Treatment outcome in early diffuse cutaneous systemic sclerosis : The European Scleroderma Observational Study (ESOS)

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Herrick, Ariane LGreat Ormond Street Hospital,University of Manchester (author)

Treatment outcome in early diffuse cutaneous systemic sclerosis : The European Scleroderma Observational Study (ESOS)

Article/chapterEnglish2017

Publisher, publication year, extent ...

2017-02-10
BMJ,2017
12 s.

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LIBRIS-ID:oai:lup.lub.lu.se:c78f40d0-0b38-43bf-b220-76e05526147b
https://lup.lub.lu.se/record/c78f40d0-0b38-43bf-b220-76e05526147bURI
https://doi.org/10.1136/annrheumdis-2016-210503DOI
https://gup.ub.gu.se/publication/252021URI

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Language:English
Summary in:English

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SwePubSwePub

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Subject category:art swepub-publicationtype
Subject category:ref swepub-contenttype

Notes

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.

Subject headings and genre

MEDICIN OCH HÄLSOVETENSKAP Klinisk medicin Reumatologi och inflammation hsv//swe
MEDICAL AND HEALTH SCIENCES Clinical Medicine Rheumatology and Autoimmunity hsv//eng
Cyclophosphamide
Methotrexate
Systemic Sclerosis
Treatment

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Pan, XiaoyanUniversity of Manchester (author)
Peytrignet, SébastienUniversity of Manchester (author)
Lunt, MarkUniversity of Manchester (author)
Hesselstrand, RogerLund University,Lunds universitet,Reumatologi och molekylär skelettbiologi,Sektion III,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Hjärt-lungsjukdom - information, stöd och bemötande,Forskargrupper vid Lunds universitet,Forskargruppen för systemisk skleros, Lund,Rheumatology,Section III,Department of Clinical Sciences, Lund,Faculty of Medicine,Cardiopulmonary disease - information, support and reception,Lund University Research Groups,Lund Systemic Sclerosis Research Group(Swepub:lu)reum-rhe (author)
Mouthon, LucParis Descartes University (author)
Silman, AlanUniversity of Oxford (author)
Brown, EdithUniversity of Manchester (author)
Czirják, LászlóUniversity of Pécs (author)
Distler, Jörg H WFriedrich-Alexander University Erlangen-Nürnberg (author)
Distler, OliverUniversity of Zurich (author)
Fligelstone, KimGreat Ormond Street Hospital (author)
Gregory, William J.Great Ormond Street Hospital (author)
Ochiel, RachelGreat Ormond Street Hospital (author)
Vonk, MadelonRadboud University Nijmegen (author)
Ancuta, CodrinaGrigore T. Popa University of Medicine and Pharmacy (author)
Ong, Voon H.University College London (author)
Farge, DominiqueInstitut Gustave Roussy (author)
Hudson, MarieMcGill University (author)
Matucci-Cerinic, MarcoUniversity of Florence (author)
Balbir-Gurman, AlexandraTechnion - Israel Institute of Technology (author)
Midtvedt, ØyvindNorwegian Radium Hospital (author)
Jordan, Alison C.New Queen Elizabeth Hospital (author)
Jobanputra, PareshNew Queen Elizabeth Hospital (author)
Stevens, WendySt. Vincent's Hospital, Melbourne (author)
Moinzadeh, PiaUniversity of Cologne (author)
Hall, Frances C.University of Cambridge (author)
Agard, ChristianUniversity of Nantes (author)
Anderson, Marina E.University of Liverpool (author)
Diot, ElisabethHopital Bretonneau (author)
Madhok, RajanGlasgow Royal Infirmary (author)
Akil, MohammedSheffield Teaching Hospitals (author)
Buch, Maya HUniversity of Leeds (author)
Chung, LorindaStanford University (author)
Damjanov, NemanjaUniversity of Belgrade (author)
Gunawardena, HarshaNorth Bristol NHS Trust (author)
Lanyon, PeterNottingham University Hospitals NHS Trust (author)
Ahmad, YasmeenPeter Maddison Rheumatology Centre (author)
Chakravarty, KuntalQueen’s Hospital (author)
Jacobsen, SørenUniversity of Copenhagen (author)
MacGregor, Alexander J.University of East Anglia (author)
McHugh, NeilRoyal National Hospital for Rheumatic Diseases (author)
Müller-Ladner, UlfJustus Liebig University Giessen (author)
Riemekasten, GabrielaBritish Columbia Children's Hospital (author)
Becker, MichaelCharité - University Medicine Berlin (author)
Roddy, JanetRoyal Perth Hospital (author)
Carreira, Patricia E.12 de Octubre University Hospital (author)
Fauchais, Anne-LaureUniversity Hospital Of Limoges (author)
Hachulla, EricUniversity of Lille (author)
Hamilton, JenniferGateshead Hospitals Foundation Trust (author)
Inanç, MuratIstanbul University (author)
McLaren, John S.Whyteman's Brae Hospital (author)
van Laar, Jacob M.University Medical Center Utrecht (author)
Pathare, SanjayJames Cook University Hospital (author)
Proudman, SusannahUniversity of Adelaide (author)
Rudin, Anna,1961Gothenburg University,Göteborgs universitet,Institutionen för medicin, avdelningen för reumatologi och inflammationsforskning,Institute of Medicine, Department of Rheumatology and Inflammation Research(Swepub:gu)xrudan (author)
Sahhar, JoanneMonash University (author)
Coppere, BrigitteHôpital Edouard Herriot (author)
Serratrice, ChristineHospital Saint Joseph (author)
Sheeran, TomCannock Chase Hospital (author)
Veale, Douglas J.St Vincent's University Hospital (author)
Grange, ClaireCentre Hospitalier Lyon Sud (author)
Trad, Georges SelimCHU Ambroise Paré (author)
Denton, Christopher PUniversity College London (author)
Great Ormond Street HospitalUniversity of Manchester (creator_code:org_t)

