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3. Responsiveness and Meaningful Thresholds of PROMIS Pain Interference, Fatigue, and Physical Function Forms in Adults with Idiopathic Inflammatory Myopathies: Report from the OMERACT Myositis Working Group

Author

Saygin, D, primary, DiRenzo, D, additional, Raaphorst, J, additional, de Groot, I, additional, Bingham, CO, additional, Lundberg, IE, additional, Regardt, M, additional, Sarver, C, additional, de Visser, M, additional, Maxwell, LJ, additional, Beaton, D, additional, Kim, JY, additional, Needham, M, additional, Alexanderson, H, additional, Christopher-Stine, L, additional, Mecoli, CA, additional, and Park, JK, additional

Published
2023
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10. Accumulation of autophagosome cargo protein p62 is common in idiopathic inflammatory myopathies

Author

Milisenda, J.C., Pinal-Fernandez, I., Lloyd, T.E., Grau, J.M., Miller, F.W., Selva-O’Callaghan, A., Christopher-Stine, L., Stenzel, W., Mammen, A.L., and Corse, A.M.

Subjects
Myositis, Autophagosomes, Humans, Muscle, Skeletal, Article, Autoimmune Diseases, Myositis, Inclusion Body, Polymyositis
Abstract

OBJECTIVE: The subsarcolemmal accumulation of p62 aggregates in myofibres has been proposed to be characteristic of s poradic inclusion body myositis (sIBM). The objective of this study was to analyse the patterns and prevalence of p62 immunostaining and to quantitate p62 gene expression in muscle biopsies from a large number of patients with different types of myopathic and neurogenic disorders. METHODS: For the p62 immunostaining analysis, all patients with a muscle biopsy immunostained for p62 at the Johns Hopkins Neuromuscular Pathology Laboratory from 2013 to 2017 were included (n=303). The prevalence and pattern of p62 immunostaining were compared between patients with histologically normal muscle (n=29), inflammatory myopathies (n=136), non-inflammatory myopathies (n=53), and neurogenic disorders (n=85). p62 expression levels were analysed using an existing RNAseq dataset including data from dermatomyositis (DM; n=39), immune-mediated necrotising myopathy (IMNM; n=49), antisynthetase syndrome (AS; n=18), and sIBM (n=23) patients as well as 20 histologically normal muscle biopsies. RESULTS: p62 staining was absent in normal biopsies, but present in biopsies from those with polymyositis (29%), non-inflammatory myopathies (all

Published
2020

11. Pain Interference, Fatigue, Physical Function as Outcome Measures in Adult Myositis: Updates on the Validation Process by the OMERACT Myositis Working Group

Author

DiRenzo, D., de Groot, I., Alexanderson, H., Bingham, C., Lundberg, I.E., Needham, M., Park, J.K., Regardt, M., Sarver, C., Saygin, D., Song, Y.W., Maxwell, L., Beaton, D., Christopher-Stine, L., Mecoli, C., DiRenzo, D., de Groot, I., Alexanderson, H., Bingham, C., Lundberg, I.E., Needham, M., Park, J.K., Regardt, M., Sarver, C., Saygin, D., Song, Y.W., Maxwell, L., Beaton, D., Christopher-Stine, L., and Mecoli, C.

Published
2021

14. 2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria—methodological aspects

Author

Rider, L. G., Ruperto, N., Pistorio, A., Erman, B., Bayat, N., Lachenbruc, P. A., Rockette, H., Feldman, B. M., Huber, A. M., Hansen, P., Oddis, C. V., Lundberg, I. E., Amato, A. A., Chinoy, H., Cooper, R. G., Chung, L., Danko, K., Fiorentino, D., De la Torre, I. G., Reed, A. M., Song, Y. W., Cimaz, R., Cuttica, R. J., Pilkington, C. A., Martini, A., van der Net, J., Maillard, S., Miller, F. W., Vencovsky, J., Aggarwal, R., Christopher-Stine, L., Criscione-Schreiber, L., Crofford, L., Cronin, M. E., Gordon, P., Hengstman, G., Katz, J. D., Mammen, A., Marder, G., Mchugh, N., Schiopu, E., Wolfe, G., Wortmann, R., Apaz, M., Bowyer, S., Constantin, T., Curran, M., Davidson, J., Griffin, T., Jones, O., Kim, S., Lang, B., Lindsley, C., Lovell, D., Magalhaes, C. S., Pachman, L. M., Ponyi, A., Punaro, M., Quartier, P., Ramanan, A. V., Ravelli, A., Rennebohm, R., Van Royen-Kerkhof, A., Sherry, D. D., Silva, C. A., Stringer, E., Wallace, C., Ascherman, D., Barohn, R., Benveniste, O., De Bleecker, J., Callen, J., Charles-Schoeman, C., Danoff, S., Dastmalchi, M., Dimachkie, M., Di Martino, S., Dourmishev, L., Ernste, F., Gono, T., Isenberg, D., Katsumata, Y., Kissel, J., Leff, R. L., Levine, T., Mann, H., Marie, I., Merola, J., Olesinska, M., Olsen, N., Pipitone, N., Ramchandren, S., Rutkove, S., Saketkoo, L. A., Schiffenbauer, A., Selva-O'Callaghan, A., Shinjo, S. K., Shupak, R., Swierkocka, K., de Visser, M., Wanschitz, J., Werth, V. P., Whitt, I., Ytterberg, S., Avcin, T., Becker, M., Beresford, M. W., Dressler, F., Dvergsten, J., Ferriani, V. P. L., Flato, B., Gerloni, V., Henrickson, M., Hinze, C., Hoeltzel, M., BUNES IBARRA, MIGUEL ANGEL, Ilowite, N., Imundo, L., Kingsbury, D., Magnusson, B., Maguiness, S., Mathiesen, P., Mccann, L., Nielsen, S., de Oliveira, S. K. F., Passo, M., Rabinovich, E., Rivas-Chacon, R., Robinson, A. B., Rouster-Stevens, K., Russo, R., Rutkowska-Sak, L., Sallum, A., Sanner, H., Schmeling, H., Selcen, D., Shaham, B., Spencer, C. H., Sundel, R., Tardieu, M., Thatayatikom, A., Wahezi, D., and Zulian, F.

