DOCSLIB.ORG

Juvenile Dermatomyositis: Novel Treatment Approaches and Outcomes

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Juvenile Dermatomyositis: Novel Treatment Approaches and Outcomes REVIEW CURRENT OPINION Juvenile dermatomyositis: novel treatment approaches and outcomes Giulia C. Varniera, Clarissa A. Pilkingtona, and Lucy R. Wedderburna,b,c Purpose of review The aim of this article is to provide a summary of the recent therapeutic advances and the latest research on outcome measures for juvenile dermatomyositis (JDM). 04/06/2021 on BhDMf5ePHKav1zEoum1tQfN4a+kJLhEZgbsIHo4XMi0hCywCX1AWnYQp/IlQrHD3i3D0OdRyi7TvSFl4Cf3VC4/OAVpDDa8KKGKV0Ymy+78= by http://journals.lww.com/co-rheumatology from Downloaded Downloaded Recent findings Several new international studies have developed consensus-based guidelines on diagnosis, outcome from measures and treatment of JDM to standardize and improve patient care. Myositis-specific antibodies http://journals.lww.com/co-rheumatology together with muscle biopsy histopathology may help the clinician to predict disease outcome. A newly developed MRI-based scoring system has been developed to standardize the use of MRI in assessing disease activity in JDM. New data regarding the efficacy and safety of rituximab, especially for skin disease, and cyclophosphamide in JDM support the use of these medications for severe refractory cases. Summary International network studies, new biomarkers and outcome measures have led to significant progress in by BhDMf5ePHKav1zEoum1tQfN4a+kJLhEZgbsIHo4XMi0hCywCX1AWnYQp/IlQrHD3i3D0OdRyi7TvSFl4Cf3VC4/OAVpDDa8KKGKV0Ymy+78= understanding and managing the rare inflammatory myositis conditions such as JDM. Keywords advanced treatment, juvenile dermatomyositis, outcome measures INTRODUCTION To overcome these issues, these two international Juvenile dermatomyositis (JDM) is a rare systemic organizations joined forces and developed a new set autoimmune disease characterized by a vasculopathy of consensus-driven response criteria for adult that primarily affects muscle and skin, but may dermatomyositis/polymyositis and children with involve the lung, bowel, heart and other organs JDM. This new tool is based on a continuous model, [1,2]. JDM is the most common inflammatory myop- with a total improvement score of 0–100, and with athy of childhood, affecting 1.9 cases per million different thresholds for minimal (30), moderate children in the United Kingdom [3] and 2.4–4.1 cases (45) and major (70) clinical response based on weighted scores applied to an absolute percentage per million children in USA [4]. In this review, we will && summarize the recent developments in the clinical improvement [8 ]. The core set measures were iden- assessment, treatments and outcomes in JDM. tified by consensus among expert paediatric and adult rheumatologists, neurologists and dermatolo- gists, using the Delphi method. The agreed measures CLINICAL OUTCOMES AND CORE SET were the following: Physician global activity; Parent CRITERIA or Patient global activity; Manual Muscle testing on 04/06/2021 International collaborations have been undertaken to unify and standardize assessments and treat- aPaediatric Rheumatology Department, Great Ormond Street Hospital ments of rare diseases such as idiopathic inflamma- b tory myositis (IIM). The Paediatric Rheumatology for Children NHS Foundation Trust, Infection, Immunity and Inflamma- tion, UCL Great Ormond Street Institute of Child Health and cNIHR International Trials Organization (PRINTO) and the GOSH Biomedical Research Centre (BRC), London, UK International Myositis Assessment & Clinical Stud- Correspondence to Giulia C. Varnier, MD, Paediatric Rheumatology ies Group (IMACS) initial preliminary response cri- Department, Great Ormond Street Hospital for Children NHS Founda- teria considerably improved clinical assessment and tion Trust, London WC1N 3JH, UK. Tel: +44 020 7405 9200ext7887; therapeutic response of JDM patients, but were lack- e-mail: [email protected] ing in sensitivity and still presented several differ- Curr Opin Rheumatol 2018, 30:650–654 ences in the individual core set measurement [5–7]. DOI:10.1097/BOR.0000000000000538 www.co-rheumatology.com Volume 30 Number 6 November 2018 Copyright © 2018 Wolters Kluwer Health, Inc. All rights reserved. Novel treatment approach and outcomes in JDM Varnier et al. patients, especially for girls and ethnic minorities. KEY POINTS Ethnicity and lower family income were found to be European and American study groups proposed a associated