Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis

Study Overview
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Brief Summary
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The aim of the study is to compare the effectiveness of commonly used immunosuppressant treatments for early diffuse cutaneous systemic sclerosis (SSc).

Detailed Description
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Diffuse cutaneous systemic sclerosis (SSc) is a rare autoimmune disease characterised by excessive connective tissue in the skin (causing skin thickening called 'scleroderma'), muscle, joints and internal organs. A number of different drugs with effects on the immune system (known as 'immunosuppressants') are currently being used by clinicians in the treatment of early diffuse cutaneous SSc, but all can have significant side effects. We want to know whether any one is definitely effective and, if so, which is the most effective.

Official Title
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Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis

Conditions
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Early Diffuse Cutaneous Systemic Sclerosis

Intervention / Treatment
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* Drug: Methotrexate
* Drug: Mycophenolate mofetil
* Drug: Cyclophosphamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age > 18 Skin thickening < 3 years Immunosuppressant use < 4 months Proximal skin involvement to face/neck, elbow or knee Exclusion Criteria: Previous use of more than 4 months of methotrexate, mycophenolate mofetil, cyclophosphamide or other immunosuppressive treatments Previous use of immunosuppressant therapy other than methotrexate, mycophenolate mofetil or cyclophosphamide within previous months Previous stem cell transplantation therapy

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Methotrexate<br>Patients treated with Methotrexate at the entry of the study. | Drug: Methotrexate<br>* Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study, either oral or subcutaneous with a target dose of 20-25mg weekly.<br>|
| Mycophenolate Mofetil<br>Patients treated with Mycophenolate Mofetil at the entry of the study. | Drug: Mycophenolate mofetil<br>* Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study,recommended dose 500 mg twice daily for 2 weeks increasing to 1gm twice daily.<br>|
| Cyclophosphamide<br>Patients treated with Cyclophosphamide at the entry of the study | Drug: Cyclophosphamide<br>* Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study, recommend use (i) IV. Minimum monthly dose 500mg/m2 with a recommended duration of 6-12 months. (ii) Oral. 1-2mg/day with a recommended duration of 12 months.<br>|
| No Immunosuppressant<br>Patients without immunosuppressant treatment at the entry of the study | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Skin score as measured by modified rodnan skin score |  | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pulmonary function as measured by right ventricular systolic pressure (RVSP) or pulmonary artery pressure (PAP) |  | 24 months | 
| Echocardiographic findings |  | 24 months | 
| Cochin hand function |  | 24 months | 
| Fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT) scale |  | 24 months | 
| Short form 36 questionnaire (SF-36) |  | 24 months | 
| Scleroderma Health Assessment Questionnaire |  | 24 months | 
| Haemoglobin |  | 24 months | 
| Erythrocyte sedimentation rate |  | 24 months | 
| Estimated glomerular filtration rate (eGFR) |  | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Systemic sclerosis