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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Methotrexate | Patients treated with Methotrexate at the entry of the study. |
| |
| Mycophenolate Mofetil | Patients treated with Mycophenolate Mofetil at the entry of the study. |
| |
| Cyclophosphamide | Patients treated with Cyclophosphamide at the entry of the study |
| |
| No Immunosuppressant | Patients without immunosuppressant treatment at the entry of the study |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Methotrexate | Drug | Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study, either oral or subcutaneous with a target dose of 20-25mg weekly. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Skin score as measured by modified rodnan skin score | 24 months |
| 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 | |
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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
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Eligibility:
Age > 18 Skin thickening < 3 years Immunosuppressant use < 4 months Proximal skin involvement to face/neck, elbow or knee
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| Name | Affiliation | Role |
|---|---|---|
| Ariane Herrick, MD, phD | University of Manchester | Principal Investigator |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29306872 | Derived | Herrick AL, Peytrignet S, Lunt M, Pan X, Hesselstrand R, Mouthon L, Silman AJ, Dinsdale G, Brown E, Czirjak L, Distler JHW, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk MC, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jobanputra P, Jordan AC, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov NS, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, MacGregor AJ, McHugh N, Muller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, McLaren JS, van Laar JM, Pathare S, Proudman SM, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad GS, Denton CP. Patterns and predictors of skin score change in early diffuse systemic sclerosis from the European Scleroderma Observational Study. Ann Rheum Dis. 2018 Apr;77(4):563-570. doi: 10.1136/annrheumdis-2017-211912. Epub 2018 Jan 6. | |
| 28188239 |
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| ID | Term |
|---|---|
| D045743 | Scleroderma, Diffuse |
| D012595 | Scleroderma, Systemic |
| ID | Term |
|---|---|
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
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| ID | Term |
|---|---|
| D008727 | Methotrexate |
| D009173 | Mycophenolic Acid |
| D003520 | Cyclophosphamide |
| ID | Term |
|---|---|
| D000630 | Aminopterin |
| D011622 | Pterins |
| D011621 | Pteridines |
| D006574 | Heterocyclic Compounds, 2-Ring |
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| Mycophenolate mofetil | Drug | 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. |
|
| Cyclophosphamide | Drug | 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. |
|
| 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 |
| Derived |
| Herrick AL, Pan X, Peytrignet S, Lunt M, Hesselstrand R, Mouthon L, Silman A, Brown E, Czirjak L, Distler JHW, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk M, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jordan AC, Jobanputra P, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov N, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, MacGregor AJ, McHugh N, Muller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, McLaren JS, van Laar JM, Pathare S, Proudman S, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad GS, Denton CP. Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Ann Rheum Dis. 2017 Jul;76(7):1207-1218. doi: 10.1136/annrheumdis-2016-210503. Epub 2017 Feb 10. |
| D000072471 |
| Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |