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1. Cohort Enrichment Strategies for Progressive Interstitial Lung Disease in Systemic Sclerosis From European Scleroderma Trials and Research

Author

Hoffmann-Vold A. -M., Brunborg C., Airo P., Ananyeva L. P., Czirjak L., Guiducci S., Hachulla E., Li M., Mihai C., Riemekasten G., Sfikakis P. P., Valentini G., Kowal-Bielecka O., Allanore Y., Distler O., Vacca A., Giollo A., Balbir-Gurman A., Gheorghiu A. M., Marcoccia A., Herrick A., Radic M., Stamenkovic B., Anic B., Granel B., Ribi C., Selmi C. F., Carlos de la Puente M., de Souza Muller C., Denton C., Kayser C., Tanaseanu C. -M., Majewski D., Rimar D., Krasowska D., Veale D., Walker U., Kerzberg E., Rezus E., Zanatta E., Siegert E., De Langhe E., Oksel F., Ingegnoli F., Cantatore F. P., Szucs G., Cuomo G., Seskute G., Litinsky V., Castellvi I., Morovic-Vergles J., Sibilia J., Henes J., Solanki K., Perdan-Pirkmajer K., Herrmann K., Saketkoo L. A., Stamp L., Mouthon L., Salvador M. J., Pozzi M. R., Uprus M., Vanthuyne M., Engelhart M., Kohm M., Iudici M., Inanc M., Fathi N., Pamuk N., Garcia de la Pena Lefebv P., Carreira P. E., Bancel D. F., Moroncini L., Montecucco C., Ancuta C., Sunderkotter C., Muller-Ladner U., Rosato E., Kucharz E. J., Iannone F., Del Galdo F., Poormoghim H., Kotter I., Distler J., Cutolo M., Tikly M., Damjanov N., Hunzelmann N., Vlachoyiannopoulos P., Hasler P., Sarzi Puttini P., Wiland P., Becvar R., Yavuz S., Zdrojewski Z., Pellerito R., Foti R., Ionescu R. M., Adler S., Kahl S., Moiseev S., Stebbings S., Rednic S., Negrini S., Heitmann S., Ullman S., Agachi S., Martin T., Schmeiser T., Riccieri V., Smith V., Bernardino V., Ortiz-Santamaria V., Hsu V. M., Abdel Atty Mohamed W. A., Hoffmann-Vold, A. -M., Brunborg, C., Airo, P., Ananyeva, L. P., Czirjak, L., Guiducci, S., Hachulla, E., Li, M., Mihai, C., Riemekasten, G., Sfikakis, P. P., Valentini, G., Kowal-Bielecka, O., Allanore, Y., Distler, O., Vacca, A., Giollo, A., Balbir-Gurman, A., Gheorghiu, A. M., Marcoccia, A., Herrick, A., Radic, M., Stamenkovic, B., Anic, B., Granel, B., Ribi, C., Selmi, C. F., Carlos de la Puente, M., de Souza Muller, C., Denton, C., Kayser, C., Tanaseanu, C. -M., Majewski, D., Rimar, D., Krasowska, D., Veale, D., Walker, U., Kerzberg, E., Rezus, E., Zanatta, E., Siegert, E., De Langhe, E., Oksel, F., Ingegnoli, F., Cantatore, F. P., Szucs, G., Cuomo, G., Seskute, G., Litinsky, V., Castellvi, I., Morovic-Vergles, J., Sibilia, J., Henes, J., Solanki, K., Perdan-Pirkmajer, K., Herrmann, K., Saketkoo, L. A., Stamp, L., Mouthon, L., Salvador, M. J., Pozzi, M. R., Uprus, M., Vanthuyne, M., Engelhart, M., Kohm, M., Iudici, M., Inanc, M., Fathi, N., Pamuk, N., Garcia de la Pena Lefebv, P., Carreira, P. E., Bancel, D. F., Moroncini, L., Montecucco, C., Ancuta, C., Sunderkotter, C., Muller-Ladner, U., Rosato, E., Kucharz, E. J., Iannone, F., Del Galdo, F., Poormoghim, H., Kotter, I., Distler, J., Cutolo, M., Tikly, M., Damjanov, N., Hunzelmann, N., Vlachoyiannopoulos, P., Hasler, P., Sarzi Puttini, P., Wiland, P., Becvar, R., Yavuz, S., Zdrojewski, Z., Pellerito, R., Foti, R., Ionescu, R. M., Adler, S., Kahl, S., Moiseev, S., Stebbings, S., Rednic, S., Negrini, S., Heitmann, S., Ullman, S., Agachi, S., Martin, T., Schmeiser, T., Riccieri, V., Smith, V., Bernardino, V., Ortiz-Santamaria, V., Hsu, V. M., and Abdel Atty Mohamed, W. A.

Subjects
interstitial lung disease, Pulmonary and Respiratory Medicine, enrichment, systemic sclerosis, clinical trial, Cardiology and Cardiovascular Medicine, Critical Care and Intensive Care Medicine
Abstract

BACKGROUND: Enrichment strategies from clinical trials for progressive systemic sclerosis-associated interstitial lung disease (SSc-ILD) have not been tested in a real-life cohort.RESEARCH QUESTION: Do enrichment strategies for progressive ILD impact efficacy, repre-sentativeness, and feasibility in patients with SSc-ILD from the European Scleroderma Trials and Research (EUSTAR) database?STUDY DESIGN AND METHODS: We applied the inclusion criteria of major recent SSc-ILD trials (Study of the Efficacy and Safety of Tocilizumab in Participants With Systemic Sclerosis [focuSSced], Scleroderma Lung Study II [SLS II], and Safety and Efficacy of Nintedanib in Systemic Sclerosis [SENSCIS]) and assessed progressive ILD, which was defined as absolute change in FVC and as significant progression (FVC decline $10%). Data were compared with all patients and with patients who did not fulfill any inclusion criteria. RESULTS: In total, 2,258 patients with SSc-ILD were included: 31.2% of the patients met SENSCIS criteria; 5.8% of the patients met SLS II criteria; 1.6% of the patients met focuSSced criteria, and 67.7% (1,529) of the patients did not meet any criteria. In the first 12 + 3 months, the absolute FVC decline in all patients and in patients who fulfilled criteria from SENSCIS was -0.1%, in patients who fulfilled criteria from focuSSced was -3.7%, and in patients who fulfilled criteria from SLS II was 2.3%, with accompanying more progressors in focuSSced. The patient populations that fulfilled the different study inclusion criteria significantly differed in various clinical parameters. In the second 12-month period, SENSCIS-enriched patients had a further absolute FVC% decline as described for the total cohort. In contrast, patients who fulfilled the focuSSced and SLS II criteria showed numeric improvement of lung function. There were no significant associations of enrichment criteria and ILD progression.INTERPRETATION: The application of enrichment criteria from previous clinical trials showed enrichment for progression with variable success, which led to selected patient populations reducing feasibility of recruitment. These findings are important for future clinical trial design and interpretation of the results of published trials.CHEST 2023; 163(3):586-598

