[French guidelines. Long-term prophylaxis for severe haemophilia A and B children to prevent haemophiliac arthropathy]. / Prophylaxie de longue durée chez les enfants hémophiles A et B sévères en pévention de l'arthropathie hémophilique.

During the last decades, long-term prophylaxis has become the gold standard for the treatment of children with severe haemophilia A or B. Prophylactic replacement regimens modify the natural history of the disease by aiming at the prevention of haemarthrosis, target joints and arthropathy. This treatment represents a constraint and an enhanced exposure to anti-haemophilic concentrates, which means potential increase of related risks and significant additional cost. The context of crisis of confidence due to the blood borne infections in the 1980s, may have delayed prophylaxis as an universal gold standard.In the early 2000s, the French group CoMETH proposed recommendations based on the review of the international experience. At first, specific guidelines of long-term prophylaxis were dedicated to children with severe haemophilia A or B, aged 3 years or less, with no history of target joint or arthropathy. The main concerns of this regimen consist in the early start and the escalating intensification of the treatment. In the French haemophilia care centres, the diffusion of these guidelines has apparently induced a significant turning point in therapeutic practices for haemophilia children. In 2006, more comprehensive recommendations were diffused to take into account all the children with severe haemophilia, whatever the bleeding history and joint status. The analysis of their impact, jointly with the National cohort "France Coag Network", will first assess the widespread implementation of the recommendations and the observance of the prophylactic regimen and identify factors associated to the compliance.

The role of the physiatrist in the haemophilia comprehensive care team in different parts of the world.

The role of the physiatrist, as a member of the multidisciplinary haemophilia comprehensive care team, is to prevent and treat activity limitations and restriction of participation on the part of the patient. . This role is threefold: (i) provide education to the patients, families and healthcare providers to detect disabling injuries and take adequate precautions; (ii) provide specialized treatment of musculoskeletal disabilities striving for the highest level of functionality and (iii) Stimulate the patient's full participation in socio-economic activities of his country. Prominent physiatrists from different parts of the world describe the situation in Armenia, China, Egypt, France, Indonesia (and the Philippines), the Netherlands and South America.

Management of haemophilic arthropathy.

Despite the tremendous benefit offered by primary prophylaxis, recurrent joint bleeding with progression to chronic synovitis and haemophilic arthropathy is still a daily concern for the multidisciplinary health care teams managing patients with severe haemophilia or haemophilia complicated by inhibitor development. Advanced stages of arthropathy could be prevented by regular assessment of musculoskeletal status and thus early detection of symptoms, daily rehabilitation exercises at home, and implementation of appropriate physiotherapy and medical training. Patient's education and psychological counselling are crucial. New tools such as magnetic resonance imaging are promising for the monitoring of these patients and might promote early detection of arthropathy and thus appropriate preventive measures to avoid further joint deterioration can be implemented. Medical synovectomy such as radionucleide synoviorthesis is a simple and non-invasive procedure that often delays the need for surgery which despite considerable improvement in techniques and postoperative rehabilitation remains a high-risk strategy in patients with severe haemophilia, especially those with inhibitors. In these high risk patients, availability of specific clotting factors such as activated prothrombin complex concentrate (FEIBA, Baxter, Vienna, Austria) and more recently, recombinant factor VIIa (rFVIIa, NovoSeven, Bagsvaerd, Denmark) has allowed to perform effective and safe orthopaedic procedures. The on-going EUREKA study will undoubtedly provide additional information about the optimal use of rFVIIa in this context.

Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. The French Study Group. secretariat.haemophiles@cch.ap-hop-paris.fr.