with worse outcome, increased morbidity consensus for optimal dataset and criteria of minimal, and decreased function in another large American moderate and major response to treatment in JDM. cohort study [12]. A further study showed worse cardiovascular outcome in JDM patients (tested with New consensus-based guidelines are available for a 6-min walk test, timed ‘up and go’ test, CMAS, diagnosis and management of children with JDM, and particularly with predominant skin disease and echocardiography, lung function test, thoracic high persistent skin disease. resolution computed tomography scan and MRI and health-related quality of life questionnaire) with a New autoantibody association, especially combined mean of 17 years of disease history when compared with muscle biopsy histopathology, and a new MRI with sex-match and age-match controls, especially scoring system may help the clinician with treatment choice and disease prognosis. those with active disease [13]. One of the ongoing challenges of the manage- ment of JDM has been identifying a reliable, practi- cal tool to measure the skin disease. A prospective (MMT) or Childhood Myositis Assessment Scale study tested the PRINTO proposed criteria for clini- (CMAS); Childhood Health Assessment Question- cally inactive disease, which stated that at least three naire; Muscle enzymes (creatine kinase, aldolase, of four conditions should be met: creatinine kinase alanine aminotransferase, aspartate aminotransfer- 150 U/l or less, CMAS at least 48, MMT of eight ase and lactate dehydrogenase) or Physical Sum- groups at least 78 and physician global assessment mary Score of the Child Health Questionnaire- of overall disease activity 0.2 or less [14]. This anal- Parent Form 50 and Extramuscular activity or Dis- ysis by Almeida et al. [15] showed the importance of ease Activity Score (DAS). These new response crite- incorporating the physician global assessment of ria provide a quantitative measurement of disease overall disease activity as an essential criterion of improvement and resolve the differences between clinically inactive disease, as this helps prevent the PRINTO and IMACS criteria, enabling an easier misclassification of patients with active skin disease. comparison between different datasets and facilitat- Subsequent to this study, the same study group ing future trials. tested three different skin scoring tools in JDM, Another important step towards effective com- the Myositis Intention to Treat Activity Index, munication between different study groups by using abbreviated Cutaneous Assessment Tool and DAS standardized clinical data has been created by Inter- and correlated them with the physician’s 10-cm skin national Group of Experts (McCann et al. [9&]), who visual analogue scale (VAS). All three tools were easy have defined an optimal dataset for JDM to capture and quick to use, and this study showed that the disease subphenotype, activity, comorbidity and DAS best correlated with the physician VAS. How- damage over time. Both an international panel of ever, all three skin tools had limitations, suggesting experts took part in a Delphi process, but also that future studies should design a new tool with all parents and patients with JDM participated in the the strengths of the existing ones [16]. survey, enabling the group to highlight what patients and families feel are essential items of the clinical assessment in JDM, with good agreement ANTIBODIES with the healthcare professionals. Juvenile myositis is a highly heterogeneous disease A recent large analysis of the EuroMyositis reg- ranging from profound muscle weakness and vis- istry, which includes both adult and paediatric onset ceral involvement to normal muscle strength. In cases of all types of IIM has highlighted the differ- recent years, autoantibodies have been identified ences between JDM and adult dermatomyositis and in 60–70% children with myositis and have been polymyositis, the former being less associated with able to identify clinically homogeneous groups [17– interstitial lung disease and malignancy and having 21]. This concept has been recently further validated different skin disease characteristics [10]. in a large study including 379 juvenile myositis Little is known regarding long-term outcome in patients, which confirmed that the myositis-specific JDM, but two recent studies shed some light on this autoantibodies (MSA) are exclusively found in chil- extremely important aspect of care. Silverberg et al. dren with IIM, and not in healthy children or [11] evaluated over 14 million hospitalization of patients with other autoimmune diseases (including patients with JDM over a 10-year period and showed arthritis or lupus) or muscular dystrophy. Therefore, significantly higher odds for cardiovascular and this study suggested that the presence of MSA cerebrovascular comorbidities in this US cohort of should be considered highly suggestive of the 1040-8711 Copyright ß 2018 Wolters Kluwer Health, Inc. All rights reserved. www.co-rheumatology.com 651 Copyright © 2018 Wolters Kluwer Health, Inc. All rights reserved. Myositis and myopathies diagnosis of myositis [19]. In this study-specific MSA inversion recovery
