Randomized clinical trials in ANCA-associated vasculitis : a systematic analysis of the WHO - International Clinical Trials Registry Platform
BACKGROUND: The analysis of the main features of randomized controlled trials (RCTs) on ANCA-associated vasculitis (AAV) can inform future study design.
METHODS: We searched within the International Clinical Trials Registry Platform all registered RCTs on AAV from October 2008 to December 2018. Two reviewers selected studies according to pre-specified eligibility criteria. We retrieved information including countries, funding, design, sample sizes, eligibility criteria, primary outcomes (POs), and treatments.
RESULTS: Among the 40 RCTs identified, 22 (55%) were conducted in Europe, 29 (72,5%) in a single country, 14 (35%) were industry-funded. The median number of patients planned to enrol was 68 (IQR 36-138). Only 28% of RCTs targeted a single vasculitis, and ANCA negative patients were not included in about 40% of studies. Interventions investigated were mainly drugs given to induce (40%) or maintain (32.5%) remission. Eighty-five percent of POs were considered being 'patient-important', but discrepancies in definition of disease states, such as remission or relapse were observed. Glucocorticoids use was part of the PO in < 25% of studies. The number of trials targeting a single disease, non-industry funded, incorporating glucocorticoids in PO, as well as the planned sample size increased over time.
CONCLUSION: Despite the important achievements in the field, a better harmonization of eligibility, and outcome criteria across studies is an important objective to pursue in next future.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2020 |
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| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:15 |
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| Enthalten in: |
Orphanet journal of rare diseases - 15(2020), 1 vom: 29. Mai, Seite 130 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Iudici, Michele [VerfasserIn] |
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| Links: |
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| Themen: |
ANCA-vasculitis |
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| Anmerkungen: |
Date Completed 18.06.2021 Date Revised 18.06.2021 published: Electronic Citation Status MEDLINE |
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| doi: |
10.1186/s13023-020-01408-6 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM310519543 |
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| 100 | 1 | |a Iudici, Michele |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Randomized clinical trials in ANCA-associated vasculitis |b a systematic analysis of the WHO - International Clinical Trials Registry Platform |
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| 500 | |a Date Revised 18.06.2021 | ||
| 500 | |a published: Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a BACKGROUND: The analysis of the main features of randomized controlled trials (RCTs) on ANCA-associated vasculitis (AAV) can inform future study design | ||
| 520 | |a METHODS: We searched within the International Clinical Trials Registry Platform all registered RCTs on AAV from October 2008 to December 2018. Two reviewers selected studies according to pre-specified eligibility criteria. We retrieved information including countries, funding, design, sample sizes, eligibility criteria, primary outcomes (POs), and treatments | ||
| 520 | |a RESULTS: Among the 40 RCTs identified, 22 (55%) were conducted in Europe, 29 (72,5%) in a single country, 14 (35%) were industry-funded. The median number of patients planned to enrol was 68 (IQR 36-138). Only 28% of RCTs targeted a single vasculitis, and ANCA negative patients were not included in about 40% of studies. Interventions investigated were mainly drugs given to induce (40%) or maintain (32.5%) remission. Eighty-five percent of POs were considered being 'patient-important', but discrepancies in definition of disease states, such as remission or relapse were observed. Glucocorticoids use was part of the PO in < 25% of studies. The number of trials targeting a single disease, non-industry funded, incorporating glucocorticoids in PO, as well as the planned sample size increased over time | ||
| 520 | |a CONCLUSION: Despite the important achievements in the field, a better harmonization of eligibility, and outcome criteria across studies is an important objective to pursue in next future | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a ANCA-vasculitis | |
| 650 | 4 | |a Epidemiology | |
| 650 | 4 | |a Randomized controlled trial | |
| 700 | 1 | |a Puéchal, Xavier |e verfasserin |4 aut | |
| 700 | 1 | |a Brigante, Alejandro |e verfasserin |4 aut | |
| 700 | 1 | |a Atal, Ignacio |e verfasserin |4 aut | |
| 700 | 1 | |a Gabay, Cem |e verfasserin |4 aut | |
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