Gene therapy may be the next major advance for treatment of many diseases, and severe haemophilia (an inherited deficiency of coagulation factor VIII or IX) is a useful model. Progress in gene therapy has been slowed down following fatal multi-organ failure during an adenovirus vector trial for ornithine-transcarbamylase deficiency and two episodes of leukaemia in a retroviral vector trial for ...

The advent of AIDS not only signifies the emergence of a new disease that affects hitherto healthy members of the general population but also highlights many unresolved problems of people with haemophilia, a life-long genetic disorder of blood clotting. Although HIV infection and the threat of AIDS has been a tragedy for people with haemophilia, the reported study shows that in spite of recent ...

Higher self-efficacy in chronic disease patients is associated with higher development of self-management skills and increased quality-of-life. Quantification and monitoring of self-efficacy is therefore of importance. Self-efficacy in haemophilia patients has received little attention due to lack of standardized scales. To validate the novel Haemophilia-specific Self-Efficacy Scale (HSES) in h...

Prophylactic treatment of haemophilia has been gaining acceptance as the optimal therapeutic option in an increasing number of haemophilia centres in the developed world in recent years. This paper focus on three aspects of prophylactic therapy: when to start treatment, venous access and the dose/dose interval. Evidence is in favour of prophylactic treatment to be started at an early age using ...

Haemophilia is the most frequent hereditary haemorrhagic illness and it is due to the deficiency of coagulation factors VIII (haemophilia A, HA) or IX (haemophilia B, HB). The prevalence of this disease varies according to the country, those having better survival rates having also higher prevalences. Specifically in Costa Rica, there are around 130 HA and 30 HB families. This study reports the...

BACKGROUND Ablation of the synovium with radiopharmaceuticals, referred to as radiation synovectomy, (RS), has emerged as a simple affordable and safe procedure that is highly effective in preventing chronic disabling end stage arthritis in haemophilia patients. OBJECTIVE To provide a review of the principles and role of radiation synovectomy, (RS), in the management of haemophiliac patients ...

Orthodontic treatment is fully consistent with general health and it also improves quality-of life along with psychological considerations. Haemophilia is a disorder in which due to deficiency of some coagulation factors. Hereditary deficiency of coagulation factors has caused 90% of hereditary diseases: Haemophilia A, Haemophilia B and von Willebrand’s. Two issues need to be considered in the ...

New Zealand Maoris (72 X chromosomes) have been compared with Pacific Island Polynesians (121 X chromosomes) and Caucasian New Zealanders (51 X chromosomes) as a control group to determine the allelic frequency of six RFLPs associated with the genes for two X linked diseases (haemophilia A and haemophilia B). RFLPs examined were BclI, XbaI, and BglI within the factor VIII gene, the factor VIII ...

Department of Haematology, University Hospital of Wales, School of Medicine, Cardiff, United Kingdom; The UK National Haemophilia Database, Manchester, United Kingdom; Department of Haematology, Royal Hospital for Sick Children, Glasgow, United Kingdom; The Haemophilia Centre, The Royal London Hospital, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, Unit...

INTRODUCTION Haemophilia is a bleeding disorder associated with significant pain, emotional distress, quality of life (QoL) impairment and considerable healthcare costs. Psychosocial health and effective pain management are considered essential end points for optimal haemophilia care, but there is a significant gap in evidence-based treatments targeting these outcomes in people with haemophilia...