Results 111 to 120 of about 22,597,991 (289)

Timing of inhibitor development in >1000 previously untreated patients with severe hemophilia A.

Blood, 2019
TO THE EDITOR: Inhibitory antibodies (inhibitors) against factor VIII (FVIII) develop in 25% to 35% of previously untreated patients (PUPs) with severe hemophilia A (SHA). It is the most serious complication of classic hemophilia treatment.[1][1][⇓][2]-[
H. M. van den Berg, K. Fischer, M. Carcao, H. Chambost, G. Kenet, K. Kurnik, C. Königs, C. Male, E. Santagostino, R. Ljung   +9 more
semanticscholar   +1 more source

INDUCTION OF TOLERANCE BY ORAL ADMINISTRATION OF FACTOR VIII AND TREATMENT OF HEMOPHILA [PDF]

, 2011
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a Subject a therapeutically effective amount of the deficient anti gen, wherein the antigen is not present in a liposome.
Alpan, Oral, Kamala, Tirumalai, Matzinger, Polly, Velander, William Hugold   +3 more
core   +1 more source

Reducing Surgery for Pediatric Posttonsillectomy Hemorrhage Using Tranexamic Acid: A Quality Improvement Initiative

Otolaryngology–Head and Neck Surgery, EarlyView.
Abstract Objective Evaluate the use of tranexamic acid (TXA) and observation as a management option for pediatric patients presenting with posttonsillectomy hemorrhage (PTH). Study Design Retrospective analysis of a prospectively implemented quality improvement initiative with a historical control comparison group. Setting Tertiary children's hospital.
Laura A. Petrauskas, Janavi Sethurathnam, Ansley J. Kunnath, Rahul K. Sharma, John Ceremsak, Ryan H. Belcher, James D. Phillips, Jay A. Werkhaven, Amy S. Whigham, Lyndy J. Wilcox, Christopher T. Wootten, Frank W. Virgin, Jason S. Park   +12 more
wiley   +1 more source

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

Scientific Reports, 2019
Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as FVIII protein injection and gene therapies via AAV-delivered F8 transgene in
Hainan Chen, Mi Shi, A. Gilam, Q. Zheng, Yin Zhang, Iva Afrikanova, Jinling Li, Z. Gluzman, Ruhong Jiang, L. Kong, R. Chen-Tsai   +10 more
semanticscholar   +1 more source

Diagnosis of von Willebrand disease in Argentina: a single institution experience [PDF]

, 2017
von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostlyinherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF).
Blanco, Alicia Noemi, Kempfer, Ana Catalina, Lazzari, María Ángela, Paiva Palomino, Juvenal Hernán, Sánchez Luceros, Analía Gabriela, Woods, Adriana Inés   +5 more
core   +1 more source

International Perspectives on Vitamin K Deficiency Bleeding in Infants: A Cross‐Sectional Questionnaire‐Based Survey

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Vitamin K deficiency bleeding (VKDB) can occur in neonates and infants due to low placental transfer and storage of VK. Although the incidence has decreased with global VK prophylaxis at birth, VKDB remains a concern. This study aimed to assess the recent magnitude, risk factors, and outcomes of VKDB and barriers/limitations to VK ...
Nongnuch Sirachainan, Patcharee Komvilaisak, C. Heleen van Ommen, Shoshana Revel‐Vilk, Shouichi Ohga, Ayesha Zia, Masayuki Ochiai, Daijiro Takahashi, Shutaro Suga, Veerle Labarque, Ernest K. Amankwah, Bounpalisone Souvanlasy, Marisol Betensky, Anthony KC Chan, Ampaiwan Chuansumrit, Neil Goldenberg, Madhvi Rajpurkar   +16 more
wiley   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

Prenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada, Anthony Atala, Graça Almeida‐Porada   +2 more
wiley   +1 more source

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Catheter Ablation for Atrial Fibrillation in Patients with Hemophilia or von Willebrand Disease

TH Open, 2019
Background Management of atrial fibrillation (AF) is complex in patients with bleeding disorders. Catheter ablation such as pulmonary vein isolation (PVI) has been suggested in cases with bleeding disorders. However, data on safety are missing.
Paul R. van der Valk, Eveline P. Mauser-Bunschoten, Jeroen F. van der Heijden, Roger E. G. Schutgens   +3 more
doaj   +1 more source

Incidental Identification of Potentially Affected Individuals Through Expanded Carrier Screening During Preconception or Early Pregnancy

Prenatal Diagnosis, EarlyView.
ABSTRACT Objective Expanded carrier screening (ECS) is used to assess the reproductive probability of having an offspring affected by an autosomal recessive or X‐linked recessive genetic disorder. Rarely, a presumably healthy individual may be identified to carry variants that could influence their health.
Yan Lü, Jiazhen Chang, Yulin Jiang, Xiya Zhou, Na Hao, Yiqing Yu, Mengmeng Li, Kaili Yin, Xueting Yang, Qingwei Qi   +9 more
wiley   +1 more source