|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
Larger international studies comparing the clinical presentation and treatment modalities of mild clotting FVIII and FIX deficiencies in both haemophilia males and females should be encouraged.
|
31815335 |
2020 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
It is recommended that each haemophilia centre should ensure that appropriate laboratory assays are available for FVIII and FIX products in local clinical use.
|
31846168 |
2020 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
AHCDC: Association of Hemophilia Clinic Directors of Canada; AICE: Italian Association of Hemophilia Centres; ATHN: American Thrombosis and Hemostasis Network; EAHAD: European Association for Haemophilia and Allied Disorders; EHC: European Hemophilia Consortium; FIX: Coagulation Factor IX; FVIII: Coagulation Factor VIII; HAL: Haemophilia Activity List; HJHS: Haemophilia Joint Health Score; HTC: Hemophilia Treatment Centre; HTCCNC: Hemophilia Treatment Centre Collaborative Network of China; MASAC: Medical and Scientific Advisory Council; MDT: Multidisciplinary team; NHD: National Haemophilia Database; NHF: National Hemophilia Foundation; PK: Pharmacokinetics; POCUS: Point of care ultrasound; PWH: People with haemophilia; SHIELD: Supporting Hemophilia through International Education, Learning and Development; WFH: World Federation of Hemophilia.
|
30073913 |
2019 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
Severe hemophilia is classically characterized by a factor VIII (FVIII) or factor IX (FIX) coagulant activity below 1% of normal (spontaneous and severe bleeds).
|
31517708 |
2019 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
The standard therapy for patients with haemophilia is prophylactic treatment with replacement factor VIII (FVIII) or factor IX (FIX).
|
30411401 |
2019 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Two of these proteins, produced in the liver, factor VIII and factor IX, are deficient or present a functional defect in people with haemophilia.
|
30999657 |
2019 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Hemophilia mice that express a mutant allele of human coagulation factor IX (FIX) containing nonsense mutation R338X were treated with <i>eRF1</i>- or <i>eRF3a</i>-ASO.
|
31070517 |
2019 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
It has been proved that the level of factor IX (FIX) is lesser with haemophilia patient and the attempt here is focused to quantify FIX level by interdigitated electrode (IDE) sensor.
|
31743722 |
2019 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
Here, we explore this topic using CPDs from FVIII and FIX and data concerning carriers' hemophilia severity.
|
31267011 |
2019 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Haemophilia is a serious inherited bleeding disorder resulting from a deficiency of coagulation factor VIII (haemophilia A) or coagulation factor IX (haemophilia B).
|
31069799 |
2019 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Nor is there sound evidence as to how subcutaneous non-FVIII/FIX replacement approaches (concizumab; emicizumab; fitusiran) or single intravenous injections of adeno-associated viral vectors (when employing gene therapy) will revolutionize adherence in haemophilia.
|
30146094 |
2019 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Patients with haemophilia who have developed inhibitors against factor VIII (FVIII) or factor IX present a significant concern to those surgeons who operate on them.
|
30507046 |
2019 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
After a second control with a normal FIX level and a second genetic confirmation of hemophilia, no FIX concentrates was administered to perform the infiltration, which occurred without hemorrhagic complication.
|
31272859 |
2019 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
The development of inhibitors (antibodies against FVIII/FIX concentrates) is the main complication in the treatment of hemophilia.
|
29357868 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
On demand and prophylaxis usage of FVIII/ FIX concentrates for the therapeutic management of hemophilia has greatly changed quality of life, and healthy life span of affected patients.
|
29895509 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
Concizumab restored thrombin generation in FVIII and FIX deficient plasmas and decreased blood loss in a rabbit haemophilia model.
|
29845491 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
One of the most challenging issues facing us in the treatment of haemophilia is the development of alloantibodies against infused factor VIII (FVIII) or factor IX (FIX).
|
29517971 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
Furthermore, when combined with reagents promoting translational read-through, Upf3b-ASO treatment leads to the production of functional factor IX protein in hemophilia mice.
|
29334995 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
Clinical trials in hemophilia using adeno-associated virus (AAV) vectors to transfer functional factor IX (FIX) have reported increases in FIX activity to functional levels, reduced bleed frequency, and a lessening or abrogation of the need for costly FIX replacement.
|
29624465 |
2018 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
It is particularly encouraging that relatively stable therapeutic FVIII or FIX expression levels were reached in severe haemophilia patients in recent clinical trials after liver-directed AAV gene therapy.
|
29878653 |
2018 |
|
Hemophilia, NOS
|
0.100 |
Biomarker
|
disease |
BEFREE |
The advent of modified factor VIII (FVIII) and factor IX (FIX) molecules with extended half-lives (EHLs) compared with native FVIII and FIX represents a major advance in the field of hemophilia care, with the potential to reduce the frequency of prophylactic injections and/or to increase the trough level prior to subsequent injections.
|
28264199 |
2017 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
von Willebrand disease (VWD) reflects a loss or dysfunction in von Willebrand factor (VWF), while haemophilia represents a loss or dysfunction of clotting factors such as factor VIII (FVIII) or FIX.
|
28750474 |
2017 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis.
|
28039188 |
2017 |
|
Hemophilia, NOS
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
Varying initial doses of activated eptacog beta (recombinant human FVIIa, rhFVIIa) may provide therapeutic options when treating bleeding in patients with congenital haemophilia who have developed inhibitory antibodies to factor VIII (FVIII) or factor IX (FIX).
|
28984010 |
2017 |
|
Hemophilia, NOS
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
We found sustained therapeutic expression of factor IX coagulant activity after gene transfer in 10 participants with hemophilia who received the same vector dose.
|
29211678 |
2017 |