UNASSIGNED: To examine the burden of treatment (BoT) experienced by people with Amyotrophic Lateral Sclerosis (ALS) in Argentina.
UNASSIGNED: Qualitative methodological design based on semi-structured interviews. Nineteen semi-structured interviews were conducted (PwALS = 7, informal caregivers= 12). The interview guides were designed based on the literature and BoT theory. Data were analysed following a framework analysis approach.
UNASSIGNED: The research highlighted the arduous journey toward obtaining a diagnosis, marked by delays influenced by healthcare system inefficiencies, lack of disease awareness and pandemic-related anxiety. Receiving the diagnosis was a destabilising experience, triggering the need to reframe self-identity, a new reality. As the disease progressed, patients encountered significant challenges in their daily activities and basic tasks, affecting their ability to work, communicate, and manage personal care. The burden extended beyond the patients to their primary caregivers. Access to specialised care, bureaucratic complexities in securing treatment, and the financial impact of managing the disease posed substantial challenges.
UNASSIGNED: The findings offer valuable insights into the experiences of PwALS and their caregivers in Argentina. They underscore the need for increased disease awareness, improved access to specialised care, and enhanced support networks to alleviate the burdens PwALS and their families face.
Streamlined pathways for patients with amyotrophic lateral sclerosis in Argentina are necessary to ensure timely access to comprehensive support and interventions, laying the foundation for holistic rehabilitation.There is a need to encourage clear communication between healthcare providers and patients to establish ongoing dialogue incorporating emotional, social, and psychological aspects into rehabilitation, aligning with patient-centric approaches.There is a need to develop and implement adaptive rehabilitation strategies to sustain independence and enhance quality of life for amyotrophic lateral sclerosis patients, addressing the physical, emotional, and social implications of the disease’s progression.Rehabilitation professionals should advocate for systemic improvements to ensure equitable access to therapies and support alleviate financial burdens, enabling a comprehensive, patient-focused approach to rehabilitation.

The burden of treatment (BOT) related to propranolol treatment for infantile hemangiomas (IH) has never previously been explored. A modified validated questionnaire, the Treatment Burden Questionnaire, and one-on-one semi-structured interviews were used to assess the BOT for propranolol for IH. Out of 80 caregivers, the overall burden score was very low at 1.2 out of 10; thematic analysis of interviews grouped themes into administration, monitoring, financial, and associated anomalies. The BOT of propranolol for IH is very low but could be reduced further by offering age-based risk stratification related to feeding frequency and risk of hypoglycemia, pragmatic advice around timing of doses before sleep, and reducing frequency of vital sign monitoring.

The objective of this study was to describe and compare the burden of treatment (BOT) and the quality of life (QoL) in early high efficacy therapy (HET) vs. escalation therapy in relapsing remitting multiple sclerosis (RRMS) patients included in RelevarEM, the Argentinean registry of MS (RelevarEM, NCT 03,375,177).
METHODS: cross sectional study conducted between September and December 2022. Participating patients were adults, RRMS patients who initiated (during the last three years) their treatment with a HET (natalizumab, ocrelizumab, alemtuzumab, cladribine) or with escalation treatment (beta interferon, glatiramer acetate, teriflunomide, dimethyl fumarate or fingolimod). Clinical and demographic aspect were collected. QoL and BOT was measured with the validated to Spanish MusiQol and BOT questionnaire. Propensity score (PS)-based nearest-neighbor matching was applied to homogenize groups. Comparisons were be done using a linear regression analysis model stratified by matched pairs, with BOT and QoL assessments as main outcomes.
RESULTS: 269 patients were included in the analysis, mean age 33.7 ± 5.7 years, 193 (71.7 %) were female. A total of 136 patients were on early HET while 133 were on escalation therapy. In the entire group the mean total BOT score (±SD) was 48.5 ± 15.3 while in the group of patients receiving early HET we observed that the mean BOT score (±SD) was 43.5 ± 12.2 vs. 54.3 ± 13.3 in escalation treatment (p < 0.0001). Regarding the score QoL (±SD), in the entire sample we observed a global score of 77.4 ± 11.2. When we stratified groups, in HET (±SD) it was 81.3 ± 14 vs. 74.1 ± 18.3 in escalation therapy (p = 0.0003).
CONCLUSIONS: in this multicenter study that included 269 patients from Argentina we observed in early HET a significantly lower BOT and higher QoL than patients receiving escalation therapy.

