Article
Rheumatology
Gulnara Mamyrova, Erica McBride, Lawrence Yao, Joseph A. Shrader, Minal Jain, Jianhua Yao, Rodolfo Curiel, Frederick W. Miller, Michael O. Harris-Love, Lisa G. Rider
Summary: The study found that muscle ultrasound parameters in patients with JDM were positively correlated with disease activity measures and MRI, suggesting that MUS may be a valuable imaging modality to assess JDM disease activity and damage.
Article
Biochemistry & Molecular Biology
Ellie Gibbs, Amer Khojah, Gabrielle Morgan, Louis Ehwerhemuepha, Lauren M. M. Pachman
Summary: This study aimed to determine if vWF:Ag, a serologic indicator of vascular disease activity, is useful to assess disease activity in children with JDM. The results showed that 25% of untreated JDM patients had elevated vWF:Ag levels, which were associated with disease severity.
Article
Medicine, General & Internal
Christopher Costin, Amer Khojah, Elisa Ochfeld, Gabrielle Morgan, Saravanan Subramanian, Marisa Klein-Gitelman, Xiao-Di Tan, Lauren M. Pachman
Summary: This study aimed to determine if B lymphocytosis can be used as a biomarker for disease activity in JDM. Children with untreated JDM were divided into two groups based on B cell percentage: normal group and high B cell group. The high B cell group had higher disease activity scores and neopterin levels. Secondary analysis showed that B cell values increased after 3-6 months of therapy and decreased after completion of therapy, while JM patients had higher BAFF levels than controls. This study supports the use of B cell lymphocytosis as a biomarker and suggests B cell-directed therapies for JDM.
Article
Rheumatology
Anke Rietveld, Judith Wienke, Eline Visser, Wilma Vree Egberts, Wolfgang Schlumberger, Baziel van Engelen, Annet van Royen-Kerkhof, Hui Lu, Lucy Wedderburn, Christiaan Saris, Sarah Tansley, Ger Pruijn
Summary: Different antibody detection techniques were used to assess anti-cN-1A reactivity in children with juvenile DM and healthy controls, and no reactivity was observed in either group. Three Dutch juvenile DM patients showed weakly positive results for synthetic cN-1A peptides in ELISA.
ARTHRITIS & RHEUMATOLOGY
(2021)
Article
Immunology
Jessica Neely, George Hartoularos, Daniel Bunis, Yang Sun, David Lee, Susan Kim, Chun Jimmie Ye, Marina Sirota
Summary: This study investigated the cell types and gene expression in juvenile dermatomyositis (JDM) using single-cell sequencing, and found separate clustering of cells and an over-expression of IFN gene signature in JDM. CD16(+) monocytes expressed the highest IFN gene score and showed an inflammatory and antigen-presenting phenotype. A distinct transitional B cell population was also found in JDM.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Ting -Wei Lin, Ya-Chiao Hu, Bor-Luen Chiang
Summary: This study aimed to evaluate the clinical characteristics of children diagnosed with juvenile dermatomyositis (JDM) in Taiwan and identify biomarkers for predicting the disease course and outcomes. The study included 47 patients, with a mean age of 7.5 years at disease onset. The most common initial presentations were Gottron's sign, muscle weakness, and facial rash. The results identified factors associated with complete clinical remission and the chronic continuous course, highlighting the importance of aggressive treatment for the latter.
JOURNAL OF MICROBIOLOGY IMMUNOLOGY AND INFECTION
(2023)
Article
Medicine, General & Internal
Amer Khojah, Gabrielle Morgan, Lauren M. Pachman
Summary: This study aimed to define the immunological and clinical differences in untreated Juvenile Dermatomyositis (JDM) patients by comparing different levels of neopterin. The results showed that JDM patients with elevated neopterin had more active disease and demonstrated variations in cell populations and genetic polymorphism.
Article
Respiratory System
Minfei Hu, Chencong Shen, Fei Zheng, Yun Zhou, Liping Teng, Rongjun Zheng, Bin Hu, Chaoying Wang, Meiping Lu, Xuefeng Xu
Summary: A prediction model using non-invasive clinical features was established to early discriminate DM-ILD in clinical practice. The model was developed based on the analysis of clinical data of pediatric patients with JDM and showed promising results in risk prediction.
RESPIRATORY RESEARCH
(2023)
Article
Pediatrics
Peyman Hosseinzadeh Kassani, Louis Ehwerhemuepha, Chloe Martin-King, Ryan Kassab, Ellie Gibbs, Gabrielle Morgan, Lauren M. Pachman
Summary: This study assesses the potential of using artificial intelligence and nailfold capillaroscopy (NFC) images to diagnose and evaluate the range of disease activity in children with idiopathic inflammatory myopathies. The study found that the NFC-Net model can accurately differentiate children with JDM from healthy controls and predict disease activity scores.
