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

Abstract

Rare, chronic diseases such as hemophilia and other congenital coagulation disorders require coordinated delivery of services for optimal outcomes. Hemophilia Treatment Centers (HTCs) are specialized, multidisciplinary health-care centers providing team-based care to meet the physical, psychosocial, and emotional needs of people with hemophilia (PWH) and may serve as a model for other rare coagulation disorders. Health-care purchasers, as well as the general medical community, may not appreciate the breadth and quality of services provided by HTCs. They exemplify the acculturalization and actualization of integrated care by providing comprehensive diagnostic and treatment services that reduce morbidity, mortality, avoidable emergency room visits, hospitalizations, and overall costs, while promoting a longer lifespan and improved patient functioning and outcomes. This is accomplished by a team-based approach relying upon a shared decision-making model to effectively prevent complications and manage symptoms in PWH, who are dependent on high-cost treatments. This article provides a concise yet comprehensive description of the core components of an HTC and the regional and national networks in the United States, which together achieve their incomparable value for all stakeholders.

Graphical abstract

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Keywords:

• hemophilia
• coagulation disorders
• integrated care
• patient-centered care
• health-care delivery network
• multidisciplinary

Acknowledgments

The authors thank Ellen Riker and Johanna Gray for their thoughtful input on this review. Medical writing assistance was provided by Bill Kadish, MD of Parexel International and funded by the National Hemophilia Foundation.

Author Contributions

All authors made substantial contributions to conception and design, acquisition of data, or analysis and interpretation of data; took part in drafting the article or revising it critically for important intellectual content; agreed to submit to the current journal; gave final approval of the version to be published; and agree to be accountable for all aspects of the work.

Disclosure

LAV, JRB, NF, KK, JNP, and MRi declare no conflicts of interest. All grant support declared in the following statements was paid to the author’s institution. RB received grant support from Health Resources & Services Administration (HRSA) and American Thrombosis and Hemostasis Network (ATHN), and honoraria from Genentech and Pfizer for advisory panel participation and from Medscape for a webinar series. ME received institutional support for clinical research from ATHN, Novo Nordisk, Sanofi, Takeda, and uniQure; payments for educational lectures from Bayer, BioMarin, Genentech, Novo Nordisk, and Pfizer; and has participated on Advisory Boards of Bayer, CSL Behring, Genentech, Kedrion, NHF, Novo Nordisk, Pfizer, and Sanofi; all outside the submitted work. SK has been reimbursed for travel to and attendance at meetings of the Hemophilia Alliance, for which she was the unpaid board chair (2018–2020) and is a retiree from the University of California, San Francisco. SL has received a grant from HRSA and from ATHN and honoraria from the National Hemophilia Foundation (NHF) for speakers’ bureau/educational events and from uniQure for participation in an advisory board. DN is co-chair of the Steering Committee and member of the Executive Committee of CONNECTS (National Heart, Lund, and Blood Institute), president and founder of the Center for Inherited Blood Disorders, and a member of the Board of Directors of the National Association of Sickle Cell Centers and Pediatric Subspecialty Faculty at CHOC Children’s Hospital. MRe reports grants from Spark Therapeutics during the preparation of this manuscript. Outside the submitted work, he has received grants from Bayer, Grifols, LFB, and Octapharma; personal fees from Catalyst Biosciences and Kedrion; and grants and personal fees from BioMarin, CSL Behring, Genentech, Hema Biologics, Novo Nordisk, Pfizer, Sanofi, Takeda, and uniQure. In addition, he is on the board of directors for Foundation for Women and Girls with Blood Disorders and Partners in Bleeding Disorders. MTR has received grant support from Bayer and BioMarin; honoraria for being on the Speakers’ Bureau of Bayer, CSL Behring, Sanofi, and Takeda; and honoraria for participation on Advisory Boards of Bayer, CSL Behring, Novo Nordisk, Sanofi, and Takeda, all outside the submitted work; and is a member of NHF’s Medical and Scientific Advisory Council. CBT has received honoraria for virtual educational presentations from InFuCare RX and vWD Connect Foundation and sits on the NHF Pain Committee and Social Work Working Group. MS has received consulting fees to his institution from NHF. He is on the Board of Directors of the World Federation of Hemophilia USA and on the governing board of the Institute for Clinical and Economic Review. The authors report no other conflicts of interest in this work.

Additional information

Funding

This work was supported by the National Hemophilia Foundation (www.hemophilia.org) and the Hemophilia Alliance (https://hemoalliance.org/).