The Cutaneous Assessment Tool: development and reliability in juvenile idiopathic inflammatory myopathy.

OBJECTIVES: Clinical care and therapeutic trials in idiopathic inflammatory myopathies (IIM) require accurate and consistent assessment of cutaneous involvement. The Cutaneous Assessment Tool (CAT) was designed to measure skin activity and damage in IIM. We describe the development and inter-rater reliability of the CAT, and the frequency of lesions endorsed in a large population of juvenile IIM patients. METHODS: The CAT includes 10 activity, 4 damage and 7 combined lesions. Thirty-two photographic slides depicting IIM skin lesions were assessed by 11 raters. One hundred and twenty-three children were assessed by 11 paediatric rheumatologists at 10 centres. Inter-rater reliability was assessed using simple agreements and intra-class correlation coefficients (ICC). RESULTS: Simple agreements in recognizing lesions as present or absent were generally high (0.5-1.0). ICCs for CAT lesions were moderate (0.4-0.75) in both slides and real patients. ICCs for the CAT activity and damage scores were 0.71 and 0.81, respectively. CAT activity scores ranged from 0 to 44 (median 7, potential range 0-96) and CAT damage scores ranged from 0 to 13 (median 1, potential range 0-22). The most common cutaneous lesions endorsed were periungual capillary loop changes (63%), Gottron's papules/sign (53%), heliotrope rash (49%) and malar/facial erythema (49%). CONCLUSIONS: Total CAT activity and damage scores have moderate to good reliability. Assessors generally agree on the presence of a variety of cutaneous lesions. The CAT is a promising, semi-quantitative tool to comprehensively assess skin disease activity and damage in IIM.

Validation of the Childhood Health Assessment Questionnaire in the juvenile idiopathic myopathies. Juvenile Dermatomyositis Disease Activity Collaborative Study Group.

OBJECTIVE: To examine the validity of the Childhood Health Assessment Questionnaire (CHAQ) in patients with juvenile idiopathic inflammatory myopathy (IIM). METHODS: One hundred fifteen patients were enrolled in a multicenter collaborative study, during which subjects were assessed twice, 7-9 months apart. Physical function was measured using the CHAQ. Internal reliability was assessed using adjusted item-total correlations and item endorsement rates. Construct validity was assessed by comparing predicted and actual correlations of the CHAQ with other measures of physical function and disease activity. Responsiveness was assessed by calculating effect size (ES) and standardized response mean (SRM) in a group of a priori defined "improvers." RESULTS: Item-total correlations were high (rs range = 0.35-0.81), suggesting all items were related to overall physical function. Manual muscle testing and the Childhood Myositis Assessment Scale correlated moderate to strongly with the CHAQ (r = -0.64 and -0.75, both p < 0.001). Moderate correlations were also seen with the physician global assessment of disease activity (rs = 0.58, p < 0.001), parent global assessment of overall health (rs = -0.65, p < 0.001), Steinbrocker function class (rs = 0.69, p < 0.001), and global skin activity (rs = 0.40, p < 0.001), while global disease damage and skin damage had low correlations (rs = 0.13 and 0.07, p > or =0.17). Responsiveness of the CHAQ was high, with ES = 1.05 and SRM = 1.20. CONCLUSION: In this large cohort of patients with juvenile IIM, the CHAQ exhibited internal reliability, construct validity, and strong responsiveness. We conclude that the CHAQ is a valid measure of physical function in juvenile IIM, appropriate for use in therapeutic trials, and potentially in the clinical care of these patients.

Overview and report on international registry of sarcoid arthritis in childhood.

Interest in childhood sarcoidosis prompted the formation of an international registry in 1991. Over the next 5 years, 53 patients were registered by 23 physicians from 14 countries. All the patients had definite histologic evidence of sarcoidosis: noncaseating granulomas of the skin (31), synovium (15), liver (10), lymph node (eight), lung (five), muscle (four), conjunctiva (three), or kidney (one). All but nine patients developed polyarthritis; 38 of 44 had persistent arthritis. Of those with persistent polyarthritis, arthritis occurred at presentation in 16 of 38 patients and inflammation of the uveal tract of the eye occurred in 44 with involvement of both anterior and posterior segments in 21. One patient was blind at the time of the report. Other ocular complications included chorioretinitis, glaucoma, and phthisis bulbi. Laboratory abnormalities included mild anemia and elevated erythrocyte sedimentation rate (39 out of 45). Angiotensin converting enzyme levels were elevated in 14 out of 37 patients. Information on these patients with sarcoidosis helps develop a better understanding of this rare childhood disease. These patients are discussed in conjunction with an overview of sarcoid arthropathy.

