Use of recombinant factor VIIa (rFVIIa, NovoSeven in patients with congenital FVII deficiency has been reported for the prophylactic management of surgical bleeding and for the treatment of acute bleeding episodes. Because of its short half-life, the use of rFVIIa on a regular prophylactic regimen has not been routinely adopted. In this report, we describe our successful experience with rFVIIa prophylaxis in preventing recurrent target joint bleeding in a severely FVII-deficient adolescent.

A study of major joint outcomes, specifically range of motion and synovitis, was conducted with data from a subset of adolescents enrolled in the prospective Hemophilia Growth and Development Study (HGDS). Clinical observations were carried out over a 7-year period from 1989 to 1996. A secondary aim was to gain insight into factors that might influence decisions regarding maintaining or discontinuing prophylaxis during early adulthood. Twenty-nine participants (median age 17.4 at entry) were included. Median follow-up was 7 years (range: 4.8-7.7). Range of motion (ROM) and synovitis in six major joints (knees, elbows and ankles), were evaluated by physical examination every 6-12 months. At the baseline observation, 73.6% of joints showed no ROM abnormalities or synovitis, and all joints were normal in 11 patients. Of the 11 participants, 54.5% developed abnormalities and 28.1% of normal joints at baseline became abnormal during the follow-up. Ankles were the most severely affected and had persistent progression during late adolescence and adulthood. Elbows and knees did not show progression after the first few years of the follow-up. The progression of haemophilic arthropathy in adolescents and young adults varies from individual to individual and also in the site of affected joints. In view of this, the decision regarding discontinuation of prophylaxis in patients with haemophilia should be individualized.

Purpose: Modern prophylaxis regimens and home factor infusion have enabled hemophiliacs to be more physically active. Our study examines the attitudes of doctors, nurses, physical therapists, social workers, and patients' families towards hemophiliacs' participation in a variety of physical activities. Methods: An anonymous survey was distributed to a group of doctors, nurses, social workers, and physical therapists and also to a group of patients/parents at a meeting of hemophilia treatment centers. The survey consisted of four case studies followed by questions that measured attitudes towards diverse subjects. Results: Overall, the healthcare professionals are in agreement as to the limitations and support of activities. Neither having hemophilia nor the presence of an inhibitor should totally exclude a patient from participating in certain sports. These results are in accordance with the results of a World Federation of Hemophilia survey that examined physician's attitudes towards hemophilia and exercise. The responses of families mirror those of healthcare professionals with the exception that families are more permissive towards a greater variety of sports, but halt activities sooner and are less likely to increase prophylaxis frequency after increased bleeds. Also, the patients' families reported balancing the clinical recommendations with other parental priorities when making their decisions about their children's activities. This was done with the goal of establishing “normalcy” within the familial environment. Disagreements exist among healthcare professionals with respect to subject management for each activity. The differences range from disagreement over prophylaxis administration to the level of consideration that should be given to psychosocial factors such as the concerns of patient's families. Conclusions: These differences most likely reflect the inherent “biases” that arise as a result of the provider's varied training, although support is present for most activities. These findings underscore the necessity of having a multi-disciplinary care team that includes the input of the family so that all of the diverse ideas/viewpoints are represented. In addition, the decision to participate in physical activity as well as the degree of involvement must be individualized for each boy with hemophilia in order to maximize each individual's potential in academic and physical achievement. This survey shows the importance of collective support from healthcare providers of various disciplines as well as the patients' families in encouraging hemophiliacs' participation in sports and activities and managing their care.

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