Low-dose diazoxide is safe and effective in infants with transient hyperinsulinism.

OBJECTIVE: Transient hyperinsulinism (THI) is the most common form of recurrent hypoglycaemia in neonates beyond the first week of life. Although self-resolving, treatment can be required. Consensus guidelines recommend the lower end of the diazoxide 5-15 mg/kg/day range in THI to reduce the risk of adverse events. We sought to determine if doses <5 mg/kg/day of diazoxide can be effective in THI. DESIGN, PATIENTS, MEASURMENTS: Infants with THI (duration <6 months) were treated with low-dose diazoxide from October 2015 to February 2021. Dosing was based on weight at diazoxide start: 2 mg/kg/day in infants 1000-2000 g (cohort 1), 3 mg/kg/day in those 2000-3500 g (cohort 2) and 5 mg/kg/day in those >3500 g. RESULTS: A total of 73 infants with THI (77% male, 33% preterm, 52% small-for-gestational age) were commenced on diazoxide at a median age of 11 days (range 3-43) for a median duration of 4 months (0.3-6.8), with no difference between cohorts. The mean effective diazoxide dose was 3 mg/kg/day (range 1.5-10); 35% (26/73) required an increase from their starting dose, including 60% (9/15) of cohort 1. There was no association between perinatal stress risk factors or treatment-related characteristics and dose increase. Adverse events occurred in 13 patients (18%); oedema (12%) and hyponatraemia (5%) were the most common. Two infants developed suspected necrotising enterocolitis (NEC); none had pulmonary hypertension. CONCLUSION: Diazoxide doses <5 mg/kg/day are effective in THI. While the nature of the association between diazoxide and NEC was unclear, other adverse events were mild. We suggest considering starting doses as low as 2-3 mg/kg/day in THI to balance the side effect risk while maintaining euglycaemia.

Adding mini meals to a nasogastric refeeding protocol for patients with eating disorders can be achieved on general hospital wards.

BACKGROUND: Treatment of medically compromised patients with eating disorders is difficult in general hospital wards. There is currently no consensus on the best feeding method; however, previous research has demonstrated the safety of using enteral feeding. Because an oral diet has benefits on psychological and behavioural pathways, concurrent feeding requires further investigation. The present study aimed to examine acceptability and safety of implementing mini meals to a previously nil-by-mouth 7-day enteral feeding protocol. METHODS: This was mixed methods research including a retrospective observational study and participant survey. Patients admitted to a tertiary hospital in Brisbane, Australia, between July 2020 and March 2021 were eligible. Eligible participants were provided mini meals from day 5. Type and quantity of meals consumed alongside clinical incident data were collected. The survey examined acceptability of mini meals. Descriptive statistics were used to interpret findings. Content analyses were conducted on survey responses. RESULTS: Sixty-four participants (95%, n = 57/60 female, 25.2 ± 8.9 years; 75%, n = 45/60 diagnosed with anorexia nervosa) were included. At least half of the participants consumed some or all of the mini meals at each meal period. No clinical incidents were reported. Twenty-six (50%, n = 26/52) surveys were returned. Half (54%, n = 14/26) agreed-strongly agreed that mini meals improved their experience. Eleven participants desired more choice in menu items. CONCLUSIONS: The present study found that introducing mini meals into an enteral feeding protocol is acceptable and safe for patients with eating disorders. Participants reported benefits in returning to eating; however, some items on the menu require reconsideration to enable increased consumption.

Development of an international template to support patient submissions in Health Technology Assessments.

