麻豆果冻传媒影音

In a small study of adolescent patients with systemic lupus erythematosus (SLE), anifrolumab (Saphnelo) led to significant and sustained improvements in cutaneous lupus. Results appear in .

Seven adolescent patients with SLE (6 females; median age 17 [range, 14-20 years]) received 300 mg of intravenous anifrolumab every four weeks. All had discoid lupus erythematosus that had resisted standard therapies; participants had an average of 4 and as many as 11 prior systemic treatments.

All patients demonstrated substantial improvement in cutaneous disease activity after initiating anifrolumab. Mean decrease and percentage decrease in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI-A) scores were 18.0 (8.9) and 72.1% (9.4%), respectively, after one month (P=.01), with improvements sustained to the 6-month mark.

There is currently no FDA-approved medication for cutaneous lupus. Belimumab (Benlysta), approved by the FDA in 2011 for SLE treatment, has been demonstrated effective and is often used to treat cutaneous lupus.

First study author, Katharina Shaw, MD, is a dermatologist-researcher with the Perelman School of Medicine at the University of Pennsylvania. Her exchange with the Reading Room has been edited for length and clarity.

What was the impetus for undertaking this study?

Shaw: The 10,000-foot view is that for decades we've had very minimal advances in the treatment of cutaneous lupus, both in adolescents and adults. There's been one FDA-approved medication prior to anifrolumab, not for cutaneous lupus but for systemic lupus, and that was belimumab. Beyond that, there have been really no advances in the past several decades.

Anifrolumab was approved for systemic lupus back in 2021, and what we learned is that adults who had really bad skin disease did extremely well. When we saw these really promising results in adults, we were curious if the same kind of efficacy would translate to pediatric or adolescent patients.

We're always confronted with patients, adolescent and otherwise, who have reached the end of their therapeutic algorithm. And so the impetus for this research letter was to describe early results in adolescent patients with refractory cutaneous lupus in the setting of their systemic lupus who were treated with anifrolumab.

Would you describe your findings?

Shaw: They're really promising. These patients didn't just get a little bit better. Their skin disease got a lot better quickly.

Everything has to be taken with a grain of salt when you're describing a small number of patients with a very specific disease phenotype, by which I mean all these patients had really bad systemic lupus and pretty bad cutaneous lupus. But in these 7 patients, the results were really striking for two reasons.

First, it was almost like a flip switched in the sense that within just a few days or weeks of getting their first infusion of anifrolumab, their skin just calmed down, which is very notable when we talk about anything related to cutaneous lupus. Our current armamentarium relies on traditional immunosuppressants like methotrexate or mycophenolate, which take months to kick in.

That delay can be really problematic for a condition that can cause a lot of scarring, which discoid lupus in particular does. And so, to have a medication like anifrolumab be able to seemingly shut down cutaneous disease activity within days to weeks is really striking.

Second, something really notable was the degree of improvement that the patients had. Not only did they have quick responses in their skin when we use validated measures of cutaneous lupus disease activity, but there also were striking decreases in overall disease activity.

Based on these results, do you have any clinical advice or suggestions for dermatologists ?

Shaw: I think the recommendation is that anifrolumab should be considered in a patient with severe and refractory cutaneous lupus after traditional first-, second-, and third-line measures have either failed or been deemed not appropriate.

You can't say at this stage that anyone should consider anifrolumab earlier in a patient's treatment scheme because we just don't have the safety data to support that. At the same time, clinicians should have this in their back pocket, adding it to their therapeutic armamentarium when considering options for adolescent patients with severe refractory cutaneous disease.

Looking ahead, what's next for investigations into anifrolumab, particularly in the context of cutaneous lupus?

Shaw: I think it's important to remember that there still is no FDA-approved medication for cutaneous lupus. So the obvious next step is to obtain an FDA-approved medication for cutaneous lupus. There are ongoing phase 2 and 3 trials, including a planned phase 3 trial of anifrolumab for cutaneous lupus specifically. It hasn't started recruiting, but it is in the works.

So I think the future is bright, so to speak. There is an immense need to have effective and quick therapeutic options for patients with cutaneous lupus.

We finally have a lot of interest in the medical community and pharmaceutical industry to get an FDA-approved medication for cutaneous lupus. There's a lot of emphasis right now in dermatology to recognize the psychosocial impact of disfiguring skin diseases. Much has been written about acne and vitiligo, but cutaneous lupus, when left untreated or when it's poorly treated, can also be devastating. These are severely dispigmenting and scarring processes that can occur.

So, to be able to finally add a medication, although its treatment or its use is off-label at this point, is a real game-changer.