Medications for Scleroderma, Arthritis, Autoimmune and Rheumatic Diseases

Author: Janey Willis. Scleroderma is highly variable. See Types of Scleroderma. Read Disclaimer
Azathioprine (Imuran)
Mycophenolate mofetil (Cellcept)
Chemo Brain


Immunosuppressants are medications that suppress or prevent the immune response. (Also see What is Scleroderma?, Medical Overview, and Medications for Scleroderma, Arthritis, Autoimmune and Rheumatic Diseases)

Immunosuppressants are used to prevent rejection of a transplanted organ and to treat autoimmune diseases such as scleroderma, psoriasis, rheumatoid arthritis, and Crohn's disease. They are also referred to as immunodepressants.

Some immunosuppressants are chemotherapy drugs; however, when used to treat scleroderma and some other autoimmine diseases the dosage is lower than when used in chemotherapy to treat cancer or organ transplant patients.

Immunosuppressants fall under a category of drugs referred to as DMARDs (Disease-Modifying Anti-Rheumatic Drugs). Immunosuppressants commonly used for scleroderma and rheumatic diseases include methotrexate, azathioprine (Imuran), cyclosporine, mycophenolate mofetil (Cellcept), and cyclophosphamide. As with any drugs, immunosuppressants can have serious side effects and risks.

Treatment outcome in early diffuse cutaneous systemic sclerosis (dcSSc): the European Scleroderma Observational Study (ESOS). 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. PubMed, Ann Rheum Dis, 02/10/2017.

Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis–related interstitial lung disease. Findings suggest that intermediate–term changes in CXCL4 may have predictive significance for long–term progression of SSc–ILD in patients receiving immunosuppressive therapy. PubMed, Arthritis Res Ther, 2016 Dec 30;18(1):305. (Also see Treatments for Pulmonary Fibrosis)

Case Report: Type B insulin resistance syndrome with Scleroderma successfully treated with multiple immune suppressants after eradication of Helicobacter pylori infection. The present case suggests H pylori infection–related pathological mechanism may contribute to type B insulin resistance syndrome and autoimmune disorders. BioMed Central, BMC Endocrine Disorders.

Risk of high–grade cervical dysplasia and cervical cancer in women with systemic lupus erythematosus (SLE) receiving immunosuppressive drugs. Among women with SLE, immunosuppressive drugs may be associated with a greater, albeit not statistically significant, risk of high–grade cervical dysplasia and cervical cancer compared to patients receiving hydroxychloroquine alone. PubMed, Lupus, 10/31/2016. (Also see Treatments for Lupus and Cancer)

Advances in pathogenesis and treatment of systemic sclerosis. Immunosuppression is now established as a beneficial approach but balancing risk and benefit is vital, especially for powerful approaches such as autologous stem cell transplantation. PubMed, Clin Med, 2015 Dec;15 Suppl 6:s58-63. (Also see Stem Cell (Bone Marrow) Transplantation and Scleroderma Treatments – General)

Assessment of Risks of Pulmonary Infection During 12 Months Following Immunosuppressive Treatment for Active Connective Tissue Diseases: A Large-scale Prospective Cohort Study. Physicians should be aware of the higher risks for corticosteroids of pulmonary infection than other immunosuppressants and assess these risk factors before immunosuppressive treatment. PubMed, J Rheumatol, 02/01/2015. (Also see Steroids)

Periodontitis treatment improves systemic lupus erythematosus response to immunosuppressive therapy. Periodontal disease treatment seems to have a beneficial effect in controlling disease activity in SLE patients under immunosuppressive therapy and therefore, management of this modifiable risk factor is recommended. PubMed, Clin Rheumatol, 2014 Jan 11. (Also see Systemic Lupus Erythematosus Treatments)

Therapeutic Plasma Exchange (TPE) TPE removes large–molecular–weight substances such as harmful antibodies from the plasma and is normally combined with disease modifying treatment, such as immunosuppressive drugs, for the underlying condition. JPAC. (Also see Therapeutic Plasma Exchange)

Immunosuppressants - clinical applications. There are now over 80 autoimmune diseases and several common allergic conditions in which immunosuppressants could play a role although they may not be life-saving. Australian Prescriber.

Scleroderma Treatments and Clinical Trials ISN.

Azathioprine (Imuran)

Azathioprine (Imuran). Patient Education. American College of Rheumatology.

Azathioprine (AZA) Maintenance Therapy to Prevent Relapses in Autoimmune Pancreatitis (AIP). AZA is an effective and safe treatment to prevent AIP relapses. PubMed, Clin Transl Gastroenterol, 2017 Apr 27;8(4):e90. (Also see Pancreatitis, Autoimmune)

Azathioprine response in patients with fibrotic connective tissue disease-associated interstitial lung disease. A significant minority of patients could not tolerate azathioprine due to non–respiratory side effects. It was associated with stability in pulmonary function and a marginal improvement. PubMed, Respir Med, 2016 Dec;121:117-122.


