Kenneth N. Schikler

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

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 physicians 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 (Kendalls 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.

Endometriosis in association with uterine anomaly

Endometriosis is frequently a chronic process, which may begin soon after menarche. The process may be enhanced by mechanical obstruction. Theories of retrograde menstruation and metaplasia still remain in vogue. Endometriosis is a cause of both acute and chronic pelvic pain in the adolescent. We present case reports of müllerian lateral wall fusion defects with surgical correction and evidence for resorption of endometriosis. Clinicians must be aware that patients with uterine anomalies may develop extensive endometriosis, which upon creation of an unobstructed outflow tract results in complete resorption. Furthermore, the mechanism of formation of endometriosis in association with an outflow tract obstruction may be very different from that associated with infertility. We recommend consideration of endometriosis and/or a reproductive tract abnormality in the adolescent with persistent pelvic pain.

Consensus treatment plans for new-onset systemic juvenile idiopathic arthritis.

There is wide variation in therapeutic approaches to systemic juvenile idiopathic arthritis (JIA) among North American rheumatologists. Understanding the comparative effectiveness of the diverse therapeutic options available for treatment of systemic JIA can result in better health outcomes. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans and standardized assessment schedules for use in clinical practice to facilitate such studies.

Cognitive behavioral therapy for the treatment of juvenile fibromyalgia: A multisite, single-blind, randomized, controlled clinical trial

OBJECTIVE Juvenile fibromyalgia syndrome (FMS) is a chronic musculoskeletal pain disorder in children and adolescents for which there are no evidence-based treatments. The objective of this multisite, single-blind, randomized clinical trial was to test whether cognitive-behavioral therapy (CBT) was superior to fibromyalgia (FM) education in reducing functional disability, pain, and symptoms of depression in juvenile FMS. METHODS Participants were 114 adolescents (ages 11-18 years) with juvenile FMS. After receiving stable medications for 8 weeks, patients were randomized to either CBT or FM education and received 8 weekly individual sessions with a therapist and 2 booster sessions. Assessments were conducted at baseline, immediately following the 8-week treatment phase, and at 6-month followup. RESULTS The majority of patients (87.7%) completed the trial per protocol. Intent-to-treat analyses showed that patients in both groups had significant reductions in functional disability, pain, and symptoms of depression at the end of the study, and CBT was significantly superior to FM education in reducing the primary outcome of functional disability (mean baseline to end-of-treatment difference between groups 5.39 [95% confidence interval 1.57, 9.22]). Reduction in symptoms of depression was clinically significant for both groups, with mean scores in the range of normal/nondepressed by the end of the study. Reduction in pain was not clinically significant for either group (<30% decrease in pain). There were no study-related adverse events. CONCLUSION In this controlled trial, CBT was found to be a safe and effective treatment for reducing functional disability and symptoms of depression in adolescents with juvenile FMS.

Changes in Pain Coping, Catastrophizing, and Coping Efficacy After Cognitive-Behavioral Therapy in Children and Adolescents With Juvenile Fibromyalgia

UNLABELLED A recent randomized multisite clinical trial found that cognitive-behavioral therapy (CBT) was significantly more effective than fibromyalgia education (FE) in reducing functional disability in adolescents with juvenile fibromyalgia (JFM). The primary objective of this study was to examine the psychological processes of CBT effectiveness by evaluating changes in pain coping, catastrophizing, and coping efficacy and to test these changes as mediators of continued improvements in functional disability and depressive symptoms at 6-month follow-up. One hundred adolescents (11-18 years old) with JFM completed the clinical trial. Coping, catastrophizing, and coping efficacy (Pain Coping Questionnaire) and the outcomes of functional disability (Functional Disability Inventory) and depressive symptoms (Childrens Depression Inventory) were measured at baseline, posttreatment, and 6-month follow-up. Participants in both conditions showed significant improvement in coping, catastrophizing, and efficacy by the end of the study, but significantly greater improvements were found immediately following treatment for those who received CBT. Treatment gains were maintained at follow-up. Baseline to posttreatment changes in coping, catastrophizing, and efficacy were not found to mediate improvements in functional disability or depressive symptoms from posttreatment to follow-up. Future directions for understanding mechanisms of CBT effectiveness in adolescents with chronic pain are discussed. PERSPECTIVE CBT led to significant improvements in pain coping, catastrophizing, and efficacy that were sustained over time in adolescents with juvenile fibromyalgia. Clinicians treating adolescents with JFM should focus on teaching a variety of adaptive coping strategies to help patients simultaneously regain functioning and improve mood.

Physical activity monitoring in adolescents with juvenile fibromyalgia: Findings from a clinical trial of cognitive–behavioral therapy

Juvenile fibromyalgia (JFM) is a chronic musculoskeletal pain condition that is associated with reduced physical function. Recent research has demonstrated that cognitive–behavioral therapy (CBT) is effective in improving daily functioning among adolescents with JFM. However, it is not known whether these improvements were accompanied by increased physical activity levels. Our objective was to analyze secondary data from a randomized clinical trial of CBT to examine whether CBT was associated with improvement in objectively measured physical activity and whether actigraphy indices corresponded with self‐reported functioning among adolescents with JFM.