PEDIATRIC RESEARCH
(2023)
Editorial Material
Critical Care Medicine
Laura H. Kim, Julie Duncan, Connie L. Yang
Summary: A previously healthy 17-year-old Taiwanese girl presented with symptoms consistent with juvenile dermatomyositis, including weakness, polyarthritis, and heliotrope rash. Diagnosis was confirmed based on elevated creatine phosphokinase level and diffuse myositis on lower limb MRI.
Review
Medicine, General & Internal
Caitlan S. Pinotti, Laura Cannon, Jeffrey A. Dvergsten, Eveline Y. Wu
Summary: Calcinosis, a complication of juvenile dermatomyositis, is the deposition of insoluble calcium salts in various tissues. It affects up to 40% of patients with juvenile dermatomyositis and leads to significant morbidity. Effective treatment for calcinosis remains unknown due to a lack of comparative studies. This review aims to summarize recent literature on the pathogenesis and treatment of calcinosis in juvenile dermatomyositis and propose future research areas.
FRONTIERS IN MEDICINE
(2023)
Article
Biochemistry & Molecular Biology
Ameera Bukhari, Amer Khojah, Wilfredo Marin, Andrey Khramtsov, Galina Khramtsova, Christopher Costin, Gabrielle Morgan, Prathyaya Ramesh, Marisa S. Klein-Gitelman, I. Caroline Le Poole, Lauren M. Pachman
Summary: The expression of otoferlin mRNA is increased in JDM patients' PBMCs and muscle compared to healthy controls. This study aims to evaluate the role of otoferlin in JDM disease pathophysiology and its association with disease activity in untreated children with JDM. The findings suggest that otoferlin expression is associated with muscle weakness, making it a possible biomarker of disease activity.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Pediatrics
Avinash Sharma, Dharmagat Bhattarai, Anju Gupta, Sandesh Guleria, Amit Rawat, Pandiarajan Vignesh, Ravinder Garg, Surjit Singh
Summary: The study found that eight patients with JDM tested positive for at least one MSA or MAA, with varying presence of SRP, MDA-5, Mi-2, and SSA/Ro52 antibodies.
INDIAN JOURNAL OF PEDIATRICS
(2021)
Article
Rheumatology
Takayuki Kishi, William Warren-Hicks, Nastaran Bayat, Ira N. Targoff, Adam M. Huber, Michael M. Ward, Lisa G. Rider
Summary: The study examined a North American registry of JDM patients to investigate factors associated with corticosteroid discontinuation, complete clinical response, and remission. Results suggest that JDM patients can achieve favorable outcomes over several years, with corticosteroid discontinuation, complete clinical response, and remission being inter-related and influenced by various clinical features and myositis autoantibodies.
Article
Rheumatology
Paola Cancarini, Tomo Nozawa, Kristi Whitney, Audrey Bell-Peter, Jo-Anne Marcuz, Andrea Taddio, Jessica Guo, Saunya Dover, Brian M. Feldman
Summary: This study describes the clinical features of a large single-centre cohort of Juvenile Dermatomyositis (JDM). The study found that JDM mostly affects females, and common symptoms include muscle weakness, nailfold capillary abnormalities, and rashes. The disease course varies, with most patients requiring corticosteroid treatment.
SEMINARS IN ARTHRITIS AND RHEUMATISM
(2022)
Letter
Rheumatology
Ingrid E. Lundberg, Frederick W. Miller, Lisa G. Rider, Victoria P. Werth, Anna Tjarnlund, Matteo Bottai
ANNALS OF THE RHEUMATIC DISEASES
(2023)
Article
Rheumatology
Matthew A. Sherman, Rose Graf, Sara E. Sabbagh, Angeles S. Galindo-Feria, Iago Pinal-Fernandez, Katherine Pak, Takayuki Kishi, Willy A. Flegel, Ira N. Targoff, Frederick W. Miller, Ingrid E. Lundberg, Lisa G. Rider, Andrew L. Mammen
Summary: This study found the presence of anti-FHL1 autoantibodies in juvenile IIM patients, which were associated with MAAs and specific skin rash, but not with other clinical features or worse outcomes.