Load more

Recommended publications
  • Eye Findings in Patients with Juvenile Dermatomyositis JONATHAN D
    Eye Findings in Patients with Juvenile Dermatomyositis JONATHAN D. AKIKUSA, DHENUKA K. TENNANKORE, ALEX V. LEVIN, and BRIAN M. FELDMAN ABSTRACT. Objective. Reports of eye involvement in juvenile dermatomyositis (JDM), including significant retinopathy with visual loss, have led some to recommend routine formal ophthalmologic assess- ments for all patients with JDM at diagnosis. Our objective was to document the frequency and spec- trum of eye involvement in patients followed in a single clinic caring for children with JDM. Methods. A chart review was conducted of formal ophthalmologic consultation notes for patients with JDM followed at the Hospital for Sick Children between 1981 and 2002. Results. Ophthalmologic assessments were found for 82 of 108 patients with JDM. The mean age at diagnosis of JDM was 7.0 years and 68.3% were female. Forty-five patients (55.6%) had abnormal eye examinations. Lid manifestations, found in 37 patients (45.7%), were the most common abnor- mality. Fourteen patients (17.1%) had corticosteroid-induced cataracts. Two patients had retinal abnormalities; one had a small retinal hemorrhage, the other an incidental chorioretinal scar. Neither had impairment of vision. No patient had uveitis. Conclusion. Eyelid and lens abnormalities are common in patients with JDM, while retinopathy is rare. As lid lesions and cataracts are easily detected by non-ophthalmologists, and retinal lesions are rare, we feel that JDM patients without visual symptoms do not require routine formal ophthalmo- logic assessment for disease manifestations. (J Rheumatol 2005;32:1986–91) Key Indexing Terms: JUVENILE DERMATOMYOSITIS RETINOPATHY CATARACTS HELIOTROPE Juvenile dermatomyositis (JDM) is a systemic inflammato- described in adult patients with DM11-13, in some cases pro- ry vasculopathy in which the predominant clinical manifes- ducing permanent visual impairment11,13.
    [Show full text]
  • Juvenile Dermatomyositis - a Case Report with Review on Oral Manifestations and Oral Health Considerations
    Volume 44 Number 1 pp. 52-61 2018 Case Report Juvenile dermatomyositis - A case report with review on oral manifestations and oral health considerations Pritesh Ruparelia ([email protected]) Oshin Verma ([email protected]) Vrutti Shah ([email protected]) Krishna Shah ([email protected]) Follow this and additional works at: https://ijom.iaom.com/journal The journal in which this article appears is hosted on Digital Commons, an Elsevier platform. Suggested Citation Ruparelia, P., et al. (2018). Juvenile dermatomyositis - A case report with review on oral manifestations and oral health considerations. International Journal of Orofacial Myology, 44(1), 52-61. DOI: https://doi.org/10.52010/ijom.2018.44.1.4 This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License. The views expressed in this article are those of the authors and do not necessarily reflect the policies or positions of the International Association of Orofacial Myology (IAOM). Identification of specific oducts,pr programs, or equipment does not constitute or imply endorsement by the authors or the IAOM. International Journal of Orofacial Myology 2018, V44 JUVENILE DERMATOMYOSITIS - A CASE REPORT WITH REVIEW ON ORAL MANIFESTATIONS AND ORAL HEALTH CONSIDERATIONS. PRITESH RUPARELIA, MDS, OSHIN VERMA, BDS, VRUTTI SHAH, BDS, KRISHNA SHAH, BDS ABSTRACT Juvenile Dermatomyositis is the most common inflammatory myositis in children, distinguished by proximal muscle weakness, a characteristic rash and Gottron’s papules. The oral lesions most commonly manifest as diffuse stomatitis and pharyngitis with halitosis. We report a case of an 8 year old male with proximal muscle weakness of all four limbs, rash, Gottron’s papules and oral manifestations.
