F.R. van Genderen

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.

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.

The use of a disability model in haemophilia research

Summary.  Most haemophilia research is medically orientated. However, assessment of the impact of disease on the individual is different when viewed from a rehabilitation perspective. Several models are available to explore functioning and health from this perspective. The disablement process (DP) is such a model, and the aim of this study was to introduce this process in haemophilia research to see whether this type of research could lead to meaningful data. Forty‐three adult patients with severe haemophilia participated in this study in which the three ‘main pathway’ domains of the DP (impairments, functional limitations and disability) and two additional factors (intra‐individual and risk factors) were addressed. Three questionnaires (HAL, Dutch‐AIMS2 and IPA) were incorporated, and Pettersson scores for 21 patients were retrieved. Step‐wise and hierarchical regression analysis was used to assess relationship between the various domains. Arthropathy explained 48% of the variance in functional limitations and nearly 25% of the variance in disability. Functional limitations explained 54% of the variance in disability. Patients identified pain as an important aspect of health which addressed 22% and 13% of the variance in functional limitations and disability respectively. Age was correlated with arthropathy (r = 0.85; P < 0.001), whereas psychological health correlated with pain (r = 0.67; P < 0.001). Both variables were also correlated with functional limitations and disability. Analyses adjusting for the effects of age and psychological health were subsequently performed resulting in more insight in the associations within the DP. The use of the DP in haemophilia research proved to be useful.

Participation and risk-taking behaviour in sports in children with haemophilia.

Summary.  The aim of this study was to investigate participation in sports activities and risk‐taking behaviour in children with haemophilia and the relationship to personal and health related factors. Ninety‐nine children (mean age 12.6 years) completed questionnaires regarding participation in sports and physical education, medication, health related quality of life, and perceived motor competence. Furthermore, weight, height, active range of motion, pain, and muscle strength were assessed. Based on a risk exposure factor (REF) we defined subgroups with low, medium, and high risk when participating in sport. Most children participate in sport five times a week (mean 140 min per week), and little absence during school sports was reported. In general, prophylaxis was not tailored to sport activities. Boys with haemophilia preferred other sports than their Dutch contemporaries. The top‐5 being soccer, swimming, tennis, gymnastics, and cardio‐fitness for the former; soccer, gymnastics, tennis, hockey, and swimming for the latter. Significant differences between the low risk group and both other groups were found for sport intensity, total energy expenditure (EE) and average risk factor (ARF), however the medium and high‐risk groups did not differ in ARF. REF and sport participation increased associated with increasing interest in athletic and motor activities. No significant differences were found between REF groups regarding age, Z‐BMI, Z‐AROM, Z‐Muscle force, and the presence of painful joints. As in normal peers motivation to participate in sport depends upon the enthusiasm and interest, in children with haemophilia choice of sports differs, probably related to sport advice.

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.

Patients with chronic whiplash-associated disorders. Relationship between clinical and psychological factors and functional health status

Schmitt MA, van Meeteren NL, de Wijer A, van Genderen FR, van der Graaf Y, Helders PJ: Patients with chronic whiplash-associated disorders: Relationship between clinical and psychological factors and functional health status. Objectives:To examine the relative contribution of cervical impairments and psychosocial factors to perceived disability among people with chronic whiplash-associated disorders. Design:A total of 86 patients with chronic whiplash-associated disorders participated in this observational, cross-sectional study. All patients were presented to outpatient physical therapy clinics. All patients completed the neck disability index. Depression, anxiety, and catastrophizing were measured with the Hospital Anxiety and Depression scale and the pain coping and cognition list, respectively. Cervical function was assessed by measuring the active range of motion. Stepwise and hierarchical regression analysis was used to estimate the contribution of cervical impairment and psychosocial functions to the variance in neck disability. Results:Depressive symptomatology and catastrophizing explained 61% of the variance in neck disability index scores. Catastrophizing explained 57% of the variance in neck disability index scores and 15% of the variance in the sum scores of active cervical rotations. Conclusions:Catastrophizing explained the variance in both perceived neck disability and, to a lesser extent, active range of cervical motion, which suggests that pain-related catastrophizing plays an important role in the physical complaints of patients with chronic whiplash-associated disorders when referred to a physical therapist.

Perceived competence in children and adolescents with haemophilia: an explorative study

Summary.  With the introduction of prophylaxis, restricting children with haemophilia to participate in physical activities was no longer necessary. Subsequently, many studies report on improved physical functioning in children and adolescents with haemophilia. However, little is known about psychological aspects such as perceived competence and impact of disease. Therefore, the aims of this study were to explore: (i) perceived competence, (ii) perceived impact of illness, and (iii) analyse associations between perceived competence and demographic factors, disease‐related factors and joint status in young haemophiliacs in the Netherlands. Fifty‐four children (age 8–12 years) and 72 adolescents (12–18 years) with haemophilia participated in this cross‐sectional, multi‐centre, explorative study. Measurements included perceived competence (Self Perception Profile for Children/Adolescents; range 6–24/5–20), impact of disease (Revised Perception Illness Experience; range 1–5), demographic factors, disease‐related factors, joint status and functional status. Mean (SD) scores for perceived competence in the children ranged from 17.3 (±4.0) to 19.6 (±4.0), and for adolescents from 13.3 (±2.4) to 15.7 (±2.8) points. In general, scores were comparable with those of healthy peers, but children with haemophilia had a lower global self‐worth score and competence in close friendship was lower for adolescents when compared with those of healthy peers. Mean (SD) scores for impact of disease ranged from 1.2 (±0.4) to 2.3 (±0.8) in children and from 1.3 (±0.4) to 2.0 (±0.8) in adolescents. Severe haemophilia, prophylactic medication, high impact of disease and a shorter walking distance showed a weak to moderate association with perceived competence. Children and adolescents with haemophilia in general have a perceived competence that is nearly comparable with that of healthy peers, with the exception of a lower global self‐worth in children and a lower competence for close friendship in adolescents. Haemophiliacs seem to perceive their disease as having relatively low impact on their life. Severe disease, prophylactic treatment and low functional status seemed to be associated with lower perceived competence.

