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PubMed Journals Articles About "RFVIIa Prophylaxis In Children With Hemophilia A And Inhibitors" RSS

16:23 EST 19th January 2019 | BioPortfolio

RFVIIa Prophylaxis In Children With Hemophilia A And Inhibitors PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest RFVIIa Prophylaxis In Children With Hemophilia A And Inhibitors articles that have been published worldwide.

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Showing "rFVIIa Prophylaxis Children With Hemophilia Inhibitors" PubMed Articles 1–25 of 10,000+

Cost-Effectiveness of rFVIIa versus pd-aPCC in the Management of Mild to Moderate Bleeds in Pediatric Patients with Hemophilia A with Inhibitors in Mexico.

To compare the costs and clinical consequences of treating mild-to-moderate joint bleeds with recombinant activated factor VII (rFVIIa) versus plasma-derived activated prothrombin complex concentrate (pd-aPCC) in pediatric patients with hemophilia A with inhibitors in Mexico.


A Retrospective Analysis of Intracranial Hemorrhage in Children with Hemophilia A.

To investigate the incidence, risk factors, clinical manifestations and prognosis of intracranial hemorrhage (ICH) in children with hemophilia A in a center of China, we conducted a retrospective analysis of 126 children with hemophilia A at our hospital in recent 4 years. Thirty-six children with hemophilia A (including 19 severe cases, and 17 moderate cases complicated with joint diseases) received low dose factor VIII (FVIII) prophylaxis, and none of them had ICH. However, 13 cases of hemophilia A not gi...

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.

Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis. In a phase 3, multicenter trial, we investigated its use as prophylaxis in persons who have hemophilia A without factor VIII inhibitors.


Evaluating the safety of emicizumab in patients with hemophilia A.

Patients affected by Hemophilia A often require frequent prophylactic and therapeutic self-infusion. For those who develop inhibitors, treatment options are limited and mortality is increased. Emicizumab, a bispecific antibody to Factors IXa and X that carries out the function of Factor VIII (FVIII), represents a novel therapeutic approach. Areas covered: We review the clinical trials and key laboratory assay research for emicizumab. Emicizumab reduced the annualized bleeding rate by 87% compared to placebo...

Emicizumab for routine prophylaxis to prevent bleeding episodes in patients with hemophilia A.

Hemophilia A is an X-linked bleeding disorder caused by defects in the gene encoding factor VIII (FVIII). Routine prophylaxis with exogenous FVIII requires frequent intravenous injections. One of the most challenging issues in the treatment of hemophilia A is the development of alloantibodies against infused FVIII. Presence of inhibitors results in an ineffective factor replacement therapy and increases the risk of morbidity and mortality in these patients. Therefore, there is growing interest in the develo...

The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study.

Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis.

Prevalence of factor VIII inhibitors among Afghan patients with hemophilia A: a first report.

: Prevalence of inhibitors in Afghan hemophilia patients has not been reported previously. Our aim was to determine the prevalence of factor VIII inhibitors among hemophilia A patients from the Kabul province of Afghanistan to identify and characterize the pattern of inhibitor formation. Clinical information and blood samples were collected from three hemophilia centers in Kabul, Afghanistan. Plasma samples were obtained from 62 patients with severe (80.5%) and 15 patients with moderate hemophilia A (19.5%)...

Inpatient Health Care Utilization in Children With Hemophilia Before and After the Joint Outcome Study Publication.

The "Joint Outcomes Study" (JOS) demonstrated improved joint outcomes for patients receiving primary prophylaxis versus on-demand therapy. The impact of primary prophylaxis on inpatient health care utilization is not well-defined. To evaluate changes in hospitalization care of children with hemophilia before and after the 2007 JOS publication, this study utilized the Pediatric Health Information System (PHIS) to evaluate admissions for patients with hemophilia A or B (age, 2 to 7) admitted between January 2...

Inhibitors in Hemophilia B.

Hemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX

Value of prophylaxis vs on-demand treatment: Application of a value framework in hemophilia.

Therapeutic advances over the past 30 years have led to longer life expectancy and improved quality of life (QOL) for persons with hemophilia. Access to innovative therapy may be compromised if treatment decisions are driven solely by cost. New strategies are needed to assess true therapeutic values, along with financial cost, as physicians, policymakers, payers and manufacturers work together to improve patient care.

Perioperative haemostasis in children with haemophilia and inhibitors during central venous catheter surgery: The Karolinska model.

Impact of Exercise/Sport on Well-being in Congenital Bleeding Disorders.

Physical activity provides many benefits in patients with congenital bleeding disorders. Patients with hemophilia are encouraged to participate in exercise and sports, especially those patients receiving prophylaxis. Several publications and guidelines have explored this issue in hemophilia patients, evaluating in particular the impact of physical activity on patients' well-being and quality of life. The other rare congenital bleeding disorders are less studied; they are heterogeneous in terms of clinical b...

Reliability and Validity of Patient-Reported Outcome Instruments in US Adults with Hemophilia B and Caregivers in the B-HERO-S Study.

