Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)
Autori
Herrick, Ariane L.Pan, Xiaoyan
Peytrignet, Sebastien
Lunt, Mark
Hesselstrand, Roger
Mouthon, Luc
Silman, Alan
Brown, Edith
Czirjak, Laszlo
Distler, Joerg H. W.
Distler, Oliver
Fligelstone, Kim
Gregory, William J.
Ochiel, Rachel
Vonk, Madelon
Ancuta, Codrina
Ong, Voon H.
Farge, Dominique
Hudson, Marie
Matucci-Cerinic, Marco
Balbir-Gurman, Alexandra
Midtvedt, Oyvind
Jordan, Alison C.
Jobanputra, Paresh
Stevens, Wendy
Moinzadeh, Pia
Hall, Frances C.
Agard, Christian
Anderson, Marina E.
Diot, Elisabeth
Madhok, Rajan
Akil, Mohammed
Buch, Maya H.
Chung, Lorinda
Damjanov, Nemanja
Gunawardena, Harsha
Lanyon, Peter
Ahmad, Yasmeen
Chakravarty, Kuntal
Jacobsen, Soren
MacGregor, Alexander J.
McHugh, Neil
Mueller-Ladner, Ulf
Riemekasten, Gabriela
Becker, Michael
Roddy, Janet
Carreira, Patricia E.
Fauchais, Anne Laure
Hachulla, Eric
Hamilton, Jennifer
Inanc, Murat
McLaren, John S.
van Laar, Jacob M.
Pathare, Sanjay
Proudman, Susannah
Rudin, Anna
Sahhar, Joanne
Coppere, Brigitte
Serratrice, Christine
Sheeran, Tom
Veale, Douglas J.
Grange, Claire
Trad, Georges-Selim
Denton, Christopher P.
Članak u časopisu (Objavljena verzija)
Metapodaci
Prikaz svih podataka o dokumentuApstrakt
Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches. Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or no immunosuppressant. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival. Results Of 326 patients recruited from 50 centres, 65 were prescribed methotrexate, 118 MMF, 87 cyclophosphamide and 56 no immunosuppressant. 276 (84.7%) patien...ts completed 12 and 234 (71.7%) 24 months follow-up (or reached last visit date). There were statistically significant reductions in mRSS at 12 months in all groups: -4.0 (-5.2 to -2.7) units for methotrexate, -4.1 (-5.3 to -2.9) for MMF, -3.3 (-4.9 to -1.7) for cyclophosphamide and -2.2 (-4.0 to -0.3) for no immunosuppressant (p value for between-group differences=0.346). There were no statistically significant differences in survival between protocols before (p=0.389) or after weighting (p=0.440), but survival was poorest in the no immunosuppressant group (84.0%) at 24 months. Conclusions These findings may support using immunosuppressants for early dcSSc but suggest that overall benefit is modest over 12 months and that better treatments are needed.
Izvor:
Annals of the Rheumatic Diseases, 2017, 76, 7, 1207-1218Finansiranje / projekti:
- European League Against Rheumatism (EULAR) Orphan Disease Programme, Scleroderma and Raynauds UK
DOI: 10.1136/annrheumdis-2016-210503
ISSN: 0003-4967; 1468-2060
PubMed: 28188239
WoS: 000403074400014
Scopus: 2-s2.0-85020703821
Kolekcije
Institucija/grupa
VinčaTY - JOUR AU - Herrick, Ariane L. AU - Pan, Xiaoyan AU - Peytrignet, Sebastien AU - Lunt, Mark AU - Hesselstrand, Roger AU - Mouthon, Luc AU - Silman, Alan AU - Brown, Edith AU - Czirjak, Laszlo AU - Distler, Joerg H. W. AU - Distler, Oliver AU - Fligelstone, Kim AU - Gregory, William J. AU - Ochiel, Rachel AU - Vonk, Madelon AU - Ancuta, Codrina AU - Ong, Voon H. AU - Farge, Dominique AU - Hudson, Marie AU - Matucci-Cerinic, Marco AU - Balbir-Gurman, Alexandra AU - Midtvedt, Oyvind AU - Jordan, Alison C. AU - Jobanputra, Paresh AU - Stevens, Wendy AU - Moinzadeh, Pia AU - Hall, Frances C. AU - Agard, Christian AU - Anderson, Marina E. AU - Diot, Elisabeth AU - Madhok, Rajan AU - Akil, Mohammed AU - Buch, Maya H. AU - Chung, Lorinda AU - Damjanov, Nemanja AU - Gunawardena, Harsha AU - Lanyon, Peter AU - Ahmad, Yasmeen AU - Chakravarty, Kuntal AU - Jacobsen, Soren AU - MacGregor, Alexander J. AU - McHugh, Neil AU - Mueller-Ladner, Ulf AU - Riemekasten, Gabriela AU - Becker, Michael AU - Roddy, Janet AU - Carreira, Patricia E. AU - Fauchais, Anne Laure AU - Hachulla, Eric AU - Hamilton, Jennifer AU - Inanc, Murat AU - McLaren, John S. AU - van Laar, Jacob M. AU - Pathare, Sanjay AU - Proudman, Susannah AU - Rudin, Anna AU - Sahhar, Joanne AU - Coppere, Brigitte AU - Serratrice, Christine AU - Sheeran, Tom AU - Veale, Douglas J. AU - Grange, Claire AU - Trad, Georges-Selim AU - Denton, Christopher P. PY - 2017 UR - https://vinar.vin.bg.ac.rs/handle/123456789/1599 AB - Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches. Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or no immunosuppressant. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival. Results Of 326 patients recruited from 50 centres, 65 were prescribed methotrexate, 118 MMF, 87 cyclophosphamide and 56 no immunosuppressant. 276 (84.7%) patients completed 12 and 234 (71.7%) 24 months follow-up (or reached last visit date). There were statistically significant reductions in mRSS at 12 months in all groups: -4.0 (-5.2 to -2.7) units for methotrexate, -4.1 (-5.3 to -2.9) for MMF, -3.3 (-4.9 to -1.7) for cyclophosphamide and -2.2 (-4.0 to -0.3) for no immunosuppressant (p value for between-group differences=0.346). There were no statistically significant differences in survival between protocols before (p=0.389) or after weighting (p=0.440), but survival was poorest in the no immunosuppressant group (84.0%) at 24 months. Conclusions These findings may support using immunosuppressants for early dcSSc but suggest that overall benefit is modest over 12 months and that better treatments are needed. T2 - Annals of the Rheumatic Diseases T1 - Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS) VL - 76 IS - 7 SP - 1207 EP - 1218 DO - 10.1136/annrheumdis-2016-210503 ER -
@article{ author = "Herrick, Ariane L. and Pan, Xiaoyan and Peytrignet, Sebastien and Lunt, Mark and Hesselstrand, Roger and Mouthon, Luc and Silman, Alan and Brown, Edith and Czirjak, Laszlo and Distler, Joerg H. W. and Distler, Oliver and Fligelstone, Kim and Gregory, William J. and Ochiel, Rachel and Vonk, Madelon and Ancuta, Codrina and Ong, Voon H. and Farge, Dominique and Hudson, Marie and Matucci-Cerinic, Marco and Balbir-Gurman, Alexandra and Midtvedt, Oyvind and Jordan, Alison C. and Jobanputra, Paresh and Stevens, Wendy and Moinzadeh, Pia and Hall, Frances C. and Agard, Christian and Anderson, Marina E. and Diot, Elisabeth and Madhok, Rajan and Akil, Mohammed and Buch, Maya H. and Chung, Lorinda and Damjanov, Nemanja and Gunawardena, Harsha and Lanyon, Peter and Ahmad, Yasmeen and Chakravarty, Kuntal and Jacobsen, Soren and MacGregor, Alexander J. and McHugh, Neil and Mueller-Ladner, Ulf and Riemekasten, Gabriela and Becker, Michael and Roddy, Janet and Carreira, Patricia E. and Fauchais, Anne Laure and Hachulla, Eric and Hamilton, Jennifer and Inanc, Murat and McLaren, John S. and van Laar, Jacob M. and Pathare, Sanjay and Proudman, Susannah and Rudin, Anna and Sahhar, Joanne and Coppere, Brigitte and Serratrice, Christine and Sheeran, Tom and Veale, Douglas J. and Grange, Claire and Trad, Georges-Selim and Denton, Christopher P.", year = "2017", abstract = "Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches. Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or no immunosuppressant. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival. Results Of 326 patients recruited from 50 centres, 65 were prescribed methotrexate, 118 MMF, 87 cyclophosphamide and 56 no immunosuppressant. 