    [Show full text]
  • Adverse Drug Reactions Associated with Treatment in Patients With
    Said et al. Advances in Rheumatology (2020) 60:53 Advances in Rheumatology https://doi.org/10.1186/s42358-020-00154-4 RESEARCH Open Access Adverse drug reactions associated with treatment in patients with chronic rheumatic diseases in childhood: a retrospective real life review of a single center cohort Manar Amanouil Said* , Liana Soido Teixeira e Silva, Aline Maria de Oliveira Rocha, Gustavo Guimarães Barreto Alves, Daniela Gerent Petry Piotto, Claudio Arnaldo Len and Maria Teresa Terreri Abstract Background: Adverse drug reactions (ADRs) are the sixth leading causes of death worldwide; monitoring them is fundamental, especially in patients with disorders like chronic rheumatic diseases (CRDs). The study aimed to describe the ADRs investigating their severity and associated factors and resulting interventions in pediatric patients with CRDs. Methods: A retrospective, descriptive and analytical study was conducted on a cohort of children and adolescents with juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM). The study evaluated medical records of the patients to determine the causality and the management of ADRs. In order to investigate the risk factors that would increase the risk of ADRs, a logistic regression model was carried out on a group of patients treated with the main used drug. Results: We observed 949 ADRs in 547 patients studied. Methotrexate (MTX) was the most frequently used medication and also the cause of the most ADRs, which occurred in 63.3% of patients, followed by glucocorticoids (GCs). Comparing synthetic disease-modifying anti-rheumatic drugs (sDMARDs) vs biologic disease-modifying anti- rheumatic drugs (bDMARDs), the ADRs attributed to the former were by far higher than the latter.
    [Show full text]
  • Autoantibodies in Myositis
    Autoantibodies in Myositis Neil McHugh University of Bath and Royal National Hospital for Rheumatic Diseases Bath UK Idiopathic Inflammatory Myositis Spectrum Disease Muscle inflammation Skin disorder Interstitial lung disease Myositis spectrum disease autoantibodies (MSDA)! Myositis Spectrum Disease • Polymyositis • Anti-synthetase syndrome • Immune-mediated necrotising myopathy • Dermatomyositis • Clinically amyopathic dermatomyositis (CADM) • Cancer associated myositis (CAM) • Inclusion Body Myositis • Juvenile Dermatomyositis • Myositis associated with connective tissue disease • Otherj • Granulomatous, eosinophilic, focal, orbital, macrophagic, myofasciitis Autoantibodies in Myositis Spectrum Disease • MSDA (myositis spectrum • MAA (myositis associated disorder autoantibodies) autoantibodies) • Anti-tRNA synthetases (e.g. • Anti-PM-Scl anti-Jo-1) • • Anti-Mi-2 Anti-U1RNP • Anti-signal recognition • Anti-Ku particle • Anti-U3RNP • Anti-SAE • Anti-Ro (SSA) • Anti-TIF1-g • Anti-NXP2 • Anti-MDA5 • Anti-HMGCR • Anti-cN-1A MSDAs and target autoantigens I Autoantibodies Target autoantigen Autoantigen function Clinical phenotype Anti-ARS tRNA synthetase Intracytoplasmic protein ASS Anti-Jo-1 Histidyl synthesis Myositis Anti-PL-7 Threonyl Binding between an amino Interstitial pneumonia Anti-PL-12 Alanyl acid and its cognate tRNA Mechanics hands Anti-EJ Glycyl Arthritis Anti-OJ Isoleucyl Anti-KS Asparaginyl Fever Anti-Zo Phenylalanyl Raynauds Anti-YRS Tyrosyl Anti-Mi-2 Helicase protein part of the Nuclear transcription Adult and juvenile DM
    [Show full text]
