Responsiveness of clinical measures in children with oligoarticular juvenile chronic arthritis.

OBJECTIVE: To compare the relative responsiveness of outcome measures in children with oligoarticular course juvenile chronic arthritis (JCA). METHODS: Twenty-six consecutive children with JCA and < or =4 joints involved were assessed at admission and after 3 months of followup. Outcome measures included physician and parent global assessments, functional ability as measured by the Childhood Health Assessment Questionnaire (CHAQ), articular variables, and laboratory indicators of systemic inflammation. Responsiveness was calculated with the standardized response median, the effect size, and the Guyatt method. RESULTS: The physician global assessment of disease activity, the active joint count, the global articular severity score, and the number and score of swollen joints proved consistently more responsive with either responsiveness measure used. The parent global assessment of the child's well being, the scores of joints with pain/tenderness and limited range of motion, and the number of joints with limited range of motion showed intermediate responsiveness. The CHAQ, morning stiffness, and laboratory indicators of systemic inflammation were the least responsive measures. CONCLUSION: The subjective estimation of disease activity by the physician and the articular variables were the more responsive outcome measures in children with oligoarticular JCA. The assessment of functional ability as measured by the CHAQ showed a poor sensitivity to clinical change in this subset of patients with JCA.     

Health related quality of life survey about children and adolescents with juvenile idiopathic arthritis

This study aimed to investigate the proxy-reported Health related quality of life (HRQOL) and its determinants in patients with juvenile idiopathic arthritis (JIA). It was hypothesized that HRQOL would decrease with worsening disease and disability. Data were available in cross-sectional study on children and adolescents with JIA according to the ILAR criteria. Patient demographics, type of JIA, clinical determinants and laboratory parameters relating to JIA were obtained for each patient. Functional disability was assessed using the parent’s or children’s version of the child health assessment questionnaire (CHAQ). The HRQOL was evaluated using the juvenile arthritis quality of life questionnaire (JAQQ). These questionnaires were previously translated and validated in Moroccan children. A total of 80 participants were enrolled with mean age of 11 [6–17 years], and female predominance (59%). Many patients (42.5%) had oligoarticular subtype; 31.3% polyarticular subtypes and 26.2% systemic form. The mean global score of JAQQ was 2.6 ± 1.3 (1–6). Patients with persistant oligoarticular had better gross motor function (P < 0.0001), better fine motor function (P < 0.0001), less psychosocial impact (P = 0.001), and less symptoms (P = 0.001) in comparison with polyarticular and systemic subtypes. The HRQOL assessed by the JAQQ was worse in adolescent patients in comparison with children except for symptoms (P = 0.15). The gender (P = 0.95), age at onset of JIA (P = 0.81), and evolution duration (P = 0.34) were not correlated with global score of JAQQ. The diagnosis delay was significantly associated with decrease of HRQOL (P = 0.001). The decrease of HRQOL was correlated with disease activity [pain (VAS), painful and swollen joints, erythrocyte sedimentation rate (for P < 0.0001)], with disability index (CHAQ) (P = 0.001) and presence of hip involvement (P = 0.01). This study suggests that JIA can have a significant adverse effect on the HRQOL of moroccan patients, particularly adolescents with polyarticular and systemic subtypes. Disease duration, disability score (CHAQ) and pain were the strongest determinants of poorer HRQOL.     

Assessment of quality of life of parents of children with juvenile chronic arthritis

The aim of the study was to assess the quality of life (QOL) and the psychological status of parents of children with juvenile chronic arthritis (JCA). The QOL, anxiety and depression of the parents of 28 children with JCA were evaluated and compared to those of the parents of 28 healthy children. Mothers of JCA children and mothers of healthy children reported similar QOL. The reported anxiety and depression levels were similar for mothers and fathers in both groups. The parents of children with pauciarticular-type JCA reported lower QOL and higher levels of anxiety and depression than the parents of children with other types, namely polyarticular and systemic JCA. These findings may be explained by the fact that the pauciarticular patients had shorter disease duration and were less frequently seen in the outpatient clinic. The QOL of mothers of children with JCA was found to be slightly impaired in the group of children with pauciarticular JCA. Future larger studies are needed to confirm these results, as the number of subjects in the three groups was rather low. Received: 26 September 2001 / Accepted: 8 February 2002     

Rheumatoid arthritis quality measures and radiographic progression

Objective

Documentation of quality measures (QMs) in rheumatoid arthritis (RA) is used as a surrogate for measure of quality of care, but the association of this documentation with radiographic outcomes is uncertain. We examined documentation of RA QMs, for disease activity and functional status and the association with radiographic outcomes.

