PubMed Journals Articles About "Chugai Pharma Emicizumab Receives Priority Review Designation Hemophilia" RSS

23:39 EDT 22nd March 2018 | BioPortfolio

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Showing "Chugai Pharma Emicizumab Receives Priority Review Designation Hemophilia" PubMed Articles 1–25 of 17,000+

Emicizumab Prophylaxis in Hemophilia A with Inhibitors.

Emicizumab Prophylaxis in Hemophilia A with Inhibitors.

Emicizumab Prophylaxis in Hemophilia A with Inhibitors.

Emicizumab Prophylaxis in Hemophilia A with Inhibitors.

Emicizumab prophylaxis overcomes factor VIII inhibitors in Hemophilia A.

The spectrum of bleeding in women and girls with haemophilia B.

Although hemophilia B affects 1 in 25,000 males there may be 3 female hemophilia B carriers per affected male. This clinical review highlights the unique challenges faced by hemophilia B carriers including the under-recognition of bleeding symptoms associated with and without FIX deficiency, discrepancies in correlation between genotype and bleeding phenotype and therapeutic considerations utilizing clinical vignettes of common scenarios.

Two-incision laparoscopic appendectomy for a severe hemophilia A child patient with coagulation factor VII deficiency: Case report and review of literature.

The main complication of patients with severe hemophilia is recurrent bleeding events that usually affected musculoskeletal contractures. And replacement therapy methods were continuously improved to minimize adverse impacts brought by those complications. However, only several cases reported about the appendectomy for hemophilia A. We report a case of acute appendicitis treated by two-incision laparoscopy in a boy with hemophilia A and coagulation factor VII deficiency for the first time.

Emicizumab-kxwh: First Global Approval.

Emicizumab-kxwh (Hemlibra®) is a bispecific humanized monoclonal antibody that restores the function of missing activated FVIII by bridging activated FIX and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab-kxwh is approved in the USA for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and paediatric patients with haemophilia A (congenital FVIII deficiency) with FVIII inhibitors. Subcutaneous emicizumab-kxwh is await...

Optimization of prophylaxis for hemophilia A.

Prophylactic injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in people with hemophilia. The aim of this study was to identify optimal person-specific prophylaxis regimens for children with hemophilia A.

Hemophilia gene therapy comes of age.

Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX act...

Impact of Hemophilia B on Quality of Life in Affected Men, Women, and Caregivers - Assessment of Patient-Reported Outcomes in the B-HERO-S Study.

Health-related quality of life (HRQoL) is impaired in patients with hemophilia, however the impact in mild/moderate hemophilia B and affected women is not well characterized.

Revascularization strategies and in-hospital management in acute coronary syndromes complicated by hemophilia A or hemophilia B.

: Among adult patients with hemophilia A and hemophilia B the emergent management of acute coronary syndromes (ACSs) is challenging, and exposure to antithrombotic agents and/or revascularization procedures may confer an enhanced risk of bleeding. We sought to identify clinical characteristics and in-hospital outcomes among ACS patients with hemophilia A/hemophilia B, compared with matched noncoagulopathic ACS controls. Case discharges from the Nationwide Inpatient Sample, Healthcare Cost and Utilization Pr...

Acquired Hemophilia presenting as Gross Hematuria following Kidney Stone - A Case Report and Review of the Literature.

A rare condition in itself, acquired hemophilia A, seldom presents as isolated gross hematuria. It is a serious condition with a high mortality rate and thus clinical suspicion followed by prompt diagnosis is imperative (1). In fact, only 8 cases of such presentation of this condition have been reported thus far in the literature. Of these, none describe the initial presentation of hematuria with the inciting event of a kidney stone. We present a case of a 67-year-old man with signs and symptoms of nephroli...

FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B.

A phase 1/2 clinical trial of AAV-mediated gene therapy in patients with hemophilia B using an enhanced specific activity factor IX (FIX) transgene reports sustained levels of FIX levels, leading to the near elimination of bleeding for more than a year and without serious adverse side effects. These results are the best outcome to date for hemophilia gene therapy.

Ultrasound for Early Detection of Joint Disease in Patients with Hemophilic Arthropathy.

Joint bleeding represents the most commonly reported type of hemorrhage in patients affected by hemophilia. Although the widespread use of prophylaxis has been able to significantly reduce the onset of arthropathy, it has been shown that a non-negligible percentage of patients develop degenerative changes in their joints despite this type of treatment. Thus, periodic monitoring of the joint status in hemophilia patients has been recommended to identify early arthropathic changes and prevent the development ...

Economic Burden of Illness among Persons with Hemophilia B from HUGS Vb: Examining the Association of Severity and Treatment Regimens with Costs and Annual Bleed Rates.

To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR.

Patient and public engagement in priority setting: A systematic rapid review of the literature.

Current research suggests that while patients are becoming more engaged across the health delivery spectrum, this involvement occurs most often at the pre-preparation stage to identify 'high-level' priorities in health ecosystem priority setting, and at the preparation phase for health research.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia.

A case report of acute inferior myocardial infarction in a patient with severe hemophilia A after recombinant factor VIII infusion.

The extent of protective effects of hemophilia against thrombotic events such as myocardial infarction (MI) and other acute coronary syndromes remains to be determined, as major risk factors for cardiovascular disease exist despite factor VIII (FVIII) deficiency. We present a case report of a 41-year-old male with severe hemophilia A and several cardiovascular risk factors.

Renew the Priority for Manuscript Review.

High-priority and low-priority drug-drug interactions in different international electronic health record systems: A comparative study.

To investigate whether alert warnings for high-priority and low-priority drug-drug interactions (DDIs) were present in five international electronic health record (EHR) systems, to compare and contrast the severity level assigned to them, and to establish the proportion of alerts that were overridden.

Loss of Magnet® Designation and Changes in RN Work Engagement: A Report on How 1 Hospital's Culture Changed Over Time.

The aim of this study was to examine the loss of Magnet® designation and how RNs' work engagement changed at 1 community hospital.

Modified Primary Prophylaxis in Previously Untreated Patients With Severe Hemophilia A in Iran.

Recently the low-dose tailoring method of primary prophylaxis has been introduced for previously untreated patients with hemophilia A.

When is audit and feedback effective in dementia care? A systematic review.

Evidence-based care for people with dementia is a priority for patients, carers and clinicians and a policy priority. There is evidence that people with dementia do not always receive such care. Audit and feedback, also known as clinical audit, is an extensively-used intervention to improve care. However, there is uncertainty about the best way to use it.

What can we learn from using formal patient-reported outcome instruments to assess pain, functional impairment, anxiety, and depression in US adults with hemophilia?

People with hemophilia frequently suffer from arthropathy that leads to pain and functional impairment, ultimately resulting in reduced quality of life. The impact of pain and functional impairment on the lives of people with hemophilia was explored in the Pain, Functional Impairment, and Quality of Life (P-FiQ) study. Various patient-reported outcome (PRO) instruments were employed to assess the effect of pain and functional impairment on multiple aspects of health in people with hemophilia. The results pr...

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