OBJECTIVES: To develop an international template to support patient submissions in Health Technology Assessments (HTAs). This was to be based on the experience and feedback from the implementation and use of the Scottish Medicines Consortium's (SMC) Summary Information for Patient Groups (SIP). METHODS: To gather feedback on the SMC experience, web-based surveys were conducted with pharmaceutical companies and patient groups familiar with the SMC SIP. Semistructured interviews with representatives from HTA bodies were undertaken, along with patient group discussions with those less familiar with the SIP, to explore issues around the approach. These qualitative data informed the development of an international SIP template. RESULTS: Survey data indicated that 82 percent (18 of 22 respondents) of pharmaceutical company representatives felt that the SIP was worthwhile; 88 percent (15/17) of patient group respondents found the SIP helpful. Both groups highlighted the need for additional support and guidance around plain language summaries. Further suggestions included provision of a glossary of terms and cost-effectiveness information. Patient group interviews supported the survey findings and led to the development of a new template. HTA bodies raised potential challenges around buy-in, timing, and bias connected to the SIP approach. CONCLUSIONS: The international SIP template is another approach to support deliberative processes in HTA. Although challenges remain around writing summaries for lay audiences, along with feasibility considerations for HTA bodies, the SIP approach should support more meaningful patient involvement in HTAs.

Preparedness for advancing future health: a national qualitative exploration of dietetics graduates' experiences.

Effective health workforce preparation is critical to the health of those who stand to benefit from its services. Emerging dietitians can provide important insights on an evolving workforce that is well-placed to advance future global health. This study aimed to explore a national sample of dietetics graduates' experiences of, and challenges faced in, dietetics workforce preparation and preparedness in Australia. An interpretive description methodology guided this study whereby researchers interpreted the meanings that participants attributed to their experiences. Twenty dietitians (graduated within the last 2 years) were purposively sampled from across Australia and detailed insights were obtained through semi-structured interviews. A multi-analyst approach employing thematic and template analysis, enabled five themes to be identified across the data set. These included: (1) being held back; (2) chasing the prize; (3) valuing real learning; (4) easing the transition; and (5) encountering influencers. While graduates appreciated their preparation, they were not empowered or equipped to embrace opportunities in diverse and emerging areas of dietetics practice. Graduates were challenged by the competitive landscape of securing obvious job opportunities and by a lack of support in transitioning into the workforce. Practice exposures and encounters with influential dietitians were highly valued. Research on role-emerging dietetics placements along with enhanced support mechanisms for novice dietitians is urgently required to ensure appropriate alignment between future dietetics preparation and practice. Obtaining insights into health professional graduates' experiences of their education can be used to ensure that emerging health workforces are relevant and responsive to future market needs.

Not the story you want? Assessing the fit of a conceptual framework characterising mental health recovery narratives.

PURPOSE: Narratives of recovery have been central to the development of the recovery approach in mental health. However, there has been a lack of clarity around definitions. A recent conceptual framework characterised recovery narratives based on a systematic review and narrative synthesis of existing literature, but was based on a limited sample. The aims of this study were to assess the relevance of the framework to the narratives of more diverse populations, and to develop a refined typology intended to inform narrative-based research, practice and intervention development. METHOD: 77 narrative interviews were conducted with respondents from four under-researched mental health sub-populations across England. Deductive and inductive analysis was used to assess the relevance of the dimensions and types of the preliminary typology to the interview narratives. RESULTS: Five or more dimensions were identifiable within 97% of narratives. The preliminary typology was refined to include new definitions and types. The typology was found not to be relevant to two narratives, whose narrators expressed a preference for non-verbal communication. These are presented as case studies to define the limits of the typology. CONCLUSION: The refined typology, based on the largest study to date of recovery narratives, provides a defensible theoretical base for clinical and research use with a range of clinical populations. Implications for practice include ensuring a heterogeneous selection of narratives as resources to support recovery, and developing new approaches to supporting non-verbal narrative construction.

Mental Health Recovery Narratives and Their Impact on Recipients: Systematic Review and Narrative Synthesis.

