MASAC Minutes

March 7, 2025

Washington, DC

 

1.           Call to Order and Chair Report            Dr. Dunn

Dr. Dunn called the meeting to order at 8:11 am.

She started with the history of MASAC and its contributions. She welcomed Sarah Dunn, PT Working Group chair, as a new member. Dr. Dunn offered tribute to Dr. Marion Koerper, a former MASAC member, who recently passed away.

Dr. Dunn shared progress on the priorities developed in 2022. She said the gene therapy readiness goals were completed in March, liver health in gene therapy was completed, contraception guidance was completed, and the vaccine update was completed. For inhibitors, she said the revision of bypassing agent guidance was completed, there was an addition of TFPI rebalancing agents for review, and that HARP was actively enrolling. For joint health and pain, she said a physical therapy document was completed, a document regarding physical therapy management of bone health was up for a vote, and the geriatrics working group’s bone health document was being reviewed.

For women’s health, she noted Dr. Baldwin was continuing to advocate, an iron deficiency document was up for review, a substance use disorder document was completed, a mental health document was completed, and multiple geriatric working group documents were being reviewed. For the HEDI category, she said the team was reviewing every MASAC document and every MASAC for You document from NBDF’s Education Department. She stated that access to the best care for all remained integral to the mission, and it was only through collaboration and innovation that MASAC would achieve goals.

New topics from Dr. Dunn to be discussed included a MASAC conflicts of interest policy and form, a MASAC document template, and a policy for retiring old MASAC documents.

Dr. Dunn announced a MASAC document reboot. She said there were 291 active documents and that many were outdated with no authorship attributions, no revision timelines, and no protocol to retire guidelines. She said the new goal was to have a cohesive document format for all to use, list authorship attribution, align with WFH Living Guidelines model, and have a revision plan. She envisioned the documents to be presented like chapters in a book listed by categories.

After a vote for priority setting for 2025 moving forward, the results showed the priorities to be rebalancing agents, reviewing of existing documents, platelet disorders, laboratory monitoring, and engaging women. 

Dr. Dunn said the Research Roundtable had a goal to ensure NBDF was in open dialogue with FDA, regulatory groups, and pharma in the bleeding disorders space to ensure alignment and collaboration on topics of interest to all. She said the idea was modeled after the Epilepsy Foundation research roundtable. The first meeting was scheduled to be October 15 and 16, 2025, with a proposed topic of women and girls in clinical trials. 

Dr. Dunn proposed a one-day meeting, perhaps tied to the March 2026 MASAC meeting, to allow pharmaceutical companies to provide updates on products, devices, and clinical trials in the pipeline.

Dr. Dunn ended by saying the path to success would be to keep the health of the families that were served as our guiding star, teamwork, and collaboration.

 

2.           NBDF Update  Mr. Gattone

During his report to MASAC, Mr. Gattone shared the mission of NBDF, and he shared that NBDF remained a four-star charity on Charity Navigator. He said it was a testament to prudent management of the generous resources that had been entrusted to NBDF. He said he took pride in upholding this standard moving forward. 

Mr. Gattone discussed the new Neil Frick Resource Center which was formerly known as HANDI. He said it offered comprehensive information and resources for individuals with inheritable bleeding and blood disorders. He said it would continue to connect the community and provide vital information about treatment centers, support groups, and more.

For Pathway to Cures, he said that the portfolio included a Spark wellness product launch, Be Biopharm was taking steps to prepare for IPO in 2026, and Seawolf completed a successful series-A extension. Strategic updates included working with the scientific advisory group to focus efforts with a current emphasis on diagnostics including prenatal screening and indications not yet addressed in the portfolio, bringing venture attention and expert feedback to companies working for the benefit of the community, and supporting NBDF’s Research Round Table. To mitigate legal and financial risk, Mr. Gattone said they took legal advice to ensure the best business practices without any violation of laws. He added that poor stock market performance was stifling investments, and loss of government funding to develop new treatments was impacting companies. He said they were working to find new ways to facilitate investment in the portfolio.

In the research area, Mr. Gattone said the 2024 accomplishments included the NRB summit and the Community Voices in Research project. The latter had data migrated, increased enrollment by 25%, and launched a baseline study. He said the 2025 goals were NRB manuscripts, development of the Research Ambassador Program, inaugural research roundtables, and Community Voices in Research (CVR). He said CVR was launching Research Connect, an administration portal, and surveys.

The Research Roundtable agenda was planned as a two-day meeting October 15 and 16, 2025. The schedule would have a plenary, about five sessions, participant reflections, and a conclusion that would include next steps. Topics being considered were historical exclusion, biological differences, inadequate inclusion criteria, safety concerns, pregnancy/breastfeeding, bias in trial design, cultural and social barriers, recruitment challenges, funding, and data disaggregation. 

Mr. Gattone said shared revenue building included collaboration with chapters. He said he wanted to expand individual giving and strengthen donor cultivation strategies to increase contributions, enhance donor stewardship, and implement best practices. He hoped to retain and upgrade donors, provide scalable fundraising tools to support chapters with fundraising technology and training, and align national and local fundraising efforts to create synergies that benefit all. He said expected impacts were working together, increasing individual giving revenue, increasing donor engagement with national foundation and local chapter, higher donor retention rates, increased major gift conversions, greater alignment between national and local fundraising, and stronger financial stability. He said this would expand NBDF’s mission’s impact.

For Community Matters, he stressed chapters were at the heart of NBDF. He said that by working together, they could build capacity for chapters and the national office. He believed they could cultivate, solicitate, and steward new major donors.