  • Childhood-Onset Systemic Lupus Erythematosus: a Review and Update
    MEDICAL www.jpeds.com • THE JOURNAL OF PEDIATRICS PROGRESS Childhood-Onset Systemic Lupus Erythematosus: A Review and Update Onengiya Harry, MD, MPH†, Shima Yasin, MD, MSc†, and Hermine Brunner, MD, MSc, MBA, FAAP, FACR upus is a chronic, autoimmune multisystem inflam- Genetic Factors matory disease that is associated with sizable morbid- There is a 10-fold increase in SLE risk among monozygotic as L ity and mortality.1 When lupus commences in an compared to dizygotic twins.15,16 Further, siblings of a patient individual less than 18 years of age,2 it is commonly referred with SLE carry an 8- to 20-fold higher risk of developing SLE to as childhood-onset systemic lupus erythematosus (cSLE). as compared with a healthy general population.16,17 With a reported incidence of 0.3-0.9 per 100 000 children per SLE is considered a polygenic disease, although rare mono- year, and a prevalence of 3.3-24 per 100 000 children,3 cSLE genic causes have been described recently.18 Genetic variants is rare. About 10%-20% of all patients with SLE are diag- that are well-established include very rare mutations in genes nosed during childhood. Typically, cSLE has a more severe clini- coding for select complement factors. Indeed, a single gene mu- cal course than that seen in adults, with a higher prevalence tation that results in a complete deficiency of C1q increases of lupus nephritis, hematologic anomalies, photosensitivity, neu- the risk of SLE, or lupus-like symptoms, to more than 90%. ropsychiatric, and mucocutaneous involvement.3-5 C4 deficiency is also
    [Show full text]
  • Beneath the Surface: Derm Clues to Underlying Disorders
    Christian R. Halvorson, MD; Richard Colgan, MD Department of Family and Beneath the surface: Derm clues Community Medicine, University of Maryland School of Medicine, Baltimore to underlying disorders [email protected] Dermatologic fi ndings are frequent indicators of The authors reported no potential confl ict of interest connective tissue disorders. Here’s what to look for. relevant to this article. any systemic conditions are accompanied by skin PRACTICE manifestations. Th is is especially true for connec- RECOMMENDATIONS Mtive tissue disorders, for which dermatologic fi nd- › When evaluating patients ings are often the key to diagnosis. with suspected cutaneous In this review, we describe the dermatologic fi ndings of lupus erythematosus, use some well-known connective tissue disorders. Th e text and multiple criteria—including photographs in the pages that follow will help you hone your histologic and immuno- diagnostic skills, leading to earlier treatment and, possibly, fl uorescent biopsy fi ndings better outcomes. and American College of Rheumatology criteria—to rule out systemic disease. C Lupus erythematosus: Cutaneous › Cancer screening with a and systemic disease often overlap careful history and physi- Lupus erythematosus (LE), a chronic, infl ammatory autoim- cal examination is recom- mended for all adult patients mune condition that primarily aff ects women in their 20s and whom you suspect of having 30s, may initially present as a systemic disease or in a purely dermatomyositis. C cutaneous form. However, most patients with systemic LE have some skin manifestations, and those with cutaneous › Suspect mixed connective LE often have—or subsequently develop—systemic involve- tissue disease in patients 1 with skin fi ndings charac- ment.
