IDDRC: Biostatistics and Informatics

Staff Directors Robert McCarter, ScD, Director 202-476-5000 rmccarte@childrensnational.org     Avital Cnaan, PhD, Co-director 202-476-3109 acnaan@childrensnational.org Faculty Jichuan Wang, PhD Karen Gold, PhD Informatics Support Anne Sill, BS Saeid Goudarzi, BS Paul Moscatt, BS Data Analysis Jianping He, MS Yao Cheng, MS Administrative Support Annie Fulton

• Brainstorming of alternative designs and approaches
• Review of study descriptions including:
  • Clarity of aims and testability of hypotheses
  • Adequacy of the definition of key variables
• Review and critique of study protocol
• Sample size estimation, including statistical power
• Development of analysis plans and corresponding databases to address each aim and hypothesis
• Other support, such as randomization, based on the needs of the study

• Consultation on study implementation including selection of the appropriate information management system:
  • REDCap - Research Electronic Data Capture¹
    
    • Account creation, assistance with set-up, and training
    • Review of the information system, especially the database structure
  • CHRIS*
    • Development of web-based data collection forms
    • Definition and management of study schedules
    • Configuration of the study information system to match study requirements
    • Development of Reports, including Data and Safety Management Reports

Analysis of data to address research questions

• Implementation of analyses including advanced modeling to accommodate:
  • Longitudinal data
  • Hierarchical structures
  • Path and structural equation modeling
  • Other methods, based on the needs of the study
• Written and verbal interpretation of results

• Publication review and critique
• Development of methods sections
• Assistance producing tables and figures

• Critical evaluation of medical literature
• Writing aims and hypotheses
• Design of research studies
• Sample size and power
• Study and research information management

• Six centrally-managed servers that have been exclusively dedicated to support study databases
• Six web servers outside the institutional firewall to provide password-protected, role-based access to local and remote investigators
• A SharePoint server for document collaboration, file sharing, workflow development, and access to reporting and scheduling tools
• Three MAC Pro servers equipped with high-performance processors, RAM, and storage to support the power-computing needs of investigators.
• Expanded Internet bandwidth for improved access to true high-performance computing with massive storage through university-based partners at George Washington and Virginia Tech

Publications

Chakrabarti L, Best TK, Cramer NP, Carney RS, Isaac JT, Galdzicki Z, Haydar TF. Olig1 and Olig2 triplication causes developmental brain defects in Down syndrome. Nat Neurosci, 2010 Aug;13(8):927-34, Epub 2010.

Ah Mew N., McCarter R., Daikhin Y., Nissim I., Yudkoff M. and Tuchman M. N-carbamylglutamate augments ureagenesis and reduces ammonia and glutamine in propionic acidemia. Pediatrics, 126:208-14, 2010.

Lyon ME, Garvie PA, Briggs L, He J, D'Angelo L, McCarter R. Development, Feasibility and Acceptability of the Family-Centered (FACE) Advance Care Planning Intervention for Adolescents with HIV. J Palliative Medicine, 12(4), 363-72, 2009.

Pearl PL, Vezina LG, Saneto RP, McCarter R, Molloy-Wells E, Heffron A, Trzcinski S, McClintock WM, Conry JA, Elling NJ, Goodkin HP, de Menezes MS, Ferri R, Gilles E, Kadom N, Gaillard WD. Cerebral MRI abnormalities associated with vigabatrin therapy. Epilepsia, 50(2):184-94, 2009.

Gropman AL, Fricke ST, Seltzer RR, Hailu A, Adeyemo A, Sawyer A, van Meter J, Gaillard WD, McCarter R, Tuchman M, Batshaw M; Urea Cycle Disorders Consortium. 1H MRS identifies symptomatic and asymptomatic subjects with partial ornithine transcarbamylase deficiency. Mol Genet Metab, 95(1-2):21-30, 2008.

Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao GP, Raper SE, Wilson JM, Batshaw ML. Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther., 14(1):25-33, 2006.