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Myeloma is an incurable life-threatening hematological cancer. Recent treatment developments have seen improvements in survival; however, while patients are living longer, they are living with symptoms and treatment side effects. To evaluate myeloma patients' preferences for treatment using a discrete choice experiment (DCE). This study set out to define the relative importance of key treatment attributes, characterize the risk-benefit trade-offs in patients' decision-making, and to analyze the predictive power of basic demographic factors. Four hundred seventy-five myeloma patients in the UK were invited to participate by Myeloma UK. Data were collected using DCEs through an online survey. The DCEs presented patients with 10 choice scenarios, each with 2 treatment options described by 7 attributes, and a \"no treatment\" option. The DCE data were modelled using a latent class model (LCM). The effects of demographic characteristics were also examined. Not surprisingly, average survival was most important to all patients but there were significant contrasts between the class preferences. The LCM revealed two classes of patients. Patients in Class 1 placed greater importance on average survival and mild-to-moderate side effects, whereas patients in Class 2 focused on the mode of administration and the average out-of-pocket costs. Patients living with others and those diagnosed in the last 5 years were more likely to be in Class 1. Different treatment features were not valued equally among all myeloma patients. This has important implications for healthcare policy decisions and could be used to guide decisions around the value of new myeloma medicines.

Background: Investigational and marketed drugs for the treatment of multiple myeloma (MM) are associated with a range of characteristics and uncertainties regarding long term side-effects and efficacy. This raises questions about what matters most to patients living with this disease. This study aimed to understand which characteristics MM patients find most important, and hence should be included as attributes and levels in a subsequent quantitative preference survey among MM patients. Methods: This qualitative study involved: (i) a scoping literature review, (ii) discussions with MM patients ( n = 24) in Belgium, Finland, Romania, and Spain using Nominal Group Technique , (iii) a qualitative thematic analysis including multi-stakeholder discussions. Results: MM patients voiced significant expectations and hopes that treatments would extend their lives and reduce their cancer signs and symptoms. Participants however raised concerns about life-threatening side-effects that could cause permanent organ damage. Bone fractures and debilitating neuropathic effects (such as chronic tingling sensations) were highlighted as major issues reducing patients' independence and mobility. Patients discussed the negative impact of the following symptoms and side-effects on their daily activities: thinking problems, increased susceptibility to infections, reduced energy, pain, emotional problems, and vision problems. MM patients were concerned with uncertainties regarding the durability of positive treatment outcomes, and the cause, severity, and duration of their symptoms and side-effects. Patients feared short-term positive treatment responses complicated by permanent, severe side-effects and symptoms. Conclusions: This study gained an in-depth understanding of the treatment and disease-related characteristics and types of attribute levels (severity, duration) that are most important to MM patients. Results from this study argue in favor of MM drug development and individual treatment decision-making that focuses not only on extending patients' lives but also on addressing those symptoms and side-effects that significantly impact MM patients' quality of life. This study underscores a need for transparent communication toward MM patients about MM treatment outcomes and uncertainties regarding their long-term efficacy and safety. Finally, this study may help drug developers and decision-makers understand which treatment outcomes and uncertainties are most important to MM patients and therefore should be incorporated in MM drug development, evaluation, and clinical practice.

Aim There is a lack of literature on what patients with myeloma think about patient-reported outcome measures (PROMs). The aim of this study was to examine the views of patients and their family members about commonly used PROMs in myeloma to help guide future development and use.Method Two groups of patients were asked to review a selection of validated PROMs used in care of people with myeloma according to their perceived relevance to their lives, ease of completion, language and comprehensiveness. The PROMs selected for review were the EQ5D-5L, HADS, FACT-G and the MyPOS.Findings Participants believed that PROMs had a validating effect on their concerns. They preferred the language and content of the MyPOS and FACT-G to the EQ5D-5L and HADS, and considered that the former two had greater relevance to their illness experience.Conclusion Patients with myeloma believe PROMs are important but underused in clinical practice.

Background Few studies explore stroke survivor views and motivations towards stem cell therapy (SCT). This qualitative study explores the views and motivations of both stroke survivors and their partners/carers towards a proposed 2‐arm Phase III Randomised Controlled Trial (RCT) comparing intracerebral insertion of stem cells with placebo neurosurgery in stroke survivors with disability. Objective To explore views and motivations towards a proposed 2‐arm stem cell trial and identify factors that may impede and enhance participation. Design This study adopts a naturalistic design to explore the complexity of this field, employing a participatory action‐research approach comprising a specialized Conversation (World) Café form of focus group. Data were collected via 5 Conversation Cafés with stroke survivors (age 40‐75) and partners/carers between June and October 2016. Of 66 participants, 53 (31 male, 22 female) were stroke survivors and 13 (6 female, 7 male) were partners/carers. Qualitative data were analysed using a thematic approach. Discussion and Conclusion Stroke survivor views and motivations reflect anticipation of the personal and future benefits of regenerative medicine. Partners/carers sought to balance the value of stroke survivor hope with carrying the weight of hope as carer, a conflict burden adding to known caregiver burden. All participants expressed the need for during and post‐trial psychological support. This study provides a rare opportunity to explore the prospective views and motivations of stroke survivors and their partners/carers towards a proposed Phase III 2‐arm RCT. This adds weight to qualitative evidence exploring capacity, consent, decision making, perceptions of treatment risk and supports required for clinical trial participation.

