Validation of the Italian version of a patient-reported outcome measure for Hereditary Spastic Paraplegia

Eleonora Diella; Maria Grazia D'Angelo; Cristina Stefan; Giulia Girardi; Roberta Morganti; Andrea Martinuzzi; Emilia Biffi

doi:10.1371/journal.pone.0301452

Validation of the Italian version of a patient-reported outcome measure for Hereditary Spastic Paraplegia

PLoS One. 2024 Apr 1;19(4):e0301452. doi: 10.1371/journal.pone.0301452. eCollection 2024.

Authors

Eleonora Diella¹, Maria Grazia D'Angelo¹, Cristina Stefan², Giulia Girardi², Roberta Morganti¹, Andrea Martinuzzi³, Emilia Biffi¹

Affiliations

¹ Scientific Institute, IRCCS E. Medea, Bosisio Parini, Lecco, Italy.
² Scientific Institute, IRCCS E. Medea, Pieve di Soligo, Treviso, Italy.
³ Scientific Institute, IRCCS E. Medea, Conegliano, Treviso, Italy.

Abstract

Background and aim: Patient-reported outcome measures (PROMs) are recognized as valuable measures in the clinical setting. In 2018 we developed the Italian version of the "Hereditary Spastic Paraplegia-Self Notion and Perception Questionnaire" (HSP-SNAP), a disease-specific questionnaire that collects personal perception on motor symptoms related to HSP such as stiffness, weakness, imbalance, reduced endurance, fatigue and pain. In this study our primary aim was to assess the questionnaire validity and reliability. Our secondary aim was to characterize the symptoms "perceived" by patients with HSP and compare them with those "perceived" by age-matched healthy subjects.

Methods: The 12-item HSP-SNAP questionnaire was submitted to 20 external judges for comprehensibility and to 15 external judges for content validity assessment. We recruited 40 subjects with HSP and asked them to fill the questionnaire twice for test-retest procedure. They also completed the Medical Outcome Survey Short Form (SF-36) and were evaluated by the Spastic Paraplegia Rating Scale and the Six-Minute Walk Test. We also recruited 44 healthy subjects who completed the HSP-SNAP once to test score variability.

Results: The HSP-SNAP content validity index was high (0.8±0.1) and the test-retest analysis showed high reliability (ICC = 0.94). The mean HSP-SNAP score (score range 0-48) of the HSP group was 22.2±7.8, which was significantly lower than healthy subjects (43.1±6.3). The most commonly perceived symptom was stiffness, followed by weakness and imbalance.

Conclusion: Although HSP-SNAP does not investigate non-motor symptoms and we validated only its Italian version, it showed good validity and reliability and it could be used in combination with other objective outcome measures for clinical purposes or as endpoints for future clinical rehabilitation studies.

Trial registration: Trial Registration: ClinicalTrial.gov, NCT04256681. Registered 3 February 2020.

Copyright: © 2024 Diella et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

MeSH terms

Humans
Italy
Paraplegia
Patient Reported Outcome Measures
Reproducibility of Results
Spastic Paraplegia, Hereditary* / diagnosis

Associated data

ClinicalTrials.gov/NCT04256681

Grants and funding

This work was supported by Fondazione Cariplo and Regione Lombardia (EMPATIA@Lecco project) and by the Italian Ministry of Health (RC2022/2024 to E. Biffi). There was no additional external funding received for this study.