1. 1 An Update… CDC Surveillance Project on Bleeding Disorders Diane Aschman Administrative PI Marilyn Manco-Johnson Scientific PI

2. 2 Goals of the Surveillance Provide descriptive knowledge about the demographics, diagnoses and health service utilization of populations with bleeding disorders and venous thromboembolism receiving care at HTCs Monitor health indicators among populations with bleeding disorders – Assess trends over time – Measure rates of, and risk factors for, complications – Identify high risk populations for prevention – Identify issues that require research

3. 3 National Surveillance Program Organization National Surveillance Program Executive Committee Scientific PI Science Committee Chair/Co-Chairs 12 Regional Scientific Spokespersons HTC PIs Administrative Committee Chair/Co-Chairs 12 Multi-Site Surveillance Managers HTC Surveillance Data Officers Meets q 2 weeks Wednesday Meredith Oakley CDC Project Officer Meets monthly Tuesday Becky Dudley Chair Meets weekly Friday M. Manco-Johnson Scientific PI

4. 4 HTC Subaward Status – 97 signed as of 4/22/12

5. 5 Thank You for Signing On! Region VI Gulf States Louisiana Arkansas Fort Worth South Texas Texas Children’s Region VII Children’s Mercy Iowa John Bouhasin U of Missouri U of Nebraska

6. 6 Surveillance Design: HTC Grass Root Input 124 Respondents from 60 HTCs to the Online Survey 30% Hematologists; 50% Nursing; 20% Others

7. 7 Surveillance Components: 2 Parts Individual level data (not aggregate) De-identified (all 18 identifiers removed) Description of population from which registry data is drawn (vs. all patients served) No patient authorization required by CDC All or a subset of patients with eligible disorders Will require blood sample Patient authorization anticipated HTC Population Profile Registry for Bleeding Surveillance

8. 8 Surveillance Components: 2 Parts HTC Population Profile Registry for Bleeding Surveillance

9. 9 HTC Population Profile: Enrollment All HTC Patients Meeting Entry Criteria Inclusion Criteria Hereditary disorders – Factor VIII /IX def – VWD – Rare bleeding – Platelet (females) DVT or PE 1+ clinic/telemed encounter w/i year Exclusion Criteria Other or Non- hematologic disorders Males with platelet disorders Acquired bleeding disorders

10. 10 HTC Population Profile: Measures As Currently Proposed Race Ethnicity Gender Year of Birth Zip Code Insurance Status Year of Visit to HTC Primary Bleeding or Clotting Disorder Baseline factor level VTE Occurrence HCV Status HIV Status Unique Identifier HTC Identifier

11. 11 HTC Population Data Data Elements Originally Proposed by CDC Race Ethnicity Year of Birth Zip code Diagnosis (with severity or type) Age of diagnosis Disability status HCV/ HIV status Visits to HTC in this year Year of last visit Insurance type – primary Insurance type – secondary Who referred patient to HTC Purpose of most recent visit Unique identifier

12. 12 HTC Population Data Data Elements Originally Proposed by CDC after feedback Race Ethnicity Year of Birth Zip code Diagnosis (with severity or type) Age of diagnosis Disability status HCV/ HIV status Visits to HTC in this year Year of last visit Insurance type – primary Insurance type – secondary Who referred patient to HTC Purpose of most recent visit Unique identifier

13. 13 HTC Population Profile: Next Steps Finalize Data Elements and Approach including specifications for changes to WebTracker Step 1 Create New Data Forms in WebTracker including testing of aggregation & transmission Step 2 Train HTCs regarding data elements, human subjects issues, submission process Step 3 Submit HTC Population Profile data (CDC Surveillance Project Part 1) Step 4

14. 14 Step 1: Finalize Data Elements Finalize Data Elements including specifications for changes to WebTracker Step 1 Create New Data Forms in WebTracker including testing of aggregation & transmission Step 2 Train HTCs regarding data elements, human subjects issues, submission process Step 3 Submit HTC Population Profile data (CDC Surveillance Project Part 1 Step 4 Demographic and basic diagnosis info – Similar to HDS – Most data elements overlap with ATHNdataset core – De-identified so no patient consent

15. 15 Step 2: Create New Data Forms Finalize Data Elements including specifications for changes to WebTracker Step 1 Create New Data Forms in WebTracker including testing of aggregation & transmission Step 2 Train HTCs regarding data elements, human subjects issues, submission process Step 3 Submit HTC Population Profile data (CDC Surveillance Project Part 1 Step 4 90 days following final data elements Data currently in WebTracker will pre-fill new surveillance forms Can input data now

16. 16 Step 3: Train HTCs Finalize Data Elements including specifications for changes to WebTracker Step 1 Create New Data Forms in WebTracker including testing of aggregation & transmission Step 2 Train HTCs regarding data elements, human subjects issues, submission process Step 3 Submit HTC Population Profile data (CDC Surveillance Project Part 1 Step 4 Phase 1: Getting Started – ATHNreport Phase 2: Regional Meetings Phase 3: Webinars – w/i 75-90 days Phase 4: Ongoing support

17. 17 Surveillance Components: 2 Parts HTC Population Profile Registry for Bleeding Surveillance

