Draft MASAC Minutes
September 14, 2024
Atlanta, GA
1. Call to Order Dr. Dunn
Dr. Dunn called the meeting to order at 8:10 a.m. and reminded members to submit their conflicts of interest statements. She thanked everyone for their attendance.
2. Chair Report Dr. Dunn
Dr. Dunn shared a history of MASAC and thanked the members for their service. She welcomed two new MASAC members: Anthony Unger, MD, and orthopedist, as an adjunct member; and she welcomed Sarah Dunn, DPT, NCS as the incoming Physical Therapy Working Group Chair.
Dr. Dunn offered thanks to members who were leaving the group: Tyler Buckner and Lora Joyner.
She paid tribute to Neil Frick, the recently deceased NBDF Senior Vice President for Medical Programs and Information who she noted served many years in assistance to MASAC.
Dr. Dunn discussed the priority setting from 2022 and reminded everyone that the topics were gene therapy, inhibitors, Joint health/pain, and access to care/women’s health through Health Equity Diversion, and Inclusion. She shared examples of successes in those areas. She said that moving forward in 2025 topics could be continuing to support the rebranding of NBDF; collaborating in opportunities such as the National Research Blueprint, Pathway to Cures, Community Voices in Research, and the WFH living guidelines. She went on to say capitalizing on a rapidly changing landscape of care should include gene therapy/editing and rebalancing agents. She added that the landscape of funding was changing, and there were threats to sustainability.
Dr. Dunn said the next steps after this meeting would be to re-evaluate the priorities from 2022, set new goals for the next 3-year cycle, finalize a conflicts of interest policy, and review and revise all outdated MASAC guidance.
3. NBDF Update Mr. Gattone
After sharing the NBDF mission, Mr. Gattone shared NBDF’s strategic imperatives of access, chapters, innovation, organization, sustainability, and vision. He expanded that access included advocating for equitable access to safe and effective care and treatment for all. He said chapters included building a strong and diverse national network of aligned stakeholders through collaboration and partnership. For innovation he included implementing and supporting a national collaborative research blueprint that is grounded in community input and amplifies a diverse patient voice. He added that organization included cementing NBDF as the premier patient-centric organization supporting the inheritable blood disorders community, advancing health equity, diversity, and inclusion. For sustainability, he mentioned including diversify and increased funding and partnerships to ensure sustainability and growth. He said vision included prioritizing and advancing the 2030 Blue Sky Vision.
To support the community, Mr. Gattone shared plans for advocacy and research, educational resources, and supportive care services. Mr. Gattone shared areas NBDF could work together more effectively including new therapy development; public awareness campaigns to drive Access to Care; diversified capacity building to fund research and innovation and chapter growth; and nationwide education, advocacy, supportive care.
Mr. Gattone concluded with a tribute to Neil Frick by Dawn Rotellini.
4. MASAC Goal Setting Dr. Dunn
Dr. Dunn started by saying that between this meeting and the next, she hoped to issue a new survey about topics to move forward. She displayed the results from the previous 2022 survey with the topics that received the most votes (women’s health, gene therapy, liver health, and pain). She said imaging was on the list, but she felt MASAC had not covered it enough. Dr. Dunn suggested being sure to have the patient and family perspective might be carried over to a new three-year period of focus.
She asked everyone to think of topics and be ready to vote in a new survey about things to focus on moving forward.
5. 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 340B, Mr. Blake shared legislative activity and said all stakeholders needed to work together to protect the program for HTCs and patient access to HTCs. He added a desire that institutions not interfere with money and let the community spend money on patient care and services.
The Hemophilia Alliance-funded genetic testing program provided testing for hemophilia A, B, and VWD. He said it was available to member HTCs. He said insurance did not cover testing and out-of-pocket expenses were too high, but no other options/programs were available. He said they used Versiti and Bloodworks labs.
Mr. Blake shared the Hemophilia Alliance’s community support in 2023 was more than $3,000,000, and for 2024, $4,000,000 was budgeted.
Mr. Blake shared updates on the NBDF Unite Walk HTC Challenge Celebration/Reception. He said the Hemophilia Alliance was in its fourth year sponsoring the event. In 2023 they honored the top five HTCs for raising funds. He added that over $41,000 was raised through HTCs and community involvement.
