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1. Treatment of Systemic Sclerosis-associated Interstitial Lung Disease: Evidence-based Recommendations. An Official American Thoracic Society Clinical Practice Guideline.

Author

Raghu, Ganesh, Montesi, Sydney, Silver, Richard, Hossain, Tanzib, Macrea, Madalina, Herman, Derrick, Barnes, Hayley, Adegunsoye, Ayodeji, Azuma, Arata, Chung, Lorinda, Gardner, Gregory, Highland, Kristin, Hudson, Marie, Kaner, Robert, Kolb, Martin, Scholand, Mary, Steen, Virginia, Thomson, Carey, Volkmann, Elizabeth, Wigley, Fredrick, Burlile, Dee, Kemper, Karen, Knight, Shandra, and Ghazipura, Marya

Subjects
SSc-ILD, interstitial lung disease, systemic sclerosis, Humans, United States, Immunosuppressive Agents, Lung Diseases, Interstitial, Cyclophosphamide, Rituximab, Scleroderma, Systemic, Lung
Abstract

Background: Interstitial lung disease (ILD) is a significant cause of morbidity and mortality in patients with systemic sclerosis (SSc). To date, clinical practice guidelines regarding treatment for patients with SSc-ILD are primarily consensus based. Methods: An international expert guideline committee composed of 24 individuals with expertise in rheumatology, SSc, pulmonology, ILD, or methodology, and with personal experience with SSc-ILD, discussed systematic reviews of the published evidence assessed using the Grading of Recommendations, Assessment, Development, and Evaluation approach. Predetermined conflict-of-interest management strategies were applied, and recommendations were made for or against specific treatment interventions exclusively by the nonconflicted panelists. The confidence in effect estimates, importance of outcomes studied, balance of desirable and undesirable consequences of treatment, cost, feasibility, acceptability of the intervention, and implications for health equity were all considered in making the recommendations. This was in accordance with the American Thoracic Society guideline development process, which is in compliance with the Institute of Medicine standards for trustworthy guidelines. Results: For treatment of patients with SSc-ILD, the committee: 1) recommends the use of mycophenolate; 2) recommends further research into the safety and efficacy of (a) pirfenidone and (b) the combination of pirfenidone plus mycophenolate; and 3) suggests the use of (a) cyclophosphamide, (b) rituximab, (c) tocilizumab, (d) nintedanib, and (e) the combination of nintedanib plus mycophenolate. Conclusions: The recommendations herein provide an evidence-based clinical practice guideline for the treatment of patients with SSc-ILD and are intended to serve as the basis for informed and shared decision making by clinicians and patients.

Published
2024

2. CONQUER Scleroderma: association of gastrointestinal tract symptoms in early disease with resource utilization.

Author

Luebker, Sarah, Frech, Tracy, Assassi, Shervin, Skaug, Brian, Gordon, Jessica, Lakin, Kimberly, Bernstein, Elana, Luo, Yiming, Steen, Virginia, Shah, Ami, Hummers, Laura, Richardson, Carrie, Moore, Duncan, Khanna, Dinesh, Castelino, Flavia, Chung, Lorinda, Kapoor, Puneet, Hant, Faye, Shanmugam, Victoria, VanBuren, John, Alvey, Jessica, Harding, Monica, Shah, Ankoor, Makol, Ashima, Lebiedz-Odrobina, Dorota, Thomas, Julie, Volkmann, Elizabeth, Molitor, Jerry, and Sandorfi, Nora

Subjects
SSc, gastrointestinal tract, health outcomes, health status, Humans, Gastrointestinal Diseases, Surveys and Questionnaires, Self Report, Registries, Scleroderma, Systemic
Abstract

OBJECTIVES: SSc is associated with increased health-care resource utilization and economic burden. The Collaborative National Quality and Efficacy Registry (CONQUER) is a US-based collaborative that collects longitudinal follow-up data on SSc patients with

Published
2023

3. Systemic sclerosis associated interstitial lung disease: a conceptual framework for subclinical, clinical and progressive disease.

Author

Roofeh, David, Brown, Kevin, Kazerooni, Ella, Tashkin, Donald, Assassi, Shervin, Martinez, Fernando, Wells, Athol, Raghu, Ganesh, Denton, Christopher, Chung, Lorinda, Hoffmann-Vold, Anna-Maria, Distler, Oliver, Johannson, Kerri, Allanore, Yannick, Matteson, Eric, Kawano-Dourado, Leticia, Pauling, John, Seibold, James, Volkmann, Elizabeth, Walsh, Simon, Oddis, Chester, White, Eric, Barratt, Shaney, Bernstein, Elana, Domsic, Robyn, Dellaripa, Paul, Conway, Richard, Rosas, Ivan, Bhatt, Nitin, Hsu, Vivien, Ingegnoli, Francesca, Kahaleh, Bashar, Garcha, Puneet, Gupta, Nishant, Khanna, Surabhi, Korsten, Peter, Lin, Celia, Mathai, Stephen, Strand, Vibeke, Doyle, Tracy, Steen, Virginia, Zoz, Donald, Ovalles-Bonilla, Juan, Rodriguez-Pinto, Ignasi, Shenoy, Padmanabha, Lewandoski, Andrew, Belloli, Elizabeth, Lescoat, Alain, Nagaraja, Vivek, Ye, Wen, Huang, Suiyuan, Maher, Toby, and Khanna, Dinesh

Subjects
connective tissue disease interstitial lung disease, systemic sclerosis associated interstitial lung disease subsets, systemic sclerosis interstitial lung disease, Humans, Lung Diseases, Interstitial, Scleroderma, Systemic, Vital Capacity, Tomography, X-Ray Computed, Severity of Illness Index, Lung
Abstract

OBJECTIVES: To establish a framework by which experts define disease subsets in systemic sclerosis associated interstitial lung disease (SSc-ILD). METHODS: A conceptual framework for subclinical, clinical and progressive ILD was provided to 83 experts, asking them to use the framework and classify actual SSc-ILD patients. Each patient profile was designed to be classified by at least four experts in terms of severity and risk of progression at baseline; progression was based on 1-year follow-up data. A consensus was reached if ≥75% of experts agreed. Experts provided information on which items were important in determining classification. RESULTS: Forty-four experts (53%) completed the survey. Consensus was achieved on the dimensions of severity (75%, 60 of 80 profiles), risk of progression (71%, 57 of 80 profiles) and progressive ILD (60%, 24 of 40 profiles). For profiles achieving consensus, most were classified as clinical ILD (92%), low risk (54%) and stable (71%). Severity and disease progression overlapped in terms of framework items that were most influential in classifying patients (forced vital capacity, extent of lung involvement on high resolution chest CT [HRCT]); risk of progression was influenced primarily by disease duration. CONCLUSIONS: Using our proposed conceptual framework, international experts were able to achieve a consensus on classifying SSc-ILD patients along the dimensions of disease severity, risk of progression and progression over time. Experts rely on similar items when classifying disease severity and progression: a combination of spirometry and gas exchange and quantitative HRCT.

Published
2023

4. Expert consensus on the management of systemic sclerosis-associated interstitial lung disease.

Author

Rahaghi, Franck, Hsu, Vivien, Kaner, Robert, Mayes, Maureen, Rosas, Ivan, Saggar, Rajan, Steen, Virginia, Strek, Mary, Bernstein, Elana, Bhatt, Nitin, Castelino, Flavia, Chung, Lorinda, Domsic, Robyn, Flaherty, Kevin, Gupta, Nishant, Kahaleh, Bashar, Martinez, Fernando, Morrow, Lee, Moua, Teng, Patel, Nina, Shlobin, Oksana, Southern, Brian, Volkmann, Elizabeth, and Khanna, Dinesh

Subjects
Algorithms, Autoimmune diseases, Connective tissue diseases, Drug therapy, Pulmonary fibrosis, Humans, Consensus, Immunosuppressive Agents, Lung Diseases, Interstitial, Lung, Mycophenolic Acid, Scleroderma, Systemic
Abstract

BACKGROUND: Systemic sclerosis (SSc) is a rare, complex, connective tissue disorder. Interstitial lung disease (ILD) is common in SSc, occurring in 35-52% of patients and accounting for 20-40% of mortality. Evolution of therapeutic options has resulted in a lack of consensus on how to manage this condition. This Delphi study was initiated to develop consensus recommendations based on expert physician insights regarding screening, progression, treatment criteria, monitoring of response, and the role of recent therapeutic advances with antifibrotics and immunosuppressants in patients with SSc-ILD. METHODS: A modified Delphi process was completed by pulmonologists (n = 13) and rheumatologists (n = 12) with expertise in the management of patients with SSc-ILD. Panelists rated their agreement with each statement on a Likert scale from - 5 (complete disagreement) to + 5 (complete agreement). Consensus was predefined as a mean Likert scale score of ≤  - 2.5 or ≥  + 2.5 with a standard deviation not crossing zero. RESULTS: Panelists recommended that all patients with SSc be screened for ILD by chest auscultation, spirometry with diffusing capacity of the lungs for carbon monoxide, high-resolution computed tomography (HRCT), and/or autoantibody testing. Treatment decisions were influenced by baseline and changes in pulmonary function tests, extent of ILD on HRCT, duration and degree of dyspnea, presence of pulmonary hypertension, and potential contribution of reflux. Treatment success was defined as stabilization or improvement of signs or symptoms of ILD and functional status. Mycophenolate mofetil was identified as the initial treatment of choice. Experts considered nintedanib a therapeutic option in patients with progressive fibrotic ILD despite immunosuppressive therapy or patients contraindicated/unable to tolerate immunotherapy. Concomitant use of nintedanib with MMF/cyclophosphamide can be considered in patients with advanced disease at initial presentation, aggressive ILD, or significant disease progression. Although limited consensus was achieved on the use of tocilizumab, the experts considered it a therapeutic option for patients with early SSc and ILD with elevated acute-phase reactants. CONCLUSIONS: This modified Delphi study generated consensus recommendations for management of patients with SSc-ILD in a real-world setting. Findings from this study provide a management algorithm that will be helpful for treating patients with SSc-ILD and addresses a significant unmet need.