Purpose: To assess the impact of switching to, or adding, an intravitreal dexamethasone implant (Dex; Ozurdex®) in anti-vascular endothelial growth factor (VEGF) therapy on disease stability and treatment intervals in eyes with neovascular age-related macular degeneration (nAMD) and persistent disease activity and high treatment demand. Methods: This retrospective noncomparative multicenter longitudinal case series included pseudophakic eyes with nAMD and persistent retinal fluid despite regular anti-VEGF therapy (ranibizumab or aflibercept) that received at least 1 intravitreal Dex implant. Visual acuity, central retinal thickness (CRT), and intraocular pressure were recorded before, and after, the addition of Dex to anti-VEGF therapy. Results: Sixteen eyes of 16 patients met the inclusion criteria of persistent fluid despite anti-VEGF therapy, under treatment intervals of ≤7 weeks in 14 instances. Patients were 80.9 ± 7.4 years old and had received 25.5 ± 17.4 anti-VEGF injections before Dex over a period of 36.4 ± 21.9 months before switching. The treatment interval increased from 5.5 ± 3.2 weeks between the last anti-VEGF and first Dex injection to 11.7 ± 7.3 weeks thereafter (P = 0.022). CRT remained stable (385.3 ± 152.1, 383.9 ± 129.7, and 458.3 ± 155.2 μm before switching as well as 12 and 24 months after switching; P = 0.78 and P = 0.36, respectively). An insignificant mean short-term early increase in visual acuity was not sustained over time. Conclusions: The addition of Dex resulted in a relevant and sustained increase in treatment intervals, whereas CRT and visual acuity remained stable in these difficult-to-treat eyes. It may be discussed whether inflammation or other steroid-responsive factors play a significant role in cases of nAMD with nonsatisfactory responses to anti-VEGF.

UNASSIGNED: The workload health and social care service users and caregivers take on, and their capacity to do this work is important. It may play a key part in shaping the implementation of innovations in health service delivery and organisation; the utilisation and satisfaction with services; and the outcomes of care. Previous research has often focused on experiences of a narrow range of long-term conditions, and on factors that shape adherence to self-care regimes.
UNASSIGNED: With the aim of deriving policy and practice implications for service redesign, this evidence synthesis will extend our understanding of service user and caregiver workload and capacity by comparing how they are revealed in qualitative studies of lived experience of three kinds of illness trajectories: long-term conditions associated with significant disability (Parkinson\'s disease, schizophrenia); serious relapsing remitting disease (Inflammatory Bowel Disease, bipolar disorder); and rapidly progressing acute disease (brain cancer, early onset dementia).
UNASSIGNED: We will review and synthesise qualitative studies of lived experience of participation in health and social care that are shaped by interactions between experienced treatment burdens, social inequalities and illness trajectories. The review will involve:   1.  Construction of a theory-informed coding manual; systematic search of bibliographic databases to identify, screen and quality assess full-text papers.   2.  Analysis of papers using manual coding techniques, and text mining software; construction of taxonomies of service user and caregiver work and capacity.   3.  Designing a model of core components and identifying common factors across conditions, trajectories, and contexts.   4.  Work with practitioners, and a Patient and Public Involvement (PPI) group, to explore the validity of the models produced; to develop workload reduction strategies; and to consider person-centred service design.
UNASSIGNED: We will promote workload reduction models to support service users and caregivers and produce policy briefs and peer-reviewed publications for practitioners, policy-makers, and researchers.
Our experiences of illness are often complex. We may have to work hard too. We may need to monitor and record symptoms: take up different diets and physical activity; use different drugs and medical devices; develop expertise in using websites and information technology; coordinate input from health and care services; sometimes we have to work out how to pay for the services we need. How we get through this work is affected by our capacity to do it, and that is shaped by personal and wider resources, we can draw on. All of this is also affected by the services that are available to us, and by the ways our chances in life are shaped by income, ethnicity, education, gender, and age. The kinds of illnesses we have and how they progress, mean that these factors change over time. We call these changes trajectories. To better understand service user work and capacity, we will review published studies that tell us about people’s everyday experiences of living with illnesses. We focus on three rarely studied trajectories. These are long-term conditions associated with significant disability; serious relapsing remitting disease; and rapidly progressing acute disease. We will first use existing research to build a framework in which we can describe and understand relevant aspects of the published studies. We will use this framework to extract relevant information from the studies. This will enable us to make a model of common features of service user work and capacity across different conditions, their trajectories, service organisation and delivery, and patterns of social and economic disadvantage. Finally, we will work with groups of service users and caregivers, and with health and social care professionals to apply the model to the development of strategies to reduce workload and improve service design for people with complex health problems.

