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CARRA E-Newsletter Spring 2014

President’s Report Laura Schanberg, M.D. President CARRA Inc. Chair, CARRA Steering Committee

2014 has been, and continues to be, a busy and groundbreaking year for CARRA as we continue to grow as an organization in a variety of ways:

Both membership and sponsorship are up and research coordinators have joined as official members under the Research Associate mem-bership category.

We hosted the Annual CARRA Meeting in Or-lando, immediately prior to the PRSYM meeting in April, welcoming the most mem-bers ever.

For the first time we supported the attendance of pediatric residents and for the second year we supported the attendance of the Research Associate Advisory Committee (RAAC). We plan to maintain and expand both programs moving forward.

This newsletter includes reports by the chair of each research committee about their activi-ties. I hope this will help us all stay up to date on what is going on with the committees we don’t regularly attend.

Another rousing success at the Annual Meeting was the Fellows Committee meeting which attracted over 50 participants! This committee makes up the future of CARRA and it is exciting to see the collaboration, leader-ship, and energy generated by the group.

In addition to these activities, a particular high-light was the poolside reception with terrific sushi!

And there are other significant changes. As many of you know, CARRA has incorporated as CARRA, Inc. with creation of a Board of Directors and officers as well as hiring of an accountant

NEW CARRA CONTACT INFORMATION

CARRA, Inc. 2608 Erwin Road

Suite 148-191 Durham, NC 27705

Phone: (800) 377-5731 Fax: (800) 377-5731

Email: info@carragroup.org

Table of Contents

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President’s Report continued

and legal representation! We have also filed for 501c3 nonprofit status. Read the article by Rob Fuhlbrigge, Finance Committee Chair and CARRA, Inc. Treasurer, about these activities. Rob has done a terrific job with Vaishali Tenkale to identify an accountant, figure out what insurance we need, and plan how to pay our bills!

As part of our growth, there are changes up-coming in the operations of our organization including a search for a new Executive Direc-tor. We are moving our home base out of Stanford in favor of

(see President’s Report, on next page)

a virtual office based wherever the Steering Committee Chair/President is located. As a result, Vaishali Tenkale left CARRA on June 13, 2014, after over a decade of hard work. Carol Wallace has written a piece about Vaishali that you can find in this newsletter as well. Arisa Kapedani, based at Montefiore, also complet-ed her work with CARRA as she went out on leave for the birth of her first child. The CARRA Executive Team thanks them both for all their efforts in support of CARRA – it is hard to imagine moving forward without them.

Sheffa Ariens has joined the CARRA team as Director of Operations and is based in Durham, NC. She comes to us with 15 years of experience in marketing and project manage-ment at non-profits and science-based start-ups, based in the San Francisco Bay Area be-fore she moved to Durham last year. As part of her initial efforts, she has secured our very own CARRA email addresses, phone and fax numbers . Moving forward, Sheffa is your con-tact for all membership related questions and activities. She can be reached at sariens@carragroup.org and by phone at (800) 377-5731, ext. 1.

One familiar face that continues to be around but in a new position is Kelly Mieszkalski. Kelly will be transitioning from working primarily with the CARRA Registry and CTPs to a position

as the Director of Research for CARRA as Registry funding is secured. In her new role she will continue to work with the Research Associate Advisory Committee, and also lead efforts in re-search training, biorepository development, and data and sample use processes.

The nationwide search for a new Executive Direc-tor will launch June 16. All applications will be vetted by Sheffa and I who will present strong candidates to the steering committee to consider. The steering committee will present a slate of three ranked candidates to the Board of Directors who will lead negotiations with the chosen candidate. You can find the job de-scription on the CARRA website once it is final-ized. Please spread the word and encourage ex-ceptional candidates that you know to apply. We hope to have the new director in place by the end of 2014.

So lots of changes. And change makes for stress, anxiety, and excitement! As we grow, it is crucial that members take responsibility for communi-cating with each other. The executive team is determined to improve our communication with members, but we can only be successful if mem-bers read the newsletters and emails that are sent out, respond to queries and surveys, self-nominate for open leadership positions, and vote!

We also ask that you communicate any concerns and suggestions to members of the executive or steering committees. We are developing a formal communications plan and if you are interested in helping with this or just have suggestions to make, please contact Sheffa or any of us. The best ideas, like the idea for a Fellow Committee, come from you!

