Intravenous Immunoglobulin an Effective Treatment for Refractory Cutaneous Dermatomyositis

by Anthony P. Fernandez, MD, PhD, and Carmen Gota, MD

New insights into the treatment and diagnosis of dermatomyositis (DM) are promising for patients in terms of outcomes, yet also illuminate the need for further study.

Adult and juvenile DM are systemic immune-mediated inflammatory diseases most commonly affecting the skin and musculoskeletal system. Amyopathic DM is a subtype that affects only the skin and lacks the characteristic muscle involvement. Treatment of these conditions, especially the cutaneous manifestations, is challenging, and currently no universally effective single treatment exists.

Our recent research provides additional evidence that intravenous immunoglobulin (IVIG) is effective in the treatment of refractory cutaneous DM, regardless of DM subtype, and results in decreased need for glucocorticoids and immunosuppressive agents.

Eighty percent of patients in our 42-person cohort improved with the IVIG treatment, and the treatment was well tolerated over extended periods, with only six participants discontinuing the treatment because of suspected IVIG-related adverse events. Additionally, the study affirms that we can determine whether a patient will have a good response to IVIG in just two to three months — after an average 1.82 cycles of the treatment — which is important given the treatment’s cost and its burden on patients.

It’s not uncommon for us to have patients simultaneously on multiple systemic immunosuppressive medications and still have difficulty managing the cutaneous aspects of the disease. For decades, we have relied substantially on systemic glucocorticoids in the treatment of DM, despite their ineffectiveness in addressing cutaneous manifestations. Even combination regimens of topical and systemic immunosuppressive medications often fail to adequately control cutaneous activity.

Patients in our study, published in Clinical and Experimental Dermatology, had active cutaneous DM after receiving at least two different systemic medications and were then ultimately treated with IVIG. Of note, methotrexate, used as either a monotherapy or in a combination regimen, had failed to control the disease in more than 60 percent of cases.

Patients who respond well to IVIG can taper off systemic steroids and sometimes other immunotherapies with potentially dangerous side effects. We also often can cut back on the IVIG dosage and time between infusions. Some people may go into remission and choose to discontinue the treatment completely.

While we believe this study represents the largest refractory cutaneous DM cohort evaluated for cutaneous response to IVIG to date, it is limited in its retrospective design, relatively small cohort and lack of objective evaluation of cutaneous disease severity before and after treatment. A prospective trial to rigorously assess the effectiveness and safety of IVIG would be useful.

Other new treatments being tested

Although IVIG appears to be useful based on our research, there remains a strong need for better medications to treat DM. We are actively collaborating with pharmaceutical companies to test newer treatments that we hope will prove safe and effective for skin manifestations associated with cutaneous DM.

We currently are recruiting for two clinical trials:

The first study (NCT02245841) aims to assess the safety and efficacy of H.P. Acthar® (adrenocorticotropic hormone) gel for treating the cutaneous manifestations in refractory classic DM, juvenile DM and amyopathic DM.

H.P. Acthar gel received FDA approval in 1952 for treatment of DM and other autoimmune diseases, but there is insufficient data relative to its safety and efficacy for treating DM. Recently an open-label study was published in the Annals of Rheumatologic Diseases suggesting this treatment may be effective for refractory DM. However, this study focused primarily on myositis whereas our study is focusing primarily on the cutaneous aspects of DM.

The second study (NCT03181893) will examine efficacy, safety and tolerability of a novel monoclonal antibody for treatment of adult patients with refractory cutaneous DM. We are among a select group of dermatology departments in the United States involved in this multicenter, phase 2a study. The treatment represents one of the first, if not the first, targeted medication developed specifically for DM and based on our understanding of the cellular and molecular biology of DM.

Patients with DM, or clinicians who have DM patients interested in either of these studies can contact Dr. Fernandez’s office at 216-445-8776.

Dr. Fernandez is Director of Medical Dermatology. Dr. Gota is staff in the Department of Rheumatologic and Immunologic Diseases.