One hundred and 16 patients contributed to an analysis of the impact of the consequences of severe haemophilia A or B (factor levels < 2%) on orthopaedic status, resources consumed in relation to this status and resultant cost, and quality of life as perceived by the patient, using the MOS 36-Item-Short-Form Health Survey (SF-36). This French cross-sectional study involved outpatients regularly attending a haemophilia treatment centre. Data were collected retrospectively over a period of 1 year by the physician of the haemophilia treatment centre. Patients had a mean age of 23, and consisted of 50% students, 25% salaried workers, 17.2% with no professional activity and 7.8% physically impaired; 82.8% of them had type A haemophilia. Mean pain score was 2.5 per patient for the six main joints; 7.7 for the clinical score and 18.8 for the radiological score, with a mean number of bleeds of 16.3 per year per patient. During the year prior to inclusion, and because of their orthopaedic status, 22.4% of patients were hospitalized, 76.7% attended for an outpatient visit and 76.7% required at least one special investigation; 97.4% received replacement therapy, 41.4% required treatment for joint pain and 42.2% orthopaedic equipment. The less affected dimensions were the physical function (76.8 +/- 22. 2) and the social relations (76.1 +/- 23.1). Least good quality of life scores concerned the pain (60.2 +/- 25.2), perception of general health (59.3 +/- 23.1) and vitality (57.8 +/- 19.5) dimensions. The age was a discriminant criterion since quality of life was better in patients of the 18-23 age group for five dimensions. Mean annual treatment costs of a patient with severe haemophilia were determined as 425 762 French francs ($73 029). Loss of production was estimated at a mean of 4609 French francs ($791) per active patient over the course of the year. Results showed indirect evidence of the usefulness of early home treatment.

Open synovectomy for the prevention of recurrent hemarthrosis of the ankle in patients with hemophilia. A report of five cases with magnetic resonance imaging documentation.

Hemophilic arthropathy is an incapacitating complication of severe hemophilia resulting from recurrent bleeding in the same joint. Open synovectomy has been used since 1969 to prevent recurrent hemarthrosis of target joints. Between 1988 and 1993 we performed open synovectomy of the ankle in five hemophiliacs aged 6 to 9 years with early-stage hemophilic arthropathy. Magnetic resonance imaging proved very useful for evaluating the severity of joint damage, usually underestimated on plain radiographs; for determining the degree of synovial membrane hypertrophy, which is a critical factor in the decision to perform synovectomy; for planning the surgical procedure and for explaining treatment failures. A decrease in the frequency of hemarthrosis episodes occurred in all five ankles. A repeat synovectomy was needed in one case and in another patient the frequency of hemarthrosis episodes increased somewhat after the fourth year. There was no loss of range of motion. Our data suggest that open synovectomy is effective and safe for reducing the frequency of hemarthrosis and that magnetic resonance imaging should be routinely performed before the procedure.

MR imaging in hemophilic arthropathy.

20 children with hemophilia were examined with Magnetic Resonance Imaging (MRI) to determine if MRI could be used to assess hemophilic arthropathy, especially in the early stages. 28 joints of the appendicular skeleton were imaged, including 17 knees, 10 ankles and 1 elbow. The results were compared to clinical and plain radiographic assessments and to the surgical findings when synovectomy was performed. MRI is able to evaluate the components of hemophilic arthropathy such as fluid effussion, synovial hypertrophy, the status of the articular cartilage, and bony lesions. These lesions can be discovered at an early stage when plain radiographs are still normal. MRI seems to be useful for the selection of patients needing early treatment of hemophilic arthropathy and in monitoring response to therapy.

Clinical and biological survey of haemophilia A and B patients infused with French heat-treated concentrates.

The efficiency of heat treatment procedures of factor VIII and factor IX concentrates, prepared from voluntary, non-paid donors by three French Blood Transfusion Centres, on the inactivation of HIV and non-A, non-B hepatitis (NANB) viruses was assessed. Some 43 patients (26 haemophilia A, 17 haemophilia B) were followed for at least 1 year by testing for HIV antibodies and alanine aminotransferase (ALT). No HIV seroconversion was observed indicating that heat treatment was completely efficient. Among 26 haemophiliacs, 6 (4 haemophilia A, 2 haemophilia B) presented an elevation in ALT, indicating only a 75% reduction of NANB viral contamination.

[Importance of synoviorthesis using osmic acid in the treatment of hemophilic arthropathy. Apropos of 19 cases]. / Intérêt de la synoviorthèse à l'acide osmique dans le traitement de l'arthropathie hémophilique.

While getting synovial sclerosis and articular drying, the synoviorthesis breaks the circulus vicious which comes from iterative hemarthrosis to articular destruction. With 25 cases and a follow up of 6 years, we have got 39% of good results, 35% of medium and 26% of bad ones. The therapeutic efficiency requires a certain latent time (0 to 15 days for isotopical synoviorthesis, 2 to 3 months for those ones with osmical acid.) A new synoviorthesis can be made if needed. Neither age nor radiological stage are of any influence. The radiological worsening uses to go on unchanged. The synoviorthesis should be performed at an early stage, previous to the cartilage and bone destructions.