Mycophenolate Mofetil As a Steroid Sparing Agent in Polymyositis and Dermatomyositis: a Systematic Review of the Literature

Janet Roberts and Stephanie O. Keeling, Medicine, University of Alberta, Edmonton, AB, Canada

Meeting: 2015 ACR/ARHP Annual Meeting

Date of first publication: September 29, 2015

Keywords: Idiopathic Inflammatory Myopathies (IIM) and mycophenolate mofetil

Session Information

Date: Tuesday, November 10, 2015

Title: Muscle Biology, Myositis and Myopathies Poster II: Autoantibodies and Treatments in Inflammatory Myopathies

Session Type: ACR Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose:

Idiopathic inflammatory myopathies (IM’s) including polymyositis (PM) and dermatomyositis (DM) are rare systemic autoimmune diseases causing chronic muscle inflammation with significant morbidity and mortality. Corticosteroids are the primary treatment; however, given the chronic nature of these conditions and the inherent risk of long term steroid use, additional immunosuppressive agents are often required. Myocophenolate mofetil (MMF) may be beneficial in the treatment of IM’s with a relatively favorable side effect profile. We systematically reviewed the literature to evaluate the benefits of MMF in the treatment of PM and DM.

Methods:

A systematic review of four databases (Embase, Pubmed, Web of Science and Scopus) using keywords pertaining to IM’s, DM, PM, immunosuppression and MMF was conducted. All studies up to June 2014 including patients over the age of 18 with PM or DM defined by Bohan and Peter and treated with MMF were included. Two reviewers independently reviewed the title screen and data was extracted using a standardized form. Primary outcomes included changes in corticosteroid dose, muscle enzymes, strength, skin manifestations, and interstitial lung disease outcomes (ILD) (computed tomography (CT) scan and pulmonary function tests (PFTs)).

Results:

One hundred and ninety (190) full articles were reviewed after the title screen of 458 articles. Eighteen articles met inclusion criteria including 5 case reports, 11 case series and 2 open label trials. A total of 110 patients (PM 16; DM 46) were treated with MMF. Ninety three percent of patients were on concomitant corticosteroids (n=102), with 89% (n=91) decreasing their steroid dose after initiation of MMF. Of those with baseline elevation in muscle enzymes, 77% (n=30) showed a decrease post treatment. In 10 patients, the change in CK was not specified. In those patients with skin manifestations (n=32), 88% (n=28) showed improved skin manifestations. In the 51% (n=56) of ILD patients, improvements were noted in PFTs, perceived dyspnea and CT scan findings, including improvement in ground glass opacities and pneumonitis. Side effects (21%) were generally mild, most common being gastrointestinal upset. Only 7% (n=8) required cessation of treatment due to side effects.

Conclusion:

MMF is a potential alternative first line steroid-sparing agent in those with IM’s. High quality trials are needed to further evaluate the efficacy and safety of MMF in this patient population. Lack of consensus on diagnostic approaches and outcome measures in IM research limits the ability to compare individual patients and studies. The development of core set measures by the International Myositis Assessment and Clinical Studies Groups (IMACS)will allow collaboration between multiple disciplines and centers to increase the quality and number of trials in this area.

Table 1. Change in IM outcomes with Mycophenolate Mofetil According to Study

Study	Δ in Prednisone	Muscle Manifestations	Pulmonary Manifestations	Skin Manifestations	Adverse Events (# patients)
Caramaschi et al Case report 1PM	40% ↓	CK ↓	N/A	N/A	None
Edge et al Open label trial 12 DM	94% ↓	Strength ↑ in 11	N/A	Improved in 10	Breast CA (1); Lymphoma (1); Cytopenia (2); Nausea (1) Stopped (2)
Rowin et al Case Series 10 DM	90 % ↓ 6 weaned	Strength ↑ in 5	N/A	N/A	Opportunistic Infections (3) Stopped (3)
Schneider et al Case Report 1 PM	100% ↓	Strength ↑	N/A	N/A	None
Gelber et al Case Series 4 DM	57% ↓	CK ↓ Strength ↑	N/A	Improved	Gastrointestinal upset (1) Stopped (1)
Tausche et al Case Series 4 DM	50% ↓	CK ↓ in 3 Strength ↑ in 3	N/A	Improved in 3	None
Bandelier et al Case Series 2 PM	50% ↑ 83% ↓	CK ↓ (90%)	N/A	N/A	Active colitis (1) Stopped X 4 mos (1)
Tsuchiya et al Case Report 1 DM	66 % ↓	N/A	Resolution of Ground Glass Opacities (31 wks) Resolution of exertional dyspnea	N/A	CMV (1)
Cozzani et al Case Report 1 DM	N/A	N/A	↓ in pneumomediastinum and parenchymal abnormalities (6 wks)	N/A	None
Danieli et al Open Label Trial 4 PM; 3 DM	87% ↓	CK ↓ (92%) Strength ↑ (39%)	N/A	Improved	Nausea (2) Headache (1)
Fischer et al Case Series 32 PM/DM	75% ↓	N/A	9.7% ↑ Forced Vital Capacity (FVC) at 156 wks (p=0.04) “similar trend DLCO” (Diffusing Lung Capacity)	N/A	Unknown
Majithia et al Case Series 3 PM; 4 DM	48% ↓	CK ↓ 88% Strength ↑	N/A	Improved	Cytopenia(1);Stopped(1) Death (1)
Marie et al Case Series 6 PM/DM	Unknown	N/A	“Improved” CT in 3 “Improved” PFT (Pulmonary Function Test) in 5	N/A	None
Mira-Avendano et al Case Series 9 PM/DM	75% ↓	N/A	No Δ in FVC after 12 mos 5% ↑ DLCO (p=0.17) 80% improved MMRC score (Modified Medical Research Council Dyspnea Scale)	N/A	Non-specific (4)
Morganroth et al Case Series 4 DM	73% ↓ 3 weaned	CK ↓ Strength ↑	Improved CT scan in 1 Improved DLCO and FVC in 4 Improved Total Lung Capacity in 2 Resolution of dyspnea in 3	Improved in 3	Tinea pedis (1)
Saketoo et. al Case Series 2 PM	98% ↓	CK ↓ Strength ↑	CT stable in 2 PFT ↓ in 1; stable in 1 Resolution of dyspnea in 2	N/A	Diarrhea (1)
Hervier et. al Case Report 1 DM	Slowly Withdrawn	CK ↓	“Stable improvement” PFT’s	Improved	None
Pisoni et. al Case Series 6 PM/DM	38% ↓	CK ↓ 69% Strength ↑	N/A	N/A	Nausea & Headache(2)

Disclosure: J. Roberts, None; S. O. Keeling, None.

To cite this abstract in AMA style:

Roberts J, Keeling SO. Mycophenolate Mofetil As a Steroid Sparing Agent in Polymyositis and Dermatomyositis: a Systematic Review of the Literature [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/mycophenolate-mofetil-as-a-steroid-sparing-agent-in-polymyositis-and-dermatomyositis-a-systematic-review-of-the-literature/. Accessed .

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