Subjects
medicine.medical_specialty, dermatomyositis, Hybrid measure, 1000Minds software, Minimal Clinically Important Difference, response criteria, Klinikai orvostudományok, Logistic regression, Polymyositis, Dermatomyositis, polymyositis, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Response criteria, medicine, Humans, Pharmacology (medical), Conjoint analysis, outcome assessment, Juvenile dermatomyositis, Myositis, 030203 arthritis & rheumatology, hybrid measure, juvenile dermatomyositis, business.industry, Minimal clinically important difference, Outcome assessment, Area Under Curve, Logistic Models, Treatment Outcome, Orvostudományok, Clinical Science, medicine.disease, Adult dermatomyositis, Clinical trial, Physical therapy, conjoint analysis, business, 030217 neurology & neurosurgery
Abstract

Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM.Methods: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data.Results: Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference.Conclusion: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

Published
2017
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15. Assessing the content validity of patient-reported outcome measures in adult myositis: A report from the OMERACT myositis working group

Author

Esfandiary, T., Park, J.K., Alexanderson, H., Regardt, M., Needham, M., de Groot, I., Sarver, C., Lundberg, I.E., de Visser, M., Song, Y.W., DiRenzo, D., Bingham, C.O., Christopher-Stine, L., Mecoli, C.A., Esfandiary, T., Park, J.K., Alexanderson, H., Regardt, M., Needham, M., de Groot, I., Sarver, C., Lundberg, I.E., de Visser, M., Song, Y.W., DiRenzo, D., Bingham, C.O., Christopher-Stine, L., and Mecoli, C.A.

Abstract

Objective To investigate the content validity of several patient-reported outcome measures (PROMs) in patients with idiopathic inflammatory myopathies (IIM). Methods Seven individual PROM instruments were selected by the Outcome Measures in Rheumatology (OMERACT) Myositis Working Group relating to the following domains: pain, fatigue, physical function and physical activity. Twenty patients from the Johns Hopkins Myositis Center were selected for one-on-one face-to-face or phone interviews for cognitive interviewing of individual PROMs to assess comprehension and content validity. Additionally, patients were asked if they thought muscle symptoms, an area originally identified in qualitative studies, were encapsulated by the other four domains. Results The majority of patients (>70%) felt that each of the instruments was clear, easy to read and understand, and could be used for assessment of its domain. Two-thirds (66%) of patients felt that ‘muscle symptoms’ were captured by the other domains. Conclusions We provided evidence to support adequate content validity for several PROMs. Further research is needed to determine whether ‘muscle symptoms’ warrant a separate domain.

Published
2020

17. OMERACT 2018 Modified Patient-reported outcome domain core set in the life impact area for adult idiopathic inflammatory myopathies

Author

Regardt, M., Mecoli, C.A., Park, J.K., de Groot, I., Sarver, C., Needham, M., de Visser, M., Shea, B., Bingham, C.O., Lundberg, I.E., Song, Y.W., Christopher-Stine, L., Alexanderson, H., Regardt, M., Mecoli, C.A., Park, J.K., de Groot, I., Sarver, C., Needham, M., de Visser, M., Shea, B., Bingham, C.O., Lundberg, I.E., Song, Y.W., Christopher-Stine, L., and Alexanderson, H.

Abstract

Objective. To present and vote on a myositis modified patient-reported outcome core domain set in the life impact area at the Outcome Measures in Rheumatology (OMERACT) 2018. Methods. Based on results from international focus groups and Delphi surveys, a draft core set was developed. Results. Domains muscle symptoms, fatigue, level of physical activity, and pain reached ≥ 70% consensus and were mandatory to assess in all trials. Domains lung, joint, and skin symptoms were mandatory in specific circumstances. This core set was endorsed by > 85% at OMERACT 2018. Conclusion. We propose a life impact core set for patients with idiopathic inflammatory myopathies and will proceed with instrument selections.