Advanced therapy medicinal products such as Chimeric antigen receptor T-cell therapy offer ground-breaking opportunities for the treatment of various cancers, inherited diseases, and chronic conditions. With development of these novel therapies continuing to increase it\'s important to learn from the experiences of patients who were among the first recipients of ATMPs. In this way we can improve the clinical and psychosocial support offered to early patient recipients in the future to support the successful completion of treatments and trials.
We conducted a qualitative investigation informed by the principles of the key informant technique to capture the experience of some of the first patients to experience CAR-T therapy in the UK. A directed content analysis was used to populate a theoretical framework informed by Burden of Treatment Theory to determine the lessons that can be learnt in supporting their care, support, and ongoing self-management.
A total of five key informants were interviewed. Their experiences were described within the three domains of the burden of treatment framework; (1) The health care tasks delegated to patients, Participants described the frequency of follow-up and the resources involved, the esoteric nature of the information provided by clinicians; (2) Exacerbating factors of the treatment, which notably included the lack of understanding of the clinical impacts of the treatment in the broader health service, and the lack of a peer network to support patient understanding; (3) Consequences of the treatment, in which they described the anxiety induced by the process surrounding their selection for treatment, and the feeling of loneliness and isolation at being amongst the very first recipients.
If ATMPs are to be successfully introduced at the rates forecast, then it is important that the burden placed on early recipients is minimised. We have discovered how they can feel emotionally isolated, clinically vulnerable, and structurally unsupported by a disparate and pressured health service. We recommend that where possible, structured peer support be put in place alongside signposting to additional information that includes the planned pattern of follow-up, and the management of discharged patients would ideally accommodate individual circumstances and preferences to minimize the burden of treatment.

Children who experience a severe burn injury not only require acute medical care but may also need ongoing rehabilitation. To mitigate the potential long-term consequences of scarring and the impact of scarring on their everyday lives, children may be expected to use a variety of time-consuming and multi-component non-invasive scar treatments (such as pressure garments and silicone gels). These treatments may pose unique challenges for children and their caregivers. With limited research previously addressing how Australian children navigate the use of scar treatments at home, this study aimed to develop a grounded theory of children and their caregivers\' engagement with non-invasive burn scar treatments. Using a constructivist grounded theory approach, interviews were completed with 20 caregivers and 7 children, and a theory of children and their caregivers becoming experts in the use of non-invasive burn scar treatments was developed. Through persistence and flexibility, they continued to develop expertise. Engaging with scar treatments was an evolving process over time for children with burns and their caregivers. Theoretical categories identified included \'making it work\', \'finding the balance\' and \'seeking reassurance\' and highlighted the remarkable strengths and adaptability of children and their caregivers. Through trial and error, children and their caregivers developed their own unique strategies for engaging with scar treatments. These insightful results may inform the development of interventions to support children and their caregivers\' day-to-day engagement with non-invasive burn scar treatments and guide health professionals recommending these treatments.