Published
2023

2. What have multicentre registries across the world taught us about the disease features of systemic sclerosis?

Author

Proudman S. M., Huq M., Stevens W., Wilson M. E., Sahhar J., Baron M., Hudson M., Pope J., Allanore Y., Distler O., Kowal-Bielecka O., Matucci-Cerinic M., H. L. Low A., Teng G. G., Law W. G., Santosa A., Nikpour M., Hill C., Lester S., Nash P., Ngian G. -S., Proudman S., Rischmueller M., Roddy J., Strickland G., Thakkar V., Walker J., Zochling J., Markland J., Robinson D., Jones N., Khalidi N., Docherty P., Kaminska E., Masetto A., Sutton E., Mathieu J. -P., Ligier S., Grodzicky T., LeClercq S., Thorne C., Gyger G., Smith D., Fortin P. R., Larche M., Abu-Hakima M., Rodriguez-Reyna T. S., Cabral A. R., Fritzler M., Avouac J., Walker U. A., Guiducci S., Riemekasten G., Air P., Hachulla E., Valentini G., Carreira P. E., Cozzi F., Gurman A. B., Braun-Moscovici Y., Damjanov N., Ananieva L. P., Scorza R., Jimenez S., Busquets J., Li M., Muller-Ladner U., Maurer B., Tyndall A., Lapadula G., Iannone F., Becvar R., Sierakowsky S., Cutolo M., Sulli A., Cuomo G., Vettori S., Rednic S., Nicoara I., Vlachoyiannopoulos P., Montecucco C., Caporali R., Novak S., Czirjak L., Varju C., Chizzolini C., Kucharz E. J., Kotulska A., Kopec-Medrek M., Widuchowska M., Rozman B., Mallia C., Coleiro B., Gabrielli A., Farge D., Hij A., Hesselstrand R., Scheja A., Wollheim F., Martinovic D., Govoni M., Lo Monaco A., Hunzelmann N., Pellerito R., Bambara L. M., Caramaschi P., Black C., Denton C., Henes J., Santamaria V. O., Heitmann S., Krasowska D., Seidel M., Oleszowsky M., Burkhardt H., Himsel A., Salvador M. J., Stamenkovic B., Stankovic A., Tikly M., Starovoytova M. N., Engelhart M., Strauss G., Nielsen H., Damgaard K., Szucs G., Mendoza A. Z., de la Puente Buijdos C., Giraldo W. A. S., Midtvedt O., Garen T., Launay D., Valesini G., Riccieri V., Ionescu R. M., Opris D., Groseanu L., Wigley F. M., Mihai C. M., Cornateanu R. S., Ionitescu R., Gherghe A. M., Gorga M., Dobrota R., Bojinca M., Schett G., Distler J. H., Meroni P., Zeni S., Mouthon L., De Keyser F., Smith V., Cantatore F. P., Corrado A., Ullman S., Iversen L., Pozzi M. R., Eyerich K., Hein R., Knott E., Szechinski J., Wiland P., Szmyrka-Kaczmarek M., Sokolik R., Morgiel E., Krummel-Lorenz B., Saar P., Aringer M., Gunther C., Anic B., Baresic M., Mayer M., Radominski S. C., de Souza Muller C., Azevedo V. F., Agachi S., Groppa L., Chiaburu L., Russu E., Zenone T., Stebbings S., Highton J., Stamp L., Chapman P., O'Donnell J., Solanki K., Doube A., Veale D., O'Rourke M., Loyo E., Rosato E., Pisarri S., Tanaseanu C. -M., Popescu M., Dumitrascu A., Tiglea I., Chirieac R., Ancuta C., Furst D. E., Kafaja S., Garcia de la Pena Lefebvre P., Rubio S. R., Exposito M. V., Sibilia J., Chatelus E., Gottenberg J. E., Chifflot H., Litinsky I., Venalis A., Butrimiene I., Venalis P., Rugiene R., Karpec D., Kerzberg E., Montoya F., Cosentino V., Low A. H. L., Teng G., Chan G., Lim A. Y. N., Ng S. C., Proudman, S. M., Huq, M., Stevens, W., Wilson, M. E., Sahhar, J., Baron, M., Hudson, M., Pope, J., Allanore, Y., Distler, O., Kowal-Bielecka, O., Matucci-Cerinic, M., H. L. Low, A., Teng, G. G., Law, W. G., Santosa, A., Nikpour, M., Hill, C., Lester, S., Nash, P., Ngian, G. -S., Proudman, S., Rischmueller, M., Roddy, J., Strickland, G., Thakkar, V., Walker, J., Zochling, J., Markland, J., Robinson, D., Jones, N., Khalidi, N., Docherty, P., Kaminska, E., Masetto, A., Sutton, E., Mathieu, J. -P., Ligier, S., Grodzicky, T., Leclercq, S., Thorne, C., Gyger, G., Smith, D., Fortin, P. R., Larche, M., Abu-Hakima, M., Rodriguez-Reyna, T. S., Cabral, A. R., Fritzler, M., Avouac, J., Walker, U. A., Guiducci, S., Riemekasten, G., Air, P., Hachulla, E., Valentini, G., Carreira, P. E., Cozzi, F., Gurman, A. B., Braun-Moscovici, Y., Damjanov, N., Ananieva, L. P., Scorza, R., Jimenez, S., Busquets, J., Li, M., Muller-Ladner, U., Maurer, B., Tyndall, A., Lapadula, G., Iannone, F., Becvar, R., Sierakowsky, S., Cutolo, M., Sulli, A., Cuomo, G., Vettori, S., Rednic, S., Nicoara, I., Vlachoyiannopoulos, P., Montecucco, C., Caporali, R., Novak, S., Czirjak, L., Varju, C., Chizzolini, C., Kucharz, E. J., Kotulska, A., Kopec-Medrek, M., Widuchowska, M., Rozman, B., Mallia, C., Coleiro, B., Gabrielli, A., Farge, D., Hij, A., Hesselstrand, R., Scheja, A., Wollheim, F., Martinovic, D., Govoni, M., Lo Monaco, A., Hunzelmann, N., Pellerito, R., Bambara, L. M., Caramaschi, P., Black, C., Denton, C., Henes, J., Santamaria, V. O., Heitmann, S., Krasowska, D., Seidel, M., Oleszowsky, M., Burkhardt, H., Himsel, A., Salvador, M. J., Stamenkovic, B., Stankovic, A., Tikly, M., Starovoytova, M. N., Engelhart, M., Strauss, G., Nielsen, H., Damgaard, K., Szucs, G., Mendoza, A. Z., de la Puente Buijdos, C., Giraldo, W. A. S., Midtvedt, O., Garen, T., Launay, D., Valesini, G., Riccieri, V., Ionescu, R. M., Opris, D., Groseanu, L., Wigley, F. M., Mihai, C. M., Cornateanu, R. S., Ionitescu, R., Gherghe, A. M., Gorga, M., Dobrota, R., Bojinca, M., Schett, G., Distler, J. H., Meroni, P., Zeni, S., Mouthon, L., De Keyser, F., Smith, V., Cantatore, F. P., Corrado, A., Ullman, S., Iversen, L., Pozzi, M. R., Eyerich, K., Hein, R., Knott, E., Szechinski, J., Wiland, P., Szmyrka-Kaczmarek, M., Sokolik, R., Morgiel, E., Krummel-Lorenz, B., Saar, P., Aringer, M., Gunther, C., Anic, B., Baresic, M., Mayer, M., Radominski, S. C., de Souza Muller, C., Azevedo, V. F., Agachi, S., Groppa, L., Chiaburu, L., Russu, E., Zenone, T., Stebbings, S., Highton, J., Stamp, L., Chapman, P., O'Donnell, J., Solanki, K., Doube, A., Veale, D., O'Rourke, M., Loyo, E., Rosato, E., Pisarri, S., Tanaseanu, C. -M., Popescu, M., Dumitrascu, A., Tiglea, I., Chirieac, R., Ancuta, C., Furst, D. E., Kafaja, S., Garcia de la Pena Lefebvre, P., Rubio, S. R., Exposito, M. V., Sibilia, J., Chatelus, E., Gottenberg, J. E., Chifflot, H., Litinsky, I., Venalis, A., Butrimiene, I., Venalis, P., Rugiene, R., Karpec, D., Kerzberg, E., Montoya, F., Cosentino, V., Low, A. H. L., Teng, G., Chan, G., Lim, A. Y. N., and Ng, S. C.