 

3.           New Conflicts of Interest Policy and Forms  Dr. Dunn

Dr. Dunn noted that in the past there was not a specific policy related to conflicts of interest for MASAC and that the COI form had not been reviewed in quite a while. Her goal was to also put conflicts of interest information on the NBDF website. She referred MASAC members to the draft that was presented to them. A question arose about the maximum of $25,000 that was chosen for financial interests or gifts from entities producing products or services for bleeding disorders. A MASAC member shared $50,000 was currently more standard at their institutions. Others suggested the $25,000 was already too high. It was clarified that this amount was per year. Dr. Dunn added that research funding would also be disclosed, but this category was referring to advisory boards, speaker bureaus, and stock ownership. Dr. Dunn asked everyone to share in email the amount their individual institutions used to find the number to be used.

Dr. Dunn went on to note the usual paper conflicts of interest forms would be modified to reflect this policy and be made available to use electronically before every meeting. Those submitted forms were to be reviewed by Dr. Recht before each meeting.

 

4.           MASAC Goal Setting and Priorities    Dr. Dunn

After sharing the results for priority setting for 2025 in her chair report, Dr. Dunn asked for comments and discussion. They were rebalancing agents, reviewing of existing documents, platelet disorders, laboratory monitoring, and engaging women. After discussion, rebalancing agents and laboratory monitoring were combined, and then funding HTCs and research moved up to number five. Dr. Dunn planned to proceed with these priorities.

 

5.           MASAC Document Template Dr. Dunn

Dr. Dunn asked Mr. Skinner to share the new document template. 

Mr. Skinner shared that as the committee working on the geriatric guidelines looked to how the documents might be presented or published, they wanted a degree of formality with all questions covered, clear recommendations, and all the authors listed. They also wanted to list future research needs and references. He said they pictured a format like a book with PDF chapters.

 

6.           Pain Evaluation Template       Ms. Dunn

Ms. Dunn shared that the PT pain taskforce created a pain evaluation template that was based on MASAC Document #283 to provide more detailed guidance for practitioners. The goal was an easy-to-use form. She said the group asked her to share the document with MASAC and ask for any feedback as they continued to work on it. She said the goal would be to have it as an appendix to the current document.

Ms. Bender suggested adding factor as a treatment on the form. Dr. Kouides suggested adding an oral pain treatment option.

 

7.           Health Equity, Diversity, and Inclusion (HEDI) Update           Dr. Norris

Dr. Norris presented via video recording. She said that in moving forward with their plan, the department was being renamed Health Access & Strategic Innovation (HASI), but they would still be doing health equity, diversity, and inclusion work every day. The reason for the rebrand was a forward-looking strategy with the innovative work of the HEDI team’s strategic plan, priority populations, and framework manuscript. The new framing for equity was to be health access, the new framing for diversity was strategic, and the new framing for inclusion was innovation. She stressed nothing about the work they were doing would change.

She said health access was still the overall goal, and strategy and innovation would be in everything they did. She said health access included increasing access to care for all, empowerment and advocacy, cultural humility, and addressing drivers of health. Moving forward, she said they would continue to prioritize equitable health access and drive innovative solutions for all communities. The decision was made strategically to protect the long-term impact white ensuring alignment with current realities.

Dr. Norris said the pivot helped them stay in the conversation and continue advocating effectively rather than being sidelined by political roadblocks and that their efforts were more than race, gender identity, or ethnicity. 

Dr. Norris said she would still be the HASI contact for MASAC, and she asked MASAC to continue to support HASI work whenever popular.

After the video presentation, Dr. Dunn stressed that changing the words would not affect the work everyone was doing. Dr. Recht added that this was not a defensive decision and that it was a proactive decision to enlarge the umbrella, and NBDF was committed to serving all.

 

8.  Geriatrics Task Force            Dr. Kouides, Mr. Skinner

Dr. Kouides reminded everyone it had been a year since this initiative was started. He was happy to point out that a quarter of the members were Lived Experience Experts (LEEs). He said this effort had proven the necessity while moving forward to include a fair proportion of LEEs on MASAC. He said this effort confirmed the need to have independent specialty expert reviewers such as cardiologists and urologists as well as the collaboration of other MASAC committees and working groups. He said the group was made up of MASAC members and professionals outside of MASAC. He said the subsections included bone health; cardiovascular (atrial fibrillation, CV interventions, prevention of CVD); psychosocial; gender issues (prostate health, women’s health); orthopedic; and others such as audiology, dermatology, and ophthalmic.

He said the documents to address at this meeting were bone health, CV interventions, presentation of CVD, psychosocial, and ophthalmic.

 

Bone Health Document             Dr. Kouides

Dr. Kouides said this section’s members were Brandon Hardesty and Divya Citla Sridhar. They focused on an algorithm on vitamin D screening and replacement and developed recommendations for osteoporosis screening with DEXA scan and management. The document noted multiple studies demonstrated that hemophilia was independently associated with reduced bone mineral density (BMD) and results in an increased risk of fracture.

Dr. Kouides said the first recommendation was that MASAC agreed with guidelines recognizing hemophilia as a cause of secondary osteoporosis. Endocrine Society clinical practice guidelines recommend that DXA screening should begin at age 50 for those at increased risk due to a secondary cause of osteoporosis. The second recommendation said MASAC recommended anyone with a low trauma fracture, osteopenia or osteoporosis by BMD on DXA, or FRAX-projected 10-year risk of ≥ 3% for hip fracture and/or ≥ 20% for major osteoporotic fracture should be referred to a bone health clinic for consideration of treatment. Recommendation three said MASAC recommended prevention strategies to preserve BMD should be encouraged. Recommendation four said MASAC recommended that all people with hemophilia or von Willebrand disease over age 2 should be screened for vitamin D deficiency and treated according to the algorithm or a similar institutional protocol.

