- ASA 2021 Statistics and Information Systems for Policy Evaluation
- Edited by Bruno Bertaccini, Luigi Fabbris, Alessandra Petrucci
Patient-generated evidence in Epidermolysis Bullosa (EB): Development of a questionnaire to assess the Quality of Life
- Laura Benedan
- May El Hachem
- Carlotta Galeone
- Paolo Mariani
- Cinzia Pilo
- Gianluca Tadini
- © 2021 Author(s) |
- CC BY 4.0
- DOI: 10.36253/978-88-5518-461-8.38
Epidermolysis Bullosa (EB) is a group of genetic conditions that cause fragile and blistering skin. Although there are different types of EB, which differ in severity, their signs and symptoms overlap. As a result of this disorder, patients face an unbearable burden in their lives, and their Quality of Life (QoL) is negatively affected at every life cycle stage. Nevertheless, the assessment of the quality of life of these patients is scanty. This project aims to develop a patient-centered questionnaire to assess the QoL of EB patients. This tool will be a valid aid for clinicians to understand patients better and identify the areas that need more attention; moreover, it will allow them to follow the patients over time and evaluate the impact of any treatments. The methodological process to develop the questionnaire consisted of two phases: firstly, a critical review of scientific literature was performed; secondly, a pseudo-Delphi study was carried out. A multidisciplinary panel (including patients, caregivers, and clinicians) actively participated in round tables to discuss the main areas of interest. Starting from this initial set of areas and through the repetition of Delphi (up to three rounds), a gradual refinement of the statements was carried out to define a list of items to be included in an easy-to-use but meaningful questionnaire. The final patient-centered questionnaire is thus able to measure the QoL beyond the physical symptoms and the clinical evolution of the disease, encompassing functional autonomy, psycho-emotional state, social relations and the working field.
- Keywords:
- Epidermolysis Bullosa,
- Patient-centered approach,
- Quality of Life,
- Pseudo-Delphi,
University of Milano-Bicocca, Italy - ORCID: 0000-0003-0427-2487
Bambino Gesù Children's Hospital, Italy - ORCID: 0000-0002-8145-4797
University of Milano-Bicocca, Italy - ORCID: 0000-0003-1934-5167
University of Milano-Bicocca, Italy - ORCID: 0000-0002-8848-8893
REB Onlus Foundation, Italy
University of Milan, Italy - ORCID: 0000-0003-1164-2802
- Cestari, T., Prati, C., Menegon, D. B., Prado Oliveira, Z. N., Machado, M. C. R., Dumet, J., … Murrell, D. F. (2016). Translation, cross-cultural adaptation and validation of the Quality of Life Evaluation in Epidermolysis Bullosa instrument in Brazilian Portuguese. International Journal of Dermatology, 55(2), pp. e94–e99.
- Dănescu, S., Sălăvăstru, C., Sendrea, A., Tiplica, S., Baican, A., Ungureanu, L., … Cosgarea, R. (2019). Correlation between disease severity and quality of life in patients with epidermolysis bullosa. Journal of the European Academy of Dermatology and Venereology, 33(5), pp. e217–e219.
- Dures, E., Morris, M., Gleeson, K., & Rumsey, N. (2011). The psychosocial impact of epidermolysis bullosa. Qualitative Health Research, 21(6), pp.771–782.
- Fine, J. D. (2016). Epidemiology of inherited epidermolysis bullosa based on incidence and prevalence estimates from the national epidermolysis bullosa registry. JAMA Dermatology, 152(11), pp. 1231–1238.
- Frew, J. W., Cepeda Valdes, R., Fortuna, G., Murrell, D. F., & Salas Alanis, J. (2013). Measuring quality of life in epidermolysis bullosa in Mexico: Cross-cultural validation of the Hispanic version of the Quality of Life in Epidermolysis Bullosa questionnaire. Journal of the American Academy of Dermatology, 69(4), pp. 652–653.
- Frew, J. W., Martin, L. K., Nijsten, T., & Murrell, D. F. (2009). Quality of life evaluation in epidermolysis bullosa (EB) through the development of the QOLEB questionnaire: An EBspecific quality of life instrument. British Journal of Dermatology, 161(6), pp. 1323–1330.
- Has, C., Bauer, J. W., Bodemer, C., Bolling, M. C., Bruckner-Tuderman, L., Diem, A., … Mellerio, J. E. (2020). Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility. British Journal of Dermatology, 183(4), pp. 614–627.
- Marbach, G., Mazziotta, C., & Rizzi, A. (1991). Le previsioni. Fondamenti logici e basi statistiche. Milano: edizioni Etaslibri.
- Tadini, G., Gualandri, L., Colombi, M., Paradisi, M., Angelo, C., Zambruno, G., … Cainelli, T (2005). The Italian registry of hereditary epidermolysis bullosa. Giornale Italiano di Dermatologia e Venereologia, 140(4), pp. 359-72.
- Trevelyan, E. G., & Robinson, N. (2015). Delphi methodology in health research: How to do it? European Journal of Integrative Medicine, 7(4), pp. 423–428.
- Yuen, W. Y., Frew, J. W., Veerman, K., van den Heuvel, E. R., Murrell, D. F., & Jonkman, M. F. (2014). Health-related quality of life in epidermolysis bullosa: Validation of the dutch QOLEB questionnaire and assessment in the dutch population. Acta Dermato-Venereologica, 94(4), pp. 442–447.
Chapter Information
Chapter Title
Patient-generated evidence in Epidermolysis Bullosa (EB): Development of a questionnaire to assess the Quality of Life
Authors
Laura Benedan, May El Hachem, Carlotta Galeone, Paolo Mariani, Cinzia Pilo, Gianluca Tadini
Language
English
DOI
10.36253/978-88-5518-461-8.38
Peer Reviewed
Publication Year
2021
Copyright Information
© 2021 Author(s)
Content License
Metadata License
Bibliographic Information
Book Title
ASA 2021 Statistics and Information Systems for Policy Evaluation
Book Subtitle
BOOK OF SHORT PAPERS of the on-site conference
Editors
Bruno Bertaccini, Luigi Fabbris, Alessandra Petrucci
Peer Reviewed
Publication Year
2021
Copyright Information
© 2021 Author(s)
Content License
Metadata License
Publisher Name
Firenze University Press
DOI
10.36253/978-88-5518-461-8
eISBN (pdf)
978-88-5518-461-8
eISBN (xml)
978-88-5518-462-5
Series Title
Proceedings e report
Series ISSN
2704-601X
Series E-ISSN
2704-5846