Developing comparative effectiveness studies for a rare, understudied pediatric disease: lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study

• Main Authors: Suzanne C. Li (Author), Robert C. Fuhlbrigge (Author), Ronald M. Laxer (Author), Elena Pope (Author), Maria F. Ibarra (Author), Katie Stewart (Author), Thomas Mason (Author), Mara L. Becker (Author), Sandy Hong (Author), Fatma Dedeoglu (Author), Kathryn S. Torok (Author), C. Egla Rabinovich (Author), Polly J. Ferguson (Author), Marilynn Punaro (Author), Brian M. Feldman (Author), Tracy Andrews (Author), Gloria C. Higgins (Author), For the CARRA Registry Investigators (Author)
• Format: Book
• Published: BMC, 2019-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background We designed and initiated a pilot comparative effectiveness study for juvenile localized scleroderma (jLS), for which there is limited evidence on best therapy. We evaluated the process we used, in relation to the specific protocol and to the general task of identifying strategie...