1. Introduction to the NHLBI Context

2. Agenda  Introduction to the NMDP / CIBMTR  Forms being curated  Progress update  Description of review process 2

3. NMDP  National Marrow Donor Program  Be the Match Registry  World’s largest registry of potential bone marrow / PBSC donors and donated umbilical cord blood units  Matches donors to recipients  Facilitates transplantation from initial search through product delivery

4. CIBMTR  Center for International Blood and Marrow Transplant Research  Research division of the NMDP  Collects and maintains outcomes data  Conducts clinical trials  Maintains a repository of paired donor and recipient blood samples  Mission is to improve patient outcomes and ensure donor safety

5. Forms That Have Been Curated  Pre-transplantation  Pre-transplantation diagnosis and treatment  Infectious disease markers  HLA typing  Product collection, storage, transport and infusion  Post-transplantation  Outcome  Annual follow-up  Death 5

6. Diseases (1 of 2) * Form Builder CRF  * Acute Myelogenous Leukemia (AML)  * Acute Lymphoblastic Leukemia (ALL)  * Chronic Myelogenous Leukemia (CML)  * Chronic Lymphoblastic Leukemia (CLL)  Myelodysplasia / Myeloproliferative Disorders  Multiple Myeloma / Plasma Cell Leukemia  * Amyeloidosis 6

7. Diseases (1 of 2)  * Hodgkin and Non-Hodgkin Lymphoma  * Breast cancer  * Testicular cancer  * Central nervous system tumors  Neuroblastoma  * Aplastic anemia  Fanconi anemia  * Immune deficiencies  * Mucopolysaccharidosis 7

8. Disease Status Case Report Forms  35 diseases  Type of Information  Diagnosis date  Current status  Best response to therapy  Form Builder Reports  Pre-Transplantation Disease Status Report: 3309539v1.0  Post-Transplantation Disease Status Report: 3319777v1.0 8

9. Progress Update  Recently released more than 600 CDEs  BRIDG  Majority of CDEs have been mapped to BRIDG  In the process of adding to the CDEs  Alternate names that indicate BRIDG class and attribute  Adding a reference document that indicates the BRIDG mapping path 9

10. Review Process  CDE  Automated error and validation reports  Peer  Technical  Subject matter expert  Form Builder  Automated comparison of question text and PVs in Form Builder and the CDE  Peer  Technical  Subject matter  Pre-release checklist 10

11. Appendix 11

12. Report Generation Tool  Custom-built application that generates reports based on CDE Browser XML  Performs some automated testing  Both template and custom designed reports 12

13. Report Generation Tool User Interface 13

14. Report Generation Tool Lookup Table 14

15. Report Generation Tool Error Report 15

16. Report Generation Tool Validation Report 16

17. Report Generation Tool Peer Report 17

18. Report Generation Tool Subject Matter Expert Review 18

19. Report Generation Tool Custom Reports 19

20. Disease Status CRFs Included Diseases  Acute Lymphoblastic Leukemia  Acute Myelogenous Leukemia  Amyloidosis  Breast Cancer  Central Nervous System Tumor  Chediak-Higashi Syndrome  Chronic Granulomatous Disease  Chronic Lymphocytic Leukemia  Chronic Myelogenous Leukemia  Congenital Amegakaryocytic Thrombocytopenia  Fanconi Anemia / Constitutional Anemia  Hemophagocytic Lymphohistiocytosis  Hepatitis Serology  Hodgkin and Non-Hodgkin Lymphoma  Human Immunodeficiency Virus  Juvenile Idiopathic Arthritis  Juvenile Myelomonocytic Leukemia  Langerhans Cell Histiocytosis  Leukodystrophies  Multiple Myeloma / Plasma Cell Leukemia  Multiple Sclerosis  Myelodysplasia / Myeloproliferative Disorders  Neuroblastoma  Osteopetrosis  Ovarian Cancer  Renal Carcinoma  Rheumatoid Arthritis  Sarcoma  Sickle Cell Anemia 20

21. Disease Status CRFs Included Diseases  Small Cell Lung Cancer  Systemic Lupus Erythematosus  Systemic Sclerosis  Testicular / Germ Cell Cancer  Waldenström's Macroglobulinemia  X-Linked Lymphoproliferative Syndrome 21