Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial
Abstract Background The effectiveness of biologic therapies, primarily tumor necrosis factor inhibitors (TNFi), for children with spondyloarthritis (SpA) has made inactive disease a realistic patient outcome. However, biologic therapies are costly, primarily delivered by subcutaneous or intravenous...
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| Format: | Article |
| Language: | English |
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BMC
2023-02-01
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| Series: | Trials |
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| Online Access: | https://doi.org/10.1186/s13063-022-07038-6 |
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| author | Pamela F. Weiss Cora E. Sears Timothy G. Brandon Christopher B. Forrest Emily Neu Melanie Kohlheim Jenny Leal Rui Xiao Daniel Lovell |
| author_facet | Pamela F. Weiss Cora E. Sears Timothy G. Brandon Christopher B. Forrest Emily Neu Melanie Kohlheim Jenny Leal Rui Xiao Daniel Lovell |
| author_sort | Pamela F. Weiss |
| collection | DOAJ |
| description | Abstract Background The effectiveness of biologic therapies, primarily tumor necrosis factor inhibitors (TNFi), for children with spondyloarthritis (SpA) has made inactive disease a realistic patient outcome. However, biologic therapies are costly, primarily delivered by subcutaneous or intravenous route, and have non-trivial side effects. Many patients and families want to know if biologic medications can be discontinued after inactive disease is achieved. It remains unclear whether medication dose should remain unchanged, tapered (increase the time between doses), or discontinued once when inactive disease is attained. Methods The Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile SpA (BACK-OFF JSpA) trial is a multicenter pragmatic trial that will randomize 198 participants ages 8–21 years old with SpA and sustained inactive disease on standard TNFi dosing to (1) continue standard TNFi dosing, (2) fixed longer dosing intervals of TNFi, or (3) stop TNFi. The trial will compare the hazard rate of protocol-defined flare and participants’ emotional health among the 3 groups over 12 months. Innovative aspects of this trial are the involvement of patient and parent stakeholders in the design and conduct of the study as well as an electronic health record-based enhanced recruitment strategy. Discussion This is the first randomized pragmatic trial to assess the efficacy of TNFi de-escalation strategies in children with JSpA with sustained inactive disease. This research will improve the evidence base that patients, caregivers, and rheumatologists use to make shared decisions about continued treatment versus de-escalation of TNFi therapy in this population. Trial registration ClinicalTrials.gov NCT04891640. Registered on 18 May 2021. |
| first_indexed | 2024-04-10T15:42:28Z |
| format | Article |
| id | doaj.art-d1702cf8534e4c1ebb23eaa08b57fc0c |
| institution | Directory Open Access Journal |
| issn | 1745-6215 |
| language | English |
| last_indexed | 2024-04-10T15:42:28Z |
| publishDate | 2023-02-01 |
| publisher | BMC |
| record_format | Article |
| series | Trials |
| spelling | doaj.art-d1702cf8534e4c1ebb23eaa08b57fc0c2023-02-12T12:21:45ZengBMCTrials1745-62152023-02-0124111210.1186/s13063-022-07038-6Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trialPamela F. Weiss0Cora E. Sears1Timothy G. Brandon2Christopher B. Forrest3Emily NeuMelanie Kohlheim4Jenny LealRui Xiao5Daniel Lovell6Division of Rheumatology and Center for Pediatric Clinical Effectiveness, Roberts Center for Pediatric Research, Children’s Hospital of PhiladelphiaDivision of Rheumatology and Center for Pediatric Clinical Effectiveness, Roberts Center for Pediatric Research, Children’s Hospital of PhiladelphiaDivision of Rheumatology and Center for Pediatric Clinical Effectiveness, Roberts Center for Pediatric Research, Children’s Hospital of PhiladelphiaApplied Clinical Research Center, Children’s Hospital of PhiladelphiaDivision of Rheumatology and Center for Pediatric Clinical Effectiveness, Roberts Center for Pediatric Research, Children’s Hospital of PhiladelphiaDepartment of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of PennsylvaniaDepartment of Pediatrics and Division of Rheumatology at Cincinnati Children’s Hospital Medical Center, University of CincinnatiAbstract Background The effectiveness of biologic therapies, primarily tumor necrosis factor inhibitors (TNFi), for children with spondyloarthritis (SpA) has made inactive disease a realistic patient outcome. However, biologic therapies are costly, primarily delivered by subcutaneous or intravenous route, and have non-trivial side effects. Many patients and families want to know if biologic medications can be discontinued after inactive disease is achieved. It remains unclear whether medication dose should remain unchanged, tapered (increase the time between doses), or discontinued once when inactive disease is attained. Methods The Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile SpA (BACK-OFF JSpA) trial is a multicenter pragmatic trial that will randomize 198 participants ages 8–21 years old with SpA and sustained inactive disease on standard TNFi dosing to (1) continue standard TNFi dosing, (2) fixed longer dosing intervals of TNFi, or (3) stop TNFi. The trial will compare the hazard rate of protocol-defined flare and participants’ emotional health among the 3 groups over 12 months. Innovative aspects of this trial are the involvement of patient and parent stakeholders in the design and conduct of the study as well as an electronic health record-based enhanced recruitment strategy. Discussion This is the first randomized pragmatic trial to assess the efficacy of TNFi de-escalation strategies in children with JSpA with sustained inactive disease. This research will improve the evidence base that patients, caregivers, and rheumatologists use to make shared decisions about continued treatment versus de-escalation of TNFi therapy in this population. Trial registration ClinicalTrials.gov NCT04891640. Registered on 18 May 2021.https://doi.org/10.1186/s13063-022-07038-6SpondyloarthritisPediatricsBiologicRandomized pragmatic trialElectronic Health RecordsPatient-reported outcome |
| spellingShingle | Pamela F. Weiss Cora E. Sears Timothy G. Brandon Christopher B. Forrest Emily Neu Melanie Kohlheim Jenny Leal Rui Xiao Daniel Lovell Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial Trials Spondyloarthritis Pediatrics Biologic Randomized pragmatic trial Electronic Health Records Patient-reported outcome |
| title | Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial |
| title_full | Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial |
| title_fullStr | Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial |
| title_full_unstemmed | Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial |
| title_short | Biologic Abatement and Capturing Kids’ Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK-OFF JSpA): study protocol for a randomized pragmatic trial |
| title_sort | biologic abatement and capturing kids outcomes and flare frequency in juvenile spondyloarthritis back off jspa study protocol for a randomized pragmatic trial |
| topic | Spondyloarthritis Pediatrics Biologic Randomized pragmatic trial Electronic Health Records Patient-reported outcome |
| url | https://doi.org/10.1186/s13063-022-07038-6 |
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