Subjects
0301 basic medicine, medicine.medical_specialty, Pediatrics, Survival, Immunology, Disease, Scleroderma, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Internal medicine, Immunology and Allergy, Medicine, Multicentre registrie, 030203 arthritis & rheumatology, Clinical features, Cohort study, Multicentre registries, Systemic sclerosis, business.industry, Interstitial lung disease, Autoantibody, Clinical features, medicine.disease, 030104 developmental biology, Clinical feature, Cohort, business, Cohort study, Rheumatism
Abstract

Introduction The aim of this study is to compare the clinical features, mortality and causes of death of systemic sclerosis (SSc) patients in four large multicentre registries. Methods Patients seen at least once in the Australian Scleroderma Cohort Study (ASCS) (n = 1714), the Canadian Scleroderma Research Group (CSRG) (n = 1628), the European League Against Rheumatism Scleroderma Trials and Research (EUSTAR) Network (n = 13,996) and the Systemic Sclerosis Cohort in Singapore (SCORE) (n = 500) before August 2016 were included. Clinical manifestations and survival in cohorts and disease subtypes were compared. Results Among 17,838 SSc patients, most were female (86.1%), Caucasian (84.6%) and had the limited cutaneous subtype (lcSSc) (65.0%). The anti-centromere autoantibody was the most prevalent (37.6%). More patients in SCORE had the diffuse subtype (dcSSc) (49.3%) and Scl-70 autoantibody (38.8%) (pConclusions This meta-cohort of SSc patients, the largest reported to date, provides insights into the impact of race and sex on disease manifestations and survival and confirms the early mortality in this disease.

Published
2017

3. What have multicentre registries across the world taught us about the disease features of systemic sclerosis?.

Author

Iannone F., Schett G., Distler J.H., Meroni P., Zeni S., Mouthon L., De Keyser F., Smith V., Cantatore F.P., Corrado A., Ullman S., Iversen L., Pozzi M.R., Eyerich K., Hein R., Knott E., Szechinski J., Wiland P., Szmyrka-Kaczmarek M., Sokolik R., Morgiel E., Krummel-Lorenz B., Saar P., Aringer M., Gunther C., Anic B., Baresic M., Mayer M., Radominski S.C., de Souza Muller C., Azevedo V.F., Agachi S., Groppa L., Chiaburu L., Russu E., Zenone T., Stebbings S., Highton J., Stamp L., Chapman P., O'Donnell J., Solanki K., Doube A., Veale D., O'Rourke M., Loyo E., Rosato E., Pisarri S., Tanaseanu C.-M., Popescu M., Dumitrascu A., Tiglea I., Chirieac R., Ancuta C., Furst D.E., Kafaja S., Garcia de la Pena Lefebvre P., Rubio S.R., Exposito M.V., Sibilia J., Chatelus E., Gottenberg J.E., Chifflot H., Litinsky I., Venalis A., Butrimiene I., Venalis P., Rugiene R., Karpec D., Kerzberg E., Montoya F., Cosentino V., Low A.H.L., Teng G., Chan G., Lim A.Y.N., Ng S.C., Kowal-Bielecka O., Proudman S.M., Huq M., Stevens W., Wilson M.E., Sahhar J., Baron M., Hudson M., Allanore Y., Distler O., Bielecka O.K., Matucci-Cerinic M., H.L. Low A., Teng G.G., Law W.G., Santosa A., Nikpour M., Hill C., Lester S., Nash P., Ngian G.-S., Proudman S., Rischmueller M., Roddy J., Strickland G., Thakkar V., Walker J., Zochling J., Pope J., Markland J., Robinson D., Jones N., Khalidi N., Docherty P., Kaminska E., Masetto A., Sutton E., Mathieu J.-P., Ligier S., Grodzicky T., LeClercq S., Thorne C., Gyger G., Smith D., Fortin P.R., Larche M., Abu-Hakima M., Rodriguez-Reyna T.S., Cabral A.R., Fritzler M., Avouac J., Walker U.A., Guiducci S., Riemekasten G., Air P., Hachulla E., Valentini G., Carreira P.E., Cozzi F., Gurman A.B., Braun-Moscovici Y., Damjanov N., Ananieva L.P., Scorza R., Jimenez S., Busquets J., Li M., Muller-Ladner U., Maurer B., Tyndall A., Lapadula G., Becvar R., Sierakowsky S., Cutolo M., Sulli A., Cuomo G., Vettori S., Rednic S., Nicoara I., Vlachoyiannopoulos P., Montecucco C., Caporali R., Novak S., Czirjak L., Varju C., Chizzolini C., Kucharz E.J., Kotulska A., Kopec-Medrek M., Widuchowska M., Rozman B., Mallia C., Coleiro B., Gabrielli A., Farge D., Hij A., Hesselstrand R., Scheja A., Wollheim F., Martinovic D., Govoni M., Lo Monaco A., Hunzelmann N., Pellerito R., Bambara L.M., Caramaschi P., Black C., Denton C., Henes J., Santamaria V.O., Heitmann S., Krasowska D., Seidel M., Oleszowsky M., Burkhardt H., Himsel A., Salvador M.J., Stamenkovic B., Stankovic A., Tikly M., Starovoytova M.N., Engelhart M., Strauss G., Nielsen H., Damgaard K., Szucs G., Mendoza A.Z., de la Puente Buijdos C., Giraldo W.A.S., Midtvedt O., Garen T., Launay D., Valesini G., Riccieri V., Ionescu R.M., Opris D., Groseanu L., Wigley F.M., Mihai C.M., Cornateanu R.S., Ionitescu R., Gherghe A.M., Gorga M., Dobrota R., Bojinca M., Iannone F., Schett G., Distler J.H., Meroni P., Zeni S., Mouthon L., De Keyser F., Smith V., Cantatore F.P., Corrado A., Ullman S., Iversen L., Pozzi M.R., Eyerich K., Hein R., Knott E., Szechinski J., Wiland P., Szmyrka-Kaczmarek M., Sokolik R., Morgiel E., Krummel-Lorenz B., Saar P., Aringer M., Gunther C., Anic B., Baresic M., Mayer M., Radominski S.C., de Souza Muller C., Azevedo V.F., Agachi S., Groppa L., Chiaburu L., Russu E., Zenone T., Stebbings S., Highton J., Stamp L., Chapman P., O'Donnell J., Solanki K., Doube A., Veale D., O'Rourke M., Loyo E., Rosato E., Pisarri S., Tanaseanu C.-M., Popescu M., Dumitrascu A., Tiglea I., Chirieac R., Ancuta C., Furst D.E., Kafaja S., Garcia de la Pena Lefebvre P., Rubio S.R., Exposito M.V., Sibilia J., Chatelus E., Gottenberg J.E., Chifflot H., Litinsky I., Venalis A., Butrimiene I., Venalis P., Rugiene R., Karpec D., Kerzberg E., Montoya F., Cosentino V., Low A.H.L., Teng G., Chan G., Lim A.Y.N., Ng S.C., Kowal-Bielecka O., Proudman S.M., Huq M., Stevens W., Wilson M.E., Sahhar J., Baron M., Hudson M., Allanore Y., Distler O., Bielecka O.K., Matucci-Cerinic M., H.L. Low A., Teng G.G., Law W.G., Santosa A., Nikpour M., Hill C., Lester S., Nash P., Ngian G.-S., Proudman S., Rischmueller M., Roddy J., Strickland G., Thakkar V., Walker J., Zochling J., Pope J., Markland J., Robinson D., Jones N., Khalidi N., Docherty P., Kaminska E., Masetto A., Sutton E., Mathieu J.-P., Ligier S., Grodzicky T., LeClercq S., Thorne C., Gyger G., Smith D., Fortin P.R., Larche M., Abu-Hakima M., Rodriguez-Reyna T.S., Cabral A.R., Fritzler M., Avouac J., Walker U.A., Guiducci S., Riemekasten G., Air P., Hachulla E., Valentini G., Carreira P.E., Cozzi F., Gurman A.B., Braun-Moscovici Y., Damjanov N., Ananieva L.P., Scorza R., Jimenez S., Busquets J., Li M., Muller-Ladner U., Maurer B., Tyndall A., Lapadula G., Becvar R., Sierakowsky S., Cutolo M., Sulli A., Cuomo G., Vettori S., Rednic S., Nicoara I., Vlachoyiannopoulos P., Montecucco C., Caporali R., Novak S., Czirjak L., Varju C., Chizzolini C., Kucharz E.J., Kotulska A., Kopec-Medrek M., Widuchowska M., Rozman B., Mallia C., Coleiro B., Gabrielli A., Farge D., Hij A., Hesselstrand R., Scheja A., Wollheim F., Martinovic D., Govoni M., Lo Monaco A., Hunzelmann N., Pellerito R., Bambara L.M., Caramaschi P., Black C., Denton C., Henes J., Santamaria V.O., Heitmann S., Krasowska D., Seidel M., Oleszowsky M., Burkhardt H., Himsel A., Salvador M.J., Stamenkovic B., Stankovic A., Tikly M., Starovoytova M.N., Engelhart M., Strauss G., Nielsen H., Damgaard K., Szucs G., Mendoza A.Z., de la Puente Buijdos C., Giraldo W.A.S., Midtvedt O., Garen T., Launay D., Valesini G., Riccieri V., Ionescu R.M., Opris D., Groseanu L., Wigley F.M., Mihai C.M., Cornateanu R.S., Ionitescu R., Gherghe A.M., Gorga M., Dobrota R., and Bojinca M.

