Major improvements in health-related quality of life during the use of etanercept in patients with previously refractory juvenile idiopathic arthritis.

OBJECTIVE: To evaluate changes in health-related quality of life (HRQoL) in patients with refractory juvenile idiopathic arthritis (JIA) who are being treated with etanercept. METHODS: 53 patients with JIA from seven Dutch centres were included. HRQoL was measured by the Childhood Health Assessment Questionnaire (CHAQ), Child Health Questionnaire (CHQ) and Health Utilities Index mark 3 (HUI3) at the start and after 3, 15 and 27 months of treatment. At the same time points the following JIA disease activity variables were collected; physician's global assessment through the visual analogue scale (VAS), number of active and limited joints and erythrocyte sedimentation rate. A statistical method linear mixed models was used to assess outcomes over time. RESULTS: During etanercept treatment both disease-specific and generic HRQoL outcomes improved dramatically. Significant improvements were shown after 3 months and these improvements continued at least up to 27 months of treatment. The disease-specific CHAQ, including VAS pain and wellbeing, showed a significant improvement in all domains. The generic health-profile measure CHQ improved for all the health concepts except for "family cohesion", which was normal. The generic preference-based HUI3 showed impairment and, subsequently, significant improvement in the more specific domains ("pain", "ambulatory", "dexterity"). In accordance disease activity variables also improved significantly over time. CONCLUSION: This study shows that the HRQoL of patients with refractory JIA can be substantially improved by the use of etanercept for all aspects impaired by JIA. Information on HRQoL is crucial to understand the complete impact of etanercept treatment on patients with JIA and their families.

Long-term follow-up on effectiveness and safety of etanercept in juvenile idiopathic arthritis: the Dutch national register.

OBJECTIVE: We undertook an observational study to obtain a complete overview of the long-term effectiveness and safety of etanercept in patients with different juvenile idiopathic arthritis (JIA) subtypes. METHODS: At baseline we collected patient and disease characteristics of all Dutch patients with JIA who started treatment with etanercept. Disease activity was evaluated (at start of the study, after 3 months and then yearly) according to the JIA core set of the American College of Rheumatology paediatric definition for 30, 50 and 70% improvement (ACR Pedi 30, 50 and 70). Use of etanercept and concomitant drugs was monitored. Adverse events were recorded. RESULTS: We included 146 patients with JIA with a median follow-up of 2.5 years per patient (range 0.3-7.3). JIA subtypes represented: 27% systemic, 8% polyarticular rheumatoid factor positive, 38% polyarticular rheumatoid factor negative, 19% oligoarticular extended, 3% enthesitis-related and 5% psoriatica. Most patients (77%) met the criteria of the ACR Pedi 30 in the first 3 months of treatment. For the majority of patients this improvement was sustained; 53 (36%) of all patients met the remission criteria. No other second-line agents were needed in 43 patients. Although patients with systemic JIA responded initially less to etanercept therapy than patients from other subtypes, those who did respond showed equal effectiveness in the long term. Serious adverse events rate was low (0.029 per patient year). CONCLUSIONS: Etanercept is effective and safe in JIA, even for a large proportion of the patients with systemic JIA. The greatest improvement occurred in the first 3 months of treatment, and was sustained for a long time in most patients (up to 75 months).

Development of a web-based register for the Dutch national study on biologicals in JIA: www.ABC-register.nl.

OBJECTIVES: Most clinical studies use paper case record forms (CRFs) to collect data. In the Dutch multi-centre observational study on biologicals we encountered several disadvantages of using the paper CRFs. These are delay in data collection, lack of overview in collected data and difficulties in obtaining up-to-date interim reports. Therefore, we wanted to create a more effective method of data collection compared with CRFs on paper in a multi-centre study. METHODS: We designed a web-based register with the intention to make it easy to use for participating physicians and at the same time accurate and up-to-date. Security demands were taken into account to secure the safety of the patient data. RESULTS: The web-based register was tested with data from 161 juvenile idiopathic arthritis patients from nine different centres. Internal validity was obtained and user-friendliness guaranteed. To secure the completeness of the data automatically generated e-mail alerts were implemented into the web-based register. More transparency of data was achieved by including the option to automatically generate interim reports of data in the web-based register. The safety was tested and approved. CONCLUSIONS: By digitalizing the CRF we achieved our aim to provide easy, rapid and safe access to the database and contributed to a new way of data collection. Although the web-based register was designed for the current multi-centre observational study, this type of instrument can also be applied to other types of studies. We expect that especially collaborative study groups will find it an efficient tool to collect data.