Please help us make this your organization.

Respectfully submitted,

Laura Schanberg, M.D. President CARRA Inc. Chair, CARRA Steering Committee

Spring 2014

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Spring 2014

Arisa Kapedani Moving On

Arisa has been insuring our members are reimbursed for their travel to Annual CARRA Meetings, collecting member dues, corresponding with all of our committee leadership and helping Vaishali in any number of ways to further the mission of CARRA since June 2011. She has been on site for most CARRA meetings, guiding members through the often

confusing venues with a recognizable smile on her face (see picture). She is due to give birth to her first child any day now, going out on maternity leave for 6 months . Arisa will be returning next year to continue working in pediatric rheumatology at the Children’s Hospital at Montefiore. We wish her all the best.

Farewell to Vaishali

In the early fall of 2004, Christy Sandborg – the second Chair of the CARRA Steering Committee, hired her research database administrator and programmer, Vaishali Tenkale, as the Business Manager for CARRA at Stanford University. Since then Vaishali has provided operational support in many aspects of business develop-ment for CARRA including development of the website, as well as membership and research study related databases. Vaishali helped develop our corporate sponsors, organized and imple-mented CARRA meetings including our Annual Scientific meeting and ACR breakfast meeting, developed and submitted grant applications, assisted with strategic planning efforts and budget management and so much more! Vaishali’s attention to detail, organizational skills and hard work have been invaluable to CARRA’s growth and success. “She has been the memory and glue for our leadership as it changed every 3 years,” states Carol Wallace who worked closely with Vaishali as Vice Chair, Chair and Past Chair of the CARRA Steering Committee. We will miss Vaishali and are grateful for her instrumental role in CARRA’s success and its evolution into the complex organization it is today. “I wish all the success of this world to CARRA and its hardworking, passionate doctors who are making the difference in the lives of children with arthritis every day, “ said Vaishali. “It's been a nice journey for me; I've learned a lot from many of you and came across so many wonder-ful people. CARRA will remain close to my heart, always!”

Annual Meeting Reimbursement Update

Due to staffing changes there were delays in

processing the annual meeting reimbursements this year.

As of June 12, 2014, all reimbursement paperwork has been

processed and submitted. You should be receiving your checks within 2-4 weeks.

We appreciate your patience.

CARRA is Seeking a New Executive Director If you know of a great candidate,

contact Sheffa at info@carragroup.org

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Registry Update

Yuki Kimura, MD Vice-President CARRA Inc. Vice-Chair, CARRA Steering Committee

Through the hard work of all the CARRA Registry sites over the

past three-and-a-half years, the CARRA Registry has been hugely successful and has enrolled 9522 patients, making it the largest registry of pediatric rheumatic diseases in the world!

In addition, the Registry has produced over 20+ full-length publications and innumerable ab-stracts, and has enabled the Consensus Treat-ment Plan (CTP) pilot studies in four diseases (systemic JIA [which has now completed enroll-ment], lupus nephritis, juvenile dermatomyositis and localized scleroderma). Other Registry re-search projects include the CORA (Childhood On-set Rheumatoid Arthritis) study (Prahalad, PI) and The Learning Cohort (Natter, PI), in which JIA and SLE patients will be able to enter their own pa-tient reported outcome measures into a web por-tal. Enrollment into all of these studies is continu-ing in the original Registry (now to be referred as the CARRA Legacy Registry), even though it is closed to enrollment for all other patients.

A NIAMS clinical trials planning grant has been awarded for the PREVENT study (Beukelman and Ilowite, co-PIs), which will be a randomized study looking at whether methotrexate can prevent extension to polyarthritis and uveitis in oligoartic-ular JIA and will use the Registry for data collec-tion. Other Registry studies are also currently be-ing planned, such as the Registry-wide implemen-tation and comparative effectiveness study of the systemic JIA CTPs, a randomized Registry-based study comparing the effectiveness of biologics with differing mechanisms of action in polyarticu-lar JIA, and implementation of the polyarticular JIA CTPs through the Registry.

The CARRA Registry team has now concluded Phase 1 of the Registry and is making key im-provements to case report forms to facilitate and streamline data entry and enable collection of data that will be necessary to study the long term

safety of medications prescribed for children and adolescents with pediatric rheumatic dis-eases. The systemic and polyarticular JIA safety cohort is planned to begin enrollment in the last quarter of 2014. We are actively negoti-ating with several biopharmaceutical compa-nies who would like to use the Registry to fulfill their post-marketing requirements, use the Registry data to understand the demographics and outcomes of JIA patients in the Registry, as well as how JIA is treated in real-life.

