National Bleeding Disorders Foundation

Health education and trainingnhf launched various new initiatives in 2022 to identify barriers and improve equitable access to inclusive evidence-based education about living with an inheritable Bleeding disorder. The health equity through htc pilot project engaging black and african-american patients in better understanding their experience at their htc, as well as sharing feedback and action planning with htc staff, also led to tool kits for families and for health care providers. Nhf also launched masac for you, starting with the topic of navigating emergency department visits, ensuring that masac guidelines are explained in simple plain language accessible to all to use in self-advocacy. Nhf also created new publications in english and spanish for glanzmann's thrombasthenia and factor vii (7) deficiency. Nhf also developed vwd guidelines summary sheets to help as a patient tool for self-advocacy.the primary goals of the National hemophilia Foundation's (nhf's) victory for women and better you know programs are: 1) improve the awareness of symptoms of Bleeding Disorders in women to increase diagnosis long term; and 2) to provide affected women with education and support. 2022 highlights include the launch of a new program, journey to know, where women with symptoms join a 3 month program getting more information and support on the path to diagnosis. In 2022, over 4500 women took the better you know risk assessment tool, 75% had symptoms of a Bleeding disorder, and over 5,700 visited victoryforwomen.org.steps for living is a multimedia educational program designed to increase access to age and culturally appropriate information that can help in the management of daily challenges of living with a Bleeding disorder. Nhf continues to work on the redesign and relaunch for 2024. In 2022, stepsforliving.hemophilia.org had over 87,000 sessions. Nhf's education for empowerment program brings workshops out to local chapter and htc events. Nhf facilitated 68 workshops in english and spanish, with over 1,300 participants in 2022. Hot topics 2022 were aging with a Bleeding disorder, mental health: a deeper dive, and shared decisions making and the shift to person centered care. Nhf has designed the National youth leadership institute (nyli) to assist young people from the Bleeding Disorders community to become well-trained, recognized leaders. Nhf provides young adults with training, support and opportunities to provide education to the Bleeding Disorders community. In 2022, nhf provided trainings to the 16 members of nyli on topics including public speaking, advocacy, non-profit management and fundraising. Nhf developed numerous educational components to its gene and innovative therapies programming to ensure that community members are aware of the latest treatments in the pipeline and how to have conversations with their healthcare providers for the best treatment decisions. The webpages had over 8000 views and these sessions at the Bleeding Disorders conference continue to be the best attended. Nhf produced 4 live virtual programs on novel treatment therapies that was funded through a cooperative agreement with the centers for disease control and prevention. These were offered as a collaborative learning exchange to physicians, nurse practitioners, and physician assistants on the following topics: "understanding mechanisms of action and utility of novel treatments for hemophilia a and b", "shared decision making in the age of novel technologies", managing complex cases with novel non-factor therapies", and "navigating reimbursement for patients on non-factor therapy. "at nhf's 74th Bleeding Disorders conference, the nhf offered 52 hours of live training for health care providers over 4 tracks. Continuing education credit were offered over 8 disciplines: - physicians - md, do and physician assistants (a maximum of 11.25 ama pra category 1 credits)- pharmacists (for 11.25 acpe contact hours)- nurses / nurse practitioners (up to 13.5 ancc contact hours, with an additional 3.0 contact hours related to pharmacotherapeutic content) - social workers (up to 13 aswb-approved continuing education credits)- physical therapists (up to 14 ceus, state dependent)we offered all sessions in person and 3 hours in each track virtually. We had 297 health care providers in person and 263 online for the 3 hour virtual sessions.nhf collaborated with medscape education to offer 6 live virtual grand rounds sessions on "gene therapy for patients with hemophilia." In collaboration with impact education a primer and 2 modules on "staying current: patient evaluation and treatment strategies to achieve hemostasis and improve outcomes in hemophilia a and b" were offered to all health care providers in the us. Nhf's medical and scientific advisory council (masac) issued eight (new/updated) documents in 2022- 267 - masac recommendation concerning prophylaxis for hemophilia a and b with and without inhibitors - 268 - recommendation on the use and management of emicizumab-kxwh (hemlibra) for hemophilia a with and without inhibitors- 269- standards and criteria for the care of persons with congenital Bleeding Disorders- 270 - recommendations on screening for development of hepatocellular cancer in patients with hepatitis b and c- 271 - masac recommendation on administration of inhibitor bypassing agents in the home for patients with hemophilia and inhibitors (replaced later in the year by #274)- 272 - masac recommendations concerning products licensed for the treatment of hemophilia and other Bleeding Disorders- 273 - recommendations on genotyping for persons with hemophilia- 274 - recommendation on administration of inhibitor bypassing agents in the home for patients with hemophilia and inhibitorstwo new nhf-takeda clinical fellows were selected to receive mentored-training in 2022-2024: - hilary whitworth, md, chop/university of Pennsylvania - debbie jiang, md, bloodworks nw/university of Washington *handi, nhf's information resource center, provided customized responses to over 600+ phone/e-mail requests about Bleeding or other rare Disorders. Newly launched "handi frequently asked questions" provides targeted answers to parents/caregivers, individuals living with an inheritable Bleeding disorder and healthcare providers. We awarded the 2022 kevin child scholarship to christopher ambrosio of lynbrook, ny. Handi also worked with three nhf chapters, united va, Colorado, and Nebraska, to promote handi services and support state outreach efforts.

