Rationale for the use of high dose rFVIIa in a high‐titre inhibitor patient with haemophilia B during major orthopaedic proceduresHaemophilia - Tập 7 Số 5 - Trang 517-522 - 2001
Herbert Cooper, Connie P. Jones, Edmund Campion, Barbara A. Konkle, Ulla Hedner
Inhibitor development is a serious complication in patients with haemophilia A and B. Historically, a lack of optimal therapies and factor products for treating inhibitor patients resulted in many patients developing chronic haemophilic arthropathies and flexion contractures of the involved joints. The introduction of immune‐tolerance protocols to eradicate high‐titre inhibitors has greatl...... hiện toàn bộ
Enhancement of factor VIIa haemostatic efficacy by formulation with PEGylated liposomesHaemophilia - Tập 14 Số 3 - Trang 476-483 - 2008
Rivka Yatuv, I. DAYAN, Lea Carmel-Goren, Micah Robinson, Irit Aviv, Mirela Goldenberg-Furmanov, Moshe Baru
Summary. Recombinant activated factor VII (rFVIIa) is an effective treatment of the haemophilia patient with inhibitors and acquired haemophilia. However, on account of its relatively short half‐life (HL), achieving therapeutic efficacy with FVIIa requires repeated injections. The development of a long‐acting FVIIa product would therefore be beneficial. The formulat...... hiện toàn bộ
Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention’s (CDC) Universal Data Collection (UDC) projectHaemophilia - Tập 15 Số 6 - Trang 1281-1290 - 2009
Roshni Kulkarni, J. Michael Soucie, Jeanne M. Lusher, Rodney Presley, Amy D. Shapiro, M. John Gill, Marilyn J. Manco‐Johnson, Marion A. Koerper, Prasad Mathew, Thomas C. Abshire, Donna DiMichele, Keith Hoots, Robert L. Janco, Diane J. Nugent, S. Geraghty, Bruce L. Evatt
Summary. Lack of detailed natural history and outcomes data for neonates and toddlers with haemophilia hampers the provision of optimal management of the disorder. We report an analysis of prospective data collected from 580 neonates and toddlers aged 0–2 years with haemophilia enrolled in the Universal Data Collection (UDC) surveillance project of the Centers for D...... hiện toàn bộ
Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long‐terminal repeatsHaemophilia - Tập 9 Số 1 - Trang 94-103 - 2003
An Van Damme, Marinee Chuah, Francesco Dell’Accio, Cosimo De Bari, Frank P. Luyten, Peter Carmeliet, Thierry VandenDriessche
Summary. Bone marrow (BM) cells are attractive target cells for ex vivo gene therapy of genetic diseases, including haemophilia A. However, BM‐derived haematopoietic stem/progenitor cells (HSCs) transduced with factor VIII (FVIII) retroviral vectors, failed to express FVIII in vivo. To overcome the limitations of ...... hiện toàn bộ
Rare coagulation deficienciesHaemophilia - Tập 8 Số 3 - Trang 308-321 - 2002
Flora Peyvandi, Stefano Duga, Sepideh Akhavan, Pier Mannuccio Mannucci
Summary. Deficiencies of coagulation factors (other than factor VIII and factor IX) that cause a bleeding disorder are inherited as autosomal recessive traits and are generally rare, with prevalences in the general population varying between 1 : 500 000 and 1 : 2 000 000. In the last few years, the number of patients with recessively transmitted coagulation deficien...... hiện toàn bộ