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

Suzanne C. Li, Robert C. Fuhlbrigge, Ronald M. Laxer, Elena Pope, Maria F. Ibarra, Katie Stewart, Thomas Mason, Mara L. Becker, Sandy Hong, Fatma Dedeoglu, Kathryn S. Torok, C. Egla Rabinovich, Polly J. Ferguson, Marilynn Punaro, Brian M. Feldman, Tracy Andrews, Gloria C. Higgins, E. Anderson, K. Francis, I. GohJ. Jaquith, K. Schollaert-Fitch, C. Smith, J. Weiss, J. Wooton

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Article number43
JournalPediatric Rheumatology
Volume17
Issue number1
DOIs
StatePublished - Jul 15 2019

Fingerprint

Localized Scleroderma
Rheumatology
Arthritis
Pediatrics
Research
Therapeutics
Rare Diseases
Comparative Effectiveness Research
Research Personnel
Juvenile-onset scleroderma
Methotrexate
Observational Studies
Cohort Studies

Keywords

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

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Rheumatology
  • Immunology and Allergy

Cite this

Developing comparative effectiveness studies for a rare, understudied pediatric disease : Lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study. / Li, Suzanne C.; Fuhlbrigge, Robert C.; Laxer, Ronald M.; Pope, Elena; Ibarra, Maria F.; Stewart, Katie; Mason, Thomas; Becker, Mara L.; Hong, Sandy; Dedeoglu, Fatma; Torok, Kathryn S.; Rabinovich, C. Egla; Ferguson, Polly J.; Punaro, Marilynn; Feldman, Brian M.; Andrews, Tracy; Higgins, Gloria C.; Anderson, E.; Francis, K.; Goh, I.; Jaquith, J.; Schollaert-Fitch, K.; Smith, C.; Weiss, J.; Wooton, J.

In: Pediatric Rheumatology, Vol. 17, No. 1, 43, 15.07.2019.

Research output: Contribution to journalArticle

Li, SC, Fuhlbrigge, RC, Laxer, RM, Pope, E, Ibarra, MF, Stewart, K, Mason, T, Becker, ML, Hong, S, Dedeoglu, F, Torok, KS, Rabinovich, CE, Ferguson, PJ, Punaro, M, Feldman, BM, Andrews, T, Higgins, GC, Anderson, E, Francis, K, Goh, I, Jaquith, J, Schollaert-Fitch, K, Smith, C, Weiss, J & Wooton, J 2019, 'Developing comparative effectiveness studies for a rare, understudied pediatric disease: Lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study', Pediatric Rheumatology, vol. 17, no. 1, 43. https://doi.org/10.1186/s12969-019-0350-5
Li, Suzanne C. ; Fuhlbrigge, Robert C. ; Laxer, Ronald M. ; Pope, Elena ; Ibarra, Maria F. ; Stewart, Katie ; Mason, Thomas ; Becker, Mara L. ; Hong, Sandy ; Dedeoglu, Fatma ; Torok, Kathryn S. ; Rabinovich, C. Egla ; Ferguson, Polly J. ; Punaro, Marilynn ; Feldman, Brian M. ; Andrews, Tracy ; Higgins, Gloria C. ; Anderson, E. ; Francis, K. ; Goh, I. ; Jaquith, J. ; Schollaert-Fitch, K. ; Smith, C. ; Weiss, J. ; Wooton, J. / Developing comparative effectiveness studies for a rare, understudied pediatric disease : Lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study. In: Pediatric Rheumatology. 2019 ; Vol. 17, No. 1.
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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.",
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T1 - Developing comparative effectiveness studies for a rare, understudied pediatric disease

T2 - Lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study

AU - Li, Suzanne C.

AU - Fuhlbrigge, Robert C.

AU - Laxer, Ronald M.

AU - Pope, Elena

AU - Ibarra, Maria F.

AU - Stewart, Katie

AU - Mason, Thomas

AU - Becker, Mara L.

AU - Hong, Sandy

AU - Dedeoglu, Fatma

AU - Torok, Kathryn S.

AU - Rabinovich, C. Egla

AU - Ferguson, Polly J.

AU - Punaro, Marilynn

AU - Feldman, Brian M.

AU - Andrews, Tracy

AU - Higgins, Gloria C.

AU - Anderson, E.

AU - Francis, K.

AU - Goh, I.

AU - Jaquith, J.

AU - Schollaert-Fitch, K.

AU - Smith, C.

AU - Weiss, J.

AU - Wooton, J.

PY - 2019/7/15

Y1 - 2019/7/15

N2 - 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.

AB - 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.

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KW - Juvenile localized scleroderma

KW - Methotrexate

KW - Study design

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