Haemophilia : the official journal of the World Federation of Hemophilia
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FEIBA (factor eight inhibitor by-passing activity) is used to achieve haemostasis in haemophiliacs with inhibitor. The aim of this study was to evaluate efficacy and consumption of the product in treatment of haemorrhages in haemophiliacs with factor VIII inhibitor, and determine factors that can influence the results of treatment. We used data from our haemophilia centre from years 2000-2008. ⋯ The cumulative dose of FEIBA was lower for three episodes with re-bleeding: median 96 U kg(-1) but not in the two cases of ineffective treatment: 361 U kg(-1). FEIBA in management of bleeding episodes completely resolved the haemorrhage in 91.8% of events and in a further 4.9% if treatment was restarted. Using home treatment saved expenditure due to the lower cumulative dose needed for treatment of haemorrhage.
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Hepatitis A and B vaccines are highly effective tools that can greatly reduce infection risk in the bleeding disorder population. Although hepatitis A and B immunization for individuals with bleeding disorders is universally recommended, various advisory bodies often differ with respect to many practical aspects of vaccination. To review the published literature and guidelines and form a practical, comprehensive and consistent approach to hepatitis A and B immunization for individuals with bleeding disorders. ⋯ Published literature addressing immunization in bleeding disorder patients is sparse and mostly examines route of vaccine administration, complications and corresponding antibody response. Although the risk of hepatitis A and B infection is low, the use of simple measures such as vaccination is reasonable and advocated by haemophilia advisory bodies. Following our review of the available literature and North American guidelines, we have developed comprehensive and practical recommendations addressing hepatitis A and B immunization for the bleeding disorder population that may be applicable in Bleeding Disorder clinics.
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Although children with haemophilia are advised to participate in physical activities, their physical fitness has not been studied in a large group. In addition, children with haemophilia may be at increased risk for becoming overweight as a result of inactivity because of joint bleedings or because of overprotection. This study aimed to assess physical fitness (aerobic capacity), joint status, muscle strength, quality of life (QoL), self-reported motor competence and also prevalence of overweight and its association with physical parameters. ⋯ Dutch children with haemophilia have normal aerobic exercise capacity and muscle strength. The majority also has normal joint mobility. Prevalence of overweight is slightly increased.
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Multicenter Study
Cost and effectiveness of treatments for mild-to-moderate bleeding episodes in haemophilia patients with inhibitors in Korea.
First-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Korea is currently activated prothrombin complex concentrate (aPCC) with recombinant activated factor VII (rFVIIa) as second-line therapy or as a last resort. The aim of this study was to estimate the cost and effectiveness of aPCC vs. rFVIIa for treating mild-to-moderate bleeds in inhibitor patients from the Korean reimbursement authorities' perspective. Clinical outcomes and resource utilization data (number of doses, average dose, number of outpatient visits, inpatient stays, ambulance transport and concomitant medications) were collected from an observational study involving four Korean paediatric haemophilia centres. ⋯ Mean total direct medical costs from bleed initiation to cessation were estimated at Korean Won (KRW)12 460 thousand (US$12 311) for rFVIIa given as first-line therapy and KRW18 304 thousand (US$18 085) for aPCC given as first-line therapy. Sensitivity analyses confirmed the cost-effectiveness of rFVIIa vs. aPCC given as first-line therapy. In Korea, use of rFVIIa as first-line therapy for treatment of mild-to-moderate bleeding episodes in inhibitor patients is both clinically effective and cost-effective compared with initial aPCC treatment. rFVIIa should be considered as the first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Korea.
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Multicenter Study
Variability in bleeding phenotype in Amish carriers of haemophilia B with the 31008 C-->T mutation.
The aim of this study was to characterize the variability of bleeding phenotype and its association with plasma factor IX coagulant activity (FIX:C) in haemophilia B carriers in a large Amish pedigree with a unifying genetic mutation, C-to-T transition at base 31008 of the factor IX gene (Xq27.1-27.2). A cross-sectional survey of haemophilia B carriers included a multiple choice questionnaire evaluating symptoms of mucocutaneous bleeding, joint bleeding and bleeding after haemostatic stress [menstruation, postpartum haemorrhage (PPH), dental extractions and invasive surgeries]. Severity of bleeding was graded as 0 to 4, 0 being no bleeding whereas 4 being severe bleeding. ⋯ Subcutaneous haematoma formation and bleeding after haemostatic stress requiring treatment were associated with bleeding scores >/=3. Phenotypic variability existed among the carriers of haemophilia B who belonged to a single pedigree carrying a single unifying mutation. The utility of bleeding scores to define bleeding phenotype precisely in haemophilia B carriers needs further evaluation.