Please be on the lookout for the new contracts, protocol and ICFs that are currently being final-ized. The new site contracts will be used for all Registry-related studies and work that are not funded by government sources (such as by pharmaceutical companies and foundations). Phase 2 of the CARRA Registry will begin once these new contracts, protocols and ICFs are in place at your sites. Enrollment will initially fo-cus on new-onset (inception cohort) JIA pa-tients and JIA patients newly starting a DMARD or biologic medication. Re-enrollment of pa-tients in the existing CARRA Legacy Registry will begin gradually. Although the emphasis is on JIA currently, the same diseases from the CAR-RA Legacy Registry are eligible for enrollment in the new CARRA Registry and we are actively soliciting funding from other sources that will enable us to pay you for work related to enroll-ment of patients with other pediatric rheu-matic diseases. We hope to have that in place by the end of the year.

We are excited about these potential new ap-plications and projects of the CARRA Registry, and are looking for investigators at all levels who are interested inbeing involved.

Please contact Yuki Kimura (ykimura@hackensackumc.org) or Tim Beukelman (tbeukelman@peds.uab.edu) for more information.

Spring 2014

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Finance Report

Robert Fuhlbrigge, M.D., Ph.D. Chair, CARRA Finance Committee Treasurer, CARRA

A number of operational updates related to the incorporation of CARRA as an independent non-

profit entity were outlined to the membership at the CARRA business meeting in Orlando. All of the developments described here have occurred through a combination of efforts from the CARRA Executive Committee and staff with the assis-tance and oversight of the CARRA Finance Com-mittee and approved by the CARRA Steering Com-mittee and membership at large. A few dedicated volunteers (Diana Milojevic, Stacy Ardoin and Pe-ter Chira) deserve our appreciation and thanks.

As an independent non-profit corporation, CAR-RA, Inc. is now responsible for functions that were previously provided by our academic insti-tutional partners for compliance with fiduciary and legal standards. These include establishing bank accounts, engagement of an accounting firm and legal counsel, purchase of liability insurance and establishment of new contract relationships with our sponsors and member sites. These are now complete or in progress.

Critical to the ongoing activities of CARRA, Inc. will be the establishment of funding resources to support the activities of our organization. These activities can be segregated into three distinct categories: Registry functions, annual meetings, and core administration. At the time of incorpora-tion, CARRA, Inc. held approximately $250,000 in accounts hosted for our benefit by various aca-demic institutional centers (Stanford, Montefiore, Duke, Hackensack). As of January, we are now able to accept dues and sponsor payments direct-ly to CARRA accounts such that these monies are now available to us without paying institutional overhead. In addition, the existing financial sup-port for our annual meetings from the Wasie Foundation and the Arthritis Foundation, as well as the NIH R13 grant for meeting support, will continue for at least the next year.

Over the past year, the CARRA Executive Com-mittee has been engaged in extensive negotiations with a variety of potential sponsors for support of CARRA operations into the future. This has been an intensive learning experience for all, as each company and foundation has its own interests and goals, as well as unique mechanisms for re-questing funds. In addition to the ongoing meeting support described above, we are pleased to an-nounce a substantial new research grant (CARRA PCORI grant –described elsewhere), as well as pledges from foundation and corporate sponsors in excess of $1M. These funds will allow us to pro-ceed with plans to re-initiate the CARRA Registry, but the size and extent of our network activities will require substantially more funding for full op-erations.

In this regard, a number of interesting opportuni-ties for ongoing support remain under develop-ment or in negotiation. As these are uncertain and fluid in nature and sensitive to clarity of message, the Executive Committee would prefer not to pub-licly disclose the details of these discussions until preliminary agreements have been established. These agreements will then be brought to the Board and Steering Committee, and to the Finance Committee, for discussion and debate.

To summarize, CARRA is going through tremen-dous changes that offer great opportunity for ad-vancing our mission to improve the lives of all chil-dren with rheumatic diseases. While there is a certain amount of flux and a great deal of uncer-tainty at present, from a financial viewpoint we are solvent and have sufficient resources to ad-vance toward establishment of the new CARRA Registry. There are negotiations underway with the potential to revolutionize our capacity to sup-port the interests of the CARRA community and to enhance our impact on the care of children with rheumatic diseases.

