P. de Kleijn

Prophylactic versus on‐demand treatment strategies for severe haemophilia: a comparison of costs and long‐term outcome

Summary.  A multicentre study was performed to compare clotting factor use and outcome between on‐demand and prophylactic treatment strategies for patients with severe haemophilia. Data on treatment and outcome of 49 Dutch patients with severe haemophilia, born 1970–80, primarily treated with prophylaxis, were compared with those of 106 French patients, who were primarily treated on demand. Dutch patients received intermediate dose prophylaxis, for a median duration of 12.7 years. Patients primarily treated with prophylaxis had fewer joint bleeds per year (median 2.8 vs. 11.5), a higher proportion of patients without joint bleeds (29% vs. 9%), lower clinical scores (median 2.0 vs. 8.0), and less arthropathy as measured by the Pettersson score (median 7 points vs. 16 points). Mean annual clotting factor use was equal at 1488 ± 783 IU kg−1 year−1 (mean ± standard deviation) for patients primarily treated with prophylaxis and 1612 ± 1442 IU kg−1 year−1 for patients primarily treated on demand. These findings suggest that, compared with a primarily on‐demand treatment strategy, a primarily prophylactic treatment strategy leads to better outcome at equal treatment costs in young adults with severe haemophilia.

Measuring patients' perceptions on their functional abilities: validation of the Haemophilia Activities List

Summary.  Recently, the Haemophilia Activities List (HAL), a haemophilia‐specific self‐assessment questionnaire to assess a patients 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 (Cronbachs α = 0.93–0.95), as was internal consistency for the seven domains of the HAL (α = 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 patients 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 patients functional abilities.

Clinimetric instruments to assess functional health status in patients with haemophilia: a literature review

Summary.  The World Federation of Hemophilia scoring system (WFH‐1) evaluates primarily body functions and structures, whereas assessment of the whole area of functional health status is considered nowadays to be a better measure. In addition, the WFH‐1 lacks psychometric properties (reliability, validity and sensitivity to change). This study aimed to gain insight into the clinimetric assessment of functional health status in patients with haemophilia by way of a systematic literature search. A standardized literature search and selection was performed on the databases of CINAHL (1982–2001), Medline (1966–2001), and PubMed (January–July 2001). Clinimetric instruments applied in the selected articles were classified based on the international classification of functioning, disability, and health (ICF) and analysed for their psychometric properties. In 19 articles published between 1979 and 2001, 34 clinimetric instruments were used to assess functional health status. Instruments were classified as measuring the ICF components body structure and function (n = 17), activities (12) and participation (4). Reliability was measured in four articles on three different instruments, the validity (construct) of the instruments for patients with haemophilia was reported in six articles on five instruments, and sensitivity to change in three articles on three instruments. The populations under study varied in number (7–9 35), mean age (21.6–50.8 years), and in the distribution of haemophilia severity. Reports on the evaluation of functional health status in patients with haemophilia are increasing. Further research on the psychometric properties of the instruments is warranted in more groups of patients with haemophilia. Development of the new core set of clinimetric instruments, the WFH‐2, might benefit from this strategy.

Recent developments in clinimetric instruments

Summary.  Assessment of impairment and function is essential in order to monitor joint status and evaluate therapeutic interventions in patients with haemophilia. The improvements in the treatment of haemophilia have required the development of more sensitive tools to detect the more minor dysfunctions that may now be apparent. This paper outlines some of the recent developments in this field. The Haemophilia Joint Health Score (HJHS) provides a systematic and robust measure of joint impairment. The MRI Scoring System has been designed to provide a comprehensive scoring system combining both progressive and additive scales. The Functional Independence Score for Haemophilia (FISH) has been developed to assess performance of functional activities and can be used in conjunction with the Haemophilia Activities List (HAL) which provides a self report measure of function. It is recommended that both measures are evaluated as these tools measure different constructs. Further refinement and testing of the psychometric properties of all of these tools is in progress. More widespread use of these tools will enable the sharing of data across the world so promoting best practice and ultimately enhancing patient care.

Physiotherapy for the treatment of articular contractures in haemophilia

Articular contractures in haemophilia are impairments that can not be cured by means of physiotherapy because of the pathophysiology of the joint. Rehabilitation, however, tries to diminish the disabilities and prevent handicaps caused by the impairments. Physiotherapy aims at pain reduction by means of manual traction. Next to manual traction the intensive physiotherapy programme includes mobilization techniques, muscle strengthening exercises and stretching, joint stability training, postural and gait training, and functional training. In all 50 haemophilia patients have undergone this intensive 4‐week clinical rehabilitation programme. Data of 20 of these severe haemophilia patients show that the mean range of motion at the start of the rehabilitation period, after 4 weeks and after 5 years do not differ. In spite of progressing arthropathy after 5 years the activities of daily living (ADL), walking range and pain are equal or better according to 13 of 15 patients.

Physiotherapy following elective orthopaedic procedures

Summary.  As haemophilic arthropathy and chronic synovitis are still the most important clinical features in people with haemophilia, different kinds of invasive and orthopaedic procedures have become more common during the last decades. The availability of clotting factor has made arthroplasty of one, or even multiple joints possible. This article highlights the role of physiotherapy before and after such procedures.

