Compassionate use

BACKGROUND: A 54-year-old Hispanic OPos female with known history of anti-Rh17 antibodies was diagnosed with Philadelphia-Chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Rh17, also known as Hr0 , is a high-frequency antigen composed of several epitopes on the RhCE protein. Anti-Rh17 antibodies can be made by individuals with missing or varied C/c, E/e antigens. Anti-Rh17 antibodies are clinically significant given multiple case reports of hemolytic disease of the fetus and newborn (HDFN)...

Recent ethical debate about compassionate conservation has invoked moral theories to oppose or support traditional practices of killing animals to protect biodiversity and ecosystems. The debate has featured the mainstream moral theories of consequentialism and utilitarianism, deontology, and virtue ethics. We identify problematic applications and critique of these moral theories in conservation discussions. Problems include a lack of clarity when invoking moral theories, misunderstanding and mischaracterizing theories, and overlooking features and circumstances affecting a theory's application...

BACKGROUND: In recent years, there has been a rise in international (care) movements that prioritise community-centred initiatives such as age-friendly communities, compassionate communities or integrated community care. Although these movements have different focal points, they share common features: seeking to address systemic failures in (care) services, value the participation of end-users, focus on unmet (care) needs, through a local, neighbourhood-oriented approach. In the Flemish and Brussels regions notably the concept of Caring Neighbourhoods is experiencing rapid growth...

BACKGROUND: Healthcare and support workers play a pivotal role in delivering quality services and support to people seeking sanctuary who have experienced poor physical and mental health linked to previous trauma, relocation and loss of freedoms. However, they often encounter various challenges in their daily work, ranging from communication barriers to resource constraints. This qualitative study seeks to delve into the perspectives of healthcare and support workers' experience of workarounds, employed to overcome barriers to providing care...

BACKGROUND: Suicide bereavement entails profound social stressors, including stigma and communication barriers, which can impair social support for suicide loss survivors (SLS). Despite recognized benefits of empathetic interactions, social support, and self-disclosure in mitigating adverse mental health outcomes after suicide loss, we lack a comprehensive understanding of the factors influencing perceived social support among SLS within their broader social environments. To address this gap, our study explores the diverse social experiences of SLS beyond their immediate circles...

In France, mexiletine - a class I antiarrhythmic drug - can be prescribed for the symptomatic treatment of myotonia of the skeletal muscles in adult patients with myotonic dystrophy under a compassionate use programme. Mexiletine is used according to its summary of product characteristics, which describes its use for myotonia treatment in adult patients with non-dystrophic myotonia, a different neuromuscular condition without cardiac involvement. A cardiac assessment is required prior to initiation and throughout treatment due to potential proarrhythmic effects...

Cancer vaccines present a promising avenue for treating immune checkpoint blockers (ICBs)-refractory patients, fostering immune responses to modulate the tumor microenvironment. We revisit a phase I/II trial using Tumor Antigen-Presenting Cells (TAPCells) (NCT06152367), an autologous antigen-presenting cell vaccine loaded with heat-shocked allogeneic melanoma cell lysates. Initial findings showcased TAPCells inducing lysate-specific delayed-type hypersensitivity (DTH) reactions, correlating with prolonged survival...

CONTEXT: In today's ever-changing world, fostering personal and social responsibility is essential for building strong and compassionate communities. This study aimed to provide a quantitative synthesis focusing on the emotional and social outcomes of Teaching Personal and Social Responsibility (TPSR) model-based Physical Education (PE) programs. METHODS: A comprehensive literature review covering the period from November 2022 to September 2023 identified 637 articles published between 2005 and 2023...

Bispecific T cell engagers (BiTEs) kill B cells by engaging T cells. BiTEs are highly effective in acute lymphoblastic leukemia. Here we treated six patients with multidrug-resistant rheumatoid arthritis (RA) with the CD19xCD3 BiTE blinatumomab under compassionate use. Low doses of blinatumomab led to B cell depletion and concomitant decrease of T cells, documenting their engager function. Treatment was safe, with brief increase in body temperature and acute phase proteins during first infusion but no signs of clinically relevant cytokine-release syndrome...

Evaluating other people's moral character is a crucial social cognitive task. However, the cognitive processes by which people seek out, prioritize, and integrate multiple pieces of character-relevant information have not been studied empirically. The first aim of this research was to examine which character traits are considered most important when forming an impression of a person's overall moral character. The second aim was to understand how differing levels of trait expression affect overall character judgments...