Mr. Blake said NBDF, the Hemophilia Alliance, HTCs, and local chapters were collaborating on big projects and state-based advocacy to support HTCs and patients. He said that included the importance of HTC sustainability to support patient care, healthcare policy, access to care, Medicaid, and other state-based issues that would arise.
Dr. Dunn stated after Mr. Blake’s presentation that MASAC had never shared opinions about 340B and maybe that should be considered in the next cycle.
6. Substance Abuse and Mental Health Dr. Reding
Dr. Reding shared the members of the Members of the Mental Health Work Group: Mark T. Reding, MD: Kate Reinhalter Bazinsky, MPH; Rose Bender, MSc; Brianna Cherrier; Tyler W. Buckner, MD, MSc; Amy L. Dunn, MD; Magdalena Lewandowska, MD; Lisa Littner, MPH, MSW, LISW-S, CHES; Michael Recht, MD, PhD, MBA; Jonathan C. Roberts, MD; Miranda Solem, MA, LADC; Rebecca Taylor, MD, MA; and Amy K. Wilson, LICSW.
Dr. Reding shared proposed updates to MASAC Document 279 Recommendations on Increasing Access to Mental Health and Substance Use Disorder Treatment Facilities and Programs for Persons with Bleeding Disorders. He said the approved SUD document was the basis for this combined SUD/MH document that included new considerations specific to mental health. He shared updates on the use of mental health medications that have potential impacts on coagulation for persons with bleeding disorders with specific examples in the appendix.
He shared updates on the use of crisis intervention techniques for people with bleeding disorders including physical holds, restraints, and emergency intramuscular injections. The use of electroconvulsive therapy was also addressed. He said response to self-injury was also covered.
Other sections of the proposed document included assessing ability for self-infusion in the context of a mental health crisis, recommendations for the emergency action plan, and a recommendation that if the co-occurring mental health condition makes regular infusions or injections challenging, the bleeding disorder treatment team should consider recommending a longer-acting hemostatic product.
Dr. Reding shared a few new issues that would restructure the document to highlight the key recommendation. He said it stated, “Provided a person with a bleeding disorder is medically stable and can maintain their established treatment protocol, having a bleeding disorder should not preclude a person from receiving mental health and/or substance use disorder treatment in a residential/inpatient setting.” He said it expanded the definition of stability to include those who prefer on-demand therapy, it included considerations for assessing ability to infuse from both the MH/SUD provider and bleeding disorder provider perspectives and highlighted the updates to federal rules.
After discussion, MASAC approved Document #289 Recommendations on Increasing Access to Mental Health and Substance Use Disorder Treatment Facilities and Programs for Persons with Bleeding Disorders.
7. Geriatric Task Force Dr. Kouides
Dr. Kouides said the geriatric task force included subsections including bone health, cardiovascular, CNS-Psychosocial, gender-specific issues, orthopedic, and others including audiology, dermatology, and ophthalmologic.
He said the bone health section members were Brandon Hardesty, Divya Citla Sridhar, and Glenn Pierce. The progress contained an algorithm on vitamin D screening and replacement and recommendations for osteoporosis screening with DEXA scan and management. For osteoporosis screening, he shared multiple studies that demonstrated hemophilia was independently associated with reduced bone mineral density and resulted in an increased risk of fracture. He said fracture rates seemed to increase above that in the general population and they were associated with increased morbidity. He said the group agreed with guidelines recognizing hemophilia as a cause of secondary osteoporosis. For prevention, they said alcohol should be minimized and smoking cessation should be encouraged, medications should be reviewed, and weight bearing or resistance exercises should be encouraged. For management, the group-suggested potential treatment options included androgen supplementation if hypogonadal, bisphosphonates, denosumab, bone anabolic agents.
He said the CNS-Psychosocial section included Kathaleen Schnur, Ellen Kachalsky, Tom Caprio, and Peter Kouides. He listed their considerations as complex and challenging medical decision making; health insurance; assessment for cognitive impairment, dementia, delirium, depression screening; access to skilled facilities and receiving factor medications; social determinants; elder abuse/fraud; isolation; caregiver fatigue; grief; and aging of the mind and body.