Abstract

Introduction: The aim of this study is to compare the clinical features, mortality and causes of death of systemic sclerosis (SSc) patients in four large multicentre registries. Method(s): Patients seen at least once in the Australian Scleroderma Cohort Study (ASCS) (n = 1714), the Canadian Scleroderma Research Group (CSRG) (n = 1628), the European League Against Rheumatism Scleroderma Trials and Research (EUSTAR) Network (n = 13,996) and the Systemic Sclerosis Cohort in Singapore (SCORE) (n = 500) before August 2016 were included. Clinical manifestations and survival in cohorts and disease subtypes were compared. Result(s): Among 17,838 SSc patients, most were female (86.1%), Caucasian (84.6%) and had the limited cutaneous subtype (lcSSc) (65.0%). The anti-centromere autoantibody was the most prevalent (37.6%). More patients in SCORE had the diffuse subtype (dcSSc) (49.3%) and Scl-70 autoantibody (38.8%) (p<0.001). Patients with dcSSc were more likely to be younger and male (p<0.001) and have shorter disease duration, more calcinosis, tendon friction rubs and synovitis (all p<0.001). Interstitial lung disease (ILD) occurred more frequently in dcSSc but prevalence of pulmonary arterial hypertension (PAH) was similar in both subtypes. More deaths occurred among SCORE patients who had the shortest median survival (p<0.001). The survival of patients with early disease, males and those with dcSSc was shorter than that of patients with prevalent disease, female gender and lcSSc, respectively. SSc-related complications accounted for more than 50% of deaths, with PAH and ILD being the most common. Conclusion(s): This meta-cohort of SSc patients, the largest reported to date, provides insights into the impact of race and sex on disease manifestations and survival and confirms the early mortality in this disease.Copyright © 2017 Wichtig International

Published
2018

4. Stratification in systemic sclerosis according to autoantibody status versus skin involvement: a study of the prospective EUSTAR cohort

Author

Muriel Elhai, Nanthara Sritharan, Marouane Boubaya, Alexandra Balbir-Gurman, Elise Siegert, Eric Hachulla, Jeska de Vries-Bouwstra, Gabriela Riemekasten, Jörg H W Distler, Edoardo Rosato, Francesco Del Galdo, Fabian A Mendoza, Daniel E Furst, Carlos de la Puente, Anna-Maria Hoffmann-Vold, Armando Gabrielli, Oliver Distler, Coralie Bloch-Queyrat, Yannick Allanore, Marco Matucci Cerinic, Ulrich Walker, Florenzo Iannone, Suzana Jordan, Radim Becvar, Otylia Kowal Bielecka, Maurizio Cutolo, Giovanna Cuomo, Claudia Kedor, Simona Rednic, Jérome Avouac, P. Vlachoyiannopoulos, C. Montecucco, Jiri Stork, Murat Inanc, Patricia E. Carreira, Srdan Novak, László Czirják, Michele Iudici, Eugene J. Kucharz, Elisabetta Zanatta, Katja Perdan-Pirkmajer, Bernard Coleiro, Gianluca Moroncini, Dominique Farge Bancel, Paolo Airò, Roger Hesselstrand, Mislav Radic, Yolanda Braun-Moscovici, Andrea Lo Monaco, Nicolas Hunzelmann, Raffaele Pellerito, Alessandro Giollo, Jadranka Morovic-Vergles, Christopher Denton, Madelon Vonk, Nemanja Damjanov, Jörg Henes, Vera Ortiz Santamaria, Stefan Heitmann, Dorota Krasowska, Paul Hasler, Michaela Kohm, Ivan Foeldvari, Gianluigi Bajocchi, Maria João Salvador, Bojana Stamenkovic, Carlo Francesco Selmi, Mohammed Tikly, Lidia P. Ananieva, Ariane Herrick, Ulf Müller-Ladner, Raffaele De Palma, Merete Engelhart, Gabriela Szücs, Cristina Sobrino Grande, Øyvind Midtvedt, David Launay, Valeria Riccieri, Ruxandra Maria Ionescu, Ami Sha, Ana Maria Gheorghiu, Cord Sunderkötter, Francesca Ingegnoli, Luc Mouthon, Vanessa Smith, Francesco Paolo Cantatore, Susanne Ullman, Carlos Alberto von Mühlen, Maria Rosa Pozzi, Kilian Eyerich, Piotr Wiland, Marie Vanthuyne, Juan Jose Alegre-Sancho, Kristine Herrmann, Ellen De Langhe, Branimir Anic, Maria Üprus, Sule Yavuz, Brigitte Granel, Carolina de Souza Müller, Joanna Busquets, Svetlana Agachi, Simon Stebbings, D'Alessandro Mathieu, Percival D. Sampaio-Barros, Lisa Stamp, Kamal Solanki, Douglas Veale, Esthela Loyo, Mengtao Li, Walid Ahmed Abdel Atty Mohamed, Antonietta Gigante, Fahrettin Oksel, Cristina-Mihaela Tanaseanu, Rosario Foti, Codrina Ancuta, Britta Maurer, Jacob van Laar, Cristiane Kayser, Nihal Fathi, Paloma García de la Peña Lefebvre, Jean Sibilia, Ira Litinsky, Giuseppina Abignano, Goda Seskute, Lesley Ann Saketkoo, Eduardo Kerzberg, Washington Bianchi, Ivan Castellví, Massimiliano Limonta, Doron Rimar, Maura Couto, François Spertini, Antonella Marcoccia, Sarah Kahl, Ivien M. Hsu, Thierry Martin, Sergey Moiseev, Lorinda S. Chung, Tim Schmeiser, Dominik Majewski, Zbigniew Zdrojewski, Julia Martínez-Barrio, Vera Bernardino, Sabine Sommerlatte, Yair Levy, Elena Rezus, Omer Nuri Pamuk, Piercarlo Sarzi Puttini, Hadi Poormoghim, Ina Kötter, Francis Gaches, Laura Belloli, Petros Sfikakis, Juliana Markus, Gary R Feldman, Ana-Maria Ramazan, H.U. Scherer, Marie-Elise Truchetet, Alain Lescoat, Lorenzo Dagna, J.M. van Laar, Lidia Rudnicka, Susana Oliveira, Fabiola Atzeni, Masataka Kuwana, Arsene Mekinian, Mickaël Martin, Yoshiya Tanaka, Elhai, M., Sritharan, N., Boubaya, M., Balbir-Gurman, A., Siegert, E., Hachulla, E., de Vries-Bouwstra, J., Riemekasten, G., Distler, J. H. W., Rosato, E., Del Galdo, F., Mendoza, F. A., Furst, D. E., de la Puente, C., Hoffmann-Vold, A. -M., Gabrielli, A., Distler, O., Bloch-Queyrat, C., Allanore, Y., Matucci Cerinic, M., Walker, U., Iannone, F., Jordan, S., Becvar, R., Kowal Bielecka, O., Cutolo, M., Cuomo, G., Kedor, C., Rednic, S., Avouac, J., Vlachoyiannopoulos, P., Montecucco, C., Stork, J., Inanc, M., Carreira, P. E., Novak, S., Czirjak, L., Iudici, M., Kucharz, E. J., Zanatta, E., Perdan-Pirkmajer, K., Coleiro, B., Moroncini, G., Farge Bancel, D., Airo, P., Hesselstrand, R., Radic, M., Braun-Moscovici, Y., Lo Monaco, A., Hunzelmann, N., Pellerito, R., Giollo, A., Morovic-Vergles, J., Denton, C., Vonk, M., Damjanov, N., Henes, J., Ortiz Santamaria, V., Heitmann, S., Krasowska, D., Hasler, P., Kohm, M., Foeldvari, I., Bajocchi, G., Salvador, M. J., Stamenkovic, B., Selmi, C. F., Tikly, M., Ananieva, L. P., Herrick, A., Muller-Ladner, U., De Palma, R., Engelhart, M., Szucs, G., Sobrino Grande, C., Midtvedt, O., Launay, D., Riccieri, V., Ionescu, R. M., Sha, A., Gheorghiu, A. M., Sunderkotter, C., Ingegnoli, F., Mouthon, L., Smith, V., Cantatore, F. P., Ullman, S., Alberto von Muhlen, C., Pozzi, M. R., Eyerich, K., Wiland, P., Vanthuyne, M., Alegre-Sancho, J. J., Herrmann, K., De Langhe, E., Anic, B., Uprus, M., Yavuz, S., Granel, B., de Souza Muller, C., Busquets, J., Agachi, S., Stebbings, S., Mathieu, D. A., Sampaio-Barros, P. D., Stamp, L., Solanki, K., Veale, D., Loyo, E., Li, M., Abdel Atty Mohamed, W. A., Gigante, A., Oksel, F., Tanaseanu, C. -M., Foti, R., Ancuta, C., Maurer, B., van Laar, J., Kayser, C., Fathi, N., Garcia de la Pena Lefebvre, P., Sibilia, J., Litinsky, I., Abignano, G., Seskute, G., Saketkoo, L. A., Kerzberg, E., Bianchi, W., Castellvi, I., Limonta, M., Rimar, D., Couto, M., Spertini, F., Marcoccia, A., Kahl, S., Hsu, I. M., Martin, T., Moiseev, S., Chung, L. S., Schmeiser, T., Majewski, D., Zdrojewski, Z., Martinez-Barrio, J., Bernardino, V., Sommerlatte, S., Levy, Y., Rezus, E., Nuri Pamuk, O., Sarzi Puttini, P., Poormoghim, H., Kotter, I., Gaches, F., Belloli, L., Sfikakis, P., Markus, J., Feldman, G. R., Ramazan, A. -M., Scherer, H. U., Truchetet, M. -E., Lescoat, A., Dagna, L., van Laar, J. M., Rudnicka, L., Oliveira, S., Atzeni, F., Kuwana, M., Mekinian, A., Martin, M., and Tanaka, Y.