For future research needs, Dr. Kouides mentioned that optimal treatment of osteoporosis in hemophilia was not known, the optimal level of 25(OH)D to reduce the risk of osteoporosis was not known, optimal timing of DXA screening in hemophilia was not known, and the precise fracture risk for an individual with a bleeding disorder was unknown. Additional research to understand the risk factors of spinal stenosis was needed.

After discussion, Dr. Dunn proposed an email vote with the modifications mentioned.

 

Psychosocial Document    Ms. Schnur

The section members listed were Kathaleen Schnur and Ellen Kachalsky. The topics the document addressed were psychosocial assessment and guidance of aging persons with bleeding disorders, complex and challenging medical decision-making, health insurance, cognitive impairment, dementia, delirium, depression, access to skilled facilities, social determinants, elder abuse/elder fraud, isolation, caregiver fatigue, grief, and aging of the mind and body. Ms. Schnur discussed each of the topics and detailed the recommendations for each topic in the document. 

For future research needs she included investigating the experience of aging individuals with hemophilia and other bleeding disorders in accessing healthcare, particularly in the context of managing complex health issues which could offer insights into barriers or inequities; research could examine how bleeding disorders affect quality of life in older individuals. That included assessing the impact of physical disabilities, ongoing treatment regimens, and the emotional toll of managing a lifelong condition; and developing hemophilia and other bleeding disorders-specific measures of frailty.

Ms. Schnur also shared the appendix that included tools to go with the document. 

MASAC approved Document #292, “Psychosocial Aspects of Care,” pending HASI review.

 

Cardiovascular 

The section members listed were Nathan Connell, Miguel Escobar, Erin Espinoza, Brandon Hardesty, Ming Lim, Jonathan Roberts, Mamie Thant, and Mary Anne Williams. Dr. Kouides said two documents were up to be discussed during the meeting that covered primary prevention of CVD and managing CV interventions.

 

Cardiovascular Prevention Document     Dr. Roberts

The experts listed in this document were Brandon Hardesty, Mamie Thant, and Erin Espinoza. Dr. Roberts presented the first cardiovascular health document where the topic was primary prevention of cardiovascular disease. The background included that heart disease was the leading cause of death in the United States. The document said advances in the care of people with hemophilia led to an aging population that was developing age-related cardiovascular disease, in particular atherosclerotic cardiovascular disease. 

Dr. Roberts discussed the seven recommendations listed in the document. 

Future research needs included the absence of cardiovascular risk calculators validated in people with hemophilia, new factor replacement products' impact on the incidence of cardiovascular events in people with hemophilia should be prospectively evaluated as they are released, and the incidence of cardiovascular events in people with hemophilia who undergo gene therapy should also be prospectively examined.

MASAC approved document #293 after discussing changes that should be made.

 

Cardiovascular Intervention Document  Dr. Roberts

The experts listed were Jonathan Roberts, Nathan Connell, Erin Espinoza, Brandon Hardesty, Mamie Thant, and Mary Anne Williams. Dr. Roberts discussed this document’s topic of cardiac interventions for cardiovascular disease. For the background, he shared that hemophilia had been commonly thought to protect against thrombotic risk in underlying cardiovascular disease, but a growing number of studies demonstrated a significant risk of CVD in persons with hemophilia that increases with age. He went on to discuss each of the recommendations in the document and how the group reached them. 

Future research needs included optimal treatment of CAD with cardiovascular interventions that remained unknown in hemophilia and required further investigation; optimal hemophilia prophylaxis with factor concentrates, factor mimetic, and rebalancing agents during anticoagulation and/or antiplatelet therapy required further study; and further investigations were necessary to optimize PCI, CABG, HVR, CPB and ECMO management in hemophilia.

After discussion, MASAC approved document #294.

 

Ophthalmic Interventions              Ming Lim

The expert listed for this document was Ming Lim. Dr. Lim could not attend the meeting so Dr. Kouides presented the document.

The first topic was cataracts. The background noted they were a leading cause of visual impairment and that the prevalence of cataracts increased with age with a prevalence as high as 90% among those 80 years and older. Dr. Kouides shared the recommendations listed under that topic.

The second topic was age-related macular degeneration, and the background showed it was the most common cause of irreversible visual loss in the elderly.

Glaucoma was the third topic, and the background showed advancing age was a major risk factor for the development of glaucoma, a leading cause of irreversible blindness worldwide. Dr. Kouides shared the recommendations listed in the document for each topic.

The future research section shared that the optimal management of cataract surgery was based on small case studies and anecdotal experience. Larger case studies on peri-operative management of cataract surgery, especially among people with mild hemophilia.

With the suggestions based on the discussion being included, MASAC approved document #295.

 

Upcoming

Dr. Kouides said an atrial fibrillation document should be ready for MASAC review for the June 2025 conference call. Gender-specific documentation should be ready for MASAC review at the August meeting that would include breast health with section members Magdalena Lewandowska and Janice Staber. Their goal was to develop guidance for procedures that could cause bruising (mammogram, Breast MRI) and bruising and/or bleeding, injection for sentinel lymph node, sentinel lymph node biopsy, lumpectomy, mastectomy, and axillary dissection. That would also address prostate health with section members Tamara Bullock, Mark Skinner, and Joe Stanco. It would discuss developing guidance for procedures such as BPH: Transurethral Resection of Prostate (TURP) vs. less invasive Prostate Artery Embolization (PEA) vs. Laser-Vaporization and prostate cancer (prostate Biopsy, transrectal versus perineal); and radical vs. Simple Prostatectomy vs. Radiotherapy with spacer to reduce proctitis.

