Defining a 'cells to society' research framework for appendiceal tumours.

Tumours of the appendix - a vestigial digestive organ attached to the colon - are rare. Although we estimate that around 3,000 new appendiceal cancer cases are diagnosed annually in the USA, the challenges of accurately diagnosing and identifying this tumour type suggest that this number may underestimate true population incidence. In the current absence of disease-specific screening and diagnostic imaging modalities, or well-established risk factors, the incidental discovery of appendix tumours is often prompted by acute presentations mimicking appendicitis or when the tumour has already spread into the abdominal cavity - wherein the potential misclassification of appendiceal tumours as malignancies of the colon and ovaries also increases.

Home Health Care Research for Children With Disability and Medical Complexity.

Pediatric home health care represents a vital system of care for children with disability and medical complexity, encompassing services provided by family caregivers and nonfamily home health care providers and the use of durable medical equipment and supplies. Home health care is medically necessary for the physiologic health of children with disability and medical complexity and for their participation and function within home, school, and community settings. While the study of pediatric home health care in the United States has increased in the last decade, its research remains primarily methodologically limited to observational studies.

Lived Experience of Patients and Caregivers in Rare Genetic Neurological Gene Therapy Clinical Trials in Children.

To date, sparse attention has been paid to the importance of the "lived experience" of participants and their caregivers in pediatric gene therapy (GT) trials for rare genetic neurological disorders. Pediatric GT studies differ meaningfully from adult GT studies as the decision to participate involves a dyad: the child participant and their caregiver(s). As a multistakeholder group of authors, we are a diverse group with expert perspectives on the social, emotional, physical, and logistical burdens/benefits of trial participation and the myriad ways they affect pediatric GT research.

Decision Regret in Patients with Appendiceal Cancer Undergoing Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy.

Background: Decision regret is an emerging patient reported outcome. The aim of this study was to assess the incidence of regret in patients with appendiceal cancer (AC) who underwent cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (CRS-HIPEC).

Patients And Methods: An anonymous survey was distributed to patients through the Appendix Cancer and Pseudomyxoma Peritonei (ACPMP) Research Foundation.

Serum Response Factor Expression in Excess Permits a Dual Contractile-Proliferative Phenotype of Airway Smooth Muscle.

The transcription factors (TFs) MyoCD (myocardin) and Elk-1 (ETS Like-1 protein) competitively bind to SRF (serum response factor) and control myogenic- and mitogenic-related gene expression in smooth muscle, respectively. Their functions are therefore mutually inhibitory, which results in a contractile-versus-proliferative phenotype dichotomy. Airway smooth muscle cell (ASMC) phenotype alterations occur in various inflammatory airway diseases, promoting pathological remodeling and contributing to airflow obstruction.

Lessons Learned From Clinical Studies in Centronuclear Myopathies: The Patient Perspective-A Qualitative Study.

Background: Since 2014, several clinical studies focusing on centronuclear myopathies have been conducted, including a prospective natural history study, a gene transfer clinical trial and a clinical trial using an antisense oligonucleotide. Dedicated patient organizations have played an important role in this process. The experience of members of these organizations, either as a study participant, parent or as a patient organization member communicating with the sponsors are potentially very informative for future trial design.

Impact of Regional Metastasis on Survival for Patients with Nonfunctional Pancreatic Neuroendocrine Tumors: A Systematic Review.

Background: Controversy exists regarding the benefit of lymphadenectomy for nonfunctional pancreatic neuroendocrine tumors (NF-PNET).

Patients And Methods: MEDLINE/PubMed, EMBASE, and the Cochrane Library were searched for studies of pancreatic neuroendocrine tumors (PNET) published between 1990 and 2021. Studies of functional PNET were excluded.

Advice to Clinicians From Expectant Parents at Extreme Prematurity: A Multimethod Study.

Background And Objectives: Despite recommendations for patient-centered counseling on extreme prematurity, clinicians often miss opportunities to communicate in a way that facilitates parental knowledge, decision-making, and emotional support. In this study, we aimed to determine empirical, parent-derived recommendations and advice for clinicians counseling on extreme prematurity.

Methods: Pregnant women (and their partners) admitted at 22 0/7 to 25 6/7 weeks' estimated gestation participated in postantenatal counseling semi-structured interviews or questionnaires to explore parental preferences in the counseling process, including advice to clinicians.

The experience of clinical study and trial participation in rare diseases: A scoping review of centronuclear myopathy and other neuromuscular disorders.

The design of a clinical trial for a rare disease can be challenging. An optimal study design is required to effectively study the clinical outcomes for possible therapies for these types of disorders. Understanding the study participants' experiences as well as barriers and facilitators of participation are important to optimize future research and to inform clinical trial management.

Development of an Online Training to Engage Home Visitors as Research Stakeholders.

Background: Home visiting (HV) has demonstrated positive impacts across family well-being domains. Home visitors receive training in HV model requirements as well as to develop knowledge and various skills. Despite growth in HV research, we are not aware of existing training or required competencies in research design, research methods, or dissemination of research findings for home visitors.

The Ultramarathon of Gene Therapy Development for Rare Diseases: How Can We Cross the Finish Line Together?