UNASSIGNED: Self-injection of biologics is a mainstay of chronic disease treatment, yet the process of self-injection often causes persistent apprehension and anxiety, distinct from needle phobia. While literature alludes to the role that routines and rituals play in self-injection, there is no comprehensive study on the routines and rituals self-injectors employ, nor of the process by which they are discovered and ingrained.
UNASSIGNED: We conducted a mixed-method, observational pilot ethnography study of 27 patients with plaque psoriasis, psoriatic arthritis, or ankylosing spondylitis with and without prior biologic self-injection experience. Patients submitted self-made videos, photos, and projective exercises of an actual biologic self-injection and completed validated instruments to assess burden of treatment. Videos and photos containing routine and ritual elements were thematically categorized based on functional and emotional benefit, and analyzed for differences based on current biologic, dosing frequency, time on current biologic, and burden of treatment measures.
UNASSIGNED: During patients\' initial at-home injections, training gaps became apparent, leading to a process of experimentation aimed at reducing pain/anxiety, increasing confidence, and building a consistent injection process. Routines were present in 27/27 (100%) patients and anchored the time, place, and process for injection, and incorporated approved use steps for the injection device. Ritual elements served as emotional coping strategies for patients and were present in 21/27 (77.8%) of patients.
UNASSIGNED: Our findings suggest that providing patients device training using adult learning principles, teaching routines and rituals concurrently, and providing at-home opportunities for practice with a device trainer may be useful strategies to reduce anxiety, avoid unnecessary experimentation, and improve adherence to injection therapy. While further studies are needed to generalize our findings, we posit that routine and ritual elements can be incorporated into existing patient-clinician interactions or novel digital interventions through mobile medical applications, smart training devices, and connected injection ecosystems.

To describe how patients with CKD negotiated assigned responsibilities in the management of their disease, resulting in potential relational nonadherence.
Qualitative study performed in two healthcare facilities in Buenos Aires, Argentina, including 50 patients and 14 healthcare providers. We conducted semistructured interviews which were analysed using a frame of reference with concepts of Burden of Treatment and Cognitive Authority theories.
Adherence to treatment defined \"good patients\". Patients needed to negotiate starting treatment, its modality and dialysis schedule, although most patients felt they did not participate in the decision process and that providers did not acknowledge implications of these decisions on their routine. Some patients skipped dialysis if concerns were not attended. Regularly, patients negotiated frequency of visits, doses, dietary restrictions and redefined relationships with their support networks, sometimes with devasting effects. As a result of overwhelming uncertainty some patients refused enrolling into a transplant program. When the frequency of complications increased, patients considered abandoning dialysis.
When patients perceived demands were excessive or conflicting, they entered into negotiations. Relationally induced nonadherence may arise when professionals do not or cannot enter into negotiations over patients\' beliefs or knowledge about what is possible for them to do.

UNASSIGNED: Many people with chronic obstructive pulmonary disease (COPD) feel overburdened with the treatment and management of their illness. Although research has begun to shed light on how COPD patients experience treatment burden, most of what we know is limited to personal experiences of patients. The aim of this study is to identify and prioritise areas of treatment burden that should be discussed during the clinical encounter from the perspectives of COPD patients, carers, and respiratory physicians.
UNASSIGNED: Data were collected from participants using the nominal group technique. Five nominal group sessions were conducted in total (n = 31); three sessions with patients (n = 18), one with carers (n = 7) and another with respiratory physicians (pulmonologists or chest physicians) (n = 6). Each session was recorded and analyzed using thematic analysis.
UNASSIGNED: Going beyond understanding patients\' and carers\' experiences of treatment burden, this study offers a practical viewpoint of what should be discussed in a clinical encounter. Each group of participants contextualized treatment burden issues for discussion from their own perspectives. There was strong agreement, however, across the groups that difficulties accessing healthcare, lack of education and information, and worry about COPD treatment and prognosis were the most important treatment burden priorities for discussion.
UNASSIGNED: Understanding and creating opportunities to discuss these issues in a clinical encounter is important in not only reducing treatment burden but also improving health outcomes and quality of life for COPD patients and their carers.