Methods

Data were analyzed for 438 RA patients in a longitudinal cohort with complete data on van der Heijde-modified Total Sharp Score (TSS). All rheumatologist (N = 18) notes in the electronic medical record during a 24-month period were reviewed for RA QMs. Any mention of disease activity categorized as low, moderate, or high was considered documentation of the QM for disease activity. Functional status QM documentation included any mention of the impact of RA on function. Change in TSS was quantified with progression defined as ≥1 unit per year. We compared percent of visits with an RA QM documented and mean change in TSS.

Results

The mean age in the cohort was 56.9 years, disease duration was 10.8 years, baseline DAS28 score was 3.8 (±1.6), 67.7% were seropositive, and 33.9% used a biologic DMARD. Radiographic progression was observed in 28.5%. Disease activity was documented for 29.0% of patient visits and functional status in 74.7%; neither had any significant relationship to mean TSS change (both P > 0.10).

Conclusion

The documentation of RA QMs was infrequent and not associated with radiographic outcomes over 24 months.     

Feasibility and construct validity of the parent willingness-to-pay technique for children with juvenile idiopathic arthritis

OBJECTIVE: To assess the feasibility and construct validity of the willingness-to-pay (WTP) technique for measuring health care preferences in families of children with juvenile idiopathic arthritis (JIA). METHODS: Parents were asked to estimate the monthly US dollar amount they would be willing to pay to obtain for their child the following hypothetical drugs: ARTHRO, which guarantees complete clinical response; and NO-STOM-ACHE, a drug that eliminates gastrointestinal (GI) symptoms. A yes/no question was used with random assignment of the starting bids. Parents who agreed to pay the starting bid were then asked whether they would be willing to pay 200% and then 400% of this initial bid. Socioeconomic data and information on medications, disease activity, patient physical function, wellbeing, and health-related quality of life (HRQOL) were obtained. RESULTS: Sixty-two families of children with JIA were interviewed. GI symptoms were present in 54%, and 53% of the children had joints with active arthritis or limited range of motion. Four parents (7%) were unwilling to pay anything for any of the studied medications. The mean amount (median; mean percentage of available family income) families were willing to pay was $395 ($300; 15%) for ARTHRO and $109 ($80; 4%) for NO-STOM-ACHE. Correlation and regression analysis supported that, adjusted for the available family income, the WTP for ARTHRO was associated with disease activity, pain, and the HRQOL of the patients. After correction for the starting bids and the available family income, the WTP for NO-STOM-ACHE was associated with the patient's HRQOL, pain, and the amount of GI discomfort. CONCLUSION: The WTP technique is feasible and has construct validity for measuring health care preferences for children with JIA. Relatively large WTP estimates support a possible important negative impact of the disease on families of children with JIA.     

Sulphasalazine in the treatment of children with chronic arthritis

The ainich of study was to investigate the efficacy and toxicity of sulphasalazine (SASP) in the treatment of children with chronic arthritis. The medical records of 36 children (25 boys, 11 girls) who received SASP for the treatment of chronic arthritis were reviewed. Twenty-one patients had juvenile spondyloarthropathies (JSA) (eight juvenile ankylosing spondylitis (JAS), 13 undifferentiated JSA (uJSA) and 15 had juvenile rheumatoid arthritis (JRA). The patients received SASP therapy for a mean of 2.5 years (range 3 weeks to 8.1 years). Clinical and laboratory data were reviewed retrospectively to determine the effects of treatment. A clinically significant response occurred in 23 (64%) children: remission in 14 (39%) (JRA 5, JSA 9) and improvement (25% reduction in joint count) in nine (25%) (JRA 4, JSA 5). There was no difference in response rate between JR and JSA patients (p=0.11), but the time to remission shorter in JSA patients (mean 5 months) JRA patients (mean 25 months) (p=0.024). Twelve of the 36 patients discontinued non-steroidal anti-inflammatory drugs, and six of eight patients discontinued prednisolone. A significant fall in erythrocyte sedimentation rate and rise in haemoglobin occurred in SASP-treated patients (p<0.005) comparing most recent results with pretreatment levels. Side-effects occurred in four of 36 patients (11%); only one patient who had persisting severe diarrhoea required discontinuation of SASP. It was concluded that SASP appears to be effective and safe in the treatment of JRA and JSA patients. As a second-line agent, SASP is the drug of first choice for patients with JSA; for JRA patients SASP may be a useful, possibly less toxic alternative to methotrexate.     