OBJECTIVE: Mental health recovery narratives are often shared in peer support work and antistigma campaigns. Internet technology provides access to an almost unlimited number of narratives, and yet little is known about how they affect recipients. The aim of this study was to develop a conceptual framework characterizing the impact of recovery narratives on recipients. METHOD: A systematic review of evidence about the impact of mental health recovery narratives was conducted. Searches used electronic databases (n = 9), reference tracking, hand-searching of selected journals (n = 2), grey literature searching, and expert consultation (n = 7). A conceptual framework was generated through a thematic analysis of included articles, augmented by consultation with a Lived Experience Advisory Panel. RESULTS: In total, 8137 articles were screened. Five articles were included. Forms of impact were connectedness, understanding of recovery, reduction in stigma, validation of personal experience, affective responses, and behavioural responses. Impact was moderated by characteristics of the recipient, context, and narrative. Increases in eating disorder behaviours were identified as a harmful response specific to recipients with eating disorders. CONCLUSIONS: Mental health recovery narratives can promote recovery. Recovery narratives might be useful for clients with limited access to peers and in online interventions targeted at reducing social isolation in rural or remote locations, but support is needed for the processing of the strong emotions that can arise. Caution is needed for use with specific clinical populations. Protocol registration: Prospero-CRD42018090923.

Oxetanes: Recent Advances in Synthesis, Reactivity, and Medicinal Chemistry.

The four-membered oxetane ring has been increasingly exploited for its contrasting behaviors: its influence on physicochemical properties as a stable motif in medicinal chemistry and its propensity to undergo ring-opening reactions as a synthetic intermediate. These applications have driven numerous studies into the synthesis of new oxetane derivatives. This review takes an overview of the literature for the synthesis of oxetane derivatives, concentrating on advances in the last five years up to the end of 2015. These methods are clustered by strategies for preparation of the ring and further derivatization of preformed oxetane-containing building blocks. Examples of the use of oxetanes in medicinal chemistry are reported, including a collation of oxetane derivatives appearing in recent patents for medicinal chemistry applications. Finally, examples of oxetane derivatives in ring-opening and ring-expansion reactions are described.

Technology, Affordances and Occupational Identity Amongst Older Telecommunications Engineers: From Living Machines to Black-Boxes.

This article explores the relationship between technology and occupational identity based on working-life biographical interviews with older telecommunications engineers. In the construction of their own working-life biographical narratives, participants attached great importance to the technology with which they worked. The article contends that workers' relationship with technology can be more nuanced than either the sociology of technology literature or the sociology of work literature accommodates. Adopting the concept of affordances, it is argued that the physical nature of earlier electromechanical technology afforded engineers the opportunity to 'fix' things through the skilled application of tools and act as autonomous custodians of 'living' machines: factors that were inherent to their occupational identity. However, the change to digital technology denied the affordances to apply hands-on skill and undermined key elements of the engineering occupational identity. Rather than simply reflecting the nostalgic romanticizing of the past, the biographies captured deterioration in the material realities of work.

Physical cognition: birds learn the structural efficacy of nest material.

It is generally assumed that birds' choice of structurally suitable materials for nest building is genetically predetermined. Here, we tested that assumption by investigating whether experience affected male zebra finches' (Taeniopygia guttata) choice of nest material. After a short period of building with relatively flexible string, birds preferred to build with stiffer string while those that had experienced a stiffer string were indifferent to string type. After building a complete nest with either string type, however, all birds increased their preference for stiff string. The stiffer string appeared to be the more effective building material as birds required fewer pieces of stiffer than flexible string to build a roofed nest. For birds that raised chicks successfully, there was no association between the material they used to build their nest and the type they subsequently preferred. Birds' material preference reflected neither the preference of their father nor of their siblings but juvenile experience of either string type increased their preference for stiffer string. Our results represent two important advances: (i) birds choose nest material based on the structural properties of the material; (ii) nest material preference is not entirely genetically predetermined as both the type and amount of experience influences birds' choices.

Evaluation of a virtual practice placement: A model to increase student capacity.