Published
2023

5. The association of outdoor temperature and self-reported Raynaud's phenomenon severity among people with systemic sclerosis: a Scleroderma Patient-centered Intervention Network Cohort study

Author

Fortuné, Catherine, Adams, Claire E., Henry, Richard S., El-Baalbaki, Ghassan, Fligelstone, Kim, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Nielsen, Karen, Pope, Janet, Rannou, François, Rodriguez-Reyna, Tatiana Sofía, Shouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Alric, Laurent, André, Marc, Beaslay, Floryan, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Blaise, Sophie, Bories, Eva, Bruns, Alessandra, Cacciatore, Carlotta, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Crichi, Benjamin, Deltombe, Thylbert, Denton, Christopher, Desroche, Tannvir, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge-Bancel, Dominique, Fortin, Paul R., Gauzère, Loraine, Gerber, Anne, Gordon, Jessica, Granel-Rey, Brigitte, Guffroy, Aurélien, Gyger, Geneviève, Hachulla, Erica, Hoa, Sabrina, Hughes, Michael, Ikic, Alena, Khalidi, Nader, Lakin, Kimberly, Lambert, Marc, Launay, David, Lee, Yvonne C., Legendre, Paul, Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Martin Lopez, Maria, Martin, Thierry, Masetto, Ariel, Mekinian, Arsène, Melchor Díaz, Sheila, Mourguet, Morgane, Nikpour, Mandana, Olgane, Louis, Poindron, Vincent, Proudman, Susanna, Pugnet, Grégory, Raffray, Loïc, Régent, Alexis, Renou, Frederic, Rivière, Sébastien, Robinson, David, Rodríguez Almazar, Esther, Roux, Sophie, Smets, Perrine, Sobanski, Vincent, Spiera, Robert, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Vagner, Damien, Varga, John, Wilcox, Pearce, Cañedo Ayala, Mara, Cook, Vanessa, Dal Santo, Cassidy, Dal Santo, Tiffany, D'Onofrio, Monica, Hu, Sophie, Neyer, Marieke Alexandra, Provencher, Sabrina, Virgili-Gervais, Gabrielle, Matthews, Bianca, Nassar, Elsa-Lynn, Carrier, Marie-Eve, Kwakkenbos, Linda, Pauling, John D, Bartlett, Susan J, Gietzen, Amy, Gottesman, Karen, Guillot, Geneviève, Hudson, Marie, Hummers, Laura K, Lawrie-Jones, Amanda, Malcarne, Vanessa L, Mayes, Maureen D, Richard, Michelle, Sauvé, Maureen, Wojeck, Robyn K, Mouthon, Luc, Benedetti, Andrea, and Thombs, Brett D

Published
2024
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6. Nintedanib in Patients With Systemic Sclerosis-Associated Interstitial Lung Disease: Subgroup Analyses by Autoantibody Status and Modified Rodnan Skin Thickness Score.

Author

Kuwana, Masataka, Allanore, Yannick, Denton, Christopher, Distler, Jörg, Steen, Virginia, Khanna, Dinesh, Matucci-Cerinic, Marco, Mayes, Maureen, Volkmann, Elizabeth, Miede, Corinna, Gahlemann, Martina, Quaresma, Manuel, Alves, Margarida, and Distler, Oliver

Subjects
Autoantibodies, Disease Progression, Female, Humans, Indoles, Lung Diseases, Interstitial, Male, Protein Kinase Inhibitors, Scleroderma, Systemic, Skin, Treatment Outcome
Abstract

OBJECTIVE: Using data from the SENSCIS trial, these analyses were undertaken to assess the effects of nintedanib versus placebo in subgroups of patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), based on characteristics previously identified as being associated with the progression of SSc-ILD. METHODS: Patients with SSc-ILD were randomized to receive either nintedanib or placebo, stratified by anti-topoisomerase I antibody (ATA) status. We assessed the rate of decline in forced vital capacity (FVC) (expressed in ml/year) over 52 weeks in subgroups based on baseline ATA status, modified Rodnan skin thickness score (MRSS) ( 0.05), indicating that there was no heterogeneity in the effect of nintedanib versus placebo between these subgroups of patients. CONCLUSION: In patients with SSc-ILD, reduction in the annual rate of decline in FVC among patients receiving nintedanib compared to those receiving placebo was not found to be heterogenous across subgroups based on ATA status, MRSS, or SSc subtype.

Published
2022

8. A comprehensive framework for navigating patient care in systemic sclerosis: A global response to the need for improving the practice of diagnostic and preventive strategies in SSc.

Author

Saketkoo, Lesley, Frech, Tracy, Varjú, Cecília, Domsic, Robyn, Farrell, Jessica, Gordon, Jessica, Mihai, Carina, Sandorfi, Nora, Shapiro, Lee, Poole, Janet, Volkmann, Elizabeth, Lammi, Monika, McAnally, Kendra, Alexanderson, Helene, Pettersson, Henrik, Hant, Faye, Kuwana, Masataka, Shah, Ami, Smith, Vanessa, Hsu, Vivien, Kowal-Bielecka, Otylia, Assassi, Shervin, Cutolo, Maurizio, Kayser, Cristiane, Shanmugam, Victoria, Vonk, Madelon, Fligelstone, Kim, Baldwin, Nancy, Connolly, Kerri, Ronnow, Anneliese, Toth, Beata, Suave, Maureen, Farrington, Sue, Bernstein, Elana, Crofford, Leslie, Czirják, László, Jensen, Kelly, Hinchclif, Monique, Hudson, Marie, Lammi, Matthew, Mansour, Jennifer, Morgan, Nadia, Mendoza, Fabian, Nikpour, Mandana, Pauling, John, Riemekasten, Gabriela, Russell, Anne-Marie, Scholand, Mary, Seigart, Elise, Rodriguez-Reyna, Tatiana, Hummers, Laura, Walker, Ulrich, and Steen, Virginia

Subjects
Disability, Interstitial lung disease, Pulmonary fibrosis, Pulmonary hypertension, Quality of life, Renal crisis, Scleroderma, Symptom burden, Systemic sclerosis, Humans, Hypertension, Pulmonary, Lung, Lung Diseases, Interstitial, Patient Care, Scleroderma, Systemic
Abstract

Systemic sclerosis (SSc), the most lethal of rheumatologic conditions, is the cause of death in >50% of SSc cases, led by pulmonary fibrosis followed by pulmonary hypertension and then scleroderma renal crisis (SRC). Multiple other preventable and treatable SSc-related vascular, cardiac, gastrointestinal, nutritional and musculoskeletal complications can lead to disability and death. Vascular injury with subsequent inflammation transforming to irreversible fibrosis and permanent damage characterizes SSc. Organ involvement is often present early in the disease course of SSc, but requires careful history-taking and vigilance in screening to detect. Inflammation is potentially reversible provided that treatment intensity quells inflammation and other immune mechanisms. In any SSc phenotype, opportunities for early treatment are prone to be under-utilized, especially in slowly progressive phenotypes that, in contrast to severe progressive ILD, indolently accrue irreversible organ damage resulting in later-stage life-limiting complications such as pulmonary hypertension, cardiac involvement, and malnutrition. A single SSc patient visit often requires much more physician and staff time, organization, vigilance, and direct management for multiple organ systems compared to other rheumatic or pulmonary diseases. Efficiency and efficacy of comprehensive SSc care enlists trending of symptoms and bio-data. Financial sustainability of SSc care benefits from understanding insurance reimbursement and health system allocation policies for complex patients. Sharing care between recognised SSc centers and local cardiology/pulmonary/rheumatology/gastroenterology colleagues may prevent complications and poor outcomes, while providing support to local specialists. As scleroderma specialists, we offer a practical framework with tools to facilitate an optimal, comprehensive and sustainable approach to SSc care. Improved health outcomes in SSc relies upon recogntion, management and, to the extent possible, prevention of SSc and treatment-related complications.

Published
2021

10. Predictive Significance of Serum Interferon-Inducible Protein Score for Response to Treatment in Systemic Sclerosis-Related Interstitial Lung Disease.

Author

Assassi, Shervin, Li, Ning, Volkmann, Elizabeth, Mayes, Maureen, Rünger, Dennis, Ying, Jun, Roth, Michael, Hinchcliff, Monique, Khanna, Dinesh, Frech, Tracy, Clements, Philip, Furst, Daniel, Goldin, Jonathan, Bernstein, Elana, Castelino, Flavia, Domsic, Robyn, Gordon, Jessica, Hant, Faye, Shah, Ami, Shanmugam, Victoria, Steen, Virginia, Elashoff, Robert, and Tashkin, Donald

Subjects
Adult, Aged, Chemokine CCL19, Chemokine CCL8, Chemokine CXCL10, Chemokine CXCL9, Cyclophosphamide, Female, Humans, Immunosuppressive Agents, Lung Diseases, Interstitial, Male, Methotrexate, Middle Aged, Mycophenolic Acid, Observational Studies as Topic, Prognosis, Randomized Controlled Trials as Topic, Receptors, Tumor Necrosis Factor, Type II, Scleroderma, Systemic, Vital Capacity, beta 2-Microglobulin
Abstract

OBJECTIVE: Response to immunosuppression is highly variable in systemic sclerosis (SSc)-related interstitial lung disease (ILD). This study was undertaken to determine whether a composite serum interferon (IFN)-inducible protein score exhibits predictive significance for the response to immunosuppression in SSc-ILD. METHODS: Serum samples collected in the Scleroderma Lung Study II, a randomized controlled trial of mycophenolate mofetil (MMF) versus cyclophosphamide (CYC), were examined. Results were validated in an independent observational cohort receiving active treatment. A composite score of 6 IFN-inducible proteins IFNγ-inducible 10-kd protein, monokine induced by IFNγ, monocyte chemotactic protein 2, β2 -microglobulin, tumor necrosis factor receptor type II, and macrophage inflammatory protein 3β) was calculated, and its predictive significance for longitudinal forced vital capacity percent predicted measurements was evaluated. RESULTS: Higher baseline IFN-inducible protein score predicted better response over 3 to 12 months in the MMF arm (point estimate = 0.41, P = 0.001) and CYC arm (point estimate = 0.91, P = 0.009). In contrast, higher baseline C-reactive protein (CRP) levels were predictive of a worse ILD course in both treatment arms. The predictive significance of the IFN-inducible protein score and CRP levels remained after adjustment for baseline demographic and clinical predictors. During the second year of treatment, in which patients in the CYC arm were switched to placebo, a higher IFN-inducible protein score at 12 months showed a trend toward predicting a worse ILD course (point estimate = -0.61, P = 0.068), while it remained predictive of better response to active immunosuppression in the MMF arm (point estimate = 0.28, P = 0.029). The predictive significance of baseline IFN-inducible protein score was replicated in the independent cohort (rs = 0.43, P = 0.028). CONCLUSION: A higher IFN-inducible protein score in SSc-ILD is predictive of better response to immunosuppression and could potentially be used to identify patients who may derive the most benefit from MMF or CYC.

