RESUMEN
Background: The primary aim of this study was to explore current caregivers' expectations on possible functional changes following treatment in comparison to data obtained in the pre-pharmacological era. Methods: A questionnaire, previously used in 2016, was administered to caregivers of type II and III SMA patients of age between 3 and 71 years, and to patients over the age of 13 years. The questionnaire focuses on (1) caregivers and patients expectations, (2) meaningfulness of the changes observed on the functional motor scales, and (3) their willingness to be enrolled in a clinical trial. A comparative study was performed with data obtained using the same questionnaire soon before the advent of disease-modifying therapies. Results: We administered the questionnaire to 150 caregivers. When comparing current caregiver data to those obtained in 2016, the most obvious differences were related to disease perception over the last year (stability: 16.5% in 2016 vs. 43.6% in 2022; deterioration 70.5% vs. 12.8%, and improvement: 12.9% vs. 43.6%) and expectations from clinical trials with higher expectations in 2022 compared to 2016 (p < 0.001). Forty-five of the 150 in the current study were caregivers of patients above the age of 13. In these 45 the questionnaire was also administered to the patient. No difference was found in responses between patients and their caregivers. Conclusions: Both carers and patients reported that even small changes on functional scales, similar to those reported by clinical studies and real-world data, are perceived as meaningful. Comparing the recent responses to those obtained in 2016, before pharmacological treatment was available, we found significant changes in caregivers' perception with increased expectations. These findings will provide a better understanding of the patients' expectations and facilitate discussion with regulators.
RESUMEN
Women who received a diagnosis of breast cancer often report impairments in physical and psychological wellbeing, even some years after treatments. Individual awareness about physical changes, body image, and current sensations related to their body is important to maintain a psycho-emotional balance. Virtual reality, as an advanced human-computer interface, can be an effective tool to improve breast cancer survivors' abilities to know and manage their current sensations related to their bodies. The present study protocol proposes a virtual reality intervention aiming at promoting interoception and emotional wellbeing, fear of cancer recurrence, and body perception in breast cancer survivors, according to the three data collection times. Repeated-measure analysis of variance (ANOVA) with between-within interaction will be performed. Expected results include participants' awareness of their internal feelings, the reduction of negative emotions, and the management of symptoms related to the body, clarifying characteristics for the effective implementation of VR psychological intervention in the future.
