Parental impact during and after neonatal intensive care admission.

Parents play a pivotal role in neurodevelopmental outcomes of their children in the neonatal intensive care unit (NICU) and beyond. Integration of parents in clinical care and research is synergistic. Engaged parents yield more comprehensive clinical care and more robust and meaningful research products. Subsequently, successful clinical and research efforts improve outcomes for children. We review strategies for parental integration into NICU clinical care and research, including parental involvement in therapeutic interventions and neurodevelopmental care, and effective communication strategies for clinicians and researchers. We discuss challenges in neonatal trials and emphasize the need for building a culture of research, collaborative partnerships with patient advocacy organizations, and ongoing support beyond the NICU. Overall, we call for recognizing and fostering the impactful role of parents as teammates with clinicians and researchers in optimizing neurodevelopmental outcomes in the NICU and beyond.

Self-performed Rh typing: a cross-sectional study.

OBJECTIVE: To evaluate whether patients are capable and willing to self-administer and interpret an EldonCard test to determine their Rh status. METHODS: This was a cross-sectional study in Honolulu, HI, USA of pregnancy-capable people aged 14-50 years who did not know their blood type and had never used an EldonCard. Participants independently completed EldonCard testing, determined their Rh type and answered a survey on feasibility and acceptability. Separately, a blinded clinician recorded their interpretation of the participant's EldonCard. When available, we obtained blood type from the electronic health record (EHR). We measured Rh type agreement between participant, clinician and EHR, as well as participant comfort and acceptability of testing. RESULTS: Of the 330 total participants, 288 (87.3%) completed testing. Patients and clinicians had 94.0% agreement in their interpretation of the EldonCard for Rh status. Patient interpretation had 83.5% agreement with EHR while clinician and EHR had 92.3% agreement. Sensitivity of EldonCard interpretation by patient and clinician was 100%. Specificity was 83.2% for patients and 92.2% for clinicians. Two patients (of 117) had Rh-negative blood type in the EHR. The vast majority of participants found the EldonCard testing easy (94.4%) and felt comfortable doing the testing (93.7%). Participants with lower education levels felt less confident (p=0.003) and less comfortable with testing (p=0.038); however, their ability to interpret results was similar to others (p=0.051). CONCLUSIONS: Patient-performed Rh typing via the EldonCard is an effective and acceptable option for patients, and could be used as a primary screening test for Rh status.

[Balancing drug regulation and health democracy: The role of patient and healthcare professional advisors at the ANSM]. / Concilier régulation du médicament et démocratie sanitaire : rôle des conseillers patient ou professionnel de santé à l'ANSM.

The French National Agency for Health Products (ANSM) is a regulatory and public health agency. Its regulatory, health policing and public health protection activities require a perfect fit with the field and the various people involved in the use of health products. Since 2019, the ANSM has adapted its organisation, procedures and processes to encourage and improve interaction with its stakeholders, as part of its policy of openness towards civil society. To accompany this ambitious change and to support its staff, the Agency has recruited advisors corresponding to the main users of health products: prescribers (doctor's hospital and outpatient), pharmacists and patients. Working as a group or individually, they provide a "lived" user perspective on health products at each stage of the evaluation process. They may be involved in the assessment of dossiers, signals or applications received by the Agency, in the internal validation of reports or in discussions with stakeholders. They are particularly involved when the analysis requires expertise that goes beyond the technical, scientific or regulatory aspects. They may also work with ANSM staff to explain certain processes and difficulties in the field. Advisors help to ensure that regulatory and/or scientific expertise is clear and consistent with user experience. In addition to their scientific and therapeutic aspects, medicines are also economic, social and political issues. Their regulation is therefore particularly affected by the need for health democracy. This requires the active participation of health professionals, patients and, more broadly, civil society in the decision-making process. Civil society is a space occupied by a wide range of actors who exert pressure from different ideological positions to influence the regulation of health products. In this context, taking into account a plurality of viewpoints in the regulation of health products is necessary and complex, but its operation can be facilitated by the collective efforts of the actors and the adaptation of organisations, such as the integration of advisors.

