20%, 162 ± 43%, respectively). D-dimer median values (interquartile range) were not significantly different at baseline (442 (603-142) ng/mL) and after the second dose (477 (654-262) ng/mL). Anti-PF4 antibodies were detected in three patients with no associated clinical manifestations. No significant differences were found in flow-based analysis of thrombus formation. Our data demonstrate that in HIV-positive patients with hemophilia, SARS-CoV-2 vaccination is effective and safe, with no effects on coagulation and endothelial perturbation.

Adherence to hemophilia patients with prophylaxis: Veritas-Pro and psychometric properties adapted to Turkish.

The Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro) assesses adherence to prophylaxis treatment recommendations in hemophilia patients. This study aimed to adapt the VERITAS-Pro into Turkish and evaluate its reliability and validity. The research design used is a psychometric study. A convenience sample of 102 patients with hemophilia A or B was followed by the Aegean Adult Hemophilia and Thrombosis Center. The VERITAS-Pro was adapted to Turkish in six steps, including forward- and back-translation, committee review, and reliability and validity analysis. Based on the confirmatory factor analysis, modification indices suggested discrepancies amongst items, which were improved upon the removal of items 11 and 15. Findings from this alternative model are: χ2/df = 1.34; RMSEA = 0.05; SRMR = 0.09; and IFI = 0.92. The alternative model showed high adherence rates. Cronbach's alpha value for the Turkish version was found to be 0.83. The test-retest reliability of the Turkish scale ranged from 0.31 to 0.78. All items discriminated significantly between participants who were more adherent and those who were less adherent (t = 23.53; p<0.01). Translation of the VERITAS-Pro into local languages enables more accurate measurement of treatment adherence among people with hemophilia and facilitates cross-cultural comparison studies. According to the validity and reliability evidence obtained, the psychometric properties of the Turkish version of the VERITAS-Pro are suitable.

Pre-operative synovial hyperaemia in haemophilia patients undergoing total knee replacement and the effects of genicular artery embolization: A retrospective cohort study.

Haemophilia is characterized by recurrent joint bleeding caused by a lack of clotting factor VIII or IX. Due to repeated joint bleeding, end-stage arthropathy occurs in relatively young patients. A total knee replacement (TKR) can be a solution. However, TKR may be complicated by perioperative and postoperative bleeds despite clotting factor therapy. The aim of this study was to evaluate the prevalence of pre-operative synovial hyperaemia and the effects of Genicular Artery Embolization on synovial hyperaemia and 3-month postoperative joint bleeding. In this retrospective cohort study, all patients with haemophilia who underwent periarticular catheter angiography between 2009 and 2020 were evaluated after written informed consent. Synovial hyperaemia on angiography was scored by an interventional radiologist. Thirty-three angiography procedures in 24 patients were evaluated. Median age was 54.4 years (IQR 48.4-65.9). Preoperative synovial hyperaemia was observed in 21/33 joints (64%). Moderate and severe synovial hyperaemia was observed in 10/33 joints (30%). Synovial hyperaemia decreased in 13/15 (87%) joints after embolization. Three-month postoperative joint bleeding occurred in 5/32 joints: in 2/18 joints (11%) without synovial hyperaemia and in 3/14 joints (21%) with mild synovial hypertrophy. Non-embolized and embolized joints did not differ regarding 3-month postoperative bleeding (P=.425). No complications were observed after embolization. One-third of patients with haemophilia requiring a TKR had moderate or severe synovial hyperaemia which can be reduced safely by Genicular Artery Embolization prior to TKR. Three-month postoperative bleeding appears to occur independently of the presence of residual mild synovial hyperaemia.

HTRS2023.P2.19 Treatment burden and unmet needs in hemophilia patients with inhibitors: results from a real-world observational study in the United States

HTRS2023.P2.19 Treatment burden and unmet needs in hemophilia patients with inhibitors: results from a real-world observational study in the United States

Hemophilia patients: are they naturally anticoagulated?