Dr. Kouides also said an orthopedic section should be ready for the August MASAC meeting for review from section members Anne Griest, Magdalena Lewandoska, and Tyler Buckner with MASAC orthopedist Dr. Anthony Ungar to assist. He said detailed documents already developed for perioperative recommendations and thromboprophylaxis were pending review. A section on pain management was pending.

 

9.           Iron Deficiency Screening        Dr. Batsuli

Dr. Batsuli said the working group members were Glaivy Batsuli, Michelle Sholzberg, Magdalena Lewandowska, Ming Lim, Kelly Tickle, Jennifer Davila, Peter Kouides, Shilpa Nataraj, Shveta Gupta, Amanda Jacobsen-Kelly, Brendan Hayes, Jennifer Girard, Maureen Baldwin, Rose Bender, Michele Lambert, Roona Sinha. She said they represented pediatric hematology, adult hematology, and women’s health.

She said the group moved forward with the rationale that iron deficiency with and without anemia was a recognized complication of inherited bleeding disorders, but there was a lack of recommendations or guidance on screening in this population. Their main objective was to generate recommendations regarding screening for iron deficiency with and without anemia in individuals with inherited bleeding disorders.

The working group progress was to include cross-organizational support with ASH, FWGBD, EAHAD, and EHC. She added that none of the pending guidelines (AAP & ASH) would specifically address the inherited bleeding disorders population. Dr. Batsuli said she would share an iron deficiency screening draft, and she would appreciate feedback.

Dr. Batsuli said future activities would include a call to action on iron deficiency with or without anemia research in inherited bleeding disorders. Research ideas included an ATHN project to pull anemia data on all inherited bleeding disorders patients and an ASPHO abstract from 2022 on iron deficiency screening and prevalence in women with bleeding disorders. They hoped to perform an assessment of ferritin reference ranges across different U.S. labs and hospital systems and issue a survey querying HTCs on their practices for iron deficiency screening.

Dr. Batsuli shared the draft document. The first topic was the impact of iron deficiency. The background information showed that an estimated 24% of the world’s population, or 1.92 billion individuals, had anemia. Iron deficiency anemia was the most common cause of anemia and was associated with significant morbidity and mortality in high-risk populations. She shared a table of signs and symptoms and recommendations from the working group. Dr. Batsuli shared topic two was the frequency of laboratory screening for iron deficiency with or without anemia and the recommendations under that topic. 

The third topic regarded recommended tests for laboratory screening of iron deficiency with or without anemia. She said laboratory screening should consider that signs and symptoms of iron deficiency could occur with or without anemia. The fourth topic discussed diagnostic considerations for laboratory screening because although iron deficiency anemia was a frequent cause of anemia, it was not the only cause of anemia.

Management of iron deficiency with or without anemia was the fifth topic. A suggestion was made to add a recommendation that efforts should be made to minimize bleeding until iron repletion could be obtained.

 

10.        Genetic Testing             Dr. Singleton

Dr. Singleton shared updates for the MASAC guidance for Genetic Testing for Rare Bleeding Disorders. She said the working group members were Tammuella Chrisentery-Singleton, Amy Shapiro, Diane Nugent, Glaivy Batsuli, Sweta Gupta, Lori Luchtman-Jones, Suchitra Acharya, Shirley Williams, Osman Khan, Kristin Zajo, Rohith Jesudas, and Samantha L Cassisi.

She said their charge was to establish guidance for MASAC regarding genetic testing for rare bleeding disorders. They started by looking at how they could provide guidance to better understand the biology of non-hemophilia disorders and improve the ability to diagnose these disorders using genetic testing.

She said as a committee, they identified the priorities as confirming or refining the diagnosis, predicting severity, determining risk, identifying genetic carriers, helping with reproductive counseling and birth planning, all to lead to improved, individualized treatments.

Dr. Singleton said the goals included making recommendations that were supported strongly with evidence that would stand the test of time and provide conservative recommendations that stand on very firm footing. She said they could be aspirational, but they needed references to support the claims. She said they would need literature for almost every sentence. Ultimately, she said they wanted to have recommendations on genetic testing and a call to action.

Dr. Singleton said the first phase they developed included practice recommendations for RCDs, the second was practice recommendations for congenital platelet disorders, and the third was practice recommendations for VWD.

The first topic for the draft document was genetic testing recommendations for persons with rare coagulation disorders along with background facts shared with MASAC. 

For future research needs, she said more efforts were needed to characterize genotype-phenotype relationships in non-hemophilia rare coagulation disorders in natural history data.

MASAC members offered suggestions for additions to the document for the next draft. Dr. Dunn suggested the next draft be shared by email.

 

11.        ATHN Update  Dr. Singleton

Dr. Singleton shared ATHN updates starting with the partners. She said the ATHN/NBDF Data Commons – Better Together was the first collaborative project. The goal was to work together to provide US data for the WFH Annual Global Survey. For the ATHN – WFH Gene Therapy Data Collaboration, they were pleased to have established a collaboration for the WFH Gene Therapy Registry. 

ATHN 10 (Leveraging the ATHNdataset to Document the State of Rare Coagulation Disorders in the United States) was initiated in 2020 as a collaboration between ATHN, CIBD, and IHTC. The genetic testing was courtesy of CIBD, and it exceeded the initial target of 1,300 specimens. She said there were 83 participating ATHN affiliates. 1225 individuals with an established ultra-rare bleeding disorder had been sequenced for their factor deficiency, 20,720 genes for analysis, and 176 novel variants had been identified. She added that ATHN was issuing contract modifications to extend the project through 2025, and CIBD was working to provide outstanding results.

