A Potential New Treatment for Severe Discoid Lupus
- Efalizumab in the Treatment of Discoid Lupus Erythematosus
Archives of Dermatology, Volume 143, Number 7, July 2007, pp. 873-877
What is the topic?
Discoid lupus is a form of lupus that affects the skin (cutaneous lupus). In most cases the discoid lupus rash appears on the face, neck, or scalp, though it can also show up on other areas of the skin. Severe discoid lupus may result in scarring. The treatments that are used most often for severe discoid lupus are strong immunosuppressants that may have significant side effects, especially when used over long periods of time.
What did the researchers hope to learn?
Efalizumab (trade name, Raptiva™) is a kind of medication called a monoclonal antibody (often abbreviated mAb). The drug works by interfering with the function of overactive immune cells that are causing disease activity. Raptiva has been approved for use in psoriasis, which, like lupus, is an autoimmune disease. The researchers in this study wanted to see if Raptiva could be effective in treating discoid lupus.
Who was studied?
The researchers reviewed the cases of 13 adult patients with severe, persistent discoid lupus who were treated with Raptiva on an experimental basis at a clinic in Leeds, England. Nine of the patients were women; four were men; they ranged in age from 32 to 66. The main sites of the discoid lupus lesions in these patients were the face and the scalp. Nine of the patients tested positive for ANA antibodies.
None of the patients had had improvements in their lesions from any previous treatments, which included steroids, antimalarials, dapsone, azathioprine, cyclosporine, and methotrexate. Each of the patients then received weekly injections of Raptiva, with the doses adjusted to their weight (1mg/kg). The average period of treatment was 14.5 months. Blood tests were taken once a month for the first three months of their Raptiva treatment, then every three months after that. The discoid rashes were assessed using a special scoring tool that measures the extent of the area covered by the rash and the thickness of the lesions.
How was the study conducted?
The researchers looked at the records of the patients from the time they began their Raptiva treatments. However, they did not compare the treatment group to other discoid lupus patients who did not receive Raptiva.
What did the researchers find?
The researchers found that 12 of the 13 patients who received Raptiva responded well, with 11 showing improvement rated as very good or excellent. Also, these improvements occurred rapidly, with the average response to treatment being 5 ½ weeks.
Two of the patients developed new discoid lupus lesions after showing improvement with Raptiva and had to take other medications; with the addition of these other medicines, however, the patients once again improved, and were able to remain symptom-free with Raptiva and the second medicine. The researchers pointed out that when treated with these medications alone, before Raptiva, neither of these patients had responded.
Several of the patients experienced side effects. One patient developed an infection, two had skin disease flares, and one developed a rash. Five of the 12 patients who responded to Raptiva had joint pain (arthralgia). Two of those patients had a history of joint pain before they began Raptiva; in two others, the joint pain went away quickly after a short course of steroids; the fifth had to take other medications for a longer period of time.
What were the limitations of the study?
This study only involved 13 patients, and the length of time they had been treated with Raptiva was relatively brief, 20 months being the longest time any patient was followed in this study. Given the fact that lupus is a chronic condition that can require medical treatment for a lifetime, there is no way to know if the improvements observed in these patients taking Raptiva will last over time or if other side effects may develop. In addition to the joint pain, which occurred in some of the patients in this study, there are also reports that Raptiva can cause low platelet counts (platelets are important for normal blood clotting).
What do the results mean for you?
This study suggests that Raptiva may be a useful treatment for people with discoid lupus, especially those who have severe disease that has not responded to other medications. However, studies with much larger numbers of patients, from different ethnic backgrounds, and with long-term follow-up periods, will need to be conducted before the positive results observed in this small study can be confirmed. Also this is an early report of findings in patients who were treated in a clinic. It is not a scientific study with a comparison group of patients who do not receive the treatment, and where the study is "blinded" so that the patients and the physicians evaluating them would not know if they received Raptiva or not. Early reports such as this one do provide a good basis for further studies that are set up in a more scientific manner.