The pain puzzle: a visual and conceptual metaphor for understanding and treating pain in pediatric rheumatic disease.

Chronic pain is a primary clinical manifestation of pediatric rheumatic diseases that for some children persists into adulthood and is associated with increased disability. The "pain puzzle" is presented as a visual and conceptual metaphor for understanding and treating pediatric rheumatic disease related pain. This metaphor is consistent with a biobehavioral model of pain that focuses on the unique and interactive components of nociceptive activity, emotions, cognitions, and behavior in the experience of pain. We describe the parts of the pain puzzle and review the implications for treating pediatric rheumatic disease related pain.

Development of validated disease activity and damage indices for the juvenile idiopathic inflammatory myopathies. II. The Childhood Myositis Assessment Scale (CMAS): a quantitative tool for the evaluation of muscle function. The Juvenile Dermatomyositis Disease Activity Collaborative Study Group.

OBJECTIVE: To develop, validate, and determine the measurement characteristics of a quantitative tool for assessing the severity of muscle involvement in children with idiopathic inflammatory myopathies. METHODS: The Childhood Myositis Assessment Scale (CMAS) was developed from 2 existing observational functional assessment tools to assess muscle function in the areas of strength and endurance across a wide range of ability and ages. The 14 ordinal items included were chosen to assess primarily axial and proximal muscle groups and are ranked with standard performance and scoring methods. Following the development of the CMAS, a training video and written instructions were developed and reviewed by the physicians participating in this study. Subsequently, utilizing a randomized block design, 12 physicians independently scored 10 children (9 with dermatomyositis, 1 with polymyositis; ages 4-15 years) twice in one day (morning and afternoon) on the CMAS. A pediatric physical therapist performed quantitative manual muscle strength testing (MMT) twice on each child (morning and afternoon), including the neck, trunk, and proximal and distal extremity muscle groups. RESULTS: The CMAS has a potential range of 0-51, with higher scores indicating greater muscle strength and endurance. The observed mean for the 10 patients was 36.4 (median 44, SD 14.1, observed range 5-51). The total score for the CMAS correlated with the physician's global assessment (by visual analog scale) of disease activity, the MMT score, serum creatine kinase level, and the Juvenile Arthritis Functional Assessment Report score. The score on the CMAS was not correlated with patient age. Interrater reliability (Kendall's coefficient of concordance) ranged from 0.77 to 1.0 for individual items (all P < 0.001), and overall, it was 0.95 (P < 0.001). Intrarater reliability for the individual physicians was measured by correlation of the CMAS scores for each patient on 2 separate evaluations and ranged from 0.97 to 0.99, with an overall correlation for all physicians of 0.98 (all P < 0.001). CONCLUSION: The CMAS demonstrated an acceptable range of observed scores, excellent convergent validity, and excellent inter- and intrarater reliability. The CMAS is validated to quantitatively assess muscle function in the areas of strength and endurance in children with idiopathic inflammatory myopathies. It can be used in routine clinical care as well as therapeutic trials.

Development of validated disease activity and damage indices for the juvenile idiopathic inflammatory myopathies: I. Physician, parent, and patient global assessments. Juvenile Dermatomyositis Disease Activity Collaborative Study Group.

OBJECTIVE: To determine the reliability, content validity, and responsiveness of physician global assessments of disease activity and damage in the juvenile idiopathic inflammatory myopathies (IIM), and to investigate concordance among physician, parent, and patient global ratings. METHODS: Sixteen pediatric rheumatologists rated 10 juvenile IIM paper patient cases for global disease activity and damage, and assessed the importance of 51 clinical and laboratory parameters in formulating their global assessments. Then, 117 juvenile IIM patients were enrolled in a protocol to examine the relationship between Likert and visual analog scale global assessments, their sensitivity to change, and the comparability of physician, parent, and patient global ratings. RESULTS: Pediatric rheumatologists demonstrated excellent interrater reliability in their global assessments of juvenile IIM disease activity and damage (97.7% and 94.7% agreement among raters, respectively), and agreed on a core set of clinical parameters in formulating their judgments. Likert scale ratings correlated with those on a visual analog scale, and both were comparable in responsiveness (standardized response means -0.56 for disease activity, 0.02 [Likert] and 0.14 [visual analog] for damage, measured over 8 months). Parent global ratings of disease activity correlated with physician assessments, but were not colinear (Spearman's correlation [r] = 0.41-0.45). Patient global disease activity assessments correlated with those done by parents (r = 0.57-0.84) and physicians (r = 0.37-0.63), but demonstrated less responsiveness (standardized response means -0.21 and -0.12, respectively, over 8 months). CONCLUSION: Physician global assessments of juvenile IIM disease activity and damage demonstrated high interrater reliability and were shown to be comprehensive measures. Both physician and parent disease activity assessments should be considered valuable as quantitative measures for evaluating therapeutic responses in juvenile IIM patients.