AIM: To describe the development and evaluation of a novel virtual practice placement. BACKGROUND: Health systems around the world face the challenge of recruiting and retaining sufficient nursing staff to provide high quality care. The need to train more nurses makes it hard to provide sufficient and varied high quality student placements to all students. This paper reports the result of one approach to the provision of a novel virtual placement for pre-registration student nurses. DESIGN: Online virtual placement evaluated by a questionnaire conducted after the placement. METHODS: A total of 195 students attended the virtual practice placement between 10th October 2022 and the 10th March 2023. The survey consisted of eight questions, of which one invited a qualitative response. RESULTS: A total of 188 students completed the questionnaire and provided feedback. Of these 84 were adult nursing students, 67 child, 36 mental health and one learning disability student. The virtual placement required considerable resources to run, however was deemed as valuable by most students. When asked to rate the overall experience out of 5, the median scores were consistently high: adult (Mdn=5), child (Mdn=4), learning disability (Mdn=5) and mental health (Mdn=5) and mean values consistently high across fields: adult (M=4.73), child (M=5), learning disability (M=5) and mental health (M=4.67). Qualitatively, there were four main themes that emerged from the questionnaire responses: increased understanding of community healthcare and holistic approaches to care; developing interpersonal skills; a positive impact on their future career opportunities and the value of realistic case studies. CONCLUSIONS: Virtual placements are a viable addition to traditional placements. However, they require careful planning and considerable resources including experienced and dedicated facilitators. Principles for the delivery of virtual placements were produced to replicate and share best practice.

Accuracy and impact on quality of life of real-time continuous glucose monitoring in children with hyperinsulinaemic hypoglycaemia.

Objective: Continuous glucose monitoring (CGM) is the standard of care for glucose monitoring in children with diabetes, however there are limited data reporting their use in hyperinsulinaemic hypoglycaemia (HH). Here, we evaluate CGM accuracy and its impact on quality of life in children with HH. Methods: Real-time CGM (Dexcom G5 and G6) was used in children with HH aged 0-16years. Data from self-monitoring capillary blood glucose (CBG) and CGM were collected over a period of up to 28days and analysed. Quality of life was assessed by the PedsQL4.0 general module and PedsQL2.0 family impact module, completed by children and their parents/carers before and after CGM insertion. Analysis of accuracy metrics included mean absolute relative difference (MARD) and proportion of CGM values within 15, 20, and 30% or 15, 20, and 30 mg/dL of reference glucose values >100 mg/dL or ≤100 mg/dL, respectively (% 15/15, % 20/20, % 30/30). Clinical reliability was assessed with Clarke error grid (CEG) analyses. Results: Prospective longitudinal study with data analysed from 40 children. The overall MARD between reference glucose and paired CGM values (n=4,928) was 13.0% (Dexcom G5 12.8%, Dexcom G6 13.1%). The proportion of readings meeting %15/15 and %20/20 were 77.3% and 86.4%, respectively, with CEG analysis demonstrating 97.4% of all values in zones A and B. Within the hypoglycaemia range (<70 mg/dL), the median ARD was 11.4% with a sensitivity and specificity of 64.2% and 91.3%, respectively. Overall PedsQL child report at baseline and endpoint were 57.6 (50.5 - 75.8) and 87.0 (82.9 - 91.2), and for parents were 60.3 (44.8 - 66.0) and 85.3 (83.7 - 91.3), respectively (both p<0.001). Conclusion: Use of CGM for children with HH is feasible, with clinically acceptable accuracy, particularly in the hypoglycaemic range. Quality of life measures demonstrate significant improvement after CGM use. These data are important to explore use of CGM in disease indications, including neonatal and paediatric diabetes, cystic fibrosis and glycogen storage disorders.

Patient Confidence and Information Preferences During the Treatment Decision-making Process: Results From a Large Multiple Myeloma Patient Survey Across 12 Countries in Europe and Israel.

