A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.

Deregulation of the mTOR pathway may play an important role in tumor biology when the APC/ß-catenin pathway is disrupted in desmoid-type fibromatosis (DT). A pilot study was conducted to determine whether sirolimus can block the mTOR pathway (primary aim) as well as determine whether it can safely be given in the preoperative setting, decrease tumor size/recurrence, and decrease tumor-associated pain in children and young adults (secondary aims) with DT. Nine subjects ages 5-28 years were enrolled from 2014 to 2017 across four centers. Sirolimus was feasible and was associated with a nonstatistically significant decrease in pS706K activation.

A Time-and-Motion Study of Clinical Trial Eligibility Screening in a Pediatric Emergency Department.

OBJECTIVE: Challenges with efficient patient recruitment including sociotechnical barriers for clinical trials are major barriers to the timely and efficacious conduct of translational studies. We conducted a time-and-motion study to investigate the workflow of clinical trial enrollment in a pediatric emergency department. METHODS: We observed clinical research coordinators during 3 clinically staffed shifts. One clinical research coordinator was shadowed at a time. Tasks were marked in 30-second intervals and annotated to include patient screening, patient contact, performing procedures, and physician contact. Statistical analysis was conducted on the patient enrollment activities. RESULTS: We conducted fifteen 120-minute observations from December 12, 2013, to January 3, 2014 and shadowed 8 clinical research coordinators. Patient screening took 31.62% of their time, patient contact took 18.67%, performing procedures took 17.6%, physician contact was 1%, and other activities took 31.0%. CONCLUSIONS: Screening patients for eligibility constituted the most time. Automated screening methods could help reduce this time. The findings suggest improvement areas in recruitment planning to increase the efficiency of clinical trial enrollment.

Factors associated with participation in research conducted in a pediatric emergency department.

OBJECTIVE: To examine the association of demographic and study characteristics in eligible subjects who agree to participate compared with those who did not participate in clinical research studies in a pediatric emergency department (PED). METHODS: Information for all families approached for participation in PED-based clinical research studies during a 6-year period was recorded in an electronic database. This included demographic factors, decision to participate, primary reason for not participating, and study characteristics. Forty studies were included in this analysis. Differences in participation rate among demographic and study characteristics were examined. Multivariable logistic regression was used to predict the likelihood of participation. RESULTS: Participation rates were similar with respect to sex (50.1% in male vs 49.9% in female), whereas families with younger children were more likely to participate (mean age, 8.5 years vs 10.2 years among nonparticipants P < 0.001). White patients were more likely to participate than African American patients (54.7% vs 45.6% in African Americans, P < 0.001). The presence of compensation, brief time requirement, and older children was negatively associated with participation for moderate to very invasive studies. However, for noninvasive and mildly invasive studies, the presence of compensation and the time required were not associated with participation. CONCLUSIONS: Study characteristics including invasiveness, time required of patients, and whether compensation is offered, along with demographic factors, influence participation in clinical studies conducted in the PED. When designing a research study in the PED, these, along with novel approaches to including all races and ethnicities in PED research, should be considered.

Caring in pediatric emergency nursing.

An environment committed to providing family-centered care to children must be aware of the nurse caring behaviors important to parents of children. This descriptive study assessed the psychometrics of a revised version of the Caring Behaviors Assessment (CBA) and examined nurse caring behaviors identified as important to the parents of pediatric patients in a pediatric emergency department. Jean Watson's theory of human caring provided the study's theoretical underpinnings. The instrument psychometrics was determined through an index of content validity (CVI) and internal consistency reliability. The instrument was determined to be valid (CVI = 3.75) and reliable (Cronbach's alpha = .971). The revised instrument was completed by a stratified, systematic random sample of 300 parents of pediatric emergency patients. Participants rated the importance of each item for making the child feel cared for by nurses. Individual survey item means were computed. Items with the highest means represented the most important nurse caring behaviors. Leading nurse caring behaviors centered on carative factors of "human needs assistance" and "sensitivity to self and others." Nearly all nurse caring behaviors were important to the parents of pediatric patients, although some behaviors were not priority. It is important for nurses to provide family-centered care in a way that demonstrates nurse caring.