Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders

Figures & data

Table 1 Requirements for HTC Funding Through the Hemophilia Care Act of 1975

Core multidisciplinary team comprising:   ● Hematologist   ● Registered nurse   ● Physical therapist   ● Social worker

Extended multidisciplinary team:   ● Internist   ● Pediatrician   ● Orthopedic surgeon   ● Oral surgeon/dentist   ● Psychologist/psychiatrist   ● Genetic counselor   ● Educational/vocational rehabilitation counselor   ● Nutritionist

Facilities:   ● Specialized coagulation laboratory that satisfies defined standards   ● Blood bank that satisfies defined capability and has appropriate blood product resources

Additional:^a   ● Instruct patient/family members in administering CFC in a home setting (home visits conducted by the HTC nurse)   ● Provide a written comprehensive care plan to the patient and their primary physician   ● Establish an outreach program and encourage all PWH and health-care providers in the geographic area served by the HTC to participate in the program   ● Train professional and other personnel about hemophilia and its management   ● Commit to collecting reimbursement from third-party payers (ensuring that PWH receive product for home treatment)   ● Establish a database for reporting outcomes   ● Participate in the community through the creation of an advisory council   ● Create a patient information source

Notes: Adapted with permission from Aledort LM. The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline. Haemophilia. 2016;22(5):676–683. © 2016 John Wiley & Sons Ltd.²¹ a As HTCs continued to evolve, additional services were provided, including an infusion certification program, vocational guidance, and family planning.

Abbreviations: CFC, clotting factor concentrate; HTC, hemophilia treatment center; PWH, person/people with hemophilia.

Figure 1 MASAC Standards and Criteria for the Care of Persons with Congenital Bleeding Disorders. Data from Reding and Kenney.²¹

Figure 2 Proliferation of Knowledge Generation in Hemophilia and Other Coagulation Disorders.

Notes: EndNote™ version 20.1 (Clarivate, London, UK) was used to search the literature available in PubMed^® National Library of Medicine database and retrieve a list of references by combining the individual year of publication (2010–2020) with each of the following Medical Subject Heading (MeSH) search terms: “hemophilia”, “von Willebrand Disease”, platelet disorders”, or “other coagulation disorders”. The number of citations for each MeSH term and year were recorded in an Excel spreadsheet (Microsoft 365, Redmond, Washington).

Table 2 Comparison of the Universal Data Collection and the Community Counts Surveillance Systems

System Details	Universal Data Collection System	Community Counts System
Time frame	1998 through September 2011	October 2011 to present (however, the full data collection system was not implemented until 2013)
Primary focus	● Track blood safety through HIV and viral hepatitis monitoring ● Track hemophilia complications with a focus on infections and joint disease	● Continued tracking of bloodborne infections and joint disease ● In-depth tracking of other hemophilia complications and additional indices relevant to the aging hemophilia population
Data source	Federally funded HTCs	Federally funded HTCs
Funding	Cooperative agreement awarded to 12 HTC regional centers	Cooperative agreement awarded to ATHN to serve as Community Counts coordinating center; ATHN administers subcontracts with HTC regional centers, which then administer contracts to individual HTCs
Partnerships	● USHTCN: includes regional medical directors, regional administrators, and health-care providers at individual HTCs ● Multidisciplinary committees with representation from CDC and USHTCN established to advise on all aspects of UDC implementation	● USHTCN ● ATHN ● Multidisciplinary committees with representation from CDC, USHTCN, and ATHN established to advise on all aspects of Community Counts implementation
System components	● Patient registry: clinical data from medical records and direct patient inquiry ● Laboratory specimens ● Mortality reporting	● Patient registry: clinical data from medical records and direct patient inquiry ● Laboratory specimens ● Mortality reporting ● HTC population profile: minimal data collection about the entire hemophilia population served by HTCs
Consent for registry participation	● UDC designated by CDC as research ● Participants (or parents of minor children) asked to provide informed consent	● Community Counts designated by CDC as non-research public health surveillance not requiring consent ● Participants still asked to provide written authorization for participation in the registry
Patient registry clinical data forms	● Initial visit form: historic and current clinical data ● Subsequent annual visit forms: clinical information since the last registry submission	● Initial visit form: historic and current clinical data ● Subsequent annual visit forms: clinical information since the last registry submission
Patient registry: types of data reported on clinical forms	● Demographics ● Weight and height ● Family history ● History of HIV, hepatitis C, and liver disease ● Bleeding disorder diagnoses ● Treatment regimen and products ● Bleeding episodes ● Mobility restrictions and joint procedures ● HTC laboratory results, including levels of circulating clotting factor and inhibitor titers ● Joint range of motion ● HIV risk-reduction measures ● Optional supplemental quality of life questionnaire (since 2005)	● Demographics ● Weight and height ● Family history ● History of HIV, hepatitis C, and liver disease ● Bleeding disorder diagnoses ● Treatment regimen and products ● Bleeding episodes: more extensive information than UDC ● Mobility restrictions and joint procedures ● HTC laboratory results, including levels of circulating clotting factor and inhibitor titers ● Additional information on patient inhibitors to treatment  products ● Chronic pain ● Opioid use for chronic pain ● Health-care use ● Comorbid medical conditions (eg, cancer, cardiovascular disease)
Patient registry data submission	● Primary submission source throughout UDC: paper forms submitted through US postal system, although some HTCs developed and submitted electronic forms via secure FTP ● Forms entered and transferred to electronic database and reviewed; HTCs asked to resolve data discrepancies and provide missing data ● HTCs encouraged to submit data continuously rather than submitting in batches	● Before 2015: paper forms submitted through US postal system  and entered into an electronic database ● Since 2015: data submitted electronically via an online data  capture system developed and maintained by ATHN ● Review of forms submitted on paper and electronically; HTCs  asked to resolve data discrepancies and provide missing data ● Some data checks integrated into ATHN system (ie, to occur in  real time) ● HTCs encouraged to submit data continuously rather than in batches
Laboratory specimens and tests	● Serum specimens  ∘Hepatitis A, B, and C  ∘HIV ● Plasma specimens (for select years)  ∘Hepatitis C RNA	● Serum specimens  ∘Hepatitis C  ∘HIV ● Plasma specimens  ∘Inhibitors to treatment products
Laboratory specimen shipping timeline	● Centrifuged and shipped to CDC on cold packs within 30 hours of blood draw	● Centrifuged and shipped to CDC on cold packs within 72 hours of blood draw or frozen and shipped on dry ice within 30 days of blood draw
Laboratory accreditation	● CLIA-certified laboratory ● Test use FDA-approved kits or CLIA-approved in-house developed tests	● CLIA-certified laboratory ● Test use FDA-approved kits or CLIA-approved in-house developed tests
Biobank	Both serum and plasma specimens stored long term (with participant permission)	Both serum and plasma specimens stored long term (with participant permission)
Publication of surveillance data	● Publication of key findings in MMWR or peer-reviewed journals ● Periodic comprehensive surveillance reports published on CDC website	● Publication of key findings in MMWR or peer-reviewed journals ● Periodic comprehensive surveillance reports published on CDC website ● Data visualization tool