Continuing, Dr. Singleton said the ATHN EMR integration project was designed to support ATHN affiliates with both limited and extensive access to their institution’s EMR IT team. She said if teams could create a CSV or Excel report from their EMR that matches the ATHN template, they could import it into ATHN systems. She said three ATHN affiliates were sending data regularly, 5 ATHN affiliates were working through the authorization process with their institutions, and 25 ATHN affiliates expressed interest since the ATHN Data Summit.

For EMR Integration and data availability, she said the available elements were demographics, insurance, vital signs, and visits. Elements under development included treatments. For 2025 they planned to add diagnoses and laboratory results.

 

12.        NBDF Education Department Ms. Hayes; Ms. Scappe-Hagen

Some of the current NYLI members introduced themselves to MASAC. They were to graduate in June 2025.

The Education Department team shared that in 2024, 30,377 education publications were distributed, up from 12,000 publications in 2023. They had 269,088 consumer web views, and 2034 people attended live workshops, events, and webinars.

They added goals that included improving equitable access to inclusive evidence-based education about living with inheritable blood disorders through outreach. They wanted to increase awareness and knowledge of the safety and effectiveness of treatment options, mechanisms of action, care best practices, and developing skills to navigate them. Another goal was to Increase knowledge of physical and psychosocial complications of living with an inherited blood disorder and develop skills to improve quality of life.

The team shared an Education for Empowerment Workshops at Chapters recap for 2024. They said 89 workshops were facilitated in-person serving 33 chapters. There were over 1,200 total attendees. 86% reported learning a new skill or idea, and 84% felt prepared to implement the new skill or idea.

Another goal was to Improve the awareness of symptoms of blood disorders in women, girls and people who have the potential to menstruate and steps to navigate diagnosis to increase diagnosis long term.

They hoped to encourage an Increase in sustainability of leadership of the blood disorders community through young adult engagement.

 

13.        Hemophilia Alliance   Mr. Blake

Mr. Blake said the Hemophilia Alliance worked to ensure member hemophilia treatment centers had the expertise, resources, and public support to sustain their integrated clinical and pharmacy services for individuals with bleeding and clotting disorders.

For 2024 key results, he said it was a year of transition with changes in staff. He said that as a group purchasing organization, members saved over $130,000,000 through subceiling pricing, rebates, and prompt pay discounts. They conducted a rollout of genetics testing program for FVIII, FIX, and VWD. Their Hemophilia Alliance Network Services procured gene therapy contracts with payers, additional units back to HTC pharmacies. For administration and operations, he said they built a new website, CE Programs, and enhanced meeting experiences. For advocacy, he said they worked with 340B legislation, PBM reform, and access.

Mr. Blake said 2025 initiatives were about being a year of transformation with organizational initiatives, their group purchasing organization, member and community relations, administration and operations, advocacy, and legal/regulatory activities.

Mr. Blake mentioned the Hemophilia Alliance member meetings and invited everyone to register through the member portal hemoalliance.org. He said the spring and fall members meetings would include updates, hot topics, and sharing of best practices. He said the attendance was not capped and travel was funded by the home HTC

For continuing education, he discussed discipline-specific planning committees for pharmacists, nurses, physical therapists, and social workers. That attendance would be through a lottery with travel funded by the Alliance and manufacturing partners.

Mr. Blake said there would be a medical provider/business administer meeting. It would be a retreat and working meeting for HTC leaders to discuss strategic priorities. He said attendance priority would be given to teams that attend together. Travel would be funded by the Alliance.

For the Hemophilia Alliance genetic testing program, he reiterated there was testing for hemophilia A, B and VWD that was available to member HTCs for patients who needed testing that insurance did not cover, or the out-of-pocket expenses were high. They were using Versiti and Bloodworks labs.

For community support, he said in 2024 more than $3,000,000 was provided, and they budgeted for $4,000,000 in 2025.

Mr. Blake said the Partners Physician Academy’s program goals were to support fellows and early career physicians working or interested in working within the U.S. federally recognized HTC network​. The goal was to provide supplemental training to address hemostasis training gaps​ and to facilitate networking and community building. He said optimal candidates should be second- to fourth-year fellows or early career physicians. He said they were currently seeing patients in the field of bleeding disorders who were working or intending to work as part of the federally recognized U.S. HTC Network.

For the 2025 Partners Physician Academy, Mr. Blake said the date would be September 15-19, 2025, in Carmel, Indiana. Applications were open February 24 through April 11, 2025. Applicants were to be evaluated by the PPA Advisory Board, who would select the top 20 candidates for admission to the 2025 Academy.

Mr. Blake said that beyond the Partners Physician Academy, there were monthly enrichment webinars available to all Academy alumni covering clinical management, communication, and career development.

 

14.        Emergency Cards and EPI and SubQ Educational Documents              Dr. Tickle

Dr. Tickle shared the annual initiatives during 2024- 2025 from the Nursing Working Group. She said they issued primary care provider educational forms regarding epistaxis and subcutaneous/IM Injections. These new documents were available on the NBDF website, and she hoped everyone would share them. Dr. Dunn asked if the forms would be translated to Spanish, and Ms. Hayes said that was the plan.

Dr. Tickle also said they also developed emergency treatment cards at the request of MASAC to be issued at HTCs. The cards were designed for patients to carry in their wallets and covered VWD, hemophilia A, hemophilia B, and general bleeding disorders. She said the cards contained information about any special considerations that might be needed in an emergency or if there was severe life-threatening bleeding. Contact information for the patient’s HTC was also included. Ms. Bender suggested the emergency action information be moved to the front of the cards and the base line information be moved to the back. Dr. Dunn noted these cards were not meant to replace bracelets or other emergency identification. She hoped NBDF would have a resource toolkit for treatment centers to be able to modify instead of starting from scratch. A discussion was held to make everyone aware that emergency services in all areas might not take time to go through the patient’s wallet.