BACKGROUND: The relapsing nature of multiple myeloma (MM) means that patients typically receive different and multiple lines of therapy, requiring many treatment decisions over the disease course. The aim of this study was to explore patient confidence and information preferences during the treatment decision-making process. PATIENTS AND METHODS: A multinational, cross-sectional survey enrolled patients with MM. It was co-developed and distributed by Myeloma Patients Europe across 12 countries in Europe and Israel from May 2019 to March 2020. Eligibility criteria included a self-reported diagnosis of MM and being able to recall the decision-making process at the start of their latest treatment line. RESULTS: A total of 1559 patients were included, with complete responses received from 1081 (69%) patients. The median age range was 54 to 64 years; there was an equal gender split and 57% had their latest treatment decision made within the past year. Overall, 54% of patients felt "very confident" in the latest treatment decision. Patients deemed the most important information to be safety/tolerability and treatment effectiveness, but the latter was among the least frequently received. Most patients reported that their primary physician treating MM was their main source for all types of information (range, 62%-94%), with 87% of patients reporting a "very good" or "good" relationship with them. CONCLUSION: Over half of patients felt very confident in their latest treatment decision; however, patients reported not routinely receiving important treatment effectiveness information. Addressing the discrepancies between information that patients receive and consider important may enhance confidence in decision-making.

Re-Thinking Hyperkalaemia Management in Chronic Kidney Disease-Beyond Food Tables and Nutrition Myths: An Evidence-Based Practice Review.

Potassium dysregulation can be life-threatening. Dietary potassium modification is a management strategy for hyperkalaemia. However, a 2017 review for clinical guidelines found no trials evaluating dietary restriction for managing hyperkalaemia in chronic kidney disease (CKD). Evidence regarding dietary hyperkalaemia management was reviewed and practice recommendations disseminated. A literature search using terms for potassium, hyperkalaemia, and CKD was undertaken from 2018 to October 2022. Researchers extracted data, discussed findings, and formulated practice recommendations. A consumer resource, a clinician education webinar, and workplace education sessions were developed. Eighteen studies were included. Observational studies found no association between dietary and serum potassium in CKD populations. In two studies, 40-60 mmol increases in dietary/supplemental potassium increased serum potassium by 0.2-0.4 mmol/L. No studies examined lowering dietary potassium as a therapeutic treatment for hyperkalaemia. Healthy dietary patterns were associated with improved outcomes and may predict lower serum potassium, as dietary co-factors may support potassium shifts intracellularly, and increase excretion through the bowel. The resource recommended limiting potassium additives, large servings of meat and milk, and including high-fibre foods: wholegrains, fruits, and vegetables. In seven months, the resource received > 3300 views and the webinar > 290 views. This review highlights the need for prompt review of consumer resources, hospital diets, and health professionals' knowledge.

Patient Perceptions Regarding Ciltacabtagene Autoleucel Treatment: Qualitative Evidence From Interviews With Patients With Relapsed/Refractory Multiple Myeloma in the CARTITUDE-1 Study.

INTRODUCTION: Ciltacabtagene autoleucel (cilta-cel), a novel chimeric antigen receptor T (CAR-T) cell therapy, has demonstrated early, deep, and durable clinical responses in heavily pretreated patients with relapsed/refractory multiple myeloma (RRMM), and improvements in health-related quality of life (HRQoL) in CARTITUDE-1 (NCT03548207). Patient perspectives on treatment provide context to efficacy outcomes and are an important aspect of therapeutic evaluation. METHODS: Qualitative interviews were conducted in a subset of CARTITUDE-1 patients (n = 36) at screening, Day 100, and Day 184 post cilta-cel on living with MM, therapy expectations, and treatment experiences during the study. RESULTS: Patients most wanted to see change in symptoms with the greatest impact on HRQoL: pain (85.2%) and fatigue (74.1%). The primary treatment expectation was achieving remission (40.7%), followed by extended life expectancy (14.8%). Patients most often defined meaningful change as improvement in symptoms (70.4%) and return to normalcy (40.7%). The percentage of patients reporting symptoms (pain, fatigue, bone fracture, gastrointestinal, neuropathy, and weakness) decreased from 85.2% to 22.2% across symptom types at baseline to 29.2% to 0% on Day 184 after cilta-cel. Improved symptoms and positive sentiments corresponded with improved perception of overall health status and reduced pain level, respectively. Most patients reported that their expectations of cilta-cel treatment had been met (70.8%) or exceeded (20.8%) at Day 184, and 70.8% of patients considered cilta-cel therapy better than their previous treatments. CONCLUSION: Overall HRQoL improvements and qualitative interviews showed cilta-cel met patient expectations of treatment and suggest the long treatment-free period also contributed to positive sentiments.

