In the JournalsPerspective

Consensus treatment plans developed for juvenile dermatomyositis with persistent rash

The Childhood Arthritis and Rheumatology Research Alliance developed the following three treatment plans for patients with juvenile dermatomyositis and persistent skin rash: intravenous immunoglobulin; mycophenolate mofetil; and cyclosporine.

Researchers used a combination of two Delphi surveys and four group meetings held from 2011 through 2015 to develop a consensus that accurately reflected typical treatment, with the assumption that patients were already receiving methotrexate and corticosteroids. Adam M. Huber, MD, at Dalhousie University in Nova Scotia, and colleagues wrote that patients not previously treated with IV immunoglobulin should try 3 doses of 2g/kg (maximum 70 g) of IV immunoglobulin for every 2 weeks, then monthly. The second treatment plan included 10 mg/kg or 600 mg/m2 (maximum 1,500 mg) of mycophenolate mofetil and the third plan called for at least 3 mg/kg of cyclosporine. For mycophenolate mofetil and cyclosporine, patients can continue receiving no more than 2 mg/kg per day (maximum 60 mg) of corticosteroids, methotrexate and intravenous immunoglobulin.

To receive treatment, patients should have skin that has not improved for at least 3 months after resolution of previous muscle involvement. In addition, patients should have no ulcerative skin disease, calcinosis that is more than mild or any medical condition that would influence treatment outcome.

“[These] treatment plans may not be relevant for some providers and some patients,” the researchers wrote. “However, it is expected that these treatment plans would represent a reasonable approximation of typical treatment for the majority of patients with this phenotype by the majority of providers.” – by Will Offit

Disclosure: The researchers report no relevant financial disclosures.

The Childhood Arthritis and Rheumatology Research Alliance developed the following three treatment plans for patients with juvenile dermatomyositis and persistent skin rash: intravenous immunoglobulin; mycophenolate mofetil; and cyclosporine.

Researchers used a combination of two Delphi surveys and four group meetings held from 2011 through 2015 to develop a consensus that accurately reflected typical treatment, with the assumption that patients were already receiving methotrexate and corticosteroids. Adam M. Huber, MD, at Dalhousie University in Nova Scotia, and colleagues wrote that patients not previously treated with IV immunoglobulin should try 3 doses of 2g/kg (maximum 70 g) of IV immunoglobulin for every 2 weeks, then monthly. The second treatment plan included 10 mg/kg or 600 mg/m2 (maximum 1,500 mg) of mycophenolate mofetil and the third plan called for at least 3 mg/kg of cyclosporine. For mycophenolate mofetil and cyclosporine, patients can continue receiving no more than 2 mg/kg per day (maximum 60 mg) of corticosteroids, methotrexate and intravenous immunoglobulin.

To receive treatment, patients should have skin that has not improved for at least 3 months after resolution of previous muscle involvement. In addition, patients should have no ulcerative skin disease, calcinosis that is more than mild or any medical condition that would influence treatment outcome.

“[These] treatment plans may not be relevant for some providers and some patients,” the researchers wrote. “However, it is expected that these treatment plans would represent a reasonable approximation of typical treatment for the majority of patients with this phenotype by the majority of providers.” – by Will Offit

Disclosure: The researchers report no relevant financial disclosures.

    Perspective
    Cathy Patty-Resk

    Cathy Patty-Resk

    CARRA should be applauded for coming together to provide guidance to other pediatric rheumatology providers, as well as non-pediatric and non-rheumatology providers who are caring for these children in severely underserved areas.  CARRA has identified a pertinent sub-group of JDM in need of further research. This is the group of patients has residual skin disease after resolution of muscle involvement and fatigue. A less common pediatric small vessel vasculitis, JDM has a lack of proven effective treatments. It is possible this sub-group of patients with residual skin disease may need a different treatment regimen for undiscovered reasons. By more clearly stratifying JDM into three subclasses with CTPs, the goal is to help further advance research for a better understanding of this disease with its three most common presentations, particularly the group with residual skin disease.  There has been little progress in understanding this disease with regard to biologic targets and thus, no new biologics have proven effective. CARRA has set the stage for more organized and meaningful research into currently used JDM treatment protocols, leaving room for an experimental arm to study possible new treatments. These CTPs would mean children have access to better treatments leading to improved treatment outcomes.  The three CTPs are ones that have been used by many pediatric rheumatologists for years, but not necessarily with the group experiencing residual active skin disease. This is the group that may be undertreated. By setting the CTPs, it is more likely providers will continue to seek effective treatment for the residual skin disease instead of ignoring it. However, this is the group of patients who may be tougher to get parental “buy in” for more aggressive pharmaco-management. They often do not see the residual skin disease as problematic or threatening of health status as they did when fatigue, muscle pain and weakness was present.  Another area that may become more problematic is obtaining insurance authorization for off-label use of pharmaceuticals not FDA approved for JDM. In pediatric rheumatology, providers are experiencing this phenomenon more each day. Unfortunately, many of the drugs used in pediatric rheumatology are used off-label or with FDA orphan designation and have been used for years safely with great success. Humira for uveitis in children is a prime example of this because it is only approved for children with JIA and Crohn’s disease.  CARRA did not address the issue of criteria for treatment failure, with this paper stating it will leave it to the provider to determine. This may lead to early treatment withdrawal or not changing the treatment plan when failure has occurred. More experienced practitioners and subspecialty trained practitioners will likely have similar thresholds of failure. However, it is the less experienced, rural or non-rheumatology providers whose failure thresholds will likely differ most. Thus, would most likely affect the children who do not have access to one of the only 300 pediatric rheumatologists in the United States and possibly lead to worse outcomes.

    • Cathy Patty-Resk, MSN, RN, CPNP
    • Pediatric Rheumatology Nurse Practitioner Children’s Hospital of Michigan Specialty Center Detroit, Detroit

    Disclosures: Patty-Resk reports no relevant financial disclosures.