The Nursing Working Group was planning a mentorship program to pair nurses with others with different experiences that was part of their 2025 initiatives. She said it was an evidence-based practice initiative led by Joseph Stanco, DNP FNP-BC, CPI from Northwell Health Hemostasis and Thrombosis Center.

Dr. Tickle reminded everyone that an ongoing project was the updating of NBDF’s Nurses’ Guide to Bleeding Disorders. She said the committee was developed in January 2024. They had two chairs and 10 committee members. Four members had doctoral degrees, and there were numerous members with authorship experience. The committee’s goal was to provide mentorship to less experienced members on authorship and proper literature review and synthesis. They developed a foundational outline developed by the committee for chapter consistency. She said the initial chapters that were written and currently undergoing review included hemophilia A, hemophilia B, acquired bleeding disorders, platelet dysfunction, and rare bleeding disorders. She said the co-chairs were Maya Bloomberg from the Sylvester Comprehensive Cancer Center at Jackson Memorial Hospital and Jennifer Donkin from the Children’s Hospital Los Angeles. She listed the committee members as Julia Gardner, Tracey Gaslin, Mary Jones-Gant, Elissa Magsamen, Brenda Nielsen, Caroline Trotter, Glen Roy, Joseph Stanco, Kelly Tickle, and Mary Anne Williams.

 

15.        Research [Round Table] and Pathway to Cures Update        Dr. Recht

Dr. Recht shared that the National Research Blueprint (NRB) was newly called the Bleeding Disorders Research Collaborative (BDRC). He said it included areas to address gaps in knowledge that could improve the lives of people affected by inheritable bleeding disorders. The goal was to ensure that the lived experience experts drove and influenced the research process. They wanted to promote equity, diversity, and inclusion so that research findings included everyone.

Dr. Recht said the HEDI Foundation’s key stakeholders were LEEs, providers, researchers, chapters, and other Partners. He said education was part of the HEDI value statement, guiding principles, and implementation. Their activities included working groups, protocol development and review, research activities, mentorship and support, dissemination of findings, and collaborations.

Dr. Recht said the 2025 NBDF inaugural Research Roundtable would convene key stakeholders (NBDF, FDA, Industry, researchers, health care providers, non-profit organizations, and lived experience experts together) in a pre-competitive, product agnostic environment where stakeholders would address challenges and advancing clinical trial design for inheritable bleeding disorders. A goal would be to identify barriers to the inclusion of women, girls, and people with the potential to menstruate (WGPPM) in clinical trials for inheritable bleeding disorders, including those tied to reproductive health, hormonal cycles, and historical biases. They hoped to develop strategies to overcome identified barriers and establish shared guidelines and best practices to enhance diversity in clinical trials. They wanted to identify actionable steps, timelines, specific and measurable goals, and collaborative opportunities to implement strategies.

The roundtable agenda included a two-day meeting in October 2025. The schedule would include a plenary, approximately five sessions with NBDF/FDA/Participant Reflections, and conclusions and next steps. The topics would include things such as historical exclusion, biological differences, inadequate inclusion criteria, safety concerns, pregnancy/breastfeeding, bias in trial design, cultural and social barriers, recruitment challenges, funding and resources, and data disaggregation.

 

16.        MASAC Document #93 “Statement on Pregnancy Options for HIV-Discordant Couples” Retirement              Dr. Baldwin

Dr. Dunn shared there was no established policy to replace/review/retire MASAC documents. She said a process was initiated where all the current documents were cataloged, and she drafted a procedure to review and retire MASAC documents. She said she reviewed the oldest MASAC documents. She said some she thought should be revised, she questioned if they needed to be revised, and some she thought should be retired. She said she shared MASAC #93 with Dr. Baldwin as an example.

Dr. Baldwin reviewed MASAC Document #93 about HIV-discordant couples from 1998 that followed a 1994 guideline. She said the statement was very short and out of date. She proposed it be retired based on much more resent statements on the same topics. She proposed an entirely new document about fertility and genetic screening. 

Dr. Dunn shared the procedure that if a document was proposed for retirement at a MASAC meeting, a vote would be held to retire the document, and the NBDF staff would remove it from the website. 

Dr. Dunn asked for a motion to adopt the new adoption policy that was presented to MASAC before the meeting. MASAC approved the new policy to retire documents. 

Next, Dr. Dunn asked for a motion to retire #93, and MASAC voted to do so. 

As a third proposal, Dr. Dunn asked if there was any agreement to create a working group for fertility disorders. MASAC agreed, and she asked volunteers to contact Dr. Baldwin.

A question was asked if old documents were still available. It was noted that currently when a document was replaced, the old one disappeared from the website. Other options could be considered. Dr. Pierce noted a section for historical documents would be useful. Dr. Staber suggested those historical documents be stored where they were not publicly accessible to make sure they were not followed as current guidance. 

 

17.        Advocacy Update        Mr. Schaefer; Mr. Delaney

Mr. Schaefer and Mr. Delaney shared a federal update as of early March 2025 including an overview from the Executive Branch. They noted things were chaotic and fast-moving in Washington and moving in unprecedented directions. They said this was particularly true at the administrative level. They said Congress, to date, had largely kept to the sidelines, but the idea was that they now must act as the continuing resolution ends while a government shutdown was looming. They said NBDF was closely monitoring multiple administration level activities including executive orders on DEI, grant freezes and terminations, cutting NIH indirect research costs, health agenda firings, and downsizing. 

