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JDM

Overview

JDM is a rare but serious chronic childhood multi-system autoimmune disease, which at times resembles chronic graft-versus-host disease. JDM can result in serious morbidity and even mortality if not treated properly.

Leadership

Adam Huber, MD

Adam Huber, MD JDM Chair

Susan Kim, MD MMSc

Susan Kim, MD, MMSc JDM Vice Chair

 

JDM News

The JDM group had a very successful year in 2015.

  1. Clinical Treatment Plans (CTPs) for JDM with Persistent Skin Disease (leader Dr. Adam Huber) The CTPs were refined and finalized, with submission of manuscript November 2015.
  2. CTPs for JDM with Predominant Skin Disease (leader Dr. Susan Kim) The CTPs were refined and finalized. The manuscript is in the final stages before submission.
  3. Biologics for JDM: Review and CTPs (leader Dr. Chuck Spencer) A review of typical clinical practice was conducted, and CTPs have been developed. The associated manuscript is in the final stages before submission.
  4. Quality Measures in JDM (leader Dr. Heather Tory) The group has assessed physician and patient/family preferred quality measure and patient-oriented outcomes in JDM. This work was presented as an abstract (poster) at the ACR meeting in November 2015, and an accompanying manuscript is in development.
  5. Understanding calcinosis in Juvenile Dermatomyositis (leader Dr. Mark Hoeltzel) This group is in the process of establishing priorities and reviewing current knowledge. In the future, this information will be used in CTP development and to help determine the research agenda in this area.
  6. Long-term adult outcomes in Juvenile Dermatomyositis (leaders Drs. Phil Kahn and Chuck Spencer) The group is developing a survey to begin to assess adult outcomes of JDM patients.

JDM Sub-Committee:

  1. Pilot study of best treatments for moderate JDM (leader Dr. Ann Reed)—this pilot study was completed in October 2015. Data is now being analyzed, and will provide preliminary information regarding the comparative effectiveness of the CTPs. These were the first published CTPs for JDM. More importantly, this pilot will provide important feasibility information to facilitate the development of formal comparative effectiveness studies to compare these CTPs.
  2. Amyopathic Juvenile Dermatomyositis: Building Consensus to Improve Patient Care (leaders Dr. Phil Kahn and Susan Kim)—a consensus meeting is being planned between members of the JDM Sub-Committee and member of the Pediatric Dermatology Research Alliance (PedRA). The goals of this meeting will be discuss ways to achieve consensus in diagnosis and management of this group of patients. The meeting will occur in conjunction with the CARRA Annual Meeting in Toronto.
  3. Cross-sectional evaluation of hydroxychloroquine therapy in children with JDM enrolled in the CARRA registry (leader Dr. Dawn Wahezi) is a project using data from the legacy CARRA registry, and seeks to characterize how this medication has been used.
  4. Health Care Disparities in JDM (leaders Drs. Angela Robinson and Susan Kim) will use the legacy CARRA Registry to examine health care disparities and differences in outcome for children with JDM.

The JDM Sub-Committee has also continued to collaborate with CureJM. Members of the JDM Sub-Committee have spoken at the CureJM Annual Medical Conference and CureJM Annual Family Conference (last held October 2014, next January 2016).

The JDM Sub-Committee has also provided expert review for research funding competitions that were held summer 2014, January 2015 and July 2015. This has resulted in the following grants being awarded to JDM Sub-Committee members (results of the most recent competition are not available).

  • Novel Biomarkers Associated with Disease Activity in JDM (Dr. Hanna Kim) (2015)
  • Complement C4a copies in JDM-pathogenesis (Drs. Chack-Yung Yu and Chuck Spencer) (2015)
  • Premature atherosclerosis in Juvenile Dermatomyositis (Dr. Dawn Wahezi) (2014)
  • Predictive Modeling for treatment of inflammatory myositis (Dr. Ann Reed) (2014)
  • Lymphocyte Repertoire and JDM (Drs. Susan Kim and Lauren Henderson) (2014)

Goals for 2016: 

1. Full integration of JDM into the CARRA Registry. We consider this to be essential infrastructure that will support observational research, allow comparative effectiveness research of clinical treatment plans and facilitate biospecimen collection to support basic science research.

2. Completion and publication of the CTPs described above

  • JDM with Persistent Skin Disease
  • JDM with Predominant Skin Disease
  • Biologics in JDM

3.  Further development of the other working groups (Calcinosis, Quality Measures, Adult Outcomes).

4.  Completion of the Amyopathic JDM consensus meeting, followed by establishment of an agenda for this group to continue working towards consensus on diagnosis and management of these patients.

5.  Continued collaboration with CureJM. This will include providing expert review for grant competitions. However, we are also interested in engaging with the CureJM membership to better understand their priorities in the research agenda.

 

JDM Projects

Study of microchimerism in juvenile dermatomyositis

Principal Investigator: Ann Reed, MD, Mayo Clinic, Rochester, MN

Juvenile dermatomyositis (JDM) is a multi-system autoimmune disease, which at times resembles chronic graft-versus-host disease. This led us to suggest that non-self cells may play a role in the disease process. We are examining the relationship between HLA genotype and the presence of maternally-derived chimeric cells in JDM patients and healthy controls, and assessed immunologic activity in the chimeric cells. We are studying the genetic contribution to the presence of these chimeric cells. To date, we have identified chimeric cells more often in children with JDM (60/72) than in their unaffected siblings (11/48) and in healthy controls (5/29). The presence of chimerism in the JDM patients, their healthy siblings, and unaffected control children was associated with a HLA-DQA1*0501 allele in the mother. We are investigating the effects of other genes on the presence of these chimeric cells as well as if the lead to susceptibility or protection from developing JDM. Further, we show that maternally transferred chimeric T cells are responsive to the host’s (JDM child’s’) lymphocytes (33.75 +/- 8.4 IFNg producing cells from JDM cells vs. 5.0 +/- 1.25 from maternal cells), and that this is a memory response. These combined data indicate that chimeric cells play a direct role in the JDM disease process and that the mother’s HLA genotype facilitates the transfer and/or persistence of maternal cells in the fetal circulation.


International myositis classification criteria project

Principal Investigators: Lisa Rider, MD, and Ingrid Lundberg, MD, PhD


Completed Project: Towards standards of care for juvenile dermatomyositis

Principal Investigator: Brian Feldman, MD, MSc, University of Toronto, Toronto, ON

Summary: JDM is a rare but serious chronic illness of childhood that can result in serious morbidity and even mortality if not treated properly. We aim – by way of survey – to determine the treatment strategies in use in North America, and through consensus to define the few best treatment strategies that can be compared in clinical trials. This study will lead to improved and standardized treatments for children with JDM.

Download the practice survey map: CARRA – practice survey map 7nov061


Completed Project: Rituximab in refractory adult and juvenile myositis (RIM) study

Principal Investigator: Ann Reed, MD, Mayo Clinic, Rochester, MN