The National Hemophilia Foundation’s State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders

Figures & data

Table 1. Common themes of community-identified research priorities [6].

Continuation of the hemophilia treatment center (HTC) model of care

Training future health care professionals (HCP)

Ensuring financial sustainability of comprehensive care

Health care disparities between various communities (based on race, ethnicity, gender, education, income, etc.)

Access to research and mechanisms to conduct research

Access to specialized care, for all people with inherited BDs

Mental health (depression, anxiety, emotional impact, substance use disorders, etc.)

Pain management

Therapeutic advances for all inherited BDs (gene therapy, non-factor replacements, etc.)

Treatment specifically for rare and ultra-rare inherited BDs, beyond hemophilia and VWD

Women’s health and access to care

Treatment of other chronic disorders and issues affecting PWIBD (e.g. heart disease, diabetes, etc.)

Treatment of the aging population with inherited BDs

Mechanisms of joint disease and optimization of diagnosis and management

BD: bleeding disorder, HCP: health care professional, HTC: hemophilia treatment center, VWD: von Willebrand disease

Table 2. Six working groups convened to distill research questions from community priorities in specific focus areas [6].

WG1	Hemophilia A and B
WG2	Von Willebrand disease (VWD), platelet dysfunction, and other mucocutaneous inherited BDs
WG3	Ultra-rare inherited BDs
WG4	Women, girls, and people with the potential to menstruate
WG5	Diversity, equity, and inclusion; health services research; and implementation science
WG6	Facilitating priority research in the inherited BD community (infrastructure, workforce, resources and funding)

BD: bleeding disorder, WG: working group

Figure 1. Plotting feasibility, impact, and risk in the evaluation of individual research questions and initiatives Reproduced with permission from Valentino et al. (2022) [6].

Table 3. Common State of the Science Research Summit discussion themes [8].

The importance of people-centricity in research, involving LEEs at all levels of research, and evolving new inclusive research models

The essential nature of people-centric outcomes

The need to strive for diversity, inclusion, and equity in research and health care

The importance of future research across the spectrum of inherited BDs to advancing and optimizing an equitable and accessible standard of care

The need for innovative clinical trial designs including decentralization, adaptive strategies, and incorporation of real-world experience

The importance of data science and infrastructure in research and clinical practice

The need for a clinical trial network with integrated infrastructure with funding and oversight capabilities

The need for HTCs to evolve and realign to transform access to more holistic care

The need for a comprehensive and long-term approach to workforce expansion, recruitment, career growth, and retention

The importance of multidisciplinary collaboration within and beyond the inherited BD community

BD: bleeding disorder, HTC: hemophilia treatment center, LEE: lived experience expert

Table

ATHN	American Thrombosis and Hemostasis Network
BD	Bleeding disorder
HCP	Health care professional
HEDI	Health equity, diversity, and inclusion
HTC	Hemophilia treatment center
LEE	Lived experience expert
NHF	National Hemophilia Foundation
PWIBD	Person with an inherited bleeding disorder
SOSRS	State of the Science Research Summit
U.S.	United States
VWD	Von Willebrand Disease
WG	Working group

Valentino LA, Witkop ML, Santaella ME, et al. Building the blueprint: formulating a community-generated national plan for future research in inherited bleeding disorders. Haemophilia. 2022;28(5):760–768.

 PubMedGoogle Scholar

Rauch A, Valentino LA, Mills K, et al. Big picture initiatives in bleeding disorders. Haemophilia. 2022;28(Suppl 4):53–60.

 PubMedGoogle Scholar