Results 71 to 80 of about 48,361 (234)

HAEMFIX: Impact of Switching From SHL‐FIX to EHL‐FIX in Patients With Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia B is an X‐linked recessive bleeding disorder caused by coagulation factor IX (FIX) deficiency. Treatment involves intravenous replacement of FIX. Recently, extended half‐life (EHL) FIX products have been introduced alongside standard half‐life (SHL) products to optimize therapy. Aim This study evaluated bleeding rates,
Jasmin Lonardi, Susan Halimeh, Sylvia von Mackensen, Lisa Kleinlein, Juliet Fleischer, Henri Funk, Julia Hölz, Johannes Holzapfel, Sabrina Juranek, Victoria Lieftüchter, Christoph Bidlingmaier, Martin Olivieri   +11 more
wiley   +1 more source

Real‐world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center

Pediatric Investigation
Importance Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China. Objective To explore the possibility of using reduced‐dosage EMI in Chinese HA children.
Qianqian Mao, Zhenping Chen, Guoqing Liu, Gang Li, Yingzi Zhen, Xiaoling Cheng, Zekun Li, Wanru Yao, Di Ai, Zhengping Li, Nan Wang, Man‐Chiu Poon, Runhui Wu   +12 more
doaj   +1 more source

Molecular Aggregation of Marketed Recombinant FVIII Products: Biochemical Evidence and Functional Effects

TH Open, 2019
Background Recombinant (rec-) coagulation factor VIII concentrates available for hemophilia A (HA) treatment differ in cell line production and structure, which could affect their pharmacodynamics and immunogenicity.
Raimondo De Cristofaro, Monica Sacco, Stefano Lancellotti, Federico Berruti, Isabella Garagiola, Carla Valsecchi, Maria Basso, Enrico Di Stasio, Flora Peyvandi   +8 more
doaj   +1 more source

Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice [PDF]

, 2007
Denise E. Sabatino, Tippi C. MacKenzie, William H. Peranteau, Shyrie Edmonson, Cesare Campagnoli, Yilin Liu, Alan W. Flake, Katherine A. High   +7 more
openalex   +1 more source

Elucidating the Molecular Basis in a Cohort of Patients With Combined Bleeding Tendencies and Joint Hypermobility Manifestations

Haemophilia, EarlyView.
ABSTRACT Background In patients with unexplained bleeding and normal haemostatic parameters, heritable disorders of connective tissue (HDCT) may be an underlying cause due to vascular fragility, as observed in Ehlers–Danlos syndrome (EDS) or Marfan syndrome (MS). This study aims to investigate the molecular profile of patients with joint hypermobility (
Perla Bandini, Nina Borràs, Carme Altisent, Judith Sánchez‐Raya, Laura Martin‐Fernandez, Iris Garcia‐Martínez, Natàlia Comes, Lorena Ramírez, Marta Martorell, Carlos Hobeich, Francisco Vidal, Irene Corrales   +11 more
wiley   +1 more source

Improving access to hemophilia care in sub-Saharan Africa by capacity building [PDF]

, 2019
Saliou Diop, Assad Haffar, Johnny Mahlangu, Irene Chami, Steve Kitchen, Glenn F. Pierce   +5 more
openalex   +1 more source

Safety, Efficacy and Treatment Patterns of rIX‐FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan

Haemophilia, EarlyView.
Abstract Background Limited real‐world data exist on recombinant fusion protein‐linking coagulation factor IX (FIX) with albumin (rIX‐FP) in paediatric previously untreated patients (PUPs) with haemophilia B, particularly in infants. Aims To evaluate the treatment patterns, safety and efficacy of rIX‐FP in paediatric PUPs in Japan.
Keiji Nogami, Chiai Nagae, Masatoshi Yono, Naoki Terasaka   +3 more
wiley   +1 more source

Canine Hemophilia and Hemopoietic Grafting [PDF]

, 1972
Rainer Storb, Thomas L. Marchioro, Theodore Graham, Mary Willemin, Cecil Hougie, E. Donnall Thomas   +5 more
openalex   +1 more source

PB1233 A Single-Center Study of Reduced-Dose Corticosteroids Therapy for Patients with Acquired Hemophilia A

, 2023
Yoshiyuki Ogawa, Kunio Yanagisawa, Akira Matsumoto, Mikiya Kajita, Yuri Miyazawa, Noriyuki Kobayashi, Tsukasa Osaki, Masayoshi Souri, Akitada Ichinose, Hiroshi Handa   +9 more
openalex   +1 more source

Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors

Haemophilia, EarlyView.
ABSTRACT Introduction The bispecific monoclonal antibody emicizumab was approved for prophylactic treatment of congenital haemophilia A (HA) in Japan in 2018. Aim To monitor long‐term safety and effectiveness of emicizumab, including appropriate concomitant use of bypassing agents (BPAs), in Japanese patients with congenital HA with inhibitors who ...
Midori Shima, Katsuyuki Fukutake, Masanori Matsumoto, Yoshihiko Sugimura, Chiaki Sugita, Lyu Ji, Mika Kawano, Tadashi Matsushita   +7 more
wiley   +1 more source