Radiological Score in Paediatric Haemophilic Patients with Early and Late Onset of Factor VIII - Prophylaxis

Abstract

Dear Sir, Osteoathropathic alteration is a major cause of morbidity in patients with severe haemophilia. During infancy and adolescence, prophylaxis is undoubtedly preferable to on-demand therapy, but as yet poor data exist comparing the onset of prophylactic treatment at an early age with treatment initiated at a more advanced age (1,2,3). In order to determine which therapy regime is needed to prevent haemophilic arthropathy we prospectively compared 17 patients with moderate and severe haemophilia A (age: 3-14 years; mean age: 6 2/12 years). All 17 patients were treated with factor VIII (30-40 IU/kg bw/three times per week; Humate P®, Beriate HS®, Behring, Germany). Prior to radiological and clinical examinations of the knee, elbow and ankle joints patients were subdivided into two groups according to therapy regimes. In group 1 prophylactic treatment was initiated in

Haemophilia prophylaxis in young patients–a long‐term follow‐up

“…Only in patients with adequate prophylaxis did joint scores remain at zero, or very low levels. In a recent German publication the authors concluded that prophylaxis is undoubtedly preferable to on-demand treatment during infancy and adolescence and not only patients with severe haemophilia but also those with moderate disease may benefit from early prophylaxis [8]. Our investigation, a 5-year review of the 34 youngest patients from the aforementioned series [2], yielded further evidence in favour of prophylactic treatment.…”

Total hip replacement in patients with severe bleeding disorders

“…Prophylactic replacement factor treatment, which has to start in early childhood, reduces the severity of spontaneous bleeding significantly [1,7,20] and can prevent or at least delay joint destruction [5]. The development of purified clotting factor concentrates, and the increased use of radiosynovectomy are two factors in current improvements in treatment leading to a significant reduction of the frequency of haemarthrosis [15].…”

The impact of inhibitors on the cost of clotting factor replacement therapy in haemophilia A in Canada

“…It should also be emphasized that the introduction of new therapeutic approaches will change the economic analysis. For example, with the demonstration of the efficacy of long‐term prophylactic therapy of severe haemophilia [ 12–14], wider use of these costly regimens will increase the cost of treating noninhibitor patients [ 15, 16]. More detailed analyses should also include other direct and indirect health care costs of delivering health care to people with haemophilia, as factor concentrates comprise only the most obvious, albeit the greatest, of these costs [ 15].…”