The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary

Figures & data

Table 1. Major categories of community-prioritized research revealed by consultations [5].

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

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

Access to research and mechanisms (ways) to conduct research

Access to specialized care

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

Pain management

Treatment for all BDs (gene therapy, non-factor replacements, etc.)

Specific treatment for other RBDs (non-hemophilia, non-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 BDs

Joint disease management

BD: bleeding disorder, HCP: health care provider, HTC: hemophilia treatment center, RBD: rare bleeding disorder, VWD: von Willebrand diseaseReproduced with permission from Valentino et al. (2022) [5]

Table 2. Six working groups convened to distill research questions from community priorities in specific focus areas [5].

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 groupReproduced with permission from Valentino et al. (2022) [5]

Table

ATHN	American Thrombosis and Hemostasis Network
BD	Bleeding disorder
CDC	Centers for Disease Control and Prevention
HCP	Health care provider
HRSA	Health Resources and Services Administration
HTC	Hemophilia treatment center
LEE	Lived experience expert
NHF	National Hemophilia Foundation
PPM	Person who has or had the potential to menstruate
PWH	Person with hemophilia
PWIBD	Person with an inherited bleeding disorder
RBD	Rare bleeding disorder
SOS RS	NHF State of the Science Research Summit
US	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.

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