Published
2019

20. A Draft Modified Core Domain Set for Patient-Reported Outcomes (PRO) in Patients with Idiopathic Inflammatory Myopathies (IIM): An Omeract Report

Author

Regardt, M., Mecoli, C.A., Park, J.K., Needham, M., De Groot, I., Sarver, C., Lundberg, I.E., Shea, B., De Visser, M., Song, Y.W., Bingham, C.O., Christopher-Stine, L., Alexanderson, H., Regardt, M., Mecoli, C.A., Park, J.K., Needham, M., De Groot, I., Sarver, C., Lundberg, I.E., Shea, B., De Visser, M., Song, Y.W., Bingham, C.O., Christopher-Stine, L., and Alexanderson, H.

Abstract

Background/Purpose: The OMERACT Myositis special interest group (SIG) represents clinicians, patients, and researchers from four continents. Focus groups were conducted including 61 patients on three countries resulting in a list of 26 domains (1). In collaboration with International Myositis Assessment Clinical Study Group (IMACS), our goal was to identify a set of core patient-reported outcomes (PRO) in regards to life impact important to assess in clinical trials and clinical practice in myositis. Methods: Patients with adult polymyositis, dermatomyositis, antisynthetase syndrome, or immune-mediated necrotizing myopathy (IMNM) in South Korea, Sweden and USA (N=638) responded to the first online modified Delphi in 2016. The second modified Delphi included patients (N=563), health care providers (HCP) (N=101), care givers (N=27) and regulatory agencies (n=xx) from multiple countries in 2017. A third modified Delphi was administered in 2018 including 410 patients, 109 HCP, 22 caregivers. Results: From this work, four domains were deemed mandatory to measure in all clinical trials for IIM: fatigue, pain, levels of physical activity, and muscle symptoms (Figure 1). Additional optional domains include skin symptoms, lung symptoms, and joint symptoms. Several other domains were deemed important to study with further research efforts including sleeping difficulty, cognitive distress, ability to work, and emotional distress. Conclusion: A draft set of core PRO has been developed through validated methods based on OMERACT guidelines. Fatigue, pain, levels of physical activity and muscle symptoms were included in the inner circle and should always be used in clinical trials in IIM. We next seek to develop corresponding instruments with each of these domains with future efforts.

Published
2018

21. Perceptions of patients, caregivers, and healthcare providers of idiopathic inflammatory myopathies: An international OMERACT Study

Author

Mecoli, C.A., Park, J.K., Alexanderson, H., Regardt, M., Needham, M., de Groot, I., Sarver, C., Lundberg, I.E., Shea, B., de Visser, M., Song, Y.W., Bingham, C.O., Christopher-Stine, L., Mecoli, C.A., Park, J.K., Alexanderson, H., Regardt, M., Needham, M., de Groot, I., Sarver, C., Lundberg, I.E., Shea, B., de Visser, M., Song, Y.W., Bingham, C.O., and Christopher-Stine, L.

Abstract

Objective. Patient-reported outcome measures (PROM) that incorporate the patient perspective have not been well established in idiopathic inflammatory myopathies (IIM). As part of our goal to develop IIM-specific PROM, the Outcome Measures in Rheumatology (OMERACT) Myositis special interest group sought to determine which aspects of disease and its effects are important to patients and healthcare providers (HCP). Methods. Based on a prior qualitative content analysis of focus groups, an initial list of 24 candidate domains was constructed. We subsequently conducted an international survey to identify the importance of each of the 24 domains to be assessed in clinical research. Patients with IIM, their caregivers, and HCP treating IIM completed the survey. Results. In this survey, a total of 638 respondents completed the survey, consisting of 510 patients, 101 HCP, and 27 caregivers from 48 countries. Overall, patients were more likely to rank “fatigue,” “cognitive impact,” and “difficulty sleeping” higher compared with HCP, who ranked “joint symptoms,” “lung symptoms,” and “dysphagia” higher. Both patients and providers rated muscle symptoms as their top domain. In general, patients from different countries were in agreement on which domains were most important. One notable exception was that patients from Sweden and the Netherlands ranked lung symptoms significantly higher compared to other countries including the United States and Australia (mean weighted rankings of 2.86 and 2.04 vs 0.76 and 0.80, respectively; p < 0.0001). Conclusion. Substantial differences exist in how IIM is perceived by patients compared to HCP, with different domains prioritized. In contrast, patients’ ratings across the world were largely similar.

