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Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)

INTRODUCTION: RLBP1 RP is an autosomal recessive form of retinitis pigmentosa (RP), characterized by night blindness, prolonged dark adaptation, constricted visual fields and impaired macular function. This study aimed to better understand the patient experience of RLBP1 RP and evaluate the content...

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Autores principales: Green, Jane, Tolley, Chloe, Bentley, Sarah, Arbuckle, Rob, Burstedt, Marie, Whelan, James, Holopigian, Karen, Stasi, Kali, Sloesen, Brigitte, Spera, Claudio, Deslandes, Jean-Yves, Mullins, Anmol
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7467452/
https://www.ncbi.nlm.nih.gov/pubmed/32372289
http://dx.doi.org/10.1007/s12325-020-01275-4
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author Green, Jane
Tolley, Chloe
Bentley, Sarah
Arbuckle, Rob
Burstedt, Marie
Whelan, James
Holopigian, Karen
Stasi, Kali
Sloesen, Brigitte
Spera, Claudio
Deslandes, Jean-Yves
Mullins, Anmol
author_facet Green, Jane
Tolley, Chloe
Bentley, Sarah
Arbuckle, Rob
Burstedt, Marie
Whelan, James
Holopigian, Karen
Stasi, Kali
Sloesen, Brigitte
Spera, Claudio
Deslandes, Jean-Yves
Mullins, Anmol
author_sort Green, Jane
collection PubMed
description INTRODUCTION: RLBP1 RP is an autosomal recessive form of retinitis pigmentosa (RP), characterized by night blindness, prolonged dark adaptation, constricted visual fields and impaired macular function. This study aimed to better understand the patient experience of RLBP1 RP and evaluate the content validity of existing patient reported outcome (PRO) instruments in this condition. METHODS: Semi-structured concept elicitation and cognitive debriefing interviews were conducted with RLBP1 RP patients in Canada and Sweden. Interviews started with open-ended concept elicitation questioning, and then patients were cognitively debriefed on The National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25), the Low Luminance Questionnaire (LLQ) and four light/dark adaptation items of the Visual Activities Questionnaire (VAQ). Qualitative interviews were also conducted with three expert clinicians. Anonymized, verbatim transcripts were analyzed using thematic analysis. RESULTS: Twenty-one patients were interviewed (Canada n = 10; Sweden n = 11). Symptoms reported included night blindness (n = 21), difficulty adapting to changes in lighting (n = 21) and difficulties seeing in bright lighting (n = 18). Patients experienced substantial impacts on daily activities (n = 21) and physical functioning (n = 17). Patients had difficulty interpreting and selecting a response for some items in the NEI VFQ-25 and LLQ. Some items were not relevant to patients’ disease experience. There were both gaps and overlaps in the conceptual coverage of the instruments. CONCLUSIONS: Visual impairment due to RLBP1 RP has a substantial impact on physical functioning and daily activities. To adequately assess all important symptoms and associated functional impacts in RLBP1 RP, it is recommended to either modify one or more existing instruments or to develop a new non-syndromic RP specific instrument. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s12325-020-01275-4) contains supplementary material, which is available to authorized users.
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spelling pubmed-74674522020-09-11 Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP) Green, Jane Tolley, Chloe Bentley, Sarah Arbuckle, Rob Burstedt, Marie Whelan, James Holopigian, Karen Stasi, Kali Sloesen, Brigitte Spera, Claudio Deslandes, Jean-Yves Mullins, Anmol Adv Ther Original Research INTRODUCTION: RLBP1 RP is an autosomal recessive form of retinitis pigmentosa (RP), characterized by night blindness, prolonged dark adaptation, constricted visual fields and impaired macular function. This study aimed to better understand the patient experience of RLBP1 RP and evaluate the content validity of existing patient reported outcome (PRO) instruments in this condition. METHODS: Semi-structured concept elicitation and cognitive debriefing interviews were conducted with RLBP1 RP patients in Canada and Sweden. Interviews started with open-ended concept elicitation questioning, and then patients were cognitively debriefed on The National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25), the Low Luminance Questionnaire (LLQ) and four light/dark adaptation items of the Visual Activities Questionnaire (VAQ). Qualitative interviews were also conducted with three expert clinicians. Anonymized, verbatim transcripts were analyzed using thematic analysis. RESULTS: Twenty-one patients were interviewed (Canada n = 10; Sweden n = 11). Symptoms reported included night blindness (n = 21), difficulty adapting to changes in lighting (n = 21) and difficulties seeing in bright lighting (n = 18). Patients experienced substantial impacts on daily activities (n = 21) and physical functioning (n = 17). Patients had difficulty interpreting and selecting a response for some items in the NEI VFQ-25 and LLQ. Some items were not relevant to patients’ disease experience. There were both gaps and overlaps in the conceptual coverage of the instruments. CONCLUSIONS: Visual impairment due to RLBP1 RP has a substantial impact on physical functioning and daily activities. To adequately assess all important symptoms and associated functional impacts in RLBP1 RP, it is recommended to either modify one or more existing instruments or to develop a new non-syndromic RP specific instrument. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s12325-020-01275-4) contains supplementary material, which is available to authorized users. Springer Healthcare 2020-05-05 2020 /pmc/articles/PMC7467452/ /pubmed/32372289 http://dx.doi.org/10.1007/s12325-020-01275-4 Text en © The Author(s) 2020 Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder.To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
spellingShingle Original Research
Green, Jane
Tolley, Chloe
Bentley, Sarah
Arbuckle, Rob
Burstedt, Marie
Whelan, James
Holopigian, Karen
Stasi, Kali
Sloesen, Brigitte
Spera, Claudio
Deslandes, Jean-Yves
Mullins, Anmol
Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title_full Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title_fullStr Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title_full_unstemmed Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title_short Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP)
title_sort qualitative interviews to better understand the patient experience and evaluate patient-reported outcomes (pro) in rlbp1 retinitis pigmentosa (rlbp1 rp)
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7467452/
https://www.ncbi.nlm.nih.gov/pubmed/32372289
http://dx.doi.org/10.1007/s12325-020-01275-4
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