Spring 2014

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PCORI Grant: PARTNERS Patient Powered Research Network

The Patient Centered Outcomes Research Insti-tute (PCORI) is a nonprofit health research or-ganization formed in 2010. PCORI funds re-search to compare the effectiveness of various treatments patients and doctors must choose between. PCORI then shares the research results with the nation. The vision of PCORI is to provide the patients with the information they need to make decisions that reflect their desired health outcomes. As part of this vision, PCORI has started a mas-sive project to join together patient-power re-search networks (PPRNs) and clinical data re-search networks (CDRNs) into a huge system that will allow treatments to be compared across millions of people. PARTNERS (Patients, Advocates and Rheumatology Teams Network for Research and Service) is one of the PPRNs created as part of this grand network. PARTNERS formally links together five organizations who share a common vision of improving the lives of families living with any of the many forms of pe-diatric rheumatic disease. These organizations are Childhood Arthritis and Rheumatology Re-search Alliance (CARRA), The Pediatric Rheuma-tology Care and Outcomes Improvement Net-work (PR-COIN), the Arthritis Foundation (AF), the Lupus Foundation of America (LFA) and Friends of CARRA (FoC). CARRA was created to ease research aimed at finding the cause and cure for childhood rheu-matic diseases. Nearly every pediatric rheuma-tologist in North America is a member of CARRA. PR-COIN is also a network of pediatric rheuma-tologists whose goal is to transform the care of children with rheumatic disease. The AF is the largest nonprofit organization dedicated to the prevention, control and cure of arthritis. LFA is devoted to solving the mystery of lupus while giving caring support to those who suffer with it. FOC supports the discovery of a cure and im-

proved treatments for childhood arthritis and related diseases and to increase public aware-ness of childhood rheumatic conditions. Over the next 18 months PARTNERS will build a network to improve the lives of children with rheumatic diseases through research that matters to you. Patient/ family volunteers are at the core of the network. How should PARTNERS be organized? How should we reach out to the patient? Which research questions are most im-portant to the families? How will the patient data be collected, protected and shared? How will the results of the studies be shared to you and your doctors have the information needed to choose your treatment? PARTNERS needs the patient voice to answer these questions. Volun-teers need not have prior experience. The time commitment includes being available during the day to participate in conference calls and willing-ness to read, learn and provide input. Once the PARTNERS network is built, all interest-ed patients can come together and let their voic-es be heard. Should I use drug X or drug Y? When should I start treatment? When should I stop? What causes a flare? Would you like to provide input into how you are doing and how that compares to other patients? By joining PARTNERS you will help answer these questions.

Spring 2014

2014 Annual Meeting By the Numbers

214 CARRA members attended the 2014 Annual Meeting in Orlando, including 133 voting mem-bers, 55 fellows, 14 research associate mem-bers, 6 pediatric residents, 1 international asso-ciate member, and 5 associate members.

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Workgroup Report:

Juvenile Idiopathic Arthritis (JIA)

Chair: Timothy Beukelman, MD, MSCE Vice Chair: Pamela Weiss, MD, MSCE

The JIA Research Committee continued progress on several CARRA Consensus Treatment Plans (CTPs). The uveitis group held a consensus meeting and furthered the development of CTPs for the treatment of uveitis patients who are naïve to systemic treatment and for the treat-ment of patients who are resistant to metho-trexate.

The membership was given a presentation by Brian Feldman about the advantages of using Bayesian methods to analyze the results of clini-cal studies. Members then provided expert opin-ion about the probability of success for the different treatment approaches in the CTPs for systemic JIA and polyarticular JIA. These re-sponses will inform the prior probabilities for the Bayesian methods approach to analyzing results from the CTPs. Grant funding to conduct these studies is being actively pursued.

In the afternoon, the JIA committee split into newly formed workgroups: JIA, systemic JIA, and outcome measures. One of the tasks was to de-fine one or more projects to develop over the next 12 months. There was considerable interest expressed in many new project ideas, including the treatment of juvenile spondyloarthropathy and the optimal management of patients with-polyarticular disease who have achieved clinical inactive disease status (e.g., when and how to stop medications). The outcome measures group aims to create the list of patient reported measures for use in the PCORI-funded patient powered research network (PARTNERS) and the Pediatric Rheumatology Care and Outcomes Im-provement Network (PR-COIN).

