Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis

University of Manchester320 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

320

Conditions

Early Diffuse Cutaneous Systemic Sclerosis

Interventions

MethotrexateDRUG

Immunosuppressant agent. Patients treated with Methotrexate at the entry of the study, either oral or subcutaneous with a target dose of 20-25mg weekly.

Mycophenolate mofetilDRUG

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.

CyclophosphamideDRUG

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.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Skin score as measured by modified rodnan skin score

Time frame: 24 months

Secondary Outcomes

Pulmonary function as measured by right ventricular systolic pressure (RVSP) or pulmonary artery pressure (PAP)

Time frame: 24 months

Echocardiographic findings

Time frame: 24 months

Cochin hand function

Time frame: 24 months

Fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT) scale

Time frame: 24 months

Short form 36 questionnaire (SF-36)

Time frame: 24 months

Scleroderma Health Assessment Questionnaire

Time frame: 24 months

Haemoglobin

Time frame: 24 months

Erythrocyte sedimentation rate

Time frame: 24 months

Estimated glomerular filtration rate (eGFR)

Time frame: 24 months

Data from ClinicalTrials.gov

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