Developing Disability Advocates Through Deliberate Inter-Professional Conversations Regarding Quality of Life After Stroke.

OBJECTIVE: Stroke can result in significant mental and physical impairment. Training healthcare professionals on effective strategies for mitigating stroke-related quality-of-life issues is crucial in facilitating comprehensive stroke management. This study aims to evaluate the impact of an interprofessional education (IPE) experience on students' attitudes regarding post-stroke disability. METHODS: In this pre-post interventional study, pharmacy and medical students received an electronic patient chart and a store-and-forward video depicting physical and cognitive impairment in a stroke patient. Students were instructed to discuss the acute management and post-discharge needs of the patient from an advocacy perspective. After the IPE experience, students completed the student perceptions of interprofessional clinical education-revised instrument (SPICE-R2) and an unvalidated disability attitudes survey. The surveys were analyzed using a paired t-test. Additionally, students reflected on the prompt, "What are some things you had NOT considered prior to this IPE?". RESULTS: Seven hundred and eight students completed the surveys. Post-IPE, there was a significant improvement in all domains of the SPICE-R2. On the disability survey, there was significant improvement on all statements, including rate your comfort with; 1.) discussing the expected disabilities associated with new-onset stroke and 2.) discussing strategies for improving the quality of life of a patient who has long term disabilities. On the self-reflections, 31.7% (N=211) had not considered the need for post-stroke care services prior to this IPE. CONCLUSION: This IPE experience was instrumental in improving student perspectives regarding post-stroke disability.

Challenges and opportunities in NMIBC management across Latin America: insights from healthcare providers and a patient advocacy group.

Non-muscle invasive bladder cancer (NMIBC) is characterised by high rates of recurrence and progression, requiring substantial healthcare resources. In Latin America, the incidence of NMIBC is set to increase due to an aging population and lifestyle changes. To better understand the current challenges for NMIBC treaters and patients, a mixed-methods approach was leveraged combining secondary research with qualitative interviews from healthcare providers in Brazil, Colombia, Mexico and Argentina. Our analysis found that significant challenges persist across the region, particularly due to Bacillus Calmette-Guérin shortages, inconsistent adherence to clinical guidelines and significant socioeconomic disparities for patients accessing healthcare services. Addressing these challenges requires improved patient advocacy, strategic use of clinical trials and better resource distribution to enhance NMIBC management across Latin America.

Seeking abortion accompaniment: experiences and self-managed abortion preferences of hotline callers after abortion legalisation in Argentina.

BACKGROUND: Until the legalisation of abortion in Argentina in 2021, the Socorristas en Red, a network of feminist collectives, provided support and information ('accompaniment') to people self-managing their abortion with medications. Following legalisation, the Socorristas continued accompanying people self-managing or accessing abortion through the healthcare system. We conducted a cross-sectional study to understand preferences, experiences and choices about abortion when contacting a Socorristas hotline after legalisation of abortion in Argentina. METHODS: We surveyed callers to the Socorristas' hotline in Neuquén, Argentina about their demographics, pregnancy history, reasons for calling, and experiences seeking abortion through the hotline and the healthcare system. We assessed overall prevalence of these experiences, and analysed differences between people who contacted the health system before calling the hotline and those who first called the hotline for services. RESULTS: Of the 755 callers in the study, the majority (63.3%) contacted the Socorristas because they trusted them, and 21.7% called because they both trusted the Socorristas and did not want to go to the healthcare system. At the end of the call, most people (95.4%) chose to self-manage their abortion with accompaniment outside the healthcare system. People who called the healthcare system prior to contacting the hotline frequently reported being referred to the Socorristas, as well as challenges scheduling appointments. CONCLUSIONS: After legalisation of abortion in Argentina there is continued demand for accompaniment. Globally, ensuring that accompaniment and self-management of abortion is legal and protected will provide individuals with the support and facilitated access to the abortion care they desire.

