Results 81 to 90 of about 82,032 (316)

Changes over the last two decades in Hemophilia A and B management in a Tertiary Care Pediatric Centre

Clinical Epidemiology and Global Health
Objective: The treatment of Hemophilia A and B has significantly evolved. Study design: we conducted a retrospective, observational study among patients with HA and HB managed in a Pediatric Hematology and Oncology Unit for almost 22 years for a total of
M.P. Esposto, E. Bonetti, R. Balter, V. Pezzella, A. Zaccaron, V. Vitale, G. Caddeo, M. Chinello, G. Tridello, G. Poli, S. Cesaro   +10 more
doaj   +1 more source

Hemophilia Severity and Its Association With Mental Health and Health‐Related Quality of Life—Results From a Cross‐Sectional Multicenter Study

Haemophilia, EarlyView.
ABSTRACT Background Limited existing research on mental health and health‐related quality of life (HRQoL) in people with hemophilia (PwH) suggests these patients still may have poor mental health despite treatment advances significantly improving somatic outcomes.
Francesca Schmitt, Laura Maier, Stefan Lerch, Manuela Albisetti, Alice Trinchero, Lukas Graf, Heinz Hengartner, Pierre Fontana, Nicolas von der Weid, Katrin Scheinemann, Sylvia von Mackensen, Corinna Reichl, Marialuisa Cavelti, Ines Mürner‐Lavanchy, Johanna A. Kremer Hovinga, Michael Kaess, Mutlu Kartal‐Kaess   +16 more
wiley   +1 more source

On a break with the X: the role of repair of double-stranded DNA breaks in X-linked disease [PDF]

, 2012
The problem of managing free reactive DNA ends in eukaryotic cells has resulted in the development of a number of mechanisms in order to ensure that free ends are rendered non-reactive, or that the double-strand DNA breaks generating the free ends are ...
Cecceroni, Lucia, De Caris, Laura, Tummala, Hemanth   +2 more
core   +4 more sources

TNF‑α Gene Polymorphisms as Determinants of Alloantibody Emergence in Hemophilia: A Systematic Review and Meta‐Analysis

Haemophilia, EarlyView.
ABSTRACT Introduction Inhibitor development remains one of the most serious complications of replacement therapy in patients with hemophilia. Tumour necrosis factor‐alpha (TNF‐α) is a key pro‐inflammatory cytokine, and its genetic variants have been implicated in immune‐related conditions.
Alessandra Faustino da Conceição Bezerra, Natã Abner Andrade Carito de Sousa, Suely Meireles Rezende, Renan Pedra de Souza   +3 more
wiley   +1 more source

Use of andexanet alfa: A British Society for Haematology position statement

British Journal of Haematology, EarlyView.
Richard J. Buka, Deepa J. Arachchillage, Catherine N. Bagot, Gary Benson, Charlotte A. Bradbury, Nicola Curry, Beverley J. Hunt, David J. Sutton, Mari R. Thomas, John Paul Westwood, Karen Breen   +10 more
wiley   +1 more source

National Bleeding Disorder Foundation Clinical Practice Recommendations for Laboratory Screening of Iron Deficiency With and Without Anemia in the Inherited Bleeding Disorders Population

Haemophilia, EarlyView.
ABSTRACT Introduction The National Bleeding Disorder Foundation (NBDF) Medical and Scientific Advisory Council (MASAC) was formed in 1954 and issues recommendations and advisories pertinent to the bleeding disorders community. MASAC is comprised of > 25 rotating members from different medical disciplines and lived experience experts.
Glaivy Batsuli, Magdalena D. Lewandowska, Ming Y. Lim, Kelly Tickle, Maureen K. Baldwin, Rose Bender, Jennifer Davila, Brendan Hayes, Amanda Jacobson‐Kelly, Peter Kouides, Michele P. Lambert, Shilpa Nataraj, Michelle Sholzberg, Roona Sinha, Hanny Al‐Samkari, Amy L. Dunn   +15 more
wiley   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Blood Advances, 2018
: Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie, Paul E. Monahan, Roshni Kulkarni, Barbara A. Konkle, Marshall A. Mazepa   +4 more
doaj   +1 more source

Bleeding-Coagulation System Disorders and Retina (Disseminated Intravascular Coagulopathy, Von Willebrand Disease, Vitamin K Deficiency, Hemophilia A and Hemophilia B)

, 2022
BOZKURT, Erdinç, LİMON, Utku, ÖZSOY SAYGIN, Işılay   +2 more
openalex   +1 more source

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source