Validation of the Index for Facial Angiofibromas: Data analysis from a randomized controlled trial of sirolimus gel treatment in patients with tuberous sclerosis complex Article Swipe
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· 2024
· Open Access
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· DOI: https://doi.org/10.1111/1346-8138.17220
The Index for Facial Angiofibromas (IFA), a novel scoring system for angiofibromas, has been validated in patients with tuberous sclerosis complex (TSC). The objective of this analysis was to further validate the IFA using data from a clinical trial of topical sirolimus in patients with TSC. This was an analysis of photographs from a Phase III trial conducted in Japan (NCT02635789). Patients ( n = 62) were randomized 1:1 to receive sirolimus or placebo gel for 12 weeks. Changes in angiofibromas were independently assessed using the primary composite endpoint, the Facial Angiofibroma Severity Index (FASI), and the IFA. Thresholds for a clinically meaningful change in IFA score were evaluated using receiver operating characteristic (ROC) analysis. The IFA scores had good‐to‐excellent inter‐assessor reliability, very high intra‐assessor reliability, and could be used to evaluate the distribution of disease severity at baseline. High correlations were observed between the categorized change from baseline in IFA scores and the primary composite endpoint (Kendall's coefficient of concordance, W = 0.8655, p < 0.0001), and between the change from baseline in IFA and FASI scores (Kendall's coefficient of concordance, W = 0.745, p < 0.0001). By ROC analysis, an optimal IFA cut‐off point of 1.667 was determined to distinguish patients with markedly improved or improved angiofibromas from those with slightly improved or unchanged angiofibromas (area under the curve 0.937) as determined by the primary composite endpoint. The IFA score is potentially clinically useful because of its high validity and reliability. A decrease in score from baseline of ≥1.667 may be considered clinically meaningful.
Related Topics
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1111/1346-8138.17220
- https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/1346-8138.17220
- OA Status
- hybrid
- Cited By
- 1
- References
- 13
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4394809244
Raw OpenAlex JSON
- OpenAlex ID
-
https://openalex.org/W4394809244Canonical identifier for this work in OpenAlex
- DOI
-
https://doi.org/10.1111/1346-8138.17220Digital Object Identifier
- Title
-
Validation of the Index for Facial Angiofibromas: Data analysis from a randomized controlled trial of sirolimus gel treatment in patients with tuberous sclerosis complexWork title
- Type
-
articleOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2024Year of publication
- Publication date
-
2024-04-15Full publication date if available
- Authors
-
Izumi Hamada, Yoshinori Yukutake, Yusuke Morita, Norifumi Ishikawa, Kenji Shimizu, Mari Wataya‐KanedaList of authors in order
- Landing page
-
https://doi.org/10.1111/1346-8138.17220Publisher landing page
- PDF URL
-
https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/1346-8138.17220Direct link to full text PDF
- Open access
-
YesWhether a free full text is available
- OA status
-
hybridOpen access status per OpenAlex
- OA URL
-
https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/1346-8138.17220Direct OA link when available
- Concepts
-
Medicine, Concordance, Clinical endpoint, Tuberous sclerosis, Angiofibroma, Receiver operating characteristic, Placebo, Randomized controlled trial, Internal medicine, Surgery, Pathology, Alternative medicineTop concepts (fields/topics) attached by OpenAlex
- Cited by
-
1Total citation count in OpenAlex
- Citations by year (recent)
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2024: 1Per-year citation counts (last 5 years)
- References (count)
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13Number of works referenced by this work
- Related works (count)
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10Other works algorithmically related by OpenAlex
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