Published
2021

12. Riociguat in patients with early diffuse cutaneous systemic sclerosis (RISE-SSc): open-label, long-term extension of a phase 2b, randomised, placebo-controlled trial

Author

Distler, Oliver, Allanore, Yannick, Denton, Christopher P, Kuwana, Masataka, Matucci-Cerinic, Marco, Pope, Janet E, Atsumi, Tatsuya, Bečvář, Radim, Czirják, László, Hachulla, Eric, Ishii, Tomonori, Ishikawa, Osamu, Johnson, Sindhu R, De Langhe, Ellen, Stagnaro, Chiara, Riccieri, Valeria, Schiopu, Elena, Silver, Richard M, Smith, Vanessa, Steen, Virginia, Stevens, Wendy, Szücs, Gabriella, Truchetet, Marie-Elise, Wosnitza, Melanie, Laapas, Kaisa, Kramer, Frank, and Khanna, Dinesh

Published
2023
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13. Racial Disparities in Systemic Sclerosis: Short- and Long-Term Outcomes Among African American Participants of SLS I and II.

Author

Volkmann, Elizabeth, Steen, Virginia, Li, Ning, Roth, Michael, Clements, Philip, Furst, Daniel, Assassi, Shervin, Khanna, Dinesh, Kim, Grace-Hyun, Goldin, Jonathan, Elashoff, Robert, and Tashkin, Donald

Abstract

OBJECTIVE: To evaluate short- and long-term outcomes of African American (AA) participants of Scleroderma Lung Studies (SLS) I and II. METHODS: SLS I randomized 158 participants with systemic sclerosis-interstitial lung disease (SSc-ILD) to 1 year of oral cyclophosphamide (CYC) versus placebo. SLS II randomized 142 participants with SSc-ILD to 1 year of oral CYC followed by 1 year of placebo versus 2 years of mycophenolate (MMF). Joint models compared the course of forced vital capacity (FVC) and diffusing capacity for carbon monoxide (DLCO) between AA and non-AA, and Cox proportional hazard models assessed long-term morbidity and mortality outcomes. RESULTS: In SLS I, there was no difference in the course of the FVC or DLCO between AA and non-AA in either treatment arm. In SLS II, AA had an improved course of the FVC compared with non-AA in the CYC arm; in the MMF arm, there was no difference in FVC course. There was no difference in DLCO course in either arm. Time to death and respiratory failure were similar for AA and non-AA in SLS I. There was a trend for improved survival and time to respiratory failure in AA compared with non-AA in SLS II. AA race was not independently associated with mortality in the SLS I or II in the Cox models. CONCLUSION: Data from two randomized controlled trials demonstrated that AA patients with SSc-ILD have similar morbidity and mortality outcomes compared with non-AA patients. These findings contrast with the racial disparities described in prior observational studies and warrant further investigation.

Published
2021

14. Patterns of patient-reported symptoms and association with sociodemographic and systemic sclerosis disease characteristics: a scleroderma Patient-centered Intervention Network (SPIN) Cohort cross-sectional study

Author

Adams, Claire E., Henry, Richard S., Fortuné, Catherine, Gottesman, Karen, Guillot, Geneviève, Hummers, Laura K., Lawrie-Jones, Amanda, Mayes, Maureen D., Richard, Michelle, Sauvé, Maureen, Assassi, Shervin, El-Baalbaki, Ghassan, Fligelstone, Kim, Frech, Tracy, Gietzen, Amy, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Nielsen, Karen, Pope, Janet, Rannou, François, Rodriguez-Reyna, Tatiana Sofia, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Abdallah, Nassim Ait, André, Marc, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Crichi, Benjamin, Denton, Christopher, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge-Bancel, Dominique, Fortin, Paul R., Gordon, Jessica, Granel-Rey, Brigitte, Guffroy, Aurélien, Gyger, Genevieve, Hachulla, Eric, Hoa, Sabrina, Ikic, Alena, Kafaja, Suzanne, Khalidi, Nader, Lakin, Kimberly, Lambert, Marc, Launay, David, Lee, Yvonne C., Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Lopez, Maria Martin, Martin, Thierry, Masetto, Ariel, Maurier, François, Mekinian, Arsene, Díaz, Sheila Melchor, Nikpour, Mandana, Olagne, Louis, Poindron, Vincent, Proudman, Susanna, Régent, Alexis, Rivière, Sébastien, Robinson, David, Almazar, Esther Rodríguez, Roux, Sophie, Smets, Perrine, Sobanski, Vincent, Spiera, Robert, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Varga, John, Wilcox, Pearce, Ayala, Mara Cañedo, Cook, Vanessa, Hu, Sophie, Matthews, Bianca, Nassar, Elsa-Lynn, Neyer, Marieke Alexandra, Nordlund, Julia, Provencher, Sabrina, Wojeck, Robyn K., Knisely, Mitchell R., Bailey, Donald E., Somers, Tamara J., Kwakkenbos, Linda, Carrier, Marie-Eve, Nielson, Warren R., Bartlett, Susan J., Malcarne, Vanessa L., Hudson, Marie, Levis, Brooke, Benedetti, Andrea, Mouthon, Luc, Thombs, Brett D., and Silva, Susan G.

Published
2023
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15. Patient Experience of Systemic Sclerosis–Related Calcinosis: An International Study Informing Clinical Trials, Practice, and the Development of the Mawdsley Calcinosis Questionnaire

Author

Saketkoo, Lesley Ann, Gordon, Jessica K., Fligelstone, Kim, Mawdsley, Anne, Chaudhry, Humza A., Valenzuela, Antonia, Christensen, Angela, Khalique, Samara M., Jensen, Kelly, Weinmann, Sophia C., Busman, Evan, Chung, Lorinda, Hsu, Vivien M., Russell, Anne-Marie, and Steen, Virginia D.

Published
2023
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17. Validity, Reliability, and Differential Item Functioning of English and French Versions of the 10‐Item Connor‐Davidson Resilience Scale in Systemic Sclerosis: A Scleroderma Patient‐Centered Intervention Network Cohort Study

Author

Neyer, Marieke A., Henry, Richard S., Carrier, Marie‐Eve, Kwakkenbos, Linda, Wojeck, Robyn K., Gietzen, Amy, Gottesman, Karen, Guillot, Geneviève, Lawrie‐Jones, Amanda, Mayes, Maureen D., Mouthon, Luc, Nielson, Warren R., Richard, Michelle, Worron‐Sauvé, Maureen, Harel, Daphna, Malcarne, Vanessa L., Bartlett, Susan J., Thombs, Brett D., Fortuné, Catherine, Hudson, Marie, Benedetti, Andrea, Hummers, Laura K., Adams, Claire Elizabeth, Ayala, Mara Cañedo, Cook, Vanessa, Hu, Sophie, Matthews, Bianca, Nassar, Elsa‐Lynn, Nordlund, Julia, Provencher, Sabrina, Assassi, Shervin, El‐Baalbaki, Ghassan, Fligelstone, Kim, Frech, Tracy, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Khalidi, Nader, Leite, Catarina, Nguyen, Christelle, Rannou, François, Nielsen, Karen, Pope, Janet, Rodriguez‐Reyna, Tatiana Sofia, Schouffoer, Anne A., Suarez‐Almazor, Maria E., Agard, Christian, André, Marc, Olagne, Louis, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Masetto, Ariel, Roux, Sophie, Cacciatore, Carlotta, Crichi, Benjamin, Farge‐Bancel, Dominique, Carreira, Patricia, Fare, Regina, Lopez, Maria Martin, Díaz, Sheila Melchor, Almazar, Esther Rodríguez, Casadevall, Marion, Chaigne, Benjamin, Dunogue, Bertrand, Régent, Alexis, Chung, Lorinda, Domsic, Robyn, Dunne, James V., Wilcox, Pearce, Fortin, Paul R., Ikic, Alena, Gordon, Jessica, Lakin, Kimberly, Spiera, Robert, Granel‐Rey, Brigitte, Guffroy, Aurélien, Martin, Thierry, Poindron, Vincent, Gyger, Genevieve, Hachulla, Eric, Hoa, Sabrina, Jones, Niall, Lambert, Marc, Launay, David, Maillard, Hélène, Sobanski, Vincent, Lee, Yvonne C., Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Maurier, François, Mekinian, Arsene, Rivière, Sébastien, Nikpour, Mandana, Proudman, Susanna, Robinson, David, Smets, Perrine, Steen, Virginia, Sutton, Evelyn, and Thorne, Carter

Published
2023
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18. HLA and autoantibodies define scleroderma subtypes and risk in African and European Americans and suggest a role for molecular mimicry

Author

Gourh, Pravitt, Safran, Sarah A, Alexander, Theresa, Boyden, Steven E, Morgan, Nadia D, Shah, Ami A, Mayes, Maureen D, Doumatey, Ayo, Bentley, Amy R, Shriner, Daniel, Domsic, Robyn T, Medsger, Thomas A, Ramos, Paula S, Silver, Richard M, Steen, Virginia D, Varga, John, Hsu, Vivien, Saketkoo, Lesley Ann, Schiopu, Elena, Khanna, Dinesh, Gordon, Jessica K, Kron, Brynn, Criswell, Lindsey A, Gladue, Heather, Derk, Chris T, Bernstein, Elana J, Bridges, S Louis, Shanmugam, Victoria K, Kolstad, Kathleen D, Chung, Lorinda, Kafaja, Suzanne, Jan, Reem, Trojanowski, Marcin, Goldberg, Avram, Korman, Benjamin D, Steinbach, Peter J, Chandrasekharappa, Settara C, Mullikin, James C, Adeyemo, Adebowale, Rotimi, Charles, Wigley, Fredrick M, Kastner, Daniel L, Boin, Francesco, and Remmers, Elaine F

Subjects
Biomedical and Clinical Sciences, Immunology, Autoimmune Disease, Rare Diseases, Biotechnology, Minority Health, Genetics, Scleroderma, Clinical Research, 2.1 Biological and endogenous factors, Inflammatory and immune system, Black or African American, Alleles, Amino Acid Sequence, Antigens, Viral, Autoantibodies, Autoantigens, Computational Biology, Datasets as Topic, Female, Genetic Predisposition to Disease, HLA Antigens, Humans, Male, Mimiviridae, Molecular Mimicry, Phycodnaviridae, Protein Structure, Secondary, Risk Assessment, Scleroderma, Systemic, Sequence Homology, Amino Acid, White People, scleroderma, HLA, autoantibodies, molecular mimicry, mimivirus
Abstract