Dr. Kouides said the cardiovascular section members were Nathan Connell, Miguel Escobar, Erin Espinoza, Brandon Hardesty, Jonathan Roberts, and Mamie Thant. He said cardiovascular disease was the leading cause of death in the United States and advances in the care of persons with hemophilia led to an aging population that was developing age-related CVD. He said the prevalence of hypertension increased in people with hemophilia and that blood pressure should be measured at every clinic visit. Continuing, he said dyslipidemia was best managed by a primary care physician. The group also suggested that tobacco use should be reviewed at every clinical encounter.
The gender-specific issues section had two parts: breast health (Magdalena Lewandowska and Janice Staber) and prostate health (Tamara Bullock, Mark Skinner, and Joe Stanco). For breast health, they were developing guidance for procedures that can cause bruising and bruising and/or bleeding. For prostate health, they were developing guidance about prostate cancer including prostate biopsy and radical vs. simple prostatectomy.
Dr. Kouides said the ophthalmologic section is nearly completed by Ming Lim. An orthopedics document is being led by Anne Griest, Magdalena Lewandowska, and Tyler Buckner. This includes detailed documents for perioperative recommendations and thromboprophylaxis recommendations pending review. He added that a section on pain management was pending.
8. NBDF Educational Programs Ms. Hayes; Ms. Nammacher; Dr. Volland
The NBDF education team shared updates on the Own Your Path program and said it was an app-based program to help navigate life. Eligibility was 18 years or older, United States residency, von Willebrand Disease or hemophilia A or B, and currently on prophylaxis treatment. The program components were education on treatment management, health coaching, goal setting, decreasing stress, improving sleep, increasing physical activity, and virtual meetups with peers.
The Better You Know campaign was to raise awareness of signs and symptoms of a bleeding disorder. They said betteryouknow.org included tools and resources to assess risk and get care and treatment. A subcomponent was the Journey to Know program geared toward undiagnosed WGPPM.
They said the T.H.R.I.V.E. Campaign was a nationwide campaign and part of the CDC cooperative agreement. Its goals were to educate individuals with bleeding disorders and their support networks about six key steps for leading a healthy life and providing resources to empower community members and their loved ones to thrive in everyday life.
In MASAC for You, they said there were four new documents in English and Spanish and a document on Emergency Department management was available in English and Spanish.
The group shared information about the clotting cascade video series of short 3D videos to learn more about how blood clots and how treatments work in the body. They said there was an introductory video on the clotting cascade, a bispecific antibody video, and that videos were available in English and Spanish.
9. Pathway to Cures Update Ms. Willey
Ms. Willey said Pathway to Cures was the National Bleeding Disorders Foundation Venture Fund, LLC, and she said it existed to support NBDF in enabling cures for inheritable blood and bleeding disorders and to address and prevent the complications of these disorders. She added they had a goal to create future revenue sources to ensure NBDF continues to support the blood and bleeding disorders community.
Ms. Willey said they were focused on cures but also invested across areas with the potential to transform patients’ lives with a goal to offer first-in-class therapeutics; novel, precision diagnostics, including biomarkers; and novel technologies.
She said the current portfolio included Anvesana, Spark Biomedical, Afimmune, and Be Biopharma.
10. MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System Dr. Buckner
Before Dr. Buckner started, Dr. Dunn asked if anyone would be interested in taking over this document since Dr. Buckner’s MASAC term was ending. Dr. Lewandowska expressed interest.
Dr. Buckner shared updates to Document #284 MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System. He said the primary changes regarded a new gene therapy product. He said fidanacogene elaparyovec was a gene therapy product approved for adults with moderate to severe congenital hemophilia B who were using FIX prophylaxis therapy or who had current or historical life-threatening bleeding or repeated, serious spontaneous bleeding episodes.
A discussion was held about whether this document should be a comprehensive compendium of treatment products available for certain blood disorders. That purpose could be reviewed in the future.
MASAC approved Document #290 MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System.
11. Anemia Task Force Dr. Batsuli
Dr. Batsuli said the working group the rationale was based on the fact that iron deficiency with and without anemia is a recognized complication of bleeding disorders management. She said there was a lack of recommendations or guidance on anemia screening for individuals with inherited bleeding disorders. She said the main objective was to generate guidance regarding screening.
