2008© COPYRIGHT Thrombosis/thrombophilia patients captured at HTCs participating in ATHN Ellis J. Neufeld MD, PhD Director, Boston Hemophilia Center Director, Thrombosis Service, Children’s Hospital Boston Associate Professor of Pediatrics, Harvard Medical School

2008© COPYRIGHT 2 Topics How does the over-arching ATHN structure allow for thrombosis (or thrombophilia or both) to fit in with hemophilia data efforts? How are sites which responded to ATHN surveys seeing thrombosis patients? Regardless of how the patients are seen, how are these sites collecting data about thrombosis/thrombophilia? Audience participation – what are we doing now? Where do we go from here? Light blue text: Neufeld editorial comments

2008© COPYRIGHT 3 Shared and distinct characteristics between bleeding disorders and thrombosis, clinical research, public health Incidence and prevalence: thrombotic disorders are vastly more common than inherited bleeding disorders. – Hemophilia ~20,000 individuals, life-long disorders – Thrombophilia -5% of Caucasian population with factor V Leiden, but only a minority get clots. – Thrombosis - >>100,000 new cases per year of DVT, plus hundreds of thousands of arterial thrombotic events – Closest relative incidence: thrombosis in thrombophilia patients plus severe thrombophilias, e.g. protein C or AT3 deficiency Caregivers: Expertise in bleeding disorders and thrombophilias tends to reside in the same group of hematologists. – But Hemophilia Centers are often distinct and relatively few in number, and – Anticoagulation clinics, which deal with warfarin or heparin for any indication are even more often distinct Funding streams – – CDC thrombosis centers vs HTCs – 340B funding and mission statement of HTCs – Relationship to industry VERY different for thrombosis and bleeding disorders (except the rare severe protein deficiencies)

2008© COPYRIGHT 4 ATHN’s Strategic Direction Network Infrastructure Data Management Public Health and Safety Outcomes Improvement Data Analysis and Research Data safety, back-up, and portability Support surveillance and patient safety Use data to drive quality, service, and patient centered care Facilitate knowledge creation and advocacy Develop electronic database capacity (In principle, each of these five elements can apply to thrombosis and thrombophilia)

2008© COPYRIGHT 5 HTC Needs Assessment eSurvey on ATHN website – Initiated mid-October 2007 Respondents – 79 of 134 (59%) HTCs responded – All 6 (100%) Region VII responded Objectives of survey – Assess state of current data collection and use – Facilitate improved data collection – Develop fact base to provide data manager funding/support to HTC’s Focus on thrombosis/thrombophilia responses

2008© COPYRIGHT 6 Currently Using Lab Tracker (Based on 79 Respondents) We may be hearing back mostly from the choir

2008© COPYRIGHT 7 Data Routinely Computerized in Lab Tracker (Based on 78 Respondents)

2008© COPYRIGHT 8 Patient Types Entered into Lab Tracker

2008© COPYRIGHT 9 Thrombosis Patients (Based on the initial 78 respondents) Audience participation – what does “within hemophilia Ctr mean to you?

2008© COPYRIGHT 10 Patients with Thrombotic Disorder at HTC 51 of 78 HTCs reporting This adds up to AT LEAST 6000 patients “without even trying”. But 2/3 of these at 15 sites

2008© COPYRIGHT 11 Types of Thrombosis Patients Seen These categories are very different, but not mutually exclusive

2008© COPYRIGHT 12 ATHN’s Strategic Opportunities Network Infrastructure Develop electronic database capacity Gain and engage ATHN Affiliates re: database Build community trust Expand local capacity by funding and training data managers Establish regional data management structure Define and establish the core dataset: bleeding and clotting

2008© COPYRIGHT 13 How Will Data Flow? HTC Central Server: Secure and Encrypted Data Central Server: Secure and Encrypted Data Non-identifiable Consented Limited Dataset Non-identifiable Consented Limited Dataset Patient Consented Patients Only Access by Certificate Only Care Management (virtual HTC safe deposit boxes in a secure server vault) Public Health & Research (includes ATHN)

2008© COPYRIGHT 14 ATHN’s Strategic Opportunities Get data to clinical decision makers Help insure the survival of treatment centers by demonstrating and driving quality and service (Future) Explore opportunities to provide clinical decision support tools as components of the web infrastructure Outcomes Improvement Use data to drive quality, service, and patient centered care Can we map these opportunities to thrombosis? How?

2008© COPYRIGHT 15 ATHN’s Strategic Opportunities Clarify roles vis-à-vis CDC, HTRS, MCHB re: research Attract research proposals to appropriately use the database Publish related to our work Formalize relationship with the for- profit sector Data Analysis and Research Facilitate knowledge creation and advocacy These all mean something very different in thrombosis/thrombophilia than they do for hemophilia

2008© COPYRIGHT 16 Audience participation – what are we doing now? Our center uses same data tools for thrombotic disorders as we do for bleeding disorders? – Thrombosis – Anticoagulation clinic – Thrombophilia Our center would like to do this if ATHN made it easy to do! Our center uses same STAFF for thrombotic disorders as we do for bleeding disorders – Thrombosis – Anticoagulation clinic – Thrombophilia We have SOME overlap of these activities, but not much – Thrombosis consultation – Anticoagulation clinic – Thrombophilia We already participate in formal thrombosis data collection for other studies – HTRS – Other

2008© COPYRIGHT 17 Thrombosis Data – where shall we go from here? Phase IStructured Core Data Elements Diagnoses? If so, which? Medications? Labs? Demographics? Phase IIHTC Data Queries Phase IIIUDC? Outcomes reports? Studies? Can we facilitate the pediatric thrombosis registry in HTRS or other ongoing projects?