Hemophilia is an X-linked bleeding disorder, characterized by low plasma levels of coagulation factor VIII (FVIII) (hemophilia A) or FIX (hemophilia B). Because of this, hemophilia patients (HP) were considered as naturally-anticoagulated and therefore protected from thrombosis. Over the last decades hemophilia care underwent striking changes by the introduction of prophylaxis with repeated injections of standard or modified coagulation factor products that maintain steady-state trough levels of the deficient factor. Meanwhile, new medications, not based on replacement therapy, were developed (i.e., emicizumab and others). However, emicizumab (the only licensed drug) can be used only for prophylaxis; during acute bleeding or surgery, HP require additional therapies, supplementing emicizumab with FVIII/IX concentrates or with bypassing agents (e.g., recombinant activated FVII or activated prothrombin complex concentrate). Owing to the new therapeutic strategies, the hemostatic competency of HP is now much better assured than in the past and therefore their life expectancy is considerably improved. Furthermore, the combined effects of the improved life-expectancy and of the steady-state hemostatic competence achieved by prophylaxis, make HP to be near(normal). They are, therefore, liable to be affected by the circumstantial risk factors of venous thromboembolism (VTE) that are common in the general population. Furthermore, HP undergo frequent surgery/invasive procedures (especially major orthopedic surgery) when they are treated with coagulation factor concentrates or bypassing agents that may increase the risk of post-operative VTE. Therefore, one wonders if HP should be considered for perioperative antithrombotic prophylaxis to prevent postoperative VTE.Clinical data on the value of antithrombotic prophylaxis in this setting are scanty. Indeed, data from an observational multicentre prospective study of 46 HP who underwent orthopedic surgery concluded that the prevalence of postoperative symptomatic VTE was similar to that estimated in the general population. Multicenter prospective trials are warranted to address the value of antithrombotic prophylaxis to avoid post-operative VTE in HP, especially during major surgery when regular prophylaxis is supplemented with additional coagulation factor products or bypassing agents. Until this information is available, HP undergoing major surgery whilst on antihemorrhagic prophylaxis supplemented with coagulation factor concentrates or bypassing agents, should at least receive intermittent pneumatic compression.

Hematuria in a young severe hemophilia patient – A case report on a rare radiological finding

Renal ectopia or ectopic kidney is one of the common renal anomalies in which there is faulty migration of the fetal pelvis during the embryonic period. Hemophilia is an inherited coagulation disorder characterized by recurrent and spontaneous bleeding due to a deficiency of clotting factor VIII or IX. Clotting factor concentrates are used as standard therapy for the management of bleeding episodes. Hematuria is the presence of blood in urine that is either visible to the naked eye (macroscopic) or nonvisible (microscopic). Common etiologies of hematuria include congenital anomalies, calculus, inflammation, and infectious and malignancy of the renal and urogenital tract. Here, we present a 14-year-old male with severe hemophilia A who presented with severe pain abdomen and hematuria. On evaluation, he was found to have a left-sided ectopic kidney. In addition to the overdue discovery of the left-sided ectopic kidney, there is the coexistence of a bleeding disorder which may pose a challenge in the patient’s management.

Altered frequency of FOXP3+ regulatory T cells is associated with development of inhibitors in patients with severe hemophilia A.

The development of anti-factor VIII (FVIII) antibodies or "inhibitors" is a major complication following FVIII replacement therapy in patients with severe hemophilia A (HA), rendering the treatment inefficient. Data on the role of regulatory T cells (Tregs) in inhibitor formation in these patients are rare. Herein, we aimed to investigate whether a difference in the FOXP3+ Tregs is linked to the formation of the inhibitors in severe HA patients. In this cross-sectional study, 32 patients with severe HA (8 patients with inhibitors and 24 without inhibitors) and 24 healthy controls were enrolled. The frequency of FOXP3+ Tregs was determined using multicolor flow cytometry method. Our results showed that the median level of CD4+ CD25+ FOXP3+ Tregs did not significantly differ between HA patients and healthy controls and between HA patients with and without inhibitors (P > 0.05). However, patients with inhibitors had significantly lower amounts of CD4+ CD25- FOXP3+ Tregs compared to those without inhibitors as well as healthy controls (*P = 0.012 and *P = 0.004, respectively). The frequency of CD4+ CD25+ T cells was significantly higher in HA patients who developed inhibitors compared to the inhibitor-negative ones whereas they were lower in inhibitor-negative patients compared to the healthy controls (*P = 0.013 and *P = *0.029, respectively). The percentages of CD4+ CD25+ T cells were positively correlated with the levels of inhibitors in HA patients (r = 0.45, *P = 0.021). Our data demonstrated for the first time that the CD4+ CD25- FOXP3+ Tregs might be implicated in the prevention of inhibitor formation in severe HA patients.