    [Show full text]
  • 329.Full.Pdf
    Recommendations Ann Rheum Dis: first published as 10.1136/annrheumdis-2016-209247 on 11 August 2016. Downloaded from EXTENDED REPORT Consensus-based recommendations for the management of juvenile dermatomyositis Felicitas Bellutti Enders,1,2 Brigitte Bader-Meunier,3 Eileen Baildam,4 Tamas Constantin,5 Pavla Dolezalova,6 Brian M Feldman,7 Pekka Lahdenne,8 Bo Magnusson,9 Kiran Nistala,10 Seza Ozen,11 Clarissa Pilkington,10 Angelo Ravelli,12 Ricardo Russo,13 Yosef Uziel,14 Marco van Brussel,15 Janjaap van der Net,15 Sebastiaan Vastert,1 Lucy R Wedderburn,10 Nicolaas Wulffraat,1 Liza J McCann,4 Annet van Royen-Kerkhof1 Handling editor Tore K Kvien ABSTRACT clinicians in the care of patients with JDM as no ▸ Additional material is Background In 2012, a European initiative called international consensus regarding diagnosis and published online only. To view Single Hub and Access point for pediatric Rheumatology treatment is currently available and management please visit the journal online in Europe (SHARE) was launched to optimise and therefore varies. (http://dx.doi.org/10.1136/ annrheumdis-2016-209247). disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic METHODS fi For numbered af liations see diseases. Juvenile dermatomyositis ( JDM) is a rare A committee of 19 experts in paediatric rheumatol- end of article. disease within the group of paediatric rheumatic ogy, 2 experts in exercise physiology and physical therapy was established to develop recommenda- Correspondence to diseases (PRDs) and can lead to significant morbidity. Dr Annet van Royen-Kerkhof, Evidence-based guidelines are sparse and management tions for JDM based on consensus, but evidence Department of Paediatric is mostly based on physicians’ experience.
    [Show full text]
  • Adverse Cutaneous Reactions to Hydroxychloroquine Are More Common in Patients with Dermatomyositis Than in Patients with Cutaneous Lupus Erythematosus
    EVIDENCE-BASED DERMATOLOGY: ORIGINAL CONTRIBUTION Adverse Cutaneous Reactions to Hydroxychloroquine Are More Common in Patients With Dermatomyositis Than in Patients With Cutaneous Lupus Erythematosus Michelle T. Pelle, MD; Jeffrey P. Callen, MD Background: Hydroxychloroquine sulfate and other an- Main Outcome Measures: Presence or absence of docu- timalarial drugs have been used successfully as adjunc- mented drug eruption due to hydroxychloroquine exposure. tive therapy for patients with cutaneous lesions of der- matomyositis over the past 20 years. An increased Results: Of 39 patients, 12 (31%) with dermatomyositis incidence of cutaneous reactions to hydroxychloro- developed a cutaneous reaction to hydroxychloroquine. quine has been postulated to occur in patients with Among age-, sex-, and race-matched patients with cuta- dermatomyositis. neous lupus erythematosus, only 1 developed a cutane- ous reaction to hydroxychloroquine. None of the reac- Objective: To determine if adverse cutaneous erup- tions observed in our patients resulted in serious morbidity tions due to hydroxychloroquine are more common in or mortality. Additionally, 4 patients with dermatomyo- patients with dermatomyositis than in those with cuta- sitis who reacted to hydroxychloroquine were treated with neous lupus erythematosus. oral chloroquine phosphate, 2 of whom also reacted to chlo- roquine phosphate. Design: Retrospective, age-, sex-, and race-matched case- control study. Conclusions: When contemplating antimalarial therapy for dermatomyositis, both the physician and the patient Setting: University-affiliated practice. should recognize that non–life-threatening cutaneous re- actions may occur in approximately one third of patients Patients: The study comprised 42 patients with and that perhaps one half of those who react to hydroxy- dermatomyositis (39 adults) and 39 age-, sex-, and chloroquine will also react to chloroquine.
