PP111 Toward Healthy Coagulation In Hemophilia

Abstract

Introduction:

Healthcare advances in hemophilia have led to near-normal life expectancies in a disorder previously associated with early death. Unlike other disorders where the therapeutic goal is to restore deficiencies to normal levels, prophylaxis in hemophilia is used to achieve a plasma level of FVIII >1%, such that severe hemophilia may be reduced to a moderate/mild phenotype. With the development of new therapies, treatment goals are evolving from on-demand treatment or prevention of bleeds to one where the risk of bleeding is minimal/absent. To accelerate this development, a new treatment paradigm is needed, with consensus from key stakeholder communities, to facilitate a shared vision for the future of hemophilia healthcare.

Methods:

A panel of hemophilia providers, patient advocates, and industry representatives convened to develop a new treatment model that establishes specific treatment milestones and target outcomes in a stepwise fashion, culminating in a progressive definition of cure.

Results:

To represent the collective experience of hemophilia for patients and treaters around the world, the following treatment milestones were defined based on optimized outcomes: (i) Sustain Life – prevention of premature death; (ii) Minimal Joint Impairment – improved quality of life; participation in activities of daily living; (iii) Freedom From Spontaneous Bleeds – ability to engage in low-risk activities; (iv) Attainment of ‘Normal’ Mobility – participation in work, career, and family life without restriction; (v) Able to Sustain Minor Trauma – more unrestricted lifestyle; (vi) Ability to Sustain Major Surgery or Trauma Without Additional Intervention – no dependency on specialized healthcare; (vii) Normal Hemostasis – optimal health and well-being; and (viii) Cure – health equity.

Conclusions:

With milestones for disease management leading toward normalized hemostasis, this treatment model provides a vision to improve hemophilia care for all patients. And by providing achievable outcomes, the community—patients, treaters, and their industry partners—has a clear path to achieve that goal.