Effects of perceived stress on pediatric chronic pain.

The dearth of theoretically driven research on the predictors of pediatric chronic pain may unwittingly contribute to needless suffering in children and adolescents by underinvestigating a potentially treatable condition. The objective of the present study was to investigate the hypothesized predictive effects of perceived stress on pediatric chronic pain intensity in 148 children and adolescents. Consistent with the a priori Biobehavioral Model of Pediatric Pain, higher perceived stress was predictive of greater pediatric pain intensity. The results are discussed with regard to the implications for cognitive-behavioral pediatric pain treatment.

Legal rights of children with musculoskeletal disabilities.

As market forces increasingly control medicine, we must not forget the disabled, especially children. Although the costs of appropriate comprehensive services are substantial, it is well worth the cost to facilitate an adulthood in which these patients are on their own, possibly earning their livelihood, and experiencing the best quality of life our society can provide.

Chronic pain and emotional distress in children and adolescents.

Pediatric chronic pain continues to be relatively underinvestigated and undertreated. The objective of the present cross-sectional study was to investigate the emotional distress hypothesized to be concurrently associated with the chronic pain experience in children and adolescents. One hundred and sixty children and adolescents with chronic pain and their parents completed standardized assessment instruments measuring pain intensity, depressive symptoms, state anxiety, trait anxiety, general self-esteem, and internalizing and externalizing behavior problems. Consistent with the a priori Biobehavioral Model of Pediatric Pain, higher patient-perceived pain intensity was associated with higher depressive and anxious symptoms, lower general self-esteem, and higher behavior problems. The results are discussed in regard to preventing and treating pain and suffering in children and adolescents with chronic pain.

Assessing chronic musculoskeletal pain associated with rheumatic disease: further validation of the pediatric pain questionnaire.

Evaluated the validity, stability, and clinical utility of the Pediatric Pain Questionnaire (PPQ), a comprehensive, multidimensional instrument for assessing childhood pain. Previous studies demonstrated adequate psychometric properties of the PPQ using small samples. Results of the current study, using a large sample (N = 100) of children and adolescents with chronic pain associated with rheumatic disease, were consistent with initial validation studies. High correlations were found among visual analog scale (VAS) pain intensity ratings obtained from parents, physicians, and patients. Significant correlations also were found between pain ratings and measures of disease activity and functional status. VAS ratings evidenced moderate stability over a 6-month period in this sample of youth with relatively stable disease activity. Other components of the PPQ provide comprehensive information that is clinically useful for treatment planning and evaluation.

Juvenile rheumatoid arthritis and spondyloarthropathies.

Further insight into the etiology and pathogenesis of juvenile rheumatoid arthritis (JRA) is presented in recent immunogenetic studies, particularly the allele associations of the pauciarticular pattern of disease. Evidence suggests that bacterial heat-shock proteins may be significant in the chronic inflammatory response in children with arthritis. Data on the role of complement activation and cytokines and their receptors also are presented. Coagulopathy in JRA may have more than one etiologic factor, including a viral agent, as may the disease itself. In the treatment of growth abnormalities in JRA, the neuroendocrine system, recombinant growth hormone, intravenous iron therapy, and nutritional supplementation are all areas of recent investigation. In outcome studies, ocular involvement and the presence of circulating IgM rheumatoid factor appear to be risk factors for disability. However, disease of less than 2 years' duration and absence of radiographic lesions likely predict good response to methotrexate therapy.

Evaluation of growth hormone secretion in children with juvenile rheumatoid arthritis and short stature.

