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Skeletal Myopathy in Systemic Sclerosis: Devising a Classification Schema Predictive of Outcomes

系统性硬化症中的骨骼肌病：设计预测结果的分类模式

基本信息

批准号：
10626940
负责人：
Julie Jisun Paik
金额：
$ 15.88万
依托单位：
JOHNS HOPKINS UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-05-01 至 2025-04-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10626940
关键词：
Address Advisory Committees Age Algorithms Biological Biological Process Biopsy Cardiac Death Cessation of life Chronic Classification Clinical Clinical Investigator Clinical Trials Consensus Creatine Kinase Data Development Diffusion Magnetic Resonance Imaging Disease Early Diagnosis Early identification Edema Electromyography Enrollment Fatty acid glycerol esters Fibrosis Foundations Future Gender Goals Heterogeneity Histologic Histopathology Image Imaging Techniques Incidence Inclusion Body Myositis Individual Inflammation Inflammatory Infrastructure International Intramuscular Knowledge Laboratories Logistic Regressions Magnetic Resonance Imaging Manuals Maps Measures Medicine Mentored Patient-Oriented Research Career Development Award Mentors Metabolic Methodology Methods Modeling Muscle Muscle Weakness Musculoskeletal Myopathy Myositis Necrosis Outcome Outcome Measure Pathogenesis Pathologic Patients Pattern Phenotype Prediction of Response to Therapy Process Prospective cohort Protocols documentation Pulmonary Heart Disease Radiology Specialty Research Resources Retrospective Studies Rheumatology Sampling Biases Scleroderma Serology Signal Transduction Skeletal Muscle Systemic Scleroderma Techniques Testing Therapeutic Therapeutic Trials Translational Research Universities Validation career clinical development clinical epidemiology clinically significant cohort diffusion weighted disability disorder control epidemiology study high risk illness length imaging biomarker interest member mortality muscle form muscle strength novel observational cohort study outcome prediction predictive marker professor prospective quantitative imaging radiological imaging random forest recruit skeletal skills treatment response uptake

项目摘要

PROJECT SUMMARY Skeletal myopathy in systemic sclerosis (SSc) is associated with increased disability and mortality. There is no consensus classification of SSc-associated myopathy that can be utilized to lay the groundwork for understanding muscle disease in scleroderma. We know that there is pathological, serological, and outcome heterogeneity that may be informative in defining distinct subsets of myopathy in SSc. Our preliminary data demonstrate that (1) muscle histopathology is heterogeneous in SSc, (2) patients with a fibrosing myopathy have poor outcomes, in particular cardiac death, and (3) there is intense signal uptake on advanced MRI imaging such as diffusion weighted and tensor imaging and T2 mapping in SSc-associated myopathy which may indicate early disease related changes when compared to conventional muscle MRI. This K23 proposal will first establish a Myopathy cohort (Aim 1), devise a classification schema and test its utility in predicting long term outcomes such as trajectory, cardiopulmonary disease, disability, mortality, and treatment responsiveness(Aim 2), and detect and quantify early disease related muscle changes on advanced muscle MRI (Aim 3). This application is for a K23 award for Julie Paik, MD, MHS, Assistant Professor of Medicine in the Division of Rheumatology at the Johns Hopkins University. Her long term career goal is to become a leading clinical investigator in scleroderma muscle disease in the context of observational cohort studies, state of the art imaging biomarkers, and clinical trial methodology. Her short term goal of devising this classification schema will provide a new gold standard for the validation of external myopathy cohorts in SSc and lay the framework for future studies directed at understanding biological mechanism and developing a therapeutic trial using measurable outcomes such as quantitative muscle MRI. This proposal takes advantage of the rich resources and infrastructure at the Johns Hopkins Scleroderma Center, a recently designated Precision Center of Excellence in 2018. Her mentoring team includes Dr. Laura Hummers and Dr. Fredrick Wigley, both renowned experts in SSc and clinical epidemiology, Dr. Andrew Mammen is an internationally recognized myositis expert, and Dr. Scott Zeger a leader in bio statistical and epidemiological research. Dr. Paik has recruited vital members to her Advisory Committee, composed of mentors and collaborators who have specific expertise relevant to the proposal and are fully supportive of Dr. Paik’s research and career goals. These include Dr. Michael Jacobs (advanced radiological imaging), Dr. Laura Fayad (musculoskeletal radiology), Dr. Andrea Corse (muscle histopathology), Dr. Tom Lloyd (classification schemas in inclusion body myositis), and Dr. Ami Shah (scleroderma translational research). This K23 award will allow Dr. Paik to gain the skills needed to analyze and develop a classification schema of skeletal myopathy in scleroderma, develop expertise in advanced muscle MRI analysis, and build her clinical trial skill set to ultimately support a future R01 proposal.

项目总结

项目成果

期刊论文数量（16）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Segmental arterial mediolysis: a rare cause of abdominal pain masquerading as vasculitis.

节段性动脉中溶解症：一种罕见的腹部疼痛原因，伪装成血管炎。

DOI：
10.1093/rheumatology/keab326
发表时间：
2021
期刊：
Rheumatology (Oxford, England)
影响因子：
0
作者：
Connolly,CaoilfhionnM;Yu,Alice;Seo,Philip;Paik,JulieJ
通讯作者：
Paik,JulieJ

Otalgia, Hearing Loss, and Mastoiditis Refractory to Antibiotic Therapy.

抗生素治疗难治的耳痛、听力损失和乳突炎。

DOI：
10.1016/j.amjmed.2022.05.027
发表时间：
2022
期刊：
The American journal of medicine
影响因子：
0
作者：
Baum,Rebecca;Rangaswamy,Shivani;Mikula,MichaelW;Meredith,RebeccaR;Lichtenstein,StephenT;Agrawal,Yuri;Gelber,AllanC;Paik,JulieJ
通讯作者：
Paik,JulieJ

International Guideline for Idiopathic Inflammatory Myopathy-Associated Cancer Screening: an International Myositis Assessment and Clinical Studies Group (IMACS) initiative.

特发性炎症性肌病相关癌症筛查国际指南：国际肌炎评估和临床研究小组 (IMACS) 倡议。

DOI：
10.1038/s41584-023-01045-w
发表时间：
2023
期刊：
Nature reviews. Rheumatology
影响因子：
0
作者：
Oldroyd,AlexanderGS;Callen,JeffreyP;Chinoy,Hector;Chung,Lorinda;Fiorentino,David;Gordon,Patrick;Machado,PedroM;McHugh,Neil;Selva-O'Callaghan,Albert;Schmidt,Jens;Tansley,SarahL;Vleugels,RuthAnn;Werth,VictoriaP;International
通讯作者：
International

Humoral response following SARS-CoV-2 vaccination: not all immunosuppressants are created equal.

DOI：
10.1016/s2665-9913(22)00066-2
发表时间：
2022-05
期刊：
The Lancet. Rheumatology
影响因子：
0
作者：
Connolly CM;Paik JJ
通讯作者：
Paik JJ

Troponin elevation independently associates with mortality in systemic sclerosis.

DOI：
10.55563/clinexprheumatol/fytfmy
发表时间：
2022-10
期刊：
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
影响因子：
3.7
作者：
Paik, J. J.;Choi, D. Y.;Mukherjee, M.;Hsu, S.;Wigley, F.;Shah, A. A.;Hummers, L. K.
通讯作者：
Hummers, L. K.