Posted on 03/09/2023 7:51:14 PM PST by ConservativeMind
Juvenile dermatomyositis, a rare but often severe and chronic systemic autoimmune disease, includes a large number of patients who are treatment resistant, requiring long term immunosuppressive therapy. A small open-label study shows promise using a targeted biologic therapy called abatacept to treat such patients.
In the 24-week trial, ten patients with juvenile dermatomyositis ages 7 years or older with moderate disease activity who all received abatacept therapy had significant improvements in their muscle strength, physical function, endurance and skin rashes, as well as in the amount of muscle inflammation on MRI.
In addition, patients were able to lower the daily dose of corticosteroids during the trial, said Rodolfo V. Curiel, M.D.
"This study makes an important contribution to the field of juvenile dermatomyositis," Curiel said. "Treatment resistant patients require long-term immunosuppressive therapy with glucocorticoids, methotrexate and other medications. This 24-week clinical trial suggests abatacept may be beneficial and a potentially safe option for refractory juvenile dermatomyositis."
Curiel says the study is the first clinical trial conducted to evaluate the safety and efficacy of abatacept in such patients. Further study must be conducted to verify the findings of this small, pilot study, he says.
"This is a breakthrough for juvenile dermatomyositis. The majority of these treatment-refractory patients improved at least moderately in clinical disease activity measures, as well as in two objective blinded measures, interferon genes and protein markers, reflecting downregulation of a key cytokine signaling pathway in JDM, and muscle inflammation on MRI.
(Excerpt) Read more at medicalxpress.com ...
It’s fantastic that there is another treatment option!
My youngest daughter was diagnosed with JDMS back in the early 90s. Her treatment was steroids and physical therapy. I don’t remember if she took any other meds.
For a while she was on a prednisone IV , and then took it orally. We had to crush her pills into a powder and mix it in with applesauce for her to be able to get it down. I’m happy to say she has been in remission since she was about 10yrs old.
She’s now almost 36, she’s married and enjoying life.
She doesn’t seem to have suffered any long term effects from all the steroids she took.
I have to give credit to her pediatrician-when he first saw her, she presented with a butterfly rash. He took a blood sample, examined it, and told us
“If I didn’t know better, I’d say she had Lupus, but that’s impossible, no 3yr old gets Lupus!” So he made some calls and directed us to -then- Children’s Memorial Hospital in Chicago and the Immunology Department.
After tests, they made a positive diagnosis and started her treatment.
The rest is history.
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