This article examines how myeloma patients experience their care in NHS England, compared with patients with other cancers, using data from the National Cancer Patient Experience Survey (NCPES). This research was conducted to provide insights into the treatment and care of myeloma patients, and to better meet their needs and those of their families. Descriptive statistics were used to explore differences in care experienced by myeloma patients, compared with the average care experience, as reported by the NCPES. Findings suggest that myeloma patients are more likely to have been invited to participate in clinical trials than individuals with other cancers. However, myeloma patients report worse care experiences compared with the all-cancer average in: length of time before seeing a myeloma specialist, getting a diagnosis, length of wait before referral and support in treatment decision making. Possible reasons for these differences are discussed. This article also discusses the value of clinical nurse specialists in myeloma and makes some recommendations for nursing practice based on findings from the NCPES.

Background: This thesis examines the impact of dream or wish fulfilment on seriously ill children and their families. Dream or wish fulfilment is operationalised as the actualisation of a seriously ill child’s wish by a charity that provides desired experiences. Anecdotal reports suggest that the experience of having a dream or a wish fulfilled can provide seriously ill children and their families with a sense of hope and time away from illness. However, little empirical research has been conducted in this area. This thesis reports the impact of dream fulfilment on the psychosocial well-being of ill children and their families. The research questions are: what is the experience of having a dream fulfilled for the child? What is the impact of dream fulfilment on the family? Methods: A constructivist grounded theory methodology was adopted, using theoretical sampling to recruit families from across the UK. Twenty-one families were interviewed, including 15 dream recipients, 8 siblings, and 24 parents. Analysis followed the grounded theory methodology of simultaneous data collection and development of theory, resulting in analytic interpretations of participants’ worlds. Results and Conclusions: This thesis reports for the first time a theory and accompanying theoretical model, that explain the impact of dream fulfilment on families’ lives. The generated theory suggests that dream fulfilment was conceptualised as an alternative milestone in seriously ill children and their families’ lives. Additionally, the dream experience shifted perceptions of illness by providing instances and experiences where illness did not underscore family life. Findings additionally suggest that the dream fulfilment process provided families with ill children, who often felt excluded and stigmatised from services, with a period of much needed support. Findings also highlight the unintended negative consequences of dream fulfilment. Implications for Dreams Come True, and other dream and wish fulfilment organisations are discussed.

Dream fulfilment provides a positive focus, hope and a sense of future for children and young people whose lives may be filled with hospital visits, pain or worry. This paper will present the results of an audit of a dream fulfilment charity’s database. The audit systematically analyses user demographics to find out more about who uses charities such as these. This project fits with the strategic exercise conducted by the NHS in England to ‘map’ palliative care services across England so that both health professionals and the public can see what services are, and where they are available. The data reported in this paper are drawn from Dreams Come True’s records of children and young people with life limiting and long term conditions and their families, and the dreams that they have had. Descriptive statistics are used to summarise the clinical and socio-demographic characteristics of the children/ families who have used the charity’s services over the last five years. Results highlight the range of health conditions that children who use the charity have, as well as reporting on other factors such as the child’s age, family size, gender and the type of dream requested. Analysis also suggests gaps in service reach which may reflect the unmet need in the sector as a whole. Results will be of interest to health care professionals working in the fields of paediatric palliative care, and care of children with long term conditions, as well as to other dream fulfilment charities. The audit’s findings have implications for referring organisations, such as hospices, and potential lessons for the adult sector.

Predictive biomarkers can potentially meet the need for improved drug assignment in acute myeloid leukemia (AML). Fewer than half of AML patients have actionable mutations: consequently, targeted therapy achieves remission in only a fraction of those who have them. Dynamic BH3 Profiling (DBP), a functional assay, can measure changes in drug-induced apoptotic priming in multiple cancers. To assess the feasibility and predictive capacity of DBP in AML, we prospectively tested DBP using a fixed-drug panel in myeloblasts from 92 patients. We generated a database combining genetic and functional annotation. Established AML clinical and genetic prognostic characteristics were associated with drug-induced apoptotic priming. We observed distinct patient sensitivities to single drugs or combinations with the BCL2-inhibitor venetoclax, and patient apoptotic priming differences based on CD123-expression within distinct cell subpopulations. DBP further predicted the likelihood of remission to chemotherapy and targeted agents, supporting its use to identify optimal personalized therapy. Dynamic BH3 profiling provides patient-specific drug vulnerability data in real-time to inform prognosis and therapy selection. Dynamic BH3 profiling can be performed on bone marrow and leukemic blood from AML patients in 48 hours.Known clinical prognostic factors associate with drug-induced apoptotic priming in AML.Drug-induced apoptotic priming identifies drug vulnerabilities in individual patients and predicts clinical response to chemotherapy and small molecule inhibitors.