Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score
Introduction and objective: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often necessary in severe forms. This study aims to assess t...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-01-01
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| Series: | Journal of Market Access & Health Policy |
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| Online Access: | http://dx.doi.org/10.1080/20016689.2020.1748492 |
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| author | Andrea Leonardi Maëva Dupuis-Deniaud Dominique Bremond-Gignac |
| author_facet | Andrea Leonardi Maëva Dupuis-Deniaud Dominique Bremond-Gignac |
| author_sort | Andrea Leonardi |
| collection | DOAJ |
| description | Introduction and objective: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often necessary in severe forms. This study aims to assess the validity of a new composite endpoint: the penalties-adjusted corneal staining score (PACS-S) proposed as primary endpoint in VEKTIS trial evaluating the efficacy of a new corticosteroid-sparing treatment, VERKAZIA® (ciclosporin 1 mg/ml eye drops), in severe VKC patients. Methodology: This research comprised a systematic literature review to identify efficacy endpoints being proposed in clinical trials for pediatric patients with severe VKC, followed by a remote expert advisory board assessing the validity of the PACS-S. Results: While no agreed or validated endpoint for assessing efficacy in VKC was identified when VEKTIS trial started, the experts’ board acknowledged a high face validity of PACS-S as a subjective integrated measure matching the current clinical practice. A fair external validity was considered with regards to VEKTIS trial secondary endpoints. Conclusion: PACS-S appears to be a reliable, valid and clinically meaningful primary endpoint that allows significant improvement over existing endpoints in severe VKC trials. Additional research is needed to validate this endpoint. |
| format | Article |
| id | doaj-art-bbde86baff57484e87bb46715c8a4314 |
| institution | DOAJ |
| issn | 2001-6689 |
| language | English |
| publishDate | 2020-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj-art-bbde86baff57484e87bb46715c8a43142025-08-20T02:54:40ZengMDPI AGJournal of Market Access & Health Policy2001-66892020-01-018110.1080/20016689.2020.17484921748492Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining scoreAndrea Leonardi0Maëva Dupuis-Deniaud1Dominique Bremond-Gignac2University of PaduaMDSTAT ConsultingParis Descartes UniversityIntroduction and objective: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often necessary in severe forms. This study aims to assess the validity of a new composite endpoint: the penalties-adjusted corneal staining score (PACS-S) proposed as primary endpoint in VEKTIS trial evaluating the efficacy of a new corticosteroid-sparing treatment, VERKAZIA® (ciclosporin 1 mg/ml eye drops), in severe VKC patients. Methodology: This research comprised a systematic literature review to identify efficacy endpoints being proposed in clinical trials for pediatric patients with severe VKC, followed by a remote expert advisory board assessing the validity of the PACS-S. Results: While no agreed or validated endpoint for assessing efficacy in VKC was identified when VEKTIS trial started, the experts’ board acknowledged a high face validity of PACS-S as a subjective integrated measure matching the current clinical practice. A fair external validity was considered with regards to VEKTIS trial secondary endpoints. Conclusion: PACS-S appears to be a reliable, valid and clinically meaningful primary endpoint that allows significant improvement over existing endpoints in severe VKC trials. Additional research is needed to validate this endpoint.http://dx.doi.org/10.1080/20016689.2020.1748492severe vernal keratoconjunctivitisallergic keratoconjunctivitis penalties-adjusted corneal staining scoreendpointrescue therapyciclosporin |
| spellingShingle | Andrea Leonardi Maëva Dupuis-Deniaud Dominique Bremond-Gignac Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score Journal of Market Access & Health Policy severe vernal keratoconjunctivitis allergic keratoconjunctivitis penalties-adjusted corneal staining score endpoint rescue therapy ciclosporin |
| title | Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score |
| title_full | Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score |
| title_fullStr | Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score |
| title_full_unstemmed | Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score |
| title_short | Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score |
| title_sort | clinical efficacy assessment in severe vernal keratoconjunctivitis preliminary validation of a new penalties adjusted corneal fluorescein staining score |
| topic | severe vernal keratoconjunctivitis allergic keratoconjunctivitis penalties-adjusted corneal staining score endpoint rescue therapy ciclosporin |
| url | http://dx.doi.org/10.1080/20016689.2020.1748492 |
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