Experience of prophylaxis treatment in children with severe haemophilia

Abstract

The practice of prophylactic treatment of boys with severe haemophilia has been evaluated in our centre. Prophylaxis was started at the median age of 3.7 years (range 0.4-12.7 years) in 38/41 children (93%) under 17 years of age. Median follow-up was 4.1 years (range 0.4-12.7 years). The criteria of primary prophylaxis according to the definition by the European Paediatric Network of Haemophilia Management was fulfilled by 9/38 (24%). Although a majority [76%, 29/38] of the children started prophylaxis after a median number of joint bleeds of 3.5, 70% of the children in this group had clinical joint scores of 0. Intravenous catheter insertion was required at a median age of 15.5 months (range 5-36 months) in 21% of the children, resulting in a catheter infection rate of 1.74 per 1000 catheter days. None developed an inhibitor on prophylaxis and three patients who had low-titre inhibitors (< 5 Bethesda units) prior to prophylaxis had undetectable inhibitors after prophylaxis. The home-treatment training programme required considerable time and cost. As a result, 87% of the children used peripheral venous access and hospital visits declined as prophylaxis became established. Parents' incentives for prophylaxis were that the children undertook many physical activities and sports previously not recommended, there was less parental anxiety and an overall improvement in the quality of life for the whole family.

Assessment of Treatment Practice Patterns for Severe Hemophilia A: A Global Nurse Perspective

“…After 12–30 months of follow-up, every-other-day prophylaxis was shown to significantly reduce the incidence of joint hemorrhage and the relative risk for joint damage compared with enhanced episodic therapy. While factor consumption was significantly higher in the prophylaxis group, potential savings may be realized from fewer complications and a reduced need for orthopedic surgery [6, 21, 22]. These compelling results, coupled with previous observational studies and case reports [4,5,6,7,8,9,10,11], not only establish prophylaxis as optimal therapy for patients with severe hemophilia A, but argue for its use as standard of care.…”

“…CVAD implantation makes home-administered prophylaxis possible, but safe management of these devices requires close cooperation between families and HTCs. Extensive patient and family education is imperative and should be reinforced on a continuing basis [22]. Caregivers should receive thorough instruction about the use of CVADs and should also be educated about the signs of complications.…”

Cost effectiveness of haemophilia treatment: a cross-national assessment

“…1). Other studies have also demonstrated significant improvements in outcomes with the use of prophylactic treatment [3,4,8,[10][11][12][22][23][24].…”

Eltern von chronisch kranken Kindern

“…Dies ist vermutlich darauf zurückzuführen, dass aufgrund medizinischer Fortschritte in der Behandlung dieser Erkrankung inzwischen eine prophylaktische Therapie durchgeführt und auf diese Weise Blutungen vorgebeugt werden kann [29]. Dies wird von den Eltern als sehr entlastend wahrgenommen und geht mit einer deutlichen Verringerung der krankheitsbezogenen Einschränkungen der betroffenen Kinder einher [21,30].…”