This study is being conducted to evaluate the safety and effectiveness of lenalidomide (Revlimid®) in subjects with Cutaneous Lupus Erythematosus (CLE). The study drug will be used in an off-label indication to treat 6 subjects for 12 months each. Men and women over the age of 18, who have a biopsy proven diagnosis of CLE and who have failed standard treatment, will be included in the study.
Cutaneous lupus erythematosus (CLE) is a chronic and often disabling disease which affects the skin. Many patients experience scarring and inflammation of the skin, which often occur on the face. Moderate to severe CLE is most frequently treated with antimalarial drugs such as hydroxychloroquine, quinacrine or chloroquine. Up to 70% of CLE patients treated with antimalarials experience a beneficial clinical response, while the remainder of patients show no response or continue to experience progression of the disease. Thalidomide has been used successfully in such patients, with up to 75% clinical response rate in refractory CLE patients. However, thalidomide is a known teratogen and can cause severe birth defects, including short, malformed limbs and damage to peripheral nerves in the extremities, requiring patients to be monitored for pregnancy. In addition, up to 25% of patients on thalidomide develop peripheral neuropathy. A new drug, lenalidomide (REVLIMID®), an analogue of thalidomide, has been developed to treat neoplastic and inflammatory conditions, including various oncologic conditions such as multiple myeloma, myelodysplastic syndrome and solid tumors. Unlike thalidomide, lenalidomide (REVLIMID®) is not known to cause the extent of serious side effects caused by thalidomide; however, it must also be monitored for side effects and be distributed under the RevAssist program authorized by the drug manufacturer, Celgene Corporation. The primary goal of this investigator-initiated, small pilot study is to evaluate the safety and effectiveness of lenalidomide (REVLIMID®) in CLE subjects using measurements such as the CLASI (Cutaneous Lupus Activity and Severity Index). The study drug will be used in an off-label indication to treat 6 subjects, for whom lenalidomide (REVLIMID®) will be provided at no cost by the drug manufacturer. Men and women over the age of 18, who have a biopsy proven diagnosis of refractory CLE and who have failed standard treatment with hydroxychloroquine for up to three months, will be included in the study. Secondarily, the study will evaluate the biologic effects of lenalidomide on pathogenic and immunologic mechanisms of the CLE disease process during the treatment period by collecting skin specimens (biopsies) and blood samples.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Hospital of the University of Pennsylvania, Department of Dermatology
Philadelphia, Pennsylvania, United States
Cutaneous Lupus Area and Severity Index (CLASI)
The Cutaneous Lupus Area and Severity Index (CLASI); range of disease activity is 0-70. Lower scores reflect less activity.
Time frame: Weeks 0 through 52
Number of Participants With Change in IFN and CD4 Levels at 6 Weeks
CXCL10, an interferon-inducible chemokine, and immunophenotyping by immunostaining. Measurement of interferon-inducible genes from peripheral blood mononuclear cells before and after treatment.
Time frame: 6 weeks
Physician Global Assessment (PGA) for Skin
General skin scores were recorded on a 10 cm visual analogue scale at each visit. At each visit on scale 0-10, 0 corresponding to worst skin condition imaginable and 10 to perfect health.
Time frame: Weeks 0 through 52
Patient General Assessment (PtGA) for Skin
General skin scores were recorded by the patient on a 10 cm visual analogue scale at each visit by the patient. At each visit on scale 0-10, 0 corresponding to worst skin condition imaginable and 10 to perfect health.
Time frame: Weeks 0 through 52
Pain in Skin
Pain in skin was recorded on a 10 cm visual analogue scale at each visit by the patient. At each visit on scale 0-10, 0 corresponding to no pain and 10 to pain as bad as you can imagine.
Time frame: Weeks 0 through 52
Itch in Skin
Itch scores were recorded on a 10 cm visual analogue scale at each visit by the patient. At each visit on scale 0-10, 0 corresponding to no itch and 10 to itch as bad as you can imagine.
Time frame: Weeks 0 through 52
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Fatigue
Fatigue scores were recorded on a 10 cm visual analogue scale at each visit by the patient. At each visit on scale 0-10, 0 corresponding to no fatigure and 10 to fatigue as bad as you can imagine.
Time frame: Weeks 0 through 52
Skindex Symptoms
Skindex symptoms scores are scored 1-5 and then normalized to 100, with a higher score corresponding to a worse impression. The absence of symptom impact on QoL is scored as "20" (this is represented as a 1 on the 1-5 scale. The worst impact of symptoms on QoL is 100. This is represented as a 5 on the 1-5 scale.
Time frame: Weeks 0 through 52
Skindex Function
Skindex function scores are scored 1-5 and then normalized to 100, with a higher score corresponding to a worse impression. The absence of function impact on QoL is scored as "20" (this is represented as a 1 on the 1-5 scale. The worst impact of function on QoL is 100. This is represented as a 5 on the 1-5 scale.
Time frame: Weeks 0 through 52