The purpose of this paper is to explore the effect of low-dose IL-2 on refractory dermatomyositis and immunological indexes.
A randomized, double-blind, placebo-controlled, multicenter clinical trial was designed. Patients were treated with low-dose IL-2 regularly to explore its efficacy and safety. The improvement of clinical and laboratory indexes was evaluated. Changes of immune cell subsets and cytokines were monitored.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
240
low dose interleukin-2 injected subcutaneously, at a dose of 1 x 10\~6 IU once every other day, for 6 months.
Peking university people's hospital
Beijing, Beijing Municipality, China
Proportion of subjects achieving minimal improvement (TIS≥20).
The primary outcome will be to compare the proportion of subjects achieving minimal improvement (TIS≥20). The TIS (total improvement score) is the sum of all 6 improvement scores associated with the change in each core set measure. A total improvement score of ≥20 represents minimal improvement, a score of ≥40 represents moderate improvement, and a score of ≥60 represents major improvement.
Time frame: week 12
MMT-8 (Manual Muscle Testing), (potential score 0 - 80);
MMT-8 is a set of 8 designated muscles tested unilaterally; test on right side (use left side if right side cannot be tested). Higher scores mean a better outcome.
Time frame: week12 and 24
CDASI activity score (cutaneous dermatomyositis disease area and severity index), (potential score 0-100 for cutaneous dermatomyositis disease area and 0-32 for severity index);
The CDASI is a clinician-scored single page instrument that separately measures activity and damage in the skin of DM patients for use in clinical practice or clinical/therapeutic studies. Higher scores mean a worse outcome.
Time frame: week12 and 24
Physician's Global Disease Activity VAS, (potential score 0 - 10);
Physician's Global Disease Activity (10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Higher scores mean a worse outcome.
Time frame: week12 and 24
Patient's Global Disease Activity VAS, (potential score 0 - 80);
Patient's Global Disease Activity (10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Higher scores mean a worse outcome.
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Time frame: week 12 and 24
Health assessment question, (potential score 0 - 3);
Patients reported how their illness affects their ability to function in daily life , Higher scores mean a better outcome.
Time frame: week 12 and 24
Myositis disease activity assessment tool (MDAAT) - 2005, VERSION 2
This is a combined tool that captures the physician's assessment of disease activity of various organ systems using (1) the 0-4 scale described below and (2) a visual analog scale (VAS) \[potential score 0 - 10\]. Please assess the clinical features (items 1-26) of each organ system. Higher scores mean a worse outcome.
Time frame: week 12 and 24
CD4 T cells
number and proportion of CD4 T cells in peripheral blood.
Time frame: week 12 and 24
Serum cytokines
concentration of serum cytokines
Time frame: week 12 and 24
glucocorticoid dosage
Daily dosage of glucocorticoid
Time frame: week 12 and 24
Rate of Participants with adverse effects associated with experimental drugs
Adverse effects include fever, rash, abnormal liver function, rate of new-onset infection and any abnormal measures associated with low-dose IL-2 therapy.
Time frame: up to 24 weeks
Proportion of subjects meeting the definition of improvement (DOI)
The DOI for this trial is a composite utilizing the six CSM: 3 of 6 CSM improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (a worsening measure cannot be the MMT).
Time frame: week12 and 24
Number of subjects achieving minimal improvement (TIS≥20).
The primary outcome will be to compare the proportion of subjects achieving minimal improvement (TIS≥20). The TIS (total improvement score) is the sum of all 6 improvement scores associated with the change in each core set measure. A total improvement score of ≥20 represents minimal improvement, a score of ≥40 represents moderate improvement, and a score of ≥60 represents major improvement.
Time frame: week 24