Subjects
Rheumatology, Immunology, Immunology and Allergy
Abstract

Background: The current subclassification of systemic sclerosis into cutaneous subtypes does not fully capture the heterogeneity of the disease. We aimed to compare the performances of stratification into LeRoy's cutaneous subtypes versus stratification by autoantibody status in systemic sclerosis. Methods: For this cohort study, we assessed people with systemic sclerosis in the multicentre international European Scleroderma Trials and Research (EUSTAR) database. Individuals positive for systemic-sclerosis autoantibodies of two specificities were excluded, and remaining individuals were classified by cutaneous subtype, according to their systemic sclerosis-specific autoantibodies, or both. We assessed the performance of each model to predict overall survival, progression-free survival, disease progression, and different organ involvement. The three models were compared by use of the area under the curve (AUC) of the receiver operating characteristic and the net reclassification improvement (NRI). Missing data were imputed. Findings: We assessed the database on July 26, 2019. Of 16 939 patients assessed for eligibility, 10 711 patients were included: 1647 (15·4%) of 10 709 were male, 9062 (84·6%) were female, mean age was 54·4 (SD 13·8) years, and mean disease duration was 7·9 (SD 8·2) years. Information regarding cutaneous subtype was available for 10 176 participants and antibody data were available for 9643 participants. In the prognostic analysis, there was no difference in AUC for overall survival (0·82, 95% CI 0·81–0·84 for cutaneous only vs 0·84, 0·82–0·85 for antibody only vs 0·84, 0·83–0·86 for combined) or for progression-free survival (0·70, 0·69–0·71 vs 0·71, 0·70–0·72 vs 0·71, 0·70–0·72). However, at 4 years the NRI showed substantial improvement for the antibody-only model compared with the cutaneous-only model in prediction of overall survival (0·57, 0·46–0·71 for antibody only vs 0·29, 0·19–0·39 for cutaneous only) and disease progression (0·36, 0·29–0·46 vs 0·21, 0·14–0·28). The antibody-only model did better than the cutaneous-only model in predicting renal crisis (AUC 0·72, 0·70–0·74 for antibody only vs 0·66, 0·64–0·69 for cutaneous only) and lung fibrosis leading to restrictive lung function (AUC 0·76, 0·75–0·77 vs 0·71, 0·70–0·72). The combined model improved the prediction of digital ulcers and elevated systolic pulmonary artery pressure, but did poorly for cardiac involvement. Interpretation: The autoantibody-only model outperforms cutaneous-only subsetting for risk stratifying people with systemic sclerosis in the EUSTAR cohort. Physicians should be aware of these findings at the time of decision making for patient management. Funding: World Scleroderma Foundation.

Published
2022

5. Progressive skin fibrosis is associated with a decline in lung function and worse survival in patients with diffuse cutaneous systemic sclerosis in the european scleroderma trials and research (eustar) cohort

Author

Wu, Wanlong, Jordan, Suzana, Graf, Nicole, Pena, Janethe de Oliveira, Curram, John, Allanore, Yannick, Matucci-Cerinic, Marco, Pope, Janet E., Denton, Christopher P., Khanna, Dinesh, Distler, Oliver, Guiducci, Serena, Walker, Ulrich, Jaeger, Veronika, Bannert, Bettina, Lapadula, Giovanni, Becvarare, Radim, Cutolo, Maurizio, Valentini, Gabriele, Siegert, Elise, Rednic, Simona, Montecucco, C., Carreira, Patricia E., Novak, Srdan, Czirjak, Laszlo, Varju, Cecilia, Chizzolini, Carlo, Allai, Daniela, Kucharz, Eugene J., Cozzi, Franco, Rozman, Blaz, Mallia, Carmel, Gabrielli, Armando, Bancel, Dominique Farge, Airo, Paolo, Hesselstrand, Roger, Martinovic, Duska, Balbir-Gurman, Alexandra, Braun-Moscovici, Yolanda, Hunzelmann, Nicolas, Pellerito, Raffaele, Caramaschi, Paola, Black, Carol, Damjanov, Nemanja, Henes, Joerg, Ortiz Santamaria, Vera, Heitmann, Stefan, Seidel, Matthias, Pereira Da Silva, Jose Antonio, Stamenkovic, Bojana, Selmi, Carlo Francesco, Tikly, Mohammed, Denisov, Lev N., Mueller-Ladner, Ulf, Engelhart, Merete, Hachulla, Eric, Riccieri, Valeria, Ionescu, Ruxandra Maria, Mihai, Carina, Sunderkoetter, Cord, Kuhn, Annegret, Schett, Georg, Distler, Joerg, Meroni, Pierluigi, Ingegnoli, Francesca, Mouthon, Luc, De Keyser, Filip, Smith, Vanessa, Cantatore, Francesco Paolo, Corrado, Ada, Ullman, Susanne, Iversen, Line, Pozzi, Maria Rosa, Eyerich, Kilian, Hein, Ruediger, Knott, Elisabeth, Wiland, Piotr, Szmyrka-Kaczmarek, Magdalena, Sokolik, Renata, Morgiel, Ewa, Madej, Marta, Jose Alegre-Sancho, Juan, Krummel-Lorenz, Brigitte, Saar, Petra, Aringer, Martin, Guenther, Claudia, Anne, Erler, Westhovens, Rene, De Langhe, Ellen, Lenaerts, Jan, Anic, Branimir, Baresic, Marko, Mayer, Miroslav, Uprus, Maria, Otsa, Kati, Yavuz, Sule, Radominski, Sebastiao Cezar, Mueller, Carolina de Souza, Azevedo, Valderilio Feijo, Popa, Sergei, Zenone, Thierry, Stebbings, Simon, Highton, John, Mathieu, Alessandro, Vacca, Alessandra, Stamp, Lisa, Chapman, Peter, O'Donnell, John, Solanki, Kamal, Doube, Alan, Veale, Douglas, O'Rourke, Marie, Loyo, Esthela, Li, Mengtao, Rosato, Edoardo, Amoroso, Antonio, Gigante, Antonietta, Oksel, Fahrettin, Yargucu, Figen, Tanaseanu, Cristina-Mihaela, Popescu, Monica, Dumitrascu, Alina, Tiglea, Isabela, Foti, Rosario, Visalli, Elisa, Benenati, Alessia, Amato, Giorgio, Ancuta, Codrina, Chirieac, Rodica, Villiger, Peter, Adler, Sabine, Dan, Diana, de la Pena Lefebvre, Paloma Garcia, Rodriguez Rubio, Silvia, Valero Exposito, Marta, Sibilia, Jean, Chatelus, Emmanuel, Gottenberg, Jacques Eric, Chifflot, Helene, Litinsky, Ira, Del Galdo, Francesco, Venalis, Algirdas, Saketkoo, Lesley Ann, Lasky, Joseph A., Kerzberg, Eduardo, Montoya, Fabiana, Cosentino, Vanesa, Limonta, Massimiliano, Brucato, Antonio Luca, Lupi, Elide, Spertini, Francois, Ribi, Camillo, Buss, Guillaume, Martin, Thierry, Guffroy, Aurelien, Poindron, Vincent, Chung, Lori, Schmeiser, Tim, Zebryk, Pawel, Riso, Nuno, Riemekasten, Gabriela, Rezus, Elena, Puttini, Piercarlo Sarzi, Wu, W., Jordan, S., Graf, N., de Oliveira Pena, J., Curram, J., Allanore, Y., Matucci-Cerinic, M., Pope, J. E., Denton, C. P., Khanna, D., Distler, O., Guiducci, S., Walker, U., Jaeger, V., Bannert, B., Lapadula, G., Becvarare, R., Cutolo, M., Valentini, G., Siegert, E., Rednic, S., Montecucco, C., Carreira, P. E., Novak, S., Czirjak, L., Varju, C., Chizzolini, C., Allai, D., Kucharz, E. J., Cozzi, F., Rozman, B., Mallia, C., Gabrielli, A., Bancel, D. F., Airo, P., Hesselstrand, R., Martinovic, D., Balbir-Gurman, A., Braun-Moscovici, Y., Hunzelmann, N., Pellerito, R., Caramaschi, P., Black, C., Damjanov, N., Henes, J., Santamaria, V. O., Heitmann, S., Seidel, M., Pereira Da Silva, J. A., Stamenkovic, B., Selmi, C. F., Tikly, M., Denisov, L. N., Muller-Ladner, U., Engelhart, M., Hachulla, E., Riccieri, V., Ionescu, R. M., Mihai, C., Sunderkotter, C., Kuhn, A., Schett, G., Distler, J., Meroni, P., Ingegnoli, F., Mouthon, L., De Keyser, F., Smith, V., Cantatore, F. P., Corrado, A., Ullman, S., Iversen, L., Pozzi, M. R., Eyerich, K., Hein, R., Knott, E., Wiland, P., Szmyrka-Kaczmarek, M., Sokolik, R., Morgiel, E., Madej, M., Alegre-Sancho, J. J., Krummel-Lorenz, B., Saar, P., Aringer, M., Gunther, C., Anne, E., Westhovens, R., De Langhe, E., Lenaerts, J., Anic, B., Baresic, M., Mayer, M., Uprus, M., Otsa, K., Yavuz, S., Radominski, S. C., de Souza Muller, C., Azevedo, V. F., Popa, S., Zenone, T., Stebbings, S., Highton, J., Mathieu, A., Vacca, A., Stamp, L., Chapman, P., O'Donnell, J., Solanki, K., Doube, A., Veale, D., O'Rourke, M., Loyo, E., Li, M., Rosato, E., Amoroso, A., Gigante, A., Oksel, F., Yargucu, F., Tanaseanu, C. -M., Popescu, M., Dumitrascu, A., Tiglea, I., Foti, R., Visalli, E., Benenati, A., Amato, G., Ancuta, C., Chirieac, R., Villiger, P., Adler, S., Dan, D., de la Pena Lefebvre, P. G., Rubio, S. R., Exposito, M. V., Sibilia, J., Chatelus, E., Gottenberg, J. E., Chifflot, H., Litinsky, I., Del Galdo, F., Venalis, A., Saketkoo, L. A., Lasky, J. A., Kerzberg, E., Montoya, F., Cosentino, V., Limonta, M., Brucato, A. L., Lupi, E., Spertini, F., Ribi, C., Buss, G., Martin, T., Guffroy, A., Poindron, V., Chung, L., Schmeiser, T., Zebryk, P., Riso, N., Riemekasten, G., Rezus, E., Sarzi Puttini, P., Ege Üniversitesi, Chizzolini, Carlo, Allali, Danièle, University of Zurich, and Distler, Oliver

Subjects
INVOLVEMENT, Male, BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences, diffuse, Time Factors, Databases, Factual, Skin Diseases/etiology/mortality/physiopathology, PREDICTION, Fibrosi, 2745 Rheumatology, Diffuse/complications/mortality/pathology, Immunology, General Biochemistry, Genetics and Molecular Biology, Immunology and Allergy, Rheumatology, Kaplan-Meier Estimate, ddc:616.07, Severity of Illness Index, Scleroderma, Cohort Studies, PROGNOSTIC-FACTORS, Fibrosis, Medicine and Health Sciences, scleroderma, Lung, Skin, integumentary system, progressive skin fibrosis, Lung function decline, BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti, 10051 Rheumatology Clinic and Institute of Physical Medicine, DEATH, Middle Aged, ddc, Europe, VARIABILITY, factual, Cohort, Visceral organ progression, 2723 Immunology and Allergy, Disease Progression, Female, Survival Analysi, Life Sciences & Biomedicine, Cohort study, Human, Adult, Skin/pathology, medicine.medical_specialty, databases, All-cause death, risk analysis, diffuse cutaneous systemic sclerosis, 610 Medicine & health, IMPROVEMENT, Systemic Sclerosis, Skin Diseases, THICKNESS SCORE, VALIDATION, Databases, FEV1/FVC ratio, 1300 General Biochemistry, Genetics and Molecular Biology, Internal medicine, medicine, Humans, Factual, Survival analysis, 2403 Immunology, Science & Technology, Proportional hazards model, business.industry, Surrogate endpoint, MORTALITY, Skin Disease, fibrosis, Progressive skin fibrosi, Lung/physiopathology, biomarkers, Diffuse cutaneous systemic sclerosi, medicine.disease, Survival Analysis, all-cause death, lung function decline, visceral organ progression, adult, cohort studies, databases, factual, disease progression, female, humans, Scleroderma, Diffuse, Cohort Studie, business
Abstract