[Successful treatment with infliximab of a patient with refractory sarcoidosis]. / Succesvolle behandeling met infliximab van een patient met therapieresistente sarcoïdose

A 35-year-old patient had persistent, refractory symptomatic pulmonary sarcoidosis, complicated by a rare congenital muscular disease: McArdle's disease (a glycogen storage disease caused by myophosphorylase deficiency). As the desaturations during mild exercise caused by the sarcoidosis aggravated the negative consequences of his muscle disease and he failed to respond adequately to corticosteroids and methotrexateimmunosuppressive agents, the patient was successfully treated experimentally with infliximab, a monoclonal antibody and specific tumour necrosis factor alpha (TNF-alpha) inhibitor. The results were favourable: after 17, 21 and 36 months there was an improvement in various lung function parameters, his fatigue was reduced and the patient had been able to resume his work as a taxi driver. TNF-alpha appears to be an important mediator of clinical disease in sarcoidosis and infliximab could be a promising therapy for patients with refractory sarcoidosis.

Rhemulatologists' performance in dailiy practice.

OBJECTIVE: To assess rheumatologists' performance for 8 rheumatologic conditions and to explore possible explanatory factors. METHODS: After written informed consent was obtained, 27 rheumatologists (21% of all Dutch rheumatologists) practicing in 16 outpatient departments were each visited by 8 incognito "standardized patients" (SPs). The diagnoses of these 8 cases account for about 23% of all new referred patients in the Netherlands. Results for ordered lab tests as well as real radiographs with corresponding results from a radiologist were simulated. Information from the visits was obtained from the SPs, who completed predefined case-specific checklists, and by collecting data on resource utilization. Feedback was provided. RESULTS: Altogether 254 encounters took place, of which 201 were first visits and 53 were followup visits. SPs were unmasked twice during a visit. There was considerable variation in resource utilization (lab tests and imaging) between cases and between rheumatologists. Mean costs per rheumatologist ranged from US $ 4.67 to $ 65.36 per visit for lab tests and from US $ 33.15 to $ 226.84 per visit for imaging tests. No significant correlations were seen between resource utilization costs and number of years of clinical experience or performance on checklist scores. Rheumatologists with longer experience had lower total item checklist scores (r = -0.47; P < 0.05). CONCLUSION: A considerable variation in resource utilization was found among 27 Dutch rheumatologists. The information obtained is an excellent source for discussion on the appropriateness of care.

The social network characteristics of fibromyalgia patients compared with healthy controls.

OBJECTIVE: To assess structural social network characteristics and perceived loneliness in fibromyalgia syndrome (FMS) patients and healthy controls. METHODS: A cross-sectional, retrospective, case-control design was employed using a structured interview and a self-report questionnaire. We studied 25 female FMS patients and 25 matched healthy female controls. RESULTS: FMS patients had statistically significantly (P < 0.05) more intimate friends (mean 4.5) and more health care providers (mean 1.5) than did controls (2.3 and 0.0, respectively). The FMS patients more often initiated the contact with family members (mean 2.1) than did controls (mean 0.8). FMS patients did not perceive themselves as lonelier than controls perceived themselves. While there was a significant negative correlation between loneliness and social network variables among the controls, this relationship was not significant among the FMS patients. Among the patients, there was a strong correlation between the total social network size and the number of intimate friends, whereas in controls, the mean number of acquaintances was strongly correlated with the total network size. CONCLUSION: Compared to healthy controls, the social networks of FMS patients presented more linkages with intimate friends, family members, and health care providers. The lack of correlation between loneliness and social network variables for FMS patients is not what might be expected from social support theory. The assessment of structural social network characteristics along with social support variables may add to our understanding of the social functioning of FMS patients.

Patient utilities in fibromyalgia and the association with other outcome measures.