Published
2018

22. Developing standardised treatment for adults with myositis and different phenotypes: an international survey of current prescribing preferences

Author

Tansley S, Shaddick G, Christopher-Stine L, Sharp C, Dourmishev L, Maurer B, Hector Chinoy, McHugh N, University of Zurich, and Tansley, Sarah

Subjects
Health Knowledge, Attitudes, Practice, 2403 Immunology, Consensus, Myositis, Attitude of Health Personnel, 2745 Rheumatology, 10051 Rheumatology Clinic and Institute of Physical Medicine, 610 Medicine & health, Drug Prescriptions, Phenotype, Rheumatology, Health Care Surveys, 2723 Immunology and Allergy, Humans, Practice Patterns, Physicians', Rheumatologists, Immunosuppressive Agents
Abstract

The evidence base for treatment of the idiopathic inflammatory myopathies is extremely limited. The rarity and heterogeneity of these diseases has hampered the development of good quality clinical trials and while a range of immunomodulatory treatments are commonly used in clinical practice, as yet there are no clear guidelines directing their use. We aimed to establish current prescribing regimens used to treat adults with myositis internationally.An electronic survey based on different clinical scenarios was distributed internationally to clinicians involved in the treatment of patients with myositis. Participants were asked to select their first-line treatment preferences in each situation. A multinomial regression analysis was used to assess the influence of clinical scenario, respondent expertise and country of origin on first-line treatment choice.107 survey responses were received. 57% of respondents considered themselves an expert in myositis and the majority of respondents were rheumatologists although responses from other specialities were also received. Pharmacological treatment with steroids and additional immunotherapy was the preference in most scenarios. First-line immunosuppressant choice was significantly influenced by the clinical scenario, the expertise of the treating physician and country of practice. Azathioprine, methotrexate and mycophenolate mofetil were the most commonly chosen agents.In the absence of available evidence, clinical experience and expert consensus often forms the basis of treatment guidelines. These results suggest that an international consensus approach would be possible in myositis and would overcome an urgent, yet unmet need for patients suffering with this difficult disease.

Published
2016

24. Reconciling Healthcare Professional and Patient Perspectives in the Development of Disease Activity and Response Criteria in Connective Tissue Disease Related Interstitial Lung Diseases

Author

Saketkoo, La, Mittoo, S, Frankel, S, Lesage, D, Sarver, C, Phillips, K, Strand, V, Matteson, El, OMERACT Baughman RP, Brown, Kk, Christmann, Rb, Dellaripa, P, Denton, Cp, Distler, O, Fischer, A, Flaherty, K, Huscher, D, Khanna, D, Kowal Bielecka, O, Merkel, Pa, Oddis, Cv, Pittrow, D, Sandorfi, N, Seibold, Jr, Swigris, J, Wells, A, Antoniou, K, Castelino, Fv, Christopher Stine, L, Collard, Hr, Cottin, V, Danoff, S, Hedlund, R, Highland, Kb, Hummers, L, Lynch, Da, Kim, Ds, Ryu, Jh, Miller, Fw, Nichols, K, Proudman, Sm, Richeldi, L, Shah, Aa, van den Assum, P, Aggarwal, R, Ainslie, G, Alkassab, F, Allanore, Y, Anderson, Me, Andonopoulos, Ap, Antin Ozerkis, D, Arrobas, A, Ascherman, Dp, Assassi, S, Baron, M, Bathon, Jm, Baughman, Rp, Behr, J, Beretta, L, Bingham, Co, Binnie, M, Birring, Ss, Boin, F, Bongartz, T, Bourdin, A, Bouros, D, Brasington, R, Bresser, P, Buch, Mh, Burge, Ps, Carmona, L, Carreira, Pe, Carvalho, Cr, Catoggio, Lj, Chan, Km, Chapman, J, Chatterjee, S, Chua, F, Chung, L, Conron, M, Corte, T, Cosgrove, G, Costabel, U, Cox, G, Crestani, B, Crofford, Lj, Csuka, Me, Curbelo, P, Czirják, L, Daniil, Z, D'Arsigny, Cl, Davis, Gs, de Andrade JA, Dellaripa, Pf, De Vuyst, P, Dempsey, Oj, Derk, Ct, Distler, J, Dixon, Wg, Downey, G, Doyle, Mk, Drent, M, Durairaj, L, Emery, P, Espinoza, Lr, Farge, D, Fathi, M, Fell, Cd, Fessler, Bj, Fitzgerald, Je, Flaherty, Kr, Foeldvari, I, Fox, Ga, Frech, Tm, Freitas, S, Furst, De, Gabrielli, A, García Vicuña, R, Georgiev, Ob, Gerbino, A, Gillisen, A, Gladman, Dd, Glassberg, M, Gochuico, Br, Gogali, A, Goh, Ns, Goldberg, A, Goldberg, Hj, Gourley, Mf, Griffing, L, Grutters, Jc, Gunnarsson, R, Hachulla, E, Hall, Fc, Harari, S, Herrick, Al, Herzog, El, Hesselstrand, R, Highland, K, Hirani, N, Hodgson, U, Hollingsworth, Hm, Homer, Rj, Hoyles, Rk, Hsu, Vm, Hubbard, Rb, Hunzelmann, N, Isasi, Me, Isasi, Es, Jacobsen, S, Jimenez, Sa, Johnson, Sr, Jones, Ch, Kahaleh, B, Kairalla, Ra, Kalluri, M, Kalra, S, Kaner, Rj, Kinder, Bw, Kiter, G, Klingsberg, Rc, Kokosi, M, Kolb, Mr, Kowal Bielecka OM, Kur Zalewska, J, Kuwana, M, Lake, Fr, Lally, Ev, Lasky, Ja, Laurindo, Im, Able, L, Lee, P, Leonard, Ct, Lien, Dc, Limper, Ah, Liossis, Sn, Lohr, Km, Loyd, Je, Lundberg, Ie, Mageto, Yn, Maher, Tm, Mahmud, Th, Manganas, H, Marie, I, Marras, Tk, Martinez, Ja, Martinez, Fj, Mathieu, A, Matucci Cerinic, M, Mayes, Md, Mckown, Km, Medsger, Ta, Meehan, Rt, Mendes, Ac, Meyer, Kc, Millar, Ab, Moğulkoc, N, Molitor, Ja, Morais, A, Mouthon, L, Müller, V, Müller Quernheim, J, Nadashkevich, O, Nador, R, Nash, P, Nathan, Sd, Navarro, C, Neves, S, Noth, I, Nunes, H, Olson, Al, Opitz, Cf, Padilla, M, Pappas, D, Parfrey, H, Pego Reigosa JM, Pereira, Ca, Perez, R, Pope, Je, Porter, Jc, Renzoni, Ea, Riemekasten, G, Riley, Dj, Rischmueller, M, Rodriguez Reyna TS, Rojas Serrano, J, Roman, J, Rosen, Gd, Rossman, M, Rothfield, N, Sahn, Sa, Sanduzzi, A, Scholand, Mb, Selman, M, Senécal, Jl, Seo, P, Shah, A, Silver, Rm, Solomon, Jj, Steen, V, Stevens, W, Strange, C, Sussman, R, Sutton, Ed, Sweiss, Nj, Tornling, G, Tzelepis, Ge, Undurraga, A, Vacca, A, Vancheri, Carlo, Varga, J, Veale, Dj, Volkov, S, Walker, Ua, Wells, Au, Wencel, M, Wesselius, Lj, Wickremasinghe, M, Wilcox, P, Wilsher, Ml, Wollheim, Fa, Wuyts, Wa, Yung, G, Zanon, P, Zappala, Cj, Groshong, Sd, Leslie, Ko, Myers, Jl, Padera, Rf, Desai, Sr, Goldin, J, Kazerooni, Ea, Klein, Js, and Keen, Kj

