Mast Cell Diseases in Practice and Research: Issues and Perspectives Raised by Patients and Their Recommendations to the Scientific Community and Beyond.

BACKGROUND: Since 2010, patients and physicians have collaborated to understand unmet needs of patients with mast cell diseases, incorporating mastocytosis and mast cell activation disorders, which include mast cell activation syndromes. OBJECTIVE: This Open Innovation in Science project aims to expand understanding of the needs of patients affected by mast cell diseases, and encourage global communication among patient advocacy groups, physicians, researchers, industry, and government. A major aim is to support the scientific community's efforts to improve diagnosis, management, therapy, and patients' quality of life by addressing unmet needs. METHODS: In collaboration with mast cell disease specialists, 13 patient advocacy groups from 12 countries and regions developed lists of top patient needs. A core team of leaders from patient advocacy groups collected and analyzed the data and proposed possible actions to address patient needs. RESULTS: Findings identified similarities and differences among participating countries in unmet needs between patients with mastocytosis and those with mast cell activation syndromes. Issues emphasized struggles relating to the nature and rarity of mast cell diseases, their impact on quality of life, the diagnostic process, access to appropriate care, more effective treatment, and the need for research. CONCLUSIONS: Solutions vary across countries because situations differ, in particular regarding the existence of and access to centers of excellence and reference centers. Multifaceted mast cell activation syndrome barriers necessitate innovative approaches to improve access to appropriate care. The outcomes of this project should greatly support scientists and clinicians in their efforts to improve diagnosis, management, and treatment of patients with mastocytosis and mast cell activation disorders.

Newborn Resuscitation Skills in Health Care Providers at a Zambian Tertiary Center, and Comparison to World Health Organization Standards.

BACKGROUND: Birth asphyxia is a leading cause of early neonatal death. In 2013, 32% of neonatal deaths in Zambia were attributable to birth asphyxia and trauma. Basic, timely interventions are key to improving outcomes. However, data from the World Health Organization suggest that resuscitation is often not initiated, or is conducted suboptimally. Currently, there are little data on the quality of newborn resuscitation in the context of a tertiary center in a lower-middle income country. We aimed to measure the competencies of clinical practitioners responsible for newborn resuscitation. METHODS: This observational study was conducted over 5 months in Zambia. Health care professionals were recruited from anesthesia, pediatrics, and midwifery. Newborn skills and knowledge were examined using the following: (1) multiple-choice questions; (2) a ventilation skills test; and (3) 2 low-medium fidelity simulation scenarios. Participant demographics including previous resuscitation training and a self-efficacy rating score were noted. The primary outcome examined performance scores in a simulated scenario, which assessed the care of a newborn that failed to respond to basic interventions. Secondary outcome measures included apnea times after delivery and performance in the other assessments. RESULTS: Seventy-eight participants were enrolled into the study (13 physician anesthesiology residents, 13 pediatric residents, and 52 midwives). A significant difference in interprofessional performance was observed when examining checklist scores for the unresponsive newborn simulated scenario (P = .006). The median (quartiles) checklist score (out of 18) was 14.0 (13.0-14.75) for the anesthesiologists, 11.0 (8.5-12.3) for the pediatricians, and 10.8 (8.3-13.9) for the midwives. A score of 14 or more was required to pass the scenario. There was no significant difference in performance between participants with and without previous newborn resuscitation training (P = .246). The median (quartiles) apnea time after delivery was significantly different between all groups (P = .01) with anesthetic and pediatric residents performing similarly, 61 (37-97) and 63 (42.5-97.5) seconds, respectively. The midwifery participants displayed a significantly longer apnea time, 93.5 (66.3-129) seconds. Self-efficacy rating scores displayed no correlation between confidence level and the primary outcome, Spearman coefficient 0.06 (P = .55). CONCLUSIONS: Newborn resuscitation skills among health care professionals are varied. Midwives lead the majority of deliveries with anesthesiologists and pediatricians only being present at operative or high-risk births. It is therefore common that midwifery practitioners will initiate resuscitation. Despite this, midwives perform poorly when compared to anesthesia and pediatric residents. To address this discrepancy, a multidisciplinary, simulation-based newborn resuscitation program should be considered with continual clinical reenforcement of best practice.

Does the 1H-NMR plasma metabolome reflect the host-tumor interactions in human breast cancer?

Breast cancer (BC) is the most common diagnosed cancer and the leading cause of cancer death in women worldwide. There is an obvious need for a better understanding of BC biology. Alterations in the serum metabolome of BC patients have been identified but their clinical significance remains elusive. We evaluated by 1H-Nuclear Magnetic Resonance (1H-NMR) spectroscopy, filtered plasma metabolome of 50 early (EBC) and 15 metastatic BC (MBC) patients. Using Principal Component Analysis, Partial Least-Squares Discriminant Analysis and Hierarchical Clustering we show that plasma levels of glucose, lactate, pyruvate, alanine, leucine, isoleucine, glutamate, glutamine, valine, lysine, glycine, threonine, tyrosine, phenylalanine, acetate, acetoacetate, ß-hydroxy-butyrate, urea, creatine and creatinine are modulated across patients clusters. In particular lactate levels are inversely correlated with the tumor size in the EBC cohort (Pearson correlation r = -0.309; p = 0.044). We suggest that, in BC patients, tumor cells could induce modulation of the whole patient's metabolism even at early stages. If confirmed in a lager study these observations could be of clinical importance.