Caregiver-reported impact on quality of life and disease burden in patients diagnosed with metachromatic leukodystrophy: Results of an online survey and a qualitative interview
adolescent; adult; biomedical technology assessment; bone marrow transplantation; Caregiver; child; clinical article; conference abstract; controlled study; disease burden; enzyme replacement; European Quality of Life 5 Dimensions questionnaire; female; follow up; France; Germany; human; human tissue; infant; institutional review; interview; investment; juvenile; male; metachromatic leukodystrophy; multicenter study; palliative therapy; patient advocacy; patient care; preliminary data; quality of life; recall; sample size; wellbeing
Metachromatic leukodystrophy (MLD) is a rare autosomal recessive lysosomal disorder caused by a deficiency of the enzyme arylsulfatase A (ARSA). Symptoms include motor decline, developmental regression, decreased intellectual capabilities, and behavioural/psychiatric abnormalities. Until now, little data exists on the burden of illness of MLD for patients and their caregivers. This multinational study aims to quantify caregiver-reported impacts across several key domains including symptoms, treatment burden, time investment, social & emotional well-being, and professional and financial impact. Data were collected using a 45 min web survey and follow-up interview. The survey incorporated the PedsQLTM, a well-validated QoL measure for adolescents and children, and other questions specifically designed to measure the resource use relevant for MLD families based on a majority recall period of 4 weeks. The EQ-5D was also administered to capture the impact of MLD on caregivers (health disutilities). Extensive validation of the survey was conducted through feedback from clinical experts (US and France), and MLD patient advocacy groups (US and UK). This study was approved by an independent Institutional Review Board. Respondents were recruited from the US, UK, France, and Germany, representing a mix of families based on onset types (late infantile, juvenile, adult) and treatment received (supportive/palliative care, bone marrow transplant, enzyme replacement therapy). Due to the rarity of the disease, this study has no upper limit on sample size and is actively recruiting to December 2019 with a sample of >=30 respondents. Preliminary results based on current available data indicate that there is significant burden of MLD on patients and their caregivers, with some degree of variability depending on onset type. This multinational study enhances our understanding of the burden of disease of MLD, which ultimately should improve patient care and assist in the health technology assessment (HTA) of interventions for MLD.
Pang F; Shapovalov Y; Howie K; Wilds A; Calcagni C; Walz M
Molecular Genetics and Metabolism
2020
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.ymgme.2019.11.329" target="_blank" rel="noreferrer noopener">10.1016/j.ymgme.2019.11.329</a>
The chameleon project: A children's end of life care quality improvement project: Preliminary results
child; child death; human; palliative therapy; controlled study; pediatric intensive care unit; terminal care; bereavement; conference abstract; hospital admission; newborn; questionnaire; documentation; cost control; England; preliminary data; teaching; total quality management
Background As there was no specialist paediatric palliative care team in the region, we made a successful bid to NHS England's Marginal rate Emergency Threshold (MRET) and Readmission Fund for the Chameleon Project1 in 2018. The project has been funded for 12 months to audit and improve children's end of life care across the region. It provides a regional specialist team: consultant paediatrician 2 days a week, clinical nurse specialist 3 days, clinical academic researcher 2 days, network administrator 2 days, 3 paediatricians with expertise in palliative care in each of 3 district general hospitals 0.5 days. Methods The team developed tools to aid identification of children with end of life care needs. These were embedded by weekly attendances by the team at ward rounds and board rounds in the region's hospitals. We also delivered lectures and case based teaching sessions to clinical teams and at regional study days. A bereaved family feedback tool was adapted from ones used by the London Neonatal Network. Children with life limiting conditions who died an expected death in the 12 months of the project were identified from the child death review databases. Numbers of specialist hospital admissions, bed days, and costs were identified. Quality of care will be evaluated from documentation of care plans and post bereavement family feedback questionnaires. Results Compared to the year before the project started, the mean number of non-elective admissions reduced from 2.3 to 1.9; the number of non-elective bed days reduced from 51 to 32; and the number of non-elective bed days on neonatal and paediatric intensive care units reduced from 28 to 20, in the last 12 months of life per child. Quality of care is currently being assessed against NICE Quality Standards QS160.2 Conclusions These preliminary results indicate that a dedicated paediatric palliative care team can reduce admissions and bed days for children in their last year of life. We hope that further analyses will demonstrate improved family experience and cost savings for local NHS providers.
Haynes S; Kelly N; Turnbull J; Griffin H; Connolly A; Dorsett C; Brown J; Wolff A
Archives of Disease in Childhood
2019
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1136/archdischild-2019-rcpch.456" target="_blank" rel="noreferrer noopener">10.1136/archdischild-2019-rcpch.456</a>
An economic examination of private insurance claims among adolescents and young adults who were enrolled in hospice during the last year of life
adolescent; retrospective study; hospital patient; health care utilization; human; article; female; male; adult; young adult; emergency ward; primary medical care; hospice care; length of stay; preliminary data; private health insurance; hospital service
Purpose: The purpose of this study was to generate baseline data on the health characteristics, health care utilization, and health care spending among privately insured adolescents and young adults (AYA), who were enrolled in hospice care during their last year of life. Methods: A retrospective, nonexperimental design was used to collect and analyze longitudinal claims data from the Truven Health MarketScanTM database. The sample included AYA (aged 15-24 years) who utilized hospice during their last year of life. Results: Totally, 17,408 AYA were included in this analysis. Mean hospice length of stay (LOS) was low overall, but there was a statistically significant difference in hospice LOS in ages 15-19 years (mean 3.56, SD 15.17 days) compared with those aged 20-24 years (mean 2.26, SD 8.24; P<0.001 days). More than a third (37%) of the AYAs used the emergency department during the last year of life and 83% sought care from a primary care visit. However, only 6% of the sample who were hospice enrollees used frequent inpatient hospital services. Conclusions: This study provides preliminary data for private insurance expenditures and clinical utilization for AYA who were enrolled in hospice. This analysis also provides initial evidence to suggest extremely short hospice LOS for AYAs prior to the end of life and represents an area of future research need.
Keim-Malpass J; Cohrs AC; Lindley LC; Leslie DL
Adolescent Health, Medicine and Therapeutics
2018
<a href="http://doi.org/%2010.2147/AHMT.S168501" target="_blank" rel="noreferrer noopener">10.2147/AHMT.S168501</a>