Subjects
Male, medicine.medical_specialty, Delphi Technique, Consensus Development Conferences as Topic, Health Personnel, Immunology, Context (language use), Disease, Severity of Illness Index, Article, Idiopathic pulmonary fibrosis, Rheumatology, medicine, Immunology and Allergy, Humans, Disease management (health), Intensive care medicine, Connective Tissue Diseases, Randomized Controlled Trials as Topic, business.industry, Interstitial lung disease, Disease Management, respiratory system, Focus Groups, medicine.disease, Comorbidity, Connective tissue disease, respiratory tract diseases, Clinical trial, Treatment Outcome, Patient Satisfaction, Physical therapy, Quality of Life, ÍNDICE DE GRAVIDADE DA DOENÇA, Interdisciplinary Communication, business, Lung Diseases, Interstitial
Abstract

Interstitial lung diseases (ILD), including those related to connective tissue disease (CTD), and idiopathic pulmonary fibrosis (IPF) carry high morbidity and mortality. Great efforts are under way to develop and investigate meaningful treatments in the context of clinical trials. However, efforts have been challenged by a lack of validated outcome measures and by inconsistent use of measures in clinical trials. Lack of consensus has fragmented effective use of strategies in CTD-ILD and IPF, with a history of resultant difficulties in obtaining agency approval of treatment interventions. Until recently, the patient perspective to determine domains and outcome measures in CTD-ILD and IPF had never been applied. Efforts described here demonstrate unequivocally the value and influence of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/comorbidity of a rheumatic disease (ILD) as well as a disease not considered rheumatic (IPF). The OMERACT 11 proceedings of the CTD-ILD Working Group describe the forward and lateral process to include both the medical and patient perspectives in the urgently needed identification of a core set of preliminary domains and outcome measures in CTD-ILD and IPF.

Published
2014

25. Mortality and causes of death in patients with sporadic inclusion body myositis: Survey study based on the clinical experience of specialists in Australia, Europe and the USA

Author

Price, M.A., Barghout, V., Benveniste, O., Christopher-Stine, L., Corbett, A., de Visser, M., Hilton-Jones, D., Kissel, J.T., Lloyd, T.E., Lundberg, I.E., Mastaglia, F., Mozaffar, T., Needham, M., Schmidt, J., Sivakumar, K., DeMuro, C., Tseng, B.S., Price, M.A., Barghout, V., Benveniste, O., Christopher-Stine, L., Corbett, A., de Visser, M., Hilton-Jones, D., Kissel, J.T., Lloyd, T.E., Lundberg, I.E., Mastaglia, F., Mozaffar, T., Needham, M., Schmidt, J., Sivakumar, K., DeMuro, C., and Tseng, B.S.