Four Decades of Carrier Detection and Prenatal Diagnosis in Hemophilia A: Historical Overview, State of the Art and Future Directions.

Hemophilia A (HA), a rare recessive X-linked bleeding disorder, is caused by either deficiency or dysfunction of coagulation factor VIII (FVIII) resulting from deleterious mutations in the F8 gene encoding FVIII. Over the last 4 decades, the methods aimed at determining the HA carrier status in female relatives of HA patients have evolved from phenotypic studies based on coagulation tests providing merely probabilistic results, via genetic linkage studies based on polymorphic markers providing more accurate results, to next generation sequencing studies enabling highly precise identification of the causative F8 mutation. In parallel, the options for prenatal diagnosis of HA have progressed from examination of FVIII levels in fetal blood samples at weeks 20-22 of pregnancy to genetic analysis of fetal DNA extracted from chorionic villus tissue at weeks 11-14 of pregnancy. In some countries, in vitro fertilization (IVF) combined with preimplantation genetic diagnosis (PGD) has gradually become the procedure of choice for HA carriers who wish to prevent further transmission of HA without the need to undergo termination of pregnancies diagnosed with affected fetuses. In rare cases, genetic analysis of a HA carrier might be complicated by skewed X chromosome inactivation (XCI) of her non-hemophilic X chromosome, thus leading to the phenotypic manifestation of moderate to severe HA. Such skewed XCI may be associated with deleterious mutations in X-linked genes located on the non-hemophilic X chromosome, which should be considered in the process of genetic counseling and PGD planning for the symptomatic HA carrier. Therefore, whole exome sequencing, combined with X-chromosome targeted bioinformatic analysis, is highly recommended for symptomatic HA carriers diagnosed with skewed XCI in order to identify additional deleterious mutations potentially involved in XCI skewing. Identification of such mutations, which may profoundly impact the reproductive choices of HA carriers with skewed XCI, is extremely important.

Hemophilia in Jordan: An Economic Burden Dilemma of Rare Disease

Jordan is an upper-middle-income country with high expenditures on pharmaceuticals. The development of new rare disease (e.g. hemophilia) therapies has encountered significant obstacles with respect to economic the cost of the disease and treatment. The aim of this overview was to estimate current annual spending on hemophilia treatment in Jordan and estimate the financial impact of adapting a new medication (Emicizumab) recently used for treating hemophilia patients in Jordan. Methods: based on the literature review, direct medical costs were quantified, required items’ costs from the actual practice in Jordan were elicited from an expert panel, and a focus group meeting with the same was conducted one month later to determine the current estimated number of hemophilia Patients in Jordan, identify current treatment on demand quantities and their prices. All related medical costs were also identified (e.g. bleeding, the estimated number of hospital days, and/or Intensive Care Unit per bleeding episodes). Estimation of the annual consumption of current on-demand treatment quantities and cost were calculated and compared with scenarios of adding the new therapy (Emicizumab). Results: showed that the financial impact of using Emicizumab S.C. instead of Recombinant Factor VIIa I.V. on the budget of the Jordanian government will be 425,747 JOD ($601,338) annually. Conclusions: the economic advantages of a new hemophilia treatment might be very substantial for patients.

Exploring the application of doggybone DNA to facilitate non-viral gene therapy delivery to hemophilia patients

Exploring the application of doggybone DNA to facilitate non-viral gene therapy delivery to hemophilia patients