    [Show full text]
  • Review of Disease-Modifying Anti Rheumatic Drugs in Paediatric
    18th Expert Committee on the Selection and Use of Essential Medicines (21 to 25 March 2011) Section 2 Analgesics, antipyretics, NSAIMs, DMARDs 2.4 Disease-modifying agents used in rheumatoid disorders Review of Disease‐Modifying Anti Rheumatic Drugs in Paediatric Rheumatic disease September 2010 Prepared by: Peter Gowdie Rheumatology and Clinical Pharmacology Fellow 2009 Royal Children’s Hospital Melbourne, Australia 1 Contents 1. Intent of review 2. Identification of priority conditions 3. Review of priority rheumatic disease 1. Juvenile Idiopathic arthritis 1. Epidemiology 2. Disease burden and outcome 3. Clinical manifestations 4. Complications • macrophage activation syndrome • uveitis • amyloidosis 2. Idiopathic Inflammatory Myopathies (Juvenile Dermatomyositis) 1. Epidemiology 2. Clinical Manifestations 3. Complications 4. Course and Outcome 5. Overview of management 3. Systemic Lupus Erythematosus 1. Epidemiology 2. Clinical Manifestations 3. Course and outcome 4. Overview of management 4. DMARDs 1. Methotrexate 1. Mechanism of action and Pharmacology 2. Efficacy in Juvenile Idiopathic Arthritis 3. Efficacy in Juvenile Dermatomyositis 4. Dose and administration 5. Drug interaction and folate supplementation 6. Safety 7. Monitoring and supervision 8. Formulary 9. Summary recommendations 2. Leflunomide 1. Mechanism of action and Pharmacology 2. Efficacy in Juvenile Idiopathic Arthritis 3. Dose and administration 4. Safety 5. Drug interaction 6. Monitoring and supervision 7. Formulary 8. Summary recommendations 2 3. Sulphasalazine 1. Mechanism of action and Pharmacology 2. Efficacy in Juvenile Idiopathic Arthritis 3. Dose and administration 4. Safety 5. Drug interaction 6. Monitoring and supervision 7. Formulary 8. Summary recommendations 4. Cyclosporin 1. Mechanism of action and Pharmacology 2. Efficacy in Juvenile Idiopathic Arthritis and Macrophage Activation Syndrome 3.
    [Show full text]
  • Juvenile Dermatomyositis and the Inflammatory Myopathies
    Published online: 2020-04-06 342 Juvenile Dermatomyositis and the Inflammatory Myopathies Collin Swafford, DO1 E. Steve Roach, MD1 1 Department of Neurology, Dell Medical School, University of Texas, Address for correspondence Collin Swafford, DO, Department of Austin, Texas Neurology, Dell Medical School, University of Texas, 1701 Trinity St, Stop Z0700, Austin, TX 78712 Semin Neurol 2020;40:342–348. (e-mail: [email protected]). Abstract The inflammatory myopathies comprise disorders of immune-mediated muscle injury. The histopathology and clinical features help distinguish them. Juvenile dermatomyo- sitis (JDM) is the most common form of myositis in children and adolescents. Children with JDM present with proximal muscle weakness and characteristic rashes. The Keywords presentation is similar in children and adults, but JDM is a primary disorder and the ► Inflammatory adult form often is concerning for a paraneoplastic syndrome. Proximal muscle myopathies weakness occurs with dermatomyositis, polymyositis, and immune-mediated necro- ► juvenile tizing myopathy, but the latter two conditions have no dermatologic findings or dermatomyositis distinct tissue changes which set them apart from dermatomyositis. Inclusion body ► inclusion body myositis, also included in the inflammatory myopathies, presents with more distal myositis involvement, and microscopically exhibits identifiable rimmed vacuoles. We review key ► immune-mediated features of these disorders, focusing in more detail on JDM because it is more often necrotizing myopathy encountered by the child neurologist. The inflammatory myopathies are characterized by immune- with median age at the time of diagnosis around 7.5 years.1,2 mediated skeletal muscle injury. The pattern of muscle The exact cause of JDM is unknown, but several theories have involvement along with the histopathological findings helps emerged over the years.