Abstract

Background: There is a paucity of data on mortality and causes of death (CoDs) in patients with sporadic inclusion body myositis (sIBM), a rare, progressive, degenerative, inflammatory myopathy that typically affects those aged over 50 years. Objective: Based on patient records and expertise of clinical specialists, this study used questionnaires to evaluate physicians’ views on clinical characteristics of sIBM that may impact on premature mortality and CoDs in these patients. Methods: Thirteen physicians from seven countries completed two questionnaires online between December 20, 2012 and January 15, 2013. Responses to the first questionnaire were collated and presented in the second questionnaire to seek elaboration and identify consensus. Results: All 13 physicians completed both questionnaires, providing responses based on 585 living and 149 deceased patients under their care. Patients were reported to have experienced dysphagia (60.2%) and injurious falls (44.3%) during their disease. Over half of physicians reported that a subset of their patients with sIBM had a shortened lifespan (8/13), and agreed that bulbar dysfunction/dysphagia/oropharyngeal involvement (12/13), early-onset disease (8/13), severe symptoms (8/13), and falls (7/13) impacted lifespan. Factors related to sIBM were reported as CoDs in 40% of deceased patients. Oropharyngeal muscle dysfunction was ranked as the leading feature of sIBM that could contribute to death. The risk of premature mortality was higher than the age-matched comparison population. Conclusions: In the absence of data from traditional sources, this study suggests that features of sIBM may contribute to premature mortality and may be used to inform future studies.

Published
2016

26. FRI0284 Anti-Srp-Associated Autoimmune Myopathy: Younger Age at Onset Is Associated with More Severe Disease and Worse Outcome

Author

Pinal-Fernandez, I., primary, Parks, C., additional, Tiniakou, E., additional, Albayda, M., additional, Paik, J., additional, Lahouti, A., additional, Casal-Dominguez, M., additional, Pak, K., additional, Huang, W., additional, Lloyd, T.E., additional, Danoff, S., additional, Casciola-Rosen, L., additional, Christopher-Stine, L., additional, and Mammen, A.L., additional

Published
2016
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27. OP0010 Thigh Magnetic Resonance Imaging Reveals Increased Active and Chronic Muscle Damage in Necrotizing Myositis Compared To Polymyositis and Dermatomyositis

Author

Pinal-Fernandez, I., primary, Casal-Dominguez, M., additional, Lahouti, A., additional, Basharat, P., additional, Albayda, M., additional, Paik, J., additional, Ahlawat, S., additional, Danoff, S., additional, Lloyd, T.E., additional, Mammen, A.L., additional, Carrino, J., additional, and Christopher-Stine, L., additional

Published
2016
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29. Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: Provisional core sets of domains and instruments for use in clinical trials