Systemic sclerosis (SSc) is a clinically heterogeneous autoimmune disease characterized by mutually exclusive autoantibodies directed against distinct nuclear antigens. We examined HLA associations in SSc and its autoantibody subsets in a large, newly recruited African American (AA) cohort and among European Americans (EA). In the AA population, the African ancestry-predominant HLA-DRB1*08:04 and HLA-DRB1*11:02 alleles were associated with overall SSc risk, and the HLA-DRB1*08:04 allele was strongly associated with the severe antifibrillarin (AFA) antibody subset of SSc (odds ratio = 7.4). These African ancestry-predominant alleles may help explain the increased frequency and severity of SSc among the AA population. In the EA population, the HLA-DPB1*13:01 and HLA-DRB1*07:01 alleles were more strongly associated with antitopoisomerase (ATA) and anticentromere antibody-positive subsets of SSc, respectively, than with overall SSc risk, emphasizing the importance of HLA in defining autoantibody subtypes. The association of the HLA-DPB1*13:01 allele with the ATA+ subset of SSc in both AA and EA patients demonstrated a transancestry effect. A direct correlation between SSc prevalence and HLA-DPB1*13:01 allele frequency in multiple populations was observed (r = 0.98, P = 3 × 10-6). Conditional analysis in the autoantibody subsets of SSc revealed several associated amino acid residues, mostly in the peptide-binding groove of the class II HLA molecules. Using HLA α/β allelic heterodimers, we bioinformatically predicted immunodominant peptides of topoisomerase 1, fibrillarin, and centromere protein A and discovered that they are homologous to viral protein sequences from the Mimiviridae and Phycodnaviridae families. Taken together, these data suggest a possible link between HLA alleles, autoantibodies, and environmental triggers in the pathogenesis of SSc.

Published
2020

19. Abatacept in Early Diffuse Cutaneous Systemic Sclerosis: Results of a Phase II Investigator‐Initiated, Multicenter, Double‐Blind, Randomized, Placebo‐Controlled Trial

Author

Khanna, Dinesh, Spino, Cathie, Johnson, Sindhu, Chung, Lorinda, Whitfield, Michael L, Denton, Christopher P, Berrocal, Veronica, Franks, Jennifer, Mehta, Bhavan, Molitor, Jerry, Steen, Virginia D, Lafyatis, Robert, Simms, Robert W, Gill, Anna, Kafaja, Suzanne, Frech, Tracy M, Hsu, Vivien, Domsic, Robyn T, Pope, Janet E, Gordon, Jessica K, Mayes, Maureen D, Schiopu, Elena, Young, Amber, Sandorfi, Nora, Park, Jane, Hant, Faye N, Bernstein, Elana J, Chatterjee, Soumya, Castelino, Flavia V, Ajam, Ali, Wang, Yue, Wood, Tammara, Allanore, Yannick, Matucci‐Cerinic, Marco, Distler, Oliver, Singer, Ora, Bush, Erica, Fox, David A, and Furst, Daniel E

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Brain Disorders, Clinical Research, Clinical Trials and Supportive Activities, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Abatacept, Adult, Double-Blind Method, Female, Gene Expression, Gene Expression Profiling, Humans, Male, Middle Aged, Patient Reported Outcome Measures, Scleroderma, Diffuse, Sequence Analysis, RNA, Severity of Illness Index, Skin, Treatment Outcome, Tumor Necrosis Factor Inhibitors, Visual Analog Scale, Vital Capacity, Immunology, Public Health and Health Services, Arthritis & Rheumatology, Clinical sciences
Abstract

ObjectiveT cells play a key role in the pathogenesis of early systemic sclerosis. This study was undertaken to assess the safety and efficacy of abatacept in patients with diffuse cutaneous systemic sclerosis (dcSSc).MethodsIn this 12-month, randomized, double-blind, placebo-controlled trial, participants were randomized 1:1 to receive either subcutaneous abatacept 125 mg or matching placebo, stratified by duration of dcSSc. Escape therapy was allowed at 6 months for worsening disease. The coprimary end points were change in the modified Rodnan skin thickness score (MRSS) compared to baseline and safety over 12 months. Differences in longitudinal outcomes were assessed according to treatment using linear mixed models, with outcomes censored after initiation of escape therapy. Skin tissue obtained from participants at baseline was classified into intrinsic gene expression subsets.ResultsAmong 88 participants, the adjusted mean change in the MRSS at 12 months was -6.24 units for those receiving abatacept and -4.49 units for those receiving placebo, with an adjusted mean treatment difference of -1.75 units (P = 0.28). Outcomes for 2 secondary measures (Health Assessment Questionnaire disability index and a composite measure) were clinically and statistically significantly better with abatacept. The proportion of subjects in whom escape therapy was needed was higher in the placebo group relative to the abatacept group (36% versus 16%). In the inflammatory and normal-like skin gene expression subsets, decline in the MRSS over 12 months was clinically and significantly greater in the abatacept group versus the placebo group (P < 0.001 and P = 0.03, respectively). In the abatacept group, adverse events occurred in 35 participants versus 40 participants in the placebo group, including 2 deaths and 1 death, respectively.ConclusionIn this phase II trial, abatacept was well-tolerated, but change in the MRSS was not statistically significant. Secondary outcome measures, including gene expression subsets, showed evidence in support of abatacept. These data should be confirmed in a phase III trial.

Published
2020

20. Evaluation of Measurement Properties and Differential Item Functioning in the English and French Versions of the University of California, Los Angeles, Loneliness Scale‐6: A Scleroderma Patient‐Centered Intervention Network (SPIN) Study

Author

S. Rapoport, Chelsea, Choi, Alyssa K., Kwakkenbos, Linda, Carrier, Marie‐Eve, Henry, Richard S., Mouthon, Luc, Roesch, Scott C., Thombs, Brett D., Malcarne, Vanessa L., Fortuné, Catherine, Gietzen, Amy, Guillot, Geneviève, Lewis, Nancy, Nielsen, Karen, Sauvé, Maureen, Richard, Michelle, Welling, Joep, Varga, John, Adams, Claire E., Ayala, Mara Cañedo, Cook, Vanessa, Hu, Sophie, Nassar, Elsa‐Lynn, Neyer, Marieke Alexandra, Nordlund, Julia, Provencher, Sabrina, Bartlett, Susan J., Hudson, Marie, Benedetti, Andrea, Gottesman, Karen, Hummers, Laura K., Lawrie‐Jones, Amanda, Mayes, Maureen D., Assassi, Shervin, Nielson, Warren R., El‐Baalbaki, Ghassan, van den Ende, Cornelia, Fligelstone, Kim, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Khalidi, Nader, Leite, Catarina, Nguyen, Christelle, Rannou, François, Pope, Janet, Reyna, Tatiana Sofia Rodriguez, Schouffoer, Anne A., Suarez‐Almazor, Maria E., Agard, Christian, Abdallah, Nassim Ait, Crichi, Benjamin, Farge‐Bancel, Dominique, André, Marc, Olagne, Louis, Smets, Perrine, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Masetto, Ariel, Roux, Sophie, Carreira, Patricia, Fare, Regina, Martin, Maria, Díaz, Sheila Melchor, Almazar, Esther Rodríguez, Casadevall, Marion, Chaigne, Benjamin, Dunogue, Bertrand, Régent, Alexis, Chung, Lorinda, Denton, Christopher, Domsic, Robyn, Dunne, James V., Wilcox, Pearce, Fortin, Paul R., Ikic, Alena, Gordon, Jessica, Lakin, Kimberly, Spiera, Robert, Granel‐Rey, Brigitte, Guffroy, Aurélien, Martin, Thierry, Poindron, Vincent, Gyger, Genevieve, Hachulla, Eric, Lambert, Marc, Launay, David, Maillard, Hélène, Sobanski, Vincent, Hoa, Sabrina, Jones, Niall, Kafaja, Suzanne, Lee, Yvonne C., Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Maurier, François, Mekinian, Arsene, Rivière, Sébastien, Nikpour, Mandana, Proudman, Susanna, Robinson, David, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, and Varga, John

Published
2023
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21. Short-term progression of interstitial lung disease in systemic sclerosis predicts long-term survival in two independent clinical trial cohorts

Author

Volkmann, Elizabeth R, Tashkin, Donald P, Sim, Myung, Li, Ning, Goldmuntz, Ellen, Keyes-Elstein, Lynette, Pinckney, Ashley, Furst, Daniel E, Clements, Philip J, Khanna, Dinesh, Steen, Virginia, Schraufnagel, Dean E, Arami, Shiva, Hsu, Vivien, Roth, Michael D, Elashoff, Robert M, Sullivan, Keith M, and groups, SLS I and SLS II study

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Autoimmune Disease, Lung, Scleroderma, Rare Diseases, Clinical Research, 4.2 Evaluation of markers and technologies, Detection, screening and diagnosis, Inflammatory and immune system, Respiratory, Good Health and Well Being, Adult, Carbon Monoxide, Cyclophosphamide, Disease Progression, Drug Administration Schedule, Female, Humans, Immunosuppressive Agents, Lung Diseases, Interstitial, Male, Middle Aged, Mycophenolic Acid, Proportional Hazards Models, Pulmonary Diffusing Capacity, Risk Factors, Scleroderma, Systemic, Skin, Time Factors, Treatment Outcome, Vital Capacity, SLS I and SLS II study groups, interstitial lung disease, survival, systemic sclerosis, treatment, Immunology, Public Health and Health Services, Arthritis & Rheumatology, Clinical sciences
Abstract

ObjectiveTo assess survival and identify predictors of survival in patients with systemic sclerosis-interstitial lung disease (SSc-ILD) who participated in the Scleroderma Lung Studies (SLS) I and II.MethodsSLS I randomised 158 patients with SSc-ILD to 1  year of oral cyclophosphamide (CYC) vs placebo. SLS II randomised 142 patients to 1 year of oral CYC followed by 1 year of placebo vs 2 years of mycophenolate mofetil. Counting process Cox proportional hazard modelling identified variables associated with long-term mortality in SLS I and II. Internal validation was performed using joint modelling.ResultsAfter a median follow-up of 8 years, 42% of SLS I patients died, and when known the cause of death was most often attributable to SSc. There was no significant difference in the time to death between treatment arms in SLS I or II. Higher baseline skin score, older age, and a decline in the forced vital capacity (FVC) and the diffusing capacity for carbon monoxide (DLCO) over 2 years were independently associated with an increased risk of mortality in SLS I. The Cox model identified the same mortality predictor variables using the SLS II data.ConclusionIn addition to identifying traditional mortality risk factors in SSc (skin score, age), this study demonstrated that a decline in FVC and DLCO over 2 years was a better predictor of mortality than baseline FVC and DLCO. These findings suggest that short-term changes in surrogate measures of SSc-ILD progression may have important effects on long-term outcomes.