Children with juvenile rheumatoid arthritis (JRA) often exhibit delayed skeletal development. Previous evaluations of growth hormone (hGH) levels in these children have used single-value blood determinations. We sought to extend information on possible hGH deficiency in children with short stature and JRA by measuring 24-hour hGH pulsatile secretion. Five children with JRA were identified as having a height less than the 3rd percentile, and one child with a height at the 25th percentile. Three of these had abnormally low 24-hour serum hGH secretion. Two underwent a 24-month trial of human recombinant hGH; both exhibited only marginally accelerated growth. These results suggest that children with JRA and persistent short stature may have low hGH secretion without an adequate physiologic response to exogenous hGH administration.

Effects of nutritional supplementation on bone mineral status of children with rheumatic diseases receiving corticosteroid therapy.

OBJECTIVE: Because children with rheumatic disease receiving longterm corticosteroids are at high risk for developing osteoporosis, we attempted to determine whether nutritional supplementation would improve bone status in this group of children. METHODS: In a crossover design study, 10 corticosteroid treated children with rheumatic disease and osteoporosis received calcium and vitamin D supplementation for 6 months to determine their effect on bone density. They were then studied for 6 months without added nutrition supplements. The mean age was 13.1 years with a mean duration of disease of 4.2 years. Six patients had juvenile rheumatoid arthritis, 2 had systemic lupus erythematosus and 2 had mixed connective tissue disease. These children obtained a minimum of 1 g of calcium and 400 IU of vitamin D daily from diet and added supplements. Dual photon absorptiometry, laboratory and dietary data were obtained at baseline, 6 months, and one year. RESULTS: Spinal bone density significantly improved with supplementation. Osteocalcin values remained low throughout the study. CONCLUSION: Our results suggest some children with rheumatic disease receiving corticosteroids would benefit from calcium and vitamin D supplementation.

Uses of nonsteroidal anti-inflammatory drugs in pediatrics.

Nonsteroidal anti-inflammatory drugs have an expanding role in pediatrics. The indications include (1) treatment of childhood rheumatic disease and other chronic inflammatory conditions; (2) symptomatic treatment of clinical problems such as fever, musculoskeletal pain, or dysmenorrhea; and (3) induction of closure of patent ductus arteriosus. Nonsteroidal anti-inflammatory drugs are generally well tolerated with a low incidence of serious side effects. However, potentially serious gastrointestinal, hepatic, central nervous system, and renal adverse effects have occurred. The pediatrician should be familiar with the pharmacokinetics and dosages of the various nonsteroidal anti-inflammatory drugs as well as laboratory tests necessary to monitor their use. Guidelines are suggested.

The validity and reliability of parental ratings of disease activity in juvenile rheumatoid arthritis.

This study assessed the validity and reliability of parental ratings of morning stiffness, activity limitations, and pain complaints on a sample of 31 patients with juvenile rheumatoid arthritis (JRA). Parental ratings were found to be significant predictors of active joint counts, accounting for 40% of the variance in joint counts. Internal consistency reliability was moderately high (0.819), but test-retest reliability was moderate to low; this is not unexpected given the fluctuations in symptoms of JRA over time. The results suggest that parental ratings are valid and moderately reliable measures of disease activity in JRA. Regular monitoring of symptoms by parents could be a useful source of information for health care providers in making treatment decisions and can increase parental and patient involvement in the treatment process.

Multicentric osteolysis: an infrequently recognized renal-rheumatologic syndrome.

We describe the case of a young girl with Type III multicentric osteolysis, a rare syndrome characterized by osteolysis and renal insufficiency. Our report is intended to make physicians aware of this uncommon cause of renal and rheumatologic abnormalities.

Seronegative juvenile rheumatoid arthritis and mast cell-associated gastritis.

We describe 4 children with seronegative inflammatory arthritis who had persistent, severe nausea and abdominal pain over several months, in spite of vigorous medical therapy, including antacids and histamine H2 receptor antagonists. Endoscopy and biopsy of gastric and duodenal mucosa showed antral gastritis and an increased number of mast cells in 3 of the 4 patients. In the fourth patient, urinary histamine levels were elevated. These findings suggest an association between inflammatory arthritis and localized mast cell disease in some individuals. Further studies are needed to determine whether this association represents an independent syndrome or whether mast cell-related disease is secondary to long-term treatment with nonsteroidal antiinflammatory drugs in children with mild arthritis.