Pneumococcal pneumonia remains a clear unmet medical need for adults worldwide. Despite advances in vaccine technology, vaccination coverage remains low, putting many people at risk of significant morbidity and mortality. The herd effect seen with paediatric vaccination is not enough to protect all older and vulnerable people in the community, and more needs to be done to increase the uptake of pneumococcal vaccination in adults. Several key groups are at increased risk of contracting pneumococcal pneumonia, and eligible patients are being missed in clinical practice. At present, community-acquired pneumonia costs over €10 billion annually in Europe alone. Pneumococcal conjugate vaccination could translate into preventing 200,000 cases of community-acquired pneumonia every year in Europe alone. This group calls on governments and decision makers to implement consistent age-based vaccination strategies, and for healthcare professionals in daily clinical practice to identify eligible patients who would benefit from vaccination strategies.
Introduction
Eptacog beta is a new recombinant activated human factor VII bypassing agent approved in the United States for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors 12 years of age or older.
Aim
To prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children <12 years of age with haemophilia A or B with inhibitors.
Methods
Using a randomised crossover design, subjects received initial doses of 75 or 225 μg/kg eptacog beta followed by 75 μg/kg dosing at predefined intervals (as determined by clinical response) to treat bleeding episodes (BEs). Treatment success criteria included a haemostasis evaluation of ‘excellent’ or ‘good’ without use of additional eptacog beta, alternative haemostatic agent or blood product, and no increase in pain following the first ‘excellent’ or ‘good’ assessment.
Results
Treatment success proportions in 25 subjects (1–11 years) who experienced 546 mild or moderate BEs were 65% in the 75 μg/kg initial dose regimen (IDR) and 60% in the 225 μg/kg IDR 12 h following initial eptacog beta infusion. By 24 h, the treatment success proportions were 97% for the 75 μg/kg IDR and 98% for the 225 μg/kg IDR. No thrombotic events, allergic reactions, neutralising antibodies or treatment‐related adverse events were reported.
Conclusion
Both 75 and 225 μg/kg eptacog beta IDRs provided safe and effective treatment and control of bleeding in children <12 years of age.
A 3 2 3 -A 6 3 6 at local level; inadequate data collection and follow-up process at later stages of life; poor awareness of immunisation schedule by health care practitioners (HCPs); lack of structural and operational policies to promote and deliver vaccination at later stages of life; infrequent interactions with the health care system during adult-hood; limited and inconsistent information dissemination by HCPs and government; negative messages on vaccination through media; complacent public attitude towards the risk posed by vaccine preventable diseases. ConClusions: Three main domains of barriers exist: low institutional facilitation to recommend lifespan immunisation as part of NIP; inadequate mechanisms at regional and local level to facilitate citizen access to immunisation / vaccination; low patient demand for immunisation at older stages of life. To overcome these barriers a broad-ranging approach based on awareness is required, which includes providing comprehensive pharmacoeconomic evidence to policy makers and payers, developing information tracking systems that enable follow up, and HCP education to facilitate information dissemination.
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