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Pediatr Rheumatol Online J. 2019 Jul 15;17(1):43. doi: 10.1186/s12969-019-0350-5.

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

Author information

1
Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Imus PC337, 30 Prospect Ave, Hackensack, NJ, 07061, USA. suzanne.li@hackensackmeridian.org.
2
Hackensack Meridian School of Medicine at Seton Hall University, Clifton, NJ, USA. suzanne.li@hackensackmeridian.org.
3
University of Colorado- Denver and Children's Hospital Colorado, Denver, CO, USA.
4
University of Toronto and The Hospital for Sick Children, Toronto, Canada.
5
Children's Mercy, Kansas City, MO, USA.
6
Texas Scottish Rite Hospital and UT Southwestern, Dallas, TX, USA.
7
Mayo Clinic, Rochester, MN, USA.
8
Duke University School of Medicine, Durham, NC, USA.
9
University of Iowa Stead Family Children's Hospital, Iowa City, IA, USA.
10
Boston Children's Hospital, Boston, MA, USA.
11
UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
12
University of Iowa Carver College of Medicine, Iowa City, IA, USA.
13
Rutgers School of Public Health, Rutgers University, Newark, NJ, USA.
14
The Ohio State University and Nationwide Children's Hospital, Columbus, OH, USA.

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 strategies for implementing studies in rare pediatric diseases.

METHODS:

This was a prospective, multi-center, observational cohort study of 50 jLS patients initiating treatment, designed and conducted by the jLS group of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) from 2012 to 2015. A series of virtual and physical meetings were held to design the study, standardize clinical assessments, generate and refine disease activity and damage measures, and monitor the study. Patients were initiated on one of three standardized methotrexate-based treatment regimens (consensus treatment plans, CTPs) and monitored for 1 year. An optional bio-banking sub-study was included.

RESULTS:

The target enrollment of 50 patients was achieved over 26 months at 10 sites, with patients enrolled into all CTPs. Enrolled patients were typical for jLS. Study eligibility criteria were found to perform well, capturing patients thought appropriate for treatment studies. Minor modifications to the eligibility criteria, primarily to facilitate recruitment for future studies, were discussed with consensus agreement reached on them by the jLS group. There were marked differences in site preferences for specific CTPs, with half the sites treating all their patients with the same CTP. Most patients (88%) completed the study, and 68% participated in the bio-banking substudy.

CONCLUSIONS:

We demonstrate the feasibility of our approach for conducting comparative effectiveness research in a rare pediatric disease. Multi-center collaboration by dedicated investigators who met regularly was a key factor in the success of this project. Other factors that facilitate these studies include having a sufficient number of investigators to enroll in each regimen, and streamlining study approval and management.

KEYWORDS:

Assessment tools; Comparative effectiveness trial; Consensus treatment plan; Corticosteroids; Juvenile localized scleroderma; Methotrexate; Study design

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