UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Efficacy and Safety of Cyclophosphamide Treatment in Severe Juvenile Dermatomyositis Shown by Marginal Structural Modeling

Deakin, CT; Campanilho-Marques, R; Simou, S; Moraitis, E; Wedderburn, LR; Pullenayegum, E; Pilkington, CA; (2018) Efficacy and Safety of Cyclophosphamide Treatment in Severe Juvenile Dermatomyositis Shown by Marginal Structural Modeling. Arthritis & Rheumatology , 70 (5) pp. 785-793. 10.1002/art.40418. Green open access

[img]
Preview
Text
Efficacy and Safety of Cyclophosphamide Treatment in Severe Juvenile Dermatomyositis Shown by Marginal Structural Modeling.pdf - Published version

Download (334kB) | Preview

Abstract

OBJECTIVE: In patients with severe or refractory juvenile dermatomyositis (JDM), second-line treatments may be required. Cyclophosphamide (CYC) is used to treat some connective tissue diseases, but evidence of efficacy in JDM is limited. This study aimed to describe clinical improvement in JDM patients treated with CYC and model efficacy of CYC compared to patients not treated with CYC. METHODS: Clinical data on skin, global and muscle disease were analyzed from patients recruited to the Juvenile Dermatomyositis Cohort and Biomarker Study. Clinical improvement following CYC treatment was described using unadjusted analysis. Marginal structural models (MSMs) were used to model treatment efficacy and adjust for confounding by indication. RESULTS: Compared to CYC start, there were reductions at 6, 12 and 24 months in skin disease (p=1.3×10-10 ), global disease (p=2.4×10-8 ), and muscle disease (p=8.0×10-10 ) for n=56 patients treated with CYC in unadjusted analysis. Limited evidence suggested reduction in glucocorticoid dose (p=0.047) in patients treated with CYC. MSM analysis showed reduced global disease and skin disease in patients who started CYC treatment over 12 months ago compared to patients never or not yet treated with CYC. In these patients, modified disease activity score for skin disease was 1.19 units lower (p=0.0085) and physician's global assessment was 0.66 units lower (p=0.027). Minor adverse events were reported in 3 patients within 1 year of stopping CYC. CONCLUSION: CYC is efficacious with no short-term side-effects seen in this study. Improvements in skin, global and muscle disease were observed. Further studies are required to evaluate longer-term side-effects. This article is protected by copyright. All rights reserved.

Type: Article
Title: Efficacy and Safety of Cyclophosphamide Treatment in Severe Juvenile Dermatomyositis Shown by Marginal Structural Modeling
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/art.40418
Publisher version: http://doi.org/10.1002/art.40418
Language: English
Additional information: Copyright © 2018 The Authors. Arthritis & Rheumatology published by Wiley Periodicals, Inc. on behalf of American College of Rheumatology. This is an open access article under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Keywords: Cyclophosphamide, epidemiologic methods, juvenile dermatomyositis, juvenile myositis and pediatric rheumatology
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Biology and Cancer Dept
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > ICH - Laboratory Management
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10041956
Downloads since deposit
22Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item