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.

Scientists Are Teaching the Body to Accept New Organs. Patients receiving new kidneys and livers must take damaging anti–rejection drugs for the rest of their lives, but now researchers hope to train the immune system instead of just tamping it down. New York Times, 01/22/2019. (Also see Dendritic Cells)

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, 2016 Feb;16(1):55-60. (Also see Stem Cell (Bone Marrow) Transplantation and Scleroderma Treatments – General)

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.


Treatment of Antineutrophil Cytoplasmic Antibody-Associated Vasculitis (AAV): Is There Still a Role for Cyclophosphamide (CYC)? With the emergence of newer agents with more favorable safety profiles, CYC is no longer the cornerstone of management of AAV. PubMed, Rheum Dis Clin North Am, 2019 Aug;45(3):379-398. (Also see Vasculitis)

Long–term effects of immunosuppressive therapy on lung function in scleroderma patients. Cyclophosphamide does not appear to result in clinically significant improvement of pulmonary function but fulfilled criteria of stable disease. PubMed, Clin Rheumatol, 2018 Nov;37(11):3043-3050. (Also see Treatments for Pulmonary Fibrosis)

Case Report: Successful treatment of cerebral large vessel vasculitis in systemic lupus erythematosus with intravenous pulse cyclophosphamide. Here, we present a rare case of cerebral large vessel vasculitis treated successfully with a clear visual presentation. PubMed, Lupus. (Also see Vasculitis and Symptoms and Complications of Lupus)

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. (Also see Raynaud's Treatments)

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)

Predictors of disease activity and structural progression after treatment with adalimumab plus methotrexate or continued methotrexate monotherapy in patients with early rheumatoid arthritis. Findings suggested a relationship between the addition of adalimumab at 6 months after methotrexate failure and improved outcomes. PubMed, Ann Rheum Dis, 08/03/2018. (Also see Treatments for Rheumatoid Arthritis)

Comparing ultraviolet light A photo(chemo)therapy with Methotrexate (MTX) protocol in childhood localized scleroderma: Evidence from systematic review and meta–analysis approach. Our study supports the combination of MTX and glucocorticoids in patients with a high risk of complication and UVA1 could represent a therapeutic option in patients with limited scleroderma. Seminars in Arthritis and Rheumatism, 03/30/2018. (Also see Morphea Scleroderma Treatments)

Thymidylate synthase (TYMS) polymorphisms and responsiveness to or toxicity of methotrexate (MTX) in rheumatoid arthritis (RA). This meta–analysis demonstrates that the TYMS 2R/3R and 6 bp I/D polymorphisms may not be associated with non–responsiveness to or toxicity of MTX therapy in RA patients. PubMed, Z Rheumatol, 01/29/2018. (Also see Medications for Rheumatoid Arthritis)

Eosinophilic Fasciitis (EF): an Updated Review on Diagnosis and Treatment. Methotrexate has emerged as the leading corticosteroid–sparing agent for EF. PubMed, Curr Rheumatol Rep, 2017 Nov 4;19(12):74. (Also see Eosinophilic Fasciitis)

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)

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.

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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