Researchnational research blueprint: building upon the nhf state of the science research summit (sos), nhf has begun the construction of the National research blueprint (nrb). This process has engaged partners throughout the Bleeding disorder community to establish a clear understanding of the most important issues and challenges for people and families living with Bleeding Disorders. This process is patient centric, engaging the multidisciplinary research enterprise to create a National research infrastructure expansion; and reinvigorate a sustainable workforce. The global themes are people centricity, collaboration, and health equity, diversity, and inclusion. The themes focus on understanding the pathobiology of Bleeding, novel and improved diagnostics, novel and adapted therapeutics, and impediments to care. Research priorities include bone and joint health, influence of sex and gender on disease, aging, immunogenicity, mental health, pain, and research infrastructure. The sos working groups evolved in 2021 into seven working groups: research & development, infrastructure, workforce, health equity, diversity, & inclusion, lived experience experts (patients and families), community engagement, and policy. Publications: three editorials from the sos supporting the nrb and seven original articles summarizing this work were submitted for publication at the end of 2022.phase ii began in 2022 with two research workshops in the spring in Washington dc. Nhf presented the sos priorities to the over 150 in attendance with the goal of obtaining additional community feedback. These meetings also provided an opportunity to outline the next steps: defining the nrb. The goal of this phase is to (1) develop a multidisciplinary research enterprise centered in a network of both specialty and community-based care; driven by pwibds as lived experience experts (lees); embedded in the principles of social justice; and that steadily advances the standard of care for pwibds through impactful basic, translational, clinical, health outcomes, and implementation research, advocacy and education; (2) expand the National research infrastructure to support the envisioned research enterprise through facilitation collaboration and inclusion, and (3) reinvigorate a sustainable workforce across comprehensive care and scientific disciplines that incorporates the lee perspective to advance health through the seamless integration of care and research as well as active community engagement to foster a research culture. Cvr (community voices in research):a patient powered registry intended to capture a 360-degree view of living with a Bleeding disorder, directly from those affected and their immediate relatives to enhance the ability of researchers to understand the lived experience, improve quality of life (qol), identify research questions important to community members, discover transformational therapies. A redesigned registry will launch in august of 2023.virtual advisory panels (vaps): vaps are virtual focus groups. In 2022 seven vaps occurred. 50 lees participated in total of the noted vaps. Topics spanned from patient education, clinical trial design, and engagement in Bleeding disorder programs. Advanced therapeutic & medicinal products (atmp): this initiative focused on engaging stakeholders from industry and academic research to focus on novel science. In 2022, five virtual meetings occurred with 115 attendees combined. Clinical trial psychosocial template: the nhf research team and an ad hoc committee from the nhf social work working group developed a psychosocial template for clinical social work evaluation of individuals considering participation in a clinical trial. The need was identified in 2021 with the evolution of gene therapy. The tool was published on the nhf website, provided to the social work working group, and referenced at the ash conference in a panel discussion. Clinical trial toolkit: clinical trial essentials: a virtual education tool was created and launched on the nhf website in 2022 to assist the community in understanding the benefits and considerations of participation in a clinical trial. Six lived experience experts (lees) and six health care providers engaged in brief interviews. Modules include: why clinical trials important, understanding clinical trials, what to expect, understanding consent, what happens in a trial, when the trial ends, other ways to participate in research, additional resources. Gene therapy novel technology workshop planning: steering committee: two meetings occurred in 2022 regarding the potential collaborations and communication of the outcomes of the workshop. Nhf met with wfh, asgct, ash, versiti, fnih, isth, athn, cdc, arm, fda scientific planning committee: one formal meeting occurred with nhf staff and nine external experts who reviewed the latest scientific advances in preparation for an event to be held in q1 of 2023. Research journal clubs (rjc): the virtual research journal club open to the community, researchers, clinicians, and chapters, held two sessions in 2022. Medical education credits for physicians, nurses, and mid-level providers were sponsored by nhf. These events were recorded and will be accessible on the nhf website with enduring credits. - spring 2022: dynamics of hemarthrosis & vascular remodeling: long-term impact of hypertension and joint bleeds for people living with hemophilia dr. annette von drygalski 57 attendees. - fall 2022: depressive Disorders among adults with hemophilia a (dr. joanne wu & randy curtis) 27 attendees. Researchers funded