Validity of health status measurement with the Dutch Arthritis Impact Measurement Scale 2 in individuals with severe haemophilia

The aim of this study was to investigate the validity of the Dutch Arthritis Impact Measurement Scales 2 (D‐AIMS2). Hence, D‐AIMS2 data of individuals with severe haemophilia were correlated with clinical and perceived health‐related quality of life data. Patients with severe haemophilia, who visit the Van Creveldkliniek on a regular basis, were administered the D‐AIMS2. In addition, health‐related quality of life was measured by the Sickness Impact Profile (SIP). As clinical indices, range of movement (which was converted into Joint Alignment and Motion scores) and muscle strength were recorded during the routine visit. Extensive descriptive and correlational (linear) analyses between corresponding datasets were performed. Thirty‐one individuals with severe haemophilia were included. Their scores on the D‐AIMS2 demonstrated moderate to very high internal consistency for scales and components (Cronbach’s α=0.62–0.92). The physical health components of the D‐AIMS2 and the SIP were significantly correlated (Pearson’s r=0.53; P < 0.05). The psychological health and social interaction components of the D‐AIMS2 did not correlate significantly with the psychosocial component of the SIP. The physical health component of the D‐AIMS2 correlated significantly with the clinical data for the lower extremities (r=0.52 and r =−0.45; P < 0.05). These data support the reliability and validity of the physical aspects of the D‐AIMS2 in patients with severe haemophilia. The next step should be to extend the investigation of psychometric qualities of this health‐related questionnaire in a larger population.

Comprehensive elements of a physiotherapy exercise programme in haemophilia – a global perspective

Summary.  Exercise programmes for people with haemophilia are usually designed and implemented to help manage the recovery after a haemarthrosis or a muscle bleed, or as a tool to help prevent bleeding episodes from occurring. In this article, we have identified individual components of exercise that are often applied as separate entities, but may also need to be implemented in concert for optimized impact. Although it may be necessary on occasion to bias an exercise programme towards one component over the others, it is important to recognize that the various elements of exercise are not mutually exclusive. Decreased flexibility, strength and proprioception, will result in an impairment of balance and a loss of function. Programme design should whenever possible be guided by proven methodology in terms of how each component is incorporated, and more specifically how long to perform the exercise for and how many repetitions should be performed. We recognize, however, that this is not always possible and that there is significant value in drawing from the experience of clinicians with specialized training in the management of haemophilia. In this study, both perspectives are presented, providing reference‐based reviews of the mechanics of the various elements of exercise as well as the expert opinions of the authors. Research that has been completed using patients with conditions other than haemophilia may or may not have a direct application with the bleeding disorders population, but the programme design based on principles of tissue healing in addition to disease specific knowledge should be encouraged.

Assessing functional health status in adults with haemophilia: towards a preliminary core set of clinimetric instruments based on a literature search in Rheumatoid Arthritis and Osteoarthritis

Summary.  People with haemophilia experience a progressive deterioration of their functional health status. Regular clinical assessment of functional health status provides insight into their process of disablement. As such, the development of a core‐set of measurement tools is warranted. The aim of this study was to gather data to prepare a (preliminary) core set of clinically relevant and feasible instruments to assess the functional health status of adults with haemophilia, and to indicate their psychometric qualities. Therefore, clinimetric instruments frequently used in two haemophilia‐resembling diseases (Rheumatoid Arthritis and Osteoarthritis) were reviewed from the literature. An extensive search in Medline yielded 13 relevant review articles, incorporating a total of 182 instruments, of which 40 were appropriate for haemophilia. Of these 40 instruments 3 measure body structures, 13 body functions, 19 activities (of which 5 are performance based and 14 self‐report based), and 3 measure participation. This classification is based on the International Classification of Functioning, Disability and Health. Detailed information regarding the psychometrics (reliability, validity and responsiveness) of four instruments is described fully in the literature, whereas the psychometrics of the majority of the other instruments are only partly described. The results of this literature study may contribute to the formation of a (preliminary) core set of clinimetric instruments to assess the functional health status of adults with haemophilia. Decisions on the final core set should be held within the Musculoskeletal Committee of the World Federation of Haemophilia.

The utility of the Dutch Arthritis Impact Measurement Scales 2 for assessing health status in individuals with haemophilia: a pilot study.

The aim of this pilot study was to examine the usefulness of the Dutch version of the Arthritis Impact Measurement Scales 2 (D‐AIMS2)in assessing the health status of Dutch individuals with haemophilia. Sixty‐eight individuals with mild, moderate, and severe haemophilia attending our clinic for their annual check‐up participated. They first completed the Canadian Occupational Performance Measure (COPM). The D‐AIMS2 was filled in afterwards at home. With the COPM, individuals rated their specific problematic activities of daily life (ADL), as well as the severity and importance of each problem. The D‐AIMS2 is a comprehensive, self‐administered questionnaire that evaluates functional health status. Fifty‐seven individuals completed and returned the D‐AIMS2. Reliability analysis demonstrated good internal consistency for the scales (Cronbach’s α=0.76–1.00), as well as for the components (α=0.80–0.88), except for the component ‘social interaction’ (α=0.44). Criterion validity of the D‐AIMS2 was assessed by comparison with COPM outcomes; 80% of the problematic ADLs were included in the questionnaire, 20% were missing. Correlations between the D‐AIMS2 components ‘physical health’ and ‘symptoms’ with predicted scores of those individuals by a highly experienced physiotherapist (r=0.63 and 0.53, respectively) substantiated its concurrent validity. Based on these results we concluded that the D‐AIMS2, with minor adjustments, can be an appropriate tool for assessing the health status of Dutch haemophilia patients.