Notes: Reproduced from Schieve L, Byams V, Dupervil B, et al. Evaluation of CDC’s Hemophilia Surveillance Program — universal data collection (1998–2011) and community counts (2011–2019), United States. MMWR Surveill Summ. 2020;69(5):1–18.⁴⁵Abbreviations: ATHN, American Thrombosis and Hemostasis Network; CDC, Centers for Disease Control and Prevention; CLIA, Clinical Laboratory Improvement Amendments; FDA, [US] Food and Drug Administration; FTP, file transfer protocol; HIV, human immunodeficiency virus; HTC, Hemophilia Treatment Center; MMWR, Morbidity and Mortality Weekly Report; UDC, Universal Data Collection; USHTCN, US HTC Network.

Figure 3 Benefits of Care at an HTC are Associated with Lower Total Cost of Care. Data from National Hemophilia Foundation.⁶

Abbreviation: ED, emergency department.

Table 3 Final HTC- and Payer-Reported Metrics for Use in Pilot Programs

Payer-Reported Metrics	HTC-Reported Metrics
Cost of factor ● Total factor cost ● Total factor cost per patient ● Site of care  ∘Facility (hospital/ED)  ∘Ambulatory (infusion center, physician’s office, HTC)  ∘Home/self	Patient classification ● Diagnosis (A or B)  ∘Severity (mild, moderate, or severe)  ∘Inhibitor status (yes or no)
Prescribed dose/dispensed dose/weight (± range) when dispensed via a specialty pharmacy ● Product ● Total units ● U/kg ● Units dispensed ● Prescribed dose/dispensed dose  ∘±10% according to MASAC guidelines; payers desire ±5%	Prescribed dose/dispensed dose/weight (± range) when dispensed at an integrated HTC pharmacy ● Product ● Total units ● U/kg ● Units dispensed ● Prescribed dose/dispensed dose  ∘±10% according to MASAC guidelines; payers desire ±5%
ED visits/hospitalizations ● With hemophilia listed as first/second diagnosis code (ie, in first 2  lines of claim)	ED visits/hospitalizations ● With hemophilia listed as first/second diagnosis code (ie, in first 2 lines of  claim)
Total cost per patient ● Total cost of pharmacy claims ● All other medical claims costs ● Total cost per patient	Home infusion ● PWH/families independently infusing at home (%) ● PWH/families infusing at home with nursing assistance (%)
	Patient contacts (eg, in person, telephone, e-mail) ● Comprehensive care visits ● Other visits ● Case management contacts ● Collaboration with other providers

Notes: Reproduced from Tarantino MD, Pindolia VK. Hemophilia management via data collection and reporting: initial findings from the Comprehensive Care Sustainability Collaborative. J Manag Care Spec Pharm. 2017;23(1):51–56. Available from: https://www.jmcp.org/doi/10.18553/jmcp.2017.23.1.51.⁵⁹Abbreviations: ED, emergency department; HTC, Hemophilia Treatment Center; MASAC, Medical and Scientific Advisory Council; PWH, person/people with hemophilia.

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