Early Diffuse Cutaneous Systemic Sclerosis
Conditions
Keywords
Systemic sclerosis
Brief summary
The aim of the study is to compare the effectiveness of commonly used immunosuppressant treatments for early diffuse cutaneous systemic sclerosis (SSc).
Detailed description
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.
Interventions
DRUGMethotrexate
Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study, either oral or subcutaneous with a target dose of 20-25mg weekly.
DRUGMycophenolate mofetil
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.
DRUGCyclophosphamide
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.
Sponsors
University of Manchester
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Skin score as measured by modified rodnan skin score | 24 months |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| Pulmonary function as measured by right ventricular systolic pressure (RVSP) or pulmonary artery pressure (PAP) | 24 months |
| Haemoglobin | 24 months |
| Erythrocyte sedimentation rate | 24 months |
| Estimated glomerular filtration rate (eGFR) | 24 months |
| Scleroderma Health Assessment Questionnaire | 24 months |
Outcome results
None listed