For working group progress, Dr. Batsuli said there were two working group meetings in July 2024, and they established a framework to devise anemia screening guidance. She said there was a draft document in progress and key areas to address were guidance for providers and consumers, age and sex-based consideration, frequency of testing, and lab tests and reference ranges.
There were pending formal anemia guidelines with new AAP anemia guidelines anticipated for 2026, upcoming ASH guidelines, and FWGBD guidelines, but none of those would directly address the bleeding disorders population. Dr. Batsuli said a general problem was a lack of data so reaching out to other organizations and the community would be a good focus.
12. NBDF Research Update Ms. Santaella
Ms. Santaella shared an update on the National Research Blueprint. She said it was an ambitious initiative to transform research through full community engagement.
She shared updates from the NRB Summit in January 2024. She said the NRB webpage was updated to include refined research priorities, numerous resources, and ways to get involved. The Progress Report brochure was updated and sent to all involved to date.
She said the research priorities included lived experiences, bleeding, female bleeding, joint and bone health, lifespan, mechanisms of bleeding, immune response, diagnostics, and treatment.
For the NRB Summit in January 2024, she said they updated the NRB webpage to include refined research priorities, numerous resources, and ways to get involved. The Progress Report brochure was also sent to all involved to date and could be found on the website. She said they were seeking various funding opportunities, engaging with potential partners, and involved in manuscript development. To refine the concept of “minimum viable product” she said they were operationalizing a process to receive research proposals using Proposal Central. She said new working groups will be formed based on the expertise required and the development of Research Ambassador Program was in progress.
For the Lived Experience Expert program, she said the plan was to leverage Community Voices in Research (CVR) and expand its capabilities by developing curricula and using CVR as a platform to connect researchers and LEEs with educational programs, to keep track of those who have gone through the program, to connect the team according to specific research projects, and to follow projects over time.
Dr. Santaella said the Research Processes aimed for a Minimum Viable Product. She said research concepts are evaluated by topical working groups. They were to collaborate with researchers on protocol development. She said the protocols were refined for budget, LEE/HEDI considerations, and IRB review as needed.
She said CVR was a Community Powered Registry. The goal was to collect information directly from people affected by inheritable bleeding disorders. It was to provide two-way communication and would complement other research efforts in the community. It centered research on the lived experience. She said a family baseline survey and Spanish translation were coming soon.
13. Hemophilia A Analytical Cohort Research Program Dr. Meeks
Dr. Meeks shared an update on the Hemophilia A Analytical Cohort Research Program (HARP). She said it was an intergenerational precision medicine research program to study factor VIII immunogenicity in severe hemophilia A. She said it was an NIH-funded study. She said it had evolved a great deal over the previous 24 months, but they now had the first decentralized subject enrolled. She said they were moving forward.
Dr. Meeks said it was a novel study of hemophilia A in mothers and babies. They were seeking a better understanding of bleeding risks associated with hemophilia in pregnancy and identifying causes of inhibitor formation in early childhood. HARP is an observational, longitudinal study. They would recruit pregnant people, follow mothers through pregnancy, and develop a shareable research resource of data samples.
Dr. Meeks invited everyone to sign up for the HARP mailing list. https://harpf8.org
14. Testing for Coagulation Assays Document Dr. Meeks
1:22 PM
Dr. Meeks shared updates for MASAC Document #228 Statement Regarding Use of Various Clotting Factor Assays to Monitor Factor Replacement Therapy. She said the updates were more difficult than anticipated. She said it was originally written as a guidance document for laboratories to encourage them to have the support that hemophilia treatment centers need to provide information for patient care. Dr. Meeks shared that since the last update, they have had four special coagulation lab professionals assist in review.
Sections of the document included diagnostic challenges and an expanded section on clotting factor concentrates to include factor produced by gene therapy. The document included four recommendations for laboratories routinely performing coagulation factor assays.
Dr. Meeks said she would share the draft document with everyone for feedback.
