Page 16 - Roche Hemlibra Non-inhibitors - Product Monograph

• Episodic therapy, regardless of the doses used, while essential in reducing the pain and
debilitating impact of individual bleeds, does not alter the bleeding profile significantly and
hence does not change the natural history of hemophilia leading to musculoskeletal damage
and other complications due to bleeding.

• Therefore, the use of prophylaxis is always recommended over episodic therapy. In countries
with healthcare constraints and for patients with limited access to CFCs, less intensive
prophylaxis regimens may be used. Still, in all countries the ideal is for patients to not
experience any bleeds (i.e., achieve “zero” bleeds).

• With the advent of innovative non factor replacement therapies (e.g. emicizumab), which for
the most part can be administered subcutaneously, prophylaxis is being redefined as the regular
administration (intravenously, subcutaneously, or otherwise) of a hemostatic agent/agents to
enhance hemostasis and effectively prevent bleeding in people with hemophilia.

Recommendation:
For patients with hemophilia A or B with a severe
phenotype (note that this may include patients with
moderate hemophilia with a severe phenotype), the WFH
strongly recommends that such patients be on
prophylaxis sufficient to prevent bleeds at all times, but
that prophylaxis should be individualized, taking into
consideration patient bleeding phenotype, joint status,
individual pharmacokinetics, and patient
self assessment and preference (WHF guidelines. 2020).

STANDARD OF CARE IN HEMOPHILA (WHF guidelines. 2020)
• According to WFH, the standard of care for all patients with severe hemophilia is regular
replacement therapy (prophylaxis) with CFCs, or other hemostasis products (emicizumab) to
prevent bleeding, started early in life (before age 3) to prevent musculoskeletal complications
from recurrent joint and muscle bleeds.

• Episodic (“on demand”) clotting factor replacement therapy should no longer be considered to
be a long term treatment option.

• Implementation of home based prophylaxis programs increases compliance and allows
people with hemophilia to live relatively normal lives. These programs should be accompanied
by education of patients, families, and healthcare providers on the benefits of prophylaxis and
the importance of adherence to treatment regimens.
• Prophylaxis in young children may be the best way for a country to begin implementing
universal prophylaxis for people with hemophilia.

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