Published
2019

24. Pain levels and associated factors in the Scleroderma Patient-centered Intervention Network (SPIN) cohort: a multicentre cross-sectional study

Author

Henry, Richard S., Gottesman, Karen, Hudson, Marie, Hummers, Laura K., Malcarne, Vanessa L., Mayes, Maureen D., Nielson, Warren R., Riggs, Robert, Assassi, Shervin, El-Baalbaki, Ghassan, Ells, Carolyn, Fligelstone, Kim, Fortuné, Catherine, Frech, Tracy, Gietzen, Amy, Guillot, Geneviève, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Nielsen, Karen, Pope, Janet, Rannou, François, Richard, Michelle, Rodriguez-Reyna, Tatiana Sofia, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Ait Abdallah, Nassim, Albert, Alexandra, André, Marc, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Correia, Chase, Crichi, Benjamin, Denton, Christopher, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge-Bancel, Dominique, Fortin, Paul R., Gordon, Jessica, Granel-Rey, Brigitte, Gyger, Genevieve, Hachulla, Eric, Herrick, Ariane L, Hoa, Sabrina, Ikic, Alena, Jones, Niall, Kafaja, Suzanne, Khalidi, Nader, Lambert, Marc, Launay, David, Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Martin, Maria, Martin, Thierry, Masetto, Ariel, Maurier, François, Mekinian, Arsene, Melchor, Sheila, Nikpour, Mandana, Olagne, Louis, Poindron, Vincent, Proudman, Susanna, Régent, Alexis, Rivière, Sébastien, Robinson, David, Rodriguez, Esther, Roux, Sophie, Smets, Perrine, Sobanski, Vincent, Spiera, Robert, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Wilcox, Pearce, Bourgeault, Angelica, Cañedo Ayala, Mara, Carboni Jiménez, Andrea, Discepola, Marie-Nicole, Gagarine, Maria, Nordlund, Julia, Østbø, Nora, Lee, Yvonne C, Fox, Rina S, Kwakkenbos, Linda, Levis, Brooke, Carrier, Marie-Eve, Welling, Joep, Sauvé, Maureen, Mouthon, Luc, Benedetti, Andrea, Bartlett, Susan J, Varga, John, and Thombs, Brett D

Published
2021
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25. Clinical and serological features of systemic sclerosis in a multicenter African American cohort

Author

Morgan, Nadia D, Shah, Ami A, Mayes, Maureen D, Domsic, Robyn T, Medsger, Thomas A, Steen, Virginia D, Varga, John, Carns, Mary, Ramos, Paula S, Silver, Richard M, Schiopu, Elena, Khanna, Dinesh, Hsu, Vivien, Gordon, Jessica K, Gladue, Heather, Saketkoo, Lesley A, Criswell, Lindsey A, Derk, Chris T, Trojanowski, Marcin A, Shanmugam, Victoria K, Chung, Lorinda, Valenzuela, Antonia, Jan, Reem, Goldberg, Avram, Remmers, Elaine F, Kastner, Daniel L, Wigley, Fredrick M, Gourh, Pravitt, and Boin, Francesco

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Lung, Autoimmune Disease, Rare Diseases, Scleroderma, Clinical Research, 4.2 Evaluation of markers and technologies, Detection, screening and diagnosis, Inflammatory and immune system, Adult, Black or African American, Chromosome Mapping, Cohort Studies, Cross-Sectional Studies, Databases, Factual, Female, Humans, Male, Prevalence, Prospective Studies, Retrospective Studies, Scleroderma, Systemic, Severity of Illness Index, Socioeconomic Factors, United States, African Americans, autoantibodies, systemic sclerosis
Abstract

Racial differences exist in the severity of systemic sclerosis (SSc). To enhance our knowledge about SSc in African Americans, we established a comprehensive clinical database from the largest multicenter cohort of African American SSc patients assembled to date (the Genome Research in African American Scleroderma Patients (GRASP) cohort).African American SSc patients were enrolled retrospectively and prospectively over a 30-year period (1987-2016), from 18 academic centers throughout the United States. The cross-sectional prevalence of sociodemographic, clinical, and serological features was evaluated. Factors associated with clinically significant manifestations of SSc were assessed using multivariate logistic regression analyses.The study population included a total of 1009 African American SSc patients, comprised of 84% women. In total, 945 (94%) patients met the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification criteria for SSc, with the remaining 64 (6%) meeting the 1980 ACR or CREST (calcinosis, Raynaud's phenomenon, esophageal dysmotility, sclerodactyly, telangiectasia) criteria. While 43% were actively employed, 33% required disability support. The majority (57%) had the more severe diffuse subtype and a young age at symptom onset (39.1 ± 13.7 years), in marked contrast to that reported in cohorts of predominantly European ancestry. Also, 1 in 10 patients had a severe Medsger cardiac score of 4. Pulmonary fibrosis evident on computed tomography (CT) chest was present in 43% of patients and was significantly associated with anti-topoisomerase I positivity. 38% of patients with CT evidence of pulmonary fibrosis had a severe restrictive ventilator defect, forced vital capacity (FVC) ≤50% predicted. A significant association was noted between longer disease duration and higher odds of pulmonary hypertension, telangiectasia, and calcinosis. The prevalence of potentially fatal scleroderma renal crisis was 7%, 3.5 times higher than the 2% prevalence reported in the European League Against Rheumatism Scleroderma Trials and Research (EUSTAR) cohort.Our study emphasizes the unique and severe disease burden of SSc in African Americans compared to those of European ancestry.

Published
2017

26. Reliability and Validity of the Tender and Swollen Joint Counts and the Modified Rodnan Skin Score in Early Diffuse Cutaneous Systemic Sclerosis: Analysis from the Prospective Registry of Early Systemic Sclerosis Cohort.

Author

Gordon, Jessica K, Girish, Gandikota, Berrocal, Veronica J, Zhang, Meng, Hatzis, Christopher, Assassi, Shervin, Bernstein, Elana J, Domsic, Robyn T, Hant, Faye N, Hinchcliff, Monique, Schiopu, Elena, Steen, Virginia D, Frech, Tracy M, and Khanna, Dinesh

Subjects
Joints, Skin, Humans, Scleroderma, Diffuse, Disability Evaluation, Physical Examination, Severity of Illness Index, Registries, Prospective Studies, Reproducibility of Results, Adult, Middle Aged, Female, Male, Young Adult, DIAGNOSTIC IMAGING, INFECTIONS AND ARTHRITIS, JOINT COUNT, OUTCOME MEASURES, SYSTEMIC SCLEROSIS, ULTRASONOGRAPHY, Clinical Research, Clinical Sciences, Immunology, Public Health and Health Services, Arthritis & Rheumatology
Abstract

ObjectiveTo determine the inter/intraobserver reliability of the tender and swollen joint counts (TJC, SJC) and the modified Rodnan Skin Score (mRSS) in diffuse cutaneous systemic sclerosis (dcSSc) and to assess content validity of the TJC/SJC.MethodsTen rheumatologists completed the SJC, TJC, and mRSS on 7 patients. Musculoskeletal ultrasound (MSUS) was performed.ResultsInterobserver and intraobserver reliability for the TJC was 0.97 and 0.99, for the SJC was 0.24 and 0.71, and for the mRSS was 0.81 and 0.94, respectively. MSUS abnormalities did not correspond with SJC/TJC.ConclusionWe demonstrate excellent inter- and intraobserver reliability for the mRSS and TJC in dcSSc. However, the SJC and TJC did not correspond to MSUS.

Published
2017

27. Minimal Detectable Changes of the Health Assessment Questionnaire–Disability Index, Patient‐Reported Outcomes Measurement Information System‐29 Profile Version 2.0 Domains, and Patient Health Questionnaire‐8 in People With Systemic Sclerosis: A Scleroderma Patient‐Centered Intervention Network Cohort Cross‐Sectional Study

Author

Alkan, Afra, Carrier, Marie‐Eve, Henry, Richard S., Kwakkenbos, Linda, Bartlett, Susan J., Gietzen, Amy, Gottesman, Karen, Guillot, Geneviève, Lawrie‐Jones, Amanda, Hudson, Marie, Hummers, Laura K., Malcarne, Vanessa L., Mayes, Maureen D., Mouthon, Luc, Richard, Michelle, Wojeck, Robyn K., Worron‐Sauvé, Maureen, Benedetti, Andrea, Thombs, Brett D., Fortuné, Catherine, Adams, Claire Elizabeth, El‐Baalbaki, Ghassan, Fligelstone, Kim, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Nielsen, Karen, Pope, Janet, Rannou, François, Rodriguez‐Reyna, Tatiana Sofia, Schouffoer, Anne A., Suarez‐Almazor, Maria E., Agard, Christian, Alric, Laurent, André, Marc, Beaslay, Floryan, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Blaise, Sophie, Bories, Eva, Bruns, Alessandra, Cacciatore, Carlotta, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Crichi, Benjamin, Deltombe, Thylbert, Denton, Christopher P., Desroche, Tannvir, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge‐Bancel, Dominique, Fortin, Paul R., Gauzère, Loraine, Gerber, Anne, Gordon, Jessica K., Granel‐Rey, Brigitte, Guffroy, Aurélien, Gyger, Geneviève, Hachulla, Eric, Hughes, Michael, Ikic, Alena, Jones, Niall, Kafaja, Suzanne, Khalidi, Nader, Lakin, Kimberly S., Lambert, Marc, Launay, David, Lee, Yvonne C., Legendre, Paul, Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Lopez, Maria Martin, Martin, Thierry, Masetto, Ariel, Mekinian, Arsene, Díaz, Sheila Melchor, Mourguet, Morgane, Nikpour, Mandana, Olagne, Louis, Poindron, Vincent, Proudman, Susanna, Pugnet, Grégory, Raffray, Loïc, Régent, Alexis, Renou, Frederic, Rivière, Sébastien, Robinson, David, Almazar, Esther Rodríguez, Roux, Sophie, Smets, Perrine, Sobanski, Vincent, Spiera, Robert F., Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Vagner, Damien, Varga, John, Wilcox, Pearce, Ayala, Mara Cañedo, Research, Medical, Cook, Vanessa, Santo, Cassidy Dal, Santo, Tiffany Dal, D'Onofrio, Monica, Hu, Sophie, Nassar, Elsa‐Lynn, Neyer, Marieke Alexandra, and Provencher, Sabrina