15. Bone Health Task Force Document Ms. Joyner
Ms. Joyner shared updates to the Physical Therapy Management for Bone Health in People with Bleeding Disorders. She said the PT Task Force developed these recommendations based on request from MASAC in BDC 2023. She said the task force was had of three PTWG members (Lora Joyner, Sarah Dunn, Anne Gonzales) and one HTC PT (Nikki Clark). She said the document had been reviewed by the PTWG and the Geriatric Task Force Bone Health subcommittee. All agreed they were In line with recommendations of the subcommittee. The PT recommendations referred to medical guidance for detail and sent out to MASAC Committee prior to this meeting.
Ms. Joyner said there were two assessments and four Intervention recommendations. For assessment, she said they screened for modifiable and non-modifiable risk factors and screened all people with bleeding disorders over the age of 30 for fall risk at least annually. She said interventions included education, pediatrics, therapeutic Exercise, and therapeutic Modalities.
She invited comments, and MASAC approved Document #291 Physical Therapy Management for Bone Health in People with Bleeding Disorders
Ms. Joyner shared the current MASAC Document 283 and said an issue was discovered in the process of publishing recommendations. She said the iteration of the document that was approved by MASAC was not the version that was published. She said there were minor editorial issues but no content changes and questioned if the document would require a new number. She said the minor changes were that the title should say “for” People with Bleeding Disorders not “of” People. She said the last statement was missing on posted document: “PWBD experiencing chronic pain should be offered therapeutic modalities as part of a multimodal treatment approach for chronic pain. She added there should be ten total recommendations (1 assessment and 9 treatment statements).
MASAC decided that since the version shared with MASAC was already approved, it did not need a new number.
16. MASAC Guidance for Genetic Testing for Non-Hemophilia Disorders Dr. Singleton
Dr. Singleton said their working group members were Tammuella “Tami” Chrisentery-Singleton, MD; Suchitra Acharya, MD; Glavy Batsuli, MD; Samantha Cassisi, MS; Sweta Gupta, MD; Nala Hamilton; Jill Johnsen, MD; Lori Luchtman-Jones, MD; Osman Khan, MD; Diane Nugent, MD; Amy Shapiro, MD; Shirley Williams, PhD; Kristin Zajo, CGC.
She shared the guidance for Genetic Testing for Rare Bleeding Disorders and said their charge was to establish guidance for MASAC regarding genetic testing for rare bleeding disorders. She said they started by deciding how they could provide guidance to better understand the biology of non-hemophilia disorders and improve our ability to diagnose these disorders using genetic testing. She said as a committee they identified the priorities as confirming or refining the diagnosis, predicting the severity, determining risk, identifying genetic carriers, helping with reproductive counseling and birth planning, and leading to improved, individualized treatments.
Dr. Singleton said their goals included making recommendations that were supported strongly with evidence and stand the test of time, providing conservative recommendations that stand on very firm footing. She said they needed literature for almost every sentence and ultimately have recommendations on genetic testing and a call to action.
To follow up from the spring MASAC meeting, she said they had three meetings in May, June, and July 2024, added a LEE to their committee, and developed separate recommendations for VWD and ultra-rare bleeding disorders. She said the genetic testing recommendations would include CLIA-approved laboratories with expertise. She said genetic counseling would be included in the recommendations. She added that further discussion was needed to address genetic studies to have counseling and education written into the study. Dr. Singleton said the committee would need to refine the statement on novel variants and VUS and decide what to do when patients present serendipitously following testing for other conditions.
Dr. Singleton said the next steps included guidance for genetic testing for ultra-rare bleeding disorders with follow up recommendations from She hoped to finalize the recommendations for presentation to vote during the spring 2025 MASAC meeting.
17. My Life, Our Future Mr. Skinner
Mr. Skinner said their vision was to bring a national program to the hemophilia community that offered free genotyping to people with hemophilia and their families. The hope was that may help improve hemophilia care by increasing understanding of the disorder and build a foundation for the scientific breakthroughs of tomorrow. Mr. Skinner said the MLOF program findings had been published.
Mr. Skinner said a high rate of novel DNA variants was found with 1914 unique DNA variants clinically reported, 744 (38.9%) of variants were novel, with a conclusion that there were likely still many other DNA variants yet to be seen that can cause hemophilia. They were characterized by DNA variants in hemophilia A and B at-scale including all kinds of DNA variations were detected and severe hemophilia A and B skewed have the most gene-disrupting kinds of DNA variants.