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In:Annals of the Rheumatic Diseases: BMJ76:7, s. 1207-12180003-49671468-2060

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By the author/editor: Herrick, Ariane ...; Pan, Xiaoyan; Peytrignet, Séba ...; Lunt, Mark; Hesselstrand, Ro ...; Mouthon, Luc; show more...; Silman, Alan; Brown, Edith; Czirják, László; Distler, Jörg H ...; Distler, Oliver; Fligelstone, Kim; Gregory, William ...; Ochiel, Rachel; Vonk, Madelon; Ancuta, Codrina; Ong, Voon H.; Farge, Dominique; Hudson, Marie; Matucci-Cerinic, ...; Balbir-Gurman, A ...; Midtvedt, Øyvind; Jordan, Alison C ...; Jobanputra, Pare ...; Stevens, Wendy; Moinzadeh, Pia; Hall, Frances C.; Agard, Christian; Anderson, Marina ...; Diot, Elisabeth; Madhok, Rajan; Akil, Mohammed; Buch, Maya H; Chung, Lorinda; Damjanov, Nemanj ...; Gunawardena, Har ...; Lanyon, Peter; Ahmad, Yasmeen; Chakravarty, Kun ...; Jacobsen, Søren; MacGregor, Alexa ...; McHugh, Neil; Müller-Ladner, U ...; Riemekasten, Gab ...; Becker, Michael; Roddy, Janet; Carreira, Patric ...; Fauchais, Anne-L ...; Hachulla, Eric; Hamilton, Jennif ...; Inanç, Murat; McLaren, John S.; van Laar, Jacob ...; Pathare, Sanjay; Proudman, Susann ...; Rudin, Anna, 196 ...; Sahhar, Joanne; Coppere, Brigitt ...; Serratrice, Chri ...; Sheeran, Tom; Veale, Douglas J ...; Grange, Claire; Trad, Georges Se ...; Denton, Christop ...; show less...

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