Health‐related quality of life of patients with juvenile dermatomyositis: Results from the paediatric rheumatology international trials organisation multinational quality of life cohort study

OBJECTIVE To investigate the health-related quality of life (HRQOL) change over time, as measured by the Child Health Questionnaire (CHQ), and its determinants in patients with active juvenile dermatomyositis (DM). METHODS We assessed patients with juvenile DM at both baseline and 6 months of followup, and healthy children age < or =18 years. Potential determinants of poor HRQOL included demographic data, physicians and parents global assessments, muscle strength, functional ability as measured by the Childhood Health Assessment Questionnaire (C-HAQ), global disease activity assessments, and laboratory markers. RESULTS A total of 272 children with juvenile DM and 2,288 healthy children were enrolled from 37 countries. The mean +/- SD CHQ physical and psychosocial summary scores were significantly lower in children with juvenile DM (33.7 +/- 11.7 versus 54.6 +/- 4.1) than in healthy children (45.1 +/- 9.0 versus 52 +/- 7.2), with physical well-being domains being the most impaired. HRQOL improved over time in responders to treatment and remained unchanged or worsened in nonresponders. Both physical and psychosocial summary scores decreased with increasing levels of disease activity, muscle strength, and parents evaluation of the childs overall well-being. A C-HAQ score >1.6 (odds ratio [OR] 5.06, 95% confidence interval [95% CI] 2.03-12.59), childs overall well-being score >6.2 (OR 5.24, 95% CI 2.27-12.10), and to a lesser extent muscle strength and alanine aminotransferase level were the strongest determinants of poor physical well-being at baseline. Baseline disability and longer disease duration were the major determinants for poor physical well-being at followup. CONCLUSION We found that patients with juvenile DM have a significant impairment in their HRQOL compared with healthy peers, particularly in the physical domain. Physical well-being was mostly affected by the level of functional impairment.

Increased sensitivity of the European medicines agency algorithm for classification of childhood granulomatosis with polyangiitis.

Objective. Granulomatosis with polyangiitis (Wegener’s; GPA) and other antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAV) are rare in childhood and are sometimes difficult to discriminate. We compared use of adult-derived classification schemes for GPA against validated pediatric criteria in the ARChiVe (A Registry for Childhood Vasculitis e-entry) cohort, a Childhood Arthritis and Rheumatology Research Alliance initiative. Methods. Time-of-diagnosis data for children with physician (MD) diagnosis of AAV and unclassified vasculitis (UCV) from 33 US/Canadian centers were analyzed. The European Medicines Agency (EMA) classification algorithm and European League Against Rheumatism/Paediatric Rheumatology International Trials Organisation/Paediatric Rheumatology European Society (EULAR/PRINTO/PRES) and American College of Rheumatology (ACR) criteria for GPA were applied to all patients. Sensitivity and specificity were calculated (MD-diagnosis as reference). Results. MD-diagnoses for 155 children were 100 GPA, 25 microscopic polyangiitis (MPA), 6 ANCA-positive pauciimmune glomerulonephritis, 3 Churg-Strauss syndrome, and 21 UCV. Of these, 114 had GPA as defined by EMA, 98 by EULAR/PRINTO/PRES, and 87 by ACR. Fourteen patients were identified as GPA by EULAR/PRINTO/PRES but not by ACR; 3 were identified as GPA by ACR but not EULAR/PRINTO/PRES. Using the EMA algorithm, 135 (87%) children were classifiable. The sensitivity of the EMA algorithm, the EULAR/PRINTO/PRES, and ACR criteria for classifying GPA was 90%, 77%, and 69%, respectively, with specificities of 56%, 62%, and 67%. The relatively poor sensitivity of the 2 criteria related to their inability to discriminate patients with MPA. Conclusion. EULAR/PRINTO/PRES was more sensitive than ACR criteria in classifying pediatric GPA. Neither classification system has criteria for MPA; therefore usefulness in discriminating patients in ARChiVe was limited. Even when using the most sensitive EMA algorithm, many children remained unclassified.

Solvent abuse associated cortical atrophy

Eleven of 42 toluene inhalers were evaluated with computed tomography scans because of neurologic abnormalities. Six of the 11 were found to have cerebral cortical atrophy. In addition, two of the six had cerebellar atrophy. All six had been exposed to toluene for at least 10 years. This study suggests that atrophy of the central nervous system may occur in chronic toluene abusers.

The role of benign joint hypermobility in the pain experience in Juvenile Fibromyalgia: an observational study

BackgroundJuvenile Fibromyalgia (JFM) is characterized by chronic widespread musculoskeletal pain and approximately 40% of children and adolescents with JFM also suffer from benign joint hypermobility (HM). It is not currently known if the presence of HM affects the pain experience of adolescents with JFM. The objective of this study was to examine whether there were any differences in self-reported pain intensity and physiologic pain sensitivity between JFM patients with and without joint HM.MethodsOne hundred thirty-one adolescent patients with JFM recruited from four pediatric rheumatology clinics completed a daily visual analogue scale (VAS) pain rating for one week and underwent a standardized 18-count tender point (TP) dolorimeter assessment. Medical records were reviewed for the presence of joint HM. Average pain VAS ratings, tender point count and tender point sensitivity were compared between JFM patients with and without hypermobility (HM+ and HM-).ResultsNearly half (48%) the sample of JFM patients were found to be HM+. HM+ and HM- patients did not differ in their self-reported pain intensity. However, HM + patients had significantly greater pain sensitivity, with lower TP thresholds (p = 0.002) and a greater number of painful TPs (p = 0.003) compared to HM- patients.ConclusionThe presence of HM among adolescent patients with JFM appears to be associated with enhanced physiologic pain sensitivity, but not self-report of clinical pain. Further examination of the mechanisms for increased pain sensitivity associated with HM, especially in adolescents with widespread pain conditions such as JFM is warranted.