Author

Saketkoo, La, Mittoo, S, Huscher, D, Khanna, D, Dellaripa, Pf, Distler, O, Flaherty, Kr, Frankel, S, Oddis, Cv, Denton, Cp, Fischer, A, Kowal Bielecka OM, Lesage, D, Merkel, Pa, Phillips, K, Pittrow, D, Swigris, J, Antoniou, K, Baughman, Rp, Castelino, Fv, Christmann, Rb, Christopher Stine, L, Collard, Hr, Cottin, V, Danoff, S, Highland, Kb, Hummers, L, Shah, Aa, Kim, Ds, Lynch, Da, Miller, Fw, Proudman, Sm, Richeldi, L, Ryu, Jh, Sandorfi, N, Sarver, C, Wells, Au, Strand, V, Matteson, El, Brown, Kk, Seibold, Jr, Aggarwal, R, Ainslie, G, Alkassab, F, Allanore, Y, Descartes, P, Anderson, Me, Andonopoulos, Ap, Antin Ozerkis, D, Arrobas, A, Ascherman, Dp, Assassi, S, Baron, M, Bathon, Jm, Behr, J, Beretta, L, Bingham, Co, Binnie, M, Birring, Ss, Boin, F, Bongartz, T, Bourdin, A, Bouros, D, Brasington, R, Bresser, P, Buch, Mh, Burge, Ps, Carmona, L, Carreira, Pe, Carvalho, Cr, Catoggio, Lj, Chan, Km, Chapman, J, Chatterjee, S, Chua, F, Chung, L, Conron, M, Corte, T, Cosgrove, G, Costabel, U, Cox, G, Crestani, B, Crofford, Lj, Csuka, Me, Curbelo, P, László, C, Daniil, Z, D'Arsigny, Cl, Davis, Gs, de Andrade JA, De Vuyst, P, Dempsey, Oj, Derk, Ct, Distler, J, Dixon, Wg, Downey, G, Doyle, Mk, Drent, M, Durairaj, L, Emery, P, Espinoza, Lr, Farge, D, Fathi, M, Fell, Cd, Fessler, Bj, Fitzgerald, Je, Fox, Ga, Foeldvari, I, Frech, Tm, Freitas, S, Furst, De, Gabrielli, A, García Vicuña, R, Georgiev, Ob, Gerbino, A, Gillisen, A, Gladman, Dd, Glassberg, M, Gochuico, Br, Gogali, A, Goh, Ns, Goldberg, A, Goldberg, Hj, Gourley, Mf, Griffing, L, Grutters, Jc, Gunnarsson, R, Hachulla, E, Hall, Fc, Harari, S, Herrick, Al, Herzog, El, Hesselstrand, R, Hirani, N, Hodgson, U, Hollingsworth, Hm, Homer, Rj, Hoyles, Rk, Hsu, Vm, Hubbard, Rb, Hunzelmann, N, Isasi, Me, Isasi, Es, Jacobsen, S, Jimenez, Sa, Johnson, Sr, Jones, Ch, Kahaleh, B, Kairalla, Ra, Kalluri, M, Kalra, S, Kaner, Rj, Kinder, Bw, Klingsberg, Rc, Kokosi, M, Kolb, Mr, Kur Zalewska, J, Kuwana, M, Lake, Fr, Lally, Ev, Lasky, Ja, Laurindo, Im, Able, L, Lee, P, Leonard, Ct, Lien, Dc, Limper, Ah, Liossis, Sn, Lohr, Km, Loyd, Je, Lundberg, Ie, Mageto, Yn, Maher, Tm, Mahmud, Th, Manganas, H, Marie, I, Marras, Tk, Antônio Baddini Martinez, J, Martinez, Fj, Mathieu, A, Matucci Cerinic, M, Mayes, Md, Mckown, Km, Medsger, Ta, Meehan, Rt, Cristina, Ma, Meyer, Kc, Millar, Ab, Moğulkoc, N, Molitor, Ja, Morais, A, Luc Mouthon, P, Müller, V, Müller Quernheim, J, Nadashkevich, O, Nador, R, Nash, P, Nathan, Sd, Navarro, C, Neves, S, Noth, I, Nunes, H, Olson, Al, Opitz, Cf, Padilla, M, Pappas, D, Parfrey, H, Pego Reigosa JM, Pereira, Ca, Perez, R, Pope, Je, Porter, Jc, Renzoni, Ea, Riemekasten, G, Riley, Dj, Rischmueller, M, Rodriguez Reyna TS, Rojas, Serrano, Roman, J, Rosen, Gd, Rossman, M, Rothfield, N, Sahn, Sa, Sanduzzi, A, Scholand, Mb, Selman, M, Senécal, Jl, Seo, P, Silver, Rm, Solomon, Jj, Steen, V, Stevens, W, Strange, C, Sussman, R, Sutton, Ed, Sweiss, Nj, Tornling, G, Tzelepis, Ge, Undurraga, A, Vacca, A, Vancheri, Carlo, Varga, J, Veale, Dj, Volkov, S, Walker, Ua, Wencel, M, Wesselius, Lj, Wickremasinghe, M, Wilcox, P, Wilsher, Ml, Wollheim, Fa, Wuyts, Wa, Yung, G, Zanon, P, Zappala, Cj, Groshong, Sd, Leslie, Ko, Myers, Jl, Padera, Rf, Desai, Sr, Goldin, J, Kazerooni, Ea, Klein, Js, Cenac, Sl, Grewal, Hk, Christensen, Am, Ferguson, S, Tran, M, Keen, K. J., Costabel, Ulrich (Beitragende*r), Raynauds & Scleroderma Association, Arthritis Research UK, The Scleroderma Society, and British Lung Foundation

Subjects
Lung Diseases, Connective tissue disease associated lung disease, CTD-ILD Special Interest Group, International Cooperation, Respiratory System, Medizin, Rheumatoid lung disease, Idiopathic pulmonary fibrosis, Quality of life, QUALITY-OF-LIFE, CYCLOPHOSPHAMIDE, SCLERODERMA LUNG, Registries, Connective Tissue Diseases, Societies, Medical, Randomized Controlled Trials as Topic, Interstitial lung disease, respiratory system, Connective tissue disease, Interstitial Fibrosis, medicine.anatomical_structure, Life Sciences & Biomedicine, Pulmonary and Respiratory Medicine, medicine.medical_specialty, Consensus, Clinical Sciences, END-POINT, Interstitial Lung Disease, Systemic disease and lungs, Medical, medicine, Humans, ENSAIO CLÍNICO CONTROLADO RANDOMIZADO, VALIDITY, Intensive care medicine, Lung, Science & Technology, COUGH, business.industry, Clinical study design, MORTALITY, SYSTEMIC-SCLEROSIS, 1103 Clinical Sciences, Congresses as Topic, medicine.disease, GEORGES RESPIRATORY QUESTIONNAIRE, respiratory tract diseases, Clinical trial, IPF, Physical therapy, Interstitial, Societies, business, Lung Diseases, Interstitial
Abstract

Rationale: Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods: The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results: A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion: Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.

Published
2014
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41. Interstitial lung disease associated with the idiopathic inflammatory myopathies: what progress has been made in the past 35 years?

