Haemophilia : the official journal of the World Federation of Hemophilia
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Multicenter Study
Use of Haemate(®) P as immune tolerance induction in patients with severe haemophilia A who failed previous induction attempts: a multicentre observational study.
Immune tolerance induction (ITI) can eliminate factor VIII (FVIII) inhibitory antibodies that appear during FVIII replacement therapy. If first-line ITI fails, switching to a different FVIII concentrate, especially one containing von Willebrand factor (VWF), has been advocated. The objective of the study was to assess the efficacy and safety of Haemate(®) P, a plasma-derived FVIII concentrate containing high levels of VWF, as ITI in severe haemophilia A patients who had failed at least one prior ITI attempt with a different FVIII concentrate. ⋯ Five patients experienced seven adverse events (AEs), including two serious AEs (sepsis). Haemate(®) P was discontinued due to an AE in one patient with a partial response. Haemate(®) P salvage ITI resulted in complete or partial tolerization in four of nine patients (44%) who had failed previous ITI attempts using different FVIII concentrates.
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Effects of desmopressin (DDAVP) in platelet disorders and primary haemostasis cannot be attributed solely to the increase in FVIII/VWF (von Willebrand factor), as VWF/FVIII concentrates have no effect in these circumstances. Microparticles (MP) can support haemostasis by expression of phospholipids, tissue factor and VWF on their surface. We hypothesized that significant amounts of VWF are bound to MP after DDAVP administration and that consequently depletion of MP should influence VWF:Ag and VWF:RCo plasma levels. ⋯ MP depletion by magnetic bead selection led to a significant reduction in VWF:Ag (-18.0%) and VWF:RCo (-27.7%) plasma levels without changes in VWF multimer composition. As results were similar for DDAVP control subjects, the amount of VWF bound to circulating microparticles was significantly higher after DDAVP administration compared with healthy controls (reduction -11.7%). DDAVP leads to a release of microparticles and increases the amount of VWF bound to microparticles which might explain the clinical efficacy of DDAVP in platelet disorders.
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Data on the health-related quality of life (HRQoL) of congenital haemophilia patients with inhibitors (CHwI) and their caregivers are limited. To understand the association between patient demo-graphics/clinical characteristics with HRQoL among CHwI patients and caregivers, a survey was developed to assess HRQoL with haemophilia-specific QoL questionnaires (HAEMO-QoL/HAEM-A-QoL). In the cross-sectional study, paper-pencil questionnaires were mailed to 261 US CHwI patients/caregivers in July 2010. ⋯ Within ChwI patients, HRQoL impairments increased with age and existed across a range of physical/psychosocial domains. In addition, caregiver burden also affected the perceived HRQoL of paediatric CHwI patients. Additional research is considered necessary to further understand the support caregivers need while caring for children with CHwI.
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Currently, haemophilia care aims to provide the best possible quality of life for individuals living with this chronic disease. Many factors are known to influence treatment adherence, including treatment satisfaction. Health-related quality of life (HRQoL) and treatment satisfaction are, therefore, important outcomes in clinical trials and clinical practice. ⋯ Equivalent concepts for 22 items that were difficult to adapt culturally for particular languages were identified and classed as semantic/conceptual (17 items), cultural (three items), idiomatic (one item), and grammatical (one item) problems. This has resulted in linguistically validated versions of these instruments, which can be used to assess HRQoL and treatment satisfaction in clinical trials and clinical practice. They will provide new insights into areas of haemophilia that remain poorly understood today.
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Congenital haemophilia is a rare and complex condition for which dedicated specialized and comprehensive care has produced measurable improvements in clinical outcomes and advances in patient management. Among these advances is the ability to safely perform surgery in patients with inhibitor antibodies to factors VIII and IX, in whom all but the most necessary of surgeries were once avoided due to the risk for uncontrollable bleeding due to ineffectiveness of replacement therapy. ⋯ Coordinated, standard pre-, intra- and postoperative assessments and planning are intended to optimize surgical outcome and utilization of resources, including costly factor concentrates and other haemostatic agents, while minimizing the risk for bleeding and other adverse consequences both during and after surgery. This article will review the special considerations for patients with inhibitors as they prepare for and move through surgery and recovery, with an emphasis on the roles and responsibilities of individual members of the multidisciplinary team in facilitating this process.