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Rare Diseases Update

Featuring Ruth Ann Vleugels, MD |

Heidi and Scott C. Schuster Distinguished Chair in Dermatology 
Brigham and Women's Hospital  
Boston, MA

| Published February 22, 2024

Ruth Ann Vleugels, MD, took us through a review of rarer dermatologic conditions and new therapeutics to be aware of when treating these patients. Starting with DLE, Dr Vleugels reviewed 2 cases that had exhausted the typical treatment ladder from photoprotection through antimalarials and oral steroids and finally to IVIG or rituximab. She presented a promising new drug, anifrolumab, an anti-type I IFN receptor monoclonal antibody, which has recently garnered approval for SLE. However, in the TULIP-2 trial, it showed promising results for CLE as well in patients who had tried and failed many other systemic medications, indicating this may play a role earlier in the treatment algorithm than systemics with more side effects like rituximab. Not only did mucocutaneous lesions show vast improvement, but almost half of patients had more than a 50% reduction in CLASI score, a measurement of cutaneous lupus activity, compared to 25% on placebo (total n=89). Dosing is 300-mg IV every 4 weeks, which has also been successfully used in adolescent studies. 

She then moved on to reviewing a few different options for dermatomyositis, including IVIG, which demonstrates superiority from placebo within 4 weeks and is now FDA approved for the condition, and tofacitinib, a JAK inhibitor. Larger randomized controlled trials are required to demonstrate efficacy of JAK inhibitors for dermatomyositis, but open-label pilot studies and retrospective studies have shown promising results. Given the strong type 1 interferon signature in dermatomyositis, JAK inhibitors may be a viable option to interrupt this pathway. Dazukibart, a monoclonal antibody directed against IFN-beta, is currently undergoing phase 3 trials for dermatomyositis. Anifrolumab also demonstrates evidence in the treatment of this disease. 

Lastly, Dr Vleugels reviewed the use of tofacitinib for cutaneous sarcoidosis. JAK inhibitors may be especially useful in treating overlapping autoimmune conditions. 


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