Mr. Schaefer said President Trump issued more than 80 executive orders since taking office with several taking aim at DEI policies inside and outside of government. They said there was a coordinated effort for the termination of all federal government DEI programs, mandates, and activities, including in federal contracts and awards. This was the basis for OMB’s January 27 memo ordering all federal agencies to freeze payment of grants. Another order directed all federal agencies to “combat illegal private-sector DEI preferences, mandates, policies, programs, and activities.” Mr. Schaefer said that executive order instructed agencies to include in every contract or grant a term requiring the grantee to certify that it does not operate “any programs promoting DEI that violate any applicable Federal anti-discrimination laws.” He said courts were intervening. 

Mr. Schaefer said additional executive actions included an HHS communications freeze and unfreeze, “Fork in the road” email; agency firings and downsizing, purges of agency websites, halt on ACIP and other advisory committee activities, and a proposed cut to NIH indirect cost rate.

For the legislative developments, Mr. Schaefer said that while the Executive branch activity takes up most of the limelight, Congress is considering changes to Medicaid financing and structure via the budget reconciliation process. He said the House budget resolution called for $880 billion in savings that would have to come from Medicaid. The idea was that Medicaid savings would go to pay for other priorities. He said implementation of these policies would shift massive costs to states, forcing state cuts to Medicaid eligibility, benefits, and/or provider reimbursement while jeopardizing coverage and care for the ~30% of the bleeding disorder community members enrolled in Medicaid. 

To address this, he said Washington Days included 248 Congressional meetings from 384 attendees where 49 states were represented. 

Mr. Schaefer that that in the present environment, NBDF should make strategic decisions about where and when to engage based on backlash. He feared threats to Medicaid via the budget resolution process in Congress, threats to federal health programs that benefit the bleeding disorder community, and threats to the affordability and availability of health insurance.

Mr. Delaney discussed that the advocates were given three issues to address when talking to officials during Washington Days. They were Medicaid cuts and policy changes, supporting Federal programs, and threats to the affordability and availability of health insurance.

Mr. Schaefer reiterated the policy landscape was challenging, but the community had compelling stories, a powerful cohort of motivated advocates, and strong partners.

 

18.        4th National HTC Patient Satisfaction Survey (PSS4) Ms. Lattimore

Ms. Lattimore shared results from the 4th National HTC Patient Satisfaction Survey. She shared how the survey was organized. She said it consisted of a steering committee or regional HTC Administrators with a background in nursing, public health, research, pharmacy, social work; health communications professionals; data analysts, and a research manager. She added there were national partners consisting of leaders from NHPCC, HRSA, CDC, ATHN, NBDF, HFA, FWGBD, Coalition for Hemophilia B, Hemophilia Alliance, and HTC Staff. All participation was voluntary with no dedicated funding. She said these surveys were conducted in 2015, 2018, 2021, and 2024 with each focused on HTC care in the prior year. She stressed that no protected health information was included.

Ms. Lattimore said the 2023 Patient Satisfaction Survey was a two-page survey that was self-administered in paper form or online and available in English or Spanish. She said participation was voluntary, and responses were anonymous. Regional leaders oversaw survey implementation, and HTCs independently managed distribution while parents and caregivers were asked to complete surveys for patients under 18 years. The aggregated results were returned to HTCs.

She said satisfaction questions addressed overall HTC care, HTC core team members (MD, RN, NP/PA, SW, PT, Pharmacy, Genetic Counselor), HTC Services, HTC care processes, and pediatrics to adult care transition.

She added that demographics included age, race and ethnicity, gender, diagnosis and severity, inhibitor status, number of HTC Encounters, and video visits.

She noted that barriers included insurance, language, COVID, and distance.

Ms. Lattimore shared that overall HTC satisfaction remained high, satisfaction with HTC staff remained high, and satisfaction with HTC processes and services also remained high. She said there was also high satisfaction with pediatric to adult care transition.

Barriers to care included insurance, language, COVID-19, and distance. 

Ms. Lattimore said the next steps were to use the data to improve care through HTCs, regions & partners to identify areas to improve, and analyze and disseminate results. She said one-page briefs, posters, and articles were made available. The next survey was planned for 2027 to cover care received in 2026.

 

19.        WFH Update   Dr. Pierce

Dr. Pierce discussed the background on the WHO Essential Medicines List and said that in the 2023 WHO EML, pathogen-reduced cryoprecipitate was listed as a core medicine and non-pathogen-reduced cryoprecipitate was listed as an alternative. PD-CFCs were listed as complimentary products.

Dr. Pierce raised safety and efficacy concerns in multiple interactions, WHO said they have no mechanism to change EML in mid-cycle. He they should file a new proposal. He said there was no guarantee of change if others refute their statements. 

Dr. Pierce said a proposal was submitted for revisions to the essential medicines list (EML) and the essential medicines list for children (EMLc) to include removal of cryoprecipitate (Cryo) (non-pathogen-reduced) for all indications, imitation of the use of pathogen-reduced cryoprecipitate (PR Cryo) to evidence-based indications outside the treatment of hemophilia A and von Willebrand Disease (VWD), transfer of the listings of plasma-derived Factor VIII (FVIII) concentrates for the treatment of hemophilia A and von Willebrand Disease (VWD) and factor IX (FIX) concentrates for the treatment of hemophilia B from the complimentary list to the core list, and removal of FIX complex as a therapeutic alternative to FIX concentrates for the treatment of hemophilia B.