Letter
Critical Care Medicine
Cheilonda Johnson, Adam I. Schiffenbauer, Frederick W. Miller, Jamie Perin, Sonye K. Danoff, Avantika R. Diwadkar, Jaehyun Joo, Blanca E. Himes, Nuala J. Meyer
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2023)
Article
Rheumatology
Danlei Zhou, Emily H. King, Simon Rothwell, Olga Krystufkova, Antonella Notarnicola, Samantha Coss, Rabheh Abdul-Aziz, Katherine E. Miller, Amanda Dang, G. Richard Yu, Joanne Drew, Emeli Lundstrom, Lauren M. Pachman, Gulnara Mamyrova, Rodolfo Curiel, Boel De Paepe, Jan L. De Bleecker, Antony Payton, William Ollier, Terrance P. O'Hanlon, Ira N. Targoff, Willy A. Flegel, Vidya Sivaraman, Edward Oberle, Shoghik Akoghlanian, Kyla Driest, Charles H. Spencer, Yee Ling Wu, Haikady N. Nagaraja, Stacy P. Ardoin, Hector Chinoy, Lisa G. Rider, Frederick W. Miller, Ingrid E. Lundberg, Leonid Padyukov, Jiri Vencovsky, Janine A. Lamb, Chack-Yung Yu
Summary: C4A deficiency is relevant in dermatomyositis, HLA-DRB1*03 is important in IBM, and both C4A deficiency and HLA-DRB1*03 contribute interactively to the risk of polymyositis.
ANNALS OF THE RHEUMATIC DISEASES
(2023)
Article
Rheumatology
Simon I. Rothwell, Christopher W. Amos, Frederick G. Miller, Lisa E. Rider, Ingrid K. Lundberg, Peter Gregersen, Jiri Vencovsky, Neil McHugh, Vidya Limaye, Albert G. Selva-O'Callaghan, Michael M. Hanna, Pedro M. Machado, Lauren M. Pachman, Ann Reed, Oyvind Molberg, Olivier Benveniste, Pernille Mathiesen, Timothy Radstake, Andrea L. Doria, Jan De Bleecker, Boel De Paepe, Britta E. Maurer, William Ollier, Leonid P. Padyukov, Terrance O'Hanlon, Annette R. Lee, Lucy Wedderburn, Hector A. Chinoy, Janine Lamb
Summary: In this study, genetic analysis of samples from patients with idiopathic inflammatory myopathies (IIMs) identified novel gene variants and localized signals to specific immune cell types. These findings provide new insights into the pathogenesis of IIMs.
ARTHRITIS & RHEUMATOLOGY
(2023)
Article
Rheumatology
Briana A. Cervantes, Prateek Gowda, Lisa G. Rider, Frederick W. Miller, Marcus Y. Chen, Adam Schiffenbauer
Summary: This study used whole-body CT scans and calcium scoring techniques to assess calcinosis in patients with adult and juvenile dermatomyositis. Different patterns and new locations of calcinosis were observed using CT scans and the Agatston scoring method, which can effectively evaluate and track calcinosis in these patients.
Article
Rheumatology
Rie Karasawa, Kazuo Yudoh, Toshiko Sato, Megumi Tanaka, Sara E. Sabbagh, Willy A. Flegel, Andrew L. Mammen, James N. Jarvis, Lisa G. Rider
Summary: By using Western blotting, the expression of TPM4 protein in normal human dermal microvascular ECs was investigated. Using ELISA, anti-TPM4 autoantibodies were detected in 30% of JDM plasma samples. These autoantibodies were associated with certain clinical features of JDM, such as cutaneous ulcers, shawl sign rash, mucous membrane lesions, and subcutaneous edema.
Article
Rheumatology
Didem Saygin, Hanna Kim, Christian Douglas, Brian Erman, Jesse Wilkerson, John A. McGrath, Chester Oddis, Ingrid E. Lundberg, Anthony A. Amato, Ignacio Garcia-De La Torre, Hector Chinoy, David Fiorentino, Lorinda Chung, Yeong-Wook Song, Frederick W. Miller, Nicolino Ruperto, Jiri Vencovsky, Rohit Aggarwal, Lisa G. Rider
Summary: This study further validated the ACR-EULAR Myositis Response Criteria (MRC) by assessing the contribution of each core set measure, the frequency of improvement in strength vs extramuscular activity, the representation of patient-reported outcome measures (PROM), and the frequency of core set measure worsening. The results showed that improvement in strength was the main contributing factor, and the majority of patients also had improvement in PROM.