through nhf research grant awards & fellowships: the nhf research department evaluated the research grant awards & fellowships program through a "deep dive" process. This explored the current grant structure, process, and areas of research support; as well as potential collaborations with other organizations to streamline efforts and enhance efficiency. This process engaged thirteen external subject matter experts across the community in three meetings and various surveys. Research funded by nhf in 2022: grant awards & fellowships: i. judith graham pool postdoctoral fellowships (jgp): - new jgp 2022: kenneth childers, structural investigation of activating factor viii and the intrinsic tenase complex by single particle cryoem, western Washington universityii. Jeanne marie lusher (jml) diversity research fellowships established in 2021, the jeanne marie lusher (jml) diversity research fellowships aims to increase diversity among benign hematologists by fostering a career-long interest in inherited bleeding/blood Disorders (ibds). Open to black, indigenous, and people of color (bipoc) who are enrolled in a doctoral program in a biomedical field relevant to inheritable bleeding/blood Disorders, the goal of the jml is to identify clinicians from under-represented communities and genders early in their career as well as those who may be in a fellowship program demonstrating interest in basic science and pre-clinical research blood Disorders with the exception of thrombophilia and establish an interest in ibds that will sustain them throughout their careers. 2022 jml award - luisanna sanchez, clinical and molecular profiles associated with robust and sustained hydroxyurea response for patients with sickle cell disease baylor college of medicine iii. Excellence fellowshipsthe excellence fellowships provide support to persons employed in the allied health disciplines with the federally funded treatment center system to expand the knowledge base specific to Bleeding Disorders. The following fellowships were awarded to a nurse, physical therapist and social worker:2022 new excellence fellowships: - k. klutz, social work fellowship, arnold palmer hospital for children's research - ramirez, physical therapy fellowship, the university of Florida- ciralo & j. wolf, nursing fellowship, western new york bloodcare Foundation

Community servicesnhf's public policy department works to advocate for policies that promote the health, safety, rights and access to care for people with inheritable blood Disorders by educating federal and state policymakers, other government agencies and officials, as well as industry and allied health education and training organizations. Two key areas of focus are payer and consumer education, and self-advocacy.the public policy department provides training, tools, and hands-on support to consumers to help them become effective self-advocates.through the state-based advocacy program, nhf awards grants to chapters, competitively, to support their advocacy efforts. Successful grant recipients are required to attend several virtual or in-person training events annually, hold regular advocacy committee meetings, include nhf staff in state advocacy events, provide a midyear progress report, collect, submit specified program metrics quarterly, host a stakeholder planning meeting and submit a final report at year's end. This program is housed in the state government relations department and is named the state based advocacy coalition (sbac) program. Grants are issued on an annual basis, with chapters applying each december. All chapters are eligible to apply for financial and programmatic support.members of the inheritable blood Disorders community throughout the country are encouraged to participate in nhf's annual Washington days program, which is a grassroots advocacy event that brings patients and their families to Washington, dc to meet with their members of congress and educate them about the Disorders impacting the community. This event takes place each year in early march. Recently, we adapted to a virtual format in the years 2020-2022. We returned to an in person event in 2023 and plan to do the same in 2024-2025. Consumers are also encouraged to attend state advocacy days at their local state capital. Nhf also provides educational opportunities for payers to help them better understand the unique healthcare needs of those affected by blood Disorders, including live presentations, webinars, online educational modules, and a joint collaborative bringing together payers, medical providers and patients. Nhf participate in about 25-30 state advocacy days per year.

Chapter services nhf's chapter services department provides community support by helping its 53 member chapters offer education, resources and referrals to affected members of the Bleeding Disorders community in the areas that each chapter serves. Chapter services offers the chapters financial support in the form of grants and provides some travel expenses support for education and advocacy meetings. Department staff members coach chapter leaders on how to create, execute and evaluate programs and services designed for their affected constituents. In 2022 chapter services offered a 16 session educational webinar series, held one National leadership seminar offering training, and offered a full "chapter training track" at nhf's annual Bleeding Disorders conference. These education offerings focused on diversification of funding strategies, health equity and inclusion, board development, and building community of volunteers and advocates.