Spring 2014

Workgroup Report:

Juvenile Dermatomyositis (JDM)

Chair: Charles Spencer, MD Vice Chair: Adam Huber, MD

The JDM group is continuing work on Consensus Treatment Plans for skin predominant JDM, skin resistant JDM, and biologics in JDM, using confer-ence calls and surveys. The progress of these CTPs was discussed at the CARRA meeting. New leaders were chosen in these areas of interest including calcinosis, quality improvement, exercise and JDM, and quality of life issues in JDM and will continue work by conference calls.

Lisa Rider briefed the JDM Research Committee on other national and international JDM projects that CARRA is involved in including JDM outcome measures. The committee reviewed the exciting new research opportunities that Cure JM has de-cided to offer CARRA members with 1 large 2 year grant of $200,000 and 1-3 small 2 year JDM grants of less than $100,000 per grant. The research must be clinical or translational work on JDM and prefer-ence will likely be given for the smaller grants to junior CARRA members. The deadline is July 1st for the 4 page grant application in the format of the previous Friends of CARRA grants and decisions by August. The grants are to over the period Sept 1, 2014 to Sept 1, 2016. Grantees will be asked to attend the Cure JM meeting in San Jose, CA on Oc-tober 3rd, 2014 with expenses paid.

Reminder: CURE JM/CARRA Grants Available

Deadline: July 1, 2014 Cure JM Foundation will fund up to three Pilot

Study research awards and one Established Investigator research award this year.

For more details, contact Sheffa at info@carragroup.org.

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Workgroup Report:

Pain

Chair: Mark Connelly, MD Vice-Chair: Natalya Fish, MD

During the CARRA April pain subcommittee meeting, we discussed both small and larger projects that are at various stages of develop-ment (from prenatal to late adolescent!) with the objective of maximizing the number of peo-ple contributing to group research projects in some way.

Participants reviewed and provided input to Dr. Jennifer Stinson’s “iCanCope” project involving a smartphone-based individualized pain manage-ment intervention for youth with JIA that con-tains a consensus decision algorithm derived from group work during the CARRA 2012 meeting. Dr. Stinson is in the process of sub-mitting for funding to support preliminary testing of this novel treatment strategy.

Dr. Susmita Kashikar-Zuck discussed her work to date on establishing a combined physical thera-py and cognitive-behavioral therapy intervention for juvenile primary fibromyalgia and reviewed possibilities of various levels of involvement in the future; she is submitting a federal planning grant this summer involving a few collaborators from our group.

We also reviewed our results to date from ana-lyzing Registry data from the fibromyalgia da-taset and discussed continuation of Registry data collection through (a) establishing a minimal pain dataset for the new CARRA Registry; and (b) identifying avenues to support collaborative col-lection of patient-reported outcomes data for patients with persistent idiopathic pain seen in rheumatology practices.

Additional new potential projects discussed as a group included secondary analyses of pain data

from prior CARRA trials, a small multisite evalua-tion of punch biopsy of the skin to evaluate small fiber sensory neuropathy in juvenile fibromyal-gia, a preliminary efficacy trial of naltrexone in specifically improving pain intensity in chronic widespread pain, and development and evalua-tion of musculoskeletal pain education modules for providers. The next steps for each project were discussed and leaders were established, with the request to briefly report on progress on a monthly basis.

Activities at the recent SLE Research Committee meeting at CARRA included progress reports on ongoing projects, visiting speakers, and initial work on a new project related to Quality indica-tors. The committee continues its pilot project on Lupus Nephritis Consensus Treatment Plans (LN-CTP). Sixty-eight patients with newly diag-nosed LN have been screened for the CTP pro-ject. Thirty two (47%) were treated according to one of the CTPs. Several reasons were provided for not using one of the CTPs. Only 60% were enrolled in the CARRA Registry—a requirement for enrollment in the CTP project. This data was presented in abstract form at PRSYM.

