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2017 ; 76
(7
): 1207-1218
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English Wikipedia
Treatment outcome in early diffuse cutaneous systemic sclerosis: the European
Scleroderma Observational Study (ESOS)
#MMPMID28188239
Herrick AL
; Pan X
; Peytrignet S
; Lunt M
; Hesselstrand R
; Mouthon L
; Silman A
; Brown E
; Czirják L
; Distler JHW
; Distler O
; Fligelstone K
; Gregory WJ
; Ochiel R
; Vonk M
; Ancu?a C
; Ong VH
; Farge D
; Hudson M
; Matucci-Cerinic M
; Balbir-Gurman A
; Midtvedt Ř
; 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
; Müller-Ladner U
; Riemekasten G
; Becker M
; Roddy J
; Carreira PE
; Fauchais AL
; Hachulla E
; Hamilton J
; ?nanç 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 GS
; Denton CP
Ann Rheum Dis
2017[Jul]; 76
(7
): 1207-1218
PMID28188239
show ga
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. TRIAL REGISTRATION NUMBER:
NCT02339441.