Abstract

Systemic sclerosis (SSc) is a rare, chronic autoimmune disorder associated with disability, diminished physical function, fatigue, pain, and mental health concerns. We assessed minimal detectable changes (MDCs) of the Health Assessment Questionnaire–Disability Index (HAQ‐DI), Patient‐Reported Outcomes Measurement Information System‐29 Profile version 2.0 (PROMIS‐29v2.0) domains, and Patient Health Questionnaire (PHQ)‐8 in people with SSc. Scleroderma Patient‐Centered Intervention Network Cohort participants completed the HAQ‐DI, PROMIS‐29v2.0 domains, and PHQ‐8 at baseline assessments from April 2014 until August 2023. We estimated MDC95 (smallest change that can be detected with 95% certainty) and MDC90 (smallest change that can be detected with 90% certainty) with 95% confidence intervals (CIs) generated via the percentile bootstrapping method resampling 1,000 times. We compared MDC estimates by age, sex, and SSc subtype. A total of 2,571 participants were included. Most were female (n = 2,241; 87%), and 38% (n = 976) had diffuse SSc. Mean (±SD) age was 54.9 (±12.7) years and duration since onset of first non‐Raynaud phenomenon symptom was 10.8 (±8.7) years. MDC95 estimate was 0.41 points (95% CI 0.40–0.42) for the HAQ‐DI, between 4.88 points (95% CI 4.72–5.05) and 9.02 points (95% CI 8.80–9.23) for the seven PROMIS‐29v2.0 domains, and 5.16 points (95% CI 5.06–5.26) for the PHQ‐8. MDC95 estimates were not materially different across subgroups. MDC95 and MDC90 estimates were precise and similar across age, sex, and SSc subtype groups. HAQ‐DI MDC95 and MDC90 were substantially larger than previous estimates of HAQ‐DI minimal important difference from several small studies. Minimally important differences of all measures should be evaluated in large studies using anchor‐based methods.

Published
2024
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29. Factors associated with fears due to COVID-19: A Scleroderma Patient-centered Intervention Network (SPIN) COVID-19 cohort study

Author

Fortuné, Catherine, Gietzen, Amy, Guillot, Geneviève, Lewis, Nancy, Richard, Michelle, Sauvé, Maureen, Welling, Joep, Fligelstone, Kim, Gottesman, Karen, Leite, Catarina, Pérez, Elisabet, Baron, Murray, Malcarne, Vanessa, Mayes, Maureen D., Nielson, Warren R., Riggs, Robert, Assassi, Shervin, Ells, Carolyn, van den Ende, Cornelia, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Hudson, Marie, Johnson, Sindhu R., Larche, Maggie, Nguyen, Christelle, Pope, Janet, Rannou, François, Reyna, Tatiana Sofia Rodriguez, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Albert, Alexandra, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Carreira, Patricia, Chaigne, Benjamin, Chung, Lorinda, Correia, Chase, Denton, Christopher, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Farge-Bancel, Dominique, Fortin, Paul R., Gordon, Jessica, Granel-Rey, Brigitte, Hatron, Pierre-Yves, Herrick, Ariane L., Hoa, Sabrina, Jones, Niall, Fernandes, Artur Jose de B., Kafaja, Suzanne, Khalidi, Nader, Launay, David, Manning, Joanne, Marie, Isabelle, Martin, Maria, Mekinian, Arsene, Melchor, Sheila, Nikpour, Mandana, Olagne, Louis, Proudman, Susanna, Régent, Alexis, Rivière, Sébastien, Robinson, David, Rodriguez, Esther, Roux, Sophie, Sobanski, Vincent, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Wilcox, Pearce, Ayala, Mara Cañedo, Carboni-Jiménez, Andrea, Gagarine, Maria, Nordlund, Julia, Østbø, Nora, Rice, Danielle B., Turner, Kimberly A., Culos-Reed, Nicole, Dyas, Laura, El-Baalbaki, Ghassan, Hebblethwaite, Shannon, Bustamante, Laura, Duchek, Delaney, Ellis, Kelsey, Wu, Yin, Kwakkenbos, Linda, Henry, Richard S., Carrier, Marie-Eve, Harb, Sami, Bourgeault, Angelica, Tao, Lydia, Negeri, Zelalem, Patten, Scott, Bartlett, Susan J., Mouthon, Luc, Varga, John, Benedetti, Andrea, and Thombs, Brett D.

Published
2021
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31. Safety and efficacy of abatacept in early diffuse cutaneous systemic sclerosis (ASSET): open-label extension of a phase 2, double-blind randomised trial

Author

Chung, Lorinda, Spino, Cathie, McLain, Richard, Johnson, Sindhu R, Denton, Christopher P, Molitor, Jerry A, Steen, Virginia D, Lafyatis, Robert, Simms, Robert W, Kafaja, Suzanne, Frech, Tracy M, Hsu, Vivien, Domsic, Robyn T, Pope, Janet E, Gordon, Jessica K, Mayes, Maureen D, Sandorfi, Nora, Hant, Faye N, Bernstein, Elana J, Chatterjee, Soumya, Castelino, Flavia V, Ajam, Ali, Allanore, Yannick, Matucci-Cerinic, Marco, Whitfield, Michael L, Distler, Oliver, Singer, Ora, Young, Amber, Nagaraja, Vivek, Fox, David A, Furst, Daniel E, and Khanna, Dinesh

Published
2020
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32. Changes in mental health symptoms from pre-COVID-19 to COVID-19 among participants with systemic sclerosis from four countries: A Scleroderma Patient-centered Intervention Network (SPIN) Cohort study

Author

Fortuné, Catherine, Gietzen, Amy, Guillot, Geneviève, Lewis, Nancy, Richard, Michelle, Sauvé, Maureen, Welling, Joep, Fligelstone, Kim, Gottesman, Karen, Leite, Catarina, Pérez, Elisabet, Baron, Murray, Malcarne, Vanessa, Mayes, Maureen D., Nielson, Warren R., Riggs, Robert, Assassi, Shervin, Ells, Carolyn, van den Ende, Cornelia, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Hudson, Marie, Johnson, Sindhu R., Larche, Maggie, Nguyen, Christelle, Pope, Janet, Rannou, François, Reyna, Tatiana Sofia Rodriguez, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Albert, Alexandra, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Bruns, Alessandra, Carreira, Patricia, Chaigne, Benjamin, Chung, Lorinda, Correia, Chase, Denton, Christopher, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Farge-Bancel, Dominique, Fortin, Paul R., Gordon, Jessica, Granel-Rey, Brigitte, Hatron, Pierre-Yves, Herrick, Ariane L., Hoa, Sabrina, Jones, Niall, Fernandes, Artur Jose de B., Kafaja, Suzanne, Khalidi, Nader, Launay, David, Manning, Joanne, Marie, Isabelle, Martin, Maria, Mekinian, Arsene, Melchor, Sheila, Nikpour, Mandana, Olagne, Louis, Proudman, Susanna, Régent, Alexis, Rivière, Sébastien, Robinson, David, Rodriguez, Esther, Roux, Sophie, Sobanski, Vincent, Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Wilcox, Pearce, Ayala, Mara Cañedo, Carboni-Jiménez, Andrea, Gagarine, Maria, Nordlund, Julia, Østbø, Nora, Rice, Danielle B., Turner, Kimberly A., Culos-Reed, Nicole, Dyas, Laura, El-Baalbaki, Ghassan, Hebblethwaite, Shannon, Bustamante, Laura, Duchek, Delaney, Ellis, Kelsey, Thombs, Brett D., Kwakkenbos, Linda, Henry, Richard S., Carrier, Marie-Eve, Patten, Scott, Harb, Sami, Bourgeault, Angelica, Tao, Lydia, Bartlett, Susan J., Mouthon, Luc, Varga, John, and Benedetti, Andrea

Published
2020
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34. Tocilizumab in systemic sclerosis: a randomised, double-blind, placebo-controlled, phase 3 trial

Author

Lucero, Eleonora, Pons-Estel, Bernardo, Rivero, Mariano, Tate, Guillermo, Smith, Vanessa, De Langhe, Ellen, Rashkov, Rasho, Batalov, Anastas, Goranov, Ivan, Stoilov, Rumen, Dunne, James, Johnson, Sindhu R., Pope, Janet E., Martinović Kaliterna, Dušanka, Mogensen, Mette, Olesen, Anne Braae, Allanore, Yannick, Henes, Joerg Christoph, Müller-Ladner, Ulf, Riemekasten, Gabriela, Skapenko, Alla, Vlachoyiannopoulos, Panayiotis, Kiss, Emese, Minier, Tünde, Beretta, Lorenzo, Gremese, Elisa, Matucci-Cerinic, Marco, Valentini, Gabriele, Asano, Yoshihide, Atsumi, Tatsuya, Ihn, Hironobu, Ishii, Tomonori, Ishikawa, Osamu, Kuwana, Masataka, Shima, Yoshihito, Takahashi, Hiroki, Takehara, Kazuhiko, Tanaka, Yoshiya, Yamasaki, Yoshioki, Bukauskiene, Loreta, Butrimiene, Irena, Medrano Ramirez, Gabriel, Ramos-Remus, Cesar, Sofia Rodriguez Reyna, Tatiana, de Vries-Bouwstra, Jeska, van Laar, Jacob M., Batko, Bogdan, Jeka, Slawomir, Kucharz, Eugeniusz, Majdan, Maria, Olesinska, Marzena, Smolenska, Zaneta, Alves, Jose, Santos, Maria, Mihai, Carmen Marina, Rednic, Simona, Castellvi Barranco, Ivan, Lopez Longo, Francisco Javier, Simeon Aznar, Carmen, Carreira, Patricia, Distler, Oliver, Walker, Ulrich A., Derrett-Smith, Emma, Griffiths, Bridget, McKay, Neil, Denton, Christopher P., Aelion, Jacob, Borofsky, Michael, Fleischmann, Roy, Forstot, Joseph Z., Furst, Daniel E., Kafaja, Suzanne, Khan, M. Faisal, Khanna, Dinesh, Kohen, Michael D., Martin, Richard W., Mendoza-Ballesteros, Fabian, Nami, Alireza, Pang, Shirley, Rios, Grissel, Simms, Robert, Sullivan, Keith Michael, Steen, Virginia D., Lin, Celia J F, Furst, Daniel E, Goldin, Jonathan, Kim, Grace, van Laar, Jacob M, Spotswood, Helen, Wagner, Bridget, Siegel, Jeffrey, Jahreis, Angelika, and Denton, Christopher P