Mr. Skinner said the new genotype-inhibitor risk findings showed “no variant found” is a high-risk genotype in severe hemophilia and that a new class of high-risk F8 genotype showed complex F8 inversions. He said race and ethnicity independently contribute to inhibitor risk race and ethnicity independently contribute to inhibitor risk.
The MLOF Research Repository was formed to fulfill the original MLOF vision to support the scientific breakthroughs of tomorrow. The MLOF Research Repository contained F8 and F9 clinical genotype and DNA sequence, and the sequence from both genes is available for all participants.
For the NHLBI Trans-Omics for Precision Medicine Program (TOPMed), he said TOPMed supported scientific advances through the integration of whole-genome sequencing and other -omics data with other data from studies focused on heart, lung, blood and sleep disorders. He said the TOPMed has sequenced ~180,000 genomes.
18. WFH Update Mr. Skinner
Mr. Skinner shared the scope and methodology of the ISTH guidelines. He said for evidence regarding the effects of interventions, randomized trials were considered the main source of data. If no randomized trial was available, the search was expanded to include nonrandomized intervention studies.
He said the stated limitations of the guidelines were due to a scarcity of robust studies in the hemophilia field and that most recommendations were based on a low-certainty or very low–certainty evidence. He added that since questions included in this guideline were prioritized in 2021, it did not include therapies approved after 2021. He said this first document for this guideline was developed in March 2019, and the panel was appointed at the end of 2020, during the COVID-19 pandemic. Within the last five years, hemophilia care has changed dramatically. The main outcomes assessed were annual bleeding rate, annual joint bleeding rate, and inhibitor formation.
Mr. Skinner shared information about how to use GRADE when there is less evidence through a case study of the expert evidence approach. He said the background & methods included preparing practice guidelines for treatment of VTE in the pediatric population, extensive search of published literature was performed. He said the results showed the evidence synthesis team was unable to identify published direct or indirect evidence for several questions or for critical outcomes. He said the proposed conclusion was to solve the issue of lack of evidence. They administered an expert evidence survey asking panel members about their unpublished observations and case series.
Mr. Skinner said the key findings showed that guideline developers can apply GRADE and use a structured approach of summarizing collective expert experience when published evidence is limited or non-existing.
Mr. Skinner said WFH Guidelines for management of hemophilia included ISTH Guidelines for Treatment of Hemophilia - The WFH Guidelines (2020) – A living document!. He said it was a comprehensive document created through a trustworthy consensus-based statement process to produce evidence-informed recommendations supported by a comprehensive and systematic search for relevant scientific literature, which was first screened based on predetermined inclusion/exclusion criteria, then followed by data extraction of the available and relevant evidence
Mr. Skinner shared updates about the Essential Medicines List. He said priority conditions were selected based on current and estimated future public health relevance and the potential for safe and cost-effective treatment. He said the core list presented a list of minimum medicine needs for a basic health-care system, listing the most efficacious, safe and cost-medicines for priority conditions. He said the complementary list presented essential medicines for priority diseases, for which specialized diagnostic or monitoring facilities, and/or specialist medical care, and/or specialist training are needed.
For WFH actions, Mr. Skinner said that given that many countries rely upon the WHO EML for accurate information and due to the irreversible nature of many pathogen infections, WFH called upon the WHO to follow their previously well-established principles for accessibility, efficacy, and safety and take immediate action to correct this action and not wait until the next scheduled revision of the WHO-EML in 2025.
19. Advocacy Update Mr. Schaefer
Mr. Schaefer started his update with APLUS recent & future initiatives including 2024. He said presentations were made of sole patient representative to Advisory Committee on Blood and Tissue Safety and Availability (ACBTSA), four plasma caucus briefings and meetings. He said other activities were held with Caucus/Congress, engagement with PLUS, HHS Giving=Living campaign, and state policy regarding plasma donation.
The Voices for Change Video Update included partnership with Believe Limited, unveiling 14 videos at BDC. He said there was a diverse representation of community members and completion of four more videos by the end of the year.
Mr. Schaefer shared updates about the election for 2024 Get Out the Vote toolkit. He stressed there were major elections in November. He said 1,000 Postcards with Get out the Vote and Voter Registration Information were distributed at BDC, a chapter toolkit was distributed, social media suggestions were shared, and these were all within the mindset of what nonprofits can do legally
For Washington Days Highlights, he said 428 participants were registered representing 45 states. He said the 2025 dates were March 5-7.
