Teaming Up to Treat People with Bleeding Disorders

The National Bleeding Disorders Foundation relies on partners in the U.S. and around the world as it works to ensure worldwide access to treatment and care.
Author: James Langford

The National Bleeding Disorders Foundation (NBDF) celebrates its 75th anniversary in 2023. Throughout the year, will be commemorating this special milestone with articles that look back at notable programs, initiatives, and events in NBDF’s history.

Changing the world isn’t the kind of job you tackle alone — even in fiction.

Superman, after all, has Lois Lane and Jimmy Olsen at his side, not to mention a virtual platoon of superpowered allies known as the Justice League.

In real life, the inventor Thomas Edison depended on the company that eventually became General Electric to produce and distribute his incandescent lightbulb in the late 1800s, and Henry Ford relied on not only his assembly lines but also the thousands of factory workers who staffed them to introduce affordable automobiles to the American public in the early 1900s.

Which illustrates why the National Bleeding Disorders Foundation — whose overarching goal is making sure everyone who has a bleeding disorder also has access to treatment and care —counts on the help of partners around the globe, including the World Federation of Hemophilia, to achieve it.

While the names of those partners have shifted over the organization’s 75-year history, active ones today also include the U.S. Centers for Disease Control and Prevention, the Coalition of the Americas, the European Haemophilia Consortium, the Medical and Scientific Advisory Council, and the American Thrombosis and Hemostasis Network.

“We want to partner with anyone we can to further access to care and treatment,” says Dawn Rotellini, chief operating officer for the National Bleeding Disorders Foundation. “We approach things so much better holistically, looking at all sides of the problem instead of only one little area.”

World Federation of Hemophilia

The advantages of that strategy are apparent in the NBDF’s work with the World Federation of Hemophilia. It’s a founding member of the organization, which was formed in 1963 and today comprises national member organizations in 147 countries cooperating to provide global treatment.

“We utilize 80% of the medicine that’s developed just in the U.S., which leaves 20% for the rest of the world, so there’s an obligation to make sure we are working closely with the World Federation to make sure others have access,” Rotellini says.

Federation members also collaborate on improving education about bleeding disorders and developing support organizations, in part through the Twinning Program, which sets up short-term partnerships between hemophilia treatment centers, patient organizations, and youth groups in developing and developed countries.

“When you approach twinning as a true partnership, it can be life-changing,” Rotellini says.

In 2021, for example, the bleeding disorders foundation and Hemophilia Federation India won an award for their work the year before on a pilot project for a youth program meant to evaluate the effectiveness of twinning with participants under 30. This year, the NBDF is twinning with Kosovo, whose bleeding disorders group was founded in 2018.

Rotellini, who is on the World Federation of Hemophilia’s board, also chairs its Women and Girls with Inherited Bleeding Disorders Committee, which works to eliminate disparities in access to treatment that date to the 19th century identification of hemophilia.

Because the disease primarily affects men, health care providers believed for decades that women were immune, and often dismissed their symptoms as unrelated. Women and girls were also excluded from clinical trials of treatments, creating a gender gap in data on effectiveness that still exists.

U.S. Centers for Disease Control and Prevention

Through its current cooperative agreement with the CDC, the latest of many, the foundation works to provide credible health information on bleeding disorders, in part by setting up evidence-based educational programs to reduce complications from hemophilia and von Willebrand disease.

In past agreements, the foundation and the CDC developed campaigns such as Better You Know, to encourage women with symptoms of bleeding disorders to seek diagnosis and treatment, as well as awareness programs focused on inhibitors that render some treatments ineffective in certain people.

One of their joint successes was establishing HANDI, a national resource for information on bleeding disorders, more than 30 years ago.

Today, HANDI continues to respond to questions submitted online, through email, and by phone. “We have providers who call us, patients who call us, the general public and other organizations,” says Neil Frick, NBDF’s senior vice president for medical programs and information. “It’s the only resource center of its kind in the world, so we get requests from anybody, anywhere.”

American Thrombosis and Hemostasis Network

The NBDF has teamed with the American Thrombosis and Hemostasis Network on initiatives including the My Life, Our Future project, which provided free genotyping to people with hemophilia from 2013 through 2017 in a partnership with Bloodworks Northwest and Bioverativ/Sanofi, formerly known as Biogen.

Genotyping — historically difficult to obtain, costly, and not covered by insurance — reveals specific genetic mutations responsible for inherited bleeding disorders, such as those found in the factor VIII and factor IX genes in hemophilia A and hemophilia B.

In addition to gaining new insight on their own health concerns, participants could opt to have a blood sample with de-identified genomic data stored with the My Life, Our Future Research Repository, which ultimately gathered 6,000 samples. That data proved particularly beneficial for researchers studying genetic variations that affect bleeding severity, who could seek access to the repository to support their work.

Medical and Scientific Advisory Council

Made up of medical professionals, government representatives, and people with bleeding disorders, the foundation’s Medical and Scientific Advisory Council was formed in 1954 to issue recommendations and advisories on treatment, research, and general health concerns.

In the decades since, the group — known as MASAC — has issued more than 400 communiques covering medical issues from prevention and treatment to infectious disease complications. The standard treatment guidelines that it sets each year are used by groups including medical schools, pharmacists, hospital emergency departments, and insurance companies.

“That gives us the strength, as a leading country, to say our experts are together, and these are their recommendations,” Rotellini says. “We can then share those recommendations with other countries and their health ministries.”

Coalition of the Americas, European Haemophilia Consortium

As part of cooperative efforts with the Coalition of the Americas, which includes hemophilia advocacy groups in North, South, and Central America, the NBDF recently hosted seven female leaders from Latin America at its Bleeding Disorders Conference.

“We were focused this year on awareness of women and girls with bleeding disorders, and we invited these female leaders to come in, meet our chapters, go to sessions, and take what they learned back to their own countries,” Rotellini says.

On the other side of the Atlantic, the NBDF works on advocacy and training programs with the European Haemophilia Consortium, which includes dozens of European nations.

“It’s crucial to develop and maintain relationships to continue driving forward education and better access to care and treatment,” Rotellini says. “Knowledge-sharing is key.”