A grounded theory study of dream fulfilment in children and young people with life-threatening and long-term conditions and their families

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

Patient Preferences for Multiple Myeloma Treatments : A Multinational Qualitative Study

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.Peer reviewe

Individual Trade-Offs Between Possible Benefits and Risks of Cancer Treatments:Results from a Stated Preference Study with Patients with Multiple Myeloma

BackgroundThe objectives of this study were to elicit the preferences of patients with multiple myeloma regarding the possible benefits and risks of cancer treatments and to illustrate how such data may be used to estimate patients' acceptance of new treatments. Patients and MethodsPatients with multiple myeloma from the cancer charity Myeloma UK were invited to participate in an online survey based on multicriteria decision analysis and swing weighting to elicit individual stated preferences for the following attributes: (a) 1-year progression-free survival (PFS, ranging from 50% to 90%), (b) mild or moderate toxicity for 2 months or longer (ranging from 85% to 45%), and (c) severe or life-threatening toxicity (ranging from 80% to 20%). ResultsA total of 560 participants completed the survey. The average weight given to PFS was 0.54, followed by 0.32 for severe or life-threatening toxicity and 0.14 for mild or moderate chronic toxicity. Participants who ranked severe or life-threatening toxicity above mild or moderate chronic toxicity (56%) were more frequently younger, working, and looking after dependent family members and had more frequently experienced severe or life-threatening side effects. The amount of weight given to PFS did not depend on any of the collected covariates. The feasibility of using the collected preference data to estimate the patients' acceptance of specific multiple myeloma treatments was demonstrated in a subsequent decision analysis example. ConclusionStated preference studies provide a systematic approach to gain knowledge about the distribution of preferences in the population and about what this implies for patients' acceptance of specific treatments. Implications for PracticeThis study demonstrated how quantitative preference statements from a large group of participants can be collected through an online survey and how such information may be used to explore the acceptability of specific treatments based on the attributes studied. Results from such studies have the potential to become an important new tool for gathering patient views and studying heterogeneity in preferences in a systematic way, along with other methods, such as focus groups and expert opinions. The objectives of this study were to ascertain the treatment preferences of patients with multiple myeloma, considering benefits and risks of particular cancer treatments, and to illustrate how such data may be used to estimate patients' acceptance of new treatments

P53 Patient Preferences for Multiple Myeloma Treatment: A Stated Preference Survey Using Discrete Choice Experiment and Swing Weighting

info:eu-repo/semantics/publishe

What matters most to patients with multiple myeloma? A Pan-European patient preference study

Introduction Given the rapid increase in novel treatments for patients with multiple myeloma (MM), this patient preference study aimed to establish which treatment attributes matter most to MM patients and evaluate discrete choice experiment (DCE) and swing weighting (SW) as two elicitation methods for quantifying patients’ preferences. Methods A survey incorporating DCE and SW was disseminated among European MM patients. The survey included attributes and levels informed by a previous qualitative study with 24 MM patients. Latent class and mixed logit models were used to estimate the DCE attribute weights and descriptive analyses were performed to derive SW weights. MM patients and patient organisations provided extensive feedback during survey development. Results 393 MM patients across 21 countries completed the survey ( M years since diagnosis =6; M previous therapies =3). Significant differences (p<01) between participants’ attribute weights were revealed depending on participants’ prior therapy experience, and their experience with side-effects and symptoms. Multivariate analyses showed that participants across the three MM patient classes identified via the latent class model differed regarding their past number of therapies ( F =4.772, p =.009). Patients with the most treatments (class 1) and those with the least treatments (class 3) attached more value to life expectancy versus quality of life-related attributes such as pain, mobility and thinking problems. Conversely, patients with intermediary treatment experience (class 2) attached more value to quality of life-related attributes versus life expectancy. Participants highlighted the difficulty of trading-off between life expectancy and quality of life and between physical and mental health. Participants expressed a need for greater psychological support to cope with their symptoms, treatment side-effects, and uncertainties. With respect to patients’ preferences for the DCE or SW questions, 42% had no preference, 32% preferred DCE, and 25% preferred SW. Conclusions Quality of life-related attributes affecting MM patients’ physical, mental and psychological health such as pain, mobility and thinking problems were considered very important to MM patients, next to life expectancy. This underscores a need to include such attributes in decision-making by healthcare stakeholders involved in MM drug development, evidence generation, evaluation, and clinical practice. This study highlights DCE as the preferred methodology for understanding relative attribute weights from a patient’s perspective.info:eu-repo/semantics/publishe

DataSheet_1_What matters most to patients with multiple myeloma? A Pan-European patient preference study.docx

IntroductionGiven the rapid increase in novel treatments for patients with multiple myeloma (MM), this patient preference study aimed to establish which treatment attributes matter most to MM patients and evaluate discrete choice experiment (DCE) and swing weighting (SW) as two elicitation methods for quantifying patients’ preferences.MethodsA survey incorporating DCE and SW was disseminated among European MM patients. The survey included attributes and levels informed by a previous qualitative study with 24 MM patients. Latent class and mixed logit models were used to estimate the DCE attribute weights and descriptive analyses were performed to derive SW weights. MM patients and patient organisations provided extensive feedback during survey development.Results393 MM patients across 21 countries completed the survey (Myears since diagnosis=6; Mprevious therapies=3). Significant differences (pConclusionsQuality of life-related attributes affecting MM patients’ physical, mental and psychological health such as pain, mobility and thinking problems were considered very important to MM patients, next to life expectancy. This underscores a need to include such attributes in decision-making by healthcare stakeholders involved in MM drug development, evidence generation, evaluation, and clinical practice. This study highlights DCE as the preferred methodology for understanding relative attribute weights from a patient’s perspective. </p