Pharmaceutical company funding of cancer patient advocacy organizations in the Netherlands.

BACKGROUND: Financial conflicts of interest (FCOI) of medical professionals and associated organizations with pharmaceutical companies (pharma) might contribute to the use of low value oncological treatments. Value criteria for oncological drug approvals in the Netherlands have recently become more stringent leading to objections by cancer patient advocacy organizations (cPAOs). Considering the importance of cPAOs input in cancer patient care we analyzed whether pharma funding of cPAOs occurs in the Netherlands. METHODS: The cPAO websites and available annual reports were evaluated for disclosure of pharma funding for the years 2021 and 2022. Also, data from the Dutch Healthcare Transparency Registry (DHTR) were extracted. RESULTS: Twenty-one of 34 (61.8 %) cPAOs received pharma funding (with 20 registered in the DHTR), and for 13 (29.4 %) cPAOs no reporting of pharma funding could be found. Three of the cPAOs disclosed pharma funding directly on their main website. Online educational material was available from 22 cPAOs on their websites with pharma funding disclosed on the educational material in 5. The total registered amount of pharmaceutical funding was €667,232.00 in 2021 and €536,098.00 in 2022. The median (and interquartile ranges) DHTR registered amount of support per cPAO that received funding in the studied period was €23,799.50 (14,823.75-84,663.30). The most common funding category as defined in the DHTR was project sponsorship. CONCLUSIONS: Financial support by the pharmaceutical industry is common for Dutch cPAOs. Given the importance of cPAOs and their objective input in the societal debate on the availability of cancer drugs, the potential influence of pharma sponsoring should be critically evaluated.

Engaging Youth Voice and Family Partnerships to Improve Children's Mental Health Outcomes.

Promoting active participation of families and youth in mental health systems of care is the cornerstone of creating a more inclusive, effective, and responsive care network. This article focuses on the inclusion of parent and youth voice in transforming our mental health care system to promote increased engagement at all levels of service delivery. Youth and parent peer support delivery models, digital innovation, and technology not only empower the individuals involved, but also have the potential to enhance the overall efficacy of the mental health care system.

What is loneliness? A reflection on my Imperial College London Patient Project.

Loneliness has emerged as a significant public health concern, with profound implications for health outcomes, which can manifest in physical, psychological, and social affliction. Working alongside Fulham Medical Centre, I sought out to create a range of both offline and online resources (YouTube video, ten-episode Spotify podcast, online website, GP practice brochure and poster) designed to provide base learning, practical strategies, community connections and a sense of support to those grappling with loneliness. These resources were well-received by the practice and were implemented on a practice basis, to provide support to the local community. Reflection on this project, highlights the need for student projects, and emphasises the tangible impact that we can have on community support and care for individuals tackling feelings of loneliness.

STXBP1: fast-forward to a brighter future - a patient organization perspective.

Syntaxin-binding protein 1 related disorder (STXBP1-RD) is a rare neurologic disorder associated with global neurodevelopmental delay, intellectual disability, early-onset epilepsy, motor abnormalities, and autism. The underlying pathophysiology stems from a de novo mutation in the STXBP1 gene, which codes for the STXBP1 protein. The STXBP1 protein is involved in synaptic vesicle fusion and neurotransmitter release. Pathogenic variants in the STXBP1 gene generally result in haploinsufficiency, an impairment in neurotransmitter release, and subsequent dysfunction in neuronal communication. The STXBP1 Foundation was founded in 2017 to support families of children with STXBP1-RD and accelerate the development of effective therapies and, ultimately, a cure for the disorder. The Foundation initially supported research aimed at better understanding the complex phenotypic presentation of the disease as well as the development of animal and cellular models usable by the research community to more fully characterize STXBP1 function and disease pathogenicity. In 2023, the Foundation embarked on its STXBP1 Fast Forward Strategic Plan, which includes a prospective natural history study and substantive biomarker work to drive forward the development of new precision therapies for STXBP1-RD.