To assess the reliability and validity of 6 patient-reported outcomes (PRO) instruments for evaluating health-related quality of life in adults with mild-severe hemophilia B and caregivers of children with hemophilia B, including affected women/girls.

Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: Pooled data from the paradigm™ trials.

Prophylaxis with replacement factor IX (FIX) reduces bleeding frequency and improves quality of life in haemophilia B patients. With prophylaxis, the likelihood of bleeding is lowered with increasing trough levels. New products with extended half-life (EHL) can maintain high factor activity levels over prolonged periods, compared with standard FIX products.

Prevalence of FVIII inhibitors in severe haemophilia A patients: Effect of treatment and genetic factors in an Indian population.

Factor replacement therapy in treatment of haemophilia A is complicated by the production of neutralising antibodies known as inhibitors. The formation of inhibitors is multifactorial being associated with both genetic and environmental factors.

Oral hygiene and dentition status in children and adults with hemophilia: A case-control study.

People with hemophilia constitute a significant proportion of the population and an oral health care professional faces a considerable challenge while treating them. This study aimed to assess the oral health and dentition status as well as fear of dental treatment in patients with hemophilia and compare it with age-matched healthy subjects.

Preventing or Eradicating Factor VIII Antibody Formation in Patients with Hemophilia A: What Can We Learn from Other Disorders?

Eradication of factor VIII (FVIII) specific neutralizing antibodies (also known as inhibitors) by the traditional method of immune tolerance induction (ITI) is costly and unsuccessful in one out of three patients. Furthermore, effective inhibitor prevention strategies are presently lacking. An overview is given in this narrative review of antidrug antibody prevention or eradication strategies that have been used in disorders beyond hemophilia A, with the aim of analyzing what we can learn from these strateg...

Impact of Trimethoprim-Sulfamethoxazole Urinary Tract Infection Prophylaxis on Non-UTI Infections.

In this secondary analysis of the Randomized Intervention for Children with Vesicoureteral Reflux (RIVUR) cohort, we found that daily prophylaxis with trimethoprim-sulfamethoxazole was not associated with an increased or decreased risk of skin and soft tissue infections, pharyngitis or sino-pulmonary infections in otherwise healthy children aged 2-71 months.

The impact of GPIbα on platelet-targeted FVIII gene therapy in hemophilia A mice with pre-existing anti-FVIII immunity.

We have previously demonstrated that VWF is essential in platelet-specific FVIII (2bF8) gene therapy of hemophilia A (HA) with inhibitory antibodies (inhibitors). At the site of injury, platelet adherence is initiated by VWF binding to the platelet GPIb complex.

Effect of Levofloxacin Prophylaxis on Bacteremia in Children With Acute Leukemia or Undergoing Hematopoietic Stem Cell Transplantation: A Randomized Clinical Trial.

Bacteremia causes considerable morbidity among children with acute leukemia and those undergoing hematopoietic stem cell transplantation (HSCT). There are limited data on the effect of antibiotic prophylaxis in children.

Antibiotic prophylaxis in neutropenic children with acute leukemia: do the presently available data really support this practice?

Antibiotics are frequently administered for prophylaxis of fever in neutropenic children with cancer. This strategy is mainly derived from adults' data, and various pediatric studies evidenced the effectiveness of antibiotics (e.g. fluorquinolones) in the prevention of febrile neutropenia. However, only two pediatric randomized, double blind, placebo-controlled trials have been performed, with a total of 262 leukemic children enrolled, and no other one was ever powered for analyzing effectiveness over other...

Alternative treatment options for pediatric hemophilia B patients with high-responding inhibitors: A thrombin generation-guided study.

Little is known about the challenging treatment of pediatric patients with hemophilia B and inhibitors due to disease rarity. We describe three patients diagnosed in childhood and followed up to 9 years. All three had allergic reactions to Factor IX, but two were later safely treated for bleeding episodes with activated prothrombin complex concentrates (APCC = FEIBA). The third was given only recombinant activated Factor VIIa. Based on ex vivo thrombin generation analysis, a new alternative treatment of c...

Impact of intermediate-dose prophylaxis on progression of haemarthropathy in patients with severe haemophilia A: A 10-year, single-centre experience in Korea.

To determine the impact of 10-year intermediate-dose prophylaxis on haemarthropathy progression in patients with severe haemophilia A (SHA).

Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing.

Standard prophylaxis dosing based on bodyweight may result in over- or under-dosing due to interpatient variability. Adopting individual pharmacokinetic (PK) based tailoring may improve adherence to treatment guideline, and consequently clinical outcomes. Here we report clinical observations performed across the adoption of individual PK based tailoring in a single center in Japan.

Use of proton pump inhibitors among Danish children: a 16-year register-based nationwide study.

Proton pump inhibitors (PPI) are among the most frequently used drugs in the developed countries. In recent years, their use among children and adolescents has been on the increase. Guidelines recommend use for a period no longer than 4-8 weeks. The aim of this study was to describe time trends in prescribing patterns of PPI use among children, with emphasis on persistence to therapy.


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