Author

Connors GR, Christopher-Stine L, Oddis CV, Danoff SK, Connors, Geoffrey R, Christopher-Stine, Lisa, Oddis, Chester V, and Danoff, Sonye K

Abstract

Interstitial lung disease is commonly associated with the autoimmune inflammatory myopathies dermatomyositis and polymyositis and accounts for significant morbidity and mortality in these conditions. In the 35 years since the association between inflammatory myopathy and interstitial lung disease was initially described, there has been progress in diagnosing and treating this dis-order. Nevertheless, there remains much about pathogenesis and therapeutics to be learned. This review examines the changes in the understanding of this complex condition, highlighting recent advances and areas deserving of further study. [ABSTRACT FROM AUTHOR]

Published
2010
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43. Clinical profile of anti-PL-12 autoantibody. Cohort study and review of the literature.

Author

Kalluri M, Sahn SA, Oddis CV, Gharib SL, Christopher-Stine L, Danoff SK, Casciola-Rosen L, Hong G, Dellaripa PF, Highland KB, Kalluri, Meena, Sahn, Steven A, Oddis, Chester V, Gharib, Suzanne L, Christopher-Stine, Lisa, Danoff, Sonye K, Casciola-Rosen, Livia, Hong, Grace, Dellaripa, Paul F, and Highland, Kristin B

Abstract

Background: The antisynthetase syndrome consists of interstitial lung disease (ILD), arthritis, myositis, fever, mechanic's hands, and Raynaud phenomenon in the presence of an antisynthetase autoantibody, most commonly anti-Jo-1. It is believed that all the antisynthetases are associated with a similar clinical profile, but definitive data in this diverse group are lacking. The purpose of this study was to examine the clinical profile of anti-PL-12, an antisynthetase autoantibody directed against alanyl-transfer RNA synthetase.Methods: Thirty-one subjects with anti-PL-12 autoantibody were identified from the databases at the Medical University of South Carolina, the University of Pittsburgh Medical Center, Johns Hopkins Medical Center, and Brigham and Women's Hospital. The medical charts were reviewed and the following data were recorded: demographic information; pulmonary and rheumatologic symptoms; connective tissue disease (CTD) diagnoses; serologic autoantibody findings; CT scan results; BAL findings; pulmonary function test results; lung histopathology; and treatment interventions.Results: The median age at symptom onset was 51 years; 81% were women and 52% were African American. Ninety percent of anti-PL-12-positive patients had ILD, 65% of whom presented initially to a pulmonologist. Ninety percent of anti-PL-12-positive patients had an underlying CTD. Polymyositis and dermatomyositis were the most common underlying diagnoses. Raynaud phenomenon occurred in 65% of patients, fever in 45% of patients, and mechanic's hands in 16% of patients. Test results for the presence of antinuclear antibody were positive in 48% of cases.Conclusions: Anti-PL-12 is strongly associated with the presence of ILD, but less so with myositis and arthritis. Idiopathic ILD diagnosed as idiopathic pulmonary fibrosis may, in fact, be associated with anti-PL-12 and be a "forme fruste" of an underlying autoimmune disorder. [ABSTRACT FROM AUTHOR]

Published
2009
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50. Comprehensive Enteroviral Serology Links Infection and Anti-Melanoma Differentiation-Associated Protein 5 Dermatomyositis.

Author

Jayaraman S, Tiniakou E, Morgenlander WR, Na M, Christopher-Stine L, and Larman HB

Abstract

Objective: Idiopathic inflammatory myopathies (IIMs) are a group of heterogeneous, systemic autoimmune diseases characterized by specific clinical features and, frequently, skeletal muscle inflammation. Specific subtypes of IIMs can be characterized by myositis-specific autoantibodies and are associated with distinct clinical phenotypes. Here, we focus on anti-melanoma differentiation-associated protein 5 (MDA5)-positive myositis and anti-signal recognition particle (SRP)-positive myositis, both of which exhibit seasonality but lack known environmental triggers., Methods: We employed Phage ImmunoPrecipitation Sequencing to profile serum antibodies against the human proteome, the human virome, and a comprehensive enterovirus library. We analyzed sera from 57 patients with anti-MDA5 autoantibodies and 57 patients with anti-SRP autoantibodies, as well as 57 healthy controls. All groups were matched for age, sex, and race., Results: Our autoantibody profiling results define specific immunogenic regions within the MDA5 and SRP autoantigens. We also discovered that in MDA5 sera, versus SRP sera, there was an elevated antibody response to the viral capsid protein 1 (VP1) of enterovirus B, which was accompanied by a decreased antibody response to rhinovirus A., Conclusion: Considering the role of MDA5 as a sensor of picornaviral infections and a mediator of inflammatory signaling, our data suggest a novel etiologic link between enterovirus infection and anti-MDA5 dermatomyositis., (© 2024 The Author(s). ACR Open Rheumatology published by Wiley Periodicals LLC on behalf of American College of Rheumatology.)

Published
2025
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