Outcomes research in rheumatic diseases The goals of the K24 application are to support my continued development and impact as a mentor, to establish a research training program for patient-oriented research (POR) in the Division of Rheumatology at the University of Michigan (UM), and to expand my current POR program. In the past few years, I have actively mentored trainees and junior faculty on different POR projects and the immediate objective of the proposed K24 is to secure protected time to continue my efforts and develop future clinical investigators with a focus on patient-reported outcome measures and trial design in rheumatic diseases. In addition to directing a large Scleroderma program, I continue to work extensively in other arthritides (gout and rheumatoid arthritis) and have support of established clinical researchers in systemic lupus erythematosus and fibromyalgia, providing a rich environment of POR at UM. Given my commitment to mentoring, recent collaborations with the UM Department of Biostatistics, UM's rich resources dedicated to POR, and strong institutional support to create this program, the K24 Award provides an invaluable opportunity to expand my mentoring efforts and to establish a specialized structured POR program. The program will ensure that medical and biostatistical students and fellows interested in becoming independent investigators will be supported by an interdisciplinary team of mentors and will acquire training in clinical research methods and biostatistics. K24 grant would preserve my time at 50% effort and relieve future clinical and administrative responsibilities. A diverse portfolio of currently funded research opportunities exists for future trainees in POR. Research proposed includes two current projects and two new projects addressing POR areas of patient reported outcomes, clinical trial design, and database development. The two current NIH funded studies include: 1. To develop and evaluate the psychometric properties of gastrointestinal symptoms item bank developed under the NIH Patient-Reported Outcomes Measurement Information System initiative;and 2. To develop a combined response index for scleroderma clinical trials in an ongoing NIH funded longitudinal observational study.
The aims of two new proposed studies are: 1. To determine the relationship between the vitamin D status and physiological, radiological, and dyspnea measures of scleroderma-associated interstitial lung disease (SSc-ILD) in an ongoing NIH funded clinical trial comparing oral mycophenolate mofetil vs. oral cyclophosphamide in 150 patients with active SSc-ILD. 2. To develop an early diffuse scleroderma observational registry through the coordinated efforts of several young investigators at large U.S. scleroderma centers. The goals of the registry include: i) to determine whether the clinical phenotypes and autoantibodies at baseline in early, diffuse scleroderma are associated with, and predict, clinical, radiological, and patient-reported outcomes and survival;and ii) to develop a repository of scleroderma blood and tissue to explore the cellular and molecular mediators of SSc and their association with clinical phenotypes and autoantibodies.

Public Health Relevance

The goals of the K24 application are to support my continued development as a mentor and to establish a research training program for patient oriented research. The proposed program is interdisciplinary, and focuses in expanding clinical research opportunities and mentoring in rheumatic diseases. The K24 Award would ensure that I have the protected time to be able to expand my current mentoring activities while continuing to advance my patient oriented research program in patients with rheumatic disease. Research includes two current projects and two new projects addressing patient-oriented research areas of patient-reported outcomes, clinical trial design, and database development.

National Institute of Health (NIH)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Midcareer Investigator Award in Patient-Oriented Research (K24)
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Study Section
Arthritis and Musculoskeletal and Skin Diseases Special Grants Review Committee (AMS)
Program Officer
Witter, James
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University of Michigan Ann Arbor
Internal Medicine/Medicine
Schools of Medicine
Ann Arbor
United States
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Altorok, Nezam; Tsou, Pei-Suen; Coit, Patrick et al. (2015) Genome-wide DNA methylation analysis in dermal fibroblasts from patients with diffuse and limited systemic sclerosis reveals common and subset-specific DNA methylation aberrancies. Ann Rheum Dis 74:1612-20
Flaherty, Kevin R; Khanna, Dinesh (2014) Idiopathic or connective tissue disease-associated interstitial lung disease: a case of HRCT mimicry. Thorax 69:205-6
Ghrenassia, Etienne; Avouac, Jerome; Khanna, Dinesh et al. (2014) Prevalence, correlates and outcomes of gastric antral vascular ectasia in systemic sclerosis: a EUSTAR case-control study. J Rheumatol 41:99-105
Schiopu, Elena; Au, Karen M; McMahon, Maureen A et al. (2014) Prevalence of subclinical atherosclerosis is increased in systemic sclerosis and is associated with serum proteins: a cross-sectional, controlled study of carotid ultrasound. Rheumatology (Oxford) 53:704-13
Gladue, Heather; Altorok, Nezam; Townsend, Whitney et al. (2014) Screening and diagnostic modalities for connective tissue disease-associated pulmonary arterial hypertension: a systematic review. Semin Arthritis Rheum 43:536-41
Tsou, Pei-Suen; Haak, Andrew J; Khanna, Dinesh et al. (2014) Cellular mechanisms of tissue fibrosis. 8. Current and future drug targets in fibrosis: focus on Rho GTPase-regulated gene transcription. Am J Physiol Cell Physiol 307:C2-13
Nagaraja, Vivek; Hays, Ron D; Khanna, Puja P et al. (2014) Construct validity of the Patient-Reported Outcomes Measurement Information System gastrointestinal symptom scales in systemic sclerosis. Arthritis Care Res (Hoboken) 66:1725-30
Haak, Andrew J; Tsou, Pei-Suen; Amin, Mohammad A et al. (2014) Targeting the myofibroblast genetic switch: inhibitors of myocardin-related transcription factor/serum response factor-regulated gene transcription prevent fibrosis in a murine model of skin injury. J Pharmacol Exp Ther 349:480-6
Khanna, Dinesh; Georges, George E; Couriel, Daniel R (2014) Autologous hematopoietic stem cell therapy in severe systemic sclerosis: ready for clinical practice? JAMA 311:2485-7
Song, J; Boscardin, W J; Furst, D E et al. (2014) A cautionary tale: dealing with missing data in clinical trials for rheumatic diseases. Clin Exp Rheumatol 32:S-122-6

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