Last year the Lupus Committee developed scien-tific priorities for childhood lupus research. The results were presented at the meeting and are currently being prepared for publication. The five priority areas include lupus nephritis, neuro-psychiatric disease, understanding and optimiz-ing long term outcome, understanding patho-genesis and biomarker development, and deter-mining best therapies. These scientific priorities are meant to guide future work and funders

(see Lupus on next page)

Spring 2014

Workgroup Report:

Lupus

Chair: Emily von Scheven, MD Vice-Chair: Ann Eberhard, MD

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(Lupus, continued) We invited two outside speakers to the meeting. Paola Daley, from the Lupus Foundation of Amer-ica (LFA) presented on the work of the LFA and their participation in the PCORI-funded PARNTERS project. Scott Wenderfer, MD, pediat-ric nephrologist, presented on the work of the pediatric nephrology community and their guide-lines related to treatment of lupus nephritis. We have partnered with the pediatric nephrology community on a survey aimed as assessing the practice around renal biopsies and hope that this will serve as the foundation for future collabora-tions. The committee initiated new work in the area of Quality improvement. Background information was provided to the committee by Rina Mina (Development of Quality Indicators for Lupus ), Julia Harris (Childhood Lupus Benchmarking Pro-ject), Esi Morgan DeWitt (Improving Outcomes with Learning Networks), Stacy Ardoin (Extending the JIA Experience to Lupus), and Aimee Hersh (Leveraging the CARRA Registry). Then the com-mittee broke into breakout groups for a struc-tured discussion about the type, extent and goals of quality improvement work for the CARRA Lu-pus Research Committee. Issues of importance and feasibility were discussed. Several individuals volunteered to continue this work after the meeting, and all interested members are invited to participate.

Spring 2014

The Scleroderma, Vasculitis and Rare Diseases (SVRD) committee is now divided into three sep-arate research subgroups. The juvenile Localized Scleroderma (jLS) group has filled 92% of its target enrollment in a pilot consensus treatment plan study funded by the Arthritis Foundation. Patients with active jLS who are initiating treat-ment with one of three methotrexate-based standardized treatment regimens were eligible for enrollment in this prospective observational study based in 10 CARRA centers, with data cap-tured in the web-based CARRA Registry EDC sys-tem. Over 70% of the study subjects are partici-pating in the optional sample biobanking substudy; these samples enable future transla-tional studies to identify biomarkers and further understand disease pathogenesis. Patients are being evaluated with recently developed clinical activity and damage measures to assess their operational characteristics. Initial testing showed excellent validity of the activity measure but poor validity of the damage measure. The group has therefore been modifying the damage measure to improve its validity. The modified damage measure was discussed in the jLS ses-sion at the annual CARRA meeting, with case

Workgroup Report:

Scleroderma, Vasculitis and Rare Diseases (SVRD)

Chair: Robert Sundel, MD Vice Chair and Incoming Chair: Suzanne Li, MD

Call for Nominations

We are currently accepting nominations for the following roles. If you would like to be considered or have a great candidate in mind, contact Sheffa at info@carragroup.org.

(see SVRD on next page)

Vice Chair, SVRD Vice Chair, TRTC Early Investigators Committee

Publications Committee Chair, Research Associate Advisory Committee (RAAC)

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The role of the Translational Research and Tech-nology Committee (TRTC) is to support high-quality translational research within CARRA. Over the last year, the TRTC has pursued this goal in several ways. Building on past efforts, the TRTC worked with investigators to identify con-sensus biospecimen protocols for proposed translational studies. The central TRTC bioreposi-tory at Cincinnati Children’s Hospital Medical Center completed its adoption of new biobank-ing software capable of integration with clinical databases and with research labs external to CCHMC. The Protocol Evaluation Subcommittee of the TRTC performed review for the Friends of CARRA grant applications, resulting in funding for two outstanding translational projects arising out of the CARRA network. The TRTC Chair, Dr. Susan Thompson, represented CARRA in the PCORnet Biorepository Task Force, comprised of representatives from about 25 research net-works to address biorepository logistics, annota-tions standards, sample sharing and governance, and sustainability/cost recovery. At this year’s Annual Meeting, the TRTC began the transition to the leadership of Dr. Peter Ni-grovic as the new TRTC Chair. Participants as-sisted with completion of a questionnaire re-quired for the new CARRA PCORI award, and planned a CARRA-wide survey of translational research capacity to support biospecimen collec-tion for the systemic JIA CTP and similar studies. Given the increased availability of samples col-lected under CARRA-sponsored studies, planning was also begun for governance documents regu-lating the fair and scientifically productive utili-zation of existing samples.