Published
2020
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36. Protocol for a partially nested randomised controlled trial to evaluate the effectiveness of the scleroderma patient-centered intervention network COVID-19 home-isolation activities together (SPIN-CHAT) program to reduce anxiety among at-risk scleroderma patients

Author

Fortuné, Catherine, Gietzen, Amy, Guillot, Geneviève, Lewis, Nancy, Nielsen, Karen, Richard, Michelle, Sauvé, Maureen, Welling, Joep, Baron, Murray, Furst, Daniel E., Gottesman, Karen, Malcarne, Vanessa, Mayes, Maureen D., Mouthon, Luc, Nielson, Warren R., Riggs, Robert, Wigley, Fredrick, Assassi, Shervin, Boutron, Isabelle, Ells, Carolyn, van den Ende, Cornelia, Fligelstone, Kim, Frech, Tracy, Godard, Dominique, Harel, Daphna, Hinchcliff, Monique, Hudson, Marie, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Pope, Janet, Portales, Alexandra, Rannou, François, Reyna, Tatiana Sofia Rodriguez, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Albert, Alexandra, André, Marc, Arsenault, Guylaine, Benzidia, Ilham, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Boire, Gilles, Bruns, Alessandra, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Cohen, Pascal, Correia, Chase, Dagenais, Pierre, Denton, Christopher, Domsic, Robyn, Dubois, Sandrine, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge-Bancel, Dominique, Fortin, Paul R., Gill, Anna, Gordon, Jessica, Granel-Rey, Brigitte, Gyger, Genevieve, Hachulla, Eric, Hatron, Pierre-Yves, Herrick, Ariane L., Hij, Adrian, Hoa, Sabrina, Ikic, Alena, Jones, Niall, de B. Fernandes, Artur Jose, Kafaja, Suzanne, Khalidi, Nader, Lambert, Marc, Launay, David, Liang, Patrick, Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Martin, Maria, Martin, Thierry, Masetto, Ariel, Maurier, François, Mekinian, Arsene, Melchor, Sheila, Nikpour, Mandana, Olagne, Louis, Poindron, Vincent, Proudman, Susanna, Régent, Alexis, Rivière, Sébastien, Robinson, David, Rodriguez, Esther, Roux, Sophie, Smets, Perrine, Smith, Doug, Sobanski, Vincent, Spiera, Robert, Steen, Virginia, Stevens, Wendy, Sutton, Evelyn, Terrier, Benjamin, Thorne, Carter, Varga, John, Wilcox, Pearce, Ayala, Mara Cañedo, Ostbo, Nora, Thombs, Brett D., Kwakkenbos, Linda, Carrier, Marie-Eve, Bourgeault, Angelica, Tao, Lydia, Harb, Sami, Gagarine, Maria, Rice, Danielle, Bustamante, Laura, Ellis, Kelsey, Duchek, Delaney, Wu, Yin, Bhandari, Parash Mani, Neupane, Dipika, Carboni-Jiménez, Andrea, Henry, Richard S., Krishnan, Ankur, Sun, Ying, Levis, Brooke, He, Chen, Turner, Kimberly A., Benedetti, Andrea, Culos-Reed, Nicole, El-Baalbaki, Ghassan, Hebblethwaite, Shannon, Bartlett, Susan J., Dyas, Laura, and Patten, Scott

Published
2020
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38. The American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis

Author

Khanna, Dinesh, Berrocal, Veronica J, Giannini, Edward H, Seibold, James R, Merkel, Peter A, Mayes, Maureen D, Baron, Murray, Clements, Philip J, Steen, Virginia, Assassi, Shervin, Schiopu, Elena, Phillips, Kristine, Simms, Robert W, Allanore, Yannick, Denton, Christopher P, Distler, Oliver, Johnson, Sindhu R, Matucci-Cerinic, Marco, Pope, Janet E, Proudman, Susanna M, Siegel, Jeffrey, Wong, Weng Kee, Wells, Athol U, and Furst, Daniel E

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Scleroderma, Inflammatory and immune system, Adult, Female, Humans, Male, Middle Aged, Psychometrics, Randomized Controlled Trials as Topic, Rheumatology, Scleroderma, Diffuse, Severity of Illness Index, Public Health and Health Services, Psychology, Clinical sciences, Allied health and rehabilitation science
Abstract

ObjectiveEarly diffuse cutaneous systemic sclerosis (dcSSc) is characterized by rapid changes in the skin and internal organs. The objective of this study was to develop a composite response index in dcSSc (CRISS) for use in randomized controlled trials (RCTs).MethodsWe developed 150 paper patient profiles with standardized clinical outcome elements (core set items) using patients with dcSSc. Forty scleroderma experts rated 20 patient profiles each and assessed whether each patient had improved or not improved over a period of 1 year. Using the profiles for which raters had reached a consensus on whether the patients were improved versus not improved (79% of the profiles examined), we fit logistic regression models in which the binary outcome referred to whether the patient was improved or not, and the changes in the core set items from baseline to followup were entered as covariates. We tested the final index in a previously completed RCT.ResultsSixteen of 31 core items were included in the patient profiles after a consensus meeting and review of test characteristics of patient-level data. In the logistic regression model in which the included core set items were change over 1 year in the modified Rodnan skin thickness score, the forced vital capacity, the patient and physician global assessments, and the Health Assessment Questionnaire disability index, sensitivity was 0.982 (95% confidence interval 0.982-0.983) and specificity was 0.931 (95% confidence interval 0.930-0.932), and the model with these 5 items had the highest face validity. Subjects with a significant worsening of renal or cardiopulmonary involvement were classified as not improved, regardless of improvements in other core items. With use of the index, the effect of methotrexate could be differentiated from the effect of placebo in a 1-year RCT (P = 0.02).ConclusionWe have developed a CRISS that is appropriate for use as an outcome assessment in RCTs of early dcSSc.

Published
2016

39. Clinical characteristics, visceral involvement, and mortality in at-risk or early diffuse systemic sclerosis: a longitudinal analysis of an observational prospective multicenter US cohort

Author

Jaafar, Sara, Lescoat, Alain, Huang, Suiyuan, Gordon, Jessica, Hinchcliff, Monique, Shah, Ami A., Assassi, Shervin, Domsic, Robyn, Bernstein, Elana J., Steen, Virginia, Elliott, Sabrina, Hant, Faye, Castelino, Flavia V., Shanmugam, Victoria K., Correia, Chase, Varga, John, Nagaraja, Vivek, Roofeh, David, Frech, Tracy, and Khanna, Dinesh

Published
2021
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41. Prognostic significance of pericardial effusion in systemic sclerosis-associated pulmonary hypertension: analysis from the PHAROS Registry.

Author

Luo, Yiming, Gordon, Jessica K, Xu, Jiehui, Kolstad, Kathleen D, Chung, Lorinda, Steen, Virginia D, Bernstein, Elana J, and Investigators, PHAROS

Subjects
PERICARDIAL effusion, RESEARCH funding, PULMONARY hypertension, SCIENTIFIC observation, FISHER exact test, DESCRIPTIVE statistics, MANN Whitney U Test, LONGITUDINAL method, KAPLAN-Meier estimator, LOG-rank test, SYSTEMIC scleroderma, RESEARCH, STATISTICS, DATA analysis software, PROPORTIONAL hazards models, DISEASE complications
Abstract

Objectives Pulmonary hypertension (PH) is a leading cause of death in patients with SSc. The purpose of this study was to determine the prognostic significance of pericardial effusion in patients with SSc-PH. Methods Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma (PHAROS) is a prospective multicentre registry which enrolled patients with newly diagnosed SSc-PH from 2005 to 2016. The prognostic impact of pericardial effusion status, including those who ever or never had pericardial effusion, and those who had persistent or intermittent pericardial effusion, was analysed. Kaplan–Meier survival analyses, log-rank test, and multivariable Cox proportional hazards regression were performed. Results Of the 335 patients with SSc-PH diagnosed by right heart catheterization and documentation of pericardial effusion presence or absence on echocardiogram, 166 (50%) ever had pericardial effusion. Ever having pericardial effusion was not predictive of survival (log-rank test P  = 0.49). Of the 245 SSc-PH patients who had at least two echocardiograms, 44% had a change in pericardial effusion status over an average of 4.3 years of follow up. Having a persistent pericardial effusion was an independent predictor of survival [adjusted hazard ratio (aHR)=2.34, 95% CI 1.20, 4.64, P  = 0.002], while intermittent pericardial effusion was not a predictor of survival (aHR = 0.89, 95% CI 0.52, 1.56, P  = 0.68), in a multivariable-adjusted analysis. Conclusion Persistent pericardial effusion, but not ever having had pericardial effusion or intermittent pericardial effusion, was independently associated with poorer survival. Incorporating information from serial echocardiograms may help clinicians better prognosticate survival in their SSc-PH patients. [ABSTRACT FROM AUTHOR]

Published
2024
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44. Experiences of telehealth during and after the COVID-19 pandemic and preferences for future care of people with systemic sclerosis: a cross-sectional study

