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Nine areas with outstanding challenges for hemophilia B research [PDF]

Therapeutic Advances in Hematology
Hemophilia A and B are rare, X-linked bleeding disorders characterized by deficiencies in coagulation factor VIII (FVIII) and factor IX (FIX), respectively. Numerous advances have helped to reduce disease burden.
Jan Astermark, Cédric Hermans, Peter J. Lenting, James S. O’Donnell,   +4 more
doaj   +2 more sources

Cardiac surgery in an infant hemophilia B carrier with moderate hemophilia: a case report [PDF]

Frontiers in Cardiovascular Medicine
IntroductionThis is the first reported case of a female infant with moderate hemophilia B undergoing complex cardiac surgery, highlighting challenges in managing bleeding disorders in this rare clinical setting.Patient concerns and clinical findingsA 4 ...
Julia Hölz, Chiara Nobile, Johannes Holzapfel, Victoria Lieftüchter, Nicole De Winkel, Martin Olivieri   +5 more
doaj   +2 more sources

Assessing the health-state utility values of rare disease-hemophilia B using EQ-5D-5L: a study based on the Chinese population [PDF]

Orphanet Journal of Rare Diseases
Background Obtaining health-state utility values (HSUVs) aids in making scientific decisions in patient health management, especially for rare disease patients.
Chuchuan Wan, Haotao Li, Yulin Zhang, Qiqi Wang, Yiwen Huang, Tao Guan, Xiaoyu Xi   +6 more
doaj   +2 more sources

Hemophilia B

The bleeding disorder known as hemophilia B (HB) is caused by a deficiency or abnormality in the blood clotting factor IX (FIX) gene, which is inherited in an X-linked manner. This disease results from one of more than 1000 classified pathogenic variations in the FIX gene F9, and genetic missense and frameshift changes predominate.
Alshaikhli A, Killeen RB, Rokkam VR.
europepmc   +2 more sources

Medical costs and hospital utilization for hemophilia A and B urban inpatients in China: a national cross-sectional study

BMC Health Services Research, 2022
Background Hemophilia care in mainland China has been greatly improved since the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC), and most of drugs for hemophilia have been covered by basic medical insurance ...
Zhengwei Huang, Stephen Nicholas, Yong Yang, Xiaoping Chen, Elizabeth Maitland, Yong Ma, Xuefeng Shi   +6 more
doaj   +1 more source

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort

Research and Practice in Thrombosis and Haemostasis, 2022
Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce, Izabela James, Savita Rangarajan, Nicola Curry, Catherine Bagot, Steven Austin, Mike Laffan, Sarah Mangles, Kandiah Chandrakumaran, Carina Mundy   +9 more
doaj   +1 more source

CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs

Haematologica, 2020
Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes.
Jiahuan Chen, Beiying An, Biao Yu, Xiaohuan Peng, Hongming Yuan, Qiangbing Yang, Xue Chen, Tingting Yu, Lingyu Wang, Xinwei Zhang, He Wang, Xiaodong Zou, Daxin Pang, Hongsheng Ouyang, Xiaochun Tang   +14 more
doaj   +1 more source

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

Haematologica, 2016
Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre, Silvia Linari, Mirko Manetti, Eloisa Romano, Francesco Sofi, Marco Matucci-Cerinic, Christian Carulli, Massimo Innocenti, Lidia Ibba-Manneschi, Giancarlo Castaman   +9 more
doaj   +1 more source

Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]

, 2017
The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo, Liumbruno, Giancarlo Maria, Marano, Giuseppe, Mengoli, Carlo, Piccinini, Vanessa, Pupella, Simonetta, Vaglio, Stefania   +6 more
core   +1 more source