Page 17 - Roche Hemlibra Non-inhibitors - Product Monograph

BENEFITS OF PROPHYLAXIS

PROPHYLAXIS USING CLOTTING FACTOR CONCENTRATES (WHF guidelines. 2020).

• All forms of prophylaxis (high/intermediate/low dose with CFCs or prophylaxis with non factor
replacement agents, e.g., emicizumab) provide superior benefits over episodic therapy.
Conventional high dose and intermediate dose prophylaxis, initiated early in life, have been
associated with over 90% reduction in joint bleeding rates, annualized joint bleeding rates
(AJBRs) below 3 per year, and a significant reduction in joint deterioration and degenerative
joint disease.
• Prophylaxis also provides protection from other types of hemorrhages in hemophilia,
including preventing or substantially reducing the risk of intracranial hemorrhage.

• Longer term benefits include reduction of chronic musculoskeletal pain, functional limitations
and disability, need for orthopedic surgery, hospitalization, emergency room visits, and
reduced length of hospital stays; all of this leads to greater participation (i.e., regular
attendance) in educational, recreational, and professional activities, with improved quality of
life.

• Because of these benefits, the World Health Organization (WHO), the WFH, and many national
and international hemophilia organizations have endorsed early prophylaxis as the standard
of care for children with a severe phenotype hemophilia and recommend that prophylaxis be
continued lifelong. Additionally, adults with severe phenotype hemophilia (if not already on
prophylaxis) should initiate prophylaxis as well.

PROPHYLAXIS USING NON ¬FACTOR REPLACEMENT THERAPIES (WHF guidelines. 2020).

• Emicizumab prophylaxis in a number of clinical trials has been shown to be associated with
very low rates of bleeding (an annualized bleeding rate [ABR] of 1.5) and ABRs lower than
what patients previously reported while on prophylaxis with CFCs.

• More research is needed regarding long term outcomes with emicizumab.
• Data on the use of other non factor therapies for prophylaxis are at present much more
limited.

Details on emicizumab will be discussed in the following section.

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