Well-being in children with juvenile chronic arthritis

OBJECTIVE: The aim of this study was to describe a model for predicting well-being in children with juvenile chronic arthritis (JCA). METHODS: 125 children (43 boys) (median age 14.1 yrs; range 10.3-17.8) rated disability and discomfort (Childhood Health Assessment Questionnaire). Pain control, pain reduction and fatigue were evaluated (visual analogue scales). In addition, variation of pain intensity was rated by a pain intensity scale. Analysis by the stepwise regression technique was used to explain the variability in well-being. Eight independent variables were included as possible predictors in the model (p < 0.1). RESULTS: The analyses indicated that well-being in children with JCA is related to three clusters of variables; pain "as it normally is", number of pain-free days and attending physical education classes. The analysis explained a substantial portion of the total variance in the children's well-being (55.1%). CONCLUSION: Pain is a robust predictor of well-being in children with JCA. This supports the concept of the benefits of reducing chronic joint pain as a major goal in caring of these children.     

Health-related quality of life in children with sickle cell disease: child and parent perception

Health-related quality of life (HRQL) is an outcome that may be used to measure the impact of sickle cell disease on the child and their family but has not been routinely assessed in this disease. The objective of this study was to describe the HRQL of children with sickle cell disease as reported by the parent and the child, to compare the relationship between the two, and to determine the association of parent, child and disease characteristics on HRQL. Ninety-five parents completed the Child Health Questionnaire (CHQ)-Parent Form28 and 53 children completed the CHQ-Child Form87. Compared with the child report, parents reported worse HRQL in the overall perception of health, physical functioning, behaviour and self esteem domains of HRQL (P < 0.005). Parent and child reports of HRQL correlated strongly in assessment of the impact of bodily pain (r = 0.58) on HRQL and moderately in physical functioning (r = 0.44), behaviour (r = 0.45), general health (r = 0.44), self esteem (r = 0.40) and changes in health (r = 0.33) domains. Disease status, neurobehavioral co-morbidities, and parent education were associated with the HRQL of the child. Both the parent and child perspectives are needed to fully understand the impact of sickle cell disease on the HRQL of the child and effect of this disease on the family.     

Health-related quality of life in children with arthritis

Health status results from an individual's values placed on the interactions among a person's physical state, associated mental perceptions and emotions, resulting behaviors, and environment. Improving health-related quality of life is the goal of all disease treatment. This article reviews the components of health status, summarizes relevant studies in children with rheumatic and related diseases, and considers the role that future studies will play in improving care for children with rheumatic diseases.     

The uveitis and its relationship with disease activity and quality of life in Moroccan children with juvenile idiopathic arthritis

