Haemophilia : the official journal of the World Federation of Hemophilia
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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.
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Review
More than a decade of international experience with a pdFVIII/VWF concentrate in immune tolerance.
Approximately 20-30% of patients with severe haemophilia A develop alloantibodies ('inhibitors') to infused FVIII rendering use of such replacement therapy ineffective. Once an inhibitor emerges, immune tolerance induction (ITI) is the standard treatment. ITI involves giving regular doses of FVIII concentrate to eradicate the inhibitor and achieve immunogenic acceptance of administered FVIII. ⋯ However, more than half the patients in the US cohort receiving rescue ITI achieved success (33% complete success; 20% partial success). These results should encourage clinicians to consider the use of pdFVIII/VWF concentrates for rescue ITI. Published outcomes data from the total global G-ITI cohort (95 patients) are awaited with anticipation.
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Little is known about the impact of the recent US economic downturn and health care reform on patient, caregiver and health care provider (HCP) decision-making for haemophilia A. To explore the impact of the recent economic downturn and perceived impact of health care reform on haemophilia A treatment decisions from patient, caregiver and HCP perspectives. Patients/caregivers and HCPs completed a self-administered survey in 2011. ⋯ Both patients/caregivers and HCPs reported that the economic downturn had a negative impact on haemophilia A treatment. Suboptimal treatment modifications were made due to the economic downturn. Health care reform, especially the elimination of lifetime caps, was perceived as positive for haemophilia A treatment and as a potential avenue for contributing to more optimal treatment behaviours.