18. 18 Registry: Enrollment Patients must be enrolled in the HTC Population Profile Inclusion Criteria Hereditary disorders – Factor VIII /IX def – VWD – Rare bleeding – Platelet (females) 1+ clinic/telemed encounter w/i year Exclusion Criteria Other or Non- hematologic disorders Males with platelet disorders Acquired bleeding disorders

19. 19 Registry: Data Elements Being defined by Science Committee in collaboration with CDC Striving for consistency across disorders where ever possible Narrowing down to most salient measures – Intracranial hemorrhage - CVAD – Inhibitors - Genetics/Family HX – Joint disease - Healthy Life-style – Product use – Prophylaxis

20. 20 We Need & Want Your Involvement Weigh in on Registry Data Elements – Contact regional leadership – Review at www.htcnetwork.orgwww.htcnetwork.org Start amassing HTC Population Profile data Keep up to date – ATHNreport – Trainings

21. 21 Announcing… ATHNdataset A Growing Resource for Bleeding Disorders Community Diane Aschman

22. 22 Announcing ATHNdataset: A Growing Resource Created by American Thrombosis and Hemostasis Network (ATHN) in collaboration with its 133 affiliated hemophilia treatment centers Brings together standardized demographic and clinical data into one national dataset Is a “Limited Data Set” as defined by Privacy Rule – Stripped of 16 specified direct identifiers – Used or disclosed for public health, research or health care operations – Disclosure covered by data use agreements

23. 23 Infrastructure Rollout Status September 2010 – March 2012

24. 24 ATHNdataset is Growing Patient authorizations as of March 31, 2012 9240 Patients choose to “opt in” by signing a patient authorization - 9240 patients are in! No special lab tests or clinic visits Patient identity is protected with a unique patient identifier

25. 25 Patient Authorizations, All Regions

26. 26 Sample Data Fields Applicable to Cohort Studies in WebTracker & ATHNadvoy DEMOGRAPHICSCLINICAL INFOOUTCOMES Date of birth*Primary*/other diagnoses1 st */all Bleed events Race*Date of diagnosis*1 st Bleed location* Ethnicity*Disease severity*Prophylaxis used Gender*Factor levels*Treatments used Age- 1 st home infusion*Treatments prescribed*Date of death* Marital statusAge at 1 st exposure*Cause of death* Education levelSurgeries/procedures*Anemia Language*Inhibitor status* Payer type*Weight/Height* Co-morbidities-HIV, Hep* Molecular defect Standardized using SNOMED CT, LOINC and First DataBank’s NDDF * Core data element

27. 27 What’s not in the ATHNdataset? Identifiers NOT in the ATHNdataset, whichis a limited data set NameSocial security #Certificate/license # Postal addressMedical records #Vehicle ID/serial# Telephone #Health plan beneficiary #Device ID/serial # Fax #Account #Web URL Email addressFull faced photosIP address Biometric identifiers Data of patients who did not sign the authorization 16 identifiers excluded from a limited data set (HIPAA)

28. 28 Why opt in? The Power is in the Numbers – A Large Pool of Data is Needed Community The HTCPatients Many critical questions need answers – Scientific, public health, policy – ATHNdataset supports research Also supports delivery of care, public health reporting, outcomes assessment and advocacy Same data enables HTC to create your ATHNready Personal Health Report for emergency preparedness

29. 29 ATHNdataset Demographics as of March 31, 2012 Authorized Patients (n=9,240) # of Patients % of Patients SEX Males6,32968.5% Females2,91131.5% AGE < 18 years4,67650.6% 18+ years4,56449.4% RACE* White7,80884.5% Non-White1,40815.2% ETHNICITY* Hispanic or Latino1,06311.5% Non-Hispanic or Latino8,14888.2% * 29 unknown

30. 30 Patients By Primary Diagnosis (9,240 Authorized Patients as of March 31, 2012) FVIII VWD

31. 31 ATHNdataset by Diagnosis as of March 31, 2012 Primary Diagnosis Group# of Patients% of Patients Factor VIII Carrier430.47% Factor VIII Deficiency3,58838.83% Factor IX Carrier200.22% Factor IX Deficiency1,10011.90% Von Willebrand Disease2,24424.29% Rare Disorders3964.29% Platelet Disorders7728.35% Thrombophilias3263.53% Venous Thrombosis2432.63% Other/Unknown5085.50% TOTAL AUTHORIZED PATIENTS9,240100.00%

32. 32 Patient Disease Severity & Inhibitors (3,588 FVIII Deficiency Authorized Patients as of March 31, 2012) 9.92%

33. 33 Prophylaxis Across Age Groups in Factor VIII Deficiency Patients* ATHNdataset as of March 31, 2012 (n=3588) 44% *unaudited

34. 34 Payer Type: Primary Factor Payer (2,733 of 9,240 Authorized Patients as of March 31, 2012)

35. 35 Dashboard Report Tracks HTC Data Capture and Patient Authorizations

36. 36 ATHNdataset Patient Registry with Standard Building Blocks…Multiple Uses Care Performance Mgt. ResearchPublic Health HTC patient profiles, accessible/reportable data Comparisons with peers or best practices Eligibility screening, cohort tracking, hypothesis testing Combining core and enriched data elements