Fewer Mycophenolate Adverse Events in Scleroderma Lung Study. Mycophenolate mofetil can be substituted for cyclophosphamide for the immunosuppressive treatment of scleroderma-related interstitial lung disease and might even be safer. Medscape, 11/02/2015. (Also see Pulmonary Fibrosis Treatments and Clinical Trials)

Systemic Sclerosis(SSc) Sera Impair Angiogenic Performance of Dermal Microvascular Endothelial Cells: Therapeutic Implications of Cyclophosphamide (CYC). In SSc, CYC treatment might boost angiogenesis and consequently improve peripheral microangiopathy. PLOS One, 06/15/2015. (Also see Raynaud's Treatments)

Low-dose pulse cyclophosphamide (CYC) in interstitial lung disease associated with systemic sclerosis (SSc-ILD): Efficacy of maintenance immunosuppression in responders and non-responders. The study supports the use of low-dose pulse CYC as induction therapy of recently deteriorated SSc-ILD. Moreover, it suggests that azathioprine should be administered to CYC-responsive patients but does not show any definite effect of micophenolic acid in unresponsive patients. ScienceDirect, 09/08/2014.

Cyclophosphamide Clinical Trials. ISN.

Scleroderma Lung Study II. ISN.

Results from the Scleroderma Lung Study I. This study showed that 1 year of oral cyclophosphamide (CYC) was effective in improving lung function, symptoms of shortness of breath, and quality of life but the effect on lung function and quality of life only lasted for another 6 months after CYC was stopped. UCLA.

Cyclophosphmide. Patient Education. Chemocare.


Cyclosporine (Neoral, Sandimmune, Gengraf).


Methotrexate Clinical Trials. ISN.

Methotrexate. Patient Education. American College of Rheumatology.

The Drug-Induced Repiratory Disease Website. List of over a hundred medications that can cause lung disease or symptoms and also shows the associated patterns. Pneumotox. (Also see Pulmonary Involvement)

Inadequate response or intolerability to oral methotrexate (MTX): Is it optimal to switch to subcutaneous (SC) methotrexate prior to considering therapy with biologics? SC MTX can prove to be more efficacious in patients refractory to oral MTX therapy or in patients experiencing severe gastrointestinal adverse effects. PubMed, Rheumatol Int, 03/02/2016. (Also see Treatments for Rheumatoid Arthritis)

Eosinophilic fasciitis (EF): clinical characteristics and response to methotrexate (MTX). MTX represents an effective treatment option for EF although the rarity of this disease would make a double-blind controlled trial study difficult to perform. PubMed, Int J Rheum Dis, 2015 Jan;18(1):91-8.

Three decades of low-dose methotrexate in rheumatoid arthritis: Can we predict toxicity? A summary of current data on low-dose MTX-associated toxicity, its prevention and predictors, keeping in mind practical RA clinical care. PubMed, Immunol Res, 2014 Nov 13. (Also see Medications for Rheumatoid Arthritis)

Methotrexate (MTX) in systemic lupus erythematosus (SLE). The use of MTX is associated with significant reductions in SLE Disease Activity Index and the average dose of corticosteroids in adult patients with SLE. PubMed, Lupus, 2014 Jan 7. (Also see Systemic Lupus Erythematosus Treatments)

Mycophenolate mofetil (Cellcept)

Mycophenolate Mofetil (Cellcept). CellCept lowers your body's immune system. Genentech.

Efficacy of mycophenolate mofetil (MMF) and oral cyclophosphamide (CYC) on skin thickness: Post–hoc analyses from the Scleroderma Lung Study I and II. (SLS-I and II) In SLS-II, MMF and CYC resulted in improvements in the modified Rodnan skin score in diffuse cutaneous systemic sclerosis over 24 months. PubMed, Arthritis Care Res (Hoboken), 05/23/2017. (Also see Clinical Trials)

Long-term efficacy and tolerability of mycophenolate mofetil (MMF) therapy in diffuse scleroderma (dcSSc) skin disease. MMF was associated with a modest improvement in modified Rodnan skin score (mRSS) and was well tolerated in the treatment of dcSSc. PubMed, Int J Rheum Dis, 03/24/2017.

Safety and effectiveness of mycophenolate in systemic sclerosis. A systematic review. Mycophenolate-associated gastrointestinal adverse events are common in SSc, but not severe enough to preclude its use and it may be effective in improving or stabilizing interstitial lung disease and skin involvement. PubMed, PLoS One, 2015 May 1;10(5):e0124205.

Chemo Brain

Some immunosuppressants are chemotherapy drugs, but used at higher dosages than when used to treat autoimmune disease.

Chemobrain: When cancer treatment disrupts your thinking and memory, Dealing with thinking and memory problems caused by cancer or cancer treatment can be frustrating. Find out more about chemobrain and how to cope. MayoClinic.

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