Results 1 to 10 of about 31,792 (167)

Shaping hemophilia care: lessons and legacy of the SIPPET trial after 10 years [PDF]

Research and Practice in Thrombosis and Haemostasis
The advent of nonreplacement therapies, such as emicizumab, has contributed to a marked reduction of the observed incidence of factor (F)VIII inhibitors in patients with severe hemophilia A.
Flora Peyvandi, Roberta Palla, Isabella Garagiola, Pier Mannuccio Mannucci   +3 more
doaj   +2 more sources

Hemophilia in pediatric age [PDF]

Romanian Journal of Pediatrics, 2023
Hemophilia represents the most common inherited bleeding disorder linked to the X chromosome, which, if not properly treated, can lead to lifelong disabilities.
Cristina Elena Singer, Carmen Simona Cosoveanu, Jaqueline Abdul-Razzak, Alina Popescu, Iulian Iliescu, Maria Madalina Singer, Mihaela Popescu, Alin Iulian-Silviu Popescu   +7 more
doaj   +1 more source

HEMATURIA IN HEMOPHILIA: WHAT DO WE KNOW? A CHALLENGING CASE STUDY AND LITERATURE REVIEW [PDF]

Euromediterranean Biomedical Journal, 2022
Hemophilia is a genetically determined bleeding disorder, which, if not properly managed, can cause lifelong disabilities. Hemorrhages in the joints and soft tissues are largely studied.
Alessandra Di Nora
doaj   +1 more source

Emicizumab-induced photosensitivity

TH Open, 2022
Emicizumab constitutes a novel and effective prophylaxis for hemophilia A patients with and without inhibitors. In this case report, we describe an emicizumab-induced photosensitivity that forced permanent sun-exposure suppression.
Pedro Asensi Cantó, Mercedes Rodríguez Serna, Pilar Lloret Madrid, Jürgen Solís Ruiz, Ana Rosa Cid Haro, Santiago Bonanad Boix, Saturnino Haya Guaita   +6 more
doaj   +1 more source

Surgical Procedures Requiring Hospitalization and Perioperative Management for Patients with Hereditary Bleeding Disorders

Clinical Pediatric Hematology-Oncology, 2022
Background : : Hemophilia requires a lifetime care for bleeding control and complications. Although patients diagnosed with hemophilia receive factor replacement, they also experience a variety of medical problems as they age.
Hyeun Su Seo, Won Kee Ahn, Seung Min Hahn, Jung Woo Han, Chuhl Joo Lyu   +4 more
doaj   +1 more source

Updates on Novel Non-Replacement Drugs for Hemophilia

Pharmaceuticals, 2022
Over the last decade, the world of hemophilia has experienced an unprecedented therapeutic advance, thanks to the progress in bioengineering technologies, leading to the introduction of drugs with novel mechanisms of action based on restoring thrombin ...
Roberta Gualtierotti, Samantha Pasca, Alessandro Ciavarella, Sara Arcudi, Andrea Giachi, Isabella Garagiola, Chiara Suffritti, Simona Maria Siboni, Flora Peyvandi   +8 more
doaj   +1 more source

Emicizumab initiation and bleeding outcomes in people with hemophilia A with and without inhibitors: A single‐center report

Research and Practice in Thrombosis and Haemostasis, 2021
Background Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.
Beth Boulden Warren, Adrian Chan, Marilyn Manco‐Johnson, Brian R. Branchford, Tyler W. Buckner, Genevieve Moyer, Elizabeth Gibson, Dianne Thornhill, Michael Wang, Christopher J Ng   +9 more
doaj   +1 more source

New therapies for von Willebrand disease

Blood Advances, 2019
: The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C ...
Pier Mannuccio Mannucci
doaj   +1 more source

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

Research and Practice in Thrombosis and Haemostasis, 2022
Background N8‐GP (turoctocog alfa pegol; Esperoct) is a glycoPEGylated human recombinant factor VIII (FVIII). Objectives Pathfinder8 (NCT01480180) was a phase 3, multinational, open‐label, nonrandomized trial to investigate the long‐term safety and ...
Steven R. Lentz, Kaan Kavakli, Robert Klamroth, Mudi Misgav, Azusa Nagao, Alberto Tosetto, Pernille Juul Jørgensen, Marek Zak, Laszlo Nemes   +8 more
doaj   +1 more source