Article
Rheumatology
Hanna Kim, Didem Saygin, Christian Douglas, Jesse Wilkerson, Brian Erman, Angela Pistorio, John A. McGrath, Ann M. Reed, Chester Oddis, Claudia Bracaglia, Annet van Royen-Kerkhof, Blanca Bica, Pavla Dolezalova, Virginia P. L. Ferriani, Berit Flato, Ana G. Bernard-Medina, Troels Herlin, Frederick W. Miller, Jiri Vencovsky, Nicolino Ruperto, Rohit Aggarwal, Lisa G. Rider
Summary: The 2016 ACR-EULAR response criteria for JDM is a composite measure with six core set measures to calculate a total improvement score. This study evaluated the contribution of each measure, representation of muscle-related and patient-reported measures, and frequency of measure worsening across improvement categories. The results support the consistent performance of the ACR-EULAR MRC across multiple studies and its further use as an efficacy endpoint in JDM trials.
Article
Rheumatology
Laura E. Schanberg, Lily (Yeruk) Mulugeta, Bolanle Akinlade, Hermine I. Brunner, Jianmeng Chen, Robert A. Colbert, Vincent Delgaizo, Marc R. Gastonguay, Rachel Glaser, Lisa Imundo, Daniel J. Lovell, Jocelyn H. Leu, Nael M. Mostafa, Robert M. Nelson, Peter A. Nigrovic, Nikolay P. Nikolov, Lisa G. Rider, Rebecca Rothwell, Chandrahas Sahajwalla, Renu Singh, Vikram Sinha, Carolyn L. Yancey, Lynne Yao
Summary: This report summarizes a workshop on drug development for polyarticular juvenile idiopathic arthritis (pJIA), highlighting the importance of accelerating drug development and the necessity of collaboration between stakeholders.
ARTHRITIS & RHEUMATOLOGY
(2023)
Article
Rheumatology
James M. Ward, Mythri Ambatipudi, Terrance P. O'Hanlon, Michael A. Smith, Melissa de Los Reyes, Adam Schiffenbauer, Saifur Rahman, Kamelia Zerrouki, Frederick W. Miller, Miguel A. Sanjuan, Jian-Liang Li, Kerry A. Casey, Lisa G. Rider
Summary: Transcript and protein expression were analyzed in active adult dermatomyositis (DM) and juvenile DM patients receiving immunosuppressive therapies. A total of 1,124 gene loci showed significant alterations in expression, with 70 genes shared between DM and juvenile DM. Interferon-stimulated genes and innate immune markers specific to neutrophils were up-regulated in both DM and juvenile DM. Pathway analysis revealed up-regulation of PI3K/AKT, ERK, and p38 MAPK signaling in DM, with different regulation of upstream and downstream components in both DM and juvenile DM. Therapeutic targets may include these pathways as well as neutrophil degranulation.
ARTHRITIS & RHEUMATOLOGY
(2023)
Article
Rheumatology
Matthew A. Sherman, Katherine Pak, Iago A. Pinal-Fernandez, Willy A. N. Flegel, Ira N. W. Targoff, Frederick W. G. Miller, Lisa G. L. Rider, Andrew L. Mammen
Summary: This study investigated the prevalence and clinical features associated with anti-Sp4 autoantibodies in juvenile-onset idiopathic inflammatory myopathies (IIM). The results showed that anti-Sp4 autoantibodies were present in 7% of the juvenile myositis patients and were associated with coexisting anti-TIF1 autoantibodies and a reduced risk of cancer.
ARTHRITIS & RHEUMATOLOGY
(2023)
Meeting Abstract
Rheumatology
Danlei Zhou, Emily H. King, Simon Rothwell, Olga Krystufkova, Antonella Notarnicola, Rabheh Abdul Aziz, Emeli Lundstrom, Stiburkova Blanka, Lauren M. Pachman, Gulnara Mamyrova, Boel de Paepe, Jan de Bleecker, Terrance P. O'Hanlon, Samantha Coss, Katherine Lintner Miller, Amanda Dang, G. Richard Yu, Joanne Drew, Vidya Sivaraman, Edward Oberle, Shoghik Akoghlanian, Kyla Driest, Charles H. Spencer, Yee Ling Wu, Stacy P. Ardoin, Lisa G. Rider, Hector Chinoy, Frederick W. Miller, Ingrid E. Lundberg, Leonid Padyukov, Jiri Vencovsky, Janine A. Lamb, Chack-Yung Yu
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
(2023)
Meeting Abstract
Rheumatology
Amer Khojah, Gabrielle Morgan, Najah Ahsan, Lauren M. Pachman
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
(2023)
Meeting Abstract
Rheumatology
Gabrielle Morgan, Amer Khojah, Sonia Savani, Najah Ahsan, Lauren M. Pachman
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
(2023)