Patient and parent oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in SMA. Rome, 13 July 2019

Summary: Background: Idiopathic Multicentric Castleman Disease (iMCD) is a rare inflammatory lymphoproliferative disorder with heterogeneous clinical presentations. The aim of this study was to identify the type, frequency, and severity of iMCD-related symptoms and their impact on the daily lives of iMCD patients and informal caregivers. Methods: Two bespoke online surveys were conducted for iMCD patients and informal caregivers, collecting descriptive data and using a Likert scale to quantify the impact of symptoms on daily life. Findings: The study included 51 iMCD patients and 11 informal caregivers. Patients reported experiencing up to 27 unique symptoms, with a mean number of 6.7 symptoms per patient. Most symptoms had a moderate to severe impact on patients' daily lives, with pain/discomfort, ability to travel, and sexual functioning being the most affected. Various patient characteristics were significantly associated with adverse impacts on daily life. Caregivers also experienced significant impacts on their own social life and freedom, emotional wellbeing, travel/relocation, and work. Interpretation: iMCD patients have diverse and underappreciated symptomatology, which adversely affects multiple aspects of their daily lives as well as their caregivers. Funding was provided by EUSA Pharma.

Summary: In this study, the IBD-disk score was found to be well correlated with IBD daily-life burden, demonstrating its potential for clinical use in assessing IBD-related disability. The high completion rate of the IBD-disk questionnaire suggests that it could be a valuable tool in evaluating the impact of IBD on patients' daily lives. The study also showed that an optimal cut-off of 40 for the IBD-disk score could effectively identify patients with high IBD daily-life burden.

Summary: This study suggests that parents of children with autism spectrum disorder gain greater autism-specific parenting self-efficacy when they are more involved in their child's therapy and are more satisfied with intervention-related training. Feeling pressure related to being caregivers of children with autism spectrum disorder can undermine this self-efficacy, but the severity of the child's symptoms does not limit parents' confidence. There is an opportunity for providers to help parents feel more confident in managing the complexities of parenting a child with autism spectrum disorder by fostering involvement in treatment and creating more satisfying training experiences.

Summary: The burden of classic galactosemia on patients and families is significant, with most adults unable to live independently and requiring support with daily activities. Symptoms such as memory difficulties and tremors greatly impact daily functioning and quality of life. This underscores the need for pharmacological intervention to improve the condition and reduce the burden on patients and caregivers.

Summary: Cancer patients can benefit from massages provided by family caregivers as part of hospice care. This pilot study explored the feasibility of training caregivers to give massages and assessed the burden on caregivers. The findings suggest that repeated-dose massages by caregivers are feasible and further research is needed to determine the benefits and burden of this therapy.

Summary: This study aimed to investigate the relationship between care burden and psychological distress among family caregivers. The findings showed that most participants reported low levels of care burden and psychological distress, and there was a significant association between the two. Therefore, promoting caregivers' understanding of caring and involvement in treatment is crucial.

Summary: Family caregivers may experience burdens from various aspects such as physical, psychological, and socio-economic when dealing with the needs of palliative care patients. The study aims to identify assessment instruments for the burden of care for family caregivers that are suitable for clinical practice.

Summary: The study found significant negative correlations between burden and quality of life of caregivers looking after moderately to totally dependent elderly. This correlation was observed in all four domains: physical, psychological, social relationships, and environmental.

Summary: This study found significant differences in caregiver burden, BADLs, and IADLs between compound and noncompound caregivers, but the correlations between caregiver burden and ADLs did not differ significantly between the two groups. It suggests that caregiver burden is negatively affected by the lack of independence in functional ability of care recipients with ASD for both compound and noncompound caregivers.