The aim of our study is to investigate ocular involvement in juvenile idiopathic arthritis (JIA) and its relationship with disease activity and quality of life in Moroccan patients who suffer from JIA. This is a cross-sectional study conducted between January and June 2012 which includes patients with juvenile idiopathic arthritis (n?=?30). All patients have undergone clinical and paraclinical assessment of JIA and a complete eye examination. Functional impairment is assessed by the Childhood Health Assessment Questionnaire while visual function is studied by the Effect of Youngsters’ Eyesight in Quality of Life instrument (EYE-Q). Quality of life is assessed using the Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0). Four patients (13.33 %) have uveitis with a confidence interval between 3.4 and 30.7. Involvement is bilateral in three children (75 %). One patient (25 %) has elevated intraocular pressure with loss of the right eye due to glaucoma. There is a strong but not significant relationship between uveitis and the number of awakenings (r?=?0.71, p?=?0.69) and morning stiffness (r?=?3.05, p?=?0, 21). This relationship is moderate with erythrocyte sedimentation rate (r?=?0.48, p?=?0.78) and C-reactive protein (r?=?0.25, p?=?0.88). A strong but not significant association is found between the overall quality of life assessed by the PedsQL 4.0 and visual function assessed by EYE-Q in the uveitis group (r?=??0.64, p?=?0.55). This study suggests that uveitis associated with JIA can present serious complications and could have a direct relationship with the activity of the JIA as well as with the quality of life of the patient.     

Sleep and its relationship to health-related quality of life in children and adolescents with inactive juvenile idiopathic arthritis

Aim of the work

To describe and compare sleep disturbance in children and adolescents with inactive juvenile idiopathic arthritis (JIA) and to study their relation to health-related quality of life (HRQoL).

Synovial fluid levels of E-selectin and intercellular adhesion molecule-1: relationship to joint inflammation in children with chronic arthritis

E-selectin and intercellular adhesion molecule (ICAM)-1 are crucial to the inflammatory response in chronic inflammatory arthritis. Soluble (s) levels of these molecules in sera and synovial fluid (SF) correlate with some clinical parameters and synovial tissue expression of the same molecules in rheumatoid arthritis. Studies of sera from children with chronic inflammatory arthritis corroborate this information; corresponding SF data are relatively lacking. We thus studied SF sE-selectin and sICAM-1 in 28 children with active juvenile rheumatoid arthritis or a spondyloarthropathy. Levels were correlated with erythrocyte sedimentation rate (ESR), SF leukocyte counts, duration of disease, and duration of response to concomitant intra-articular corticosteroid injection. Levels were compared according to use of methotrexate and/or sulfasalazine. Synovial fluid sE-selectin correlated with ESR and SF leukocyte counts. There was a trend toward lower sICAM-1 in patients treated with sulfasalazine and/or methotrexate. We conclude that SF levels of sE-selectin accurately reflect intra-synovial inflammation. Soluble ICAM-1 levels may reflect the effects of disease-modifying agents.     

Juvenile chronic arthritis and imaging: comparison of different techniques

OBJECTIVE: The aim of this study was to compare imaging findings obtained with different techniques in a patient with juvenile chronic arthritis. METHODS: The patient was a 12 years-old child with a 7-months history of arthritis of the first metatarsophalangeal joint of the right foot. The involved area was explored with the following imaging techniques: X-ray, technetium bone scintigraphy, magnetic resonance, gray-scale and power-Doppler ultrasonography. RESULTS: No abnormalities were detected with conventional X-ray. Scintigraphy showed an abnormal uptake of the radionuclide in the first metatarsophalangeal joint of the right foot. Magnetic resonance without contrast revealed clearly evident features of an active process of synovitis. Ultrasonography was able to detect the presence of joint effusion, synovial proliferation, bone erosion of the first metatarsal head. Power-Doppler examination revealed evident signs of soft tissue hyperaemia. CONCLUSIONS: Comparative assessment of different imaging techniques in this patient with recent-onset juvenile chronic arthritis indicates that high resolution ultrasonography provides the most detailed evaluation of the joint involvement with respect to the other imaging techniques.     

The relationship between very-low-density lipoprotein lipid and measures of carbohydrate metabolism in children with different lipoprotein profiles: Bogalusa Heart Study