20. ATHN Update Dr. Singleton
Dr. Singleton said the ATHN Data Summit was date driven and research focused. The research team focus was data quality, availability of the data to investigators, projects with young investigators, fluidity of study and site start-up/site participation, site communications and support, medical monitoring/adverse event reporting, and compliance models, processes, and infrastructure.
For ATHN Transcends she shared the goal of Exploration Through Collaboration included leveraging ATHN Transcends as an avenue for ATHN affiliates to meaningfully participate in the ideation, formation, and execution of research initiatives. She said they were expanding their collaborative research process to support ATHN affiliate investigators in developing concepts to answer scientific and clinically meaningful questions. They also wanted to enable ATHN affiliate investigators to transform research concepts into fully funded studies seamlessly using ATHN’s infrastructure and services.
For Data Commons, she said ATHN and NBDF were collaborative partners in the inheritable bleeding disorders field, sharing many key aspects of our missions in improving the lives of people affected by these conditions. She said they were utilizing ATHN systems of electronic data capture, data management and biostatistical services, and research support.
She said LEEs should be moved to the forefront of transformational research and examine where research can have the most community impact.
21. CDC Liaison Dr. Burton
Dr. Burton shared two updates from CDC. The first was a status update on Community Counts Hemophilia Inhibitor Surveillance Report which was still moving forward. He said it was their first comprehensive look at the data they had on inhibitors from 2013-2022. They were to describe the impact of inhibitors of 15,000 persons.
He also shared there were some changes coming to the viral antibody testing that was done as part of Community Counts. He said that for year five of the Community Counts cooperative agreement, which was to start at the end of the current month, they would be discontinuing the viral HIV, hepatitis C, and SARS-CoV-2 testing.
22. Upcoming Nursing Documents Dr. Tickle
Dr. Tickle shared new Nursing Working Group Projects including primary care provider educational forms, the nurses’ guide to bleeding disorders, and emergency treatment pocket forms.
She said the primary care provider educational forms covered injections, epistaxis, and school nurses.
Dr. Tickle said the Nurses Guide to Bleeding Disorders group was formed in 2024. She said it had two Co-Chairs and 11 Committee Members.
For the Emergency Treatment Pocket Card, she said they would include name, date of birth, bleeding disorder diagnosis, inhibitor status, and HTC information. She said there would be diagnosis and disease specific cards.
23. New and Deferred Business Dr. Dunn
Mr. Petty presented an idea addressing transition. He put forward a motion to see if MASAC would support a working group focused on the transition and retention of patients. Others added that all the transitions through life could be included, not just childhood to adult.
Dr. Dunn asked Mr. Petty to be sure to propose the transition topic when the call for goal setting ideas. Members suggested getting a diverse perspective by having working group members representing several different backgrounds.
Mr. Petty shared a draft description, mission statement, and committee makeup. He proposed working group topics could be: How to guide the transition from pediatric to adult. What are the barriers of entry/conversation to a new healthcare team. Education gaps. Patient-physician relation and how to be a team/partnership.
Mr. Petty was asked to continue developing this idea.
Dr. Lewandowska said that plasminogen deficiency was not a bleeding disorder, but it was an absence of a clotting factor and coagulation factor that could have severe consequences. She shared several problems that could arise for these patients. She noted that there was a new medication approved for plasminogen replacement therapy. She said the belief that this condition was best treated at a hemophilia treatment center, but many times show signs and symptoms for the first time at many places such as ophthalmology, dentistry, pulmonary, etc. and should be referred to the HTC. Once the disease is suspected, she shared the belief that the HTCs should provide the care for these patients.
She proposed drafting a document to say they should be treated at an HTC.
Dr. Dunn and MASAC agreed this idea should be explored.
Dr. Lim reminded everyone that in the previous March meeting, MASAC discussed if MASAC should make treatment recommendations that were not FDA approved. As an update, she said since that time a group drafted a recommendation but even internally they were having debates.
24. Adjourn Dr. Dunn
Dr. Dunn adjourned the meeting at 4:24 p.m.