PubMed: 30852552, Objectives To determine whether progressive skin fibrosis is associated with visceral organ progression and mortality during follow-up in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods We evaluated patients from the European Scleroderma Trials and Research database with dcSSc, baseline modified Rodnan skin score (mRSS) ?7, valid mRSS at 12±3 months after baseline and ?1 annual follow-up visit. Progressive skin fibrosis was defined as an increase in mRSS >5 and ?25% from baseline to 12±3 months. Outcomes were pulmonary, cardiovascular and renal progression, and all-cause death. Associations between skin progression and outcomes were evaluated by Kaplan-Meier survival analysis and multivariable Cox regression. Results Of 1021 included patients, 78 (7.6%) had progressive skin fibrosis (skin progressors). Median follow-up was 3.4 years. Survival analyses indicated that skin progressors had a significantly higher probability of FVC decline ?10% (53.6% vs 34.4%; p, Bayer Bayer, 1Department of Rheumatology, University Hospital Zurich, Zurich, switzerland 2Graf Biostatistics, Winterthur, switzerland 3Clinical Development Pulmonology, Bayer Us llC, Whippany, new Jersey, Usa 4Data science and analytics, Bayer plc, Reading, UK 5Rheumatology a Department, Paris Descartes University, inseRM U1016, sorbonne, Paris Cité, Cochin Hospital, Paris, France 6Division of Rheumatology, University of Florence, Florence, italy 7Department of Medicine, Division of Rheumatology, University of Western Ontario, st. Joseph’s Health Care, london, Western Ontario, Canada 8Department of Rheumatology, Royal Free Hospital, University College london, london, UK 9scleroderma Program, Department of internal Medicine, Division of Rheumatology, University of Michigan, ann arbor, Michigan, Usa Acknowledgements The authors thank nicole schneider for excellent administration and data entry into the eUsTaR cohort. Medical writing assistance was provided by adelphi Communications ltd (Bollington, UK), funded by Bayer aG (Berlin, Germany)., This study was supported by a grant from Bayer aG.

Published
2019

6. Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: A European Scleroderma Trials and Research (EUSTAR) analysis

Author

Becker, Mike, Graf, Nicole, Sauter, Rafael, Allanore, Yannick, Curram, John, Denton, Christopher P., Khanna, Dinesh, Matucci-Cerinic, Marco, Pena, Janethe de Oliveira, Pope, Janet E., Distler, Oliver, Guiducci, Serena, Walker, Ulrich, Jaeger, Veronika, Bannert, Bettina, Lapadula, Giovanni, Becvarare, Radim, Cutolo, Maurizio, Valentini, Gabriele, Siegert, Elise, Rednic, Simona, Montecucco, C., Carreira, Patricia E., Novak, Srdan, Czirjak, Laszlo, Varju, Cecilia, Chizzolini, Carlo, Allai, Daniela, Kucharz, Eugene J., Cozzi, Franco, Rozman, Blaz, Mallia, Carmel, Gabrielli, Armando, Bancel, Dominique Farge, Airo, Paolo, Hesselstrand, Roger, Martinovic, Duska, Balbir-Gurman, Alexandra, Braun-Moscovici, Yolanda, Hunzelmann, Nicolas, Pellerito, Raffaele, Caramaschi, Paola, Black, Carol, Damjanov, Nemanja, Henes, Joerg, Ortiz Santamaria, Vera, Heitmann, Stefan, Seidel, Matthias, Pereira Da Silva, Jose Antonio, Stamenkovic, Bojana, Selmi, Carlo Francesco, Tikly, Mohammed, Denisov, Lev N., Mueller-Ladner, Ulf, Engelhart, Merete, Hachulla, Eric, Riccieri, Valeria, Ionescu, Ruxandra Maria, Mihai, Carina, Sunderkoetter, Cord, Kuhn, Annegret, Schett, Georg, Distler, Joerg, Meroni, Pierluigi, Ingegnoli, Francesca, Mouthon, Luc, De Keyser, Filip, Smith, Vanessa, Cantatore, Francesco Paolo, Corrado, Ada, Ullman, Susanne, Iversen, Line, Pozzi, Maria Rosa, Eyerich, Kilian, Hein, Ruediger, Knott, Elisabeth, Wiland, Piotr, Szmyrka-Kaczmarek, Magdalena, Sokolik, Renata, Morgiel, Ewa, Madej, Marta, Jose Alegre-Sancho, Juan, Krummel-Lorenz, Brigitte, Saar, Petra, Aringer, Martin, Guenther, Claudia, Anne, Erler, Westhovens, Rene, De langhe, Ellen, Lenaerts, Jan, Anic, Branimir, Baresic, Marko, Mayer, Miroslav, Uprus, Maria, Otsa, Kati, Yavuz, Sule, Radominski, Sebastiao Cezar, Mueller, Carolina de Souza, Azevedo, Valderilio Feijo, Popa, Sergei, Zenone, Thierry, Stebbings, Simon, Highton, John, Mathieu, Alessandro, Vacca, Alessandra, Stamp, Lisa, Chapman, Peter, O'Donnell, John, Solanki, Kamal, Doube, Alan, Veale, Douglas, O'Rourke, Marie, Loyo, Esthela, Li, Mengtao, Rosato, Edoardo, Amoroso, Antonio, Gigante, Antonietta, Oksel, Fahrettin, Yargucu, Figen, Tanaseanu, Cristina-Mihaela, Popescu, Monica, Dumitrascu, Alina, Tiglea, Isabela, Foti, Rosario, Visalli, Elisa, Benenati, Alessia, Amato, Giorgio, Ancuta, Codrina, Chirieac, Rodica, Villiger, Peter, Adler, Sabine, Dan, Diana, de la Pena Lefebvre, Paloma Garcia, Rodriguez Rubio, Silvia, Valero Exposito, Marta, Sibilia, Jean, Chatelus, Emmanuel, Gottenberg, Jacques Eric, Chifflot, Helene, Litinsky, Ira, Del Galdo, Francesco, Venalis, Algirdas, Saketkoo, Lesley Ann, Lasky, Joseph A., Kerzberg, Eduardo, Montoya, Fabiana, Cosentino, Vanesa, Limonta, Massimiliano, Brucato, Antonio Luca, Lupi, Elide, Spertini, Francois, Ribi, Camillo, Buss, Guillaume, Martin, Thierry, Guffroy, Aurelien, Poindron, Vincent, Chung, Lori, Schmeiser, Tim, Zebryk, Pawel, Riso, Nuno, Riemekasten, Gabriela, Rezus, Elena, Puttini, Piercarlo Sarzi, Ege Üniversitesi, University of Zurich, Distler, Oliver, Chizzolini, Carlo, Allai, Daniela, Becker, M., Graf, N., Sauter, R., Allanore, Y., Curram, J., Denton, C. P., Khanna, D., Matucci-Cerinic, M., de Oliveira Pena, J., Pope, J. E., Distler, O., Guiducci, S., Walker, U., Jaeger, V., Bannert, B., Lapadula, G., Becvarare, R., Cutolo, M., Valentini, G., Siegert, E., Rednic, S., Montecucco, C., Carreira, P. E., Novak, S., Czirjak, L., Varju, C., Chizzolini, C., Allai, D., Kucharz, E. J., Cozzi, F., Rozman, B., Mallia, C., Gabrielli, A., Bancel, D. F., Airo, P., Hesselstrand, R., Martinovic, D., Balbir-Gurman, A., Braun-Moscovici, Y., Hunzelmann, N., Pellerito, R., Caramaschi, P., Black, C., Damjanov, N., Henes, J., Santamaria, V. O., Heitmann, S., Seidel, M., Pereira Da Silva, J. A., Stamenkovic, B., Selmi, C. F., Tikly, M., Denisov, L. N., Muller-Ladner, U., Engelhart, M., Hachulla, E., Riccieri, V., Ionescu, R. M., Mihai, C., Sunderkotter, C., Kuhn, A., Schett, G., Distler, J., Meroni, P., Ingegnoli, F., Mouthon, L., De Keyser, F., Smith, V., Cantatore, F. P., Corrado, A., Ullman, S., Iversen, L., Pozzi, M. R., Eyerich, K., Hein, R., Knott, E., Wiland, P., Szmyrka-Kaczmarek, M., Sokolik, R., Morgiel, E., Madej, M., Alegre-Sancho, J. J., Krummel-Lorenz, B., Saar, P., Aringer, M., Gunther, C., Anne, E., Westhovens, R., De Langhe, E., Lenaerts, J., Anic, B., Baresic, M., Mayer, M., Uprus, M., Otsa, K., Yavuz, S., Radominski, S. C., de Souza Muller, C., Azevedo, V. F., Popa, S., Zenone, T., Stebbings, S., Highton, J., Mathieu, A., Vacca, A., Stamp, L., Chapman, P., O'Donnell, J., Solanki, K., Doube, A., Veale, D., O'Rourke, M., Loyo, E., Li, M., Rosato, E., Amoroso, A., Gigante, A., Oksel, F., Yargucu, F., Tanaseanu, C. -M., Popescu, M., Dumitrascu, A., Tiglea, I., Foti, R., Visalli, E., Benenati, A., Amato, G., Ancuta, C., Chirieac, R., Villiger, P., Adler, S., Dan, D., de la Pena Lefebvre, P. G., Rubio, S. R., Exposito, M. V., Sibilia, J., Chatelus, E., Gottenberg, J. E., Chifflot, H., Litinsky, I., Del Galdo, F., Venalis, A., Saketkoo, L. A., Lasky, J. 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Subjects
INVOLVEMENT, SELECTION, BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences, diffuse, predictive factors, systemic sclerosis, 2745 Rheumatology, epidemiologic methods, morbidity, Disease, Immunology, General Biochemistry, Genetics and Molecular Biology, Immunology and Allergy, Rheumatology, INTERSTITIAL LUNG-DISEASE, disease worsening, mortality, predictive factors, systemic sclerosis, predictive factor, disease worsening, DESIGN, middle aged, Medicine and Health Sciences, FIBROSIS, scleroderma, SKIN THICKNESS SCORE, mortality, skin and connective tissue diseases, Prospective cohort study, humans, lung diseases, ddc:616, education.field_of_study, heart diseases, integumentary system, clinical trials as topic, BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti, 10051 Rheumatology Clinic and Institute of Physical Medicine, Interstitial lung disease, follow-up studies, ddc, female, Cohort, 2723 Immunology and Allergy, europe, Life Sciences & Biomedicine, CLINICAL-TRIALS, survival rate, medicine.medical_specialty, Population, 610 Medicine & health, disease progression, male, 1300 General Biochemistry, Genetics and Molecular Biology, Internal medicine, Severity of illness, REGRESSION, medicine, severity of illness index, education, Survival rate, METAANALYSIS, 2403 Immunology, Science & Technology, Scleroderma, Systemic, business.industry, MORTALITY, systemic, medicine.disease, prospective studies, Clinical trial, prognosis, scleroderma, diffuse, scleroderma, systemic, Scleroderma, Diffuse, business
Abstract

