Summary.  Background: Clotting factor products have been safe for HIV since 1985, and for hepatitis C since 1992. Few studies have reported on mortality in the total population of hemophilia patients after the period of risk of viral infection transmission. Objectives: We studied the mortality, causes of death, and life expectancy of hemophilia patients between 1992 and 2001. We compared these findings with those of previous cohorts, together spanning the periods before, during, and after the use of potentially contaminated clotting products. Patients and methods: We performed a prospective cohort study among 967 patients with hemophilia A and B. Death rates, overall and cause‐specific, were compared with national mortality figures for males adjusted for age and calendar period as standardized mortality ratio (SMRs). Results: Between 1992 and 2001, 94 (9.7%) patients had died and two patients were lost to follow‐up (0.2%). Mortality was 2.3‐times higher in hemophilia patients than in the general male population (SMR 2.3 95% confidence interval 1.9–2.8). In patients with severe hemophilia, life expectancy decreased from 63 (1972–1985) to 59 years (1992–2001). Exclusion of virus‐related deaths resulted in a life expectancy at birth of 72 years. Conclusions: AIDS was the main cause of death (26%) and 22% of deaths were because of hepatitis C. In patients not affected by viral infections, there still appeared to be a trend toward a moderately increased mortality compared with the Dutch male population. Thus, mortality of patients with hemophilia is still increased; this is largely because of the consequences of viral infections.

Recently, the Haemophilia Activities List (HAL), a haemophilia-specific self-assessment questionnaire to assess a patient's self-perceived functional ability, was introduced and a limited pilot study warranted its further development. The present study finalizes the HAL and assesses the convergent and construct validity, as well as the internal consistency of its definitive version. Three questionnaires (HAL, Dutch-Arthritis Impact Measurement Scales 2 and the Impact on Participation and Autonomy questionnaire) were completed by 127 patients with severe haemophilia (<1% clotting activity), as well as four performance tests (button test, 50 metre walking test, timed-up-and-go test and figure-8 walking test). After removal of 15 non-informative items from the provisional HAL, three components within the questionnaire were identified (upper extremity activities, basic lower extremity activities and complex lower extremity activities). The internal consistency of these components was high (Cronbach's alpha = 0.93-0.95), as was internal consistency for the seven domains of the HAL (alpha = 0.61-0.96). The convergent validity of the HAL when compared to the other two questionnaires was good (r = 0.47-0.84). The construct validity of the HAL when compared to the four performance tests was generally lower (r = 0.23-0.77). The final version of the HAL has good internal consistency and convergent validity and gives the clinician insight into a patient's self-perceived ability to perform activities of daily life. It is likely that self-assessment instruments (questionnaires) and performance tests consider different concepts of functional health status and it is therefore recommended that both types are included when clinicians assess a patient's functional abilities.

We designed a double-blind trial to study the effect of an "activated" prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in hemophiliacs with antibodies to factor VIII. Fifteen patients received either FEIBA or the control preparation (a nonactivated prothrombin-complex concentrate) for a total of 150 bleeding episodes (four mucocutaneous bleeding, 117 joint bleeding, and 29 muscle bleeding). In 64 per cent of the episodes, FEIBA was judged by the physician to be effective; the control preparation was perceived as effective in 52 per cent of the episodes in which it was used. Pairwise comparison of FEIBA and the control preparation for bleeding in the same joint or muscle showed a significantly better result with FEIBA (P = 0.0085). Joint mobility after the use of FEIBA was significantly improved (P = 0.006). There was a high incidence of hepatitis (three of the 15 patients) and of transient disturbances of liver function (nine of 15) during the 15-month observation period.

Several instruments can be used to evaluate the functional status of patients with haemophilia, but none of these instruments is specific for haemophilia. We developed a haemophilia-specific self-assessment questionnaire to evaluate and monitor a patient's perceived functional health status: the Haemophilia Activities List (HAL). In three separate but interlinked substudies, the questionnaire was constructed and tested for face, expert, and convergent validity, as well as internal consistency and patient-evaluated relevance. Items for the questionnaire were collected by interviewing 162 patients, using the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR). The items were combined to generate the first version of the questionnaire [HAL(1)]. This version was evaluated and commented on by two focus groups (patients and caregivers), and then the questionnaire was adapted on the basis of these comments, forming the final version, HAL(2). This version was then validated in a pilot study with 50 consecutive patients using the Dutch Arthritis Impact Measurements Scales 2 (Dutch-AIMS2) and the Impact on Participation and Autonomy (IPA) questionnaires. The HAL(2) showed good convergent validity (Pearson correlation 0.80-0.91; P < 0.01), and the internal consistency was good for six of the eight domains (Cronbach's alpha 0.83-0.95). Patients considered the content of the HAL to be more relevant to their situation than the content of the other questionnaires (P < 0.01). Three major factors (upper extremity function, lower extremity function, key activities/major problem activities) were identified by factor analysis. The questionnaire seems to be a useful tool to identify problematic activities as part of the functional health status of patients with haemophilia. The construct validity, test-retest reliability, and responsiveness of the HAL will be established in the future.

Most health care professionals involved in the management of people with haemophilia (PWH) believe that exercise is beneficial and its practice is widely encouraged. This article aims to demonstrate that appropriate exercise (adapted to the special needs of the individual PWH) may be beneficial for all PWH through improved physical, psychosocial and medical status. Based on evidence gathered from the literature, many PWH, particularly those using long-term prophylaxis or exhibiting a mild/moderate bleeding phenotype, are as active as their healthy peers. PWH experience the same benefits of exercise as the general population, being physically healthier than if sedentary and enjoying a higher quality of life (QoL) through social inclusion and higher self-esteem. PWH can also gain physically from increased muscle strength, joint health, balance and flexibility achieved through physiotherapy, physical activity, exercise and sport. Conversely, very little data exist on activity levels of PWH in countries with limited resources. However, regarding specific exercise recommendations in PWH, there is a lack of randomized clinical trials, and consequently formal, evidence-based guidelines have not been produced. Based on published evidence from this review of the literature, together with the clinical experience of the authors, a series of recommendations for the safe participation of PWH in regular physical activities, exercises and sport are now proposed. In summary, we believe that appropriately modified programmes can potentially allow all PWH to experience the physical and psychosocial benefits of being physically active which may ultimately lead to an improved QoL.