eCase - Hemostatic Balance in Hemophilia

Introduction

In this module, Miguel A. Escobar, MD; Margareth C. Ozelo, MD, PhD; and Runhui Wu, MD, PhD, provide an overview of the evolving treatment landscape of hemophilia, including novel agents for rebalancing homeostasis, best practices for integrating these agents into your clinical practice, and how to address common barriers to care and patient adherence.

The key points discussed in this module are illustrated with thumbnails from the accompanying downloadable PowerPoint slideset, which can be found here or downloaded by clicking any of the slide thumbnails in this module.

CCO plans to measure the educational impact of this activity. Several questions will be asked twice: once at the beginning of the activity and then once again after the discussion that informs the best choice. Your responses will be aggregated for analysis, and your specific responses will not be shared.

If you are a practicing healthcare professional (HCP), how many patients with hemophilia do you provide care for in a typical month?

A.
1-4
B.
5-10
C.
11-15
D.
16-20
E.
>20

Rebalancing hemostasis in patients with hemophilia includes all of the following strategies EXCEPT which one?

A.
Monoclonal antibodies directed against tissue factor pathway inhibitor (TFPI) at the initiation of the clotting cascade
B.
Small interfering RNA (siRNA) directed against antithrombin III (ATIII) during propagation of the clotting cascade
C.
Serpin molecules directed against activated protein C (APC)
D.
Gene therapy with an adeno-associated virus vector to produce FVIII protein

Which of the following was reported with the use of concizumab prophylaxis compared with on-demand bypassing agent (BPA) in the phase IIIa explorer7 trial in patients older than 12 years of age with hemophilia A or B with inhibitors who had been receiving on-demand BPA?

A.
There was no difference in mean annualized bleeding rate (ABR) with concizumab compared with on-demand BPA
B.
Concizumab decreased mean ABR compared with on-demand BPA
C.
Concizumab increased mean ABR compared with on-demand BPA
D.
Concizumab increased the rate of thromboembolic events regardless of individualized dose adjustments

Based on current clinical trial data, which of the following strategies could you consider to mitigate vascular thrombosis risk with fitusiran while maintaining efficacy?

A.
Monitor antithrombin (AT) levels and escalate or de-escalate fitusiran dose to maintain optimal AT target range
B.
Do not use concurrent factor replacement for breakthrough bleeding events
C.
Use fitusiran only for people with hemophilia A without inhibitors
D.
Use fitusiran only for people with hemophilia A or B if they have not previously received factor or BPA prophylaxis

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Research Focus: Achieving Hemostatic Balance in Hemophilia With Emerging Therapies

Miguel A. Escobar, MD

Margareth C. Ozelo, MD, PhD

Runhui Wu, MD, PhD

Activity

Introduction

If you are a practicing healthcare professional (HCP), how many patients with hemophilia do you provide care for in a typical month?

Rebalancing hemostasis in patients with hemophilia includes all of the following strategies EXCEPT which one?

Which of the following was reported with the use of concizumab prophylaxis compared with on-demand bypassing agent (BPA) in the phase IIIa explorer7 trial in patients older than 12 years of age with hemophilia A or B with inhibitors who had been receiving on-demand BPA?

Based on current clinical trial data, which of the following strategies could you consider to mitigate vascular thrombosis risk with fitusiran while maintaining efficacy?