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Title: | Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study |
Author: | Vázquez Costa, Juan F. Branas Pampillón, María Medina Cantillo, Julita Povedano, Mónica Pitarch Castellano, Inmaculada López Lobato, Mercedes Fernández Ramos, Joaquín A. Lafuente Hidalgo, Miguel Rojas García, Ricard Caballero Caballero, José M. Málaga, Ignacio Eirís Puñal, Jesús Lemus, Mencía De Cattinari, María G. Cabello Moruno, Rosana Díaz Abós, Paola Sánchez Menéndez, Victoria Rebollo, Pablo Maurino, Jorge Madruga Garrido, Marcos |
Keywords: | Qualitat de vida Atròfia muscular Quality of life Muscular atrophy |
Issue Date: | 21-Oct-2022 |
Publisher: | Springer Science and Business Media LLC |
Abstract: | Introduction Outcome measures traditionally used in spinal muscular atrophy (SMA) clinical trials are inadequate to assess the full range of disease severity. The aim of this study was to assess the psychometric properties of a set of existing questionnaires and new items, gathering information on the impact of SMA from the patient and caregiver perspectives. Methods This was a multicenter, prospective, noninterventional study including patients with a confirmed diagnosis of 5q-autosomal-recessive SMA aged 8 years and above, or their parents (if aged between 2 and 8 years). The set of outcome measurements included the SMA Independence Scale (SMAIS) patient and caregiver versions, the Neuro-QoL Fatigue Computer Adaptive Test (CAT), the Neuro-QoL Pain Short Form-Pediatric Pain, the PROMIS adult Pain Interference CAT, and new items developed by Fundacion Atrofia Muscular Espana: perceived fatigability, breathing and voice, sleep and rest, and vulnerability. Reliability, construct validity, discriminant validity, and sensitivity to change (4 months from baseline) were measured. Results A total of 113 patients were included (59.3% 2-17 years old, 59.3% male, and 50.4% with SMA type II). Patients required moderate assistance [mean patient and caregiver SMAIS (SD) scores were 31.1 (12.8) and 7.6 (11.1), respectively]. Perceived fatigability was the most impacted domain, followed by vulnerability. Cronbach's alpha coefficient for perceived fatigability, breathing and voice, and vulnerability total scores were 0.92, 0.88, and 0.85, respectively. The exploratory factor analysis identified the main factors considered in the design, except in the sleep and rest domain. All questionnaires were able to discriminate between the Clinical Global Impression-Severity scores and SMA types. Sensitivity to change was only found for the SMAIS caregiver version and vulnerability items. Conclusions This set of outcome measures showed adequate reliability, construct validity, and discriminant validity and may constitute a valuable option to measure symptom severity in patients with SMA. |
Note: | Reproducció del document publicat a: https://doi.org/10.1007/s40120-022-00411-2 |
It is part of: | Neurology and Therapy, 2022, vol. 12, num. 1, p. 89-105 |
URI: | http://hdl.handle.net/2445/205340 |
Related resource: | https://doi.org/10.1007/s40120-022-00411-2 |
ISSN: | 2193-6536 |
Appears in Collections: | Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL)) |
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