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.

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.

Despite an almost uniformly positive attitude, physiotherapists infrequently use the outcome measures recommended in the CPGPS. Robust setting-specific tailored implementation strategies based on the reported barriers and facilitators are needed.

Patients with severe haemophilia often experience pain caused by haemophilic arthropathy, and it is known that pain acts as a predictor of disability. The role of pain in functional limitations in these patients, however, has not been systematically investigated. The aim of this study was to describe pain as experienced by patients with haemophilia and to address the role of pain in subsequent functional limitations. Seventy-eight adult patients (18-70 years) with severe haemophilia completed one questionnaire on pain and one on self-perceived functional abilities. Two-thirds of the patients suffered from more than one painful joint, the ankle being mentioned most frequently (n=43). Analgesics were used by 36% of the patients indicating having pain. Six of 10 most frequently mentioned pain descriptors were of evaluative nature. Evaluative qualities of pain were associated with pain intensity, which in turn was associated with the impact of pain on daily life. Pain outcomes, after adjusting for age and treatment modality, explained 3-22% of the variance in self-perceived functional abilities. Data on radiological joint damage (Pettersson scores) were retrieved for a subgroup of 28 younger patients on prophylaxis and no significant correlations with pain parameters were found. Over two-thirds of the patients with severe haemophilia had one or more painful joints. Pain plays a small, but significant role in functional limitations. In the identified subgroup, arthropathy, as measured by the Pettersson score, seemed to have no role in pain experience. Promoting analgesic use might decrease the impact of pain on functional limitations.

Although children with haemophilia are advised to participate in physical activities, their physical fitness has not been studied in a large group. In addition, children with haemophilia may be at increased risk for becoming overweight as a result of inactivity because of joint bleedings or because of overprotection. This study aimed to assess physical fitness (aerobic capacity), joint status, muscle strength, quality of life (QoL), self-reported motor competence and also prevalence of overweight and its association with physical parameters. Weight and height were measured. Skin folds were measured unilaterally at biceps, triceps, subscapular and supra-iliac sites. Aerobic capacity was determined on a cycle ergometer or with a 6-min walk test (6MWT). Muscle strength and active range of motion of elbows, knees and ankle joints were measured. Self-reported motor competence was measured with the 'Competentie BelevingsSchaal voor Kinderen'. Joint pain was scored on a Visual Analogue Scale. The Haemo-QoL Index was used to measure QoL. In 158 Dutch boys with haemophilia, with a mean age of 12.7 years (SD 2.9), normal aerobic capacity and muscle strength were found. Joint pain was reported by 16% of the participants. The prevalence of overweight (16%) was slightly increased when compared with healthy Dutch boys (13.5%). Being overweight had a negative association with the6MWT and QoL. Dutch children with haemophilia have normal aerobic exercise capacity and muscle strength. The majority also has normal joint mobility. Prevalence of overweight is slightly increased.