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Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany
Background and ObjectiveHaemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads...
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Bleeding pattern and consumption of factor VIII concentrate in adult patients with haemophilia A without inhibitors in the Czech Republic between 2013 and 2021 (Czech National Haemophilia Programme registry data)
The manuscript provides an overview of treatment and its changes in adult patients with haemophilia A without inhibitors in the Czech Republic...
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Eftrenonacog Alfa: A Review in Haemophilia B
Eftrenonacog alfa (Alprolix ® ) is an extended half-life recombinant factor IX (rFIX)-Fc fusion protein (hereafter referred to as rFIXFc). Administered...
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Health-related quality of life, direct medical and societal costs among children with moderate or severe haemophilia in Europe: multivariable models of the CHESS-PAEDs study
BackgroundHaemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of...
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Differential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study
BackgroundThe lifelong nature of haemophilia makes patient-centred and societal assessments of its impact important to clinical and policy decisions....
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Barriers to prophylactic treatment among patients with haemophilia A in Shandong Province, China: a qualitative study
BackgroundHaemophilia A is a rare, hereditary haemorrhagic disease that manifests as induced spontaneous bleeding and leads to disability or...
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Decline in health-related quality of life and foot and ankle patient reported outcomes measures in patients with haemophilia and ankle haemarthropathy
BackgroundHaemophilia is an X-linked recessive genetic disorder characterised by bleeding within soft tissue and joints. The ankle is...
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Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors
IntroductionRecombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement...
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Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe
BackgroundReal-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL)...
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Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study
BackgroundGene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment...
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Risk of low bone mineral density in patients with haemophilia: a systematic review and meta-analysis
IntroductionPatients with haemophilia (PWH) may have lower bone mineral density (BMD). The risk of low BMD in PWH has not been comprehensively...
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A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient
BackgroundAdeno-associated virus (AAV)-based gene therapy for haemophilia has advanced substantially in the last 13 years; recently, three products...
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Intra-articular injection of tranexamic acid in patients with haemophilia arthritis: retrospective controlled study in total knee arthroplasty
PurposeTotal knee arthroplasty is the main method for the treatment of advanced haemophilic knee arthritis. Due to the particularity of hemophilia,...
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Incidence and treatment-related risk factors of inhibitor development after intensive FVIII replacement for major orthopaedic surgery in previous treated haemophilia A
IntroductionHaemophilia A (HA) is an X-linked recessive bleeding disorder caused by lack or deficiency of coagulation factor VIII.
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Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries
IntroductionThe current study describes real-world clinical outcomes and factor usage among patients with haemophilia B switching from standard...
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Current status and future prospects of activated recombinant coagulation factor VIIa, NovoSeven®, in the treatment of haemophilia and rare bleeding disorders
rFVIIa, a human recombinant activated coagulation factor VII, has been used worldwide for more than two decades for the treatment of bleeding...
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The impact of bleeding event frequency on health-related quality of life and work productivity outcomes in a European cohort of adults with haemophilia A: insights from the CHESS II study
BackgroundHaemophilia A carries a substantial healthcare burden, affecting health-related quality of life (HRQoL). The Cost of Haemophilia in Men: a...
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Severe haemophilia A in a preterm girl with Turner syndrome: case report – a diagnostic and therapeutic challenge for a paediatrician (Part 2)
BackgroundHaemophilia A is an X-linked genetic condition which manifests itself mainly in male children in the first 2 years of life, during gross...
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Haemophilia
Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX),...
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Iliopsoas haemorrhage complicated by femoral neuropathy in patients with haemophilia: a case series report
ObjectivesThe study aimed to determine the incidence of femoral neuropathy in patients with haemophilia exhibiting iliopsoas haemorrhage.
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