‘A plethora of emerging treatments’ coming for idiopathic inflammatory myopathies
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SAN DIEGO — Clinical trials exploring intravenous immunoglobulin and Janus kinase inhibition headline a robust pipeline for idiopathic inflammatory myopathies, according to a speaker at the 2023 Congress of Clinical Rheumatology West.
“For the first time, we have a plethora of emerging treatments,” Lisa Christopher-Stine, MD, director of the Johns Hopkins Myositis Center, told attendees. “Boy, have treatments come a long way.”
According to Christopher-Stine, a key concern is that current treatment approaches are based more on expert opinion than on clinical trial data. Small cases series have been published, as have open-label studies, but there are few randomized, controlled trials in myositis, she said.
Christopher-Stine likened treatment of the various manifestations of inflammatory myopathies — including Raynaud’s phenomenon, skin rash, interstitial lung disease and arthritis — to the whack-a-mole arcade game. With this in mind, she stressed that treatments should be stratified by clinical phenotype.
“This is really the way we think about this in the myositis center,” she said.
For patients with mild to moderate skin-dominant disease, topical interventions should come first, according to Christopher-Stine. Specifically, topical steroids or tacrolimus have been mainstays of treatment for these patients and continue to show efficacy.
Looking into the pipeline, topical JAK inhibitors additionally may have efficacy in patients with mild skin disease, but further investigation is necessary.
“JAK inhibitors, systemically, have a strong signal, but they do not always have great penetration under the skin,” Christopher-Stine said. “So, we are TBD on that, but they are of interest.”
When skin disease is more severe, methotrexate, mycophenolate or azathioprine, in addition to steroids, have been most effective, she added.
“Mycophenolate can really take 12 to 16 weeks to show efficacy,” she said. “It is a waiting game, but it is a way to spare steroids.”
Methotrexate is the optimal intervention in these cases, with azathioprine as the third choice, according to Christopher-Stine.
Meanwhile, for patients with the most severe skin disease, IVIG is recommended.
“If you ask patients what is the most potent therapy we have on the market, they chose IVIG or steroids,” Christopher-Stine said.
Although some rheumatologists may not view skin disease as a severe complication, Christopher-Stine encouraged them to listen to their patients.
“Patients describe it as disfiguring, isolating, and itching,” she said. “Some patients say they have borderline suicidal thoughts due to the itching of skin on their body.”
Turning to patients with lung-dominant disease, Christopher-Stine addressed concerns about methotrexate and lung injury.
“The signal for these events is not actually strong, but if you have other options, why not try them?” she said.
Those options include tacrolimus or azathioprine.
“Tacrolimus is a very effective agent for lung involvement,” Christopher-Stine said.
IVIG may also demonstrate efficacy in lung disease when combined with immunosuppression, she added.
Further trials in IVIG and JAK inhibition are likely to emerge soon, according to Christopher-Stine. In addition, experimental approaches, such as the neonatal Fc receptor, specific TYK2/JAK1 inhibition, TLR7 and TLR8 blockade, along with dendritic cells, all are in early trials.
“Novel therapeutic mechanisms are currently being investigated in several clinical trials,” she said. “The myositis clinical trial landscape in 2023 is everything, everywhere, all at once.”
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Christopher-Stine L. Update in Idiopathic Inflammatory Myopathies. Presented at The Congress of Clinical Rheumatology West; Sept. 7-10, 2023 (hybrid meeting).
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