The relationship of VLDL lipid (cholesterol and triglycerides) levels to fasting and postglucose plasma glucose, plasma glucose, insulin, and free fatty acid (FFA) levels were examined in four subgroups of children (n = 311, ages 6 to 18 years) from a total biracial population whose earlier beta- or pre-beta-lipoprotein cholesterol levels (or both) were in the extreme quintiles or quartiles. High beta-lipoprotein cholesterol strata with or without elevated pre-beta-lipoprotein cholesterol showed significantly high levels of FFA and glucose response (mean, 30 and 60 minutes) to oral glucose load, whereas postglucose insulin responses were markedly higher in the high pre-beta-lipoprotein cholesterol strata. VLDL triglycerides related closely with fasting plasma glucose levels (r = 0.53 to 0.60, P less than 0.001) and to a lesser extent with postglucose plasma glucose response (r = 0.37 to 0.44, P less than 0.001) in all cases. For insulin and FFA, however, correlations were significant only in certain subgroups. Similar relationships were noted for VLDL cholesterol. Measurements relating to carbohydrate tolerance, age, and race accounted for 35% to 48% of the variability in VLDL lipid values. Surprisingly, fasting plasma glucose showed the highest partial regression coefficient for VLDL lipid in all subgroups except high pre-beta-lipoprotein cholesterol and low beta-lipoprotein cholesterol category, in which age was the major predictor variable. These results demonstrate that subtle abnormalities in the above-mentioned metabolic interrelationships are established early in life.     

Recombinant human erythropoietin improves health-related quality of life in patients with rheumatoid arthritis and anaemia of chronic disease; utility measures correlate strongly with disease activity measures

Treatment with recombinant human erythropoietin (r-hu-Epo) in patients with rheumatoid arthritis (RA) and anaemia of chronic disease (ACD) resulted in improvement of both anaemia and disease activity. Utilities represent a generic and comprehensive quality of life measure, capable of integrating domain-specific information into one overall value which a patient assigns to his state of health. Therefore, the effect of r-hu-Epo on quality of life was studied by measuring utilities, derived from the rating scale and standard gamble, in a 52-week placebo-controlled randomised double-blind study with r-hu-Epo in 70 patients with active RA and ACD. Furthermore, the relation between anaemia as assessed by haemoglobin levels (Hb), disease activity as assessed with the Disease Activity Score (DAS), and utilities was investigated. Compared to the placebo group, significant improvement of Hb (P<0.001), DAS (P = 0.01) and rating scale utilities (P = 0.002), but not of standard gamble utilities, was observed in the Epo group. Rating scale utilities correlated strongly with DAS (r = –0.47, P<0.01), Hb (r = 0.37, P<0.01) and changes in both DAS (r = –0.74, P<0.01) and Hb (r = 0.44, P<0.01). Both DAS and Hb contributed significantly to the variance in rating scale utilities (21% and 3% respectively) and to changes in rating scale utilities (43% and 3% respectively). Standard gamble utilities correlated less well with clinical disease variables than rating scale utilities did. These results indicate, that r-hu-Epo improves utility-derived health-related quality of life, most probably by improving both disease activity and anaemia. Utilities, particularly rating scale utilities, correlated well with conventional disease activity variables and proved sensitive to change. Utilities may be a useful tool for investigating quality of life in RA-patients. Received: 16 December 1998 / Accepted: 5 March 1999     

Assessment of the reporting of quality and outcome measures in hepatic resections: a call for 90-day reporting in all hepatectomy series

BackgroundThe aim of this paper is to assess the current state of quality and outcomes measures being reported for hepatic resections in the recent literature.MethodsMedline and PubMed databases were searched for English language articles published between 1 January 2002 and 30 April 2013. Two examiners reviewed each article and relevant citations for appropriateness of inclusion, which excluded papers of liver donor hepatic resections, repeat hepatectomies or meta-analyses. Data were extracted and summarized by two examiners for analysis.ResultsFifty-five studies were identified with suitable reporting to assess peri-operative mortality in hepatic resections. In only 35% (19/55) of the studies was the follow-up time explicitly stated, and in 47% (26/55) of studies peri-operative mortality was limited to in-hospital or 30 days. The time period in which complications were captured was not explicitly stated in 19 out of 28 studies. The remaining studies only captured complications within 30 days of the index operation (8/28). There was a paucity of quality literature addressing truly patient-centred outcomes.ConclusionQuality outcomes after a hepatic resection are inconsistently reported in the literature. Quality outcome studies for a hepatectomy should report mortality and morbidity at a minimum of 90 days after surgery.     