STXBP1: fast-forward to a brighter future STXBP1-related disorder (STXBP1-RD) is a rare and severe brain condition. It causes delays in development, learning problems, seizures starting at an early age, movement challenges, and sometimes autism. The main problem comes from a new mutation in the STXBP1 gene, which makes a protein needed for brain cells to communicate properly. When this gene doesn't work right, there's not enough of the protein, leading to trouble with brain cell communication. To help families dealing with this disorder and speed up the search for effective therapies, the STXBP1 Foundation started in 2017. At first, they funded studies to understand the disease better and create models for testing treatments. Then, in 2023, they launched the STXBP1 Fast Forward Strategic Plan. This plan includes studying how the disorder progresses naturally and researching markers that could help develop precise treatments for STXBP1-RD.

Exploring the relationship between spiritual care and patient advocacy of nurses from generations X, Y and Z working in intensive care clinics: A cross-sectional study.

OBJECTIVE: To determine the relationship between spiritual care and patient advocacy across three generations of nurses working in intensive care units. DESIGN: Cross-sectional survey. METHODS: Data collection took place from July to August 2022 with 120 nurses in Turkey. Data collection tools included the Spiritual Caregiving Competency Scale, the Spirituality and Spiritual Care Assessment Scale, and the Patient Advocacy Scale for Nurses. Data on nurses' demographics were evaluated using descriptive statistical methods (number, percentage, mean, standard deviation). Independent sample t-test, one-way ANOVA, Pearson correlation, and linear multiple regression analysis were used to evaluate the relationships between variables, with results reported as 95% confidence intervals (CI). RESULTS: More than half of the nurses were from Generation Y (39.2 %) and Generation Z (42.5 %). Generation Z's mean patient advocacy score (156.96 ± 23.16) was statistically significantly higher than Generation X's (139.32 ± 34.26). We determined that the spiritual competence scale communication sub-dimension score of Generation Y nurses working between 1-10 years was higher than that of Generation Z nurses. Additionally, as the patient advocacy scores of all generations increased, so did spiritual competence scores. CONCLUSION: The study found differences in patient advocacy and spiritual care competencies between generations. Thus, we recommend organizing courses, seminars, and in-service training on patient advocacy and spiritual care for intensive care nurses. IMPLICATIONS FOR CLINICAL PRACTICE: This study estimates nurses' spiritual care competencies and patient advocacy levels from different generations and sheds light on the literature to eliminate differences in care between generations in nursing practices that evolve and change over time. It is recommended that courses, seminars, in-service training, spiritual activities, and interactive meetings be organized to encourage the participation of intensive care nurses to minimize the differences in spiritual care and patient advocacy among all generations of intensive care nurses.

Ophthalmological research in Germany: suggestions by an international expert panel.

PURPOSE: To evaluate the research performance in ophthalmology in Germany based on the findings of the recent research map of the German Ophthalmological Society (DOG) and to suggest strategies for future improvements on a national level both to DOG as well as to politics. The focus is on preclinical and translational clinical research. METHODS: International expert panel evaluation and discussion organized by the Task Force Research of the German Ophthalmological Society (DOG). RESULTS: The international view on the German ophthalmological research landscape was generally positive. The value for money relationship was judged as very good. As Germany is facing an aging society and vision impairment will create an ever-increasing socioeconomic burden, the reviewers suggested several lines of future activities: an increased activity of securing intellectual property, more lay audience lobbying, intensified collaboration and critical mass building between "lighthouses" of ophthalmic research in Germany, as well as the establishment of a German national eye institute equivalent. CONCLUSION: The ophthalmological research performance in Germany was rated to be very good by an international expert panel. Nonetheless significant improvements were requested in the fields of translation (clinical trials, IP), synergy between specialized institutions and governmental funding for a German center for eye research.