Standardised practices in the networked management of congenital hyperinsulinism: a UK national collaborative consensus.

Congenital hyperinsulinism (CHI) is a condition characterised by severe and recurrent hypoglycaemia in infants and young children caused by inappropriate insulin over-secretion. CHI is of heterogeneous aetiology with a significant genetic component and is often unresponsive to standard medical therapy options. The treatment of CHI can be multifaceted and complex, requiring multidisciplinary input. It is important to manage hypoglycaemia in CHI promptly as the risk of long-term neurodisability arising from neuroglycopaenia is high. The UK CHI consensus on the practice and management of CHI was developed to optimise and harmonise clinical management of patients in centres specialising in CHI as well as in non-specialist centres engaged in collaborative, networked models of care. Using current best practice and a consensus approach, it provides guidance and practical advice in the domains of diagnosis, clinical assessment and treatment to mitigate hypoglycaemia risk and improve long term outcomes for health and well-being.

Online Virtual Nursing Placements: A Case Study on Placement Expansion.

Introduction: A need to increase student placements, in a culture that is under unprecedented demand, means that practice providers and partner higher education institutions need to find new and innovative ways to ensure quality learning experiences for students. Virtual placements are just one way of providing student placements, and this study presents a case for this. Objective: This research aims to explore the student's experience of virtual blended placements and to consider future sustainability. Method: This is a qualitative, exploratory study observing the attitudes, thoughts, and feelings of student nurse experiences of online virtual nursing placements. Key cases were identified, and two participants were asked to reflect upon their experiences of the blended virtual learning placement. Student evaluations, and virtual placement day plans, form part of the data collection, and results are presented in a theory building and comparative approach, adopting pattern matching. Results: Three themes emerged from the narratives, supported by student evaluations and day plans. Students reviewed the placement positively in that they were able to achieve their required practice proficiencies. Second, they discussed the way in which the virtual days were facilitated and that building relationships with professionals and peers while on placement was of high importance. Conclusion: This case study presents a novel and unique student experience within a 0-19 community nursing service, which through 3 days of practice placement supported by two virtually taught days, students were able to reach their practice proficiencies. This virtual placement model offers a unique way to reduce the pressures on frontline staff and enriches the students' experiences.

POTS and Pregnancy: A Review of Literature and Recommendations for Evaluation and Treatment.

Postural Orthostatic Tachycardia Syndrome (POTS) is a disorder of the autonomic nervous system most commonly affecting women of reproductive age. Studies on POTS and pregnancy are limited, and there is a lack of clinical guidelines regarding assessment and management of pregnant women with POTS. The purpose of this review is to summarize data from the available studies on the topic of pregnancy in POTS and common comorbid conditions and to provide the clinical recommendations regarding evaluation and treatment of POTS in pregnant women, based on the available studies and clinical experience. We conclude that pregnancy appears to be safe for women with POTS and is best managed by a multi-disciplinary team with knowledge of POTS and its various comorbidities. Importantly, large, prospective studies are needed to better delineate the course and outcomes of pregnancy, as well as possible pregnancy-related complications in women with POTS. Clinicians should be aware of the clinical presentation, diagnostic criteria, and treatment options in pregnant women with POTS to optimize outcomes and improve medical care during pregnancy and post-partum period.

Observed High Adherence to Recombinant Human Growth Hormone Treatment Using a Multi-Component Approach to Improve Adherence in Individuals with Growth Disorders.