Novel prostate-specific membrane antigen (PSMA) ligands labeled with α-emitting radionuclides are sparking a growing interest in prostate cancer treatment. These targeted alpha therapies (TATs) have attractive physical properties that deem them effective in progressive metastatic castrate-resistant prostate cancer (mCRPC). Among the PSMA TAT radiopharmaceuticals, [225Ac]Ac-PSMA has been used extensively on a compassionate basis and is currently undergoing phase I trials. Notably, TAT has the potential to improve quality of life and has favorable antitumor activity and outcomes in multiple scenarios other than in mCRPC...

Regulatory agencies must balance patient demands to access new treatments for fatal diseases with limited treatment options while ensuring drug safety and efficacy. However, questionable U.S. regulatory actions resulted in the early approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS) by reconvening advisory commissions to obtain positive decisions and designating the drug as a new molecular entity. Data from one randomized clinical trial suggests minimal delays in disease progression and longer survivability, but debate remains about the lack of confirmatory evidence of effectiveness owing to study limitations...

Background Nursing is a compassionate profession that carries occupational hazards, including work-related injuries. Back pain is a common concern due to the physically demanding tasks performed by nurses. Utilizing proper techniques of body mechanics is vital to prevent work-related back pain and enhance overall well-being. Aim To assess the knowledge and practice of the nurses working in Jordanian hospitals about the appropriate techniques of body mechanics and their relation to non-specific back pain. Methods A cross-sectional design with a convenience sample of 280 participants was randomly selected from hospitals...

Using an experimental posttest-only control group design, the purpose of this study was to examine the effects of self-compassionate writing exercises on transgender and non-binary participants' body satisfaction, gender identity pride, and internalized transphobia. A total of 238 transgender people participated in this study (Mage = 28.27, SD = 8.12). Participants were randomly assigned to a self-compassionate writing task focusing on their gender identity, a self-compassionate writing task focusing on their body image as a transgender person (i...

BACKGROUND: When it comes to pandemic response, preparation can be the key. Between 2020 and 2024, the fast-paced development of COVID-19-often compounded by pubic policies' failures to reflect the latest reality and the public's divergent reactions to the pandemic and the policies-means that society should prepare for exit strategies that can reflect the reality of the pandemic and the interests of the people. Yet oftentimes societies only have one exit strategy with limited scope. This paper investigates the dangers of having only one pandemic exit strategy for pandemics like COVID-19...

INTRODUCTION: In adults, sodium-glucose cotransporter type 2 inhibitors have revolutionised the treatment of type 2 diabetes mellitus, heart failure, and chronic kidney disease. OBJECTIVE: We aimed to review information on compassionate use, clinical pharmacology, efficacy, and safety of dapagliflozin and empagliflozin in children. METHODS: We conducted a systematic review of published clinical trials, case reports, and observational studies in Medline, Excerpta Medica, and Web of Science databases from inception to September 2023...

OBJECTIVES: This research study aimed to discover how dementia affecting older people was perceived, experienced, and managed by stakeholders in the Pacific Island country of Fiji. METHOD: A transformational grounded theory approach was used. Semi-structured interviews and focus groups with key stakeholders in the major towns of Suva, Lautoka, and Nadi were carried out. Transcripts were analysed in line with transformational grounded theory methods. RESULTS: A total of 50 participants (40 service providers, eight family caregivers, one person with dementia, and one village elder) shared their views and experiences about dementia...

Intersex individuals, encompassing people with diverse sex characteristics that do not fit binary frameworks of sex, have long faced a history of medical secrecy, discrimination, and societal stigma, contributing to their limited social visibility. In recent years, increased awareness of intersex issues and a robust advocacy movement have drawn significant attention to the experiences of intersex individuals and their families. This study contributes to the existing literature by examining the experiences and needs of parents of intersex individuals within genetic healthcare systems, bridging a critical gap, and advocating for more comprehensive and supportive healthcare practices...

BACKGROUND: Caring in Africa is often viewed from sociopolitical and humanity perspectives. OBJECTIVE: The aim of this study was to investigate the meanings and expressions of caring from the narratives of persons living with cancer and their nurses. METHODS: This study adopted a qualitative ethnonursing methodology. A purposive sampling technique was used to select the key participants, who were grouped into 7 nurses and 6 patients. General participants included 4 physicians, 3 ward assistants, and 6 patients' relatives...

Anxiety is one of the common psychological problems among infertile women, which affects their quality of life. The purpose of this study was to compare the effects of self-compassion intervention based on a religious and non-religious perspective regarding the anxiety and quality of life of infertile women. A randomized clinical trial design with experimental and control groups was used. A total of 78 infertile women who lived in Yazd province, Iran, were referred to Yazd reproductive sciences institute, were selected by available sampling and randomly assigned to two experimental and one control groups...