    [Show full text]
  • Recent Advances in Juvenile Idiopathic Inflammatory Myopathies
    REVIEW CURRENT OPINION Recent advances in juvenile idiopathic inflammatory myopathies Floranne C. Ernstea and Ann M. Reedb Purpose of review Ongoing research continues to advance our understanding of the juvenile idiopathic inflammatory myopathies (JIIMs). We review the recent contributions from the published literature about the classification, pathogenesis, assessment, and treatment of JIIMs in basic and translational science and clinical research in 2013 through early 2014. Recent findings Large registries, such as the Childhood Arthritis and Rheumatology Research Alliance registry, are conducting trials to enhance our understanding of JIIMs. Ultraviolet radiation exposure 1 month prior to juvenile dermatomyositis (JDM) may trigger the onset of disease. Myositis-specific autoantibodies define clinical phenotypes in JIIMs. MRI is useful in diagnosing JDM and may be used as a disease assessment tool. Type 1 interferon genes and proteins are increasing in use as disease assessment tools, but larger, prospective, validation studies are needed. Moderate-to-intense physical activity is effective in increasing the aerobic capacity of JDM patients in remission. New criteria developed by the Paediatric Rheumatology International Trials Organization for classifying inactive disease in JDM have practical applicability to the current clinical practice and clinical trials as even after 16.8 years of symptom onset, over half of JDM patients still have active disease. Summary There has been significant progress in understanding the clinical characteristics, diagnostic workup, treatment, disease assessment, and prognosis of JIIM patients, but more prospective treatment trials are needed, especially in light of the paucity of the current biologic treatment agents available. Keywords clinical characteristics, immunopathogenesis, juvenile dermatomyositis, prognosis, therapy INTRODUCTION common myositis is JDM, comprising approxi- The juvenile idiopathic inflammatory myopathies mately 85% of JIIM patients.
    [Show full text]
  • A Case of Dermatomyositis with Secondary Sjögren's Syndrome
    162 A Case of Dermatomyositis with Secondary Sjögren’s Syndrome- Diagnosis with Follow-up Study of Technetium-99m Pyrophosphate Scintigraphy Ching-Tang Huang1, Ying-Chu Chen1, Chingtsai Lin2, Yu-Chun Hsiao3, Lai-Fa Sheu4, Min-Chien Tu1 Abstract Purpose: To report a case of dermatomyositis (DM) with secondary Sjögren’s syndrome (SS) and propose the clinical application of technetium-99m pyrophosphate (99mTc-PYP) scan. Case Report: A 50-year-old woman had progressive proximal muscle weakness of bilateral thighs, myalgia, tea-colored urine, and exercise intolerance for 6 months. Physical examination showed malar rash, V-sign, periungual erythema, and mechanic hands. Neurological assessment showed symmetric pelvic-girdle weakness, myopathic face, waddling gait, but preserved deep tendon reflex and sensory functions. DM was diagnosed on the basis of typical rashes and serum creatinine kinase elevation (7397 IU/L). Aside from myopathic symptoms, dry eye and mouth were reported. Thorough autoantibody searches showed positive anti-SSA/Ro antibody (198 U/ml). Both Schirmer's test and sialoscintigraphy were positive, leading secondary SS as diagnosis. Initial 99mTc-PYP scan revealed increased radiouptake in the muscles of bilateral thighs, compatible with clinical assessment. Follow- up scan three months later shows abnormal but attenuated radiouptake at bilateral thighs, in the presence of nearly-complete clinical recovery. Conclusion: DM with secondary SS in adult is a unique disease entity, with predominantly myopathic symptoms and satisfactory therapeutic response as its characteristics. Our serial muscle imaging studies suggest that 99mTc-PYP scan is at once anatomically-specific and persistently-sensitive to microstructural damages within inflammatory muscles, enabling clinician to monitor disease activity and therapeutic response.
    [Show full text]