(SVRD, continued)

scenarios presented to test the modified measure’s feasibility and validity among attendees. Other tools needed for conducting comparative effectiveness treatment studies are also being evaluated in this pilot study. The Autoinflammatory Diseases group is work-ing to characterize clinical subtypes of one of the periodic fever syndromes (PFAPA) in preparation for refining clinical criteria to allow for a more homogenous study group from which samples for a biorepository may be collected. Members of the SVRD are participating in a questionnaire to explore current diagnostic criteria and treat-ment approaches in preparation for future trials. In parallel with this, the CRMO group is evalu-ating the possibility of a comparative effective-ness study comparing DMARDs, bisphospho-nates, and biologic agents for the treatment of children who fail to respond to NSAIDs. This ne-cessitates a more precise definition of treatment response, including defining disease activity in a quantifiable and reproducible manner. Finally, the Vasculitis group continues to consid-er treatment approaches to the most common types of pediatric vasculitis in preparation for development of clinical effectiveness studies. These discussions include initial treatment of ANCA-associated vasculitides and Henoch-Schonlein Purpura (HSP), and augmented thera-py for children with Kawasaki Disease at high risk of developing aneurysms. Hand-in-hand with these efforts are participation of members of the SVRD with other international organiza-tions, including OMERACT and PReS, to develop disease activity and outcome measures for pedi-atric vasculitides. These are essential if studies of vasculitis in children are to be harmonized with the far larger studies currently underway in adults.

Spring 2014

Workgroup Report:

Translational Research and Technology Committee (TRTC)

Chair: Susan Thompson, MD Vice Chair and Incoming Chair: Peter Nigrovic, MD

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CARRA Research Associate Membership Update Kelly Mieszkalski, MA Member, Research Associate Advisory Committee Director of Research, CARRA Inc. kelly.m@duke.edu

Invitation for Coordinators to Join CARRA

CARRA began offering membership to coordina-tors via the Research Associate Membership in August of 2012. Joining CARRA as a Research Associate Member is free. Research Associate Members must be clinical research professionals who actively work with studies related to chil-dren with rheumatic disease. CARRA Research Associate Members will have access to the CARRA Coordinator online forum/communication tool that will be released in the coming months. The online forum will be a great way to help connect coordinators working in pe-diatric rheumatology for brainstorming, problem solving, networking and mentoring. CARRA Re-search Associate Members will also be able to participate in the upcoming election of the Re-search Associate Chair that will take place in May and June of 2014. To date, there are 37 Research Associate members from 29 of 114 CARRA sites. We strongly encour-age all coordinators and other research profes-sionals (such as regulatory specialists, data tech-nicians, and research managers) who are working on pediatric rheumatology research studies at CARRA sites to join our research coordinator net-work by joining CARRA today.

CARRA Research Associate Members in Orlando at 2014 Annual CARRA Meeting Front Row: Lori Ponder, Amy Rakestraw Back Row: Kelly Mieszkalski, Ann Kunkel, Jennifer Woo, Janet Wootton, Mary Ellen Riordan, Mary Gervasini, Kimberly Francis, Heather Benham, Joni Dean, Ching Hung Present at CARRA but not in photo: Andrea Hudgins, Marsha Malloy, Heidi Stapp

Spring 2014

CARRA Research Associate Advisory Committee (RAAC)

The CARRA RAAC (formerly the SCAC/Study Co-

ordinator Advisory Committee) was formed back

in August of 2011. The mission of the CARRA

Research Associate Advisory Committee (RAAC)

is to provide guidance and support to CARRA in

the conduct of high quality Pediatric Rheumatol-

ogy Research. The RAAC currently has 10 mem-

bers and together they have over 150 years of

research experience:

Heather Benham, DNP, RN, CPNP, Texas Scottish Rite Hospital

Katie Caldwell Mims, RN, BSN, MA, Medical University of South Carolina

Kimberly Francis, MS, MPA, Children's Hospi-tal of Pittsburgh

Mary Gervasini, BA, CCRP, Baystate Medical Center

Andrea Hudgins, BA, CCRP, Indiana University Kelly Mieszkalski, MA, Duke and CARRA Marsha Malloy, RN, BSN, MBA, CCRC, Medical

