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Research
Michele IUDICI
Hopitaux Universitaires de Geneve
Corresponding Author
ORCiD: https://orcid.org/0000-0001-5871-8806
License:
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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 (38%) were conducted in Europe, 29 (72%) 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 40% of studies. Interventions investigated were mainly drugs given to induce (40%) or maintain (32%) 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, the design of RCTs on AAV could be further improved.
Keywords
ANCA-vasculitis, randomized controlled trial, epidemiology.
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CURRENT STATUS: Published
View the published article at Orphanet Journal of Rare Diseases.
Version 2
Posted 14 May, 2020
No community comments so far
Editorial decision: Accept
On 08 May, 2020
Reviewer #2 agreed
On 05 May, 2020
Review #2 received
Received 05 May, 2020
Editor assigned
On 30 Apr, 2020
Reviewers invited
Invitations sent on 30 Apr, 2020
Reviewer #1 agreed
On 30 Apr, 2020
Review #1 received
Received 30 Apr, 2020
Submission checks complete
On 29 Apr, 2020
Editor invited
On 29 Apr, 2020
No comments provided
Review #2 received
Received 21 Apr, 2020
Editorial decision: Minor revision
On 21 Apr, 2020
Review #1 received
Received 17 Apr, 2020
Reviewer #2 agreed
On 07 Apr, 2020
Editor assigned
On 02 Apr, 2020
Reviewers invited
Invitations sent on 02 Apr, 2020
Reviewer #1 agreed
On 02 Apr, 2020
First submitted
On 01 Apr, 2020
Submission checks complete
On 01 Apr, 2020
Editor invited
On 01 Apr, 2020
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Internal Medicine
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A new preprint design is coming!
We have improved readability, clarity, accessibility, and opportunities for engagement.
See the published version of this preprint at Orphanet Journal of Rare Diseases.
This is a preprint, a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
Learn more about In Review
Research
Michele IUDICI
Hopitaux Universitaires de Geneve
Corresponding Author
ORCiD: https://orcid.org/0000-0001-5871-8806
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
Peer Review Timeline
CURRENT STATUS: Published
View the published article at Orphanet Journal of Rare Diseases.
Version 2
Posted 14 May, 2020
No community comments so far
Editorial decision: Accept
On 08 May, 2020
Reviewer #2 agreed
On 05 May, 2020
Review #2 received
Received 05 May, 2020
Editor assigned
On 30 Apr, 2020
Reviewers invited
Invitations sent on 30 Apr, 2020
Reviewer #1 agreed
On 30 Apr, 2020
Review #1 received
Received 30 Apr, 2020
Submission checks complete
On 29 Apr, 2020
Editor invited
On 29 Apr, 2020
No comments provided
Review #2 received
Received 21 Apr, 2020
Editorial decision: Minor revision
On 21 Apr, 2020
Review #1 received
Received 17 Apr, 2020
Reviewer #2 agreed
On 07 Apr, 2020
Editor assigned
On 02 Apr, 2020
Reviewers invited
Invitations sent on 02 Apr, 2020
Reviewer #1 agreed
On 02 Apr, 2020
First submitted
On 01 Apr, 2020
Submission checks complete
On 01 Apr, 2020
Editor invited
On 01 Apr, 2020
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Internal Medicine
License:
This work is licensed under a CC BY 4.0 License. Read Full License
View the published article at Orphanet Journal of Rare Diseases.
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 (38%) were conducted in Europe, 29 (72%) 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 40% of studies. Interventions investigated were mainly drugs given to induce (40%) or maintain (32%) 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, the design of RCTs on AAV could be further improved.
Figure 1
Figure 2
Figure 3
Figure 4
This is a list of supplementary files associated with this preprint. Click to download.
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