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Efficacy and Safety of Cyclophosphamide Treatment in Severe Juvenile
Dermatomyositis Shown by Marginal Structural Modeling
#MMPMID29342499
Deakin CT
; Campanilho-Marques R
; Simou S
; Moraitis E
; Wedderburn LR
; Pullenayegum E
; Pilkington CA
Arthritis Rheumatol
2018[May]; 70
(5
): 785-793
PMID29342499
show ga
OBJECTIVE: In patients with severe or refractory juvenile dermatomyositis (DM),
second-line treatments may be required. Cyclophosphamide (CYC) is used to treat
some connective tissue diseases, but evidence of its efficacy in juvenile DM is
limited. This study was undertaken to describe clinical improvement in juvenile
DM patients treated with CYC and model the efficacy of CYC treatment compared to
no CYC treatment. METHODS: Clinical data on skin, global, and muscle disease for
patients recruited to the Juvenile DM Cohort and Biomarker Study were analyzed.
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 the start of CYC
treatment, 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 56 patients treated with CYC in unadjusted analysis. Limited
evidence suggested a 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 an ~6-month course of CYC treatment >12 months ago compared
to patients never or not yet treated with CYC. In the treated patients, the
modified skin Disease Activity Score for juvenile DM was 1.19 units lower (P =
0.0085) and the 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: Our findings indicate that CYC is efficacious with no short-term side
effects. Improvements in skin, global, and muscle disease were observed. Further
studies are required to evaluate longer-term side effects.
|Antirheumatic Agents/therapeutic use
[MESH]
|Azathioprine/therapeutic use
[MESH]
|Child
[MESH]
|Child, Preschool
[MESH]
|Cohort Studies
[MESH]
|Cyclophosphamide/*therapeutic use
[MESH]
|Dermatomyositis/*drug therapy
[MESH]
|Female
[MESH]
|Glucocorticoids/therapeutic use
[MESH]
|Humans
[MESH]
|Hydroxychloroquine/therapeutic use
[MESH]
|Immunoglobulins, Intravenous/therapeutic use
[MESH]
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