Author

Adams, Claire E., Gottesman, Karen, Hudson, Marie, Hummers, Laura K., Lawrie-Jones, Amanda, Malcarne, Vanessa L., Mayes, Maureen D., Richard, Michelle, Wojeck, Robyn K., El-Baalbaki, Ghassan, Fligelstone, Kim, Frech, Tracy, Harel, Daphna, Hinchcliff, Monique, Johnson, Sindhu R., Larche, Maggie, Leite, Catarina, Nguyen, Christelle, Nielsen, Karen, Pope, Janet, Rannou, François, Rodriguez-Reyna, Tatiana Sofia, Schouffoer, Anne A., Suarez-Almazor, Maria E., Agard, Christian, Alric, Laurent, André, Marc, Beaslay, Floryan, Bernstein, Elana J., Berthier, Sabine, Bissonnette, Lyne, Blaise, Sophie, Bories, Eva, Bruns, Alessandra, Cacciatore, Carlotta, Carreira, Patricia, Casadevall, Marion, Chaigne, Benjamin, Chung, Lorinda, Crichi, Benjamin, Deltombe, Thylbert, Denton, Christopher, Desroche, Tannvir, Domsic, Robyn, Dunne, James V., Dunogue, Bertrand, Fare, Regina, Farge-Bancel, Dominique, Fortin, Paul R., Gauzère, Loraine, Gerber, Anne, Gordon, Jessica K., Granel-Rey, Brigitte, Guffroy, Aurélien, Gyger, Geneviève, Hachulla, Eric, Hoa, Sabrina, Hughes, Michael, Ikic, Alena, Khalidi, Nader, Lakin, Kimberly S., Lambert, Marc, Launay, David, Lee, Yvonne C., Legendre, Paul, Maillard, Hélène, Maltez, Nancy, Manning, Joanne, Marie, Isabelle, Martin Lopez, Maria, Martin, Thierry, Masetto, Ariel, Mekinian, Arsène, Melchor Díaz, Sheila, Mourguet, Morgane, Nikpour, Mandana, Olagne, Louis, Poindron, Vincent, Proudman, Susanna, Pugnet, Grégory, Raffray, Loïc, Régent, Alexis, Renou, Frederic, Rivière, Sébastien, Robinson, David, Rodríguez Almazar, Esther, Roux, Sophie, Smets, Perrine, Sobanski, Vincent, Spiera, Robert F., Steen, Virginia, Sutton, Evelyn, Thorne, Carter, Vagner, Damien, Wilcox, Pearce, Ayala, Mara Cañedo, Cook, Vanessa, Dal Santo, Cassidy, Dal Santo, Tiffany, D'onofrio, Monica, Neyer, Marieke Alexandra, Nassar, Elsa-Lynn, Virgili-Gervais, Gabrielle, Carrier, Marie-Eve, Kwakkenbos, Linda, Henry, Richard S, Hu, Sophie, Provencher, Sabrina, Golberg, Meira, Bartlett, Susan J, Mouthon, Luc, Patten, Scott B, Varga, John, Benedetti, Andrea, and Thombs, Brett D

Published
2024
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45. Long-Term Outcomes in Systemic Sclerosis-Associated Pulmonary Arterial Hypertension From the Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma Registry (PHAROS)

Author

Bolster, Marcy B., Csuka, Mary Ellen, Derk, Chris T., Domsic, Robyn T., Fischer, Aryeh, Frech, Tracy, Furie, Richard, Furst, Daniel, Goldberg, Avram Z., Gomberg-Maitland, Mardi, Gordon, Jessica, Hant, Faye, Hill, Nicholas, Hinchcliff, Monique, Horn, Evelyn, Hummers, Laura, Hsu, Vivien, Kafaja, Susanna, Kassab, Firas, Khanna, Dinesh, Medsger, Thomas A., Preston, Ioana, Molitor, Jerry, Saketkoo, Lesley, Shapiro, Lee, Schiopu, Elena, Silver, Rick, Simms, Robert, Varga, John, Wigley, Frederick, Kolstad, Kathleen D., Li, Shufeng, Steen, Virginia, and Chung, Lorinda

Published
2018
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47. Combination of Echocardiographic and Pulmonary Function Test Measures Improves Sensitivity for Diagnosis of Systemic Sclerosis-associated Pulmonary Arterial Hypertension: Analysis of 2 Cohorts

Author

Gladue, Heather, Steen, Virginia, Allanore, Yannick, Saggar, Rajeev, Saggar, Rajan, Maranian, Paul, Berrocal, Veronica J, Avouac, Jerome, Meune, Christophe, Trivedi, Mona, and Khanna, Dinesh

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Rare Diseases, Lung, Clinical Research, Scleroderma, Autoimmune Disease, Detection, screening and diagnosis, 4.1 Discovery and preclinical testing of markers and technologies, 4.2 Evaluation of markers and technologies, Cardiovascular, Adult, Aged, Cohort Studies, Familial Primary Pulmonary Hypertension, Female, Humans, Hypertension, Pulmonary, Male, Middle Aged, Pulmonary Artery, Respiratory Function Tests, Scleroderma, Systemic, Sensitivity and Specificity, Ultrasonography, ECHOCARDIOGRAM, PULMONARY FUNCTION TESTS, SYSTEMIC SCLEROSIS, PULMONARY HYPERTENSION, DIAGNOSIS, PULMONARY ARTERIAL HYPERTENSION, Immunology, Public Health and Health Services, Arthritis & Rheumatology, Clinical sciences
Abstract

ObjectiveTo evaluate routinely collected non-invasive tests from 2 systemic sclerosis (SSc) cohorts to determine their predictive value alone and in combination versus right heart catheterization (RHC)-confirmed pulmonary arterial hypertension (PAH).MethodsWe evaluated 2 cohorts of patients who were at risk or with incident PAH: (1) The Pulmonary Hypertension Assessment and Recognition Outcomes in Scleroderma (PHAROS) cohort and (2) an inception SSc cohort at Cochin Hospital, Paris, France. Estimated right ventricular systolic pressure (eRVSP) as determined by transthoracic echocardiogram (TTE) and pulmonary function test (PFT) measures was evaluated, and the predictive values determined. We then evaluated patients with PAH missed on TTE cutoffs that were subsequently identified by a PFT measure.ResultsIn the PHAROS cohort (n = 206), 59 (29%) had RHC-defined PAH. An eRVSP threshold of 35-50 mm Hg failed to diagnose PAH in 7% to 31% of patients, 50% to 70% of which (n = 2-13) were captured by PFT measures. In the Cochin cohort (n = 141), 10 (7%) patients had RHC confirmed PAH. An eRVSP threshold of 35-50 mm Hg missed 0% to 70% (n = 0-7) of patients, of which 0% to 68% (n = 0-6) were met by PFT measures. The combination of TTE and PFT improved the negative predictive value for diagnosing PAH.ConclusionIn 2 large SSc cohorts, screening with TTE and PFT captured a majority of patients with PAH. TTE and PFT complement each other for the diagnosis of PAH.

Published
2013

48. Pregnancy

Author

Chakravarty, Eliza F., Steen, Virginia, Varga, John, editor, Denton, Christopher P., editor, Wigley, Fredrick M., editor, Allanore, Yannick, editor, and Kuwana, Masataka, editor

Published
2017
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50. Patterns of patient-reported symptoms and association with sociodemographic and systemic sclerosis disease characteristics: a scleroderma Patient-centered Intervention Network (SPIN) Cohort cross-sectional study

Author

Wojeck, Robyn K., primary, Knisely, Mitchell R., additional, Bailey, Donald E., additional, Somers, Tamara J., additional, Kwakkenbos, Linda, additional, Carrier, Marie-Eve, additional, Nielson, Warren R., additional, Bartlett, Susan J., additional, Malcarne, Vanessa L., additional, Hudson, Marie, additional, Levis, Brooke, additional, Benedetti, Andrea, additional, Mouthon, Luc, additional, Thombs, Brett D., additional, Silva, Susan G., additional, Adams, Claire E., additional, Henry, Richard S., additional, Fortuné, Catherine, additional, Gottesman, Karen, additional, Guillot, Geneviève, additional, Hummers, Laura K., additional, Lawrie-Jones, Amanda, additional, Mayes, Maureen D., additional, Richard, Michelle, additional, Sauvé, Maureen, additional, Assassi, Shervin, additional, El-Baalbaki, Ghassan, additional, Fligelstone, Kim, additional, Frech, Tracy, additional, Gietzen, Amy, additional, Harel, Daphna, additional, Hinchcliff, Monique, additional, Johnson, Sindhu R., additional, Larche, Maggie, additional, Leite, Catarina, additional, Nguyen, Christelle, additional, Nielsen, Karen, additional, Pope, Janet, additional, Rannou, François, additional, Rodriguez-Reyna, Tatiana Sofia, additional, Schouffoer, Anne A., additional, Suarez-Almazor, Maria E., additional, Agard, Christian, additional, Abdallah, Nassim Ait, additional, André, Marc, additional, Bernstein, Elana J., additional, Berthier, Sabine, additional, Bissonnette, Lyne, additional, Bruns, Alessandra, additional, Carreira, Patricia, additional, Casadevall, Marion, additional, Chaigne, Benjamin, additional, Chung, Lorinda, additional, Crichi, Benjamin, additional, Denton, Christopher, additional, Domsic, Robyn, additional, Dunne, James V., additional, Dunogue, Bertrand, additional, Fare, Regina, additional, Farge-Bancel, Dominique, additional, Fortin, Paul R., additional, Gordon, Jessica, additional, Granel-Rey, Brigitte, additional, Guffroy, Aurélien, additional, Gyger, Genevieve, additional, Hachulla, Eric, additional, Hoa, Sabrina, additional, Ikic, Alena, additional, Kafaja, Suzanne, additional, Khalidi, Nader, additional, Lakin, Kimberly, additional, Lambert, Marc, additional, Launay, David, additional, Lee, Yvonne C., additional, Maillard, Hélène, additional, Maltez, Nancy, additional, Manning, Joanne, additional, Marie, Isabelle, additional, Lopez, Maria Martin, additional, Martin, Thierry, additional, Masetto, Ariel, additional, Maurier, François, additional, Mekinian, Arsene, additional, Díaz, Sheila Melchor, additional, Nikpour, Mandana, additional, Olagne, Louis, additional, Poindron, Vincent, additional, Proudman, Susanna, additional, Régent, Alexis, additional, Rivière, Sébastien, additional, Robinson, David, additional, Almazar, Esther Rodríguez, additional, Roux, Sophie, additional, Smets, Perrine, additional, Sobanski, Vincent, additional, Spiera, Robert, additional, Steen, Virginia, additional, Sutton, Evelyn, additional, Thorne, Carter, additional, Varga, John, additional, Wilcox, Pearce, additional, Ayala, Mara Cañedo, additional, Cook, Vanessa, additional, Hu, Sophie, additional, Matthews, Bianca, additional, Nassar, Elsa-Lynn, additional, Neyer, Marieke Alexandra, additional, Nordlund, Julia, additional, and Provencher, Sabrina, additional

Published
2023
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