Shared Vision for Improving Outcomes for Serious Fungal Diseases: Report of a Patient, Caregiver, and Clinician Summit.

Background: Recently, increasing focus on patient input into research and healthcare improvements has fostered expanded patient-centered advocacy efforts. This first pan-fungal disease summit, part of the MYCology Advocacy, Research, & Education effort, brought together patients, caregivers, and mycology experts to better document patient experiences with invasive fungal disease (IFD) and establish priorities for mycology education, advocacy, and research. Methods: Patients who had suffered from IFD, their caregivers, clinicians, industry representatives, government officials, and patient advocacy professionals were invited. Patients and caregivers shared their stories and struggles with IFD. Breakout sessions separated mycology experts from patients and caregivers for further discussions to identify commonalities and perceived gaps and to formulate recommendations. The 2 groups then reconvened to develop consensus recommendations. Results: IFD patients and their caregivers shared experiences reflecting the typically lengthy prediagnosis, acute treatment, long-term treatment, and posttreatment recovery stages of IFD. They reported substantial physical, psychological, and financial burdens associated with the IFD experience, particularly related to delayed diagnoses. They reaffirmed a need for coordinated patient-centered education, peer support, and advocacy to document the burden of serious fungal infections. Mycology experts discussed strategies to address gaps in the mycology field, such as insufficient training, inadequate workforce support, and a need to partner more with patient groups. Conclusions: A summit involving patients with IFD, family caregivers, and mycology experts identified a substantial nonclinical burden of disease associated with IFD. Patients and mycology experts prioritized several goals for education, advocacy, and research to raise awareness of IFD and improve outcomes.

[Ophthalmological research in Germany: evaluation by an international expert panel]. / Ophthalmologische Forschung in Deutschland: Evaluation durch ein internationales Expertengremium.

PURPOSE: To evaluate the research performance in ophthalmology in Germany based on the findings of the recent research map of the German Ophthalmological Society ( DOG) and to suggest strategies for future improvements on a national level both to DOG as well as to politics. The focus is on preclinical and translational clinical research. METHODS: International expert panel evaluation and discussion organized by the Task Force Research of the German Ophthalmological Society (DOG). RESULTS: The international view on the German ophthalmological research landscape was generally positive. The value for money relationship was judged as very good. As Germany is facing an aging society and vision impairment will create an ever-increasing socioeconomic burden, the reviewers suggested several lines of future activities: an increased activity of securing intellectual property, more lay audience lobbying, intensified collaboration and critical mass building between "lighthouses" of ophthalmic research in Germany, as well as the establishment of a German national eye institute equivalent. CONCLUSION: The ophthalmological research performance in Germany was rated to be very good by an international expert panel. Nonetheless significant improvements were requested in the fields of translation (clinical trials, IP), synergy between specialized institutions and governmental funding for a German center for eye research.

What Is a Sarcoma 'Specialist Center'? Multidisciplinary Research Finds an Answer.

The management of sarcomas in specialist centers delivers significant benefits. In much of the world, specialists are not available, and the development of expertise is identified as a major need. However, the terms 'specialist' or 'expert' center are rarely defined. Our objective is to offer a definition for patient advocates and a tool for healthcare providers to underpin improving the care of people with sarcoma. SPAGN developed a discussion paper for a workshop at the SPAGN 2023 Conference, attended by 75 delegates. A presentation to the Connective Tissue Oncology Society (CTOS) and further discussion led to this paper. Core Principles were identified that underlie specialist sarcoma care. The primary Principle is the multi-disciplinary team discussing every patient, at first diagnosis and during treatment. Principles for optimal sarcoma management include accurate diagnosis followed by safe, high-quality treatment, with curative intent. These Principles are supplemented by Features describing areas of healthcare, professional involvement, and service provision and identifying further research and development needs. These allow for variations because of national or local policies and budgets. We propose the term 'Sarcoma Intelligent Specialist Network' to recognize expertise wherever it is found in the world. This provides a base for further discussion and local refinement.