Advances in Hemophilia: From Joint Health to FVIII Guidelines and the Clinical Integration of Rebalancing Agents

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* 1. A 22-year-old woman with hemophilia A on prophylaxis reports no symptoms of joint bleeding or impacts on daily functioning or quality of life over the past 2 years. During a routine visit, point-of-care musculoskeletal ultrasound reveals blood within the left knee. Which of the following management approaches is most appropriate?

A. Continue the current prophylaxis regimen with closer follow-up.

B. Reassess FVIII activity and prophylaxis treatment.

C. Administer a single on-demand dose of FVIII concentrate.

D. Consider arthroscopic joint lavage or synovectomy.

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* 2. Which of the following statements best reflects current evidence on FVIII trough targets in hemophilia A prophylaxis?

A. A trough level of 1% or higher eliminates spontaneous joint bleeding in the majority of patients.

B. Extended half-life products increase peak FVIII levels without affecting time spent above protective activity thresholds.

C. Increasing trough levels to a 3-5% range or higher is associated with further reductions in spontaneous and subclinical joint bleeding.

D. Raising FVIII trough levels above 8% offers little clinical benefit and increases the risk of adverse events with factor-based replacement therapies.

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* 3. Which of the following statements best reflects key evidence from the pivotal clinical trials of rebalancing agents used in the treatment of hemophilia?

A. In the ATLAS trial program, fitusiran required daily subcutaneous dosing to achieve bleeding rates comparable to factor concentrate-based prophylaxis.

B. The approval of marstacimab for hemophilia A or B, with or without inhibitors, was based on the BASIS trial program, which evaluated this small interfering RNA therapy targeting antithrombin and showed reduced annualized bleeding rates comparable to factor concentrate‑based prophylaxis.

C. In the EXPLORER program, concizumab significantly reduced annualized bleeding rates but was associated with thrombotic events, leading to the implementation of a mitigation plan.

D. Across the ATLAS, EXPLORER, and BASIS trial programs, rebalancing agents shared the same mechanism of action and demonstrated equivalent hemostatic efficacy.

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* 4. A 25-year-old man with hemophilia A without inhibitors is on extended half-life FVIII prophylaxis. Despite excellent adherence, he reports finding IV access increasingly stressful. He has had no recent target joint bleeds on his current regimen. Which of the following is the most appropriate next step?

A. Adjust the prophylaxis regimen to target FVIII troughs ≥ 1% and reduce administration frequency.

B. Transition to on-demand therapy to reduce treatment burden.

C. Switch to a rebalancing agent as these are administered subcutaneously.

D. Transition the patient to a different extended half‑life FVIII regimen to reduce infusion frequency.

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* 5. What is your highest attained credential?

MD/DO

PA/PA-C

NP/APRN

PharmD/RPh

PhD

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* 6. What is your specialty?

Hematology

Hematology/oncology

Internal Medicine

Orthopedics

Family Medicine/Primary Care

Research

Other (please specify)

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* 7. Which of the following describes your primary practice setting?

Hemophilia Treatment Center

Group practice

Solo practice

University/Teaching System

Hospital-Based Specialty Clinic

Specialty Pharmacy Setting

HMO/managed care

Other (please specify)

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* 8. How many years have you been in practice? (Please use numbers only)

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* 9. How many patients with hemophilia A or B do you see per month? (Please use numbers only.)

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* 10. Please let us know how this activity will help you with patient care.

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* 11. Please rate how well the activity met the identified objective:

	Strongly Agree	Agree	Neutral	Disagree	Strongly Disagree
Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice.	Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice. Strongly Agree	Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice. Agree	Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice. Neutral	Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice. Disagree	Formulate strategies to address the joint health-related unmet needs of patients with hemophilia in clinical practice. Strongly Disagree
Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines.	Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines. Strongly Agree	Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines. Agree	Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines. Neutral	Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines. Disagree	Assess the impact of factor-based replacement therapies on FVIII levels according to the guidelines. Strongly Disagree
Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management.	Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management. Strongly Agree	Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management. Agree	Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management. Neutral	Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management. Disagree	Evaluate the safety and efficacy of newly approved rebalancing agents and implement integration strategies that support personalized, patient-focused hemophilia management. Strongly Disagree

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* 12. Please rate the effectiveness of the presenting faculty:

	Strongly Agree	Agree	Neutral	Disagree	Strongly Disagree
The faculty was effective in presenting the material	The faculty was effective in presenting the material Strongly Agree	The faculty was effective in presenting the material Agree	The faculty was effective in presenting the material Neutral	The faculty was effective in presenting the material Disagree	The faculty was effective in presenting the material Strongly Disagree
The faculty was knowledgeable about the subject matter	The faculty was knowledgeable about the subject matter Strongly Agree	The faculty was knowledgeable about the subject matter Agree	The faculty was knowledgeable about the subject matter Neutral	The faculty was knowledgeable about the subject matter Disagree	The faculty was knowledgeable about the subject matter Strongly Disagree

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* 13. Please rate the effectiveness of the CME/CE content:

	Strongly Agree	Agree	Neutral	Disagree	Strongly Disagree
The content addressed the learning objectives (purpose)	The content addressed the learning objectives (purpose) Strongly Agree	The content addressed the learning objectives (purpose) Agree	The content addressed the learning objectives (purpose) Neutral	The content addressed the learning objectives (purpose) Disagree	The content addressed the learning objectives (purpose) Strongly Disagree
The activity enhanced my current knowledge base	The activity enhanced my current knowledge base Strongly Agree	The activity enhanced my current knowledge base Agree	The activity enhanced my current knowledge base Neutral	The activity enhanced my current knowledge base Disagree	The activity enhanced my current knowledge base Strongly Disagree
The information was relevant to my practice and my educational needs	The information was relevant to my practice and my educational needs Strongly Agree	The information was relevant to my practice and my educational needs Agree	The information was relevant to my practice and my educational needs Neutral	The information was relevant to my practice and my educational needs Disagree	The information was relevant to my practice and my educational needs Strongly Disagree
The information will help me improve patient care	The information will help me improve patient care Strongly Agree	The information will help me improve patient care Agree	The information will help me improve patient care Neutral	The information will help me improve patient care Disagree	The information will help me improve patient care Strongly Disagree
The activity provided new ideas or information I expect to use	The activity provided new ideas or information I expect to use Strongly Agree	The activity provided new ideas or information I expect to use Agree	The activity provided new ideas or information I expect to use Neutral	The activity provided new ideas or information I expect to use Disagree	The activity provided new ideas or information I expect to use Strongly Disagree
The activity provided appropriate learning assessment activities	The activity provided appropriate learning assessment activities Strongly Agree	The activity provided appropriate learning assessment activities Agree	The activity provided appropriate learning assessment activities Neutral	The activity provided appropriate learning assessment activities Disagree	The activity provided appropriate learning assessment activities Strongly Disagree
The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge	The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge Strongly Agree	The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge Agree	The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge Neutral	The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge Disagree	The activity addressed the ABMS/ACGME core competencies of patient care, professionalism, and medical knowledge Strongly Disagree

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* 14. The content presented:

	Strongly Agree	Agree	Neutral	Disagree	Strongly Disagree
Was scientifically rigorous and evidence based	Was scientifically rigorous and evidence based Strongly Agree	Was scientifically rigorous and evidence based Agree	Was scientifically rigorous and evidence based Neutral	Was scientifically rigorous and evidence based Disagree	Was scientifically rigorous and evidence based Strongly Disagree
Was fair, balanced, objective, and free of commercial bias	Was fair, balanced, objective, and free of commercial bias Strongly Agree	Was fair, balanced, objective, and free of commercial bias Agree	Was fair, balanced, objective, and free of commercial bias Neutral	Was fair, balanced, objective, and free of commercial bias Disagree	Was fair, balanced, objective, and free of commercial bias Strongly Disagree

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* 15. Based upon your participation in this activity, do you intend to change your practice behavior? Choose only one of the following options:

I do plan to implement changes in my practice based on the information presented.

My current practice has been reinforced by the information presented.

I need more information before I will change my practice.

Not applicable to my scope of practice

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* 16. If you plan to change your practice behavior, what types of changes do you plan to implement? Check all that apply:

Modify my approach to monitoring and managing joint health in patients with hemophilia

Modify my approach to identifying and addressing subclinical bleeding

Modify my approach to applying evolving evidence on FVIII targets in prophylaxis planning

Modify my approach to selecting and optimizing factor-based replacement strategies

Modify my approach to safety monitoring and adverse event mitigation with hemophilia therapies

Incorporate rebalancing therapies into care for appropriate patients with hemophilia

Incorporate extended half-life therapies into care for appropriate patients with hemophilia

Incorporate shared decision-making when selecting prophylaxis strategies

Incorporate multidisciplinary approaches to hemophilia care

Other (please specify)

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* 17. How confident are you that you will be able to make your intended changes?

Very confident

Confident

Somewhat confident

Unsure

Not very confident

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* 18. In what time frame do you anticipate making these intended changes?

Immediately

1-2 months

3-6 months

Some point in the future

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* 19. Which of the following parameters do you anticipate will be the primary barrier to implementing these changes? Please choose only one of the following options and provide any additional details in the box below:

I do not anticipate any significant barriers

Limited access to diagnostic tools or monitoring resources

Lack of familiarity with current guidelines

System or institutional constraints

Patient adherence or treatment burden

Time or resource constraints

Lack of familiarity with new and evolving therapies

Safety concerns or treatment-related adverse events

Other (please specify)

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* 20. How confident were/are you in your ability to identify and address joint health–related unmet needs in patients with hemophilia, including subclinical bleeding and long-term joint protection?

	Very Confident	Confident	Somewhat Confident	Unsure	Not Very Confident
Before this activity?	Before this activity? Very Confident	Before this activity? Confident	Before this activity? Somewhat Confident	Before this activity? Unsure	Before this activity? Not Very Confident
After this activity	After this activity Very Confident	After this activity Confident	After this activity Somewhat Confident	After this activity Unsure	After this activity Not Very Confident

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* 21. How confident were/are you in applying FVIII targets and optimizing factor-based replacement strategies to reduce bleeding risk and improve long-term outcomes in hemophilia?

	Very Confident	Confident	Somewhat Confident	Unsure	Not Very Confident
Before this activity?	Before this activity? Very Confident	Before this activity? Confident	Before this activity? Somewhat Confident	Before this activity? Unsure	Before this activity? Not Very Confident
After this activity	After this activity Very Confident	After this activity Confident	After this activity Somewhat Confident	After this activity Unsure	After this activity Not Very Confident

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* 22. How confident were/are you in evaluating and integrating rebalancing therapies into individualized hemophilia care?

	Very Confident	Confident	Somewhat Confident	Unsure	Not Very Confident
Before this activity?	Before this activity? Very Confident	Before this activity? Confident	Before this activity? Somewhat Confident	Before this activity? Unsure	Before this activity? Not Very Confident
After this activity	After this activity Very Confident	After this activity Confident	After this activity Somewhat Confident	After this activity Unsure	After this activity Not Very Confident

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* 23. Did you view and/or download the slides accompanying the activity?

Yes

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* 24. Please let us know what you found most helpful in this activity.

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* 25. Complete to receive your credit

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* 26. Email

Thank you for participating! Please click "Done" to submit your answers. You will receive your certificate via email within 3 weeks.