OBJECTIVE: To compare in patients with fibromyalgia (FM) utilities derived by rating scale and standard gamble methods; to gain insight into construct validity by relating utility values to other outcome measures; to assess the sensitivity to change of utilities. METHODS: A total of 73 patients with FM were randomized into one of 3 groups: low impact fitness training, biofeedback, or controls. At baseline and after 6 mo the Maastricht Utility Measurement Questionnaire was applied. By means of both the rating scale and standard gamble method patients were asked to value their own health status. Construct validity of patient utility measurements was evaluated by Spearman correlation and multiple regression of baseline values with pain, stiffness, patient's global assessment, Sickness Impact Profile (SIP), modified Health Assessment Questionnaire and Arthritis Impact Measurement Scale (AIMS). Sensitivity to change was assessed against changes in these outcomes. RESULTS: Rating scale utilities correlated significantly (p < 0.05) with patient's global assessment (rs = 0.53), pain (rs = -0.47), SIP (rs = -0.43), and with 9 of 11 dimensions of the AIMS (rs ranging from 0.23 to 0.62). Standard gamble utilities correlated significantly with mobility, pain, and arthritis impact of the AIMS scale (rs from 0.22 to 0.36) and with pain by visual analog scale (rs = -0.24) and patient's global assessment (rs = 0.32). Multiple regression analysis showed that patient's global assessment explained 41% (rating scale) and 10% (standard gamble) of total variance in baseline utilities. Also, 16% of the variance in change in rating scale utility values was explained by changes in patient's global assessment. In contrast, variance of changes in standard gamble utility values was not explained significantly by changes in other disease outcomes. CONCLUSION: Rating scale utilities correlated more strongly with disease outcome measures than standard gamble utilities. Also, construct validity for the rating scale was better than for the standard gamble. In FM, utility measurement is sensitive to the method chosen to elicit patient priorities.

Problem elicitation to assess patient priorities in ankylosing spondylitis and fibromyalgia.

OBJECTIVE: To elicit patient priorities as outcome measures in ankylosing spondylitis (AS) and fibromyalgia (FM); to relate these measures to other outcomes; to assess construct validity and sensitivity to change of the problem elicitation technique (PET) questionnaire. METHODS: One hundred thirty-four patients with AS were randomly allocated to weekly sessions of group physical therapy or daily exercises at home, whereas 73 patients with FM were randomized into one of 3 groups (low impact fitness, biofeedback, controls). The PET questionnaire was applied by trained interviewers at baseline and at 6 (FM) and 9 (AS) month followup. A PET score was calculated at each assessment. Construct validity of the PET was assessed by correlation and multiple regression of baseline values with other disease outcomes (pain, stiffness, patient's global assessment, Sickness Impact Profile (SIP), Health Assessment Questionnaire (HAQ), Arthritis Impact Measurement Scale (AIMS), patient utilities). Sensitivity to change of PET was assessed against changes in these outcomes and by comparing the efficiency of the PET with other outcomes. RESULTS: Patients with FM identified more problems (mean 6.8) than patients with AS (mean 4.4). Moreover, more patients with AS than with FM were unable to identify any problem at baseline (10% compared to 1%). The PET score improved from 14.9 to 11.3 (p = 0.0001) in patients with AS but did not change from 21.8 to 21.1 (p = 0.24) in patients with FM. Construct validity testing of the PET score showed statistically significant (p < 0.05) correlations with AIMS, utilities, SIP, HAQ, pain, stiffness, and patient's global health in both groups of patients (r varying from 0.22 to 0.66). By multiple regression pain explained 29% of the variance in PET scores among patients with AS. In FM patient global assessment accounted for 39% of total variance of PET scores, whereas pain explained another 15%. Changes in PET scores correlated significantly (p < 0.05) with changes in AIMS, utilities, pain, stiffness, and patient global health in both AS and FM (r varying from 0.22 to 0.51). Some 6% of the variance in changes in PET scores was explained by changes in pain in patients with AS and 35% by changes in pain and subjective health in patients with FM. Assessment of sensitivity to change revealed that efficiency of the PET score was 0.6 in patients with AS and 0.09 in those with FM. Compared to other outcomes this was reasonable in patients with AS but low in those with FM. CONCLUSION: Obtaining patient priorities was generally feasible. In both groups of patients construct validity of the PET questionnaire was satisfactory. The PET was much more sensitive to change in patients with AS than in patients with FM.

Social network characteristics in fibromyalgia or rheumatoid arthritis.

PURPOSE: To describe trends in the personal social network characteristics of the fibromyalgia patients compared with the network of patients with another chronic disorder such as rheumatoid arthritis (RA). METHODS: A structured interview was applied to 10 fibromyalgia patients and 10 matched RA patients. RESULTS: The networks of the 10 fibromyalgia and 10 RA patients were comparable in most respects, namely: the small number of intimate friends, the reliance for support on the spouse and the physician, and the relative lack of new social contacts. Furthermore, the fibromyalgia networks were closed networks within a small geographic area. These networks lacked initiative to establish and maintain relations, and can hardly fulfill the patients' psychosocial needs. CONCLUSION: Based on small numbers, the study provides some evidence that social networks of patients with fibromyalgia are more restricted than those of RA patients. Further studies should include larger numbers of patients to clarify cause and effect relationships and to suggest new directions in the treatment of chronic disorders such as fibromyalgia.