The Neck Bournemouth Questionnaire Cross-Cultural Adaptation Into Dutch and Evaluation of Its Psychometric Properties in a Population With Subacute and Chronic Whiplash Associated Disorders

Study Design. Cross-cultural adaptation of an outcome questionnaire. Objective. The aim of the study was to cross-culturally adapt the Neck Bournemouth Questionnaire (NBQ) for the Dutch language in a population of people with subacute and chronic whiplash associated disorders (WAD), and to assess its psychometric qualities. Summary of Background Data. The NBQ covers the salient dimensions of the biopsychosocial model of pain, and has been shown to be reliable, valid, and responsive to clinically significant change in patients with non specific neck pain. However, no Dutch validated version was available for patients with WAD at the time our study was initiated. Methods. The English version of the NBQ was translated into Dutch (NBQ-NL) and back-translated according to established guidelines. The internal consistency (with help of Cronbachs α), construct validity, and convergent validity were estimated in a different group of 92 patients. Agreement and correlation between the NBQ-NL scores and counterpart questionnaires (SF-36, Neck Disability Index, Hospital Anxiety and Depression Scale, the General Perceived Self-Efficacy and a numerical rating scale for perceived pain) were investigated, using Bland and Altman method and Spearman rank correlation coefficient. Subsequently, 34 patients with subacute WAD completed the questionnaire twice over 1 to 3 weeks, to assess its test-retest reliability. Results. Ninety-two patients, with subacute and chronic WAD, completed the study. Their mean age was 41 years (SD = 11). There were relative high mean scores on the Neck Disability Index (25.5; SD = 8.2), and the NBQ (43.9; SD = 14.8). The NBQ-NL was granted face-validity. Spearman rank correlation coefficient was 0.51 to 0.82 (except for General Perceived Self-Efficacy (0.21). The limits of agreement of normalized scores were relative variable, from small to wide. Cronbachs α (internal consistency) for the NBQ-NL whole scale was 0.87. The Intraclass correlation coefficient for the test-retest reliability was excellent (0.92) and the SEM was relatively low (3.7). Conclusion. Overall, the rank correlation level in general was good, whereas the agreement between questionnaires outcome was variable, most of which seems to be attributable to absolute scale differences. The NBQ-NL is a useable patient-orientated tool for assessing disability in clinical studies and clinical diagnosis in Dutch speaking patients with WAD.

Pain in patients with chronic idiopathic axonal polyneuropathy

Background/Aims: Pain in patients with chronic idiopathic axonal polyneuropathy (CIAP) has never been studied in detail. The aim of the study was to investigate the pain experienced by patients with CIAP, and to determine whether pain is associated with health-related quality of life (HRQoL). Methods: The McGill Pain Questionnaire (MPQ) and the RAND-36 were used in a cross-sectional study. Results: Sixty-three of 91 patients with CIAP reported experiencing pain, describing it as nagging (56%) and annoying (52%). Of these patients, 27 were classified in a subgroup with neuropathic pain (median VAS = 33 mm), 25 in a subgroup with non-neuropathic pain (median VAS = 34 mm), and 11 in a mixed-pain subgroup (median VAS = 25 mm). Non-neuropathic pain was as common and as painful as neuropathic pain. Pain was strongly associated with the physical functioning domain of the RAND-36 in patients in the mixed pain subgroup (r = –0.71, p < 0.05). Conclusion: Neuropathic and non-neuropathic pain syndromes should be distinguished in patients with CIAP who experience pain, to enable appropriate tailoring of treatment.

Determinants of participation in patients with severe haemophilia

The multifactorial nature of disability makes it difficult to point out a specific cause for limitations in participation. The conceptual framework of the WHO‐ICF (International Classification of Function, Disability and Health) was used to study the determinants participation in patients with severe haemophilia. Outcome was assessed in a single‐centre cohort of 124 patients with severe haemophilia. Joint mobility and muscle strength of the elbows, knees and ankles, in combination with recent X‐ray findings (N = 39 only) and the MPQ‐DLV pain questionnaire were used to assess Body Functions and Structures. Four performance‐based functional tasks and the HAL questionnaire were used to assess Activities. The IPA questionnaire was used to assess Participation. Stepwise and hierarchical regression analysis adjusted for age and psychological health (Dutch‐AIMS 2) was used to associate the various domains of the ICF. Irrespective of age, joint mobility was an important factor in explaining self‐reported and performance‐based activities. Muscle strength had no significant association with participation. Self‐reported activities showed a stronger association with participation than performance‐based activities. Adjusted for age and psychological health, joint mobility and pain explained none of the variation in participation. Self‐reported activities, however, significantly contributed in explaining participation (25%), whereas performance‐based activities (3%) did not. This study adds to the knowledge of determinants of participation in haemophilia. As the currently used instruments on joint status and activities only partially explain differences in participation, this aspect of clinical outcome should be included to fully assess outcome in haemophilia.