Publication of data collection forms from NHLBI funded sickle cell disease implementation consortium (SCDIC) registry.

BACKGROUND: Sickle cell disease (SCD) is an autosomal recessive blood disorder affecting approximately 100,000 Americans and 3.1 million people globally. The scarcity of relevant knowledge and experience with rare diseases creates a unique need for cooperation and infrastructure to overcome challenges in translating basic research advances into clinical advances. Despite registry initiatives in SCD, the unavailability of descriptions of the selection process and copies of final data collection tools, coupled with incomplete representation of the SCD population hampers further research progress. This manuscript describes the SCDIC (Sickle Cell Disease Implementation Consortium) Registry development and makes the SCDIC Registry baseline and first follow-up data collection forms available for other SCD research efforts. RESULTS: Study data on 2400 enrolled patients across eight sites was stored and managed using Research Electronic Data Capture (REDCap). Standardized data collection instruments, recruitment and enrollment were refined through consensus of consortium sites. Data points included measures taken from a variety of validated sources (PHENX, PROMIS and others). Surveys were directly administered by research staff and longitudinal follow-up was coordinated through the DCC. Appended registry forms track medical records, event-related patient invalidation, pregnancy, lab reporting, cardiopulmonary and renal functions. CONCLUSIONS: The SCDIC Registry strives to provide an accurate, updated characterization of the adult and adolescent SCD population as well as standardized, validated data collecting tools to guide evidence-based research and practice.

Strategies to Improve Management of Shoulder Dystocia Under the AHRQ Safety Program for Perinatal Care.

OBJECTIVE: To assess implementation of safety strategies to improve management of births complicated by shoulder dystocia in labor and delivery units. DESIGN: Mixed-methods implementation evaluation. SETTING/LOCAL PROBLEM: Labor and delivery units (N = 18) in 10 states participating in the Safety Program for Perinatal Care (SPPC). Shoulder dystocia is unpredictable, requiring rapid and coordinated action. PARTICIPANTS: Key informants were labor and delivery unit staff who implemented SPPC safety strategies. INTERVENTION/MEASUREMENTS: The SPPC was implemented by using the TeamSTEPPS teamwork and communication framework and tools, applying safety science principles (standardization, independent checks, and learn from defects) to shoulder dystocia management, and establishing an in situ simulation program focused on shoulder dystocia to practice teamwork and communication skills. Unit staff received training, a toolkit, technical assistance, and unit-specific feedback reports. Quantitative data on unit-reported process improvement measures and qualitative data from staff interviews were used to understand changes in use of safety principles, teamwork/communication, and in situ simulation. RESULTS: Use of shoulder dystocia safety strategies improved on the units. Differences between baseline and follow-up (10 months) were as follows: in situ simulation (50% vs. 89%), teamwork and communication (67% vs. 94%), standardization (67% to 94%), learning from defects (67% vs. 89%), and independent checks (56% vs. 78%). Interview data showed reasons to address management of shoulder dystocia, various approaches to implement safety practices, and facilitators and barriers to implementation. CONCLUSION: Successful management of shoulder dystocia requires a rapid, standardized, and coordinated response. The SPPC strategies to increase safety of shoulder dystocia management are scalable, replicable, and adaptable to unit needs and circumstances.

Automatically quantified diffuse excessive high signal intensity on MRI predicts cognitive development in preterm infants.

BACKGROUND: Cognitive and language impairments constitute the majority of disabilities observed in preterm infants. It remains unclear if diffuse excessive high signal intensity on magnetic resonance imaging at term represents delayed white matter maturation or pathology. METHODS: We hypothesized that diffusion tensor imaging-based objectively quantified diffuse excessive high signal intensity measures at term will be strong predictors of cognitive and language development at 2 years in a cohort of 41 extremely low birth weight (≤1000 g) infants. Using an automated probabilistic atlas, mean diffusivity maps were used to objectively segment and quantify diffuse excessive high signal intensity volume and mean, axial, and radial diffusivity measures. Standardized neurodevelopment was assessed at 2 years of age using the Bayley Scales of Infant Development, third edition. RESULTS: Thirty-six of the 41 infants (88%) had complete developmental data at follow-up. Objectively quantified diffuse excessive high signal intensity volume correlated significantly with cognitive and language scores at 2 years (P < 0.001 for both). The sum values of the three diffusivity measures in detected diffuse excessive high signal intensity regions also correlated significantly with the Bayley scores (r(2) 34.7%; P < 0.001 for each). Infants in the highest quartile for diffuse excessive high signal intensity volumes had scores between 19 and 24 points lower than infants in the lowest quartile (P < 0.01). When diagnosed subjectively by neuroradiologists however, Bayley scores were not significantly lower in infants with extensive diffuse excessive high signal intensity. CONCLUSIONS: These findings lend further evidence that diffuse excessive high signal intensity is pathologic and that objectively quantified diffusion-based diffuse excessive high signal intensity volume at term is associated with cognitive and language impairments. Our approach could be used for risk stratification and early intervention for such high-risk extremely preterm infants.

Perinatal clinical antecedents of white matter microstructural abnormalities on diffusion tensor imaging in extremely preterm infants.

OBJECTIVE: To identify perinatal clinical antecedents of white matter microstructural abnormalities in extremely preterm infants. METHODS: A prospective cohort of extremely preterm infants (N = 86) and healthy term controls (N = 16) underwent diffusion tensor imaging (DTI) at term equivalent age. Region of interest-based measures of white matter microstructure - fractional anisotropy and mean diffusivity - were quantified in seven vulnerable cerebral regions and group differences assessed. In the preterm cohort, multivariable linear regression analyses were conducted to identify independent clinical factors associated with microstructural abnormalities. RESULTS: Preterm infants had a mean (standard deviation) gestational age of 26.1 (1.7) weeks and birth weight of 824 (182) grams. Compared to term controls, the preterm cohort exhibited widespread microstructural abnormalities in 9 of 14 regional measures. Chorioamnionitis, necrotizing enterocolitis, white matter injury on cranial ultrasound, and increasing duration of mechanical ventilation were adversely correlated with regional microstructure. Conversely, antenatal steroids, female sex, longer duration of caffeine therapy, and greater duration of human milk use were independent favorable factors. White matter injury on cranial ultrasound was associated with a five weeks or greater delayed maturation of the corpus callosum; every additional 10 days of human milk use were associated with a three weeks or greater advanced maturation of the corpus callosum. CONCLUSIONS: Diffusion tensor imaging is sensitive in detecting the widespread cerebral delayed maturation and/or damage increasingly observed in extremely preterm infants. In our cohort, it also aided identification of several previously known or suspected perinatal clinical antecedents of brain injury, aberrant development, and neurodevelopmental impairments.