Prophylactic treatment of haemophilia has been gaining acceptance as the optimal therapeutic option in an increasing number of haemophilia centres in the developed world in recent years. This paper focus on three aspects of prophylactic therapy: when to start treatment, venous access and the dose/dose interval. Evidence is in favour of prophylactic treatment to be started at an early age using either a peripheral vein with 1-2 injections per week and a successive increase in the frequency depending on the child and the veins, or, using a Port-A-Cath which allows a better prophylactic coverage by infusions preferably every second day in haemophilia A and every third day in haemophilia B.
Prophylaxis is the treatment of choice for children with severe haemophilia. As prophylaxis is especially important during the period of growth, the need for continued prophylaxis in adulthood should be considered. The aim of this study was to describe the incidence and outcome of stopping prophylaxis in patients with severe haemophilia who were offered prophylaxis during childhood. All patients with severe haemophilia (factor VIII/IX
Both Sweden and the Netherlands have a long experience with primary prophylaxis in children with severe haemophilia. In these countries it has been offered to all children for the last 3-4 decades. In Sweden prophylaxis is generally started at an earlier age with a higher dosage and frequency than in the Netherlands. Patients in the Netherlands receive a more individually tailored regimen, with prophylaxis now started after the first one or two joint bleeds and dosages are increased when breakthrough bleeds occur. The current study evaluated the effect prophylaxis on long-term outcomes and the consumption of clotting factor concentrates in Dutch and Swedish cohorts. Our results show that the orthopaedic outcome in the oldest groups of patients from Sweden and the Dutch cohorts were comparable, although the Swedish patients used twice as much clotting products per year. In the youngest patients, joint status is very good and further follow-up is necessary to demonstrate the benefits of either strategy. In conclusion, more individually tailored regimens aimed at preventing bleeding prevent joint damage in children with severe haemophilia, while clotting factor consumption is about half of that in previously described regimens.
Twelve children with a severe form of haemophilia A received a totally implantable venous access system (Port-A-Cath) to facilitate regular prophylactic treatment with factor VIII. The indication for implantation was difficulty in obtaining regular access to a peripheral vein. Postoperative bleeding around the portal site occurred in two of 12 cases. After a median duration of follow-up of 26 months (range 5-79 months), none of the systems had needed replacement due to bleeding, septicaemia or thrombosis. One child, with an inhibitor against factor VIII, had an infection at the portal site and this system was removed. None of the other children had any serious side effects. Nine of the 12 children's parents learned how to use the Port-A-Cath system, thus enabling optimal prophylactic home treatment with factor VIII to be begun early in life.
Arthropathy is associated with the greatest cost and morbidity to persons with haemophilia. Clinical protocols have been developed empirically to prevent or retard the development of joint disease using routine infusions of replacement factor concentrate. However, randomized clinical trials to determine optimal therapy to prevent joint disease in persons with severe haemophilia are lacking. Two clinical trials are ongoing to answer important clinical questions about the prevention of arthropathy. The first, a US randomized clinical trial, is comparing an aggressive multiple-infusion episode-based protocol to standard alternate-day prophylaxis to determine whether prevention of joint disease requires prevention of the bleeding event, per se, or can be achieved by promoting complete resolution of each bleeding event in the joint. This study included the development and validation of sensitive new physical and imaging scales to detect the earliest signs of joint disease in young children. The second, a single-arm, open-label Canadian study, is asking whether prevention of joint disease in young children can be individualized by escalating the dose and frequency of routine replacement infusions of factor concentrate based upon the clinical course of haemophilia in the affected child. Both of these studies will contribute valuable information regarding optimal therapy and will help establish evidence-based medicine for the management of severe haemophilia.
The purpose of this study was to determine whether rural providers have adequate preparation for safe and effective haemophilia care.
This qualitative study proceeded in two phases: focus group (phase I) and telephone (phase II) interviews.
Five Canadian rural hospitals served by one urban haemophilia treatment centre and providing service to at least one haemophilia family.
Phase I: focus groups of rural health professionals (site 1: n = 5; site 2: n = 6), including nursing, medicine and lab technology. Phase II: telephone interviews with nine participants from nursing, medicine, lab technology, social work and physiotherapy across three sites.
Qualitative content analysis yielded categorical themes for specialty care resource requirements in a rural context.
Resource needs reflected five main categories: communication network, subjective knowledge, team roles, objective knowledge and partnerships (C-STOP).
The five C-STOP categories require resources and alignment of urban specialist, rural provider and family expertise. Specialty clinic efforts promoting self-care are incomplete without matched resources for rural providers.
During the 1960s, it was reported from Sweden that haemophiliacs with factor levels above 1% rarely develop arthropathy. This observation suggested that severe haemophilia could be converted to a milder form by regular infusions with factor concentrate. After several earlier publications, a report was published in 1992 that detailed 25 years' experience with prophylaxis in 60 patients from the Malmö centre. The results showed that starting prophylaxis early in life with a dose regimen that would prevent factor VIII or IX plasma levels from falling below 1% could prevent the development of haemophilic arthropathy. Also, older age groups who had received less intensive treatment, and who started prophylaxis later in life, were still in a much better condition than historic controls. In the 1970s several small but well-controlled studies from the USA, Germany and Italy clearly showed the benefit of prophylaxis in reducing bleeding frequency. Early experience from the Netherlands was published in 1971. Since these early studies, the results have been corroborated from many countries and in a large multinational study. Although the benefits of prophylaxis seem unquestionable, several research questions remain to be better elucidated, such as when to start and when to stop, dosing and dose interval, and how to assess the long-term treatment effects. These issues are of great economic importance, and the need for health economical studies is obvious.