We explored whether a multi-component approach - using a digital health device, the easypod™ auto-injector, the 'MySupport' patient support programme (PSP) and a Patient Activation Measure® (PAM®) - could improve adherence in patients receiving recombinant human growth hormone (r-hGH). A 13-item PAM was used to assess caregiver self-reported knowledge, resulting in two PAM scores for 88 patients at four UK hospitals after an average of 5.6 months. Most patients improved their PAM score by ≥1 level (43%) or maintained it (>-1 and <1; 21%). In parallel, 74% of patients maintained (-5 to +5%) or improved (≥5%) their adherence. Further studies are required to evaluate a multi-component approach to adherence in a larger population and for a longer duration.

Enhancing the clinical supervision experience of staff members working within primary and community care: a best practice implementation project.

OBJECTIVES: The project aimed to assess compliance with evidence-based criteria regarding the use of clinical supervision amongst district nurses and to improve knowledge and engagement in clinical supervision activities within the workplace. INTRODUCTION: It is important to provide clinical support to all healthcare workers that provide opportunities to develop and be listened to in a supervised environment. Clinical supervision is seen as a key element to provide this support. It provides a professional working relationship between two or more members of staff where the reflection of practice and personal emotion can be discussed, which is outlined in many policies and guidelines. METHODS: A baseline audit was carried out using the JBI Practical Application of Clinical Evidence System program involving 16 participants in one district nursing team in South Wales. The first step involved the development of the project and generating the evidence. Following this, a baseline audit was conducted, and educational training on clinical supervision was undertaken followed by clinical supervision sessions. A postimplementation re-audit was conducted following implementation. RESULTS: A total of 16 participants enrolled on the project. Receiving basic training and participating in clinical supervision was much higher than the baseline audit with both increasing to 100% compliance. Furthermore, 94% of participants were aware of clinical supervision activities and 88% knew of existing records on clinical supervision. The project results show a large increase in compliance with all of the criteria. CONCLUSION: Overall the implementation project achieved an improvement in evidence-based practice regarding clinical supervision in primary care.

The Burden of a Multiple Myeloma Diagnosis on Patients and Caregivers in the First Year: Western European Findings.

Background: This research aimed to quantify the burden of illness (BoI) in transplant eligible (TE) and transplant non-eligible (TNE) newly diagnosed multiple myeloma (NDMM) patients and their caregivers, in the first year after diagnosis: at months 0, 3, and 12. Methods: Prospective, cross-sectional, observational NDMM study of TE and TNE patients and their caregivers from France, Germany, Italy, and Spain was conducted between May 2019 and January 2021. A structured, online questionnaire measuring disease burden, direct and costs, out-of-pocket expenses, and health-related quality of life (HRQoL) was used. Descriptive statistics were performed. Results: A total of 164, 160, and 190 NDMM patients [>65 years; self-described healthy; not working; living with caregiver] answered at months 0, 3, and 12. Patients lost independence to perform daily activities; mean pain intensity rose and opioid utilization increased, more significantly among TNE patients. Overall health status and HRQoL remained stable. Median 3-month direct medical costs peaked at month 3. Specialist consultations and hospital admissions were the greatest cost amongst TE and TNE patients. Home adaptations increased out-of-pocket expenditures amongst TNE patients. Patients describing themselves as working spent a median 0 hours in the office at all time points. A total of 131, 122, and 124 caregivers answered at months 0, 3, and 12. Mean self-rated burden score rose. By month 12, half of caregivers developed stress, anxiety or depression. Most employed caregivers continued working. Productivity was low at month 0 with a trend of recovering at month 12. Caregivers of TNE compared to TE patients reported greater time burden. Caregivers' HRQoL was stable over time. Conclusion: NDMM is burdensome for patients and caregivers in the first year after diagnosis. TNE patients are more dependent on caregivers and incur higher care costs than TE patients. Despite the financial, physical, and emotional burden, HRQoL remains stable possibly indicating resilience and illness adjustment amongst patients and caregivers.