Development and content validation of a sunlight exposure diary in patients with erythropoietic protoporphyria.
Erythropoietic protoporphyria
Patient reported outcomes
Quality of life
Sun exposure diary
Journal
Journal of patient-reported outcomes
ISSN: 2509-8020
Titre abrégé: J Patient Rep Outcomes
Pays: Germany
ID NLM: 101722688
Informations de publication
Date de publication:
20 Nov 2023
20 Nov 2023
Historique:
received:
11
05
2023
accepted:
10
11
2023
medline:
21
11
2023
pubmed:
20
11
2023
entrez:
20
11
2023
Statut:
epublish
Résumé
Erythropoietic protoporphyria is a rare, inherited disorder presenting in early childhood with severe, painful phototoxicity. EPP has significant impacts on health-related quality of life, though there is variable disease severity. Accurately capturing how much time individuals with EPP can spend outdoors before they develop symptoms is critical to understanding HRQoL and measuring therapeutic response. Therefore, the goal of this study was to develop a comprehensive and content valid sun exposure diary to assess the efficacy of new therapies in individuals with EPP. Qualitative interviews were conducted with adult and adolescent EPP participants, as well as five clinical experts, to obtain their input on the content of an existing sun exposure diary. Revisions to the diary were made based on evidence generated in cognitive debriefing interviews analyzed in eight consecutive groups of EPP participant. Interviews were conducted with 17 adults and 6 adolescents with EPP. The average age of adults was 40 years and of adolescents was 14 years. Clinical experts thought the original diary needed clarification on the description of symptoms, how time outdoors was captured, and the distinction between direct vs. indirect sunlight. Participants with EPP also noted these items needed revision, and that the distinction between prodromal symptoms and full reaction symptoms should be clarified. In the final diary version, participants with EPP found most items to be clear and easy to complete/think about. Seventy-six percent of participants (13/17) asked thought the diary was easy to complete. The remainder thought the majority of the diary was easy to complete with the exception of select questions. Evaluating a new treatment for EPP requires accurately capturing time in sunlight and symptoms in this unique disorder. The newly developed sun exposure diary is content valid and can be used to assess important aspects of symptoms and daily life and therefore evaluate clinically meaningful therapeutic response.
Sections du résumé
BACKGROUND
BACKGROUND
Erythropoietic protoporphyria is a rare, inherited disorder presenting in early childhood with severe, painful phototoxicity. EPP has significant impacts on health-related quality of life, though there is variable disease severity. Accurately capturing how much time individuals with EPP can spend outdoors before they develop symptoms is critical to understanding HRQoL and measuring therapeutic response. Therefore, the goal of this study was to develop a comprehensive and content valid sun exposure diary to assess the efficacy of new therapies in individuals with EPP.
METHODS
METHODS
Qualitative interviews were conducted with adult and adolescent EPP participants, as well as five clinical experts, to obtain their input on the content of an existing sun exposure diary. Revisions to the diary were made based on evidence generated in cognitive debriefing interviews analyzed in eight consecutive groups of EPP participant.
RESULTS
RESULTS
Interviews were conducted with 17 adults and 6 adolescents with EPP. The average age of adults was 40 years and of adolescents was 14 years. Clinical experts thought the original diary needed clarification on the description of symptoms, how time outdoors was captured, and the distinction between direct vs. indirect sunlight. Participants with EPP also noted these items needed revision, and that the distinction between prodromal symptoms and full reaction symptoms should be clarified. In the final diary version, participants with EPP found most items to be clear and easy to complete/think about. Seventy-six percent of participants (13/17) asked thought the diary was easy to complete. The remainder thought the majority of the diary was easy to complete with the exception of select questions.
CONCLUSIONS
CONCLUSIONS
Evaluating a new treatment for EPP requires accurately capturing time in sunlight and symptoms in this unique disorder. The newly developed sun exposure diary is content valid and can be used to assess important aspects of symptoms and daily life and therefore evaluate clinically meaningful therapeutic response.
Identifiants
pubmed: 37982964
doi: 10.1186/s41687-023-00655-y
pii: 10.1186/s41687-023-00655-y
pmc: PMC10660587
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
119Informations de copyright
© 2023. The Author(s).
Références
Mol Genet Metab. 2019 Nov;128(3):314-319
pubmed: 30711301
J Intern Med. 2011 Mar;269(3):270-4
pubmed: 21332583
Br J Dermatol. 2008 Jul;159(1):192-7
pubmed: 18460025
N Engl J Med. 2015 Jul 2;373(1):48-59
pubmed: 26132941
J Patient Rep Outcomes. 2023 Nov 20;7(1):119
pubmed: 37982964
JAMA Dermatol. 2017 Aug 1;153(8):789-796
pubmed: 28614581
Psychother Psychosom Med Psychol. 1992 Sep-Oct;42(9-10):339-48
pubmed: 1287700
JIMD Rep. 2019 Sep 14;50(1):9-19
pubmed: 31741822
Br J Dermatol. 2006 Sep;155(3):574-81
pubmed: 16911284
Orphanet J Rare Dis. 2009 Sep 10;4:19
pubmed: 19744342
Mol Genet Metab. 2019 Nov;128(3):298-303
pubmed: 30704898