He addressed the WFH Living Guidelines model by saying it was evidence-based and consensus driven. He said the gene therapy guidelines included approximately 85 evidence based and consensus driven recommendations and a manuscript submitted to Haemophilia in early February 2025. For 2025 he said the second project was planned to have an update of hemostatic agents and prophylaxis. He said the methodology team was selected; the guidelines panel was selected; and they had collaboration with EAHAD, EHC, NBDF.

For the WFH Living Guidelines governance, he said the steering committee included Glenn Pierce, Amy Dunn, Cedric Hermans, Magdalena Lewandowska, Johnny Mahlangu, Margareth Ozelo, Alok Srivastava, and Donna Coffin. The governance oversight Committee included Kate Khair, Mark Skinner, Clive Smith, Enrique David, and Preza Hernandez.

Dr. Pierce said the WFH Living Guidelines Model dissemination plan was based on ensuring widespread adoption and application of evidence-based practices. The target audience was healthcare professionals, PWH and policy makers. They wanted to publish updated chapters as stand alone in Haemophilia with a dedicated section on the WFH website for the guidelines with PDFs, infographics, lay summaries, and video summaries.

Dr. Pierce said education and training were planned to include sessions on the WFH guidelines at regional trainings and international conferences with host webinars tailored to healthcare professionals.

He said the WFH Shared Decision-Making Tool was focused on patient-centered care. 2025 would include a pilot test with a survey-based study underway. Updates would include new therapies when approved (fitusiran, NXT-007, Mim-8) and new languages.

Dr. Pierce said the WFH World Bleeding Disorders Registry for 2024 included 127 HTCs and 15,427 PWH and 1,250 PwVWD enrolled in the WBDR. He added 46 research projects across 24 countries had been funded.

For the WFH Gene Therapy Registry, Dr. Pierce said the national registries included the USA, Canada, and the Netherlands. The ethics were approved in Seattle (Barbara Konkle) and Philadelphia (Ben Samelson Jones). Others were Milan, Naples, and an upcoming Florence, Padua and Rome.

Implementation partners in 2025 were Germany, France, Japan, Spain, and the UK.

In 2026-27, they would add Belgium, Brazil, Finland, Ireland, Korea, Saudi Arabia, South Africa, and Sweden. 

 

20.        New and Deferred Business   Dr. Dunn

 

TFPI document Dr. Lewandowska

Dr. Dunn said Dr. Lewandowska and Dr. Staber edited a document on TFPI agents, and she asked them to present.

Dr. Lewandowska presented a document about anti-tissue factor pathway inhibitors. Noting that the agents were quite novel, she said she did include a great deal of information regarding management, and she hoped for feedback about what should be included. She began with a background stating that concizumab was a monoclonal antibody that binds to the Kunitz-2 domain of tissue factor pathway inhibitor (TFPI). The reduction of TFPI inhibitory activity allows for sufficient FXa[AS1]  production by the FVIIa-tissue factor complex to achieve hemostasis. Concizumab was shown to be safe and effective in adults and children older than 12 years with hemophilia A or B with inhibitors.

She addressed the suggested recommendations that concizumab may be considered for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients 12 years of age and older with hemophilia A or B with inhibitors; concizumab is used for prophylaxis, not for treatment of bleeding episodes; and patients should be educated on the risk, signs, and symptoms of thromboembolic events. 

For future research needs, she suggested the safety and efficacy of concizumab use in patients receiving ongoing immune tolerance induction (ITI) had not been established, therefore shared decision making should be implemented to discuss risks and benefits of continued treatment; optimal surgical management of patients on concizumab is not known; the safety of concomitant use of systemic antifibrinolytic therapy in patients on concizumab has not been determined; there is no safety data on the use of concizumab in pregnancy or lactation; and the safety and efficacy has not been established in patients younger than 12 years old.

After discussion and submission of ideas Dr. Dunn asked that the document be shared with the MASAC members for further review.

 

Playing it Safe

Dr. Dunn noted it was mentioned earlier in the meeting that the NBDF Playing it Safe publication that was originally written by the PT Working Group should be updated. She asked for that to be considered for future revision based on treatment changes, and she offered MASAC to work with the PT Working Group.

 

Guidelines Review

Dr. Dunn reiterated that many MASAC guidelines had not been reviewed or updated for quite a while, and a priority set earlier in the meeting was to update old guidelines. She asked for volunteers to contact her to review the obvious guidelines she reviewed to look at them and agree they should be submitted to MASAC for retirement. She believed this could be done by electronic voting before the next in person meeting. 

After that she said she wanted to further review the documents that didn’t need to be retired but needed to be revised. Dr. Dunn planned to share her first thoughts for documents that should be reviewed right after HTRS.

 

Transition Working Group

Mr. Petty reminded everyone that at the previous MASAC meeting, he proposed starting a transition working group. He said he wondered if there was still interest. Dr. Dunn said she was approached by a group who developed the transition guidelines for the Cystic Fibrosis Foundation. She said they were currently working with the sickle cell foundation on a program. She said since there was a group with this expertise, she felt they would be very interested in working with him. When asked who would be interested in working on this, Dr. Dunn, Dr. Staber, and Ms. Bender expressed interest. Dr. Dunn suggested they each go back to their centers and regions to ask for input. Dr. Dunn felt it was an interesting project that could be easily funded.

 

21.        Adjourn            Dr Dunn

Dr. Dunn noted Dr. Meeks and Dr. Reding would end their terms after the August meeting. She noted one idea was to add a genetic counselor to one of those positions, but the goal was to always have a balance of pediatric and adult providers so she asked for suggestions after the meeting, especially from a region not recently represented.

Dr. Dunn adjourned the meeting at 4:57 pm.

 

 

 

 

 

 

 [AS1]Allows for increased generation of FXa by the FVIIa-tissue factor complex to allow hemostasis