Defining Natural History: Assessment of the Ability of College Students to Aid in Characterizing Clinical Progression of Niemann-Pick Disease, Type C

Niemann-Pick Disease, type C (NPC) is a fatal, neurodegenerative, lysosomal storage disorder. It is a rare disease with broad phenotypic spectrum and variable age of onset. These issues make it difficult to develop a universally accepted clinical outcome measure to assess urgently needed therapies. To this end, clinical investigators have defined emerging, disease severity scales. The average time from initial symptom to diagnosis is approximately 4 years. Further, some patients may not travel to specialized clinical centers even after diagnosis. We were therefore interested in investigating whether appropriately trained, community-based assessment of patient records could assist in defining disease progression using clinical severity scores. In this study we evolved a secure, step wise process to show that pre-existing medical records may be correctly assessed by non-clinical practitioners trained to quantify disease progression. Sixty-four undergraduate students at the University of Notre Dame were expertly trained in clinical disease assessment and recognition of major and minor symptoms of NPC. Seven clinical records, randomly selected from a total of thirty seven used to establish a leading clinical severity scale, were correctly assessed to show expected characteristics of linear disease progression. Student assessment of two new records donated by NPC families to our study also revealed linear progression of disease, but both showed accelerated disease progression, relative to the current severity scale, especially at the later stages. Together, these data suggest that college students may be trained in assessment of patient records, and thus provide insight into the natural history of a disease

Homogeneous and heterogeneous catalysts for multicomponent reactions

[EN] Organic synthesis performed through multicomponent reactions is an attractive area of research in organic chemistry. Multicomponent reactions involve more than two starting reagents that couple in an exclusive ordered mode under the same reaction conditions to form a single product which contains the essential parts of the starting materials. Multicomponent reactions are powerful tools in modern drug discovery processes, because they are an important source of molecular diversity, allowing rapid, automated and high throughput generation of organic compounds. This review aims to illustrate progress in a large variety of catalyzed multicomponent reactions performed with acid, base and metal heterogeneous and homogeneous catalysts. Within each type of multicomponent approach, relevant products that can be obtained and their interest for industrial applications are presented.The authors wish to gratefully acknowledge the Generalitat Valenciana for the financial support in the project CONSOLIDER-INGENIO 2010 (CSD2009-00050)Climent Olmedo, MJ.; Corma Canós, A.; Iborra Chornet, S. (2012). Homogeneous and heterogeneous catalysts for multicomponent reactions. RSC Advances. 2(1):16-58. https://doi.org/10.1039/c1ra00807bS16582

Cultural Beliefs among Latinas: The Role of Acculturation and Impact on Timeliness of Breast Cancer Care

Certain cultural beliefs related to breast cancer may act as a barrier to a woman seeking breast cancer preventive services or timely follow-up for a breast symptom. For Latinas, holding beliefs that are inconsistent with healthcare-seeking behavior may lead to low or delayed utilization of care regardless of access. This study explores factors associated with breast cancer beliefs among Latina women and how this might lead to delays in seeking care or receiving treatment for breast cancer. Data were obtained from a population-based sample of 181 urban Latina women recruited as part of the Breast Cancer Care in Chicago (BCCC) study. Women were ages 30–79 and had been diagnosed with primary in situ or invasive breast cancer. Interviews included a cultural beliefs scale spanning a range of beliefs that could be inconsistent with motivation to seek timely healthcare. The total number of beliefs was dichotomized at the sample mean, such that patients holding three or more beliefs were compared to patients holding two or less beliefs. Sociodemographic, socioeconomic, acculturation, and access/care utilization factors were examined as potential confounders. Three outcomes were examined: patient delay, clinical delay, and total delay. Seventy-five percent of women held one or more beliefs. The belief most commonly held was, “Faith in God can protect you from breast cancer” (48%). Fifty percent of the sample had an acculturation score of zero (Mean=1). Lower acculturation was associated with greater beliefs. Patients with less income, less education, and lacking private health insurance also tended to hold more beliefs (p<.05). Both clinical delay (57% versus 43%, p=.07) and total delay (59% versus 32%, p=.0005) were more common for women holding three or more beliefs versus two or less. After adjusting for age, education, income, and acculturation, holding three or more beliefs was associated with 3.35 times the odds of experiencing a total delay compared to holding two or less beliefs. Cultural beliefs may predispose certain Latina women who are less acculturated and of lower socioeconomic status to prolong seeking care for breast symptoms and may influence delays in receiving treatment for breast cancer

Patient and Other Stakeholder Engagement in Patient-Centered Outcomes Research Institute Funded Studies of Patients with Kidney Diseases

Including target populations in the design and implementation of research trials has been one response to the growing health disparities endemic to our health care system, as well as an aid to study generalizability. One type of community-based participatory research is “Patient Centered-Research”, in which patient perspectives on the germane research questions and methodologies are incorporated into the study. The Patient-Centered Outcomes Research Institute (PCORI) has mandated that meaningful patient and stakeholder engagement be incorporated into all applications. As of March 2015, PCORI funded seven clinically-focused studies of patients with kidney disease. The goal of this paper is to synthesize the experiences of these studies to gain an understanding of how meaningful patient and stakeholder engagement can occur in clinical research of kidney diseases, and what the key barriers are to its implementation. Our collective experience suggests that successful implementation of a patient- and stakeholder-engaged research paradigm involves: (1) defining the roles and process for the incorporation of input; (2) identifying the particular patients and other stakeholders; (3) engaging patients and other stakeholders so they appreciate the value of their own participation and have personal investment in the research process; and (4) overcoming barriers and challenges that arise and threaten the productivity of the collaboration. It is our hope that the experiences of these studies will further interest and capacity for incorporating patient and stakeholder perspectives in research of kidney diseases

Patient and other stakeholder engagement in patient-centered outcomes research institute funded studies of patients with kidney diseases

© 2016 by the American Society of Nephrology. Including target populations in the design and implementation of research trials has been one response to the growing health disparities endemic to our health care system, as well as an aid to study generalizability. One type of community-based participatory research is “Patient Centered-Research”, in which patient perspectives on the germane research questions and methodologies are incorporated into the study. The Patient-Centered Outcomes Research Institute (PCORI) has mandated that meaningful patient and stakeholder engagement be incorporated into all applications. As of March 2015, PCORI funded seven clinically-focused studies of patients with kidney disease. The goal of this paper is to synthesize the experiences of these studies to gain an understanding of how meaningful patient and stakeholder engagement can occur in clinical research of kidney diseases, and what the key barriers are to its implementation. Our collective experience suggests that successful implementation of a patient- and stakeholder-engaged research paradigm involves: (1) defining the roles and process for the incorporation of input; (2) identifying the particular patients and other stakeholders; (3) engaging patients and other stakeholders so they appreciate the value of their own participation and have personal investment in the research process; and (4) overcoming barriers and challenges that arise and threaten the productivity of the collaboration. It is our hope that the experiences of these studies will further interest and capacity for incorporating patient and stakeholder perspectives in research of kidney diseases