Bone mineral density in children with juvenile chronic arthritis

The aim of this study was to evaluate bone mineral density changes in patients with juvenile chronic arthritis (JCA) and to determine the most likely causes of osteoporosis in these patients. Eighteen (11 male, 7 female) patients suffering from JCA and 14 healthy controls (10 male, four female) were included in this study. The mean age of the patients and control groups were 11.0±3.2 and 10.9±2.9 years respectively. Disease activity was determined by clinical and laboratory evaluation and Articular Disease Severity Score (ADSS). Bone mineral density (BMD) of the femoral neck and lumbar spine was measured by dual photon absorptiometry.BMD of the patients at the lumbar spine was significantly lower than the control group (p<0.05). This difference was more marked in patients treated with steroids. Femoral neck BMD was also lower in the patient group but this difference was not statistically significant. There was a negative correlation between ADSS and BMD at the spine. In conclusion, trabecular bone loss is characteristic for osteoporosis in JCA. Our results indicate that steroid treatment and disease severity are important factors in the development of osteoporosis in JCA.     

Comparing two measures of quality of life for children with haemophilia: the CHO-KLAT and the Haemo-QoL

Summary. Disease‐specific measures of quality of life (QoL) for children with haemophilia are now available for use in clinical studies [Haemophilia, 10 , 2004, 9–16]. One of these measures, the Canadian Haemophilia Outcomes – Kids’ Life Assessment Tool (CHO‐KLAT), was developed in Canada with emphasis on the perspectives of children [Pediatr Blood Cancer, 47 , 2006, 305–11; Haemophilia, 10 , 2004, 34–43]. Another, the Haemo‐QoL, was developed in Europe, with emphasis on the perspectives of clinicians [Haemophilia, 8 , 2002, 47–54; Haemophilia, 10 , 2004, 17–25]. While these two measures are unique and independent, researchers from both studies were collaboratively linked throughout development and testing. This study presents the results of a joint assessment of the two measures with respect to their strengths, limitations and unique contributions. The primary questions addressed were: 1 What is the relationship between the CHO‐KLAT and the Haemo‐QoL in terms of summary scores and item content? 2 What are the methodological strengths, limitations and unique contributions of each measure? We conducted a retrospective analysis of data from field testing of both measures. The analysis included a comparative assessment of the basic validity, reliability and items used in each measure. Overall, the CHO‐KLAT and the Haemo‐QoL are promising and valuable measures of QoL for children with haemophilia. Our analyses confirmed the basic psychometric properties of both tools, but identified some discrepancies between them. Additional data will allow for greater understanding of these discrepancies and lend clarity to how the tools should be used in clinical studies (separately or merged). The present recommendation is that the measures be run independently, but preferably concurrently in studies of children with haemophilia.     

Mortality in rheumatoid arthritis: relationship to single and composite measures of disease activity

BACKGROUND: Rheumatoid arthritis (RA) is a heterogeneous disease characterized by a variable course of remissions and relapses. Single measures of disease activity at only one point in time may not reflect the overall control of disease activity. OBJECTIVE: The aim was to determine (i) the predictive value of 20 baseline demographic and disease variables on mortality, and (ii) the relationship between serial measures of the Stoke index (SI; a validated index of disease activity in RA) and mortality in RA. METHODS: Mortality in 309 RA patients followed up for a median of 14 yr was analysed retrospectively. The standardized mortality ratio (SMR) was calculated for all causes of death. The predictive values of baseline and time-integrated variables were assessed using multivariate Cox proportional hazards regression analysis. RESULTS: The SMR was 1.65. At baseline, only nodules, erosions, RA latex titre, white cell count and globulin level were predictive of mortality after correction for age, sex and disease duration. Using a stepwise Cox proportional hazards regression model, the most powerful predictors of mortality were age, nodules and RA latex titre. Individual measures of disease activity and the SI at baseline were not predictive of mortality. However, the mean level of the SI over 12 months was related to mortality (P=0.039). CONCLUSIONS: At baseline, the demographic and disease variables most significantly related to mortality in RA are age, nodules and RA latex titre. Individual measures of disease activity at a single point in time are poor predictors of mortality in RA. However, measurement of the mean level of disease activity over time using the composite SI has a significant relationship with mortality. A high level of sustained inflammation appears to be an important predictor of premature death.