PubMed: 31227488, Objectives Mortality and worsening of organ function are desirable endpoints for clinical trials in systemic sclerosis (SSc). The aim of this study was to identify factors that allow enrichment of patients with these endpoints, in a population of patients from the European Scleroderma Trials and Research group database. Methods Inclusion criteria were diagnosis of diffuse SSc and follow-up over 12±3 months. Disease worsening/organ progression was fulfilled if any of the following events occurred: new renal crisis; decrease of lung or heart function; new echocardiography-suspected pulmonary hypertension or death. In total, 42 clinical parameters were chosen as predictors for the analysis by using (1) imputation of missing data on the basis of multivariate imputation and (2) least absolute shrinkage and selection operator regression. Results Of 1451 patients meeting the inclusion criteria, 706 had complete data on outcome parameters and were included in the analysis. Of the 42 outcome predictors, eight remained in the final regression model. There was substantial evidence for a strong association between disease progression and age, active digital ulcer (DU), lung fibrosis, muscle weakness and elevated C-reactive protein (CRP) level. Active DU, CRP elevation, lung fibrosis and muscle weakness were also associated with a significantly shorter time to disease progression. A bootstrap validation step with 10 000 repetitions successfully validated the model. Conclusions The use of the predictive factors presented here could enable cohort enrichment with patients at risk for overall disease worsening in SSc clinical trials. © Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY. Published by BMJ., Bayer Bayer, United Kingdom Université Paris Descartes Li Ka Shing Foundation, LKSF University of Michigan, U-M, 1Department of Rheumatology and the Centre of experimental Rheumatology, University Hospital Zurich, Zurich, switzerland 2Graf Biostatistics, Winterthur, switzerland 3Big Data institute, li Ka shing Centre for Health information and Discovery, nuffield Department of Medicine, University of Oxford, Oxford, UK 4Rheumatology a Department, Paris Descartes University, sorbonne Paris Cité, Cochin Hospital, Paris, France 5Data science and analytics, Bayer plc, Reading, UK 6UCl Division of Medicine, Royal Free Campus, london, UK 7Division of Rheumatology, Department of internal Medicine, University of Michigan scleroderma Program, University of Michigan, ann arbor, Michigan, Usa 8Department of experimental and Clinical Medicine, University of Florence, Florence, italy 9Bayer Us llC, Whippany, new Jersey, Usa 10Department of Medicine, Division of Rheumatology, University of Western Ontario, st. Joseph’s Health Care, london, Ontario, Canada Acknowledgements The R-code for the linear Mi-lassO was received from Qixuan Chen.21 Medical writing assistance was provided by adelphi Communications ltd (Bollington, UK), funded by Bayer aG (Berlin, Germany)., Contributors study conception and design, acquisition of data, analysis and interpretation of data and drafting and revising the article: OD and MB; analysis and interpretation of data: OD, MB, Rs and nG. all authors have critically reviewed and approved the final submitted version to be published. Funding This study was supported by a grant from Bayer aG. Bayer employees are coauthors of this paper and supported the study design and interpretation of the data, but otherwise Bayer had no influence on the study., Competing interests MOB declares no conflict of interest. OD has had consultancy relationships with actelion, Bayer, Biogen idec, Boehringer ingelheim, Chemomab, espeRare foundation, Genentech/Roche, GsK, inventiva, italfarmaco, lilly, medac, Medimmune, Mitsubishi Tanabe Pharma, Pharmacyclics, novartis, Pfizer, sanofi, sinoxa and UCB in the area of potential treatments of scleroderma and its complications. OD has received research funding from actelion, Bayer, Boehringer ingelheim, Mitsubishi Tanabe Pharma and Roche in the area of potential treatments of scleroderma and its complications. OD has a patent for mir-29 licensed for the treatment of systemic sclerosis. DK has consultancy relationships and/or has received grant/research support from Bayer, Bristol-Myers squibb, Boehringer ingelheim, Genentech/Roche, niH, Pfizer, sanofi-aventis Pharmaceuticals, actelion Pharmaceuticals Us, Chemomab, Corbus, Covis, Cytori, eicos, eMD serono, Gilead, GlaxosmithKline, and UCB Pharma. He is a shareholder of eicos. CPD has consultancy relationships with and/or has received speakers’ bureau fees from actelion Pharmaceuticals Us, Bayer aG, GlaxosmithKline, Csl Behring, Merck serono, Roche Pharmaceuticals, Genentech and Biogen iDeC inc., inventiva, sanofi-aventis Pharmaceuticals and Boehringer ingelheim. JeP has consultancy relationships with and/or has received grant/research support from actelion, Bayer aG, Bristol-Myers squibb, Merck, Pfizer inc. and Roche. MM-C has consultancy relationships and/ or has received grant/research support from Pfizer, Bristol-Myers squibb, actelion, UCB Pharma, Bayer, Chemomab, Genentech/Roche, inventiva and lilly. Ya has consultancy relationships with and/or has received grant/research support from actelion, Pharmaceuticals Us, Bayer aG, Bristol-Myers squibb, inventiva, Medac, Pfizer inc., Roche Pharmaceuticals, Genentech and Biogen iDeC inc., sanofi-aventis Pharmaceuticals and servier. JdOP and JC are employees of Bayer. nTG has nothing to disclose.

Published
2019

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