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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| low-dose IL-2 | Experimental | The first stage (double-blind treatment period): One million IU of IL-2 was injected subcutaneously once every other day for 12 weeks. The second stage (open treatment period): One million IU of IL-2 was injected subcutaneously once every other day for 12 weeks. |
|
| Placebo | Placebo Comparator | The first stage (double-blind treatment period): Placebo was injected subcutaneously once every other day for 12 weeks. The second stage (open treatment period): One million IU of IL-2 was injected subcutaneously once every other day for 12 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Interleukin-2 | Drug | low dose interleukin-2 injected subcutaneously, at a dose of 1 x 10~6 IU once every other day, for 6 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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); |
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Inclusion Criteria:
Age 18-75 years old (including 18 and 75 years old);
The diagnosis of dermatomyositis conforms to Bohan/Peter Recommendation in 1975 or EULAR/ACR Classification Standard in 2017.
Active myositis was defined by baseline Manual Muscle Testing (MMT-8) no greater than 125/150 and at least two additional abnormal CSMs. To allow the enrolment of patients with active DM with a moderate to severe rash who may not meet the MMT-8 criterion noted above, patients with DM could be enrolled if their cutaneous VAS score on the Myositis Disease Activity Assessment Tool (MDAAT) was ≥3cm on the 10cm VAS scale and at least three of the five CSMs were abnormal (excluding the MMT-8).
Abnormal CSMs include:
The dose of glucocorticoid (equivalent to prednisone) was less than 0.5mg/kg/d within 4 weeks before joining the group, and/or there were no new immunosuppressants (cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, methotrexate, etc.) within 12 weeks, and the dose was stable for 4 weeks.
Voluntary signing of informed consent: When participating in the trial, the patient must be given a written notice of consent, and hope that the patient can comply with the requirements of the study follow-up plan and other protocols.
Agree to adopt effective contraceptive measures during the study period (women of childbearing age).
Exclusion Criteria:
Any subject meeting any of the following criteria should be excluded:
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| Name | Affiliation | Role |
|---|---|---|
| Zhanguo Li | Peking University People's Hospital | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking university people's hospital | Beijing | Beijing Municipality | 100044 | China |
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| ID | Term |
|---|---|
| D003882 | Dermatomyositis |
| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D007376 | Interleukin-2 |
| C082598 | aldesleukin |
| ID | Term |
|---|---|
| D007378 | Interleukins |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
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|
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. |
| 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. | 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. | 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. | 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. | week 12 and 24 |
| CD4 T cells | number and proportion of CD4 T cells in peripheral blood. | week 12 and 24 |
| Serum cytokines | concentration of serum cytokines | week 12 and 24 |
| glucocorticoid dosage | Daily dosage of glucocorticoid | 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. | 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). | 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. | week 24 |
| D009468 |
| Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D000602 |
| Amino Acids, Peptides, and Proteins |
| D008222 | Lymphokines |
| D011506 | Proteins |
| D001685 | Biological Factors |