276 (84.7%) patients completed 12 and 234 (71.7%) 24 months follow-up (or reached last visit date). There were statistically significant reductions in mRSS at 12 months in all groups: -4.0 (-5.2 to -2.7) units for methotrexate, -4.1 (-5.3 to -2.9) for MMF, -3.3 (-4.9 to -1.7) for cyclophosphamide and -2.2 (-4.0 to -0.3) for no immunosuppressant (p value for between-group differences=0.346). There were no statistically significant differences in survival between protocols before (p=0.389) or after weighting (p=0.440), but survival was poorest in the no immunosuppressant group (84.0%) at 24 months. Conclusions These findings may support using immunosuppressants for early dcSSc but suggest that overall benefit is modest over 12 months and that better treatments are needed.", journal = "Annals of the Rheumatic Diseases", title = "Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)", volume = "76", number = "7", pages = "1207-1218", doi = "10.1136/annrheumdis-2016-210503" }
Herrick, A. L., Pan, X., Peytrignet, S., Lunt, M., Hesselstrand, R., Mouthon, L., Silman, A., Brown, E., Czirjak, L., Distler, J. H. W., Distler, O., Fligelstone, K., Gregory, W. J., Ochiel, R., Vonk, M., Ancuta, C., Ong, V. H., Farge, D., Hudson, M., Matucci-Cerinic, M., Balbir-Gurman, A., Midtvedt, O., Jordan, A. C., Jobanputra, P., Stevens, W., Moinzadeh, P., Hall, F. C., Agard, C., Anderson, M. E., Diot, E., Madhok, R., Akil, M., Buch, M. H., Chung, L., Damjanov, N., Gunawardena, H., Lanyon, P., Ahmad, Y., Chakravarty, K., Jacobsen, S., MacGregor, A. J., McHugh, N., Mueller-Ladner, U., Riemekasten, G., Becker, M., Roddy, J., Carreira, P. E., Fauchais, A. L., Hachulla, E., Hamilton, J., Inanc, M., McLaren, J. S., van Laar, J. M., Pathare, S., Proudman, S., Rudin, A., Sahhar, J., Coppere, B., Serratrice, C., Sheeran, T., Veale, D. J., Grange, C., Trad, G.,& Denton, C. P.. (2017). Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). in Annals of the Rheumatic Diseases, 76(7), 1207-1218. https://doi.org/10.1136/annrheumdis-2016-210503
Herrick AL, Pan X, Peytrignet S, Lunt M, Hesselstrand R, Mouthon L, Silman A, Brown E, Czirjak L, Distler JHW, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk M, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jordan AC, Jobanputra P, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov N, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, MacGregor AJ, McHugh N, Mueller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, McLaren JS, van Laar JM, Pathare S, Proudman S, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad G, Denton CP. Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). in Annals of the Rheumatic Diseases. 2017;76(7):1207-1218. doi:10.1136/annrheumdis-2016-210503 .
Herrick, Ariane L., Pan, Xiaoyan, Peytrignet, Sebastien, Lunt, Mark, Hesselstrand, Roger, Mouthon, Luc, Silman, Alan, Brown, Edith, Czirjak, Laszlo, Distler, Joerg H. W., Distler, Oliver, Fligelstone, Kim, Gregory, William J., Ochiel, Rachel, Vonk, Madelon, Ancuta, Codrina, Ong, Voon H., Farge, Dominique, Hudson, Marie, Matucci-Cerinic, Marco, Balbir-Gurman, Alexandra, Midtvedt, Oyvind, Jordan, Alison C., Jobanputra, Paresh, Stevens, Wendy, Moinzadeh, Pia, Hall, Frances C., Agard, Christian, Anderson, Marina E., Diot, Elisabeth, Madhok, Rajan, Akil, Mohammed, Buch, Maya H., Chung, Lorinda, Damjanov, Nemanja, Gunawardena, Harsha, Lanyon, Peter, Ahmad, Yasmeen, Chakravarty, Kuntal, Jacobsen, Soren, MacGregor, Alexander J., McHugh, Neil, Mueller-Ladner, Ulf, Riemekasten, Gabriela, Becker, Michael, Roddy, Janet, Carreira, Patricia E., Fauchais, Anne Laure, Hachulla, Eric, Hamilton, Jennifer, Inanc, Murat, McLaren, John S., van Laar, Jacob M., Pathare, Sanjay, Proudman, Susannah, Rudin, Anna, Sahhar, Joanne, Coppere, Brigitte, Serratrice, Christine, Sheeran, Tom, Veale, Douglas J., Grange, Claire, Trad, Georges-Selim, Denton, Christopher P., "Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)" in Annals of the Rheumatic Diseases, 76, no. 7 (2017):1207-1218, https://doi.org/10.1136/annrheumdis-2016-210503 . .