The promising developments in gene and cell therapy bring an array of hope to the >7000 rare disease patient communities, of which 90% to 95% are still working toward a treatment. However, the pace of gene and cell therapy development pathways often resembles ultramarathon relay races that potentially span decades. It is a pace that is incongruent to the sprint that most individuals with rare diseases and their families are living and working in pursuit of life-saving treatments.

Predicting the Next-Day Perceived and Physiological Stress of Pregnant Women by Using Machine Learning and Explainability: Algorithm Development and Validation.

Background: Cognitive behavioral therapy-based interventions are effective in reducing prenatal stress, which can have severe adverse health effects on mothers and newborns if unaddressed. Predicting next-day physiological or perceived stress can help to inform and enable pre-emptive interventions for a likely physiologically and perceptibly stressful day. Machine learning models are useful tools that can be developed to predict next-day physiological and perceived stress by using data collected from the previous day.

Amplifying patient voices amid pandemic: Perspectives on tracheostomy care, communication, and connection.

Objective: To investigate perspectives of patients, family members, caregivers (PFC), and healthcare professionals (HCP) on tracheostomy care during the COVID-19 pandemic.

Methods: The cross-sectional survey investigating barriers and facilitators to tracheostomy care was collaboratively developed by patients, family members, nurses, speech-language pathologists, respiratory care practitioners, physicians, and surgeons. The survey was distributed to the Global Tracheostomy Collaborative's learning community, and responses were analyzed.

Reducing posttraumatic stress disorder symptom severity among depressed women with childhood sexual abuse histories in interpersonal psychotherapy-trauma: The role of improved social functioning.

Objective: Childhood sexual abuse (CSA) increases risk for posttraumatic stress disorder (PTSD), and both CSA and PTSD are strongly associated with impaired social functioning. Interpersonal psychotherapy (IPT) has demonstrated promise for adults with PTSD and has been shown to be effective for women with PTSD and histories of CSA, but the mechanisms by which IPT might improve PTSD are unknown. The current secondary analysis tested whether the ameliorative effect of IPT-trauma (IPT-T) on PTSD, demonstrated in a randomized effectiveness trial, was mediated by improved social function.

Prevalence of hepatitis C in sexual assault survivors presenting to a SANE clinic: A descriptive analysis.

While previous epidemiologic research has demonstrated that sexual assault survivors (SAS) may be at disproportionate risk for exposure to Hepatitis C (HCV), HCV screening in SAS is not addressed in current post-sexual assault testing recommendations. We sought to identify the prevalence of HCV among a SAS cohort along with associated basic demographics. Opt-out HCV antibody screening and RNA confirmatory testing was provided for all SAS at a Sexual Assault Nurse Examiner (SANE) clinic in Birmingham, Alabama, from April 2020 through March 2021.

Cultural Adaptation of the Mothers and Babies Intervention for Use in Tribal Communities.

Objective: While one in five women may experience mood and anxiety disorders during pregnancy and postpartum, Indigenous identity increases that risk by 62%, especially among younger Indigenous women. The need for evidence-based perinatal mental health interventions that provide culturally relevant well-being perspectives and practices is critical to improving maternal, child, and community outcomes for Indigenous peoples, and reducing health inequities.

Methods: Through a collaboration between community maternal and child health professionals, intervention researchers, and a cultural consultant, our workgroup developed cultural adaptations to Mothers and Babies, an evidence-based perinatal depression prevention intervention.

Undifferentiated carcinoma with osteoclast-like giant cells: A pathologic-radiologic correlation of a rare histologic subtype of pancreatic ductal adenocarcinoma.

Undifferentiated carcinoma with osteoclast-like giant cells (UC-OGC) is an exceedingly rare subtype of pancreatic ductal adenocarcinoma. Histologically, UC-OGC is characterized by three cell types namely, a neoplastic mononuclear cell component, non-neoplastic osteoclast-like giant cells, and a non-neoplastic mononuclear histiocytic component. The behavior of this tumor is unpredictable; but many patients survive many years after diagnosis.

Tracheostomy care and communication during COVID-19: Global interprofessional perspectives.

Objective: Investigate healthcare providers, caregivers, and patient perspectives on tracheostomy care barriers during COVID-19.

Study Design: Cross-sectional anonymous survey SETTING: Global Tracheostomy Collaborative Learning Community METHODS: A 17-item questionnaire was electronically distributed, assessing demographic and occupational data; challenges in ten domains of tracheostomy care; and perceptions regarding knowledge and preparedness for navigating the COVID-19 pandemic.

Results: Respondents (n = 115) were from 20 countries, consisting of patients/caregivers (10.

Current Controversies in Neoadjuvant Therapy for Pancreatic Cancer.

Over the last two decades, there have been significant changes in the management of patients with localized pancreatic cancer. The rationale for an evolution toward a neoadjuvant approach and summary of relevant clinical trials is reviewed. Controversies in identifying optimal neoadjuvant therapeutic approaches are discussed.

Ki67 does not predict recurrence for low-grade appendiceal mucinous neoplasms with peritoneal dissemination after cytoreductive surgery and HIPEC.

Low-grade appendiceal mucinous neoplasms (LAMN) can disseminate to become low-grade mucinous carcinoma peritonei (LGMCP), which is optimally treated with cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (CRS/HIPEC). Approximately half of the patients with LGMCP recur despite complete cytoreduction, and risk factors for recurrence are poorly understood. We sought to evaluate if Ki67 predicts progression of LGMCP after CRS/HIPEC.