College of Wisconsin Mary Ellen Riordan, BSN, CCRC, Hackensack

University Medical Center Jenna Tress, BA, CCRP, Children's Hospital of

Philadelphia Jennifer Woo, MPH, University of California,

Los Angeles and Medical College of Wisconsin

See RAAC on next page

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Spring 2014

RAAC, continued

CARRA Registry Newsletter

The Newsletter Committee (Heather Benham, Marsha Malloy, Kelly Mieszkalski, Katie Mims and Jennifer Woo) has produced 4 CARRA Registry participant newsletters (The CARRAnet Gazette) which can be found on the CARRA Registry web-site: https://www.carranetwork.org/news. Addi-tional participant newsletters are on hold pending the new protocol. Joni Dean from the American Family Children’s Hospital in Madison, Wisconsin and Ching Hung from Seattle Children’s Hospital will be joining the Newsletter Committee once newsletter production resumes.

CARRA Registry Cost Survey

Jenna Tress and the CHOP site conducted a survey of CARRA Registry Principal Investigators and Re-search Coordinators on costs related to the CAR-RA Registry. The survey has been completed and data has been analyzed. They are in the final stages of writing up a manuscript, and are dis-cussing where they would like to publish. Prelimi-nary summary of results include: 52 respondents. Almost 1/3 of the respondents reported avoiding enrollment of certain diagnoses; 89% reported supplementing funds provided by the Registry with either their own or divisional funds.

Steroid Taper Smart Phone App

CARRA RAAC members Marsha Malloy, and Jen-nifer Woo, initiated efforts to develop a smart device app that will calculate and calendar steroid taper regimens. The multidisciplinary team also includes CARRA members Dominic Co, MD, PHD and Betsy Roth Wojcicki, PNP, neurology and im-

The CARRA Research Associate Advisory Committee (RAAC) is available to provide review of CARRA protocols and associated study documents at any stage in the proto-col development process.

Whether you are a new or a seasoned investiga-tor, please consider consulting with the RAAC on your project. Contact Kelly Mieszkalski at kelly.m@duke.edu or 919.668.7531 if you have any questions or have material for which you would like input from the RAAC.

RAAC Protocol and Document Review

munology colleagues at the Medical College of Wisconsin as well as a patient representative.

The team’s Steroid Taper Calculator and Calen-dar App was one of 6 out of 86 applications re-cently awarded support from University of Wis-consin-Milwaukee Computer Science App Brew-ery for the development. Programming is ex-pected to commence this summer with Phase I ready for beta-testing in late 2014. The multi-phase design plan was presented by Betsy at the 2014 CARRA Meeting to elicit awareness and input from the CARRA membership during early stages of the app development

2014 Annual CARRA Meeting

Fifteen CARRA Research Associate members in-cluding 8 members of the Research Associate Advisory Committee attended the 2014 annual CARRA meeting in Orlando. Members attended disease-specific committee sessions and were also able to meet as a group to discuss goals for the future of CARRA Research Associate Mem-bers and the RAAC within the CARRA organiza-tion. Members will continue to pursue their own pediatric rheumatology research interests in the coming year including research on transition is-sues, environmental factors related to pediatric rheumatic disease, physical activity and out-comes, access to care, QI, and research on pedi-atric rheumatology research site resources and staff. Be on the lookout for presentations from Research Associate members at our next CARRA meeting!

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Spring 2014

CARRA Steering Committee in Orlando at Annual CARRA Meeting

Front Row: Robert Fuhlbrigge, Peter Blier, Norman Ilowite, Kathleen O’Neill, Suzanne Li Back Row: Laura Schanberg, Yuki Kimura, Charles Spencer, Ann Eberhard, Susan Thompson, Carol Wallace, Pe-ter Nigrovic, Hanisi Accetta, Mark Connelly, Christy Sandborg, Tim Beukelman, Natalya Fish, Emily Von Scheven, Pamela Weiss

JOIN CARRA TODAY

To become a CARRA Research Associate member, submit the completed CARRA membership application along with a letter of support from a current CARRA voting member to member-ship@carragroup.org or fax to (800) 377-5731.

For an application and more information on how to join CARRA, please visit the CARRA website:

www.carragroup.org/get-involved

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CARRA gratefully acknowledges the continuous support of

Save the date for CARRA’s 2015 Scientific Meeting April 17-19, 2015